Weekly Pharma News Review | PharmaSources.com (0912-0916)

This week, in China, Humanwell Healthcare's Class 1.2 traditional Chinese medicine was approved for marketing. Pearl Bio's Bozitinib enteric capsule was planned to be included for priority approval. While globally, the terlipressin injection became the world's first FDA-approved therapy to improve renal function in adult patients with hepatorenal syndrome (HRS). Besides, several drugs have obtained Fast Track Designation (FTD), and many innovative drugs achieved positive clinical results, despite the fact that there were also a few drug failures. The news reviewed this week is from September 12 to 16, covering a total of 26 pieces of information in 3 sections, i.e., drug review, R&D, and business.

Drug Review

NMPA – Marketing

[Approval]

1. On September 15, the NMPA official website revealed that Humanwell Healthcare's Desmodium styracifolium total flavonoids capsule, a class 1.2 traditional Chinese medicine, had been approved for marketing. Used for the treatment of ureteral calculi syndrome diagnosed as pent-up dampness-heat in traditional Chinese medicine, it is the world's first new drug of traditional Chinese medicine for the prevention and treatment of urolithiasis. The drug adopts macroporous resin adsorption technology for purification, extracting Desmodium styracifolium total flavonoids from the single Chinese herbal medicine of Desmodium styracifolium.

NMPA – Marketing

[Application]

2. On September 13, the CDE official website revealed that an application had been submitted for the marketing of Liporaxel (RMX3001), jointly developed by Haihe Biopharma and Daehwa Pharmaceutical of South Korea. The drug is intended for second-line treatment of patients with advanced gastric cancer. RMX3001 is an oral paclitaxel preparation developed by Daehwa Pharmaceutical based on its innovative lipid self-emulsifying drug delivery technology, as well as the world's first oral paclitaxel product successfully developed and approved for marketing.

NMPA – Clinical Trials

[Approval]

3. On September 13, the CDE official website revealed that Renocell Biotech's RY_SW01 cell injection had been approved for clinical use, intended for the treatment of active lupus nephritis. RY_SW01 cell injection is a mesenchymal stem cell drug and has been administered to the first patient in the investigator-initiated trial (IIT), with the preliminary results indicating good safety.

4. On September 14, the CDE official website revealed that Hengrui Medicine's HRS-6209 capsule had been approved for clinical use. HRS-6209 capsule is a selective cell cycle blocker, able to induce G0/G1 arrest of tumor cells, thereby having an anti-tumor effect, intended for the treatment of advanced malignant tumors. No similar products have currently been approved for marketing at home or abroad, and neither is their relevant sales data.

5. On September 16, the CDE official website revealed that the CAEL-101 injection declared by Alexion subordinate to AstraZeneca had been approved for clinical use, intended for the indication of amyloid light chain amyloidosis. CAEL-101 is a potential "first-in-class" fibroreactive monoclonal antibody, currently undergoing Phase III clinical trials overseas for the treatment of light chain amyloidosis.

6. On September 15, the CDE official website revealed that Idorsia Pharmaceuticals' selatogrel injection had been approved for clinical use and intended for the pre-admission treatment of acute myocardial infarction (AMI) in adult patients with a previous history. Selatogrel is a new-type, quick-acting, reversible P2Y12 receptor antagonist, which can be administered subcutaneously by patients themselves, currently undergoing Phase III clinical research overseas for the indications of acute myocardial infarction.

7. On September 15, the CDE official website revealed that Biogen's BIIB122 had been approved for clinical use, which is intended for the treatment of Parkinson's disease. BIIB122 is a potential "first-in-class" LRRK2 small molecule inhibitor, currently undergoing Phase III clinical trials overseas for the treatment of Parkinson's disease.

8. On September 13, the CDE official website revealed that I-Mab Biopharma's third stage registered clinical study of CD47 antibody lemzoparlimab combined with azacytidine in the treatment of newly diagnosed high-risk myelodysplastic syndrome (HR-MDS) had been approved. Lemzoparlimab can effectively target tumor cells and minimize adverse effects on red blood cells, tpreventingvent severe anemia.

[Application]

9. On September 15, the CDE official website revealed that the clinical trial application for the LY3819469 injection jointly developed by Eli Lilly and Dicerna Company had been submitted and processed. The injection, used for the treatment of cardiac metabolic diseases, is the first RNAi therapy drug declared by Eli Lilly in China. LY3819469 was approved by FDA for a clinical trial in May 2021.

10. On September 16, the CDE official website revealed that Jing Medicine’s clinical trial application for HJM-353 tablets had been submitted and processed. HJM-353 is a potent, selective, and orally bioavailable EED inhibitor, which is intended for the treatment of malignant and solid tumors of the hematological system.

[Priority Review]

11. On September 14, the CDE official website revealed that Bozitinib enteric capsule, a c-Met inhibitor created by Pearl Bio, subordinate to Avistone Pharmaceuticals, had been proposed to be given priority review for the treatment of adult patients with locally advanced or metastatic NSCLC with exon 14 skipping of interstitial-epithelial transformation factor (MET).

FDA - Marketing

[Approval]

12. On September 14, Mallinckrodt announced that the FDA had approved its terlipressin injection for the treatment of adult HRS, which became the world's first FDA-approved treatment to improve the renal function of adult HRS patients. Terlipressin, as a synthetic analogue of endogenous vasopressin secreted by human posterior pituitary glands, has the pharmacological effects of constricting blood vessels and resisting hemorrhage, enabling the reduction of liver blood flow and portal vein pressure.

[Application]

13. On September 12, Acadia Pharmaceuticals announced that the FDA had processed and granted priority review to Trofinetide's new drug application (NDA) for the treatment of Rett syndrome. The PDUFA date would be March 12, 2023. Trofinetide is a novel amino-terminal tripeptide analogue of insulin-like growth factor I (IGF-1). No drug for the treatment of Rett syndrome has ever been approved by the FDA so far.

FDA – Clinical Trials

[Approval]

14. On September 13, Luye Pharma announced that LY03015 had been approved by the FDA and would soon be under Phase I clinical trial. It is intended for the treatment of tardive dyskinesia and Huntington's disease. LY03015 is a new generation of VMAT2 inhibitors, expected to improve the clinical weaknesses of VMAT2 inhibitors in terms of activity, efficacy, and safety.

15. On September 15, Transcenta Group announced that its clinical trial application of TST003 had been approved by the FDA. TST003 is the world's first potential antibody candidate drug to target the new immunomodulatory protein Gremlin1 produced by tumor-associated fibroblasts or tumor cells with the mesenchymal phenotype.

[Fast Track Designation]

16 . On September 12, Mersana announced that its XMT-1660 had been granted FTD by the FDA for the treatment of adult patients with advanced or metastatic triple-negative breast cancer (TNBC). XMT-1660 is an ADC drug targeting B7-H4 developed by the Dolasynthen technology platform, with a drug-to-antibody ratio of 6 (DAR=6), currently undergoing Phase I clinical trial.

17. On September 14, aTyrPharma announced that its efzofitimod had been granted FTD by the FDA for the treatment of interstitial lung disease (ILD) related to systemic sclerosis (SSc). Efzofitimod is a fusion protein, formed by the fusion of the immunomodulatory domain of histidyl-tRNA synthase and the Fc region of a humanized antibody, which restores human immune balance by selectively regulating NRP2.

18. On September 15, Alphamab Oncology announced that its Envafolimab monoclonal antibody injection had been granted FTD by the FDA for the treatment of locally advanced, unresectable, or metastatic pleomorphic sarcoma (UPS)/fibrous histiocytic sarcoma (MFS) with disease progression after first-line/second-line chemotherapy. Envafolimab is the world's first and only subcutaneous PD-(L) 1 inhibitor available in the market, as well as the first approved immunotherapy drug for pan-tumor indications (MSI-H/dMMR) in China.

19. On September 15, Jasper Therapeutics announced that its JSP191 had been granted FTD by the FDA for the treatment of patients with severe combined immunodeficiency who received allogeneic hematopoietic stem cell transplantation. JSP191 is a humanized anti-CD117 monoclonal antibody under research, able to eliminate hematopoietic stem cells in bone marrow by blocking the signal transduction of stem cell factor receptors and making space for donors or transplanted stem cells after genetic correction.

R&D

Clinical trials launching

20. On September 14, MC2 Therapeutics announced that the first patients have been dosed in its Phase II clinical trial, MC2-25-C1, evaluating the safety and effectiveness of MC2-25 cream in patients with moderate to severe pruritus III-V related to chronic kidney disease (CKD-aP). MC2-25 cream is a first-in-class drug for treating CKD-aPIII-V patients. The active component of MC2-25 Cream is a dipeptide, which is formulated in MC2Therapeutics’ proprietary formulation and drug delivery system – PAD Technology.

Clinical data release

21. On September 12, Daiichi Sankyo and Sarah Cannon Research Institute published in a Proffered Paper session (Abstract # 453O) at the ESMO Conference that extended follow-up data from a Phase I/II trial of first-in-class drug DS-7300 in patients with several types of heavily pretreated cancers including lung, prostate, or esophageal cancer. The data indicated the drug's high remission rate for a variety of solid tumors. DS-7300 is an investigational B7-H3 directed ADC, designed using Daiichi Sankyo’s proprietary DXd ADC technology.

22. On September 14, Zelgen announced success in its multicenter, randomized, double-blind, placebo parallel controlled Phase II clinical trial (ZGJAK008) of the Jaktinib hydrochloride tablets for the treatment of active ankylosing spondylitis (AS). Jacktinib hydrochloride, a first-in-class new drug, is a new JAK inhibitor independently developed by Zelgen.

23. On September 14, Altimmune announced the results from its Phase Ib study of pemvidutide in patients with nonalcoholic fatty liver disease (NAFLD). At 12 weeks of the treatment, the primary endpoint of reductions in liver fat content (LFC) had been achieved in all 3 pemvidutide dosing groups (1.2 mg, 1.8 mg, 2.4 mg). At the 1.8 mg dose, pemvidutide achieved a mean reduction of the liver fat content of 68.5% relative to the baseline. Pemvidutide is a GLP-1/glucagon dual receptor (GEGR) agonist in development for the treatment of obesity and NASH.

24. On September 14, Eloxx Pharmaceuticals announced topline results from its Phase II clinical trial of ELX-02 in combination with ivacaftor in class 1 cystic fibrosis (CF) patients with at least one nonsense mutation. The combination therapy had been well tolerated but did not achieve statistical significance for efficacy endpoints, including changes from baseline in sweat chlorine concentration (SCC) or percent forced expiratory volume in the first second (FEV1).

25. On September 15, Revance Therapeutics announced the positive results of its Phase II JUNIPER clinical trial evaluating the long-term efficacy and safety of DaxibotulinumtoxinA-lanm injection for the treatment of upper limb spasm (ULS) in adults after stroke or traumatic brain injury. The injection is a new-generation neuromodulator containing stable peptide preparations and highly purified botulinum toxin type A.

Business

26. On September 15, NeuroBo and Dong-A jointly announced their conditional strategic cooperation agreement to develop and commercialize DA-1241 and DA-1726. As stipulated in the agreement, NeuroBo will be responsible for the global development, regulatory, and commercial activities of DA-1241 and DA-1726 other than for certain Asian-Pacific geographies. Dong-A, on the other hand, shall retain the rights to the development and commercialization of these two products in South Korea.