Biotech Values

[Mufaso]

VK2735 has specific liver fat reduction properties that are promising. See #msg-173042423. Time will tell if it also has an a "clinically meaningful" effect on fibrosis like the dual GLP1-GIP agonist trizepatide which does not exist as an oral.

Picking any small molecule GLP1 is not likely to be as effective.

[Mufaso]

Thanks for the link to the full article. They say it costs $70,000 to make a kilogram of semaglutide which implies 7 cents per mg. for material. It appears to me they are indeed neglecting the capital involved for building the factory which is not trivial.

[dewophile]

“The outgoing CEO of AlmataBio (the private company AVTX is acquiring) is Patrick Crutcher, who used to be a co-moderator of this message board.”

Very cool. Sounds like he is going to make out quite well. The company is due 7.5 M in cash now and 5M upon dosing the first patient into phase 2 which is a near certainty, along with stock valued at 15M. Another 15M (cash and or stock ) if it makes it to phase 3. Good for him.

[dewophile]

We might get a more definitive answer to this now that GSK might be incentivized to bear the cost of a large trial. The upside to their Shingrix franchise would be very significant if positive.

[dewophile]

A corollary here is that if you have a small molecule drug with lower efficacy but very clean safety and distinct MOA (i.e. combinable with a GLP-1 backbone) it might be written off by analysts due to efficacy when in fact it could have tremendous value in combination. That would be an entry I might eye in this area bc right now I’m late to the party and valuations are sky high. I have no idea what regimens will ultimately win the day but at least that would be an under appreciated entry

[iwfal]

IBIO—A second company issued a PR today mentioning both AI and obesity drugs—and it was good for a +192% move:

A) Suspect that specific bump is more about who bought it. See Twitter.

B) IBIO’s (unprovable) claims for AI are at least more reasonable than many since it seems to be more about molecular folding, which AI can actually do well, than the completely ridiculous sweeping claims of many. Eg zero chance AI understands all the pathways and loops of a highly pleiotropic system.

[DewDiligence]

Please see #msg-174128961.

[DewDiligence]

…if [VK2735] can be proven to work as an oral, an all oral combination with VK2809 for NASH is also appealing as we have discussed before.

My retort to that is that VK2809—or a similar agent—might combine even better with an oral small-molecule obesity drug rather than an oral peptide.

In addition to the points raised in #msg-174034595, there’s the issue of co-formulation into an FDC tablet. It ought to be easier to make an FDC from an oral small-molecule drug and a second oral agent than it is to make an FDC from an oral peptide and a second oral.

Of course an all-oral combination need not be an FDC; however, co-formulation has proven to be a major commercial benefit in many indications.

[Rocky3]

MRK and LLY have stayed near their highs. BMY and ABBV are still way off their lows,



MRK, LLY, and BMY hitting new highs. GILD too. ABBV has not returned to highs.

Well, since late 2022, LLY has continued be be a great place to be. MRK and ABBV are up near all-time highs (but not done any better than the S&P). GILD is down, and BMY has been a disaster.

[DewDiligence]

Tautological PR header?

https://www.globenewswire.com/news-release/2024/03/27/2853659/0/en/By-2035-Canada-could-have-the-most-enviable-healthcare-system-in-the-world-if-obesity-is-recognized-as-a-chronic-disease-predicts-Obesity-Canada.html

By 2035, Canada could have the most enviable healthcare system in the world if obesity is recognized as a chronic disease, predicts Obesity Canada

[DewDiligence]

VTRS Investor Day slide deck shows 2026 as “anticipated launch date” for Botox-biosimilar collaboration with RVNC:

https://investor.viatris.com/static-files/b01a07f3-e549-4522-8480-c31b359a6a4f (slide #14)

If this pans out it will generate material cash flow for RVNC in the next couple of years. The collaboration has $70M of remaining clinical and regulatory milestone payments. The collaboration also has up to $225M of sales-based milestones, although these presumably won’t be triggered until later in the decade.

[DewDiligence]

IBIO—A second company issued a PR today mentioning both AI and obesity drugs—and it was good for a +192% move:

https://finance.yahoo.com/news/ibio-astralbio-announce-transformative-ai-110000784.html

Caveat emptor, LOL.

Loosely similar PR from 2020: #msg-155310524.

[StarryEmpire4]

Here you go friend: https://archive.ph/Nh2Yq

Given what we now know about Viking's subQ and oral data, what price could the company command in a potential bidding war? $20B? $25B?

[DewDiligence]

AVTX is +250% in AH trading. The outgoing CEO of AlmataBio (the private company AVTX is acquiring) is Patrick Crutcher, who used to be a co-moderator of this message board.

This deal is structured as a regular-way merger, but it’s conceptually a reverse-merger insofar as AVTX’s lead asset is the one they just licked up from AlmataBio.

[TheFinalCD]

$avtx

Avalo acquires Phase 2-ready anti-IL-1ß mAb, AVTX-009, through acquisition of AlmataBio, Inc.
Topline results from planned Phase 2 trial in hidradenitis suppurativa expected in 2026
Executed private placement financing of up to $185 million, including initial upfront investment of $115.6 million; expected to extend cash runway into 2027
Webcast to be held tomorrow, March 28, 2024 at 8:30 a.m. E.T.

why cant they all do this

LL

[TheFinalCD]

$PDSB

$PDSB management team is living the dream with shareholders like this person.

Please, take more of my money!!!! pic.twitter.com/Y3eKD50H2L

— Adam Feuerstein ✡️ (@adamfeuerstein) March 27, 2024

[Mufaso]

NVO Ozempic costs- Bloomberg ran a similar story but most of it is behind a paywall and I don't subscribe. Here was the headline there:

Ozempic, Novo’s $1,000 Diabetes Shot, Can Be Made for Under $5 a Month
-Active ingredient in Ozempic costs 7 cents a dose, study finds
-Novo says it’s invested billions in research and production

If true, it's pretty crazy that the active ingredient itself is only 7 cents a dose. The factories aren't cheap. LLY is spending $2.5B on a German factory top make trizepatide and NVO paid quite a bit for Catalent but when you sell $billions/yr of a drug that only has an active costing pennies it still doesn't impact the cost much per dose.

https://www.bloomberg.com/news/articles/2024-03-27/ozempic-novo-s-1-000-diabetes-drug-can-be-made-for-less-than-5-a-month

[jbog]

WorstLuck

The key to me is that they're spending $1.47 in Q4 ($1.58 last year) for every $1.00 in sales after one year. So if the RHA is not growing it'll put more pressure on Daxi before we see any return.

[stocksrising]

Market watch ( as of 3/15/24) = % OF FLOAT SHORTED
17.93%

[jbog]

Ozempic manufacturing costs could be 89c per month, study finds

Novo Nordisk's (LLY) Ozempic could be manufactured for 89c to $4.73 for one month's supply, according to an investigation published in JAMA Network Open.

The study asks what prices of insulins, sodium-glucose cotransporter inhibitors and glucagonlike peptide 1 agonists could be if they were closer to the cost of production. "High prices limit access to newer diabetes medicines in many countries. The findings of this study suggest that robust generic and biosimilar competition could reduce prices to more affordable levels and enable expansion of diabetes treatment globally," the paper states.
The study argues that prices could decrease to $1.30 per month for treatment with SGLT2Is and under 75c for treatment with GLP1As. Further price reductions will likely become possible once a robust global generic and biosimilar market emerges, it adds.

Read more at:
https://thefly.com/n.php?id=3888598

[stocksrising]

On the CC , it was like the 3rd rail seeing not 1 of the analysts ( respectable peeps,-GS, MS, RJ, etc.) asked( if my recall is correct) …mgmt didn’t even guide (LYFGENIA- sickle cell) with clarity other than to say 50% of the 85-100 patient starts across 62 qualified treatment centers (QTCs) …a Q&A regarding TAM ( worldwide-all 3 fda approved indications ) was mid single digits ( ~3B+I believe)… so it’s wait and see what Q1, 24’ conf call suggests..
My take away is that they are going at this alone
( US) and will consider collaborating in other countries once they understand Payers & Regulations and who to dance with ….
Market digesting late 10k /amend and maybe short covering perhaps( thought I saw 15-18%)

[DewDiligence]

What is BLUE’s guidance with respect to attaining profitability?

[stocksrising]

Yes(accounting screw-ups), AND this management burns exorbitant amounts of cash consistently, but the Hercules Credit Facility should provide runway until Q1, 2026 per CC 🙏
Q1/2, 24’ should be decent considering several patients (ZYNTEGLO, SKYSONA) starts (cell collections) and Sickle cell( LYFGENIA) in Q3/4…
Hopefully Kevin Tang continues to build position(~1.7mm sh), as well as other Funds…
This kind of reminds me of RV*C, not sure what Rollout has been worse! ( and perhaps Mgmt) :(
However, Long both and accumulating 🙏

[DewDiligence]

PIRS terminates all R&D—becomes royalty-collection vehicle:

https://finance.yahoo.com/news/pieris-pharmaceuticals-announces-strategy-maximize-120000564.html

[DewDiligence]

Recent Biotech Bankruptcies/Liquidations/Dissolutions

[Added AMPE, BTTX, GMDA, and NBSE.]

 
                     iHub 
 Company      Date   Reference 
 ADLS         5/11   #msg-62813901 
 AFFY         6/14   #msg-103663749 
 AGIX         1/09   #msg-35019458 
 AKAO         4/19   #msg-148227772 
 ALNA         9/22   #msg-169875553 
 ALPIX.PA    11/23   #msg-173275218 
 ALTU        11/09   #msg-43509933 
 AMB.TO       8/09   #msg-40145970 
 AMPE         4/24   #msg-174111257 
 ANSV         1/10   #msg-45161615 
 ARAV         1/24   #msg-173646720 
 ARDM         2/19   #msg-146903332 
 ARLZ         8/18   #msg-142824112 
 ARNI        12/17   #msg-137120846 
 ARTE        12/08   #msg-33895436 
 ASP.TO       1/23   #msg-171046860 
 ATHX         1/24   #msg-173589749 
 ATNX         5/23   #msg-171913749 
 AXLA        12/23   #msg-173374718 
 BAXS        11/14   #msg-108147336 
 BCART.BR     9/23   #msg-172903024 
 BIND         5/16   #msg-122328088 
 BIOA         5/18   #msg-140549553 
 Biolex       7/12   #msg-77324773 
 BPUR         7/09   #msg-39629524 
 BTTX         3/24   #msg-174037486 
 BVTI‡        3/13   #msg-85440257 
 CALA         1/23   #msg-170898055 
 CBIO         6/22   #msg-169273606 
 CDAK         3/23   #msg-171544942 
 CJB.TO       7/10   #msg-52557510 
 CHKT        12/08   #msg-33949508 
 CLSC         8/09   #msg-40685152 
 CLVS        12/22   #msg-170599077 
 CRXT         9/22   #msg-170229149 
 CWBR        11/23   #msg-173138450 
 DCGN        11/09   #msg-43629379 
 DDXS         6/16   #msg-123229557 
 DMK          2/24   #msg-173773814 
 DNDN        11/14   #msg-108004147 
 DXTR        12/17   #msg-136829445 
 Egenix       1/15   #msg-109921347 
 ENDP         8/22   #msg-169707980 
 Epix†        7/09   #msg-39900524 
 EPRS         7/16   #msg-123687350 
 EYES         6/20   #msg-156388471 
 FRTX         9/23   #msg-172854853 
 GMDA         3/24   #msg-174125313 
 GNCA         5/22   #msg-168957883 
 GNTA         8/12   #msg-78123007 
 GTOP         9/08   #msg-32249160 
 HEMA         4/12   #msg-74167597 
 HGEN         7/23   #msg-172432338 
 HSTO         9/23   #msg-172847239 
 ILIU         7/17   #msg-133264435 
 IMMG         8/17   #msg-134305213 
 IMNP         2/19   #msg-146936897 
 IMV          5/23   #msg-171814610 
 INFI        10/23   #msg-172941539  
 Inspiration 10/12   #msg-81024196 
 INSY         6/19   #msg-149302493 
 Introgen††  12/08   #msg-33947783 
 Isolagen*    6/09   #msg-38746197 
 KBIO        12/15   #msg-119489701 
 KLDO         4/22   #msg-168491485 
 KV.A         8/12   #msg-78203130 
 LBPS         6/22   #msg-169255625 
 LHDX         2/22   #msg-171275021 
 LIAN         2/24   #msg-173835088 
 LMDX        12/23   #msg-173530663 
 MACK         2/24   #msg-173845049 
 MBRK         4/10   #msg-49658072 
 MBVX         3/19   #msg-151985211 
 MIIS        11/08   #msg-33720220 
 MIPI        12/10   #msg-57646943 
 MLNT        12/19   #msg-153074747 
 MNK         10/20   #msg-158843178 
 MRV.TO       3/23   PR link 
 NBSE         3/24   #msg-174119874 
 NEXI        11/23   #msg-173152495 
 NMTI         4/11   #msg-62251315 
 NMTR         7/23   SEC link 
 NOVN         7/23   #msg-172361873 
 NRGX         3/12   #msg-73415156 
 NRX          5/16   #msg-122331475 
 NSTG         2/24   #msg-173773766 
 NVDL         2/17   #msg-128923029 
 NVIV         2/24   #msg-173755991 
 NVLN         1/20   #msg-153111867 
 NVTA         2/24   #msg-173844970 
 OBSN.SW      2/24   #msg-173937364 
 ODT          3/21   #msg-162706983 
 ONCR         6/23   #msg-172037432 
 ONCS         6/23   #msg-172145325 
 OREX         3/18   #msg-139213097 
 ORPH         3/22   #msg-168169591 
 OSCI         7/09   #msg-39511719 
 OTIC        12/22   #msg-170737355 
 PEAR         4/23   #msg-171639648 
 PHAS        10/22   #msg-170272758 
 PLXP         4/23   #msg-171677346 
 PROMO.ST    10/23   #msg-173007859 
 PTX          2/19   #msg-146937356 
 PYMX         4/13   #msg-86313192 
 PZRX        12/17   #msg-136794351 
 QTNT        12/22   #msg-170676963 
 RCPI         8/16   #msg-127560117 
 REX         11/08   #msg-32189097 
 ROKA         8/17   #msg-133945967 
 ROSG         6/18   #msg-141797330 
 RUBY         2/22   #msg-171270235 
 RVALL        1/20   #msg-153358221 
 RVLP        10/23   #msg-173007859 
 RXPC         7/14   #msg-104103329 
 SCLX         2/23   #msg-171193757 
 SGYP        12/18   #msg-145394802 
 SIEN         2/24   #msg-173834941 
 SIGA         9/14   #msg-106313111 
 SIOX        12/22   #msg-170699458 
 SNNA         9/19   #msg-151196707 
 SPHS         5/20   #msg-155712447 
 SRGA         6/23   #msg-172201878 
 SRNE         2/23   #msg-171193757 
 SVNT        10/13   #msg-92997648 
 TDLP         6/11   #msg-64744114 
 TLCV        12/09   #msg-44751578 
 TMBR        11/23   #msg-173254229 
 TOMDF       12/23   #msg-173490278 
 TPPH         2/08   #msg-26765260 
 VION        12/09   #msg-44654555 
 VLRX         2/20   #msg-153741993 
 VRA         10/07   #msg-18053275 
 VRAY         7/23   #msg-172361873 
 VVUS         7/20   #msg-156752080 
 ZSAN         6/22   #msg-169035970

*Emerged from bankruptcy as FCSC.

†Not to be confused with ESSA Pharma.

††Not to be confused with Inogen.

‡Second BK filing.

[DewDiligence]

GMDA—(-82%)—announces “packaged bankruptcy” recapitalization:

https://finance.yahoo.com/news/gamida-cell-announces-commencement-restructuring-110000176.html

[DewDiligence]

AVIR completes enrollment of phase-3 COVID trial—cancels interim analyses:

https://www.globenewswire.com/news-release/2024/03/27/2853010/0/en/Atea-Pharmaceuticals-Completes-Patient-Enrollment-in-Global-Phase-3-SUNRISE-3-Trial-Evaluating-Oral-Antiviral-Bemnifosbuvir-for-COVID-19-in-High-Risk-Patients.html

Atea Pharmaceuticals…today announced that the company has completed enrollment of the global Phase 3 SUNRISE-3 trial evaluating bemnifosbuvir, an oral nucleotide polymerase inhibitor, or placebo for the treatment of COVID-19. Over 2,200 patients were randomized into the supportive care monotherapy cohort… The primary endpoint of the trial is all-cause hospitalization or death through Day 29 post-treatment in the bemnifosbuvir supportive care monotherapy cohort of high-risk patients.

The two planned interim analyses have been canceled:

“Since full enrollment was achieved ahead of the two planned interim analyses for safety and futility by the Data Safety Monitoring Board (DSMB), the analyses are no longer relevant, and in agreement with the DSMB, we will proceed to the full analysis of the trial. We look forward to reporting SUNRISE-3 results in the second half of 2024…

[WorstLuck]

Jbog: I have no idea on RHA. Expanding Daxi sales would seem to be a driver of sales there, no?

potentially as late as Friday.

Housekeeping note on my prior comment: The market is closed Friday.

[DewDiligence]

HOTH cites AI and obesity drugs_in_same_PR and gets only a 3% bump, LOL:

Hoth Therapeutics' Wholly Owned Subsidiary Merveille.ai, Advances AI-Driven Discovery for Obesity Treatment

[Mufaso]

Lots of differing opinions on how much muscle loss comes with weight loss and for what reason. Obviously, you will get different percentages based on what your base BMI is but then it is be affected by lots of factors like how much exercise one does while in caloric deficit and/or how much protein is consumed.

I posted on ALT claim as I believe going forward, how much muscle is lost while on a weight loss drug will become a battleground between different drugs and an important market segment. Their specific claim was "only 25.5% from lean mass, comparable to the effects historically associated with weight loss from diet and exercise programs,” Anecdotally, I believe the general consensus is that 30%-40% of the typical weight loss for an obese individual is muscle. (BTW- A big problem occurs when people yo-yo back up as the weight they put back on is disproportionately higher in fat) . An article I reference below says the muscle loss figure is one third, so take your pick as to what an "average/typical" number is.

“When we look at weight reduction from any intervention, about one-third of the weight we lose tends to be lean mass, and that can be problematic,” said Dr. Jaime Almandoz, an associate professor of internal medicine in the Division of Endocrinology at UT

But answering your question on comparable drugs to ALT's I previously posted in #msg-173034452 that supposedly Wegovy weight loss was 40% muscle and Mounjaro was 25% from this article:
https://www.nbcnews.com/health/health-news/weight-loss-drugs-muscle-loss-rcna84936

Here is some data in the article from the Wegovy trial saying 40% of the weight loss was Muscle mass.

A 2021 clinical trial that tested the weight loss effects of Wegovy found that while about 40% of the weight people lost tended to be lean mass, including muscle, at the end of 68 weeks, people who took the drug had a lower risk of heart disease and better physical function compared to a placebo group.

Here is some data from a Mounjaro trial saying the muscle loss was only 25%:

Studies showed similar results for the drug tirzepatide, which is sold under the brand name Mounjaro. In a phase 3 clinical trial, “a reduction in lean mass was reported at a similar rate as what is seen in lifestyle-based treatments for obesity,” Jessica Thompson, a spokesperson for tirzepatide-maker Eli Lilly, said in an emailed statement

A new analysis of data from the same trial found that after 72 weeks of taking the drug, about 25% of the weight participants lost was lean mass. However, the participants had an overall healthier body composition. Aronne, who worked on the trial, presented the findings this week at the European Congress on Obesity in Dublin.

Viking has yet to comment on muscle loss to my knowledge.

Disclaimers about the follies of comparing different trials aside, I would be very interested in any data anyone has that shows that semaglutide is better than tirzepatide for muscle mass retention or any other thoughts anyone has here. I suspect that all of these companies will slice the data differently to make their particular drug look better than competition..
.

As a final thought, BioAge is running a trial for azelaprag, an apelin receptor (APJ) agonist, in combination with Lilly’s Zepbound (tirzepatide) and therapeutic pipeline. See #msg-173838283. Hopefully when this trial runs we will see a comparator arm on tirzepatide that will show what tirzepatide muscle loss is alone in a defined patient population.

[randychub]

How do these lean tissue numbers compare to VKTX and other drugs in this space


Our data show that 74.5% of weight loss was derived from adipose tissue and only 25.5% from lean

[Mufaso]

Altimmune Announces Positive Lean Mass Preservation Data for Pemvidutide along with 4thQ/yearend results:

“We are extremely pleased with the results of the body composition analysis from our recently completed MOMENTUM 48-week Phase 2 obesity trial of pemvidutide. Our data show that 74.5% of weight loss was derived from adipose tissue and only 25.5% from lean mass, comparable to the effects historically associated with weight loss from diet and exercise programs,” said Vipin K. Garg, Ph.D., President and Chief Executive Officer of Altimmune.

“Based on compelling weight loss, a clean safety profile, robust reductions in serum lipids and blood pressure, and now preservation of lean mass observed in our clinical trials, we believe that pemvidutide has the potential to distinguish itself broadly from other therapies for the treatment of obesity.

We also remain excited about the outcome of our ongoing IMPACT Phase 2b MASH trial with topline 24-week data on the key endpoints of MASH resolution or fibrosis improvement anticipated in the first quarter of 2025. The results from a recently completed preclinical study demonstrating direct anti-fibrotic activity of pemvidutide only adds to our optimism about achieving a positive outcome in this trial.”

https://ir.altimmune.com/news-releases/news-release-details/altimmune-announces-positive-lean-mass-preservation-data

[DewDiligence]

FDA approves MRK’s Winrevair—(sotatercept)—for PAH:

https://www.businesswire.com/news/home/20240326077272/en

This is the first approved drug for PAH that is thought to be disease modifying. MRK acquired sotatercept in the $11.5B buyout of XRLN in 2021 (#msg-166156870).

[iwfal]

There is data that shingrix (and before that zostavax) is associated with lower rates of dementia.

Similarly there is also data ppl getting treated for oral herpes… have lower rates of Alzheimer’s (altho, IIRC, it was one sex and not the other). And I think I’ve seen one other vaccine that had similar. So seems likely to be real?

[iwfal]

RVNC

In 4th q 22 sales were $34.7m and 2023 sales were $30.3, $31.7, $32.1 and $34.5 for the 4 quarters. Total sales year over year were nice but I'm not sure why it looks they hit a wall.

This was, even at the time, a fairly obvious result of Daxxy being highly anticipated with only limited launch spots so they prioritized those who made big order of prior products.

[DewDiligence]

Thanks. See #msg-174114321 for more color on the same topic.

[TheFinalCD]

$VKTX conference call done- Summary

1) Safety profile similar to placebo even at high dose of 40mg
2) Expecting higher dose cohorts, no disclosure on how many in ph2
3) High placebo effect due to small N and diarrhea in placebo arm
4) Expecting placebo effect to move back…

— Pharmdca (@Pharmdca) March 26, 2024

$VKTX there’s seems to be no side effects with oral drug even at high doses

Profile like placebo

Remember- $VKTX is the only, only dual agonist and GIP agonist helping with counterbalancing GI side effects

✅✅

— Pharmdca (@Pharmdca) March 26, 2024

BTW Thanks for the reply

much appreciated

TEAMWORK makes the DREAMWORK
https://investorshub.advfn.com/boards/read_msg.aspx?message_id=173997658&txt2find=vktx

[DewDiligence]

NBSE prepares for dissolution:

https://www.sec.gov/Archives/edgar/data/1173281/000110465924038432/tm249597-1_pre14a.htm

To Our Stockholders:

You are cordially invited to attend a Special Meeting of Stockholders (the “Special Meeting”) of NeuBase Therapeutics, Inc. (the “Company”) to be held on May 13, 2024, at 8:30 a.m., Eastern Time, exclusively online via live audio-only webcast.

The purpose of the Special Meeting is to approve the liquidation and dissolution of the Company (the “Dissolution”) and the Plan of Liquidation and Dissolution (the “Plan of Dissolution”), which, if approved, will authorize the Company’s Board of Directors (the “Board”) to liquidate and dissolve the Company in accordance with the Plan of Dissolution.

LOL re the faux cordialty.

NBSE is the product of a reverse-2019 merger (#msg-145848413).

[RNsidersbuying]

NKTX 9.87 - the April19-$10-Call Option is currently bidding at $1.10. Their recent K filing showed $5+ cash remaining and they just secondaried at $10 which should put them well above $5+ cash per share accompanied by some upcoming inevitable touting...one can only hope the option premiums remain this high?

https://finance.yahoo.com/news/nkarta-announces-pricing-240-million-103000060.html

New and existing investors participated in the offering, including Adage Capital Partners LP, Boxer Capital, Commodore Capital, Cormorant Asset Management, an affiliate of Deerfield Management, EcoR1 Capital, Janus Henderson Investors, OrbiMed, RA Capital Management, Ridgeback Capital Investments, Samsara BioCapital, SR One, and a leading mutual fund.

[DewDiligence]

BLUE—(-13%)—discloses accounting screw-up—issues 2024 patient-start guidance:

https://finance.yahoo.com/news/bluebird-bio-reports-fourth-quarter-110100135.html

On March 26, 2024, bluebird announced that it will restate its consolidated financial statements for 2022, and for the first three quarters of both 2022 and 2023 in its Annual Report on Form 10-K for the year ended December 31, 2023 (the "2023 Form 10-K"). The restatements relate to the identification of embedded leases and the treatment of non-lease components contained in lease agreements with contract manufacturers. As a result, the Company anticipates recording an increase in lease assets and lease liabilities, as well as an increase in non-cash interest expense in each restated period.

Meanwhile, BLUE issued 2024 guidance for 85-105 combined patient starts for its three approved gene therapies: Lyfgenia, Zynteglo, and Skysona. In 2024 to date, BLUE has performed 9 patient starts for Zynteglo and Skysona, while Lyfgenia has not yet had a patient start. In 2023, BLUE had 26 patient starts on Zynteglo, and Skysona.

[dewophile]

VKTX up 20%, enta up 17% riding its coattails (just kidding i have no clue why enta also up today could be something on the legal front but nothing obvious that I can find https://www.pacermonitor.com/public/case/44980990/Enanta_Pharmaceuticals,_Inc_v_Pfizer_Inc)

[Whalatane]

SWAV. (Reuters)

-Johnson & Johnson is in talks to buy medical device maker Shockwave Medical, the Wall Street Journal reported on Tuesday, with a deal likely to help the healthcare giant expand its presence in cardiovascular devices.

Shockwave, a company that makes devices to treat heart disease, has a market capitalization of around $11 billion, according to LSEG data.

A deal could be finalized in the coming weeks, assuming talks don't fall apart, and it is also possible another suitor could emerge, the report said, citing people familiar with the matter.

Kiwi

[DewDiligence]

FIXX—>QTTB, pursuant to previously announced reverse-merger:

https://www.prnewswire.com/news-releases/q32-bio-announces-closing-of-merger-with-homology-medicines-and-concurrent-private-placement-of-42-million-302098557.html

[DewDiligence]

MRNA’s next-gen COVID vaccine produces_stronger immune response_than Spikevax:

https://www.accesswire.com/846784/moderna-achieves-positive-interim-results-from-phase-3-trial-of-next-generation-covid-19-vaccine

The next-gen vaccine, mRNA-1283, also has a longer shelf life and less onerous storage requirements compared to Spikevax.

[dewophile]

OT

[Dementia is] not even a little bit good for those around you. That may require some thought and personal choices made early in the progression...

My wife has the green light to take me to a state where it is legal and put me down (and vice versa)

[jfmcrr]

Yes the cause is probably multi factorial and the disease heterogeneous as well

I'm a 73 year old whippersnapper. I want to grow up to be N40K. I may get there. My family manages to get to the 90's, with a little heart trouble and cancer here and there. Dementia may or may not take the fun out of it for me, but it's not even a little bit good for those around you. That may require some thought and personal choices made early in the progression...

[Mufaso]

VKTX cc on VK2735 was very well received by the analyst community. Most seemed amazed by the tolerability/safety results. Here are some initial thoughts:

• Outstanding tolerability. "Almost no side-effects”. No clinically significant difference between study participants and placebo
  
• 2 subjects had diarrhea in placebo compared to 1 in the 2nd highest dose of 20 mg.
  
• placebo effect was high at 2.1% weight loss. Possible reason was the small numbers (n=10 for placebo) and 2 subjects had diarrhea.
  
• 2 total discontinuations- one for family reasons- other was for abdominal reasons.
  
• Noted that effect was durable 6 days after study dosing stopped while placebo group gained 0.5% weight. Had this been included the placebo adjusted weight loss would have been 3.8%. Also noted that other drugs in testing had rebounds immediately after dosage stopped.
  
• PK was such that they saw slow accumulation- seems like VK2735 is almost "self-titrating". Brian Lian when asked if there was a need to titrate said "doesn't look like it but we will continue to do titrate"
  
• Found it interesting that one analyst (Justin Zelin at BTIG) asked if oral was safer than Subq version.
  
• Viking did not disclose what the "special formulation" was for an oral peptide that made it work.
  
• Adding another cohort at higher dose- likely to see increased weight loss. Overall study numbers may change slightly. Adding a few more placebo at the same time.
  
• Question about manufacturing a peptide and whether there was a point at where it didn't make sense to dose scale- answer was "we aren't stopping and we are spending a lot of time on manufacturing- currently have plenty of material for registrational studies". Brian Lian did not seem deterred at all by this question.
  
• Regarding food, participants were asked to dose in the am after waking and not to consume food for 30 mins.
  
• Just got data so no Metabolic data available yet (e.g. for lipids, glucose etc.). Will likely release metabolic data towards end of the summer.

I think this was a home run. Placebo adjusted Weight loss close to 4% at 28 days, clear indication of dose response, incredibly good safety profile.

[Mufaso]

Viking Therapeutics Announces Results from Phase 1 Clinical Trial of Oral Tablet Formulation of Dual GLP-1/GIP Receptor Agonist VK2735

https://ir.vikingtherapeutics.com/2024-03-26-Viking-Therapeutics-Announces-Results-from-Phase-1-Clinical-Trial-of-Oral-Tablet-Formulation-of-Dual-GLP-1-GIP-Receptor-Agonist-VK2735

• Up to 3.3% Placebo-Adjusted Mean Weight Loss (5.3% from Baseline) Observed After 28 Days
  
  
• VK2735 Shown to be Safe and Well-Tolerated in 28-Day Study with Low Rates of GI-Related Adverse Events
  
  
• Phase 2 Trial in Obesity Planned for 2H24

[DewDiligence]

Insider-trading trial against Medivation executive tests_the_definition_of_the_term:

https://finance.yahoo.com/news/sec-tests-insider-trading-theory-000517436

The civil trial that began Monday in San Francisco will answer the novel question of whether buying or selling another company’s stock based on specialized industry knowledge constitutes insider trading.

… Securities traders and lawyers are closely watching the San Francisco case of Matthew Panuwat, the SEC’s first attempt to pursue so-called shadow trading. It also underscores how Congress has never explicitly defined insider trading, leaving courts to decide when the SEC oversteps its authority.

… Seven minutes after receiving an August 2016 email from Medivation’s CEO that said Pfizer Inc. was interested in acquiring it, Panuwat invested more than $117,000, or almost half of his annual salary, on call options in rival Incyte Corp., according to the agency.

Pfizer announced the $14 billion Medivation deal days later. By taking one mid-cap biopharma company off the market, the deal made Incyte a more valuable acquisition target, the SEC alleged in its suit against Panuwat.

[DewDiligence]

AMPE voluntarily delists—prepares for likely liquidation:

https://finance.yahoo.com/news/ampio-announces-voluntary-delisting-sec-210000274.html

…the Board has determined that it is in the Company's best interests that the Company take steps designed to ensure sufficient cash to adequately fund an orderly wind down of the Company's operations…

AMPE has essentially been a shell company for the past two years (#msg-168609120).