Protalix BioTherapeutics Inc (PLX)

[jcon777]

Reminder, Russell 3000 adds PLX tomorrow (Monday)

We should see a lot more volume going forward. Let's see if it helps the stock price.

[midastouch017]

Protalix BioTherapeutics to Host In-Person KOL Breakfast following the Approval of Elfabrio® in both the United States and the European Union for Adults with Fabry Disease

https://finance.yahoo.com/news/protalix-biotherapeutics-host-person-kol-105000869.html

Event will take place on Tuesday, June 27, 2023 in New York

CARMIEL, Israel, June 8, 2023 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell–based protein expression system, today announced it will host an in-person KOL breakfast in New York on Tuesday, June 27, 2023 at 8:00 a.m. Eastern Daylight Time (EDT), with presentations beginning at 8:30 a.m. EDT.

The event will feature a discussion of the recent approval by the U.S. Food and Drug Administration (FDA) of Elfabrio (pegunigalsidase alfa-iwxj) in the United States for the treatment of adult patients with Fabry disease. Ankit Mehta, M.D., FASN (Baylor University Medical Center) will discuss the opportunity for Elfabrio to address the unmet needs in Fabry disease; Giacomo Chiesi, Head of Chiesi Global Rare Diseases, the Company's commercial partner, will discuss Chiesi's commercial capabilities; and the Company's leadership will provide insight into the Company's strategy and future plans.

A live question and answer session will follow the presentations. Registration for the event is available at https://lifescievents.com/event/protalix/, or by clicking here.

About Ankit Mehta, MD, FASN

Dr. Mehta is fellowship-trained and board-certified in nephrology and internal medicine. His professional interests include management of patients with severe hypertension, glomerulonephritis, kidney stones and fluid electrolyte disorders. Dr. Mehta is a committed educator and currently serves as Program Director for the Department of Internal Medicine at Baylor University Medical Center, part of Baylor Scott & White Health. He attended medical school at Grant Medical College in Mumbai, India. Dr. Mehta's internship and residency in internal medicine took place at Baylor University Medical Center. He worked in hospital medicine for a year before going on to a fellowship in nephrology at Baylor, Dallas. Dr. Mehta is a practicing nephrologist with Dallas Nephrology Associates. He is also involved in research at Baylor Scott and White research institute and renal diseases. His research interests include anemia of chronic kidney disease, diabetic kidney disease, Alport's syndrome, polycystic kidney disease and various glomerulonephritis.

[BooDog]

Of course that would be great to see. This is one I continue to accumulate on.

[jcon777]

Zacks article: PLX target price & valuation $16
https://finance.yahoo.com/news/plx-back-envelope-150000398.html

This part was interesting:
----------------------------------------------------------------
"We performed a quick and conservative back of the envelope analysis to generate a base case valuation.
? Fabry Disease market size – $2 billion 2
? Potential market share – 15% – 50%
? Royalty from Chiesi – 15% to 40%
? Applied multiple of royalty revenues – 15x – 25x
? Convertible debt – rounded up to $30 mm
If we take the low end of each of these components and calculate a value, this produces a conservative enterprise value estimate of about $675 million. After subtracting convertible debt this gives us an equity value of $645 million or about $10 per share."

"Our target price and valuation are determined using a set of more realistic assumptions that produce a $16 target which we recently increased following the FDA approval of Elfabrio. These assumptions include peak penetration of 18% of US and ex-US markets, as well as a sum of royalty and milestone payments of up to 40% of product revenues."

[bryanb40]

This author knows almost nothing about this company, he doesn't even know how this company gets paid.

"If Protalix captures 1% of the market for 1,240 people, the company could make $372 million per year from Elfabrio. However, it will probably take 5 to 10 years to reach that point and there is no guarantee that it does. I'm just trying to provide some point of reference for Elfabrio's potential growth."

[midastouch017]

Given the low valuation, the potential for a profitable year, and strong growth expectations, I expect the stock to make strong gains over the next two years if things go well for the company. If Protalix's PE increased to the industry average of 30 and the company achieved EPS of $0.16 for 2023, the stock would increase to $4.80 within about a year. That would represent a 133% gain for the stock over the current price.

[midastouch017]

Protalix BioTherapeutics Stock: FDA Approval For Elfabrio To Drive Profitability

May 30, 2023 7:01 AM ET

Protalix BioTherapeutics, Inc. (PLX)

David Zanoni

Summary
Protalix received FDA and EU approval for Elfabrio.
This puts the company on track for profitability as early as 2023.
With a reasonable valuation and strong growth, I see a significant potential upside for PLX stock.


Protalix BioTherapeutics (NYSE:PLX) in partnership with Chiesi Global Rare Diseases achieved a milestone as it recently received FDA and EU approval for Elfabrio, a treatment for Fabry disease. The approval of Elfabrio puts the company on a realistic path to profitability as early as 2023. This catalyst along with a reasonable valuation can drive the stock for significant above-average gains.

Company Background
Protalix is currently a small cap ($141 million market cap) biopharmaceutical company with two FDA approved therapies on the market. Protalix develops plant-based proteins with better therapeutic profiles as part of its ProCellEx system. The company received its 1st FDA approval in partnership with Pfizer (PFE) for Elelyso for the treatment of Gaucher disease. The company had revenue of $47.6 million in 2022 with one FDA approval under its belt.

The company received positive news a few weeks ago for the FDA and EU approval of Elfabrio for the treatment of Fabry disease. This is an important milestone for Protalix. Elfabrio is projected to increase revenue for 2023 by about 33% to $63 million. In 2024, revenue is projected to grow 50% to nearly $95 million. Protalix is also projected to end 2023 with positive net income after many years of net losses.

Protalix is set to receive a $20 million milestone payment from its partner Chiesi upon the FDA approval of Elfabrio. Protalix is also eligible to receive up to $1 billion in regulatory and commercial milestone payments in addition to tiered royalties. The company expects cash & equivalents to increase to $51.6 million after receiving the $20 million milestone payment. That represents a 56.4% increase over Protalix's cash & equivalents of $33 million.

Protalix also has two therapeutics being developed in the pipeline. PRX-115 or Uricase is a plant-based cell-expressed recombinant human DNase I product candidate for the treatment of gout. PRX-119 is the company's therapeutic being developed for the treatment of NETs related diseases. PRX-115 is in Phase I development while PRX-119 is in a preclinical stage of development. So, it is likely to be multiple years before these therapies are considered for commercial marketing.

The Potential for Elfabrio
Elfabrio joins Sanofi's (SNY) Fabrazyme and Amicus Therapeutics' (FOLD) Galafold as FDA approved treatments for Fabry disease. Takeda (TAK) has Replagal approved in other countries for Fabry disease, but it has not been FDA approved. Sangamo Therapeutics (SGMO) has a gene therapy in a Phase 2 study for the treatment of Fabry disease. So, that may take a few years to achieve approval.

So, Protalix does have competition in the marketplace with possible future entries into the market. Elfabrio offers a new alternative treatment option for adults with Fabry disease. As a rare disease, the prevalence of Fabry disease is estimated to be about 11,000 people in the U.S. and about 113,000 people in the European Union. Treatments for Fabry disease cost over $300,000 per year. If Protalix captures 1% of the market for 1,240 people, the company could make $372 million per year from Elfabrio. However, it will probably take 5 to 10 years to reach that point and there is no guarantee that it does. I'm just trying to provide some point of reference for Elfabrio's potential growth.

One of the important factors for Protalix regarding the FDA and EU approval of Elfabrio is that the company is expected to become profitable in 2023 according to analyst estimates. Becoming profitable could have a positive effect on the stock.

One of the reasons for Protalix's potential for profitability is the company's strong gross margin of 59.6%, which is higher than the sector median GM of 55.8%. Another reason is the result of the company's plant cell production advantages which can be less expensive than competing production methods. Profitability can help the company invest back into the business for new therapeutics and possibly avoid or reduce the need for a share diluting capital raise.

Protalix's Plant Cell Production Advantages vs. mammillian cell expression and bacteria and yeast cell expression
Protalix's Plant Cell Production Advantages vs. Other Methods (Protalix 10-K)

Valuation is Attractive
Protalix is still valued attractively even after the recent FDA approval of Elfabrio. Protalix is trading with a trailing price/sales ratio of 2.57 and a forward price/sales of 2.2. This is significantly lower than the biotechnology industry's price/sales ratio of 8.88. Protalix also has an attractive forward PE of 12.9 as compared to its industry's forward PE of over 30.

One of the reasons for the attractive valuation is because the stock sold off after the FDA approval. The stock had a run up before the FDA approval decision. However, the actual FDA approval resulted in a sell-the-news reaction from investors. Therefore, investors can pick up the stock at a reasonable valuation before the next catalyst.

Technical Perspective
Protalix stock possible takeover target undervalued biotech
Protalix Stock Chart (Price, MACD, RSI) (tradingview.com)

The chart above shows the significant run up prior to the FDA approval of Elfabrio and the sell-the-news reaction after the decision was made. The stock peaked at the 52-week high of $3.55. The stock now appears to be holding the $2 level where it has multi-month support. The MACD indicator in the middle of the chart shows that the selling momentum has been reduced. However, the stock could also move lower on negative news such as the announcement of a share diluting capital raise.

Solid Balance Sheet
The recent $20 million milestone payment will bring Protalix's cash & equivalents up to $51.6 million. That is higher than the company's total debt of $33.6 million, giving them zero net debt.

Prior to the milestone payment, the company had 1.6x more current assets than current liabilities and 1.02x more total assets than total liabilities for total equity of $1 million. So, the milestone payment strengthens the company's balance sheet further. Of course, Protalix will need the extra cash to begin manufacturing Elfabrio for commercial sales. Now, the company is in a better financial position to execute it.

Possible Takeover Target
Small companies like Protalix can be takeover targets for larger companies to acquire. Protalix offers an attractive valuation with a soon to be profitable asset with Elfabrio. A takeover would typically occur at a premium price.

Outlook for Protalix
Although there is competition on the market for Fabry disease therapies, Elfabrio can allow Protalix to get its piece of the pie of the total market. Some Fabry disease patients might tolerate Elfabrio better than competing therapies.

Protalix is expected to be profitable in 2023 with projected EPS of $0.16. EPS is expected to grow to $0.63 in 2024. Revenue is expected to grow about 33% to $63 million in 2023 and increase 50% in 2024 to about $95 million. I expect the stock to make significant above-average gains if these estimates are met or exceeded.

The main risks for investors would be a share dilutive capital raise and/or sales and earnings expectations coming in worse than expected. These situations are likely to put negative pressure on the stock. Potential investors might want to wait to see if Protalix needs a capital raise to launch Elfabrio before buying the stock. Protalix should be considered as a highly risky stock due to these factors.

Given the low valuation, the potential for a profitable year, and strong growth expectations, I expect the stock to make strong gains over the next two years if things go well for the company. If Protalix's PE increased to the industry average of 30 and the company achieved EPS of $0.16 for 2023, the stock would increase to $4.80 within about a year. That would represent a 133% gain for the stock over the current price.

This article was written by

David Zanoni
9.44K Followers

[midastouch017]

Protalix BioTherapeutics Issues Letter to Stockholders

https://finance.yahoo.com/news/protalix-biotherapeutics-issues-letter-stockholders-105000485.html

CARMIEL, Israel, May 30, 2023 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, today announced the following letter from its President and Chief Executive Officer, Dror Bashan, to its stockholders and the investment community.

Dear Protalix Stockholders,

As we arrive at the halfway point of this remarkable year, I would like to take a moment to celebrate our success, as well as share our excitement for the future of Protalix.

The spring commenced with a bang. Elfabrio®, our primary development candidate, has been approved for the treatment of adult patients with Fabry disease, both in the United States and the European Union. These approvals mark a significant milestone in our history. Although we faced challenges during our path toward regulatory approval, our team persevered, and you with us. These approvals are a true testament to our commitment to delivering innovative solutions to patients in need.

Elfabrio is the second approved therapy based on our proprietary ProCellEx® plant cell-based protein expression system, a unique platform conceived of and developed by Protalix and brought to fruition by the talented scientists making up our team. ProCellEx represents a relatively new method for developing recombinant proteins in an industrial-scale manner. Elelyso®, our first approved drug product, was the first protein produced through plant cell-based expression in suspension to be approved by the FDA.

Chiesi Global Rare Diseases, our commercial partner for Elfabrio, is well prepared for a successful commercial launch, and I have every confidence that they will realize the full potential of this much needed therapeutic option. I would like to extend my heartfelt gratitude to Chiesi.

Now that we stand on the other side of this accomplishment, we are turning our attention to our pipeline programs which have shown potential for growth. We are excited to focus on these development programs, armed with the knowledge gained from our hard-earned experiences. We plan to share our insights and future strategic initiatives with you and extend an invitation for you to join us at our upcoming in-person investors event on June 27, 2023 at 8:00 am ET at the Lotte Palace Hotel in New York City. Additional details and registration information will soon be available on our website.

We are profoundly grateful for your confidence in our company and for standing by us throughout the years. We are excited about the path ahead and the opportunity to connect with you during the upcoming event where we will discuss our future plans that we are designing to drive Protalix's growth. With your continued support, we will work towards a fruitful future, creating value for you, our investors and, most importantly, for patients and their families.

Sincerely,

Dror Bashan, President & Chief Executive Officer

About Protalix BioTherapeutics, Inc.

Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. It is the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. This unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights to taliglucerase alfa for the treatment of Gaucher disease, Protalix's first product manufactured through ProCellEx, excluding in Brazil, where Protalix retains full rights. Protalix's second product, Elfabrio®, has been approved by both the FDA and the European Medicines Agency (EMA). Protalix has partnered with Chiesi Farmaceutici S.p.A. for the global development and commercialization of Elfabrio.

Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: PRX–115, a plant cell-expressed recombinant PEGylated uricase for the treatment of severe gout; PRX–119, a plant cell-expressed long action DNase I for the treatment of NETs–related diseases; and others.

[Trickshot]

"In clinical trials" the elfabrio site states 29% experience infusion related reactions. The fabrazyme site says 59% of its patients do.

[midastouch017]

Protalix stock rises as biotech set to join Russell 3000 Index

May 23, 2023 11:27 AM ETProtalix BioTherapeutics, Inc. (PLX)

By: Ravikash, SA News Editor

Protalix BioTherapeutics (NYSE:PLX) stock rose ~8% on Tuesday after
the company said it is set to join the broad-market Russell 3000 Index
at the conclusion of the 2023 Russell indexes annual reconstitution,
effective June 26 when U.S. markets open.
The preliminary list of additions were posted

[BooDog]

Excellent news. Protalix BioTherapeutics set to join Russell 3000® Index

https://finance.yahoo.com/news/protalix-biotherapeutics-set-join-russell-105000952.html

Take a look at the weekly as well, a LOT of running room.

https://finviz.com/quote.ashx?t=PLX&p=w

[ofirm]

They got like $11M in cash instead of future $25M (which we do not when they were to be
due to PLX).

[jcon777]

Protalix BioTherapeutics set to join Russell 3000® Index

CARMIEL, Israel, May 23, 2023 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell–based protein expression system, today announced that it is set to join the broad-market Russell 3000® Index at the conclusion of the 2023 Russell indexes annual reconstitution, effective after the US market opens on June 26, 2023 according to a preliminary list of additions posted May 19.

https://www.prnewswire.com/news-releases/protalix-biotherapeutics-set-to-join-russell-3000-index-301831972.html

[Trickshot]

That's correct, they gave up some potential milestone % for a debt related financial consideration, over a year ago if memory serves.

[king oil]

Most of the payments must be tied to commercial milestones and not regulatory milestones. So again we wait….

[midastouch017]

I remotely recall that Chiesi paid of some of
PLX debts in return to reduced milestone
$ amounts.
I'll try looking up this PLX-Chiesi agreement.

This was the initial agreement:
https://protalixbiotherapeutics.gcs-web.com/news-releases/news-release-details/protalix-biotherapeutics-expands-partnership-chiesi-farmaceutici


Protalix to receive $25 million upfront, an additional up to $20 million in development costs and an aProtalix BioTherapeutics Expands Partnership with Chiesi Farmaceutici to Include Exclusive U.S. Rights for the Development and Commercialization of PRX-102 (pegunigalsidase alfa) for thdditional up to $760 million in potential regulatory and commercial milestone payments for the U.S. rights

U.S. partnership includes tiered royalties ranging from 15% to 40% on net sales

CARMIEL, Israel, July 24, 2018 (GLOBE NEWSWIRE) -- Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®, today announced the expansion of its partnership with Chiesi Farmaceutici S.p.A., or Chiesi. Protalix and Chiesi entered into an exclusive U.S. license and supply agreement which grants to Chiesi the United States rights for the development and commercialization of PRX-102 (pegunigalsidase alfa), the Company’s chemically modified version of the recombinant protein alpha-Galactosidase-A protein, for the treatment of Fabry disease. In October 2017, Protalix announced an exclusive partnership with Chiesi for the development and commercialization of PRX-102 for the treatment of Fabry disease outside the United States.

Under the terms of the U.S. license and supply agreement, Protalix is entitled to an upfront payment of $25 million from Chiesi and additional payments of up to a maximum of $20 million in development costs, capped at $7.5 million per year. Protalix is also eligible to receive an additional up to a maximum of $760 million, in the aggregate, in regulatory and commercial milestone payments, and tiered royalties ranging from 15% to 40% on net sales as consideration for product supply. Protalix will continue to be the manufacturer of PRX-102 for clinical development and commercial purposes.

----------------------------------------------------------------------------------
This agreement was modified at By:
/s/ Eyal Rubin/s/ Dror Bashan
Title:CFOCEO
Name:Eyal RubinDror Bashan
Date:5/13/20215/13/2021
https://www.sec.gov/Archives/edgar/data/1006281/000155837021007157/plx-20210331xex10d3.htm

[king oil]

Well that explains the stock plummeting after approval. The payment is far lower than most had imagined.

[midastouch017]

On a proforma basis, including the receipt of the $20.0 million milestone payment, cash and cash equivalents as of May 18, 2023 is $51.6 million.

No offering needed, neither usage of ATM facility!

[midastouch017]

Protalix BioTherapeutics Announces $20 Million Milestone Payment from Chiesi Global Rare Diseases
https://finance.yahoo.com/news/protalix-biotherapeutics-announces-20-million-105000631.html

Milestone payment triggered by FDA approval of ELFABRIO® (pegunigalsidase alfa-iwxj) and payable within 30 days of approval

CARMIEL, Israel, May 18, 2023 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell–based protein expression system, today announced that it is eligible to receive a $20 million milestone payment from its commercial partner, Chiesi Global Rare Diseases, a business unit of the Chiesi Group. The milestone payment was triggered by the approval by the U.S. Food and Drug Administration (FDA) of ELFABRIO (pegunigalsidase alfa-iwxj) for the treatment of adult patients with Fabry disease, which was announced on May 10, 2023, and is payable within 30 days of the FDA approval date.

"Receiving this milestone payment resulting from the FDA's approval of ELFABRIO is an important step for Protalix as it further to strengthens our financial position," said Dror Bashan, Protalix's President and Chief Executive Officer. "We continue to be grateful to our commercial partner, Chiesi, who has the global expertise to maximize the market potential of pegunigalsidase alfa."

On July 23, 2018, the Company's wholly-owned subsidiary entered into an Exclusive License and Supply Agreement with Chiesi Farmaceutici S.p.A., or Chiesi (the Chiesi US Agreement), pursuant to which the Company granted Chiesi an exclusive license to commercialize pegunigalsidase alfa in the United States. This followed the Exclusive License and Supply Agreement entered into with Chiesi on October 19, 2017 (the Chiesi Ex-US Agreement), pursuant to which Chiesi was granted an exclusive license to commercialize pegunigalsidase alfa in all markets outside of the United States. Under the two agreements, the Company has already received a total of $95.0 million in upfront payments and to cover development costs. In addition, under the two agreements, the Company is entitled to up to $1.0 billion in potential regulatory and commercial milestone payments, tiered royalties ranging from 15% to 40% of Chiesi's net sales in the United States and tiered royalties ranging from 15% to 35% of Chiesi's net sales outside the United States, as consideration for product supply.

On a proforma basis, including the receipt of the $20.0 million milestone payment, cash and cash equivalents as of May 18, 2023 is $51.6 million.

About Protalix BioTherapeutics, Inc.

Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. It is the first company to gain U.S. Food and Drug Administration approval of a protein produced through plant cell-based in suspension expression system. This unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights to taliglucerase alfa for the treatment of Gaucher disease, Protalix's first product manufactured through ProCellEx, excluding in Brazil, where Protalix retains full rights. In addition, Protalix has partnered with Chiesi Farmaceutici S.p.A. for the global development and commercialization of PRX–102 (pegunigalsidase alfa).

Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: PRX–115, a plant cell-expressed recombinant PEGylated uricase for the treatment of severe gout; PRX–119, a plant cell-expressed long action DNase I for the treatment of NETs–related diseases; and others.

[Spideyboy]

I would disagree that the once per 4 week is very important for the market potential of Elfabrio, when compared to the equal efficacy and better safety, and probable real world better efficacy.

I think for this it is important to define the 'doing well on Fabrazyme'. Let us remember that from the clinical data, Fabrazyme causes ADAs/immunogenic response in 74% of patients.

So if we assume that doing well on Fabrazyme is in that 26% of patients, then yes, that person would be best suited to switch on to the once per 4 month dosing if/when available.

But the other 74%, even if doing well on the efficacy of Fabrazyme and generally well on more important side effects will still not be feeling great due to this ADA positive activity in their system.

Whereas in the Balance trial, patients with ADA's dropped when on Elfabrio, by 34% and dropped the neutralising antibodies by 55%.

And this is in patients who had already been on Fabrazyme for a mean of 6 years, so these are patients that are tolerating Fabrazyme well/or at least much better than others (clinical trial was in USA and 11 countries outside the USA).

So I think this type of patient (in the 74% ADA) doing well on Fabrazyme, would still be better off on Elfabrio. And this type of patient would already be accustomed to the once per 2 week regimen over many years of Fabrazyme, so it's not like Elfabrio is a detractor on that.

Thus for this type of patient in the 74% with ADAs would be a candidate for Elfabrio. Anyone doing well on efficacy and no adverse reaction and no ADAs would be fine to stay on Fabrazyme.

As remember in the Balance study, TEAE in total were 4x higher for Fabrazyme, and Infusion-Related-Reactions number and rate were 4x and 8x higher respectively for Fabrazyme.

[wgg2]

As we all know now the approvals by the EMA and FDA are for once-every-two-week treatments only, not for the once monthly treatment. I correct my post # 3860 which included patients for both. Still think it reasonable for Chiesi/Protalix to have 2000 patients under treatment in 18 months, therefore I stick by my earnings and value expectations.

I noted the J. Vandermosten from Zacks suggested " the # of patients to be 15,000 in the associated regions". If the pricing of fabrazyne is similar for Elfabrio then that implies a much bigger market and sales dollars than anticipated.

Management assures me both they and Chiesi have adequate inventory to meet an early marketing campaign.

Hope we soon get through this brutal short-term profit taking and short sale fascial.

[king oil]

It’s definitely getting shorted heavily which drives the price down. Either someone believes the stock will continue to fall or they are shorting to drive the price down. It wouldn’t surprise me if it’s part of a plan to acquire the company at a lower price.

[jcon777]

PLX is Short sale restricted for 2023-05-15

Anyone know much about this?

https://www.shortablestocks.com/?PLX

[Tex18]

Possibly because the milestones are staged payments, not all at once. If you count them as top line earnings and apply DCF, the future payments are as valuable as the up front payments from a time value sense. Also it’s conceivable the market is assuming they don’t take much market share from the incumbents who’ve been “entrenched” for decades.

Regardless, the stock is 1/5 to 1/10th of what it should be valued imo.

[BrunoGOudega]

Read into PLX latest annual report and i dont understand those milestone paymenu’s of €320M and €760M for EU and US.
Means if those payments are done the stock should be at €980M / 57.38M = €17
Can somebody explain because im missing something really important at the moment.
Thanks for your replies.

[Kronberg]

Spideyboy thanks for the detailed answer.The middle of the year is the problem of my English . I meant during the year, that is, when a person started on one medicine and, say, in the second half of the year, changed it to the another.The 2 mg/kg administered every four weeks regimen is very important IMO because it can add a significant number of new patients. let's say family working man 25-35 years old doing well on the Fabrazyme.IMO, it is very tempting for him to reduce the number of infusions by half with the same drug efficacy to be more free and flexible.
Perhaps you have noticed this information.
At month 12, the mean slopes for eGFR were -2.507 mL/min/1.73 m²/year for the
pegunigalsidase alfa arm and -1.748 for the agalsidase beta arm (difference -0.749 [-3.026, 1.507]. At month 24, the median slopes for eGFR were -2.514 [-3.788; -1.240] mL/min/1.73 m²/year for the pegunigalsidase alfa arm and -2.155 [-3.805; -0.505] for the agalsidase beta arm
(difference -0.359 [-2.444; 1.726]).
Who knows maybe by the end of the third year mean slopes for eGFR will be equal or the Elfabrio will even act better?

[BooDog]

Filled some 2.33's. Maybe a little early. I like anywhere here to the $2 mark. Lower bollinger 2.08.

[Spideyboy]

Hi Kronberg, thanks for the message.

First of all I don't think my forecasts and numbers are too optimistic.

I cannot see what patient or physician would not prefer to be on Elfabrio aside from those doing well with low ADA and low side effects from Fabrazyme. As well as the Efficacy and Safety data generated to date, one should also keep in mind earlier PK/PD data which showed significantly more enzyme going into the heart as well as kidneys. If I were a patient I might even demand to be put on Elfabrio if my insurance covers it. And given I'm probably on an isurance that covers Fabrazyme, then I would assume it would cover Elfabrio too.

I believe the above only makes sense. This is my life you're talking about, this is my family I'm going to spend time with. I want to maximise it with a few safety issues as possible.

A timeframe for 50% is too difficult to really predict as I'm sure you'll appreciate.

However I might say, quick uptake of all US & EU Fabry patients that are on very poor eGFR slopes as those recruited into the Balance trial, this being quick for both Fabrazyme & Replagal. Quick maybe 3-6 months post launch and available.

How much of the market would that be, no idea. For fun I would hazard a guess at 10% of the market.

Then we have the uptake of these doing generally poorly and considering the option with the physicians I would put that at another 20% of the market. These coming over a 6 months-1 year period.

Then those that are doing ok on eGFR but are having side-effects from Fabrazyme and want to try Elfabrio to minimise that, another 10% of the market. These 10% also coming 6 months to 1-2 years.

Then some those that are doing well on Fabrazyme eGFR and no big side effects, but that decide that they would like to switch based on the great feedback they hear from everyone else on quality of life. Anecdotal evidence that Elbabrio patients have even started sweating again, which they hadn't done so for years. it might seem small but all these things point to happier patients. And who doesn't want to be happier. These coming from the 1-2 year mark

And that is my highly speculative assumption, though is not to say that Elfabrio would not become standard of care. I only feel this chance is somewhat harder to say as I am not blind to the fact that Sanofi has plenty of expertise and cash. That said I have every confidence in both the Phase I/II and 3 Phase 3 trials data across naive and switch patients as well as all the PK/PD and organ data, as well as Chiesi capacity to maximise the value. They are a Top 50 global company with rare disease ERT experience. Very satisfied.

As far as timing of switch, yes of course this can happen 'in the middle of the year' or at anytime really. I don't quite get why you are focused on the middle of the year?? Curious??

Balance and Bright were both switch studies from patient previously on Fabrazyme and/or Replagal. So yes, any of these patients can switch over to Elfabrio at anytime.

Correct the once in 4 weeks dosing was not approved yet, and based on the clinical data I can see where the regulators were coming from. Not that things were bad, but I don't think it was specifically good enough to get the regulators to agree, especially when they can start the process with the once per 2 weeks regimen that all patients are already accustomed to. So they want more data before approving reducing the regimen by half.

First of all I think that's fine, but more importantly for us, it makes no difference to the revenues as the amount dosed over the 2 week or 4 week regimen is the same and this drug usage is the same. Where of course it could make a difference is for those patients that are doing very well on Fabrazyme or Replagal already, but just want to take less frequent dosing.

I am highly doubtful of a BO, and nor to do I wish it to happen yet, perhaps after a year of sales, and the price would be significantly higher.

But yes, once this shorting nonsense ends and the longs that were in stop freaking out, then we can see the SP take on a true image.

I expect SP to rise nicely even coming into the June strategy meeting, as this may show early indications of Chiesi commercialisation which will spook the shorts.

Just look at GMDA, just the mere suggestion of the upcoming earnings call which may show signs of market uptake or progress in commercialisation efforts has the shorts covering.

I note that yesteday on Shortablestocks, you can see that the short shares available to be seen in that site, when from 750,000 to 1.7 million. So the shorts covering significantly, and which was associated with the 20% increase in GMDA we say yesterday.

So while we will not hear of the amount of the milestone payment which will be made in the next 2-3 weeks based on the USA agreement SEC filing, I still think that much the way the chance of uncertainty and bad news in the market spooks investors tremendously, would expect that for shorts the chance of uncertainty and good news has the same effect on them and rightly so.

The future is bright for PLX and I'm just going to buy more when this nonsense bottoms out. Which could be today, let's see.

Best,
Spidey

[BooDog]

Looking like solid consolidation setting a nice support level.

glta!

[Kronberg]

Hello Spideyboy!
I like your forecasts and numbers but maybe today they are a bit optimistic. By the way what is your timeframe for Elfabrio to get half of the market?
My quite limited understanding says Elfabrio's two main benefits today are immunogenicity and 2 mg/kg every four weeks regimen.
"In clinical studies, 17 out of 111 of patients (16%) treated with 1 mg/kg Elfabrio every two weeks and 0 out of 30 patients treated with 2 mg/kg Elfabrio every four weeks developed treatment-induced antidrug antibodies (ADAs)."
Which drug is more effective Elfabrio or Fabrazyme apparently it will be possible to say only in a few years when significant comparative statistics will be possible. Unfortunately, 2 mg/kg every four weeks regimen is not yet possible and it will probably take some time to get OK on it. Do you think a patient in the middle of the year can switch from Fabrazyme or Replagal to Elfabrio?
Now it's a waiting game for a significant Fabry market uptake or BO.

[midastouch017]

Three out of four: Protlix also received marketing approval for its medicine for Fabry disease
The approval comes just after the company's stock was delisted from trading in Tel Aviv.

(Article published in Hbrew - Google translated, a non perfect translation.)

• MediWound and Gamida Cell also received marketing approvals for their drugs this year, after many
years of drought in obtaining approvals for Israeli drugs through new mechanisms
• Bioline is also awaiting the FDA's response.

Gali Vinrab 10.05.2023

The company Protelix, which produces drugs for rare diseases by engineering plant cells so that they express human proteins, has received marketing approval in the US for its drug for Fabry disease. Protelix is traded on the New York Stock Exchange at a value of 191 million dollars and in pre-opening trading the stock is up 17%. In March it was deleted The company's stock from trading in Tel Aviv. Even before that, it managed to rise nicely, in preparation for the possibility of receiving the approval. Its return in the last year on the New York Stock Exchange is 190%.

It is one of four Israeli companies that submitted innovative drugs to the FDA, the American Food and Drug Administration, for approval this year. This, after many years in which no innovative Israeli drug reached this status. Two companies have already received a positive answer: Gamida Sel, which is developing a drug that improves treatments for transplanting a new immune system in blood cancer patients, and Medivand, which has developed a drug against burns. Bioline, which is also developing a blood cancer treatment, is also waiting for the Authority's answer until the end of the year.

Protelix's drug is the only one that enters an existing market where it will compete head-to-head with Genzyme's drug Favrezyme. Genzyme's drug is produced in animal cells, while Protelix is the only one that produces it in plant cells. The entire market is estimated at about 2.2 billion dollars. Protelix has a marketing agreement in the US with the Chiesi company, which specializes in the field of rare diseases.

Protlix previously developed a drug for Gaucher's disease based on the same technology, and commercialized it to Pfizer. This drug generated revenue for the company, but due to delays in entering the market, it led to the fact that when it entered the market it was very competitive, and it was unable to take a significant place in the market, and Pfizer lost interest in it, and in the end the drug was not successful in the world market.

After learning this lesson, Protlix stated that it would only go to market with products that were superior to those already on the market. The Fabri product was designed to be better than existing products, and the company does state in its press release that it has a longer half-life. The studies conducted by the company were "non-inferiority" studies, and they did not establish that the half-life results in superiority of the product in terms of efficacy or safety.

That's why Protlix is conducting two follow-up trials for marketing purposes and obtaining insurance indemnity at the desired price. One trial was designed to support the claim that the product improves kidney function compared to Genzyme's product, and another trial was designed to support a treatment protocol according to which the injection is given once a month instead of once every two weeks.

The companies have not yet disclosed what the product's pricing will be compared to Genzyme's, but Protlix will probably not try to compete with an extremely low price, but will price it around the market price.

Amicus also competes in the American febrile market with the Galfold product, which can be swallowed and is only suitable for some patients. Outside of the USA, Shire (Takeda) also operates, which for a decade has not been able to get FDA approval.

The maximum revenue Protlix expects from the product is 150-200 million dollars per year. Protlix will produce the drug at the plant in Karmiel, where it also produces the drug for Gosha.

In the first quarter of this year, Protlix recorded revenues of 5.1 million dollars from the Ghosha product. At the end of the quarter, it had $33 million in cash. Upon approval, she is expected to receive a milestone payment from Casey.

The other two companies that received approvals from the beginning of the year, Medivand and Gamida Sel, did not, as a result, register significant increases in their shares over time. There is a difference between these two companies and Protelix: both companies market their products independently to markets where there are currently no other products, and they now need to invest in building a marketing system and educating the market, so they may require more time and significant investment before they reap the benefits of marketing their drugs . Protelix may have an easier path forward because it has already signed the deal with Casey. However, it is possible that some of these differences are already priced in the share price.

[midastouch017]

Fabry Disease Treatment Showdown: Protalix Bio's Elfabrio Joins the Ranks of Sanofi and Amicus Therapeutics

Vandana Singh

Wed, May 10, 2023 at 11:16 PM GMT+3

https://finance.yahoo.com/news/fabry-disease-treatment-showdown-protalix-201601855.html

The FDA approved Chiesi Group and Protalix BioTherapeutics Inc's (NASDAQ: PLX) Elfabrio (pegunigalsidase alfa-iwxj) for treating adult patients with Fabry disease.

The product is an enzyme replacement therapy for adults with a rare, inherited disease in which abnormal deposits of fatty substances build up and cause pain and sometimes end-organ failure.

Elfabrio is a PEGylated enzyme replacement therapy. It is a recombinant human a–Galactosidase–A enzyme expressed in a plant-cell culture designed to provide a long half-life.

Also Read: Protalix Clocks 40% Decline In Q1 Revenues Reflecting Lower Sales To Brazil.

Just last week, the European Commission approved PRX-102 (pegunigalsidase alfa) in the European Union for Fabry disease.

Elfabrio competes with Sanofi SA's (NASDAQ: SNY) Fabrazyme and Amicus Therapeutics Inc's (NASDAQ: FOLD) Galafold in treating Fabry disease.

Almost two years back, the companies received a Complete Response Letter (CRL) from the FDA related to the marketing application of pegunigalsidase alfa (PRX-102) for Fabry disease.

[jcon777]

PLX volume 1st day after FDA approval: 7,632,535

Here are the numbers for today's volume:
Volume 7,632,535
Avg. Volume 1,664,273

I was thinking it might have been even higher today, but it might be awhile until the bigger institutes start coming in.

[wgg2]

Spideyboy, I would appreciate your help in clarifying how the PRX-115 phase one trial for severe gout is conducted. In the 3/21/2023 release it states: The trial is a single ascending dose (SAD) study with up to seven cohorts, and patients are being randomized 3:1 to receive a single intravenous (IV) dose of PRX-115 or a placebo. It says a patient can get up to seven treatments (I assume if needed). Then what does the word single mean later in the release? How frequently might the "up to 7 cohorts" be administered? Even though phase one trials are not to determine effectiveness, considering the nature of gout, it seems to me they will know rather quickly if it is effective or not. Agree? As of last week, they stated that they have 9 patients already being treated. If this works, this is another major market opportunity since there is little out there to treat severe gout. Thanks.

[midastouch017]

Chiesi Global Rare Diseases and Protalix BioTherapeutics Announce FDA Approval of ELFABRIO® (pegunigalsidase alfa-iwxj) for the Treatment of Fabry Disease

https://finance.yahoo.com/news/chiesi-global-rare-diseases-protalix-100000335.html

- PEGylated enzyme replacement therapy designed to provide long half-life* -

BOSTON and CARMIEL, Israel, May 10, 2023 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, and Protalix BioTherapeutics, Inc. (NYSE American:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®, announced today that the U.S. Food and Drug Administration (FDA) has approved ELFABRIO (pegunigalsidase alfa-iwxj) in the United States for the treatment of adult patients with Fabry disease.

"While much progress has been made in the treatment of Fabry disease, there is still a need for new treatment options," said Giacomo Chiesi, head of Chiesi Global Rare Diseases. "We established Chiesi Global Rare Diseases to deliver innovative therapies and solutions for people affected by rare diseases. With the FDA approval of ELFABRIO, we can now offer people living with Fabry disease an alternative treatment option."

"We are extremely pleased to receive FDA approval of ELFABRIO for the treatment of adult patients with Fabry disease," said Dror Bashan, Protalix's President and Chief Executive Officer. "This approval is a testament to the dedication of the Protalix and Chiesi teams to deliver this much needed new therapeutic option to patients in need. The totality of clinical data suggests that ELFABRIO has the potential to be a long-lasting therapy. Together with Chiesi, we are grateful to all of the patients and investigators and their staff members who participated in our clinical trial programs and remain committed to bringing ELFABRIO to patients with Fabry disease."

ELFABRIO is a PEGylated enzyme replacement therapy (ERT). It is a recombinant human a–Galactosidase–A enzyme expressed in plant-cell culture that is designed to provide a long half-life.

The safety, tolerability, and efficacy of ELFABRIO has been studied in a comprehensive clinical development program in more than 140 patients with up to 7.5 years of follow up treatment. It has been studied in both ERT-naïve and ERT-experienced patients, including a head-to-head trial that met its primary endpoint with ELFABRIO demonstrating non-inferior efficacy to agalsidase beta in controlling estimated glomerular filtration rate (eGFR) decline, and in which ELFABRIO was generally well-tolerated with the majority of adverse events being mild or moderate in severity.

"It is important to understand that there is a lot of variability in Fabry disease and misdiagnoses are common, especially in women," said Jack Johnson, founder of the Fabry Support & Information Group (FSIG). "Growing up, a lot of people didn't know what was wrong with me. They knew I was different, but they didn't know why. Now we have made advances in screening, treatment, and monitoring for Fabry disease."

*ELFABRIO has an initial half-life of 78.9 ± 10.3 hours. Clinical studies have not established that half-life results in superior efficacy or safety based on clinically relevant end points.

Indication and Important Safety Information

Indication

Elfabrio® (pegunigalsidase alfa-iwxj) is indicated for the treatment of adults with confirmed Fabry disease.

Important Safety Information

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS

Patients treated with Elfabrio have experienced hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during Elfabrio administration. If a severe hypersensitivity reaction (eg, anaphylaxis) occurs, discontinue Elfabrio immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reaction, a desensitization procedure to Elfabrio may be considered.

Prior to Elfabrio administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. Inform patients and caregivers of the signs and symptoms of hypersensitivity reactions and infusion-associated reactions (IARs), and instruct them to seek medical care immediately if such symptoms occur.

If a severe hypersensitivity reaction (including anaphylaxis) or severe IAR occurs, immediately discontinue Elfabrio administration and initiate appropriate medical treatment.

If a mild to moderate hypersensitivity reaction or IAR occurs, consider slowing the infusion rate or temporarily withholding the dose.

In clinical trials, 20 (14%) Elfabrio-treated patients experienced hypersensitivity reactions.

Four Elfabrio-treated patients (3%) experienced anaphylaxis reactions that occurred within 5 to 40 minutes of the start of the initial infusion. The signs and symptoms of hypersensitivity reactions and anaphylaxis included headache, nausea, vomiting, throat tightness, facial and oral edema, truncal rash, tachycardia, hypotension, rigors, urticaria, intense pruritus, moderate upper airway obstructions, macroglossia, and mild lip edema.

In clinical trials, 41 (29%) Elfabrio-treated patients experienced one or more infusion-associated reactions, including hypersensitivity, nausea, chills, pruritus, rash, chest pain, dizziness, vomiting, asthenia, pain, sneezing, dyspnea, nasal congestion, throat irritation, abdominal pain, erythema, diarrhea, burning sensation, neuralgia, headache, paresthesia, tremor, agitation, increased body temperature, flushing, bradycardia, myalgia, hypertension, and hypotension.

A case of membranoproliferative glomerulonephritis with immune depositions in the kidney was reported during clinical trials. Monitor serum creatinine and urinary protein-to-creatinine ratio. If glomerulonephritis is suspected, discontinue treatment until a diagnostic evaluation can be conducted.

When switching to Elfabrio from a prior enzyme replacement therapy, the risk of hypersensitivity reactions and infusion-associated reactions may be increased in certain patients with pre-existing anti-drug antibodies (ADAs). Consider monitoring IgG and IgE ADAs and clinical or pharmacodynamic response (eg, plasma lyso-Gb3 levels).

The most common adverse reactions (=15%) were infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis.

Please see Full Prescribing Information for Elfabrio.

About Fabry Disease

Fabry disease is an X–linked inherited disease that results from deficient activity of the lysosomal a–Galactosidase–A enzyme resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in the lysosomes throughout a person's body. Fabry disease occurs in one person per 40,000 to 60,000. Fabry patients inherit a deficiency of the a–Galactosidase–A enzyme, which is normally responsible for the breakdown of Gb3. The abnormal storage of Gb3 increases with time and, accordingly, Gb3 accumulates, primarily in the blood vessel and tissues. The ultimate consequences of Gb3 deposition range from episodes of pain and impaired peripheral sensation to end-organ failure.

About ELFABRIO

ELFABRIO (pegunigalsidase alfa-iwxj), a PEGylated enzyme replacement therapy (ERT) to treat Fabry disease, is a plant cell culture-expressed, and chemically modified stabilized recombinant version of the a–Galactosidase–A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with stable pharmacokinetic parameters. In clinical studies, ELFABRIO has been observed to have an initial half-life of 78.9 ± 10.3 hours. Clinical studies have not established that half-life results in superior efficacy or safety based on clinically relevant end points.

About Chiesi Global Rare Diseases

Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system.

For more information visit www.chiesirarediseases.com.

About Chiesi Group

Chiesi is an international, research-focused biopharmaceuticals group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company's mission is to improve people's quality of life and act responsibly towards both the community and the environment.

By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi's commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, we're part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035.

With over 85 years of experience, Chiesi is headquartered in Parma (Italy), operates in 31 countries, and counts more than 6,500 employees. The Group's research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden.

For further information please visit www.chiesi.com.

About Protalix BioTherapeutics, Inc.

Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. It is the first company to gain FDA approval of a protein produced through plant cell-based in suspension expression system. This unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights to taliglucerase alfa, Protalix's first product manufactured through ProCellEx, excluding in Brazil, where Protalix retains full rights.

Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: pegunigalsidase alfa, a modified stabilized version of the recombinant human a-Galactosidase-A protein for the treatment of Fabry disease; PRX–115, a plant cell-expressed recombinant PEGylated uricase for the treatment of severe gout; PRX–119, a plant cell-expressed long action DNase I for the treatment of NETs-related diseases; and others. Protalix has partnered with Chiesi Farmaceutici S.p.A., both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa-iwxj.

Protalix's Forward-Looking Statements

To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms "expect," "anticipate," "believe," "estimate," "project," "may," "plan," "will," "would," "should" and "intend," and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on Protalix's current beliefs and expectations as to such future outcomes. Factors that might cause material differences include, among others: risks related to the commercialization of ELFABRIO; the likelihood that the FDA, European Medicines Agency (EMA) or other applicable health regulatory authorities will approve an alternative dosing regimen for ELFABRIO; risks related to the commercial success of Protalix's other product and product candidates, if approved; failure or delay in the commencement or completion of preclinical studies and clinical trials of our other product candidates which may be caused by several factors, including: slower than expected rates of patient recruitment; unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; inability to satisfactorily demonstrate non-inferiority to approved therapies; inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; and inability to monitor patients adequately during or after treatment; delays in the approval or potential rejection of any applications we file with the FDA, EMA or other health regulatory authorities for our other product candidates, and other risks relating to the review process; risks associated with the novel coronavirus disease, or COVID–19, outbreak, which may adversely impact our business, preclinical studies and clinical trials; the risk that the results of the clinical trials of our product candidates will not support the applicable claims of safety or efficacy, or that our product candidates will not have the desired effects or will be associated with undesirable side effects or other unexpected characteristics; risks related to our ability to maintain and manage our relationship with our collaborators, distributors or partners; our dependence on performance by third party providers of services and supplies, including without limitation, clinical trial services; the inherent risks and uncertainties in developing drug platforms and products of the type we are developing; the impact of development of competing therapies and/or technologies by other companies and institutions; potential product liability risks, and risks of securing adequate levels of product liability and other necessary insurance coverage; and other factors described in our filings with the U.S. Securities and Exchange Commission. The statements in this press release are valid only as of the date hereof and we disclaim any obligation to update this information, except as may be required by law.

Chiesi Group Media Contact
Chiara Travagin
Rare Communication Manager
Tel: [phone]
Email [e-mail]

Alessio Pappagallo
Press Office Manager
Tel: [phone]
Email [e-mail]

Adam Daley
Berry & Company Public Relations
1-212-253-8881
[e-mail]

Protalix Investor Contact
Chuck Padala, Managing Director
LifeSci Advisors
646-627-8390
[e-mail]

PP-EF-0306 V1.0



(PRNewsfoto/Chiesi Global Rare Diseases)
(PRNewsfoto/Chiesi Global Rare Diseases)
Cision
Cision
View original content to download multimedia:https://www.prnewswire.com/news-releases/chiesi-global-rare-diseases-and-protalix-biotherapeutics-announce-fda-approval-of-elfabrio-pegunigalsidase-alfa-iwxj-for-the-treatment-of-fabry-disease-301820680.html

SOURCE Chiesi Global Rare Diseases; Protalix BioTherapeutics, Inc.



pressed in plant-cell culture aimed to provide a long

[midastouch017]

3.6000 +0.6800 (+23.2877%)
Pre-Market: 5:13AM EDT

Nice, very nice.

[bryanb40]

The milestone payments are broken up, some are regulatory and some are sales based.
it's close to 1 billion total possible I believe when US and EU are combined, but the approval milestones will be a fraction of that. They also got an advance in place of one of the later EU ones some time ago.
There is also some language that's been shared on other boards that limits the number of milestones they can collect at one time, at least, the sales based ones, to, I think, 1 per year. The additional earned milestones in that year would then be paid later after they pay the largest earned one first. This is presumably to protect Chiesi's bank account in case it gains instant traction in the market.
This was my understanding.

[midastouch017]

(Old 2017 article) PLX $320M in milestone payments

I have my doubts in regards above $$$ amount.
Hopefully the official PR later the day will contain
the correct milestone figure.
I suspect it will be lower, but this should not ruin
our party!
Mazzel - Tov longs!

[king oil]

Very high volume after hours. Tomorrow will be exciting. They finally got fda approval.

[MiamiGent]

PLX OK, it's moving in AH..
$2.92 was the close of regular session. today

Now, in AH session:
Refresh Quote
Bid ARCX 3.24 x 5
Ask ARCX 3.25 x 3
Volume
3,828,358

[jcon777]

Congrats again longs. Enjoy the ride up.

I have been waiting around 4 years for today.

[jcon777]

FDA approved Elfabrio!!!

https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2023

[MiamiGent]

PLX Ok, it passed FDA!
Now let's see if it runs.
Presently up a little in AH from $2.92 CLOSE
Bid ARCX 3.04 x 5
Ask ARCX 3.10 x 56
Volume 3,547,022

[jcon777]

Probably FDA approval tonight per a stocktwits user

Cash_flow (probably the best PLX poster on stocktwits) just posted this. I am copying what he wrote below

https://stocktwits.com/Cash_Flow/message/527049036

"BBB and I have been briefed. While we obviously can’t disclose any material info at this moment, he will be providing the hourly rocket emojis tonight and I’ll just leave you with this [Bullish icon]

[MiamiGent]

PLX So this was the preliminary good news.
I'm up a little but a small position as I'm leery of "sell the news".

BRIEF-Chiesi Global Rare Diseases And Protalix Biotherapeutics Announce European Commission Authorization Of PRX-102
REUTERS

7:31 AM ET 05/05/2023

May 5 (Reuters) - Protalix Biotherapeutics Inc (PLX):

* CHIESI GLOBAL RARE DISEASES AND PROTALIX BIOTHERAPEUTICS ANNOUNCE EUROPEAN COMMISSION AUTHORIZATION OF PRX-102 (PEGUNIGALSIDASE ALFA) FOR THE TREATMENT OF FABRY DISEASE Source text for Eikon

https://stockcharts.com/h-sc/ui?s=PLX

[jcon777]

(Old 2017 article) PLX $320M in milestone payments

I wonder if the above statement is still accurate from this article which is over 5 years old when Protalix entered into a partnership with Chiesi Farmaceutici

https://www.reuters.com/article/brief-protalix-biotherapeutics-enters-in-idUSFWN1MT09Y

[wgg2]

Thanks. Spideboy for your analysis of how market share may be achieved. I agree, assuming FDA approval. My thoughts continue to be with the 5 elements of revenues (initial sales of the substrate to Chiesi, regulatory milestones, commercial milestones, US royalties, & non-US royalties), and virtually no marketing expenses, with only1000 two-week dosed patients and 1000 once-a-month dosed patients 18 months in the future they will have the revenue stream that could very well generate $1.50 in net tax-free earnings per share. At a 15 multiple that suggests a price target of $22. This is with PLX getting on average 35% of the total revenues with Chiesi keeping the rest. In my thinking I have used about 75 million shares outstanding at that point. This would value PLX at about $1.6 billion at that point.
As to the current price volatility: I think it may be temporarily self-inflicted. Here is why: Selling more shares under the shelf registration in place with the SEC (and Wainwright). On 1/1/23 PLX had 53,790,167 shares outstanding. During the first quarter they sold 8,212,482 shares at an average price of $1.73 and raised $14.2 million. In early April after the quarter ended, they sold another 3.412,268 at an average price of $2.32 per share raising an additional $7.9 million. (see the 10Q) Looks like some profits are being realized (at least temporarily) regardless of the very favorable long-term outlook. In my view this too shall pass with the potential growth in the second half of the year.
Any other thoughts would be welcome.

[jcon777]

That makes sense. Thank you.

[king oil]

More people in EU but also more price control on drugs. The US allows practically any price the owner wants to charge. Potential revenue is probably bigger in the US.

[midastouch017]

A very convincing analysis,
thank you Spideyboy.