Week In Review: CANbridge In-Licenses Gene Therapy For Rare Disease From UMass

Deals and Financings

Beijing CANbridge Pharma (HK: 1228) exercised an option for global rights to a novel second-gen gene therapy aimed at spinal muscular atrophy (SMA) from UMass Chan medical school. A rare disease/rare oncology company, CANbridge will have rights to develop, manufacture and commercialize the therapy. The company supported the UMass research effort. In late 2022, data was presented from preclinical tests showing the new SMA gene therapy was more effective and caused less liver toxicity than the current therapy. SMA is caused by the lack of a functional motor survival motor neuron 1 (SMN1) gene that results in loss of motor function and death.

Shanghai Zhimeng Biopharma signed an agreement to out-license global rights for its TLR8 agonist to GSK plc (GSK) as an HBV treatment (see story). Zhimeng is currently conducting a US Phase I trial of CB06, and GSK will acquire the rights – including development, manufacturing and commercialization – if the candidate posts successful results. CB06 is a potential candidate for combination with GSK’s bepirovirsen, an antisense oligonucleotide that targets all HBV messenger RNAs to decrease virus load. GSK hopes to offer a virtual cure for HBV. The financial details of the agreement were not disclosed.

Shanghai JiXing Pharma bought global rights to a preclinical hypertension asset from PhaseBio Pharmaceuticals (OTC:PHASQ), a Philadelphia area company currently in bankruptcy. PB6440 is a next-gen selective aldosterone synthase inhibitor. In preclinical tests, the candidate inhibited aldosterone synthase but did not interfere with a homologous enzyme that is key to cortisol synthesis. JiXing brings novel drugs to China patients who have serious unmet medical needs. Terms of the agreement were not disclosed.

GenScript ProBio, a Nanjing biologics CDMO, partnered with London’s Bio Immunitas to develop novel therapies using Bio Immunitas’ human Recombinant Protein Platform (hRPP). Specifically, GenScript ProBio will fast-track Bio Immunitas’ manufacturing and production from bench to bedside, providing IND-enabling CMC services for preclinical development of two recombinant protein products. Bio Immunitas is developing a novel treatment that stabilizes the immune system, acting as an anti-viral, anti-inflammatory and immunomodulator with an anti-ageing capability that clears senescent cells.

Shanghai Asieris Pharma (SHA: 688176) formed a research collaboration with San Francisco’s ReviR Therapeutics to develop novel therapies for genitourinary (GU) tumors. The companies will use ReviR’s proprietary RNA-targeted technologies to discover and develop candidates for multiple oncology targets. ReviR combines computational and high throughput drug discovery technologies for drug discovery. Asieris will use ReviR’s innovative RNA modulation technologies, named BindeR and SpliceR, to identify and develop the candidates. A genitourinary cancer company, Asieris will have an option to license any candidates.

Government and Regulatory

China is planning to prohibit any export from biotech companies engaged in certain cutting-edge sectors – cell cloning and gene editing technology for human use, along with restrictions on the export of CRISPR gene editing technology and synthetic biology technology. Officially, the ban is in limbo during a public comment phase, though the edict will probably not change significantly. The proposed China export ban follows the 2022 US law that supported semiconductor chip manufacturing in the US and restricted export of the most powerful chips to China.

Trials and Approvals

Kintor (OTCPK:KNTPF; HK: 9939), a Suzhou biopharma developing androgen products, reported positive top line data from a US Phase I trial of a degrading androgen receptor candidate for androgenetic alopecia and acne (see story). Kintor’s GT20029 is an in-house developed and fully owned proteolysis targeting chimera (PROTAC) compound. The trial data showed GT20029 was safe, well-tolerated and had good pharmacokinetic characteristics in healthy subjects as well as subjects with androgenetic alopecia or acne. Kintor said GT20029 is the first topical PROTAC compound in the world to complete Phase I trials in both China and the US.

Beijing InnoCare (OTCPK:INCPF; HK: 09969; SHA: 688428) reported the American Journal of Hematology published very positive results from a Phase II trial of its BTK inhibitor orelabrutinib (see story). The China study enrolled 80 patients with relapsed/refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma. The overall response rate was 92.5% and the complete response was 21.3%. Orelabrutinib was approved in China for three lymphoma/leukemia indications in 2020. One year ago, InnoCare out-licensed the ex-China rights for use of the drug as a MS therapy to Biogen in a deal with potential value of $937 million.

Shenzhen ImmVira reported its first intratumoral injected oncolytic virus product, MVR-T3011 IT, was effective in early tests, raising Progression-Free Survival in melanoma patients to 12.9 months. The patients had previously failed another treatment. MVR-T3011 is a 3-in-1 oncolytic herpes simplex virus that aims to achieve the best profile of attenuated HSV-1 with high levels of replication in tumor cells, while causing only highly restricted replication in normal cells. The product includes two well-validated exogenous genes, PD-1 antibody and IL-12, to enhance the immune responses in the tumor microenvironment.

Jiaxing Technoderma Medicines has begun a US Phase I trial of a clinical stage topical ointment that is intended to treat Atopic Dermatitis. TDM-180935 is a small molecule JAK1/Tyk2 small molecule inhibitor that suppresses both keratinocyte- and T cell-derived pathogenic pathways, an important channel for Atopic Dermatitis. Technoderma believes the candidate may offer better efficacy and safety than existing topical treatments. The trial enrolled healthy male volunteers who will be administered ascending single and multiple doses of TDM-180935. Technoderma develops treatments for dermatological conditions.

Disclosure: None.

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