X4 Pharmaceuticals Inc. plans to spend this year educating doctors and patients while helping the latter navigate reimbursement for Xolremdi (mavorixafor) capsules, approved by the U.S. FDA for patients 12 years and older with warts, hypogammaglobulinemia, infections and myelokathexis, or WHIM syndrome. The drug is designed to increase the number of circulating mature neutrophils and lymphocytes. A selective CXC chemokine receptor 4 (CXCR4) antagonist, Xolremdi is the first therapy for WHIM, an ultra-rare, combined primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 pathway dysfunction.
Deciphera says o-yes to $2.4B Ono acquisition
In a $2.4 billion deal, Deciphera Pharmaceuticals Inc. is merging with Ono Pharmaceutical Co. Ltd. Ono is acquiring all of Deciphera’s outstanding stock shares for $25.60 each in cash. Deciphera brings with it U.S. FDA-approved Qinlock for adults with gastrointestinal stomal tumor who have received prior treatment with three or more kinase inhibitor therapies. It also had a phase III success in October with vimseltinib in patients with tenosynovial giant cell tumor not amenable to surgery. Ono’s Opdivo (nivolumab) accounts for 60% of the Osaka, Japan-based company’s revenue. Deciphera’s stock (NASDAQ:DCPH) was trading dramatically upward at midday, with shares going for $25.20 each, 72% higher than when they started the day.
Repertoire, BMS ink $1.8B autoimmune disease discovery deal
Flagship Pioneering-backed Repertoire Immune Medicines Inc. landed its first major collaboration, picking up $65 million up front in a deal with Bristol Myers Squibb Co. that could bring up to $1.8 billion in milestone and other payments, plus tiered royalties. The companies will use Repertoire’s Decode T-cell receptor-epitope discovery platform to develop tolerizing vaccines for up to three autoimmune diseases. The deal follows BMS’s restructuring news last week as the big pharma focuses on long-term growth drivers.
Sanders pressures international industry to back Pandemic Accord
Looking beyond the U.S. biopharma industry, Sen. Bernie Sanders (I-Vt.) is now pushing the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA) to get on board with the World Health Organization’s proposed Pandemic Accord aimed at making diagnostics, treatments and vaccines available to everyone who needs them. “Your responsibility does not end with inventing breakthroughs. The products you develop must be made available around the world quickly and affordably,” Sanders said in a letter Friday to IFPMA leadership. “At the very least, you should not challenge governments negotiating new rules to make medicines more accessible.”
Biopharma index sustains Q1 growth bolstered by positive earnings
The BioWorld Biopharmaceutical Index (BBI) continued to climb during the first quarter (Q1), reaching a 6.4% increase by the end of March. A number of biopharma firms on BBI saw shares tick up after positive earnings reports, and investors are focused on top gainer Sarepta Therapeutics Inc.’s Elevidys label expansion and future sales. Meanwhile, Alnylam Pharmaceuticals Inc. saw shares in Q1 down 20%, while analysts remain hopeful for a positive Helios-B readout for Amvuttra. Biogen Inc. fell 17% in the first three months of the year, partly attributed to declining quarterly sales and profits and missing Wall Street expectations in its Q4 financials.
X chromosome silencer contributes to female autoimmune risk
Females have a much greater risk of developing an autoimmune disease than males do. Eighty percent of autoimmune disease patients are female, and specific disorders can have an even more lopsided ratio – 90% of systemic lupus erythematosus (lupus) and almost 95% of Sjögren’s disease patients are female. At a recent joint event of the NIH’s Office of Autoimmune Disease Research and Office of Research on Women’s Health, scientists presented new insights into one factor that underpins females’ greater risk: Xist, which silences all but one X chromosome per cell.
Investors highlight hot therapy areas in Asia Pacific
Antibody-drug conjugates (ADCs) are the hot spot for deals in Asia, but investors questioned whether oncology is really the place to be, during the Asia Bio Partnering Forum in Singapore April 24 to 25. Asia is ripe for neurology and infectious diseases, said Arthur Pappas, CEO and managing general partner of Pappas Capital, noting that modifications on ADCs will remain hot, but infectious disease is the hot spot in Asia. Commercializing therapies in infectious diseases remains a challenge, however, said Damian O’Connell, CEO of the Experimental Drug Development Centre, Singapore’s national platform for drug discovery and development.
Korea-US biotech Genosco passes technology review for Kosdaq IPO
Boston-based South Korean biotech Genosco Inc. said on April 25 that it passed a technology review required for the special listing track on the tech-heavy Kosdaq market, as it readies an IPO on the Korea Exchange (KRX). Genosco, of Billerica, Mass., is a subsidiary of Seongnam-si, Gyeonggi-do-based Oscotec Inc. Genosco was first founded as OCT USA Inc. in Los Angeles in November 2000, until OCT opened its Boston-based R&D Center for novel small-molecule and protein drug development in 2008 and changed its name to Genosco in 2009.
BioWorld Insider Podcast: Mark McKenna storms back with Mirador and eyes the development landscape
In one of the biggest financings of the year so far, former Prometheus Biosciences Inc. CEO Mark McKenna helped raise $400 million to launch a new company, Mirador Therapeutics Inc. He didn’t sit on the sidelines for long after Merck & Co. Inc. bought Prometheus for $10.8 billion in 2023. He recruited key Prometheus executives to focus on Mirador’s genetic approach to drug discovery and precision medicine. McKenna said there was too much left undone to just hang back. In this BioWorld Insider episode, he talks about the new company and the multi-billion-dollar drugs that he believes provide sub-optimal efficacy compared to the tailor-made therapies he wants to develop. He also has deep insights into drug pricing and why the investment market is so tough on companies that don’t have A-plus science and teams.