Pociredir

Headache drug specialist Slate Medicines said Monday it will combine with struggling biotechnology firm Fulcrum Therapeutics, two months after the latter announced it was seeking strategic alternatives following the discontinuation of its sickle cell drug.Fulcrum spent years developing pociredir, a drug that boosts fetal hemaglobin levels by inhibiting a group of proteins called PRC2 in patients with sickle cell disease. The biotech theorized that doing so would prevent the malformation of red blood cells and thus reduce the rate of vaso-occlusive crises, the sudden, severe pain that occurs when sickle-shaped blood cells block blood flow.The Food and Drug Administration previously hit Fulcrum with a six-month trial hold over concerns that targeting PRC2 could heighten the risk of developing hematological cancers. In June, Fulcrum said it would discontinue development of pociredir after regulators expressed worries over the rate of development of secondary blood cancers in patients on Ipsens Tazverik, which worked similarly to Fulcrums drug. (Tazverik was pulled from market in March.)Fulcrum has now found a new home in Slate, which started up earlier this year. The young biotech raised $130 million from backers like RA Capital Management and Forbion to fund testing of an experimental medicine that targets a protein called PACAP. Rather than go after CGRP, a popular target for other pharmaceutical companies developing headache drugs, Slate is trying to develop a PACAP inhibitor to treat migraines in patients who dont respond to approved treatments.That experimental medicine, licensed from a Chinese pharmaceutical firm and codenamed SLTE-1009, will soon start Phase 1 testing. An additional $245 million financing announced Monday will help Slate with developing another monoclonal antibody its testing to treat migraines, as well as a third undisclosed program.The merger gives Fulcrum a compelling opportunity to participate in the development of a portfolio of next generation migraine therapeutics, said Alex Sapir, Fulcrums CEO, in a statement.According to Mondays announcement, Fulcrums pre-merger shareholders will receive a cash dividend equal to the amount by which Fulcrum's net cash exceeds $20.3 million. The company held just over $333 million in cash, cash equivalents and securities at the end of the first quarter of 2026.Fulcrum stockholders will own 5% of the combined company, while Slates investors hold onto 95% of shares. Gregory Oakes, Slates CEO, will head the merged company.The new biotech will operate under Slates name and trade on Nasdaq under the ticker symbol SLTE. The deal is expected to close by the end of 2026. '

Ned Sharpless on Trump admin's shortlist to lead FDA: reportFulcrum cuts 85% of workforceInnoCare's BTK inhibitor clears mid-stage lupus trialEU approves paediatric label for Chiesi's LoxujtaNed Sharpless is on Trump admin's shortlist to lead FDA: reportThe Trump administration is reportedly considering Ned Sharpless to serve as the permanent FDA commissioner, according to CNN, which cited four people familiar with the matter. He would replace Kyle Diamantas, who stepped in as interim chief following Marty Makary's resignation in May. Sharpless, who previously led the National Cancer Institute and filled in as acting FDA head for seven months under President Donald Trump's first term, is one of a "handful" of outside candidates who are being vetted for the job — including Richard Pops, who is set to retire from his 35-year role as CEO of Alkermes — though the search process is still ongoing. Since leaving the FDA, Sharpless helped found Jupiter Bioventures, a biotech venture foundry armed with $70 million to create eight to 10 oncology-focused startups. He also, alongside several former FDA leaders, penned a letter last year condemning changes to US vaccine policy proposed by Vinay Prasad, the former head of the agency's Center for Biologics Evaluation and Research.-Elizabeth EatonFulcrum cuts 85% of workforceA few days after ending work on its only clinical programme — the oral small molecule EED inhibitor pociredir for sickle cell disease — Fulcrum Therapeutics disclosed in a securities filing that it will cut 85% of its workforce, leaving it with just nine full-time employees.The restructuring plan was approved by the company's board on May 31 in an effort to reduce operating expenses and preserve capital. The workforce reduction was communicated on June 4 and is expected to be completed in the second quarter.As previously indicated, Fulcrum is undertaking a review of strategic alternatives, which may include a merger, acquisition or business combination. -Matt DennisInnoCare's BTK inhibitor clears mid-stage lupus trialUpbeat mid-stage data for InnoCare Pharma's BTK inhibitor orelabrutinib in systemic lupus erythematosus (SLE) were presented Friday at the European Alliance of Associations for Rheumatology (EULAR) congress.The Phase IIb trial randomised 187 patients with moderate-to-severe SLE to receive one of two daily doses of orelabrutinib or placebo. Results showed the higher-dose group achieved an SLE Response Index-4 (SRI-4) response rate of 57.1% versus 34.4% for placebo at week 48, meeting the study's primary endpoint. The higher-dose arm also met secondary goals for SRI-6 and British Isles Lupus Assessment Group–Based Composite Lupus Assessment (BICLA) response rates versus placebo. Additionally, 71.1% of patients receiving the higher dose had their steroid use down to ≤7.5 mg versus 43.6% of placebo recipients. Meanwhile, orelabrutinib was safe and well tolerated, with no new signals identified. The BTK inhibitor is already marketed for certain haematologic malignancies in China, Australia and Singapore under the name Hibruka. InnoCare is currently conducting a registrational Phase III trial of orelabrutinib for SLE, while investigating it for multiple sclerosis in partnership with Biogen.-Pavan KamatEU approves paediatric label for Chiesi's LoxujtaThe European Commission authorised a broader label for Chiesi's Loxujta (lomitapide) covering children aged 5 years and older with homozygous familial hypercholesterolaemia (HoFH), in conjunction with diet and other lipid-lowering treatments, including LDL-apheresis where available. The microsomal triglyceride transfer protein (MTP) inhibitor was initially approved in the EU for adults with HoFH back in 2013.The expanded indication is supported by data from the Phase III APH-19 study. The trial met its primary endpoint, demonstrating a mean 53.5% reduction in LDL-C from baseline at week 24, with significant reductions also recorded across non-HDL-C, total cholesterol, VLDL-C, apolipoprotein B and triglycerides. Adverse events were predominantly mild and gastrointestinal or hepatic in nature; no new safety signals were identified.-Pavan Kamat