-Once-daily oral ALK2 inhibitor approved to reduce the volume of total new heterotopic ossification in patients aged 12 years and older with FOP -Atebrioz expected to be available in the United States in October through Mirum Access Plus (MAP), with eligible patients paying as little as $0 per month -PROGRESS pediatric development program continues to evaluate zilurgisertib in children aged 2 to <12 years Mirum Pharmaceuticals, Inc. (Nasdaq:MIRM) and Incyte (Nasdaq:INCY) today announced that

SLS009 increased apoptosis more than six-fold versus daraxonrasib in a MYC-amplified, daraxonrasib-resistant patient-derived pancreatic cancer organoid model - Combination of SLS009 and daraxonrasib further increased apoptosis to 36.5% and necrosis to 30.6%, supporting evaluation of CDK9 inhibition as a potential strategy to enhance RAS-directed therapy - SLS009 combined with BET inhibition demonstrated synergistic activity and sustained suppression of MYC, providing additional evidence of SLS0

Novo will explore the use of Nanexa's PharmaShell technology platform in an effort to reduce the dosing frequency of injectables for the treatment of obesity, type 2 diabetes and other cardiometabolic indications. The deal, disclosed late Thursday by Nanexa, is potentially worth up to €1.165 billion ($1.3 billion). PharmaShell is Nanexa's atomic layer deposition (ALD)-based drug delivery platform, which is designed to enable controlled and sustained release of active pharmaceutical ingredients

Kovina Therapeutics Inc., a biotechnology company developing first-in-class small molecule therapeutics targeting the Human Papillomavirus (HPV) E6 oncoprotein, today announced a $2.3 million Direct-to-Phase II Small Business Innovation Research (SBIR) award from the National Cancer Institute (NCI). The two-year award will support IND-enabling development of Kovina’s lead oral small molecule program for HPV-driven cancers. The award follows Kovina’s recent publication in the Proceedings of the N

- 48.9% placebo-adjusted reduction in psoriasis area and severity index (PASI) score achieved in mild-to-moderate plaque psoriasis patients with once-daily 200 mg 28-day treatment. - Steady-state elimination half-life after 28-day treatment in patients was 6.5 days, supporting potential once-weekly oral dosing. - Placebo-adjusted reduction in PASI score reached 65.9% in mild-to-moderate plaque psoriasis patients 15 days after last dose (28th dose), which supports potential once-weekly oral dosin

The FDA on Friday approved AbbVie's Juvmo (tavapadon) to treat adults with Parkinson's disease, according to a listing on the agency's approvals database. The pharma gained the D1/D5 partial agonist through its $8.7-billion takeout of Cerevel Therapeutics, announced in late 2023. However, the centrepiece of that deal, experimental schizophrenia treatment emraclidine, failed in mid-stage testing shortly after the acquisition closed (see – Spotlight On: Three things to watch at AbbVie). Despite

If approved, Joenja would be available to eligible patients aged 4 years and older with APDS who weigh 13 kg or more Decision follows recent approval of Joenja for children aged 4 to 11 years with APDS weighing at least 27 kg PDUFA target action date of January 30, 2027 September 25, 2026: Pharming (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune and genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its

Results presented at IMS 2026 show ZENBEXUS in combination with daratumumab and dexamethasone doubled minimal residual disease-negative complete response rates versus daratumumab, bortezomib and dexamethasone (41.1% vs. 20.7%) Data reinforce the value of the ZENBEXUS-based triplet as an effective, easily administered treatment option for patients with relapsed or refractory multiple myeloma as early as first relapse Results simultaneously published in The Lancet Oncology Bristol Myers Squibb (N

The new partnership follows Roche’s recent R&D expansion in Boston focused on development for cardiovascular, renal and metabolic diseases. Roche is doling out $70 million to pair up with Atavistik Bio in hopes of discovering new small molecule therapeutics against cardiovascular, renal and metabolic diseases, with an additional $1.9 billion in biobucks up for grabs. The Big Pharma is joining forces with the hematology-focused biotech to use its allosteric drug discovery platform, dubbed AMPS,

Ligand Pharmaceuticals Incorporated (Nasdaq: LGND) today announced that it has entered into a financing agreement with AvenCell Therapeutics, Inc., a clinical-stage cell therapy company developing controllable, allogeneic CAR-T therapies for patients with cancer, for up to $47 million. The investment will help advance AvenCell’s pipeline, including AVC-201 for the treatment of relapsed/refractory acute myeloid leukemia (AML) and AVC-203 for the treatment of B-cell malignancies. Under the terms