SOUTH SAN FRANCISCO (dpa-AFX) - The biotech sector delivered a mix of regulatory milestones, strategic deals, corporate restructuring and clinical trial developments across therapeutic areas such as hypothalamic obesity, peanut allergy, neurogenic orthostatic hypotension, and overweight and obesity this week.
Let's dive into the details.
Layoffs
Theravance Biopharma Announces Workforce Reductions After Trial Setback
Theravance Biopharma, Inc. (TBPH) announced that its Phase 3 CYPRESS study of Ampreloxetine in neurogenic orthostatic hypotension did not meet its primary endpoint. Following the setback, the company will wind down the program and reduce operating expenses by about 60%, impacting nearly half its workforce.
Theravance said the restructuring will preserve capital, with expected annualised cash flow of $60-$70 million beginning in Q3 2026, supported by YUPELRI sales and TRELEGY milestones. The board has also accelerated its strategic review to evaluate options for maximizing shareholder value.
TBPH closed Thursday's trading at $13.58, down 0.62%
BioAtla Launches Strategic Review, Cuts Workforce
BioAtla, Inc. (BCAB) announced it has initiated a formal process to evaluate strategic options to monetise its assets following recent clinical setbacks. As part of the restructuring, the company will implement workforce reductions to conserve cash while exploring alternatives, including partnerships, asset sales, and other transactions.
BioAtla said the measures are intended to extend its financial runway and support ongoing evaluation of its antibody-based oncology programs. The board emphasised that the process is focused on maximising shareholder value amid challenging market conditions.
BCAB closed Thursday's trading at $0.16, up 2.55%
FDA Approvals & Rejections
Aardvark Therapeutics Pauses Phase 3 HERO Trial Following Safety Signal
Aardvark Therapeutics, Inc. (AARD) has voluntarily paused its Phase 3 HERO trial evaluating ARD-101 for hyperphagia in Prader-Willi Syndrome (PWS). The company also halted the study's open-label extension after reversible cardiac observations were detected at above-target therapeutic doses during routine safety monitoring in a healthy-volunteer study.
Aardvark has initiated a full review of the ARD-101 program and, out of caution, suspended ongoing enrollment and dosing while the assessment is underway. As a result, the company no longer expects to deliver HERO topline data in Q3 2026. Updated guidance is now planned for Q2 of this year, once the internal review is complete.
AARD has closed Thursday's trading (March 6, 2026) at $5.74, 1.77%.
Ascendis Pharma Gains FDA Accelerated Approval for YUVIWEL
Ascendis Pharma A/S (ASND) receives FDA accelerated approval of YUVIWEL (navepegritide), the first once-weekly treatment to boost linear growth in children aged two and older with achondroplasia. The therapy provides continuous systemic CNP exposure, with continued approval dependent on confirmatory trial results.
The decision was supported by data from multiple randomized, placebo-controlled studies and long-term extension trials. Ascendis expects U.S. availability in the early second quarter of 2026, alongside patent support services through its Ascendis Signature Access Program. The FDA also granted a Rare Pediatric Disease Priority Review Voucher.
ASND closed Thursday's trading at $237.35, down 1.67%.
BioMarin Secures FDA Approval Expanding PALYNZIQ Use to Pediatric PKU Patients
BioMarin Pharmaceutical Inc. (BMRN) announced that the U.S. FDA has approved its supplemental Biologics License Application for PALYNZIQ (pegvaliase-pqpz), expanding the therapy's indication to include pediatric patients aged 12 and older with phenylketonuria (PKU). PALYNZIQ remains the only enzyme substitution therapy approved to lower blood phenylalanine levels in individuals with PKU.
The company is also pursuing a similar label expansion with the European Medicines Agency, aiming to extend access to adolescents as young as 12 across the EU.
BMRN closed Thursday's trading at $60.57, up 0.23%
Agio's PYRUKYND Gains UAE Approval for Adults with Thalassemia
Agios Pharmaceuticals, Inc.(AGIO) announced that the Emirates Drug Establishment (EDE)has approved PYRUKYND(mitapivat), for adults with both non-transfusion-dependent and transfusion-dependent alpha-or beta-thalassemia, making it the only approved treatment for this patient population in the United Arab Emirates.
The approval is based on results from the global Phase 3 ENERGIZE and ENERGIZE-T trials, which demonstrated meaningful improvements in hemoglobin levels and reduction in transfusion burden. Agios said the decision marks a key milestone for patients in a region with a high prevalence of thalassemia.
Commercialization in the Gulf region will continue through NewBridge Pharmaceuticals, which is also supporting launches following prior approval in Saudi Arabia. A marketing application for mitapivat in thalassemia is currently under review in Europe.
AGIO closed Thursday's trading at $27.68, down 1.81%.
Outlook Therapeutics Updates FDA Approval Pathway of LYTENAVA
Outlook Therapeutics Inc. (OTLK) announced it held a Type A meeting with the U.S. FDA following the December 2025 Complete Response Letter for ONS-5010/LYTENAVA (bevacizumab-vikg). The FDA requested additional confirmatory evidence of effectiveness before approval could proceed.
LYTENAVA has already shown statistically significant vision gains in the NORSE TWO Phase 3 trial, supported by NORSE EIGHT, with a favourable safety profile. If approved, it would be the first FDA-approved ophthalmic formulation of bevacizumab for wet AMD, backed by a fully domestic U.S. supply chain.
OTLK closed Thursday's trading at $0.43, down 1.53%
Deals
Esperion To Acquire Corstasis Therapeutics
Esperion Therapeutics, Inc. (ESPR) on Tuesday said it has agreed to acquire Corstasis Therapeutics Inc. for $75 million in upfront cash, with up to an additional $180 million tied to regulatory and commercial milestones, plus low double-digit royalties on sales of Enbumyst and related follow-on products.
The transaction is expected to close in the second quarter of 2026.
The acquisition adds Enbumyst, the FDA-approved nasal spray diuretic for oedema associated with congestive heart failure and hepatic and renal disease in adults, to Esperion's portfolio. In addition, it also brings Corstasis' subcutaneous pipeline in development, including a multidose pen injector with market opportunities.
ESPR closed Thursday's trade at $2.77, down 4.81%.
Select Medical Going Private In $3.9 Bln Deal
Select Medical Holdings Corporation (SEM) announced that it will be acquired by a consortium led by Robert A. Ortenzio, Martin F. Jackson, and Welsh, Carson, Anderson & Stowe (WCAS).
Under the definitive agreement, the consortium will purchase all outstanding shares of Select Medical not already owned by them for $16.50 per share in cash, valuing the company at about $3.9 billion.
SEM closed Thursday's trade at $16.25.
Clinical Trials - Breakthroughs
Rhythm Shares Additional Phase 3 TRANSCEND Trial Results
Rhythm Pharmaceuticals, Inc. (RYTM) announced additional positive results from its global Phase 3 TRANSCEND trial of Setmelanotide in patients with acquired hypothalamic obesity (HO).
According to the firm, at 52 weeks, the study demonstrated an 18.8% placebo-adjusted difference in BMI reduction across all 142 patients, including those from the Japanese cohort and supplemental participants.
In the trial, patients receiving Setmelanotide achieved a mean BMI reduction of 16.4% from baseline, compared with a +2.4% BMI increase in the placebo group.
The therapy was generally well tolerated, with a safety profile consistent with earlier studies.
Rhythm has submitted a supplemental New Drug Application (sNDA) to the U.S. FDA, with a PDUFA goal date of March 20, 2026.
RYTM closed Thursday's trade at $90.46, down 0.51%.
DBV Technologies VIASKIN Peanut Patch Meets Primary Goal In Phase 3 VITESSE Study
DBV Technologies (DBVT) announced that its investigational VIASKIN peanut patch met the primary endpoint in the pivotal Phase 3 VITESSE trial, demonstrating significant desensitisation in children aged 4 to 7 years with peanut allergy.
The VITESSE study was a multicenter, randomized, double-blinded, placebo-controlled trial evaluating daily use of the VIASKIN Peanut Patch over 12 months.
The study demonstrated that 46.6% of children in the treatment group were responders at 12 months compared to 14.8% in the placebo group and about 83% of treated children increased their tolerance threshold, versus 48% in the placebo group.
According to the firm, the therapy was generally well tolerated, with most adverse events limited to mild, localised skin reactions.
The firm plans to submit a Biologics License Application (BLA) to the U.S. FDA in the first half of 2026 based on the results.
DBVT closed Thursday's trade at 3.12%, up $22.08.
Theravance Biopharma's Phase 3 CYPRESS Study of Ampreloxetine Fails in Neurogenic Orthostatic Hypotension Due to MSA
Theravance Biopharma, Inc. (TBPH) announced that the Phase 3 CYPRESS study evaluating Ampreloxetine in patients with symptomatic neurogenic orthostatic hypotension (nOH) due to multiple system atrophy (MSA), a rare disease, did not meet its primary endpoint.
The Phase 3 CYPRESS study evaluated the efficacy and safety of ampreloxetine in patients, and the primary endpoint was the change in OHSA composite score at Week 8 during the double-blind randomised withdrawal period.
In the Phase 3 CYPRESS study, the primary endpoint was not statistically significant. Similar trends were observed in the secondary endpoints at week 8.
As a result, the firm has decided to wind down the ampreloxetine program.
TBPH closed Thursday's trade at $13.58, down 0.62%.
Can-Fite's Namodenoson Achieves Primary Goal in Phase 2a Trial in PDAC
Can-Fite BioPharma Ltd. (CANF) announced that its investigational drug Namodenoson successfully met the primary endpoint in a Phase 2a study for advanced pancreatic ductal adenocarcinoma (PDAC).
The study enrolled 20 patients with advanced PDAC who had received one or more prior lines of therapy.
The trial met its primary endpoint and demonstrated that Namodenoson was very well tolerated, with no new safety signals identified in this difficult-to-treat patient population.
Secondary endpoints included overall survival (OS) and progression-free survival (PFS). Survival follow-up remains ongoing, with 1/3 of patients currently alive at the time of data cut-off.
Namodenoson is a selective A3 adenosine receptor (A3AR) agonist also being evaluated in clinical trials for advanced liver cancer.
CANF closed Thursday's trade at $4.40, down 23.59%.
BioXcel Reports Positive Phase 2 Results For BXCL501 In Opioid Withdrawal Treatment
BioXcel Therapeutics, Inc. (BTAI) on Thursday reported positive topline results from a Phase 2 study evaluating BXCL501 for the treatment of opioid withdrawal symptoms in adults with undergoing a methadone taper.
The study indicated that BXCL501 may be as effective as or superior to Lucemyra, an FDA-approved non-opioid medicine, in reducing opioid withdrawal symptoms, while offering a more convenient dosing schedule and a favourable tolerability profile.
BXCL501 demonstrated similar or lower overall rates of cardiovascular side effects compared with lofexidine, and no cases of sedation or somnolence were reported in the BXCL501 groups, compared with 5% in the Lucemyra arm.
BTAI closed Thursday's trade at $1.61, down 2.42%.
Roche's Petrelintide Shows Positive Results In Phase 2 Trial
Roche (RHHBY) announced positive topline results from its Phase 2 ZUPREME-1 study of Petrelintide, an investigational amylin analogue for people living with overweight and obesity.
Petrelintide is once-weekly subcutaneous injection to help restore sensitivity to the satiety hormone leptin, enabling patients to feel full faster and sustain weight loss.
The trial met its primary endpoint, demonstrating that participants achieved a mean body weight reduction of 10.7% at week 42, compared to 1.7% with placebo.
Roche highlighted that female participant in the study lost considerably more weight than male participants, underscoring the drug's potential impact across diverse patient groups.
According to the firm, the treatment was well tolerated, with no cases of vomiting or discontinuations due to gastrointestinal side effects at the maximally effective dose.
RHHBY closed Thursday's trade at $54.19, down 6.34%.
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