PTC Therapeutics Inc. scored a deal with Novartis AG for its Huntington’s disease program, banking $1 billion in cash up front along with as much as $1.9 billion in development, regulatory and sales milestone rewards. Warren, N.J.-based PTC will share profits in the U.S. with Novartis, of Basel, Switzerland. The arrangement also brings tiered double-digit royalties on ex-U.S. net sales. Novartis will assume global development, manufacturing and commercial responsibilities following completion of the placebo-controlled portion of the ongoing phase II Pivot-HD study. Shares of PTC (NASDAQ:PTCT) were trading at $50, up $6.12, or almost 14%.

FDA rejects Applied Therapeutics’ govorestat for galactosemia

Following a late-cycle review meeting with the U.S. FDA in September, expectations were high heading toward the PDUFA date for Applied Therapeutics Inc.’s priority NDA for govorestat in galactosemia. So the complete response letter issued by the agency, citing deficiencies in the clinical application, caught everyone off guard. The news, announced just after market close Nov. 27, ahead of the Thanksgiving holiday in the U.S., sent shares (NASDAQ:APLT) falling 76% Friday, Nov. 29. The stock opened Dec. 2 down another 10%.

China approves Kelun-Biotech’s TROP2 ADC for breast cancer

Sichuan Kelun-Biotech Biopharmaceutical Co. Ltd. won local approval of the first China-made trophoblast cell-surface antigen 2 (TROP2)-targeted antibody-drug conjugate (ADC), sacituzumab tirumotecan (sac-TMT), as a third-line therapy for advanced breast cancer. China’s National Medical Products Administration cleared Kelun-Biotech’s NDA of sac-TMT on Nov. 27 to treat unresectable locally advanced or metastatic triple-negative breast cancer in adult patients who received at least two prior systemic therapies, with at least one in an advanced or metastatic setting. Formerly known as SKB-264 and MK-2870, sac-TMT was expected to become the first domestically developed TROP2 ADC to be approved for China since Kelun-Biotech posted positive phase III Optitrop-Breast01 study findings in August 2023, as previously reported by BioWorld.

Jiuyuan Gene raises HKD$485M in Hong Kong IPO

Hangzhou Jiuyuan Gene Engineering Co. Ltd. raised HKD$485.4 million (US$62.38 million) in a November 28 IPO on the main board of the Hong Kong Stock Exchange (HKEX:2566). The IPO consisted of 45.4 million shares priced at HKD$12.42 per share. Jiuyuan Gene Engineering applied to list on the Hong Kong exchange in July 2024, after its first filing on Jan. 22, 2024. Founded in 1993, Jiuyuan is headquartered in China’s Zhejiang Province and is developing therapies across four main therapy areas: orthopedics, metabolic diseases, oncology, and hematology. Funds raised from the IPO will be used to deepen the company’s biopharma pipeline and expand its commercial abilities. The funds will also allow Jiuyuan to explore international markets.

Biomarker could aid in psychosis diagnosis

Researchers at the University of Rochester have described a neuroimaging-based biomarker that could identify individuals with early psychosis, and improved their identification when it was added to a standard neurocognitive diagnostic test. In a group of roughly 160 participants in the Human Connectome Early Psychosis Project, individuals who were in the early stages of psychosis had stronger connections from the thalamus (a midbrain sensory processing area) to the cortex, but weaker connections between different cortical areas, than controls. Combining the two differences into a single score resulted in a single “somato-visual” biomarker that was broadly useful within psychoses and at least somewhat specific to them. It applied equally to affective psychoses, where psychosis is part of major depression or bipolar depression, and nonaffective psychoses. But it was not present in a control group with attention deficit/hyperactivity disorder.

Healing the health divide: BioWorld reports on women’s health space

It’s difficult to fathom that the health of half the world’s population is underserved. But it’s a hard truth. Women’s health has traditionally focused on what Seema Kumar, the CEO of Cure, coined as the “bikini zone.” However, reproduction and breast health are a drop in the bucket when it comes to women’s health. In this multipart, in-depth series, BioWorld provides insight into the oft-overlooked space of women’s health, exposing the disparities in research and funding while looking toward future opportunities for bridging the gender gap in health care.

BioWorld Insider podcast: Healing the health divide for women

As a new BioWorld investigative report shows, decades of research excluding women from clinical trials and investment decisions made in male-dominated boardrooms have led to half the world’s health needs being underserved. Of the drug development companies working on women's health solutions, the proportion of funding and partnering for the sector is quite small. But it’s slowly growing, as is the depth of science. Karen Carey, BioWorld’s managing editor and chief analyst, and the managing editor of Bioworld Science, Anette Breindl, join this episode of the BioWorld Insider podcast to talk about the business and the science of women’s health.

Holiday notice

BioWorld's offices were closed in observance of Thanksgiving in the U.S. No issues were published Thursday, Nov. 28, or Friday, Nov. 29.

Also in the news

Acadia, Adcendo, Advanz, Agenus, Alector, Anavex, AOP Health, Beigene, Biogene, Cervomed, Duality, Empyrean, Exelixis, Fate, GSK, Idorsia, Kelun, Kronos, Lin, Novartis, Oncopeptides, Outlook, Preveceutical, PTC, Saniona, Satsuma, Secretome, Senti, Transcode, Tuhura, Valneva