The timing is ripe for a robust biosimilar market in China, given the rapid increase of novel biologics approved to treat cancer and inflammatory diseases in the country over the past decade and the looming patent cliffs for several established biologics. As of December, the NMPA had approved more than 20 biosimilars that were developed in China. Most of those referenced just two biologics – Roche AG’s cancer drug Avastin (bevacizumab) and Abbvie Inc.’s immunology drug Humira (adalimumab). In 2022, the oncology and immunology biosimilar market in China garnered sales of about $2 billion, according to Clarivate estimates. To reach their full potential in China though, biosimilars must win over prescribers and patients.

Amylyx reports ‘encouraging’ data in rare Wolfram syndrome

With the recent removal of its amyotrophic lateral sclerosis drug from the market, Amylyx Pharmaceuticals Inc. is looking to revive investor interest with data from eight participants in the Helios phase II trial testing the same drug, AMX-0035 (sodium phenylbutyrate plus taurursodiol), in Wolfram syndrome. Interim data showed all eight patients met prespecified responder criteria, demonstrating either improvement or stabilization of disease. The trial’s principal investigator, Fumihiko Urano, noted “encouraging” improvements across multiple organ systems. Amylyx is one of few very drug developers working on treatments for Wolfram syndrome, a rare genetic disease characterized by childhood-onset diabetes, optic nerve atrophy and neurodegeneration.

Still targeting Xtandi, groups ask CMS to do what NIH wouldn’t

Having failed in its efforts to get the U.S. NIH to march in on Xtandi’s patents under the Bayh-Dole Act because of price, Knowledge Ecology International has joined with two other groups in asking the Centers for Medicare & Medicaid Services (CMS) to do what the NIH refused to do. But rather than pressing for a march-in, which can be a lengthy process, the groups are pushing for CMS to use its rights under two other statutory provisions to allow Xtandi (enzalutamide) generics to launch in the U.S. before Astellas Pharma Inc.’s three remaining patents for the prostate cancer drug expire in 2026 and 2027. First approved in 2012, Xtandi has market exclusivity until November 2026 for its newest indication, in nonmetastatic castration-sensitive prostate cancer, which was approved about five months ago.

FDA: Would a new endpoint for accelerated approvals in MM work?

The U.S. FDA thinks using minimal residual disease (MRD) as an endpoint for accelerated approval in new therapies to treat multiple myeloma (MM) might just be a good idea. The FDA now wants to know what its Oncologic Drugs Advisory Committee thinks about it, so the agency has convened a meeting of the committee for a deep dive April 12. Typically, overall response rate has supported accelerated approval of new MM therapies. The FDA said the positive impact of MRD on long-term outcomes recently has increased its interest in adding the new endpoint to MM studies. No specific products will be presented or discussed during the adcom meeting.

Newco news: Series A readies Rxcell's iPSC-derived photoreceptors for clinic

On the heels of a $4.6 million series A round in December 2023, cell therapy company Rxcell Inc. is planning to raise another $15 million in 2024 to take its iPSC-derived photoreceptors to the clinic for retinitis pigmentosa and other degenerative diseases of the retina. “We are making retinal progenitor cells, and those are going to be committed to photoreceptors,” Rxcell CEO Xianmin Zeng told BioWorld. The therapy could be useful for a range of retinal degenerative diseases, but for the first clinical trial – that will run in both the U.S. and Singapore – Rxcell will focus on retinitis pigmentosa, a hereditary disease that causes blindness and for which there is no effective treatment.

AACR 2024: New concepts suggest new targets for metastatic disease

Prior to this year’s Annual Meeting of the American Association for Cancer Research (AACR), it had been 14 years since metastasis had been the subject of a plenary session. So, the Tuesday session on “Evolution of the genome, microenvironment, and host through metastasis” had plenty of new insights to share. At the time of the last plenary, moderator Cyrus Ghajar told the audience, metastasis was conceptualized as “a linear progression of obligate steps” leading to tumor cells moving from their primary site and colonizing a distant organ. The concept of metastasis has changed a lot over the last 14 years, and at the plenary, speakers described several of those changes. One of those changes, Ghajar said, is that it is important “not just what the tumor cells are doing to the organ, it’s what they are doing to the entire host.”

Anvisa pilot program to spur biopharma innovation in Brazil

Anvisa launched a pilot program to help Brazilian startups navigate the regulatory path from the initial phases of product development. In addition to providing regulatory support, the goal of the program is to accelerate the process of biopharma innovation in the country, while gathering lessons that will help improve Anvisa's guidance and support strategies for drug innovations. Anvisa plans to select three startups for the pilot – one each developing an herbal drug, a synthetic drug and a biologic.

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