Pharma Frontiers: Daily Digest of Global Pharmaceutical News – Sep 24

1.Daiichi Sankyo/AstraZeneca TROP2 ADC Phase III study fails to meet primary OS endpoint

On September 23rd, AstraZeneca and Daiichi Sankyo jointly announced that the Phase III TROPION-Breast01 study of TROP2 ADC datopotamab deruxtecan (Dato-DXd) in second/third-line treatment for patients with HER2-low expressing or negative breast cancer did not meet the primary endpoint of overall survival (OS). This global, multicenter, randomized, open-label clinical trial enrolled 700 patients who had progressed after endocrine therapy or were unsuitable for endocrine therapy and had received at least one form of systemic treatment. The patients were either inoperable or had metastatic HR-positive, HER2-low or HER2-negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer. The study assessed the safety and efficacy of datopotamab deruxtecan compared to the investigator’s choice of single-agent chemotherapy (Eribulin, Capecitabine, Vinorelbine, or Gemcitabine). The primary endpoints were OS and progression-free survival (PFS).

The PFS primary endpoint was met in September 2023, showing that the PFS of the datopotamab deruxtecan group was significantly extended compared to the chemotherapy group (6.9 vs. 4.9 months, HR=0.63, P<0.0001), the results being statistically and clinically significant. Based on these findings, AstraZeneca submitted the second Biologics License Application (BLA) for datopotamab deruxtecan to the FDA. However, the newly presented results indicate that the OS data lacks statistical significance, adding uncertainty regarding the drug’s effectiveness. Nonetheless, AstraZeneca noted that the approval of multiple ADC products during the TROPION-Breast01 study might have influenced subsequent treatments for the trial participants, thereby potentially affecting the OS outcome.

2.Ji Xing Pharmaceuticals' Aficamten Proposes for Priority Review

On September 23rd, the CDE website disclosed that Ji Xing Pharmaceuticals has filed for Aficamten to be included in the priority review process for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Public information reveals that Aficamten, developed by Cytokinetics, is a new generation of cardiac myosin inhibitor. In July 2020, Ji Xing Pharmaceuticals entered into an exclusive collaboration with Cytokinetics to develop this drug in Greater China. In May of this year, Cytokinetics announced that Aficamten achieved both the primary and all secondary endpoints in the pivotal Phase III clinical trial SEQUOIA-HCM for patients with symptomatic oHCM, with detailed data published in the New England Journal of Medicine (NEJM). Cytokinetics previously announced plans to submit regulatory applications in the US and EU later this year.

3.Novartis’ Oral Medication Iptacopan to Potentially Enter Priority Review in China 

On September 23, according to the latest announcement on CDE's official website, Novartis' application for a new indication of Iptacopan hydrochloride capsules to treat adult patients with C3 glomerulopathy (C3G) is proposed to be included in priority review. Iptacopan (Iptacopan, LNP023) is an orally administered specific inhibitor of complement factor B. For this C3G indication, the product had previously been categorized as a breakthrough therapy by CDE. Globally, Iptacopan was approved by the FDA in December 2023 as the first oral monotherapy for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). On August 8 of this year, the FDA approved a new indication for Iptacopan to reduce proteinuria in adult patients with IgA nephropathy (IgAN) at risk of rapid disease progression. In China, Iptacopan was approved by NMPA in April this year for its first indication to treat adult patients with paroxysmal nocturnal hemoglobinuria (PNH). In August, the second indication for this product was accepted by CDE. This current submission marks the third application for Iptacopan in China, aiming to treat adult patients with C3 glomerulopathy (C3G). 

4.Allist Pharmaceuticals’ $180 Million Import! Class 1 New Lung Cancer Drug Approved for Clinical Trials

On September 23, as displayed on CDE's official website, Allist Pharmaceuticals’ AST2303 tablets (ABK3376 tablets) were approved for clinical trials, intended for use in adult patients with advanced non-small cell lung cancer (NSCLC) harboring the EGFR C797S mutation. Public information reveals that AST2303 is a highly selective, brain-penetrant, new-generation EGFR oral small molecule inhibitor, effectively targeting the C797S mutation which can develop resistance to third-generation EGFR-TKIs. In October 2023, Allist Pharmaceuticals and Abbisko Therapeutics entered into an approximately $187.9 million collaboration, securing the exclusive license to research, develop, manufacture, use, and sell this product in China (including Mainland China, Hong Kong, Macau, and Taiwan).

5.Antengene's Selinexor Officially Approved for Marketing in Thailand

On September 23, Antengene announced that the Thai Food and Drug Administration has approved the New Drug Application (NDA) for Selinexor (trade name: XPOVIO®). It is approved for use in two indications: (1) in combination with Bortezomib and Dexamethasone for the treatment of adult patients with multiple myeloma (MM) who have received at least one prior therapy; (2) in combination with Dexamethasone for the treatment of adult patients with MM who have received at least four prior therapies and are refractory to at least two proteasome inhibitors, two immunomodulatory agents, and an anti-CD38 monoclonal antibody drug, and who have shown disease progression on the last therapy. Selinexor is the first and only XPO1 inhibitor approved for marketing in Thailand. This medication has now been approved in nine Asia-Pacific countries and regions, and covered by medical insurance in Mainland China, Australia, Singapore, and Korea. Antengene has also submitted an NDA in Indonesia, with approval expected in the second half of this year.

6.Frontera Therapeutics' FT-002 Granted FDA Phase II Clinical Trial Permission

On September 21, Frontera Therapeutics' internally developed recombinant adeno-associated virus (rAAV) gene therapy product, FT-002 Injection, was granted permission by the U.S. Food and Drug Administration (FDA) to proceed with a Phase II clinical trial (IND). This IND approval marks FT-002 Injection as the first Chinese rAAV gene therapy drug to be granted FDA permission to directly conduct a Phase II clinical trial in the United States, representing a milestone in the overseas development of Chinese rAAV gene therapy for ophthalmology. FT-002 aims to treat X-linked Retinitis Pigmentosa (XLRP) caused by mutations in the RPGR gene.

7.$27 Million! Sanofi "Invests" in NLRP3 Inhibitors 

On September 23, biopharmaceutical company Ventyx Biosciences announced that Sanofi has agreed to make a strategic investment of $27 million in the company at a conversion price of $3.8243 per common share. The proceeds from the investment are expected to further strengthen the company’s cash position, ensuring operations until at least the second half of 2026. Additionally, Ventyx has agreed to grant Sanofi exclusive rights to negotiate for certain rights to the VTX3232 project. VTX3232 is an orally administered selective Central Nervous System-permeable NLRP3 inhibitor with potential therapeutic effects in treating various neuroinflammatory and neurodegenerative diseases, including Parkinson’s Disease, cardiac metabolic diseases, Alzheimer’s Disease, and Multiple Sclerosis. The NLRP3 targets the inflammatory inflammasome/IL-1β axis, disrupting the inflammatory feed-forward cycle in autoimmune and autoinflammatory diseases.

8.Vicebio Completes $100 Million Financing to Develop RSV/hMPV Vaccine 

On September 23, Vicebio announced the completion of a $100 million Series B financing round led by TCGX, with participation from Goldman Sachs Alternatives, Avoro Ventures, venBio, UniQuest, and founding investor Medicxi. Vicebio is a biopharmaceutical company developing novel vaccines against life-threatening respiratory viral infections, utilizing proprietary Molecular Clamp technology discovered at the University of Queensland. This innovative approach enables the production of easy-to-manufacture, potent vaccines to be delivered in ready-to-use prefilled syringes, suitable for a wide range of viruses including Respiratory Syncytial Virus (RSV), Human Metapneumovirus (hMPV), parainfluenza, influenza, and coronaviruses. Vicebio was incubated by Medicxi in 2019, with Dr. Emmanuel Hanon, former head of vaccine R&D at GlaxoSmithKline, serving as CEO. The rights to the Molecular Clamp technology were licensed through the commercialization arm of the University of Queensland, UniQuest.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!