This week, in terms of Drug Review, the marketing application of a first-in-class of HAISCO, HSK16149 capsule was submitted, and Fosun Kite's CD19 CAR-T autologous product for new indications was declared for marketing. In terms of R&D, several clinical trials achieved positive results, while Alpine Immune terminated two clinical trials of davoceticept due to the death of patients. In terms of Business, JW Therapeutics and 2seventybio conditionally agreed to establish a strategic alliance; In terms of the Listing, Cwbio, a molecular testing company, was listed on the STAR.
The news review of this week is from October 24 to 28, covering a total of 23 pieces of information in 4 sections, i.e., drug review, R&D, business and listing.
NMPA – Marketing
1. On October 25, the CDE official website revealed that the new indication of Axicabtagene Ciloleucel Injection from Fosun Kite was declared for marketing. It was intended for the treatment of adult large B-cell lymphoma (r/rLBCL) which failed to respond to first-line immunotherapy or recurred within 12 months after first-line immunotherapy. Axicabtagene Ciloleucel Injection is a targeted human CD19 autologous CAR-T cell therapy product that Fosun Kite imported Yescarta from Kite for technology transfer in China in early 2017 and was authorized to carry out localized production in China.
2. On October 25, the CDE official website revealed that a marketing application for Parsaclisib, the PI3Kδ inhibitor of Innovent, was submitted, and it was intended to be included in the priority review for use in adult patients with relapsed or refractory follicular lymphoma who had previously received at least two systemic treatments. Parsaclisib comes from Innovent's cooperation with Incyte in 2018.
3. On October 27, the CDE official website revealed that the marketing application of HAISCO's first-in-class HSK16149 Capsule was submitted, presumably for the treatment of diabetic peripheral neuralgia. HSK16149 is a novel voltage-gated calcium ion channel analgesic, belonging to me-better of pregabalin. Benefits from its high target selectivity, strong and long-acting analgesic effect, and small central side effects, it may have better efficacy in moderate-to-severe patients.
NMPA – Clinical Trials
4. On October 24, the CDE revealed that a clinical application for GQ1005 from GeneQuantum was approved and the clinical research for solid tumor treatment would be initiated in multiple sites in China. GQ1005 is a new generation of site-specific coupled ADC, which targets HER2 and is intended to be developed for the treatment of solid tumors such as breast cancer and gastric cancer with a low/medium expression of HER2.
5. On October 25, the CDE official website revealed that the clinical application of Abbisko's ABSK021 was approved, and it could enter the critical phase III clinical research for patients with giant cell tumors of the tendon sheath. ABSK021 is a brand-new oral, highly selective, and active small molecule inhibitor of CSF-1R researched and developed by Abbisko. Abbisko is developing this drug for the treatment of various types of tumors and non-tumor indications.
6. On October 26, the CDE official website revealed that a clinical application for Bebetter Med's BEBT-503 capsule was approved for the treatment of type 2 diabetes combined with non-alcoholic fatty liver disease. BEBT-503, a first-in-class, is an efficient and balanced pan-PPAR agonist that regulates key metabolic, inflammatory, and fibrotic pathways by efficiently activating three receptors, and is used for the treatment of diabetes combined with NAFLD and NASH.
7. On October 24, CDE’s official website revealed that a clinical trial application for UA021 capsule, a first-in-class drug of Usynova, had been submitted. UA021 is a highly effective, highly selective, and highly oral bioavailability inhibitor of TYK2 allosteric variants, intended to be developed for treating autoimmune diseases such as inflammatory bowel disease and psoriasis.
FDA – Marketing
8. On October 25, Johnson & Johnson announced that its BCMA×CD3 bispecific antibody Teclistamab (Tecvayli) was approved by FDA to be marketed in the United States for the treatment of relapsed/refractory multiple myeloma (R/RMM) which had received four lines or above treatment (including proteasome inhibitor, immunomodulator, and anti-CD38 monoclonal antibody).
FDA – Clinical Trials
9. On October 27, Simcere Pharmaceutical announced that SIM0237 had been approved for clinical use in the United States for the treatment of locally advanced unresectable or metastatic solid tumors. SIM0237 is a PD-L1/IL15v bifunctional fusion protein developed by Simcere Pharmaceutical using its protein engineering technology platform and a potential best-in-class antitumor drug with a wide treatment window obtained through mutation attenuation.
[Fast Track Designation]
10. On October 24, Acumen announced that the FDA granted ACU193 the Fast Track Designation for the treatment of early Alzheimer's disease (AD). ACU193 is an anti-Aβ oligomer (AβOs) monoclonal antibody with best-in-class potential for the treatment of AD. It helps to protect neuronal function by preventing AβO from binding to dendritic spines, thereby exerting the therapeutic effect.
[Rare Pediatric Disease Designation]
11. On October 24, Vitalgen Biomed announced that VGN-R08b was awarded the Rare Pediatric Disease Designation (RPDD) for the treatment of neuropathic Gaucher's disease by the FDA. VGN-R08b is a gene replacement therapy with AAV9 as the carrier. The functional human GBA1 gene is injected into the brain, and the replenished GBA1 replenishes the generated GCase into lysosomes and replaces the mutated endogenous GCase to restore its activity.
[Orphan Drug Designation]
12. On October 25th, OriCell Therapeutics announced that FDA had granted OriCAR-017 Orphan Drug Designation for the treatment of relapsed/refractory multiple myeloma. OriCAR-017 is a chimeric antigen receptor T cell targeted by autologous GPRC5D developed by OriCell Therapeutics. GPRC5D, a group 5 member D of the G protein-coupled receptor family C, has a high specificity in multiple myeloma (MM) cells and almost no expression in normal tissues.
Clinical trials Status
13. On October 24th, Alpine Immune announced that it had terminated two clinical trials of conditional CD28 costimulatory agent and dual PD-L1/CTLA-4 checkpoint inhibitor davoceticept due to the second patient's death. Davoceticept is a recombinant protein, which can activate the CD28 receptor when it binds to PD-L1, block the binding of PD-L1 to PD-1 and inhibit the binding of CD80/86 to CTLA-4.
14. On October 24, the Drug Clinical Trial Registration and Information Publicity Platform of CDE announced that Createrna Science and Technology started a phase II clinical trial to evaluate the efficacy and safety of QR052107B tablets in patients with refractory or unexplained chronic cough. QR052107B tablet is a new generation P2X3 antagonist, and it is a first-in-class drug under research developed by Createrna Science and Technology.
Clinical data release
15. On October 24, Novartis announced that the pivotal Phase III APPLY-PNH trial had met its two primary endpoints, showing that iptacopan was superior to anti-C5 therapies (eculizumab or ravulizumab) in adults with paroxysmal nocturnal hemoglobinuria (PNH) who still had residual anemia symptoms despite prior treatment with anti-C5s. Iptacopan is a first-in-class, oral inhibitor against complement bypass factor B which is under development by Novartis.
16. On October 24, Gilead announced that the real-world results data from the BICSTaR study and the five-year data from two Phase III studies (Study 1489 and Study 1490) of three-in-one compound medicine Biktarvy (BIC tegravir 50mg/emtricitabine 200mg/ tenofovir dipivoxil 25mg, BIC/FTC/TAF) were published. The results highlighted potent and durable efficacy and safety of Biktarvy as a generally well-tolerated and efficacious regimen regardless of prior treatment and comorbidity status in people with HIV.
17. On October 25, Gritstone Bio announced that its self-amplified mRNA (samRNA) COVID-19 vaccine showed good tolerance and immunogenicity in two Phase I CORAL Studies (CORAL-BOOST and CORAL-CEPI). The SamRNA vector is derived from a synthetic RNA molecule of a wild-type Venezuelan equine encephalitis virus (VEEV) replicon and can prolong the duration and amplitude of immunogen expression to achieve an effective and durable immune response.
18. On October 26, AstraZeneca announced that its Phase II SERENA-2 study of camizestrant in the treatment of locally advanced or metastatic breast cancer patients with estrogen receptor-positive (ER+) had met the primary endpoint. Camizestrant is a new oral SERD and selective ERα antagonist independently researched and developed by AstraZeneca. It has shown anticancer activity in a series of preclinical models (including ER activation mutation).
19. On October 26, ESSA Pharma announced the presentation of updated clinical data from the first two cohorts of phase I/II study of EPI-7386 in combination with enzalutamide in patients with metastatic castration-resistant prostate cancer (mCRPC). The results showed that the safety of combined medication was favorable, which was consistent with that of the second-generation antiandrogen drugs, and no dose-limited toxicity was observed. EPI-7386 is a highly-selective oral small molecule inhibitor of the N-terminal domain of the androgen receptor (ARNTD).
20. On October 27, Roche announced that faricimab (Vabysmo) had reached the primary endpoints in two global phases III clinical trials for branch retinal vein occlusion and central retinal vein occlusion, respectively. Faricimab is a bispecific antibody, which can simultaneously block two key pathogenic pathways —Ang-2 and vascular endothelial growth factor-A (VEGF-A).
21. On October 25th, Astellas Pharma and Taysha Gene Therapies announced that they had reached a strategic investment to support the advancement of Taysha’s adeno-associated virus (AAV) gene therapy for genetic diseases of the central nervous system (CNS). Under the terms of the agreement, Astellas Pharma will receive an exclusive option to obtain an exclusive license for TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy (GAN).
22. On October 27, JW Therapeutics announced that it had conditionally agreed to establish a strategic alliance with 2seventy bio to develop and commercialize cell therapy products for MAGE-A4 (including any mutation, fragment, variant, or derivative of engineering TCR combined with MAGE-A4) for tumor indications in Greater China, and it was possible to expand cooperation by including other projects and products.
23. On October 25, Cwbio Tech was listed on the Shanghai Stock Exchange in STAR, with an issue price of RMB 48.98 yuan/share and an issue quantity of 23,290,278 shares. Cwbio Tech's main business covers the whole industrial chain of molecular detection, including molecular detection enzyme raw materials (including reverse transcriptase, Taq enzyme, hot start Taq enzyme, high fidelity enzyme, isothermal amplification enzyme, etc.), nucleic acid preservation reagents, nucleic acid extraction and purification reagents, molecular diagnosis kits, and clinical molecular detection services.
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NMPA announced the emergency drug review and approval, and conditionally approved the first-in-class innovative drugs, Simnotrelvir Tablets/Ritonavir Tablets of Hainan Simcere Pharmaceutical and Renmindevir of Shanghai Wangshi Biological Technology.