The U.S. FDA approved Intrabio Inc.’s Aqneursa (levacetylleucine) on its PDUFA date for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg, making Aqneursa the only approved stand-alone therapy indicated for NPC. Pricing was not disclosed. On Sept. 20, Zevra Therapeutics Inc., of Celebration, Fla., won FDA clearance for Miplyffa (arimoclomol), used in combination with enzyme inhibitor miglustat, as the first treatment for NPC, a rare genetic disease linked to progressive neurological symptoms that is almost always fatal. Zevra said the wholesale acquisition cost of Miplyffa will land between $40,000 and $106,000 per month, depending on dosage. Miplyffa won’t be available for eight to 12 weeks; Aqneursa sales are to start immediately.

Amgen’s rocatinlimab data underwhelm in atopic dermatitis trial

As Eli Lilly and Co. launches its recently approved Ebglyss (lebrikizumab) in an atopic dermatitis (AD) market already dominated by established biologic Dupixent (dupilumab, Regeneron Pharmaceuticals Inc.), investors tuned into an Amgen Inc. investor call disclosing top-line phase III results for rocatinlimab, a monoclonal antibody that could potentially offer patients a new mechanism of action. Data from the Rocket Horizon study showed rocatinlimab hit all co-primary and secondary endpoints, though analysts described the early findings as “disappointing” and “underwhelming” when viewed against the increasing competition in the AD disease space. Similar reactions were noted for Amgen’s phase III readout for Uplizna (inebilizumab) in IgG4-related disease and generalized myasthenia gravis.

Should generics jump the queue in Wegovy parade?

Given the demand for Ozempic and Wegovy and the revenue the GLP-1 drugs are generating for Novo Nordisk A/S in the U.S., a lot of generic companies are clamoring to cash in on the drugs’ current popularity. And there are some U.S. lawmakers more than willing to oblige. Ahead of yesterday’s Senate Health, Education, Labor and Pensions Committee hearing on the U.S. pricing of Ozempic and Wegovy, more than a dozen congressional members wrote to Health and Human Services Secretary Xavier Becerra urging him to use his legal authority to license generic competition to the semaglutide drugs, even though they still have more than a decade’s worth of patent protection and Wegovy has some marketing exclusivity for a few more years.

Genespire raises €47M to advance Gene-202, lentiviral vectors

In one of the largest private rounds raised by an Italian biotech, Genespire Srl has closed a €46.6 million (US$51.88 million) series B, enabling it to lay the ground for a phase I/II clinical trial of its lead program, Gene-202, and to further develop its proprietary lentiviral vectors. The vectors are designed to be applicable to a range of liver-related metabolic disorders and as its first indication, the company intends to treat methylmalonic acidemia (MMA), a serious inherited condition which results in impaired metabolism of certain amino acids and lipids. Currently, there are no disease modifying treatment for MMA, which causes significant mortality and morbidity in affected children, including muscular weakness, seizures, developmental delays and organ damage.

Neurological diseases index falls; Sarepta and Intra-Cellular report gains

As of the end of August 2024, the BioWorld Neurological Diseases Index (BNDI) reported a decline of 8.89%, lagging behind the Nasdaq Biotechnology Index and the Dow Jones Industrial Average. In recent months, shares of Sarepta Therapeutics Inc. jumped following the U.S. FDA approval of Elevidys (delandistrogene moxeparvovec), while Intra-Cellular Therapeutics Inc. gained ground after positive results from Caplyta (lumateperone).

Aussie VC to triple biotech investments in undervalued market

One of Australia’s newest biotech investment funds is set to triple investments on the back of its success and strong investor demand. The three-year old Merchant Biotech Fund (MBF) invested in several high growth ASX-listed and private life sciences companies and is up 10% for the current financial year, Portfolio Manager Reece O'Connell told BioWorld. The Perth, Australia-based fund delivered a 29.8% return in its second year despite a challenging market for biotechnology and small cap stocks.

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AB Science, Abiologics, Acurx, Aditxt, Altamira, Aptadir, Araris, Arcutis, Atavistik, Aurion, Biogen, Biovaxys, Biovie, Black Diamond, Bluebird, Capricor, Cassava, Catalyst, CSL Vifor, Eli Lilly, Enlivex, Evofem, Fleming, George Medicines, GSK, Henlez, Hutchmed, Immutep, Innate, Lift, Lipella, Longbio, Metsera, Nona, Novavax, Nxera, Quell, Rion, Santhera, Septerna, Silexion, Synedgen, Takeda, UCB, Verimmune, Wave