Global Drug Approvals/R&D UpdatesGlobal New Drug ApprovalsAccording to the statistical analysis by PharmaDJ, during this statistical period (July 6, 2024 - July 12, 2024), there was 1 new drug approved for marketing globally (excluding China). Among them, 1 new indication was approved. Compared with the previous statistical period, there were 10 fewer new drug approvals in this period.On July 12, Ultomiris received MFDS approval for the treatment of anti-aquaporin-4 (AQP-4) antibody-positive NMOSD in adults aged 18 years and older. During the 73-week study period, Ultomiris reduced the risk of relapse by 98.6% compared to placebo, with no patients diagnosed with relapse. The proportion of patients with HAI worsening in the Ultomiris group was 3.4%, compared to 23.4% in the placebo group. The half-life of Ultomiris is approximately four times longer than that of Soliris, requiring dosing every eight weeks, which enhances the convenience of administration.New Drug Approvals Worldwide (Excluding China)
Global New Drug Application ProgressAccording to the statistical analysis by PharmaDJ, during this statistical period (July 6, 2024 - July 12, 2024), there were 2 new drug applications for marketing approval globally (excluding China). Among them, there were 2 NDA filings. Compared with the previous statistical period, there was a decrease of 2 NDA/BLA filings in this period.On July 9, Curium announced that it had submitted a 505(b)(2) New Drug Application for Lutetium Lu 177 Dotatate Injection. If approved by the FDA, the drug will be used to treat somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Curium's submission represents an important step in its strategy to become a key player in the promising field of nuclear medicine therapies.
According to the drugAccording to data statistics analysis, during this statistical period (2024.07.06-07.12), a total of 4 drugs worldwide (excluding China) received special regulatory designations. Among them, there were 2 biologics and 2 cell therapies. Compared with the previous statistical period, the number of drugs receiving special regulatory designations decreased by 1.
On July 12, Mabwell announced that its Nectin-4-targeting ADC innovative drug (9MW2821) had been granted Fast Track Designation (FTD) by the FDA for the treatment of locally advanced or metastatic Nectin-4-positive triple-negative breast cancer. After being injected into the body, 9MW2821 can bind to Nectin-4 on the surface of tumor cells and enter the cells, releasing cytotoxins directionally through enzymatic action to achieve precise tumor killing. In February, it was granted FTD by the FDA for the treatment of advanced, recurrent, or metastatic esophageal squamous cell carcinoma; in May, it received Orphan Drug Designation (ODD) and FTD for the treatment of esophageal cancer and recurrent or metastatic cervical cancer that had failed previous platinum-based chemotherapy.On July 15, Qrigincell Therapeutics announced that the FDA had granted Fast Track designation to the CAR-T cell injection (OriCAR-017) targeting GPRC5D for the treatment of relapsed/refractory multiple myeloma. OriCAR-017 demonstrated deep and durable responses in RRMM patients who had failed treatments with anti-CD38, proteasome inhibitors (PI), immunomodulatory drugs (IMID), and BCMA CAR-T therapy, while also showing a favorable safety profile. All 10 patients responded well to the treatment, achieving a 100% overall response rate, with all patients reaching a 100% minimal residual disease-negative status by day 28.Special Qualification Recognition
Global New Drug Development ProgressAccording to the statistical analysis by PharmaDJ, during this statistical period (July 6, 2024 - July 12, 2024), there were a total of 21 updates on the clinical R&D status of new drugs globally (excluding China), covering 10 fields including oncology, hematology and lymphatic diseases, genetic metabolic disorders, neurological diseases, and dermatological conditions.
Among them, the clinical progress updates in the field of oncology ranked first across all fields, with a total of 10 updates: five related to chemical drugs and five related to biologics in the oncology field.On July 7, Hycan Pharmaceuticals announced that the FDA had approved the Phase I/II clinical trial application for HP537 tablets for the treatment of hematological malignancies. HP537 is a small-molecule anti-tumor drug independently developed by Hycan Pharmaceuticals as a p300/CBP inhibitor. HP537 tablets achieve an anti-tumor effect by binding to the p300/CBP bromodomain (BRD), blocking the p300/CBP signaling pathway, and inhibiting the activity of the p300/CBP enzyme, thereby suppressing the growth of tumor cells.On July 9, CSPC Pharmaceutical Group announced that SYS6023 had received U.S. clinical trial approval. SYS6023 is an antibody-drug conjugate that was already approved for clinical trials in China this past March. The approved indication for this clinical trial is advanced solid tumors. SYS6023 can bind to specific receptors on the surface of tumor cells, enter the cells through endocytosis, and release toxins to kill tumor cells. Preclinical studies have shown that this product demonstrates promising anti-tumor effects across various cancers.Global New Drug Development Progress Details (Partial)
Global Pharmaceutical Transaction EventsThis statistical cycle（2024.07.05-07.12）There were a total of 46 pharmaceutical transaction events globally (including China), involving drug rights transfers, company acquisitions, and other transaction events.
Summary Table of Global Pharmaceutical Transaction Timings (Partial)
Drug Approval/Development Dynamics in ChinaNew Drug Approvals in ChinaAccording to the statistical analysis of PharmaDJ data, during this statistical period (July 6, 2024 - July 12, 2024), a total of 2 new drugs were approved for marketing by the NMPA in China. Among them, 1 was approved via NDA and 1 via BLA. Compared with the previous statistical period, there were 7 fewer new drug approvals by the NMPA in this cycle.
On July 8, Ascentage Pharma announced that Olverembatinib (trade name: Nulrex®) has been officially approved for marketing by the regulatory authorities in Macao. The indications include the treatment of adult patients with chronic myeloid leukemia (CML) in chronic phase (-CP) and accelerated phase (-AP) who are resistant to any tyrosine kinase inhibitor (TKI) and have the T315I mutation; as well as the treatment of adult patients with CML-CP who are resistant or intolerant to first- and second-generation TKIs. Nulrex® is an original Class 1 new drug developed by Ascentage Pharma. It is the first third-generation BCR-ABL inhibitor approved for marketing by the NMPA and is considered a best-in-class drug globally, showing significant efficacy against BCR-ABL and various BCR-ABL mutants, including the T315I mutation.
On July 11, Eisai and Biogen announced that the Department of Health of the Hong Kong Special Administrative Region Government of China had approved the humanized anti-soluble aggregated amyloid-beta (Aβ) monoclonal antibody lecanemab (brand name: Leqembi®) for the treatment of patients with Alzheimer's disease in the mild cognitive impairment or mild dementia stage. Lecanemab can selectively bind to soluble Aβ aggregates and insoluble Aβ aggregates, the main component of Aβ plaques in Alzheimer’s disease, thereby reducing Aβ fibrils and Aβ plaques in the brain.Approval Status of New Drugs in China (Partial)
Progress of Clinical Implied Consent for New Drugs in ChinaAccording to the data analysis by PharmaDJ, during this statistical period (July 6, 2024 - July 12, 2024), a total of 24 new drugs in China received clinical tacit approval, involving 30 application numbers. Among them, there were 8 chemical drugs, 14 therapeutic biological products, and 2 preventive biological products. Compared with the previous statistical period, the number of clinical tacit approvals decreased by 36 application numbers in this period.
Clinical Trial Approvals for New Drugs in China This Week (Partial)
Progress of New Drug Applications in ChinaAccording to the statistical analysis by PharmaDJ, during this statistical period (July 6, 2024 - July 12, 2024), there were 4 new drug marketing applications in China, involving 5 acceptance numbers. Among them, there were 2 chemical drugs and 2 therapeutic biological products. Compared with the previous statistical period, there were 3 fewer new drug marketing application acceptance numbers in this period.
New Drug Applications and Market Launches in China (Partial)
According to the statistical analysis by PharmaDJ, during this statistical period (July 6, 2024 - July 12, 2024), there were 58 new drug clinical trial applications in China, involving 95 acceptance numbers. Among them, there were 35 chemical drugs, 21 therapeutic biological products, and 2 preventive biological products. Compared with the previous statistical period, this period saw an increase of 45 clinical trial application acceptance numbers.
Clinical Trial Applications for New Drugs in China (Partial)
According to the data analysis by PharmaDJ, during this statistical period (July 6, 2024 - July 12, 2024), no drugs in China received special qualification designation from the NMPA.
Progress in New Drug Development in ChinaAccording to the statistical analysis by PharmaDJ, during this statistical period (July 6, 2024 - July 12, 2024), there were a total of 6 updates on the clinical R&D status of new drugs in China, covering four fields: oncology, pathological conditions, signs and symptoms, genetic and metabolic diseases, and cardiovascular diseases. Among them, there were 3 chemical drugs and 3 biologics.
On July 9, Hengrui released data from the multi-center, randomized, double-blind, double-dummy, positive-controlled Phase III clinical study (PROFIT) on the efficacy and safety of the innovative drug HR20013 for preventing nausea and vomiting induced by highly emetogenic chemotherapy. The results showed that, compared with Fosaprepitant + Palonosetron + Dexamethasone (FAPR+PALO+DEX), HR20013 + Dexamethasone (DEX) has the potential to improve the QoL of patients receiving highly emetogenic chemotherapy (HEC), especially during the delayed and super-delayed phases.Recently, Hengrui's innovative drug Camrelizumab for neoadjuvant treatment of rectal cancer in a Phase III study was published in the Annals of Oncology. The UNION study is the world's first Phase III study to evaluate short-course radiotherapy followed by sequential immunotherapy combined with chemotherapy for locally advanced rectal cancer. Compared with the CAPOX group, the pCR rate in the CAM+CAPOX group was significantly higher, reaching the primary endpoint. The pCR rate increased significantly from 15.3% in the CAPOX group to 39.8% in the CAM+CAPOX group. The CAM+CAPOX group demonstrated tolerable safety, and the neoadjuvant therapy did not affect the feasibility of surgery. The 3-year EFS and OS rates are still under follow-up.Progress in the Development of New Drugs in China (Partial)
Policy and Regulatory Updates in China's New Drug Development FieldNotice on Publicly Soliciting Opinions on the ICH Draft Guideline "M14: Pharmacoepidemiological Studies Using Real-World Data for Drug Safety Assessment: Planning and Design"
ICH Guideline "M14: Pharmacoepidemiological Studies Using Real-World Data for Drug Safety Assessment: Planning and Design" has now entered Stage 3 of the regional public consultation phase. According to ICH regulations, ICH's regulatory agency members are required to collect feedback on the draft of Stage 2b guidelines from their regions and submit it to ICH.
The English original and Chinese translation of the M14 draft guideline are attached. The public is now invited to provide comments on the content of the guideline and its Chinese translation.
Hot News in China's New Drug Development FieldHuahai Pharmaceutical: Stock Price and Performance Soar, What's the "Growth Secret"?
Since 2024, there have not been many pharmaceutical companies whose stock prices have risen against the trend, and Huahai Pharmaceutical is one of them.On the evening of July 4, Huahai Pharmaceutical announced: "In the first half of 2024, the net profit attributable to shareholders is expected to increase by approximately 28% to 38%," causing the stock price to hit the daily limit the next day. As of the closing on July 10, Huahai Pharmaceutical's year-to-date increase is nearly 23%, ranking among the top ten in the gainers list.It can be seen that the reversal of performance is the main reason for Huahai Pharmaceutical's counter-trend rise in stock price. This reversal of performance actually has traces to follow, and the 2023 financial report has already given us the answer.For more information, please read the original text.Phase IIb Failure Causes HilleVax, a Takeda Spin-off, to Plunge 88% in Stock PriceIn 2021, Takeda announced a collaboration with Frazier Healthcare Partners to establish the biopharmaceutical company HilleVax, which is responsible for the development and commercialization of Takeda's norovirus vaccine candidate, HIL-214.In 2022, HilleVax went public through an IPO and launched the Phase II clinical trial of HIL-214 for infants in the same year.On July 8, the failure of the NEST-IN1 study was announced — due to not meeting its primary and secondary efficacy endpoints, the company will halt further development of HIL-214 for infants and will continue to explore its potential for adults.Shiyao Roxadustat Approved for Marketing: How Far Has This Blue Ocean Track Progressed?On July 5, CSPC Zhongnuo Pharmaceutical announced that its developed Roxadustat Capsules received NMPA approval for marketing in China. This is the first generic Roxadustat Capsules to be approved for marketing in China.HIF-PH Inhibitor First Launched in ChinaRoxadustat, originally developed by FibroGen, is a small-molecule hypoxia-inducible factor-1 prolyl hydroxylase (HIF-PH) inhibitor. Its mechanism of action involves mimicking the physiological response to hypoxic conditions, activating the HIF pathway, promoting endogenous erythropoietin (EPO) production, improving iron absorption and mobilization, downregulating hepcidin, and promoting red blood cell production to correct anemia.Innovent Biologics' Hypertension siRNA Drug Granted Clinical Tacit ApprovalOn July 8, according to the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration, the small nucleic acid (siRNA) drug SGB-3908 (IBI3016) injection, co-developed by Innovent Biologics and Saint Biologics for the treatment of hypertension, has been approved for clinical trials. The indication is proposed for the treatment of essential hypertension.In December 2023, Innovent Biologics and Saint Biologics announced a strategic collaboration agreement. According to the cooperation agreement, both parties will jointly advance the development of SGB-3908 to a certain stage. Meanwhile, Innovent Biologics will obtain an exclusive option and may pay an exercise fee in the future to acquire exclusive rights for the development, production, and commercialization of SGB-3908 across different regions globally. After Innovent Biologics exercises the option, Saint Biologics will also be entitled to subsequent R&D milestone payments, sales milestone payments, and tiered royalties based on net sales post-commercialization.For more information, please read the original text.CAN103 by北海康成 Completes Last Patient's Final Visit in Phase II Clinical Trial for Gaucher Disease in ChinaOn July 8,北海康成 (CANbridge) announced that the last patient's final visit in the Phase II clinical trial of CAN103, a treatment for Gaucher disease (GD) developed in collaboration with药明生物 (WuXi Biologics, 2269.HK), has been completed. The Phase II trial, a randomized, double-blind, dose-comparison study, is designed to evaluate the efficacy, safety, and pharmacokinetics of CAN103 in treatment-naïve patients aged 12 years or older with Type I and Type III Gaucher disease.
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