Pharma Frontiers: Daily Digest of Global Pharmaceutical News – Oct 9

1.Biogen's new ALS drug Tofersen approved for market in China

On October 8th, the official website of the National Medical Products Administration (NMPA) displayed that Biogen's Tofersen injection has been approved for market in China. Tofersen, developed jointly by Biogen and Ionis Pharmaceuticals, is an antisense oligonucleotide drug used for the treatment of SOD1-ALS. It binds to the mRNA encoding SOD1, leading to its degradation by ribonuclease, thereby reducing the production of SOD1 protein. This drug is the first gene-targeted therapy for ALS and received accelerated approval by the FDA in April 2023 for the treatment of patients with Amyotrophic Lateral Sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1).

2.Livzon Pharmaceutical's Triptorelin Microspheres approved for new indication in China

On October 8th, the official website of the NMPA latest announced that the market application for a new indication of Livzon Pharmaceutical’s Triptorelin Acetate Microspheres for injection has been approved. According to previously disclosed information from Livzon Pharmaceutical, this product is a once-a-month intramuscular injection of a Gonadotropin-Releasing Hormone (GnRH) agonist, with the newly approved indication for the treatment of endometriosis. Triptorelin Acetate Microspheres, a high-end long-acting microsphere formulation developed by Livzon Pharmaceutical, was previously approved in May 2023 in China for patients requiring androgen deprivation therapy for prostate cancer. According to previous announcements by Livzon Pharmaceutical, compared to ordinary Triptorelin Acetate injections, these microspheres feature a longer onset period and less frequent dosing, which can reduce patients’ discomfort and medication burden, and improve medication tolerance and accessibility.

3.The world's first oral GLP1/GIP/IGF1/GCG receptor agonist announces clinical data

On October 7th, Biomed Industrie announced positive top-line results from a Phase I clinical trial for its once-daily quad-target receptor agonist NA-931, achieving a weight reduction of 6.4% or 5.1% relative to placebo over 28 days. At week 12, participants treated daily with 150mg NA-931 saw a weight reduction of 12.7%, or 10.4% relative to placebo. Significantly, NA-931 did not exhibit any notable gastrointestinal adverse events, nor muscle loss, making it a well-tolerated and promising option for weight management. NA-931 is the first global agonist targeting IGF-1, GLP-1, GIP, and GCG four major hormone receptors; the insulin-like growth factor 1 (IGF-1) plays a crucial role in energy metabolism and body composition regulation, thus achieving weight loss without muscle reduction. Furthermore, IGF-1 combined with GLP-1 and GIP has been proven effective in assisting dietary and exercise treatment for weight reduction in non-diabetic obese patients.

4.CSPC Pharmaceutical's CPO301 granted Fast Track designation by the U.S. FDA

On October 8th, CSPC Pharmaceutical announced that its EGFR ADC new drug, CPO301, had been granted Fast Track designation by the U.S. FDA for the treatment of patients with EGFR overexpressing recurrent or metastatic squamous NSCLC who have progressed following platinum-based chemotherapy and anti-PD-(L)1 therapy. This marks the second FDA Fast Track designation for CPO301; the first was for the treatment of metastatic EGFR-mutated NSCLC patients who are refractory/difficult to treat or have shown resistance to EGFR-targeted treatments (e.g., third-generation EGFR inhibitors, including osimertinib).

5.Ascentage Pharma's APG-2449 approved to initiate Phase III clinical trials

On October 8th, Ascentage Pharma announced that its first-class novel drug, APG-2449, received clinical trial approval from the National Medical Products Administration (NMPA) Drug Evaluation Center (CDE) to commence two registration Phase III clinical studies for patients suffering from ALK TKI-resistant or intolerant non-small cell lung cancer (NSCLC), or treatment-naïve patients with advanced or locally advanced ALK-positive NSCLC. Both studies are multicenter, open, randomized controlled, pivotal registration Phase III clinical studies. The first aims to evaluate the efficacy and safety of APG-2449 compared to platinum-based chemotherapy in advanced NSCLC patients resistant or intolerant to second-generation ALK TKIs; the second aims to evaluate the efficacy and safety of APG-2449 compared to Crizotinib in treatment-naïve ALK-positive advanced or locally advanced NSCLC patients. APG-2449 is an orally active small molecule FAK inhibitor independently developed by Ascentage Pharma, representing the third-generation ALK/ROS1 TKI, and is the first domestic FAK inhibitor approved by CDE for clinical trials.

6.Innovent Biologics Forms Commercialization Partnership with Aosaikang for Third-Generation EGFR-TKI

On October 8, Innovent Biologics announced a collaboration agreement with Aosaikang Pharmaceutical regarding the exclusive commercialization of Limertinib, a third-generation epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI). Under this agreement, Aosaikang Pharmaceutical will act as the holder of the marketing authorization, responsible for the commercial production and supply of the collaborative product and will pay Innovent Biologics a service fee for sales promotion. Innovent Biologics has obtained the exclusive rights to promote and sell the collaborative product in mainland China and will pay Aosaikang Pharmaceutical an initial payment, registration milestones, and sales milestone payments as stipulated in the agreement. Limertinib is an orally administered third-generation EGFR-TKI, featuring significant activity and originating from proprietary intellectual property as a new molecular entity. Currently, the New Drug Applications (NDA) for two indications of Limertinib tablets are under review by the National Medical Products Administration (NMPA): 1) the treatment of adults with locally advanced or metastatic non-small cell lung cancer with progression on or after EGFR-TKI therapy who have tested positive for either the acquired EGFR T790M mutation or primary EGFR T790M mutation; 2) first-line treatment of adults with locally advanced or metastatic non-small cell lung cancer harboring either EGFR exon 19 deletions (19DEL) or exon 21 substitution mutations (L858R).

7.City Therapeutics Completes $135 Million Series A Funding to Develop Next-Generation RNAi Therapeutics

On October 8, City Therapeutics announced its launch with a vision to lead the future of RNA interference (RNAi)-based therapeutics. The company aims to build a robust and sustainable product engine using next-generation engineering of small interfering RNA (siRNA), the "trigger" molecules that mediate RNAi, expanding the therapeutic range for RNAi-based drugs. City Therapeutics has completed its Series A financing round, led by ARCH Venture Partners and raising $135 million, with additional participation from leading life science investors including Fidelity, Invus, Slate Path Capital, Rock Springs Capital, Regeneron Ventures, AN Ventures, and other undisclosed investors.

8.Chia Tai Tianqing to Introduce YZY Biopharma’s Dual Antibody CD3/EpCAM

On October 8, Sino Biopharmaceutical announced that its subsidiary, Chia Tai Tianqing Pharmaceutical Group has signed an exclusive licensing and collaboration agreement with Wuhan YZY Biopharma to introduce the dual antibody new drug, M701 (CD3/EpCAM dual antibody). M701, currently in Phase III clinical trials, is intended for the treatment of malignant pleural effusion (MPE) and malignant ascites (MA) caused by cancer. M701 is the first domestically developed CD3/EpCAM bispecific antibody to enter clinical trials. Through this collaboration, Chia Tai Tianqing will have exclusive, sublicensable rights to develop, register, produce, and commercialize M701 in mainland China. Chia Tai Tianqing will make an initial payment of approximately 315 million yuan to YZY Biopharma based on research and development progress, along with research milestones, and up to 700 million yuan in sales milestone payments, plus a single to low double-digit percentage of annual net sales as tiered royalties.

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