It’s been a decade since Sandoz Inc. launched Zarxio, referencing Amgen Inc.’s Neupogen (filgrastim), as the first biosimilar in the U.S. Zarxio was expected to be the beginning of a biosimilar boom that would deliver big savings by finally providing direct competition for costly biologics. Neither the pipeline nor the uptake of biosimilars has lived up to expectations, as only 6% of the 313 biologics approved by the FDA’s CDER have been targeted by biosimilars and fewer than 5% are actually competing with the follow-ons.

Novartis’ gene therapy, Itvisma, approved for older SMA patients

More than six years after Novartis AG’s Zolgensma was approved for children under 2 with spinal muscular atrophy (SMA) with biallelic mutations in the survival motor neuron 1 (SMN1) gene, the U.S. FDA cleared a new version, under the brand name Itvisma (onasemnogene abeparvovec-brve), for those 2 and older, including teens and adults with the same mutation. For the older population, it is the first gene replacement therapy available, delivered through a one-time fixed dose that is not dependent on age or body weight. By replacing SMN1, patients can regain motor function without needing to rely on chronic treatment. The wholesale acquisition cost for Itvisma is $2.59 million, and it will be available in December. “In the U.S., Itvisma is priced at 35%-46% less than the 10-year cost of the available chronic disease-modifying therapies,” a Novartis spokesperson told BioWorld.

Phrontline raises $60M in pre-A+ round for bispecific ADCs

Phrontline Biopharma Suzhou Co. Ltd. closed a $60 million pre-A+ financing round led by Lapam Investment, with participation from nine other investors. Six new investors joined the round, including Samsung Venture Investment Corp. (SVIC), Guofang Innovation, Hankang Venture Capital, Songqing Capital, Jifeng Ventures and Sino Biopharmaceutical Ltd. Existing shareholders, including Decheng Capital, Medfine Health Fund and C&D Emerging Investment, participated and increased their stakes. The funds raised will support Phrontline’s development of next-generation antibody-drug conjugate (ADC) candidates, built on the Suzhou-based firm’s bispecific antibody and dual-linker payload ADC platforms.

Novo’s amycretin drops weight in phase II study of type 2 diabetes

In a phase II study, Novo Nordisk A/S’s amycretin reduced the weight of type 2 diabetes patients by 14.5% in 36 weeks, a statistically significant loss in weight. The results also reduced hemoglobin A1C, an average of blood glucose used to monitor blood sugar control, below 7% in up to 89.1% of the participants. The co-agonist of GLP-1 and amylin receptors was subcutaneously administered once a week to patients or once a day orally in multiple ascending doses. The results were encouraging enough for Novo to say it planned a phase III development program next year of amycretin in the same group, adults with type 2 diabetes.

Gene therapies aim for the big goal of edits in vivo

The field of gene therapy is experiencing major advances driven by precise editing technologies, such as base and prime editing, and by the design of increasingly sophisticated vectors to deliver payloads that could reverse the effects of diseases. However, in the transition to in vivo applications many approaches still fail in their attempt to effectively reach target tissues or cells.

Otsuka submits NDA for new non-stimulant ADHD therapy

Otsuka Pharmaceutical Co. Ltd. filed an NDA with the U.S. FDA for centanafadine (formerly EB-1020), a new nonstimulant therapy for treating attention-deficit hyperactivity disorder (ADHD) in children, adolescents and adults. Centanafadine is a norepinephrine, dopamine and serotonin reuptake inhibitor formulated as a once-daily extended-release capsule. “Centanafadine represents a first-in-class mechanism of action among available ADHD therapies, and if approved, may expand the range of options available to those managing this complex condition.” said John Kraus, Otsuka Pharmaceutical Development & Commercialization Inc.’s executive vice president and chief medical officer.

Also in the news

Alvotech, Astrazeneca, Avenzo, Azitra, Bayer, BCM, Biocardia, Biocortex, Biogen, Capricor, CD, Clearmind, Clearside, Day One, Dayra, Enlivex, Fibrobiologics, Galvanize, Genenta, Genprex, Harbour, Harmony, Invivyd, Johnson & Johnson, Kelun-Biotech, Merck, Mindimmune, Nervgen, Novo Nordisk, Parabilis, Pasithea, Scilex, Vaxil, Vaximm, Verrica