PROTALIX BIOTHERAPEUTICS, INC. (PLX)

[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
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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.

[Spideyboy]

Honestly I feel a very big chunk of it.

Compared to Fabrazyme it has at least equal efficacy and superiority in safety. That was in the clinical trial setting. But in the real world Elfabrio would have greater efficacy as we should remember that these patient came into the Balance trial with a terrible mean -8 eGFR slope despite being on Fabrazyme already for years.

I would not understand if being the treating physician why I would not put my more severe/classical patients on Elfabrio due to the efficacy, and I would not understand why I would not put the same classical as well as my other patient on Elfabrio due to the efficacy and safety.

Now this is compared to Fabrazyme.

Now let's consider vs Replagal, where Elfabrio results were incredible both on efficacy and safety again.

Now let us also consider this from a patient perspective. Which treatment would you rather be on? You have a choice of equal or better efficacy with better safety, for a drug that you will take every 2 weeks for the rest of your life and which is keeping you alive.

This is evidenced by the Balance clinical data vs Fabrazyme.We note that in this vs Fabrazyme trial, 97% of the patients completing the trial, 96% (45 of 47) in the Elfabrio arm opted to continue on Elfabrio, and also the 100% (24/24) of the Fabrazyme arm patient opted to initiate on Elfabrio.

The physician and patient community is small and well informed. I expect Elfabrio to make strong sales. To support this we should also note that Chiesi is a strong and well recognised partner, I would expect swift ramp-up with strong continuation of efforts. Time is money. All parties are strongly incentivised to maximise sales.

I would expect Elfabrio to take at least 50% of the US market as it will be the only other option to Fabrazyme, if approved of course, (I think we can ignore Galafold, after so many years they are only treating just about 1000 patients globally). Elfabrio would also take about 50% of the Fabrazyme market in Europe.

But in Europe I would expect Elfabrio to take another 50% from the Replagal market. Hence I would expect Elfabrio to be the dominant player in the EU.

Again just think about, as a physician and patient, why would you not switch to Elfabrio?

While I am not a Fabry patient, I would be almost certain that if my insurance covered both Elfabrio and Fabrazyme, I would most certainly want to be on Elfabrio. The Balance was a trial that was a switch from Fabrazyme, so I would feel very comfortable knowing that this would directly relate to me.

If I were the physician, I might be even more prone to switch my patient because of the clear data proven safety benefits and avoid getting sued in the future.

Again, I would like to hear any reasons as to why not switch to Elfabrio (assuming insurance coverage of course).

[midastouch017]

Protalix Biotherapeutics

https://finance.yahoo.com/news/weekly-biotech-pulse-big-catalysts-063256242.html

Another day, another PDUFA. On the docket for Tuesday is a PDUFA review for Protalix Biotherapeutics' (NYSE:PLX) PRX-102 compound, its Fabry disease candidate - news that InvestingPro subscribers will get in rapid fire.

Protalix likewise got a CRL back in April 2021 and also appears to have regained some trust among investors ahead of the key decision: Shares have gained just shy of 140% since the start of the year.

The company has 3 buy ratings from analysts, 5 holds, and 0 sells ahead of the decision.

[jcon777]

EMA & FDA markets & population sizes

Anyone have any thoughts of how much profit is in Europe versus U.S. with the populations listed below?

Population of Europe is 746.4 million (2018)
Population of U.S. is 331.9 million (2021)

Since Europe is more than double the size of the U.S., it seem there would be a lot more of the market for Protalix in Europe with EMA approval than with the U.S. with FDA approval.

[jcon777]

Here are the PLX links needed this week

I am copying this from stocktwits and then copying the full message below:
https://stocktwits.com/Taliban_Gigolo/message/526677706
----------------------------------------------------------------------------------------------------------------
FDA Drug Approvals
https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=reportsSearch.process
FDA Press Announcements
https://www.fda.gov/news-events/fda-newsroom/press-announcements
PR News Wire – PLX
https://www.prnewswire.com/news/protalix-biotherapeutics%2C-inc./
PLX Press Releases
https://protalixbiotherapeutics.gcs-web.com/press-releases

[midastouch017]

Protalix, Chiesi challenge Sanofi with EU approval for Fabry disease med as FDA decision looms

https://www.fiercepharma.com/pharma/protalix-chiesi-earn-approval-europe-fabry-disease-enzyme-replacement-therapy

By Kevin DunleavyMay 5, 2023 02:40pm

Protalix BioTherapeutics and Chiesi Farmaceutici have endured a rejection from the FDA and conducted multiple readouts of a phase 3 trial of their Fabry disease candidate.

After many stops and starts, the companies have finally scored a marketing approval in Europe for PRX-102 (pegunigalsidase alfa), an enzyme replacement therapy for the rare, genetic, progressive disorder, which causes an accumulation of fatty deposits in the lysosomes and strikes roughly 1 in 50,000 people.

The authorization is based on results from a clinical program that has tested PRX-102 in more than 140 patients with up to 7.5 years of treatment.

GlaxoSmithKline bails out of its Fabry deal with Amicus

A head-to-head trial pitting PRX-102 against Sanofi’s Fabrazyme (agalsidase beta) demonstrated noninferior efficacy in controlling the kidney disease that accompanies the disorder.

Fabrazyme, which was approved 20 years ago, is the longtime dominant drug in the Fabry disease market, generating sales of 938 million euros ($986 million) last year.

Amicus Therapeutics of Philadelphia, which earned an FDA approval for its Fabry disease drug Galafold in 2018, reported sales of $329 million last year for the treatment, which has the advantage of coming in tablet form as opposed to Fabrazyme, which—like PRX-102—is administered through IV.

Both Fabry disease treatments cost more than $300,000 annually.

Chiesi and Protalix are still awaiting a nod in the U.S. after getting a fast-track designation in 2018 and submitting for approval two years later. Inspection delays—brought on by the pandemic—helped lead to the FDA rebuff in April of 2021. The companies resubmitted 14 months ago, with an FDA decision expected next week on May 9.

Fabry patients petition NIH on shortage

With an approval in the U.S., Protalix of Israel would receive a milestone payment from Chiesi of Italy, which is commercializing the treatment worldwide.

[midastouch017]

Fabry Disease Treatment Market

https://www.gminsights.com/industry-analysis/fabry-disease-treatment-market#:~:text=Fabry%20Disease%20Treatment%20Market%20was,8%25%20from%202022%20to%202030.

With (hopefully) FDA approval, how much of a market share
could PLX achieve?

[king oil]

I gave up commenting because I had nothing new or good to say. But I held a chunk of shares in case this succeeded. I still don’t trust the FDA.

[midastouch017]

Milestones

https://finance.yahoo.com/news/plx-one-down-one-112600835.html

? PRX-115 to start Phase I – 1Q:23

? EMA approval for PRX-102 –May 5th, 2023

? FDA PDUFA date for PRX-102 – May 9th, 2023

? Investor event: Protalix Strategy – late June 2023

? PRX-115 Clinical Study Report – 1Q:24

Valuation

We update our valuation to reflect the May 5th, 2023 approval of PRX-102 by the European Commission (EC) and the increase in share count as a result of the ATM-related capital raise year to date. We had applied an 80% probability of EC approval and have now increased this to 100% following EC approval. We will adjust our probability of success for FDA approval when the status of PRX-102 is clear next week. We now have an 80% chance of success with the US regulatory body. We also shift our DCF valuation forward by one year. The net of our changes results in an increase in our target from $11 per share to $15 per share.

Summary

Protalix has received approval from one regulatory agency and should see a response from the other next week. Chiesi will take over the commercialization of PRX-102 following approval and the focus for Protalix will turn to the development assets are now moving towards center stage. The Phase I trial for PRX-115 has begun and the first 9 of an anticipated 56 subjects have been enrolled. Product sales were down year over year in 1Q:23; however, this is not a surprise as ordering and delivery for Pfizer and Fiocruz are volatile. License and R&D revenues were also down, reflecting this shift away from the development stage for PRX-102. While expectations for Elelyso are low, the product has been generally improving in its revenue contribution over the last several quarters and may benefit from additional sales in Europe following the expiration of exclusivity of Vpriv. EC approval supports a valuation increase. We update our valuation to $15.00 per share.

[jcon777]

My plan is to follow BLRX more closely

I planned to get through these PLX approvals and then look at my BLRX holdings more closely before their September PDUFA

[jcon777]

of course i'd like to be wrong!

I think this is the first time that I would like you to be wrong too. :)

But even if you are right, we can be very confident that the next few days, weeks and months should be exciting (after hopefully the FDA approves of course).

Mazel Tov!

Have a great weekend all! We waited a long time for this.

[midastouch017]

Lol, i have just posted on the BLRX board!

[midastouch017]

I do not expect a substantial price increase today,
of course i'd like to be wrong!

Fingers crossed for me being wrong in above prediction.

[kidscosttoomuch]

GMDA now PLX just need BLRX for the trifecta

[jcon777]

Stock market opens in 5 minutes.

Now the fun starts for PLX holders.

Mazel tov.

[midastouch017]

LOL, indeed so, but finally patience
has its merits!
Truth is, i thought you gave up years
ago!

[midastouch017]

FDA on Tuesday (or sooner and hopefully not much later)

From my recent GMDA experience, FDA announces sooner
or at exactly the PDUFA date.
It would be nice to enjoy a double slam-dunk today to make
a perfect weekend!

Mazzel - Tov!

[jcon777]

Congrats longs and friends!

Yes Midas, now we hopefully hear good news from the FDA on Tuesday (or sooner and hopefully not much later)

[king oil]

Feels like it’s been 100 years

[midastouch017]

Now looking forward to FDA approval!

[midastouch017]

Chiesi Global Rare Diseases and Protalix BioTherapeutics Announce European Commission Authorization of PRX-102 (pegunigalsidase alfa) for the Treatment of Fabry Disease

https://finance.yahoo.com/news/chiesi-global-rare-diseases-protalix-104600663.html

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

PARMA, Italy, BOSTON and CARMIEL, Israel, May 5, 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®, today announced that the European Commission (EC) has granted marketing authorization to PRX-102 (pegunigalsidase alfa) in the European Union (EU) for the treatment of adult patients with Fabry disease.

"People living with Fabry disease often perceive their disease as burdensome and still experience unmet medical needs," said Giacomo Chiesi, head of Chiesi Global Rare Diseases. "Our deepest gratitude to all patients and patient advocates who have stood shoulder-to-shoulder with clinical researchers, scientists and regulators during the clinical development program, providing the data needed for this approval. I believe this is a vital ingredient in bringing innovation to the real lives of patients and enabling hope and definitive, integrated solutions."

"We are delighted that the European Commission has approved PRX-102 for the treatment of adult patients with Fabry disease. The EU authorization is a testament to our commitment to deliver innovative therapies and solutions for people affected by rare diseases," said Diego Ardigò, M.D., Ph.D., head of research and development of Global Rare Diseases at the Chiesi Group. "As a certified B Corp we are committed to ensuring access to PRX-102 to as many people living with Fabry disease as possible and thank those who participated in our extensive clinical research program. It is important to deliver this new treatment option to reduce the burden of this chronic disease on patients, their families, and the healthcare system."

"The European Commission's approval of PRX-102 is a significant milestone for patients with Fabry disease and their families, providing a new therapeutic option," said Dror Bashan, Protalix's President and Chief Executive Officer. "We are proud of this achievement and believe that this approval further validates our science and technology. Based on solid results from our robust clinical programs, PRX-102 has the potential to be widely used for many years to come. Together with Chiesi, we remain committed to meeting the needs of patients with Fabry disease and bringing this new treatment option to market."

PRX-102 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 EC authorization of PRX-102 is based on results from a comprehensive clinical development program in more than 140 patients with up to 7.5 years of 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 PRX-102 demonstrating non-inferior efficacy to agalsidase beta in controlling kidney disease as evaluated by the estimated glomerular filtration rate (eGFR) decline.

Pegunigalsidase alfa, an investigational new drug product, is currently not approved by the U.S. Food and Drug Administration (FDA). The effectiveness and safety of pegunigalsidase alfa is under review, but has not yet been approved, by the FDA. Prior to FDA review and approval, no conclusions can be drawn on pegunigalsidase alfa's efficacy and safety profile. When seeking expanded access, treating physicians should consider all possible risks of treatment with pegunigalsidase alfa. Access must be compliant with all applicable federal and state laws and regulations. Investigators should not seek reimbursement for product provided to patients who participate in a government funded insurance program.

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 PRX–102

PRX–102 (pegunigalsidase alfa) is a PEGylated enzyme replacement therapy (ERT) to treat Fabry disease that is now approved by the European Medicines Agency (EMA) and is under evaluation by the FDA. PRX-102 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, PRX–102 has been observed to have a circulatory half-life of approximately 80 hours.

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.

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 timing, progress and likelihood of final approval by the FDA of the resubmitted Biologics License Application (BLA) by the PDUFA action date, if at all, and, if approved, whether the FDA will impose significant limitations on the use of PRX–102; risks related to the commercial success of PRX-102, and of Protalix's other product and product candidates, if approved; the likelihood that the FDA, EMA or other applicable health regulatory authorities will approve an alternative dosing regimen; 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]



(PRNewsfoto/Chiesi Global Rare Diseases)
(PRNewsfoto/Chiesi Global Rare Diseases)
Cision
Cision
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SOURCE Chiesi Global Rare Diseases; Protalix BioTherapeutics, Inc.