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Cool
1 day ago
Definium Therapeutics, Inc. (NASDAQ:DFTX) has now delivered a third consecutive positive Phase 3 readout for its LSD-based medication, the second in generalized anxiety disorder. The result puts the company on a viable route toward a potential FDA approval of an LSD-based treatment for generalized anxiety disorder, and considering how closely psychedelic stocks have historically traded on each other's data, the reading carries far more weight than Definium's own ticker.
On September 14, Definium Therapeutics, Inc. (NASDAQ:DFTX) announced that its drug DT120, an orally disintegrating tablet formulation of LSD, was successful in Panorama, the company's second late-stage anxiety trial. Over 12 weeks, patients who received a 100-microgram dose experienced a 9.8-point decline on the Hamilton Anxiety Rating Scale, a typical clinical measure, compared to a 4.7-point drop with placebo.
The resulting 5.1-point placebo-adjusted improvement is similar to what the company's first pivotal anxiety trial, Voyage, showed back in August: an 11.6-point improvement versus 6.2 for placebo, a 5.4-point separation that Jefferies called one of the strongest placebo-adjusted efficacy results ever seen in generalized anxiety disorder, and that Stifel simply described as "a clean win."
Psychedelic equities have a history of trading as a group rather than as individual names, and this tendency applies both ways. In February 2026, when Compass Pathways, the sector's other clinical leader developing a psilocybin-based drug for treatment-resistant depression, reported positive late-stage data, shares of Definium Therapeutics, Inc. (NASDAQ:DFTX), Atai Beckley, GH Research, and Helus Pharma all rose, some by double digits.
That correlation is significant here because Definium's win is an independent validation of the broader therapeutic argument on which Compass and others are betting: a single dose of a classic psychedelic, administered in a controlled clinical setting, can produce lasting improvements in serious mental health conditions via pathways different from existing SSRIs and other standard-of-care drugs.

#definium #placebo
hixaxedarihazana
3 days ago
Evommune, Inc. (NYSE:EVMN) announced on September 8 that EVO756 missed its primary endpoint in a randomized, double-blind, placebo-controlled Phase 2b trial involving 121 adults with moderate-to-severe atopic dermatitis. The primary measure was the percentage change from baseline in Eczema Area and Severity Index, or EASI, at week 12. No tested dose met the primary or secondary endpoints, and development in that indication will stop.
Evommune, Inc. (NYSE:EVMN) had already discontinued EVO756 for chronic spontaneous urticaria following a June trial failure. Development continues in migraine prevention. The setbacks place greater weight on EVO301, which has positive Phase 2a eczema data and a Phase 2b study planned for mid-2027.
Evommune, Inc. (NYSE:EVMN) has clinical evidence supporting its next eczema program. In February, the company reported that EVO301 met the primary endpoint in a randomized, double-blind, placebo-controlled Phase 2a trial involving 70 adults. Participants received two intravenous doses or placebo over a 12-week study.
Evommune, Inc. (NYSE:EVMN) reported a 55% EASI reduction with EVO301 versus 22% with placebo at week 12, a difference of 33 percentage points. The study met its prespecified Bayesian success criterion, and a separate conventional ****** ysis showed statistical significance at p
8zf7aot0bo3x60bw
5 days ago
Pfizer Inc. (NYSE:PFE) and Valneva SE (NASDAQ:VALN) reported on August 14 that the European Medicines Agency has validated the Marketing Authorization Application for PF-07307405, the companies' experimental Lyme disease vaccine candidate, and will now begin the official review.
For a vaccine candidate first proposed in a 2020 collaboration deal between Pfizer Inc. (NYSE:PFE) and French biotech Valneva SE (NASDAQ:VALN), this represents the formal transition from years of clinical development into European regulatory review.
The application is based on the Phase 3 VALOR trial, a placebo-controlled, randomized study of 9,437 participants aged five and older from high-incidence Lyme disease locations in US, Canada, and Europe. Participants got four doses on a phased schedule: one at months 0, 2, and 5-9, followed by a fourth dosage around a year later, shortly before the start of the next Lyme season. Topline data released in March 2026 demonstrated efficacy of over 70% in reducing confirmed Lyme disease cases, with the vaccine generally tolerated and no safety issues detected. However, the statistical picture was less clean than the headline efficacy suggests. The first prespecified **** ysis, which served as the primary endpoint, showed 73.2% efficacy but failed the trial's predefined statistical criterion because the lower bound of the 95% confidence interval was 15.8%, below the required 20%. A second prespecified **** ysis showed 74.8% efficacy and did meet that threshold.
Valneva SE (NASDAQ:VALN), the smallest of the two partners, views the vaccine as a clean competitive setup. According to the company, PF-07307405 is the most advanced Lyme disease vaccine candidate currently in clinical development, a rare spot in the vaccine industry where new entrants often face competition from existing treatments. Under the terms of the 2020 partnership and license agreement, Pfizer Inc. (NYSE:PFE) will have exclusive rights to produce and commercialize the vaccine if it is approved, while Valneva SE (NASDAQ:VALN) will benefit as a development partner.
The institutional stance is very different between the two companies, reflecting their very different sizes. Pfizer Inc. (NYSE:PFE) is still a strong name in the institutional **** e, with hedge fund ownership stable at 83 funds in the second quarter of 2026, the same as the first quarter, reflecting its position as a large, diversified pharmaceutical holding, rather than a name driven by a specific pipeline event. Valneva SE (NASDAQ:VALN), however, has little hedge fund exposure, with only 4 funds holding a position in the second quarter, up from 1 in the first quarter, reflecting the continued lack of visibility of the smaller partner among institutional investors.

#vaccine #pfizer #NYSE
rsikvi
5 days ago
Pfizer Inc. (NYSE:PFE) and Valneva SE (NASDAQ:VALN) reported on August 14 that the European Medicines Agency has validated the Marketing Authorization Application for PF-07307405, the companies' experimental Lyme disease vaccine candidate, and will now begin the official review.
For a vaccine candidate first proposed in a 2020 collaboration deal between Pfizer Inc. (NYSE:PFE) and French biotech Valneva SE (NASDAQ:VALN), this represents the formal transition from years of clinical development into European regulatory review.
The application is based on the Phase 3 VALOR trial, a placebo-controlled, randomized study of 9,437 participants aged five and older from high-incidence Lyme disease locations in US, Canada, and Europe. Participants got four doses on a phased schedule: one at months 0, 2, and 5-9, followed by a fourth dosage around a year later, shortly before the start of the next Lyme season. Topline data released in March 2026 demonstrated efficacy of over 70% in reducing confirmed Lyme disease cases, with the vaccine generally tolerated and no safety issues detected. However, the statistical picture was less clean than the headline efficacy suggests. The first prespecified **** ysis, which served as the primary endpoint, showed 73.2% efficacy but failed the trial's predefined statistical criterion because the lower bound of the 95% confidence interval was 15.8%, below the required 20%. A second prespecified **** ysis showed 74.8% efficacy and did meet that threshold.
Valneva SE (NASDAQ:VALN), the smallest of the two partners, views the vaccine as a clean competitive setup. According to the company, PF-07307405 is the most advanced Lyme disease vaccine candidate currently in clinical development, a rare spot in the vaccine industry where new entrants often face competition from existing treatments. Under the terms of the 2020 partnership and license agreement, Pfizer Inc. (NYSE:PFE) will have exclusive rights to produce and commercialize the vaccine if it is approved, while Valneva SE (NASDAQ:VALN) will benefit as a development partner.
The institutional stance is very different between the two companies, reflecting their very different sizes. Pfizer Inc. (NYSE:PFE) is still a strong name in the institutional **** e, with hedge fund ownership stable at 83 funds in the second quarter of 2026, the same as the first quarter, reflecting its position as a large, diversified pharmaceutical holding, rather than a name driven by a specific pipeline event. Valneva SE (NASDAQ:VALN), however, has little hedge fund exposure, with only 4 funds holding a position in the second quarter, up from 1 in the first quarter, reflecting the continued lack of visibility of the smaller partner among institutional investors.

#disease
vcTlD
10 days ago
Royalty Pharma plc (NASDAQ:RPRX) disclosed that pelacarsen failed the Phase 3 Lp(a)HORIZON cardiovascular-outcomes trial conducted by Novartis AG (NYSE:NVS). The randomized, double-blind study enrolled 8,323 patients with elevated lipoprotein(a), or Lp(a), and established cardiovascular disease.
Pelacarsen lowered Lp(a), but the study did not meet its primary endpoint of reducing cardiovascular events compared with placebo in the overall population. The endpoint combined cardiovascular death, nonfatal heart attack, nonfatal stroke, and urgent coronary revascularization requiring hospitalization. Complete results have not yet been presented.
Royalty Pharma plc (NASDAQ:RPRX) provided Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) with $500 million in January 2023. The transaction allocated $150 million to pelacarsen royalties and $350 million to Spinraza royalties. The clinical failure shifts the financial focus from uncertain pelacarsen upside to recovery through Spinraza.
Royalty Pharma plc (NASDAQ:RPRX) acquired 25% of the Spinraza royalties received by Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) through 2027. That share increases to 45% in 2028 on Spinraza annual sales of up to $1.5 billion.
Following the HORIZON result, the Spinraza interest will revert after aggregate payments to Royalty Pharma plc (NASDAQ:RPRX) reach $550 million, equal to 1.1 times the original funding. Management expects the structure to recover the entire investment and generate a modest positive return despite the clinical failure.

#royalty #pharma #cardiovascular #horizon
km92jgeynpape6
18 days ago
On August 26, Biohaven (NYSE:BHVN) and SK Biopharmaceuticals announced a global licensing agreement covering opakalim, Biohaven's lead epilepsy candidate, in a deal worth up to $795 million plus royalties. SK Biopharmaceuticals picks up exclusive worldwide rights to Biohaven's Kv7 ion channel platform, while Biohaven walks away with $400 million in near-term cash. The timing lines up with an August 10, 2026 earnings report that showed Biohaven still losing well over $100 million a quarter, and the gap between those two dates explains a lot about why this deal happened now.
SK Biopharmaceuticals will pay up to $795 million in upfront and milestone payments connected to the Kv7 platform, on top of tiered royalties on US net sales of opakalim that range from the mid-teens to low twenties. Biohaven collects $350 million at closing and another $50 million in 2027, with as much as $150 million more available through development and regulatory milestones plus royalties on global sales. SK Biopharmaceuticals is also taking over Kv7 program costs going forward, including certain Knopp Biosciences obligations. CEO Vlad Coric described the structure as proof Biohaven can monetize its pipeline through partnerships rather than leaning on public markets for cash.
The deal only makes sense because opakalim looks like it works. In a proof-of-concept study in idiopathic generalized epilepsy, the median time to a second generalized tonic-clonic seizure stretched to 141 days on opakalim versus 47 days on placebo, and a third of patients made it through the full 24 weeks without a second seizure. In focal epilepsy, 54% of patients in an open-label extension study saw at least a 50% drop in seizure frequency over any six months, in a group of more than 100 patients.
Opakalim is also designed as a once-daily pill with no ******* ration required, a real edge over older antiseizure drugs. Pairing that data with SK Biopharmaceuticals, the company behind XCOPRI and the only firm to bring a new focal-seizure drug to the US market since 2016, gives Opakalim a commercial path Biohaven would have struggled to build alone. The cash also buys runway for the rest of the pipeline, including protein degraders BHV-1300 and BHV-1400, which have shown rapid, selective reductions in disease-driving antibodies in Graves' disease and IgA nephropathy with clean safety data across nearly 200 patients dosed.
Biohaven is giving up full ownership of an ******* et it now believes could be a major seller. Royalties in the mid-teens to low twenties are real money, but they are a fraction of what outright ownership of an approved epilepsy drug would be worth, and $50 million of the $400 million upfront does not arrive until 2027. The Knopp Biosciences obligations SK Biopharmaceuticals is absorbing, worth up to $245 million plus mid-single-digit royalties, are a reminder that other parties already have claims on opakalim's future revenue before Biohaven sees a dollar of profit from it.

#ro
madlyna
24 days ago
The FDA approved Pasatru, whose generic name is garetosmab, of Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) for adults with fibrodysplasia ossificans progressiva. FOP is an ultra-rare genetic disorder in which bone forms inside muscles, tendons, and ligaments, progressively restricting movement. In a 56-week Phase 3 trial involving 63 adults, the approved 3-milligram-per-kilogram dose reduced the number of new abnormal bone lesions by 94% compared with placebo. The decision completed an unusual clinical recovery. Regeneron paused an earlier study in 2020 after five patients died during its open-label portion, when all participants were receiving active treatment. The published ****** ysis later considered a relationship to treatment unlikely, although causality could not be ruled out. For Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN), Pasatru is a regulatory and scientific victory whose earnings potential is constrained by an exceptionally small market.
The company's existing scale makes that limitation difficult to ignore. Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) generated second-quarter revenue of $4.29 billion, up 17%, while company-defined non-GAAP diluted EPS reached $14.29. Global Dupixent sales, recorded by partner Sanofi, increased 38% to approximately $6 billion, and U.S. Eylea HD sales rose 52% to $596 million. Against those franchises, even a successful Pasatru launch would have limited influence on consolidated results.
The approval demonstrates that Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) can recover a program after a major clinical setback. Following the 2020 dosing pause, the company worked with regulators to design a late-stage study that produced a statistically significant reduction in new bone lesions. Pasatru also validates research into Activin A, which activates the mutant ACVR1/ALK2 receptor and triggers abnormal bone formation in FOP.
That platform value may matter more than initial sales. Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) plans to begin a pediatric trial later in 2026, which could eventually expand the eligible population. The regulatory recovery may also strengthen confidence in the company's ability to advance other rare-disease programs from internally discovered biology.
Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) is entering an exceptionally small pharmaceutical market. Published prevalence estimates generally range from approximately one case per one million to one per two million people. Only approximately 800 to 900 known or confirmed cases have been identified globally, although additional patients may remain undiagnosed. Pasatru is currently approved only for adults, further narrowing its immediate market.

#regeneron #pasatru #adults #market
rbufso407
26 days ago
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) was up 7.5% in after-hours trading as of approximately 5:33 p.m. ET Wednesday after the FDA approved Genglycos for glycogen storage disease type Ia, or GSDIa. The one-time gene therapy is the first approved treatment designed to address the disorder's underlying cause. It will carry a U.S. list price of approximately $2.7 million per patient and is expected to become available through qualified treatment centers within 30 to 60 days.
The approval gives Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) first-mover status in a disease the company estimates affects only 1,500 to 2,500 Americans. The commercial question is whether a high-priced treatment for an ultra-rare population can produce meaningful revenue after diagnosis, reimbursement, and treatment-center constraints.
GSDIa prevents the liver from releasing glucose properly, leaving patients dependent on frequent raw-cornstarch doses to avoid potentially life-threatening hypoglycemia. In the Phase 3 GlucoGene trial, Genglycos reduced mean daily cornstarch intake by 41% at Week 48, compared with 10% for placebo, while maintaining glucose control. For Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), reducing that daily burden creates a clear argument for premium pricing.
Longer-term data also support the treatment's potential durability. At Week 96, the original-treatment group reported a 61% mean reduction from baseline, while the crossover group reported a 61% reduction from Week 48, when it began treatment. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) will manufacture the therapy at its Bedford, Massachusetts, facility, giving the company direct control over an important part of the supply chain.
The launch will also test infrastructure that could support future gene therapies. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) already sells rare-disease products including Crysvita, Dojolvi, Evkeeza and Mepsevii. Second-quarter revenue reached $214 million, while cash, cash equivalents, and marketable securities totaled $436 million as of June 30. Genglycos therefore enters a functioning commercial organization rather than a first-time launch platform.

#rare #pharmaceutical
mix_0157
27 days ago
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) was up 7.5% in after-hours trading as of approximately 5:33 p.m. ET Wednesday after the FDA approved Genglycos for glycogen storage disease type Ia, or GSDIa. The one-time gene therapy is the first approved treatment designed to address the disorder's underlying cause. It will carry a U.S. list price of approximately $2.7 million per patient and is expected to become available through qualified treatment centers within 30 to 60 days.
The approval gives Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) first-mover status in a disease the company estimates affects only 1,500 to 2,500 Americans. The commercial question is whether a high-priced treatment for an ultra-rare population can produce meaningful revenue after diagnosis, reimbursement, and treatment-center constraints.
GSDIa prevents the liver from releasing glucose properly, leaving patients dependent on frequent raw-cornstarch doses to avoid potentially life-threatening hypoglycemia. In the Phase 3 GlucoGene trial, Genglycos reduced mean daily cornstarch intake by 41% at Week 48, compared with 10% for placebo, while maintaining glucose control. For Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), reducing that daily burden creates a clear argument for premium pricing.
Longer-term data also support the treatment's potential durability. At Week 96, the original-treatment group reported a 61% mean reduction from baseline, while the crossover group reported a 61% reduction from Week 48, when it began treatment. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) will manufacture the therapy at its Bedford, Massachusetts, facility, giving the company direct control over an important part of the supply chain.
The launch will also test infrastructure that could support future gene therapies. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) already sells rare-disease products including Crysvita, Dojolvi, Evkeeza and Mepsevii. Second-quarter revenue reached $214 million, while cash, cash equivalents, and marketable securities totaled $436 million as of June 30. Genglycos therefore enters a functioning commercial organization rather than a first-time launch platform.

#treatment
xojuputo
30 days ago
Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) has released topline results from the Phase 3 LUCIDITY trial of avexitide on August 18, 2026. Shares closed Monday down 0.9% at $21.43, then traded 15.7% higher at $24.80 after hours after the company scheduled the readout. The rally came before investors saw any efficacy or safety data, underscoring how much hope is attached to one event. For Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX), the question is whether a successful metabolic-disease trial can finally replace the failed ALS narrative that has defined the company since Relyvrio was withdrawn.
LUCIDITY randomized 78 adults with post-bariatric hypoglycemia following Roux-en-Y gastric bypass surgery in a 3:2 ratio to receive once-daily avexitide or placebo. The FDA-agreed primary outcome measures the reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Avexitide is designed to block the exaggerated GLP-1 response that can drive excess insulin secretion and dangerous drops in blood glucose.
The trial is therefore more than another pipeline update. Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) currently has no approved product, and avexitide is its most advanced near-term opportunity to rebuild a commercial business. A positive result would validate the company's move into endocrine disease. A miss would return attention to earlier-stage programs that cannot replace a late-stage **** et quickly.
Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) enters the readout with supportive early evidence. Amylyx said five earlier Phase 1 and Phase 2 studies produced consistent signals. In a 28-day, open-label Phase 2b crossover study involving 16 patients who had undergone Roux-en-Y gastric bypass or other upper gastrointestinal surgeries, the 90 mg once-daily dose now used in LUCIDITY reduced Level 2 events by 53% and Level 3 events by 66% against the medical nutrition therapy-only run-in baseline. Avexitide was generally well tolerated across prior studies.
The FDA has granted avexitide Breakthrough Therapy designation for post-bariatric hypoglycemia, and management anticipates a potential 2027 launch if the drug is approved. Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) held $250.8 million in cash, cash equivalents, and short-term investments as of June 30. Based on its current operating plans, Amylyx expects that amount to provide a cash runway into 2028, supporting a potential regulatory filing and commercial preparations.

#pharmaceuticals #NASDAQ
5kj4sk2
1 month ago
On August 6, Corvus Pharmaceuticals (NASDAQ:CRVS) held its second-quarter 2026 earnings call, and the story was almost entirely about one molecule. Soquelitinib, the company's oral ITK inhibitor, is now being tested across five different diseases at once, from a rare blood cancer to asthma. That kind of breadth from a single-asset biotech is either a sign of real conviction in the science or a company spreading itself thin before it has proven anything. The quarter's numbers and trial updates offer evidence for both readings.
The clearest bright spot is atopic dermatitis. In the highest-dose cohort of a Phase 1 trial, presented at the Society for Investigative Dermatology meeting, 75% of soquelitinib patients hit EASI-75 after 8 weeks of 200 milligram twice-daily dosing, versus 20% of patients on placebo. A quarter of treated patients reached EASI-90, and a third achieved clear or almost-clear skin, outcomes no placebo patient matched. Management also pointed to something more unusual: the benefit persisted after dosing stopped, without the rebound seen with other drugs in the class.
Corvus is carrying that data into a roughly 200-patient Phase 2 trial, called SEERA-1, with enrollment set to finish in early 2027 and results due the following quarter. The company's finances give it room to run. It ended the quarter with $215 million in cash and marketable securities, up from $56.8 million at the end of 2025, largely thanks to $189 million raised in a Q1 follow-on offering, enough to fund operations into the second quarter of 2028.
That cash cushion exists because Corvus is spending a lot more than it used to. R&D costs jumped to $16 million in the quarter from $7.9 million a year earlier, and the net loss more than doubled to $18 million from $8 million, driven by higher trial costs and added headcount tied to soquelitinib. The company remains a one-drug story in practice, and its most advanced program, a Phase 3 trial in relapsed or refractory peripheral T-cell lymphoma, will not offer a public readout soon.
An independent committee is expected to run a futility ***** ysis around the first quarter of 2027, with no data released publicly at that time. Much of the atopic dermatitis story outside the US also runs through Angel Pharmaceuticals, a China-based partner in which Corvus just put another $5 million as part of a $13.5 million financing round. That trial's first cohort of 24 patients is not expected to finish enrolling until September, meaning part of the bull case still sits ahead of the company.

#company
dsca_mumvw
1 month ago
On August 6, Corvus Pharmaceuticals (NASDAQ:CRVS) held its second-quarter 2026 earnings call, and the story was almost entirely about one molecule. Soquelitinib, the company's oral ITK inhibitor, is now being tested across five different diseases at once, from a rare blood cancer to asthma. That kind of breadth from a single-asset biotech is either a sign of real conviction in the science or a company spreading itself thin before it has proven anything. The quarter's numbers and trial updates offer evidence for both readings.
The clearest bright spot is atopic dermatitis. In the highest-dose cohort of a Phase 1 trial, presented at the Society for Investigative Dermatology meeting, 75% of soquelitinib patients hit EASI-75 after 8 weeks of 200 milligram twice-daily dosing, versus 20% of patients on placebo. A quarter of treated patients reached EASI-90, and a third achieved clear or almost-clear skin, outcomes no placebo patient matched. Management also pointed to something more unusual: the benefit persisted after dosing stopped, without the rebound seen with other drugs in the class.
Corvus is carrying that data into a roughly 200-patient Phase 2 trial, called SEERA-1, with enrollment set to finish in early 2027 and results due the following quarter. The company's finances give it room to run. It ended the quarter with $215 million in cash and marketable securities, up from $56.8 million at the end of 2025, largely thanks to $189 million raised in a Q1 follow-on offering, enough to fund operations into the second quarter of 2028.
That cash cushion exists because Corvus is spending a lot more than it used to. R&D costs jumped to $16 million in the quarter from $7.9 million a year earlier, and the net loss more than doubled to $18 million from $8 million, driven by higher trial costs and added headcount tied to soquelitinib. The company remains a one-drug story in practice, and its most advanced program, a Phase 3 trial in relapsed or refractory peripheral T-cell lymphoma, will not offer a public readout soon.
An independent committee is expected to run a futility ******* ysis around the first quarter of 2027, with no data released publicly at that time. Much of the atopic dermatitis story outside the US also runs through Angel Pharmaceuticals, a China-based partner in which Corvus just put another $5 million as part of a $13.5 million financing round. That trial's first cohort of 24 patients is not expected to finish enrolling until September, meaning part of the bull case still sits ahead of the company.

#corvus #company #pharmaceuticals
rfhqhqlmjwh
1 month ago
Eli Lilly and Company (NYSE:LLY) cleared a notable regulatory hurdle on August 10, when Britain's drug regulator authorized the company's oral GLP-1 drug orforglipron, becoming the first country in Europe to approve the pill. Shares of LLY increased more than 2% in response to the announcement, a modest but insightful reaction for a company whose weight-loss franchise has become the single most important driver of its market value.
The UK's Medicines and Healthcare Products Regulatory Agency approved orforglipron, offered under the brand name Foundayo, for weight loss and maintenance in obese adults, as well as select overweight individuals with at least one weight-related health condition. The approval also addresses increasing blood sugar control in people with type 2 diabetes.
What sets Foundayo apart from Lilly's other weight-loss medications, as well as the majority of the GLP-1 category in general, is the format. Unlike injectable drugs like Lilly's Zepbound or Novo Nordisk A/S (NYSE:NVO)'s Ozempic, Foundayo is a small, non-peptide GLP-1 receptor agonist that is taken as a single daily tablet. It can also be taken at any time with no food or water restrictions, making it a far more convenient regimen than some oral peptide alternatives that require fasting windows around dosing.
The clinical data that supports the approval is strong. In the ATTAIN-1 trial, which tracked 3,127 individuals with obesity for 72 weeks, participants on the 36 mg dose lost an average of 11.2% of their body weight, as opposed to 2.1% on placebo, and more than half of those on that dose lost at least 10%.
The authorization is crucial, but it is not the final step. Foundayo is not currently available through the NHS in the UK, and the drug's journey to that larger market is through the National Institute for Health and Care Excellence, which is completing its own cost-effectiveness study, with a decision expected on November 18. Until NICE decides, UK patients seeking the medicine will have to pay out of pocket or get it privately, a scenario that is already playing out with competing products in the category.

#foundayo #loss #NYSE #regulatory
Warm_1
1 month ago
It was reported on July 27 that AstraZeneca PLC (NASDAQ:AZN) shares outperformed in European trading after the company reported second-quarter earnings that beat Wall Street expectations and reiterated its full-year 2026 guidance. Core earnings per share (EPS) jumped 18% on a constant exchange rate (FXN) basis year-over-year to $2.63, comfortably ahead of the $2.48 ***** yst consensus. Total revenue reached $15.38 billion, up 5% at constant exchange rates, driven primarily by sustained momentum in its Oncology and Rare Disease units. Management reconfirmed its full-year 2026 outlook of mid-to-high single-digit revenue growth and low double-digit Core EPS growth, expressing confidence in reaching its $80 billion total revenue target by 2030 despite near-term headwind shocks.
The quarter demonstrated strong commercial execution in core growth engines. Oncology revenue rose 16% to $7.33 billion, supported by strong demand for Tagrisso ($1.94 billion), Imfinzi ($1.85 billion), and Enhertu (+31%). Rare Disease contributed $4.9 billion, led by Ultomiris. These gains successfully offset severe pressures in the Cardiovascular, Renal & Metabolism (CVRM) segment, which declined 15% due to the loss of exclusivity (LOE) for Farxiga in the U.S. and ongoing Volume-Based Procurement (VBP) price cuts in China.
Meanwhile, pipeline updates presented a mixed picture. On July 27, AZN disclosed that a Phase 3 study evaluating Ultomiris in hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) failed to hit its primary endpoint of event-free survival at week 26 versus placebo. Following the readout, H.C. Wainwright noted that the trial miss represents a "clear positive" for competitor Omeros (OMER), removing a major near-term competitive overhang on its drug Yartemlea and driving Omeros shares up 11% in morning trading.
AstraZeneca PLC (NASDAQ:AZN)'s core profitability remains elite, with core operating margins expanding to 34% in Q2 despite top-line headwinds from generic entry. High gross and net margins signal durable pricing power across its branded specialty portfolio. This strong profitability generates predictable cash flow to fund heavy R&D investments, commercial rollouts, and growing shareholder returns, including a 3-cent increase in the interim dividend to $1.06 per share, while buffering the company against pricing pressure.
The company's expansive late-stage pipeline and high volume of regulatory approvals underpin a multi-year growth trajectory. With 30 major regional approvals since late 2025 and more than 20 high-value trial readouts scheduled over the next 18 months, AZN possesses broad commercial optionality. Continued expansions in oncology (e.g., Enhertu and Imfinzi) and respiratory therapies (such as Breztri and Tezspire) provide direct revenue replacement for legacy products facing patent expiration.

#high #july
yanevapo57
1 month ago
AbbVie (NYSE:ABBV) just moved a step closer to expanding one of its most recognizable brands. On August 4, the FDA accepted for review a supplemental Biologics License Application for Botox Cosmetic to treat masseter muscle prominence, the jaw-muscle bulge that can give a face a wider, squarer look. If cleared, Botox Cosmetic would become the first and only neurotoxin approved for that use in the US and the fifth aesthetic indication for a drug that already anchors AbbVie's fastest-growing units.
The filing rests on two Phase 3 studies, M21-416 and M21-417, both of which hit their main goal and showed statistically significant improvement in masseter prominence against a placebo, with p-values of 0.0046 and 0.0014. Twice as many treated patients called themselves satisfied compared with the placebo group, and the safety data lined up with Botox's long track record, with no new red flags. That is the AbbVie playbook in miniature: squeeze another approved use out of a drug it already owns rather than starting from zero.
The broader business backs that pattern up. In the quarter ended June 30, net revenue came in just under $17 billion, up more than 10% year over year, with immunology sales climbing 15% on Skyrizi and Rinvoq and neuroscience revenue jumping more than 20%. Management raised its full-year outlook for the second time this year, and the Humira patent-cliff drag that weighed on results for two years now looks largely behind the company.
Most of AbbVie's US product sales still flow through just three wholesale distributors, McKesson, Cardinal Health, and Cencora, a concentration that leaves the company exposed if any one relationship sours. The balance sheet carries real leverage too: a debt-to-equity ratio of roughly -21.1x as of its December 2025 filing means liabilities outweigh shareholder equity outright, and a current ratio near 0.7x offers less short-term cushion than many healthcare peers hold.
Patent protection for Skyrizi and Rinvoq remains a multi-year risk, and the Inflation Reduction Act already allows government price negotiation on products including Imbruvica and, notably, Botox itself. The pending $10.9 billion Apogee Therapeutics acquisition adds fresh debt and integration risk on top of that. Oncology revenue slipped almost 2% in the latest quarter, and the stock's 8% gain this year has trailed the S&P 500's 13%, with a trailing P/E near 70 that mostly reflects acquisition-related charges rather than the underlying business.

#revenue
bluntly_hawk_lynx_72
2 months ago
Baron Capital, an investment management company, released its Q2 2026 investor letter for the "Baron Health Care Fund". A copy of the letter is available to download here. The Fund gained 11.99% during the quarter, compared with the 10.48% gain for the Russell 3000 Health Care Index and the 15.44% gain for the Russell 3000 Index. Since inception, the Fund appreciated 10.61% on an annualized basis, compared with 10.02% for the Benchmark and 14.83% for the Index. Strong stock selection in pharmaceuticals, biotechnology, health care equipment, and life sciences tools and services supported the Fund's outperformance, although limited exposure to managed care stocks reduced relative returns. The Fund remains positive on health care due to improving biotechnology funding, strong acquisition activity, recovering managed care margins, and growth from an aging population, chronic disease, medical innovation, and higher health care spending. In addition, please check the Fund's top five holdings to know the best picks in 2026.
In its second-quarter 2026 investor letter, Baron Health Care Fund highlighted Veradermics, Incorporated (NYSE:MANE). Veradermics, Incorporated (NYSE:MANE), a clinical-stage biopharmaceutical company developing treatments for pattern hair loss in adults and children, contributed to the Fund's performance during the quarter. On August 03, 2026, Veradermics, Incorporated (NYSE:MANE) closed at $101.89 per share. The one-month return of Veradermics, Incorporated (NYSE:MANE) was -13.05%. Veradermics, Incorporated (NYSE:MANE) has a market capitalization of $4.26 billion.
Baron Health Care Fund stated the following regarding Veradermics, Incorporated (NYSE:MANE) in its Q2 2026 investor letter:
"Veradermics, Incorporated (NYSE:MANE), a dermatologist founded, late clinical-stage biopharmaceutical company developing VDPHL01, an extended-release oral minoxidil tablet for pattern hair loss. In April, the company reported solid Phase 3 data in male pattern hair loss. The drug demonstrated robust hair growth with up to 63% of male patients reporting improved hair coverage compared to just 13% on placebo. We await data from a second Phase 3 study of VDPHL01 in male pattern hair loss and the first clinical data from a Phase 2 study of VDPHL01 in female pattern hair loss patients, both expected in the second half of 2026. If VDPHL01 is U.S. Food and Drug Administration (FDA) approved, we think it will be uniquely positioned as the first FDA-approved oral treatment in several decades for a condition affecting 80 million Americans."

#incorporated #vdphl01
socketwhirl
2 months ago
The biopharma sector is in the middle of a transformation in which macroeconomic pressures and policy developments actively dictate corporate dealmaking. As leading pharmaceutical companies prepare for a $300 billion patent cliff in the second half of the decade, capital allocation has switched sharply toward restocking commercial pipelines with low-risk, high-conviction clinical ******* ets. At the same time, implementation of the Inflation Reduction Act has changed the relative attractiveness of drug options. Since small-molecule drugs are subject to government price negotiations earlier in their lifecycle compared to large-molecule biologics, commercial buyers are shifting premium valuations to large-molecule immunology platforms.
Against this macro context, argenx SE (NASDAQ:ARGX) entered a definitive all-cash agreement on July 27 to acquire clinical-stage biotech Forte Biosciences, Inc. (NASDAQ:FBRX) for $77 per share, valuing the company at over $2.2 billion. At first glance, the $77 offer represents a 40% premium to Forte's closing price prior to the announcement. However, the underlying deal economics demonstrate argenx's readiness to pay a premium for clinical validation: when compared to Forte's volume-weighted average pricing following its successful Phase 1b vitiligo readout on July 9, the purchase price represents an 86% premium.
The target ******* et, Forte's FB102, is a first-in-class anti-CD122 antibody that has shown clinical efficacy in vitiligo and celiac disease. In a 24-week experiment, FB102 showed a significant 29.6% mean improvement on the Facial Vitiligo Area Scoring Index, separating from placebo by day 64.
Argenx's ability to carry out a $2.2 billion cash deal stems from its strong financial condition. The company's Q1 2026 financial report showed global product net sales of $1.3 billion, a 63% increase year-over-year, as well as a quarterly net profit of $366 million and a strong balance sheet with $4.9 billion in cash and financial ******* ets. This commercial momentum is almost exclusively driven by Vyvgart, the company's blockbuster FcRn-blocking medication, which is now seeing its 17th straight quarter of growth.
That said, the explosive top-line trajectory poses a major concentration risk. According to CEO Karen Massey's "Vision 2030" framework, which targets 50,000 patients and ten designated indications worldwide, argenx SE (NASDAQ:ARGX) needs a supplementary second leg in immunology. FB102 offers that diversification by delivering a unique CD122 mechanism into unserved autoimmune indications without jeopardizing its core FcRn business.

#premium #price
94calm
2 months ago
Concerned about an AI bubble? Sign up for The Daily Upside for smart and actionable market news, built for investors.
It was just another manic market day, at least for AstraZeneca.
Shares of the British-Swedish biopharmaceutical company tumbled Thursday after it shared the surprising failure of its drug Wainua to meet its target in a recent clinical trial. AstraZeneca and Ionis Pharmaceuticals said the heart disease treatment didn't outperform the placebo in preventing heart problems in patients with transthyretin-mediated amyloid cardiomyopathy (or ATTR-CM), a rare and potentially fatal disease.
It was a blow ***** ysts weren't expecting, and AstraZeneca's shares fell as much as 10.6% in London, its largest intraday plummet since 2017, per Bloomberg. The company's New York Stock Exchange-listed shares ended Thursday down 5.7%.
Sign up for The Daily Upside at no cost for premium ***** ysis on all your favorite stocks.
zunufa_g_ni_jewozo
2 months ago
Biohaven Ltd. (NYSE:BHVN) is one of the 10 Best Performing American Stocks in June 2026.
On June 30, 2026, Biohaven Ltd. (NYSE:BHVN) announced the completion of enrollment in RISE3, a pivotal Phase 2/3 randomized, double-blind, placebo-controlled study evaluating opakalim for refractory focal epilepsy. Opakalim is Biohaven's selective Kv7.2/7.3 channel activator, and top-line results from the study are expected in 2H 2026.
On June 29, Biohaven announced enrollment of the first patient in the pivotal Phase 3 trial of BHV-1300 for Graves' disease. BHV-1300 is the first MoDE extracellular protein degrader, described by the company as a novel small molecule IgG1, 2, and 4 degrader that uses the body's clearance machinery to eliminate the IgG1 TSHR autoantibody driving Graves' disease.
On June 26, Deutsche Bank raised the firm's price target on Biohaven to $20 from $15 and kept a Buy rating on the shares. Deutsche Bank said the company's troriluzole in spinocerebellar ataxia could be the next beneficiary of recent FDA reversals.
Miriam Doerr Martin Frommherz/Shutterstock.com
vr3oa
3 months ago
Vaxcyte Inc. (NASDAQ:PCVX) is one of the 8 Hidden Multibagger Stocks to Buy Now. On June 18, Mizuho Securities ******* yst Salim Syed reiterated a Buy rating on Vaxcyte Inc. (NASDAQ:PCVX) with a price target of $163. The firm's price target reflects a significant 180% upside from current levels.
Earlier on June 16, PCVX announced an update regarding its ongoing clinical study. The company is conducting an early-stage clinical trial of VAX-A1, a vaccine designed to protect against Group A strep infections. The study will evaluate the safety, tolerability, and immunogenicity of VAX-A1 in healthy young adults. The trial is testing three different dose levels of the vaccine against a placebo. It is administered via muscle injection and is designed to support the immune system.
If the trial shows positive results, the vaccine could allow the company to enter a new market for preventing Group A strep infections. This could boost investor confidence in the company and support the stock's valuation. However, disappointing results could increase concerns about the program and shift attention toward larger vaccine competitors such as Pfizer and GSK.
Vaxcyte Inc. (NASDAQ:PCVX) is a clinical-stage vaccine innovation company that develops conjugate and novel protein vaccines to prevent or treat bacterial infectious diseases. The company develops a variety of vaccines, and its major candidate is VAX-24. The company was formerly known as SutroVax, Inc. and changed its name to Vaxcyte, Inc. in May 2020. It was incorporated in 2013 and is headquartered in San Carlos, California.
While we acknowledge the potential of PCVX as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock.
prism
3 months ago
We recently compiled a list of the 10 Best Weight Loss Drug Stocks to Buy in 2026. Merck & Co., Inc. (NYSE:MRK) is one of the best weight loss drugs on this list.
TheFly reported on June 18 that MRK announced that the U.S. Food and Drug Administration (FDA) granted an expanded approval for Capvaxive, extending its use to children and adolescents between 2 and 17 years old. The updated indication applies to pediatric patients who have completed their initial pneumococcal vaccination series and have chronic health conditions that increase their vulnerability to pneumococcal disease. With this authorization, Capvaxive becomes the only pneumococcal conjugate vaccine (PCV) in the United States specifically evaluated and approved for this patient group.
In another major operational breakthrough, on June 12, Merck & Co., Inc. (NYSE:MRK) announced that the U.S. Food and Drug Administration (FDA) approved KEYTRUDA and KEYTRUDA QLEX, its anti-PD-1 therapies, in combination with WELIREG for the adjuvant treatment of adults with clear cell renal cell carcinoma (ccRCC) at an increased risk of recurrence after surgery. The approval marks the first use of WELIREG in earlier-stage ccRCC and the first authorization of a PD-1 and HIF-2α inhibitor combination therapy. The decision was supported by results from the Phase 3 LITESPARK-022 trial, which showed the combination reduced the risk of recurrence, metastasis, or death by 28% compared with KEYTRUDA plus placebo in eligible patients.
Merck & Co., Inc. (NYSE:MRK) is a global healthcare company expanding its obesity pipeline through early-stage GLP-1 and dual agonist therapies. While it lacks a marketed weight-loss drug, its metabolic research and strong pharmaceutical portfolio make it a potential long-term obesity market contender.
While we acknowledge the potential of MRK as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock.
266prism_packet
3 months ago
We recently compiled a list of the 10 Best Weight Loss Drug Stocks to Buy in 2026. Amgen Inc. (NASDAQ:AMGN) is one of the best weight loss drugs on this list.
TheFly reported on June 16 that Mizuho ****** yst Salim Syed increased the price target for AMGN to $303 from $295 while maintaining a Neutral rating on the shares. The adjustment followed an update to the firm's valuation model after reviewing AMGN's first quarter results.
In separate news, on June 7, Amgen Inc. (NASDAQ:AMGN) presented new clinical findings at the American Diabetes ****** ociation (ADA) 86th Scientific Sessions, highlighting its efforts to address cardiovascular risks among people with cardiometabolic conditions. The company shared Phase 3 VESALIUS-CV subgroup data evaluating Repatha in 6,002 high-risk diabetes patients with elevated LDL-C levels who had not previously experienced a heart attack or stroke.
The ****** ysis showed that Repatha, used alongside statins or other LDL-C-lowering treatments, lowered the likelihood of major cardiovascular events, including coronary heart disease death, heart attack, or ischemic stroke, by 29% compared with placebo. The treatment also demonstrated a 21% reduction in a broader cardiovascular outcome that included ischemia-related revascularization. Patients receiving Repatha achieved a median LDL-C level of 45 mg/dL, compared with 106 mg/dL among those receiving placebo.
Amgen Inc. (NASDAQ:AMGN) is a global biotech company gaining investor attention in the weight-loss market through MariTide, an experimental obesity drug in Phase 3 trials. With potential monthly dosing and strong weight-loss results, MariTide could become a major competitor in the growing obesity treatment ****** e.
H4RdCEfuCcxJ
3 months ago
Vaxcyte, Inc. (NASDAQ:PCVX) is one of the most promising healthcare stocks according to Wall Street ****** ysts. On June 2, Vaxcyte, Inc. (NASDAQ:PCVX) dosed the first participant in its Phase 1 first-in-human clinical trial of VAX-A1. VAX-A1 is the company's experimental vaccine against Group A Streptococcus, or Group A Strep.
Pressmaster/Shutterstock.com
The dosing marks Vaxcyte's entry of what would be the first-in-human vaccine candidate for Group A Strep, a disease for which no approved vaccine currently exists. For context, Vaxcyte estimates that Group A Strep is responsible for 800 million illnesses globally every year, and the illnesses range from common strep throat to severe, life-threatening infections and long-term complications like rheumatic heart disease. Despite this burden, no approved vaccine exists anywhere in the world, noted Vaxcyte.
For the trial, Vaxcyte will enroll 80 healthy adults aged 18 to 40 in Australia. The company said the choice of Australia is because the country has high rates of Group A Strep disease. It also has established research networks with deep expertise in studying it. Vaxcyte will structure the study in two stages, where Stage 1 will include 12 participants who will serve as a safety checkpoint before an independent safety board reviews results and approves the expansion to Stage 2.
All participants will receive two doses of VAX-A1 or a placebo, which will be ****** ed about two months apart. They will then be monitored for six months after the final dose. The trial will test low, mid, and high dose levels to find the right balance between immune response and tolerability.
4rjUf
3 months ago
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) is one of the 8 Best Genomics Stocks to Buy According to ***** ysts.
On May 28, Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) said partner GSK reported pivotal Phase 3 data for bepirovirsen. The corporation disclosed a 19% functional cure rate as compared to 0% on standard care, while reaching 26% in patients with lower viral activity. The trials, B-Well 1 and B-Well 2, met primary endpoints. Ionis stated that 233 of 1,220 treated patients achieved a functional cure versus none in the placebo group.
The company also reported 49% of treated patients reached surface antigen levels ≤100 IU/mL one year post-treatment, while 23% achieved sustained viral suppression at week 72.
Ionis mentioned adverse events included injection site reactions and temporary liver enzyme hikes.
Chief Executive Officer Brett P. Monia called the data "an important advance," stating bepirovirsen could address chronic hepatitis B by reducing viral replication and suppressing surface antigen as well as stimulating an immune response.
D7mN5YFOs8M
3 months ago
Amgen Inc. (NASDAQ:AMGN) is one of the
7 Best Longevity Stocks to Buy Now.
On June 7, 2026, Amgen Inc. (NASDAQ:AMGN) announced new data at the American Diabetes ****** ociation 86th Scientific Sessions, including Phase 3 VESALIUS-CV subgroup results for Repatha in patients with high-risk diabetes and elevated LDL-C without prior heart attack or stroke. In an ****** ysis of 6,002 patients, Repatha, when added to statins or other LDL-C-lowering therapies, reduced the risk of the composite primary endpoint of coronary heart disease death, myocardial infarction, or ischemic stroke by 29% compared with placebo. Repatha also reduced the risk of a second composite primary endpoint that included ischemia-driven revascularization by 21%.
On June 5, 2026, Goldman Sachs lowered the firm’s price target on Amgen Inc. (NASDAQ:AMGN) to $389 from $425 and maintained a Buy rating on the shares. Goldman Sachs adjusted its model after Amgen disclosed in its Q1 earnings that the IRS audit for 2016 to 2018 had escalated into a formal dispute, with the company receiving a notice of proposed adjustment.
Earlier in June, Amgen announced that the European Commission granted marketing authorization for Imdylltra as a monotherapy for adults with extensive-stage small cell lung cancer who require systemic therapy after disease progression on or after first-line platinum-based chemotherapy.
prism
4 months ago
AstraZeneca PLC (NYSE:AZN) is one of the best stocks to buy now for long term growth. AstraZeneca PLC (NYSE:AZN) reported on May 29 that the global CARES Phase III clinical programme showed that treatment with anselamimab led to “nominally statistically significant and highly clinically meaningful benefit in adults with advanced kappa light chain (AL) amyloidosis as first-line therapy added to standard of care plasma cell dyscrasia (PCD) treatments, compared to placebo”. Anselamimab is a potential first-in-class anti-fibril therapy. The company added that in the overall population of patients with AL amyloidosis, treatment with anselamimab did not meet the primary endpoint, defined as “a hierarchical combination of time to all-cause mortality (ACM) and frequency of cardiovascular hospitalisations (CVH), as previously disclosed”.
In a separate development, AstraZeneca PLC (NYSE:AZN) reported on May 28 that its Imfinzi (durvalumab), in combination with Bacillus Calmette-Guérin (BCG) induction and maintenance therapy, received approval by the FDA in the U.S. to treat adult patients with BCG-naïve, high-risk non-muscle-invasive bladder cancer (NMIBC). Management stated that the FDA’s approval is based on positive results from the POTOMAC Phase III trial.
AstraZeneca PLC (NYSE:AZN) is a biopharmaceutical company that explores, develops, manufactures, and commercializes prescription medicines. It supplies its products and services to specialty and primary care physicians, and is involved in exploring novel immuno-oncology treatment approaches. AstraZeneca PLC (NYSE:AZN) distributes its products and services through local representative offices and distributors.
While we acknowledge the potential of AZN as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock.
READ NEXT: 15 Stocks That Will Make You Rich in 10 Years AND 12 Best Stocks That Will Always Grow.
kmzwolm_xavyuzu
4 months ago
Our ******* ysts just identified a stock with the potential to be the next Nvidia. Tell us how you invest and we'll show you why it's our #1 pick. Tap here.
Abivax SA shares suffered a spectacular collapse on Tuesday, cratering as much as 40% in Paris and New York.
The clinical-stage drugmaker dropped highly anticipated Phase 3 maintenance data for its lead ulcerative colitis pill, obefazimod. The headline numbers revealed unmatched long-term efficacy, putting the company on a direct flight path to challenge big-pharma incumbents. However, the victory was instantly derailed by the appearance of scattered cancer cases concentrated exclusively in the high-dose cohort, spooking institutional investors and prompting an immediate, high-profile ******* yst downgrade.
The results from the global 44-week ABTECT maintenance study originally looked like a best-case scenario. Evaluating patients with moderately to severely active ulcerative colitis — many of whom were completely refractory, having failed multiple advanced biologic therapies — obefazimod proved to be an absolute clinical powerhouse. Patients on either the 25 mg or 50 mg once-daily oral doses achieved clinical remission rates of 50.8% and 51.3%, respectively. This stands in contrast to a meager 10.4% baseline for the placebo group, securing a placebo-adjusted remission rate of roughly 40%.
The financial wheels fell off, however, when investors opened the safety appendix. In the higher 50 mg treatment arm, investigators recorded individual diagnoses of prostate cancer, breast cancer, and colonic dysplasia — an abnormal cell progression that often acts as a precursor to malignant tumors. The high-dose cluster also flagged four separate non-melanoma skin cancer cases.

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