PETAH TIKVA (dpa-AFX) - This week's biotech landscape witnessed key FDA meetings being scheduled, IND clearances, complete response letters, sale of oncology assets and clinical trial data readouts across key therapeutic areas such as Fabry Disease, Gaucher disease, Obesity, HbA1c Reduction and Weight Loss in Type 2 Diabetes.
Let us unpack the key developments and milestones in the biotech space this week.
FDA Approvals & Rejections
aTyr Pharma To Meet With FDA for Efzofitimod Program
aTyr Pharma, Inc. (ATYR) said it has scheduled a Type C meeting with the U.S. FDA in mid-April 2026 to discuss the clinical and regulatory path for Efzofitimod, its lead candidate for pulmonary sarcoidosis, a chronic inflammatory lung disease.
The meeting will focus on Phase 3 trial planning and key design considerations as the company prepares to advance Efzofitimod into late-stage development. aTyr said it will provide an update once it receives formal FDA feedback.
ATYR closed Thursday's trade at $0.81, down 7.93%.
Prelude Therapeutics Wins FDA Clearance for Mutant-Selective JAK2V617F Inhibitor Study
Prelude Therapeutics Inc. (PRLD) announced that the U.S. FDA has cleared its Investigational New Drug application for PRT12396, a mutant-selective JAK2V617F inhibitor being developed for myeloproliferative neoplasms driven by the JAK2 mutation.
The company said the clearance enables initiation of a Phase 1 trial evaluating safety, tolerability, and early pharmacologic activity in patients with JAK2V617F-mutated hematologic cancers. Prelude noted that PRT12396 has shown strong selectivity in preclinical studies, supporting its advancement into first-in-human testing.
PRLD closed Thursday's trade at $2.02, down 12.17%.
Teva Secures Expanded FDA Approval for Migraine Drug
Teva Pharmaceutical Industries Ltd. (TEVA) said the U.S. FDA has approved an expanded indication for AJOVY, making it the first anti-CGRP preventive treatment authorised for pediatric episodic migraine in patients aged 6-17 years who weigh at least 45 kilograms.
AJOVY was first approved in 2018 for the preventive treatment of migraine in adults, and the new decision extends its use to younger patients for the first time. The expanded label includes a once-monthly injection that can be administered in-office or at home, addressing a significant unmet need in pediatric migraine care.
Teva said the approval reinforces AJOVY's established safety and efficacy profile and broadens access to preventive therapy for children and adolescents.
TEVA closed Thursday's trade at $34.80, down 0.46%.
Aquestive Therapeutics Receives FDA Complete Response Letter for Anaphylm
Aquestive Therapeutics Inc. (AQST) said the U.S. FDA has issued a Complete Response Letter (CRL) for its New Drug Application for Anaphylm sublingual film, a non-invasive epinephrine treatment for Type I allergic reactions, including anaphylaxis, in patients weighing 30 kg or more.
The company said the deficiencies outlined in the CRL were limited to human-factors issues and a supportive pharmacokinetics (PK) study, with no concerns raised regarding chemistry, manufacturing, controls, or the clinical comparability data submitted. The FDA cited problems in the human-factors validation study, including difficulty opening the pouch and incorrect film placement, which the agency believes could pose safety risks during anaphylaxis.
Aquestive said it has already updated the pouch design, labeling, and instructions for use, and plans to conduct a new human-factors study along with the required PK study before resubmitting the application as early as Q3 2026.
AQST closed Thursday's trade at $3.84, down 4%.
FDA Declines to Approve Expanded Label of Pharming's Joenja
Pharming Group N.V. (PHAR) said the U.S. FDA has issued a Complete Response Letter (CRL) for the expanded indication of Joenja in children ages 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.
The FDA cited concerns about potential underexposure in lower-weight pediatric patients and requested additional pharmacokinetic data to reassess dosing. The agency also asked for clarification on an analytical method used for batch testing.
Pharming said it believes the issues can be addressed and will request a Type A meeting to determine next steps. The CRL does not affect Joenja's existing U.S. approval for patients 12 years and older.
PHAR closed Thursday's trade at $16.04, down 4.98%.
Deals
Polyrizon Signs MOU To Acquire 51% Stake In Global Private Aviation Company
Polyrizon Ltd. (PLRZ), a preclinical biotechnology company, has signed a non-binding Memorandum of Understanding to acquire a 51% stake in Arrow Aviation Ltd., a global private aviation operator.
Polyrizon develops intranasal protective hydrogel technologies and Arrow Aviation firm operates a fleet of executive jets and provides VIP private flights for business and leisure, as well as specialized services for government and public entities, including medical evacuations, air rescue, special-needs transport, medical tourism, and hazardous-materials cargo operations.
Arrow Aviation generates approximately $19 million in annual unaudited revenue and $3 million in adjusted EBITDA.
Under the terms of the MOU, Polyrizon intends to purchase the majority stake on a fully diluted basis through a cash investment of NIS 18 million (approximately $5.8 million). The move marks a strategic diversification into the high-growth private aviation sector.
Polyrizon said the potential acquisition aligns with its strategy to explore revenue-generating opportunities in high-growth sectors while leveraging its strong financial position. The company cited industry forecasts projecting the private aviation market to reach $41.38 billion by 2030.
A definitive agreement is expected within 30 days, subject to customary closing conditions.
PLRZclosed Thursday's trade at $12.43, down by 7.03%.
HOOKIPA Pharma To Sell Oncology Assets To NeoTrail Therapeutics
HOOKIPA Pharma Inc. (HOOK), a clinical-stage biopharmaceutical company that develops immunotherapeutics for cancer and other infectious diseases, announced the sale of its immunooncology assets, Eseba-vec, also known as HB-200, and HB-700 development programs, to NeoTrail Therapeutics, Inc., a privately held firm.
HOOKIPA's Eseba-vec (HB-200) is an investigational immunotherapeutic agent being evaluated for HPV16-positive cancers and HB-700 is an investigational arenaviral immunotherapy designed to treat KRAS-mutated lung, colorectal, pancreatic and other cancers.
The asset purchase agreement was signed on January 28, 2026, and the transaction is expected to close in the second quarter of 2026. The financial details of the sale are undisclosed.
HOOK closed Thursday's trade at $1.06, up 0.95.
Clinical Trials - Breakthroughs
Sangamo's Fabry Disease Study Of ST-920 Yields Positive Data
Sangamo Therapeutics, Inc. (SGMO), a genomic medicine firm, on Tuesday announced positive data from the registrational Phase 1/2 STAAR study evaluating its lead candidate, isaralgagene civaparvovec, for the treatment of Fabry disease.
The STAAR study enrolled patients who were on Enzyme Replacement Therapy (ERT), ERT pseudo-naïve defined as having been off ERT for six or more months, or ERT-naïve. The Phase 1/2 STAAR study is complete, and 32 patients have successfully rolled into the long-term follow-up study.
The promising results of ST-920 from the registrational Phase 1/2 STAAR study include a positive mean annualised estimated glomerular filtration rate (eGFR) slope at 52 weeks across all dosed patients in the study as an intermediate clinical endpoint reported earlier, stable cardiac function over one year, and durability of effect, with elevated expression of alpha-galactosidase A (a-Gal A) activity maintained for up to 4.5 years.eGFR estimates kidney function by measuring how well the kidneys filter waste.
The FDA provided a regulatory pathway to accelerated approval for isaralgagene civaparvovec, agreeing that data from the ongoing Phase 1/2 STAAR study can serve as the primary basis for approval under the Accelerated Approval Program, using the mean annualised eGFR slope at the 52-week clinical endpoint.
SGMO closed Thursday's trade at $1.86, down 1.86%.
Novo Nordisk's CagriSema Shows Notable HbA1c Reduction and Weight Loss In REIMAGINE 2 Trial
Novo Nordisk A/S (NVO) announced positive results from phase 3 REIMAGINE 2 trial, in which CagriSema demonstrated both superior HbA1c reduction and weight loss at week 68 versus semaglutide, across all tested doses in adults with type 2 diabetes or T2D.
The positive results from REIMAGINE 2 include CagriSema achieving superior weight loss of up to 14.2%, and a reduction of up to 1.91 % points from a mean HbA baseline of 8.2%.
CagriSema is a fixed-dose combination of a long-acting amylin receptor agonist, cagrilintide, and a long-acting GLP-1 receptor agonist, semaglutide.
REIMAGINE 2 was a phase 3, 68-week efficacy and safety trial investigating once-weekly subcutaneous CagriSema in two different doses (2.4 mg/2.4 mg and 1.0 mg/1.0 mg) compared to two different doses of semaglutide (2.4 mg and 1.0 mg), cagrilintide (2.4 mg), and placebo.
The trial included 2,728 people with type 2 diabetes inadequately controlled with metformin with or without a sodium-glucose transport protein 2(SGLT2) inhibitor.
The findings showed treatment with CagriSema 2.4mg/2.4mg resulted in a statistically significant reduction in HbA1c of 1.91% points compared to 1.76% points with semaglutide 2.4mg at week 68.
Also, from a mean baseline body weight of 101 kg, people treated with CagriSema 2.4 mg/2.4 mg achieved a 14.2% weight loss after 68 weeks without a weight-loss plateau, compared with 10.2% with semaglutide 2.4 mg.
NVO closed Thursday's trade at $43.34, down 8.16%.
Skye Reports Positive Data from Extension Phase Of CBeyond Trial In Obesity
Skye Bioscience, Inc. (SKYE), a clinical-stage biotechnology company, announced positive interim 52-week data from the combination-therapy arms in the extension Phase 2a CBeyond proof-of-concept study of nimacimab, its lead antibody candidate in obesity.
Nimacimab is a peripherally restricted monoclonal antibody inhibitor of the CB1 receptor.
The positive results from the extension phase of the Phase 2a Cbeyond study include achieving a significant total weight loss of 22.3% in the combination arm of nimacimab and semaglutide, with no weight-loss plateau observed, compared with 19.7% in the placebo plus semaglutide arm after 52 weeks of treatment.
Full topline results from the extension CBeyond Phase 2a study, including nimacimab monotherapy data and 13-week off-therapy follow-up, are expected in Q3 2026.
SKYE closed Thursday's trade at $0.79, down 8.11%.
Sanofi's Venglustat Meets Primary Goal In LEAP2MONO Gaucher Study, Falls Short In PERIDOT study For Fabry Disease
Sanofi (SNY) announced that its investigational drug Venglustat met all primary endpoints in a phase 3 study of type 3 Gaucher disease dubbed LEAP2MONO while it did not meet the primary endpoint in a phase 3 study of Fabry disease dubbed PERIDOT.
Venglustat is an investigational oral glucosylceramide synthase inhibitor (GCSi) designed to cross the blood-brain barrier.
In the phase 3 study of type 3 Gaucher disease(GD3) in adults and pediatric patients, dubbed LEAP2MONO, patients receiving Venglustat demonstrated significant improvements in the primary endpoints of neurological symptoms measured by a global test score for two assessments, the Scale for Assessment and Rating of Ataxia (SARA) modified total score and the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS), at week 52 compared with those receiving enzyme replacement therapy.
In the phase 3 study in Fabry disease, dubbed PERIDOT, Venglustat did not demonstrate superiority on the primary endpoint as both the Venglustat group and the placebo group experienced reductions in neuropathic and abdominal pain. Additional data are being analysed, with more information expected to be shared at a future medical meeting.
The U.S. Food and Drug Administration also granted fast-track designation to Venglustat for its potential use in GD3 and Fabry disease.
SNY closed Thursday's trade at $47.49, down 1.23%
Alvotech Reports Positive Top-line Pharmacokinetic Study Results for AVT80, A Biosimilar to Entyvio
Alvotech (ALVO) reported that its pivotal pharmacokinetic study dubbed AVT80-GL-P01 for AVT80, its planned biosimilar to Takeda Pharma's Entyvio, met all primary endpoints, marking a key milestone in the company's dual-formulation vedolizumab program.
Entyvio is indicated for the treatment of Ulcerative Colitis and Crohn's disease, two chronic inflammatory bowel diseases that often require long-term biologic therapy.
The pivotal pharmacokinetic study, AVT80-GL-P01, was a randomised, double-blind, single-dose trial conducted in healthy adults.
It compared AVT80 with the reference product and demonstrated pharmacokinetic similarity, supported by aligned safety, tolerability, and immunogenicity profiles, which form the core clinical requirement for biosimilar approval in major regulatory markets.
ALVO closed Thursday's trade at $4.99, down 1.96%.
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