Alnylam Submits NDA for RNAi Drug Lumasiran to FDA; Lundbeck Launches Vyepti for Migraine Prevention in the U.S.

NeuFores Completes RMB 130 Million Series A Financing to Advance Industrialization of Ophthalmic Gene Therapy Drugs

On April 8, Wuhan NewFores Biotech announced the completion of its RMB 130 million Series A financing round, led by Sequoia Capital China and Fosun Star Future Capital, with Northern Light Venture Capital as a participating investor. Currently, NewFores is building two R&D platforms: an R&D platform for gene therapy targeting inherited eye diseases, and a GMP manufacturing platform for gene therapy drugs. Professor Li Bin, the company’s founder, revealed that the company plans to establish a translational center for ophthalmic gene therapy to serve as an R&D base for animal and cell experiments related to drug development for eye diseases. Meanwhile, the company will also develop additional product lines in ophthalmology.

CR Double-Crane Amlodipine Besylate Tablets Receive Approval for Supplementary Drug Application

On April 8, China Resources Double-Crane Pharmaceutical issued an evening announcement stating that its wholly-owned subsidiary, China Resources Saike, had received the "Drug Supplemental Application Approval" from the National Medical Products Administration (NMPA) for Amlodipine Besylate Tablets (2.5 mg), approving the addition of the 2.5 mg strength. Amlodipine Besylate Tablets are primarily indicated for the treatment of hypertension and coronary heart disease. The approval of this supplemental application further expands the available strengths of Amlodipine Besylate Tablets, which will help unlock the product’s potential for future market sales and competitive advantage.

Xingqi Eye Medicine’s Marketing Application for Compound Ipratropium Bromide Solution for Inhalation Accepted by the CDE

On April 8, the marketing application for Inhalation Compound Ipratropium Bromide Solution, submitted by Shenyang Xingqi Eye Pharmaceutical and Sichuan Haimeng Zhisen Biotechnology as a generic drug under Category 4, was accepted by the Center for Drug Evaluation (CDE). Compound Ipratropium Bromide, administered as a combination of ipratropium bromide and salbutamol sulfate, is indicated for patients requiring combined bronchodilator therapy to treat reversible bronchospasm associated with obstructive airway diseases. It demonstrates superior efficacy compared to monotherapy and is used clinically for patients with moderate-to-severe asthma and chronic obstructive pulmonary disease (COPD).

Shanghai Pharmaceuticals’ Ambroxol Hydrochloride Capsules and Captopril Tablets Pass Consistency Evaluation

On April 8, Shanghai Pharmaceuticals announced that its wholly-owned subsidiary, Sine Tianping, and its controlled subsidiary, Changzhou Pharmaceutical, had respectively received the "Drug Supplemental Application Approval" issued by the National Medical Products Administration (NMPA) for Ambroxol Hydrochloride Capsules and Captopril Tablets. The aforementioned drugs have passed the consistency evaluation for generic drugs. Ambroxol Hydrochloride Capsules are primarily indicated for acute and chronic respiratory diseases, such as acute and chronic bronchitis, bronchial asthma, bronchiectasis, and pulmonary tuberculosis, which cause viscous sputum and difficulty in expectoration; Captopril Tablets are mainly used to treat hypertension and heart failure.

Health Yuan’s Tinidazole Tablets Pass the Generic Drug Consistency Evaluation

On the evening of April 7, Joincare Pharmaceutical Group Industry Co., Ltd. announced that its subsidiary, Livzon Pharmaceutical Group Inc., through its wholly-owned subsidiary Livzon Pharmaceutical Factory, had received the “Approval for Supplemental Drug Application” issued by the National Medical Products Administration (NMPA). The Tinidazole Tablets manufactured by Livzon Pharmaceutical Factory have passed the Consistency Evaluation of Quality and Efficacy for Generic Drugs. Indications for Tinidazole Tablets include: treatment of trichomoniasis of the urogenital tract in both men and women; infections caused by susceptible anaerobic bacteria, such as respiratory tract infections (e.g., pneumonia, lung abscess), intra-abdominal infections, gynecological infections (e.g., endometritis, tubal abscess), and oral infections (e.g., periodontitis, pericoronitis).

Conba's Parecoxib Sodium for Injection Approved

Recently, Zhejiang Jinhua Conba, a subsidiary of Conba Pharmaceutical Co., Ltd., received the Drug Registration Approval issued by the China Food and Drug Administration (CFDA) for Parecoxib Sodium for Injection. The active pharmaceutical ingredient (API) of parecoxib sodium and Parecoxib Sodium for Injection are products jointly developed by Jinhua Conba and Nanjing Huawei Pharmaceutical Technology Development Co., Ltd., indicated for the short-term treatment of postoperative pain.

Conba's Finasteride Tablets Pass the Generic Drug Consistency Evaluation

Recently, Hangzhou Conba Pharmaceutical, a wholly-owned subsidiary of Conba, received the "Drug Supplemental Application Approval" issued by the National Medical Products Administration (NMPA) for its Finasteride Tablets, indicating that the drug has passed the consistency evaluation for generic drugs. This medication is indicated for the treatment and control of benign prostatic hyperplasia (BPH), as well as for preventing and reducing the risk of acute urinary retention. It is suitable for the treatment of patients with prostatic hypertrophy, helping to reduce prostate size, improve urine flow, and alleviate symptoms associated with benign prostatic hyperplasia.

Lundbeck Announces U.S. Launch of Migraine Drug Vyepti

Recently, Lundbeck announced that its migraine medication Vyepti (eptinezumab) has been launched in the United States. The drug received approval from the U.S. FDA on February 21 this year for the preventive treatment of migraine in adults. Vyepti is administered via intravenous infusion, with a recommended dosage of 100 mg once every quarter (three months); some patients may benefit from a 300 mg dose. Notably, Vyepti is the first and only intravenous (IV) therapy indicated for migraine prevention, offering an effective and generally well-tolerated treatment option requiring only four IV infusions per year.

Biopharmaceutical Company Alnylam Completes Submission of Marketing Application for Rare Disease RNAi Drug Lumasiran to the FDA

Recently, biopharmaceutical company Alnylam announced that it has completed the rolling submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for lumasiran, an investigational RNAi therapeutic for the treatment of primary hyperoxaluria type 1 (PH1). Rolling review allows pharmaceutical companies to submit completed sections of their NDA or Biologics License Application (BLA) to the FDA for review as they become available, rather than waiting until all sections are complete before submitting the entire application. In the United States, lumasiran had previously been granted by the FDA Pediatric Rare Disease Priority Review Voucher eligibility, Orphan Drug Designation (ODD), and Breakthrough Therapy Designation (BTD) for the treatment of PH1.

Sanofi and Regeneron Announce Success of Phase III Clinical Trial of Dupixent for Treating Severe Atopic Dermatitis in Children Aged 6–11 Years

Recently, Sanofi and its partner Regeneron announced the results of a pivotal Phase III pediatric clinical trial (NCT03345914) evaluating the anti-inflammatory drug Dupixent (dupilumab) for the treatment of uncontrolled severe atopic dermatitis (AD) in adolescents aged 6–11 years, at the 2020 Revolutionizing Atopic Dermatitis (RAD) virtual conference. The data demonstrated that Dupixent, in combination with standard-of-care topical corticosteroids (TCS), significantly improved disease symptoms and health-related quality of life. Based on these findings, Dupixent is the first biologic agent to show positive outcomes in this pediatric population (aged 6–11 years) with AD.

FDA Approves Ridgeback’s New Drug EIDD-2801 for Human Clinical Trials in COVID-19

Recently, biotechnology company Ridgeback announced that the U.S. FDA has approved the Investigational New Drug (IND) application for the oral antiviral compound EIDD-2801 for the treatment of COVID-19. This action by the FDA enables Ridgeback to initiate human clinical trials of EIDD-2801 in the United States.

Pharmaceutical Company Can-Fite’s Innovative Therapy Namodenoson Meets Phase 2 Clinical Trial Endpoints

Recently, biopharmaceutical company Can-Fite BioPharma announced that namodenoson, its A3 adenosine receptor agonist, achieved the primary endpoint in a Phase 2 clinical trial involving patients with non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH), demonstrating dose-dependent reductions in serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels.

Biopharmaceutical Company Alnylam Submits Marketing Application for Rare Disease RNAi Drug Lumasiran to EMA

Recently, biopharmaceutical company Alnylam announced that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for lumasiran for the treatment of primary hyperoxaluria type 1 (PH1). In the European Union, lumasiran had previously been granted Orphan Drug Designation (ODD) and PRIority MEdicines (PRIME) status. Furthermore, lumasiran has been granted accelerated assessment by the EMA. Accelerated assessment is awarded to medicines considered to be of major public health interest and therapeutic innovation. Once the MAA is submitted and validated, accelerated assessment can shorten the review timeline from 210 days to 150 days.

Pharmaceutical Company Millendo’s Phase IIb ZEPHYR Study of New Rare Disease Drug Fails

On April 6, biopharmaceutical company Millendo Therapeutics announced that it would terminate the development of its non-acylated ghrelin analog livoletide for the treatment of Prader-Willi syndrome due to the failure of the Phase IIb ZEPHYR study. Millendo stated that following the discontinuation of livoletide, the company will focus on the development of nevanimibe for congenital adrenal hyperplasia and MLE-301 for vasomotor symptoms.

Biotech Firm Kazia Announces Positive Interim Data from Phase II Study of Paxalisib

Recently, biotechnology company Kazia Therapeutics announced positive interim data from its ongoing Phase II study (NCT03522298) evaluating paxalisib (formerly GDC-0084) for the treatment of glioblastoma multiforme (GBM). The interim analysis results showed that Part A (dose-escalation cohort) demonstrated a median overall survival (OS) of 17.7 months, representing a clinically meaningful extension in survival compared to the 12.7 months associated with the current standard of care, temozolomide (TMZ). The patient who received treatment for the longest duration remained progression-free 19 months after diagnosis.

Ascentage Pharma’s Bcl-2 Inhibitor APG-2575 Approved for Phase Ib/II Clinical Trial

On April 7, Ascentage Pharma announced that its Bcl-2 selective inhibitor, APG-2575, had received clinical trial approval. The company plans to conduct Phase Ib/II clinical studies in China to evaluate APG-2575 as monotherapy and in combination regimens for the treatment of relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). This open-label Phase Ib/II dose-exploration study aims to assess the safety and tolerability of APG-2575, both as monotherapy and in combination therapy, in patients with relapsed/refractory CLL/SLL, and to preliminarily evaluate its efficacy.

FDA Approves Hope’s Autologous Adipose-Derived Mesenchymal Stem Cell Therapy for Phase II Clinical Trials

Recently, biotechnology company Hope Biosciences announced that the FDA has approved a Phase II clinical trial to evaluate the safety and efficacy of autologous adipose-derived mesenchymal stem cells (HB-adMSCs) in providing immune support for individuals at high risk of COVID-19. In a Phase I/II clinical trial targeting rheumatoid arthritis, results demonstrated that HB-adMSCs were safe and effective in reducing systemic inflammation. Since inflammation drives disease progression in COVID-19 patients, early modulation of the immune system is crucial.

GenScript and Eutilex Enter into Exclusive Strategic Partnership

Recently, GenScript ProBio, the CDMO arm of Nanjing GenScript Biotech, and Eutilex, a South Korean biotechnology company dedicated to developing anti-tumor immunotherapy technologies, jointly announced a strategic collaboration on process development and manufacturing of plasmids and viruses for multiple CAR-T projects. This partnership aims to achieve mutual benefits and accelerate advancements in the global immunotherapy sector. Under the agreement, GenScript’s high-quality, one-stop plasmid and viral vector platform will be leveraged to provide plasmid and viral process development and manufacturing services for Eutilex’s multiple CAR-T programs.

Fosun Pharma and Sinopharm Holdings Sign “Framework Agreement on Mutual Supply of Products/Services”

On April 7, Fosun Pharma announced that Shanghai Fosun Pharmaceutical (Group) Co., Ltd. had entered into a Framework Agreement on Mutual Supply of Products/Services with Sinopharm Group Co., Ltd. The announcement indicated that the Group’s business spans pharmaceutical manufacturing and R&D, medical devices and medical diagnostics, healthcare services, and pharmaceutical distribution and retail. As such, it maintains upstream and downstream relationships with Sinopharm Group, which is engaged in the circulation of pharmaceuticals, diagnostics, and medical devices, leading to inevitable business interactions—including purchases, sales, and services—in the course of daily operations. These routine related-party transactions are necessary and ongoing, and do not affect the Company’s independence.

Biopharmaceutical Company Sage Announces Restructuring Plan, to Lay Off 340 Employees Local Time

On April 7, U.S. biopharmaceutical company Sage Therapeutics announced a restructuring plan that includes laying off 340 employees, representing 53% of its total workforce. Sage stated that it would also reduce external expenditures, with these measures expected to save the company $170 million annually.

Teyi Pharmaceutical Plans Private Placement to Raise Up to RMB 953 Million for Expanding TCM Production Capacity

On the evening of April 7, Teyi Pharmaceutical released a plan for a non-public offering of shares, aiming to raise no more than RMB 953 million. The funds will be used for the expansion and technological upgrading of its modern Chinese medicine product line, the construction of a modern Chinese herbal decoction pieces production facility, the development of an R&D center and headquarters operational center, as well as to supplement working capital. Through this non-public offering, Teyi Pharmaceutical seeks to expand its business scale, address future growth bottlenecks, and achieve leapfrog development. The products covered by the investment projects funded through this offering mainly include Pudilan Anti-inflammatory Tablets, Xiqingguo Granules, Fengliao Changweikang Tablets, and Pifubing Xuedu Pills.

《EBioMedicine》: Study Reveals New Therapy for Rare Neurological Disease

Recently, a study from the University of Cincinnati has shown that a targeted therapy used to treat certain cancers, including glioblastoma, may also be beneficial for treating other neurological disorders. Published online in the journal EBioMedicine, the study demonstrates that a nanoparticle drug delivery agent called SapC-DOPS can deliver targeted therapy without harming healthy cells. This approach has been proven successful in treating other neurological conditions, such as Parkinson’s disease.

*Nature Communications*: Scientists Unveil New Mechanism of Acarbose Biosynthesis

Recently, Professor Bai Linquan’s team from the School of Life Sciences and Biotechnology at Shanghai Jiao Tong University and the State Key Laboratory of Microbial Metabolism conducted an in-depth analysis of the biosynthetic mechanism of the antidiabetic drug acarbose. By employing effective metabolic engineering strategies, they further increased acarbose production. The relevant research findings were recently published in Nature Communications. Experts stated that this study not only underscores the importance of shunt products in microbial drug synthesis and yield enhancement but also reveals the diverse crosstalk between primary and secondary metabolism, thereby providing more effective guidance for the high-yield engineering of microbial drugs.

*Nature Medicine*: Preoperative Immunotherapy Effective for Colon Cancer

Recently, researchers at the Netherlands Cancer Institute announced new findings from the NICHE study, an innovative phase II clinical trial. Medical oncologist Myriam Chalabi and her colleagues published the results in Nature Medicine on April 6. The study found that patients with colon cancer but no distant metastases could receive short-term immunotherapy while awaiting surgery, enabling their own immune cells to shrink or eliminate cancer cells within a very short period. Among patients with a specific subtype of colon cancer (MSI), 100% benefited from the treatment, while 25% of patients with MSS-type tumors showed benefit.

PNAS: Convalescent Plasma Can Be Used to Treat Severe COVID-19

A recent report published by Chinese scientists indicates that a single dose of antibodies extracted from the blood of patients who have recovered from COVID-19 appears to be life-saving, shorten the duration of symptoms, increase oxygen levels, and accelerate viral clearance in 10 critically ill patients infected with the novel coronavirus. These preliminary findings come from a “pilot study” published in PNAS. The authors of this new study stated that the initial research on convalescent plasma therapy demonstrates potential therapeutic efficacy and low risk in treating severe COVID-19 cases. A single dose of convalescent plasma containing high concentrations of neutralizing antibodies can rapidly reduce viral load and help improve clinical outcomes.

《Science》: Modifying anticancer drugs to achieve optimal tumor cell killing efficacy

In a study recently published in the journal *Science*, researchers from the University of Montreal reported key structural and biochemical differences among a class of anticancer drugs known as PARP inhibitors. These distinct differences are associated with their varying abilities to kill cancer cells, thereby resolving the long-standing puzzle regarding the disparate efficacy of PARP inhibitors. Furthermore, leveraging these new structural and biochemical insights, the researchers modified existing PARP inhibitors to enhance their potency in killing cancer cells.