Shares of GRI Bio Inc. (NASDAQ:GRI) were trading at $6.28, up $3.08, or 96%, after the firm reported interim safety results from the ongoing phase IIa study testing GRI-0621 for idiopathic pulmonary fibrosis (IPF). An oral formulation of tazarotene, GRI-0621 is a RAR-βɣ dual agonist designed to inhibit the activity of human type 1 invariant natural killer T cells. The preplanned interim analysis for two-week safety results from the biomarker study in IPF showed GRI-0621 at 4.5 mg orally once daily safe and well-tolerated in the first 12 patients.

Phase III primary endpoint data disappoint Axsome but subgroup beckons

Despite missing the primary endpoint in a phase III study of treating major depressive disorder, Axsome Therapeutics Inc. still sees a path to another phase III. The study failed to show a statistically significant shift in the overall patient population given solriamfetol compared to placebo. But a subgroup with severe excessive daytime sleepiness saw improvements in depressive symptoms compared to placebo. Solriamfetol is a dopamine and norepinephrine reuptake inhibitor, and TAAR1 and 5-HT1A agonist. The company’s stock (NASDAQ:AXSM) wobbled before the opening bell, then jelled in the next few hours to a 6.2% drop as shares were selling at $109.38 each.

Where’s the ‘radical transparency’ Kennedy promised?

As the U.S. Department of Health and Human Services (HHS) begins implementing its reorganization and reduction-in-force (RIF) plan by sending out termination notices this week to 10,000 employees across its agencies, top Democratic congressional appropriators are demanding details about the plan and called on HHS Secretary Robert Kennedy to deliver on the “radical transparency” he promised when he was seeking the job. In a letter sent yesterday to Kennedy, the three lawmakers, led by Sen. Patty Murray, D-Wash., challenged the legality of the terminations, claiming that HHS’ actions will put the health and well-being of Americans at risk.

Anocca launching Europe study of gene-edited T-cell therapy

Anocca AB has secured the first regulatory authorization in Europe for a CRISPR-Cas gene-edited T-cell receptor T-cell therapy (TCR-T) trial, with the phase I/II study now due to start over the next three months at sites in Sweden, Germany, Denmark and the Netherlands. The umbrella study, Vidar-1, will test three different TCR-Ts, targeted via specific cell surface human leukocyte antigens at three different oncogenic driver mutations in KRAS, in patients with pancreatic adenocarcinoma.

Airna’s $155M series B advances RNA editing drug AIR-001 for AATD

Airna Corp. Inc. closed an oversubscribed $155 million series B financing led by Venrock Healthcare Capital Partners and Forbion Growth, with RTW Investments, Nextech Invest, Arch Venture Partners, Forbion Ventures, ND Capital and other new and existing investors also participating. Proceeds are expected to go toward moving the company’s lead RNA editing product candidate, AIR-001, for alpha-1 antitrypsin deficiency (AATD) into a phase I/II trial, as well as to advance a pipeline of RNA-editing medicines for cardiometabolic and other diseases. Airna plans to file a clinical trial application in the second half of 2025. The company, which is based in Cambridge, Mass., with research operations in Tübingen, Germany, launched in 2023 with $30 million to start, and pulled in another $60 million last July, bringing the series A round to $90 million raised.

Merck opts in for global license of Abbisko’s pimicotinib for $85M

Merck KGaA is exercising its option for a global license to commercialize Abbisko Therapeutics Co. Ltd.’s pimicotinib for $85 million. Abbisko out-licensed China rights to its colony-stimulating factor 1 receptor inhibitor, pimicotinib, to Merck in a deal worth up to $605.5 million in December 2023. Under terms of the deal, Merck was granted an exclusive license to commercialize products comprising pimicotinib (ABSK-021) for all indications in mainland China, Hong Kong, Macau and Taiwan. Merck had the option to commercialize the drug globally for an additional payment, Hua Jiang, Abbisko’s business development director told BioWorld.

BioWorld Insider Podcast: As biopharma investments stumble, med tech surges

The biopharma sector is still trying to get its wind and resume its once-powerful investment ways. Medical technology has sidestepped much of biopharma’s issues by being more flexible along the development path, according to BioWorld MedTech Editor Annette Boyle in this edition of the podcast. This year’s med-tech investment numbers have improved over those from last year and the year before by bringing in $2.76 billion by financing 56 transactions in January alone. Boyle described the current financing climate on the newest BioWorld Insider podcast and explained why the sector is performing well year to date.

Also in the news

Alnylam, Astrazeneca, Atavistik, Ayrmid, Biocardia, Biomark, Biomx, Biora, Beigene, Bluebird, BMS, Cabaletta, Corcept, Cyclo, Eli Lilly, Ensysce, Fibrogen, Gyre, Halozyme, Ideaya, Keros, LB, Marvel, Mineralys, Moderna, Modus, Nanobiotix, Nervgen, Novartis, Novo Nordisk, Opthea, Otsuka, Palatin, Polyrizon, Probiogen, Rafael, Regulus, Sensei, Talphera, Transcend, Vaxcyte, Vertex