Weekly Pharmaceutical News Roundup (Nov 11–15, 2024): Clinical Approvals, Global Collaborations, and Pipeline Highlights

In China

1. CSPC's Bivalent Human Papillomavirus mRNA Therapeutic Vaccine Approved for Clinical Trials

On November 12, CSPC announced that its bivalent messenger RNA (mRNA) vaccine SYS6026 had been approved by the National Medical Products Administration to conduct clinical trials in China. This product is CSPC's first self-developed therapeutic vaccine, consisting of an mRNA molecule combined with a lipid nanoparticle delivery system. By expressing the E6 and E7 antigens of human papillomavirus types 16 or 18, it induces specific T-cell immune responses to eliminate infected cells and block carcinogenesis. Current clinical treatments are mainly surgical, which have the disadvantages of significant trauma and high recurrence rates. As a non-surgical treatment option, this product is expected to meet a significant clinical need. The approved indication for this trial is the treatment of high-grade squamous intraepithelial lesions associated with human papillomavirus types 16 or 18. Currently, there are no HPV therapeutic vaccines available on the global market.

2. Hengrui Pharma's Innovative Drug Combination Therapy Approved for Clinical Trials

Recently, Hengrui Pharma's innovative drug HRS-2189 tablets for combination therapy in advanced solid tumors has been approved for clinical trials by the National Medical Products Administration. This combination therapy covers multiple drugs, including fulvestrant, HRS-6209, HRS-5041, etc. These drugs have their own characteristics in anti-tumor effects; for instance, HRS-5041 is a novel AR-PROTAC small molecule, and HRS-1358 is a novel PROTAC molecule targeting estrogen receptor degradation. This study will explore the safety and efficacy of various anti-tumor therapies for patients with advanced unresectable or metastatic solid tumors, offering the potential to provide more treatment options for cancer patients in China. Hengrui Pharma has always been committed to innovative drug research and development, making this breakthrough highly anticipated.

III. Lunan Pharmaceutical's Macrodor® Edoxaban Tosylate Tablets New Specification Approved for Marketing

Recently, MaiLuoda® Edoxaban Tosylate Tablets (15mg, 30mg specifications) from Shandong New Era Pharmaceutical Co., Ltd., a subsidiary of Lunan Pharmaceutical Group, received approval from the National Medical Products Administration for marketing in China and were deemed to have passed the generic drug quality and efficacy consistency evaluation. Edoxaban is a selective factor Xa inhibitor used for the prevention of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation who have risk factors, as well as for the treatment and prevention of deep vein thrombosis and pulmonary embolism in adults. In November 2023, its 60mg specification was approved. The addition of these new specifications is based on insights into market demand and concern for patient health, providing more options for clinical treatment and meeting the personalized needs of different patients.

Overseas

I. Bristol-Myers Squibb Showcases Cardiovascular Product Strength at the 2024 American Heart Association Scientific Sessions

Recently, Bristol Myers Squibb announced that it will present data from its cardiovascular portfolio at the American Heart Association Scientific Sessions, to be held in Chicago from November 16 to 18. The presentations will include nearly two-year REMS program evaluation results for CAMZYOS (mavacamten), real-world data, and long-term extension data, among others. CAMZYOS is the first approved cardiac myosin inhibitor, indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy. Additionally, data related to ELIQUIS (apixaban) and milvexian will also be showcased. A senior vice president of Bristol Myers Squibb stated that these presentations underscore the company’s commitment to delivering transformative therapies to improve clinical outcomes for patients worldwide.

2. AbbVie's Emraclidine Fails in Phase 2 Trial for Schizophrenia

On November 11 local time, AbbVie announced that its two Phase 2 EMPOWER trials studying emraclidine for the treatment of schizophrenia did not meet the primary endpoint. However, the drug was well-tolerated, with an adverse event profile consistent with the Phase 1b trial. AbbVie's Executive Vice President of Research and Development expressed disappointment but stated that they would analyze the data to determine the next steps. Neuroscience is a key focus area for AbbVie, which has expanded its neuroscience pipeline through the acquisition of Cerevel. Schizophrenia is a serious and complex mental health disorder, and emraclidine is a potential novel M4 selective positive allosteric modulator aimed at treating schizophrenia and Alzheimer's disease psychosis. The EMPOWER clinical development program also includes an open-label extension trial. AbbVie remains committed to providing innovative therapies for patients with neurological and psychiatric disorders.

3. AstraZeneca and Daiichi Sankyo Submit New BLA for Accelerated Approval of Datopotamab Deruxtecan in Advanced EGFR-Mutated Non-Small Cell Lung Cancer Patients

On November 12 local time, AstraZeneca and Daiichi Sankyo submitted a new Biologics License Application (BLA) for accelerated approval of datopotamab deruxtecan in patients with locally advanced or metastatic epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer (NSCLC) who have previously received systemic therapy. The prior BLA based on the Phase 3 TROPION-Lung01 trial for non-squamous NSCLC patients was voluntarily withdrawn. This new BLA is based on the results of the Phase 2 TROPION-Lung05 trial and supported by additional trial data. TROPION-Lung05 aims to evaluate the efficacy and safety of the drug in specific patient populations, with 137 patients recruited globally. Currently, NSCLC accounts for approximately 80% of all lung cancer cases, and treating EGFR-mutated patients remains challenging. Datopotamab deruxtecan, as a TROP2-directed DXd antibody-drug conjugate, is highly anticipated. Both companies are also conducting multiple Phase 3 trials to assess its efficacy both as a monotherapy and in combination with other treatments, offering hope for new therapeutic options for patients.

4. AstraZeneca announces positive results for Koselugo in the global Phase 3 KOMET trial

On November 12 local time, AstraZeneca announced positive results from the global Phase 3 KOMET trial. The oral selective MEK inhibitor Koselugo demonstrated statistically significant and clinically meaningful objective response rates in adult patients with neurofibromatosis type 1 (NF1). NF1 is a rare, progressive genetic disorder affecting approximately 1.7 million people worldwide, and currently, there are no approved treatments for adult patients. KOMET is the largest global randomized, double-blind, placebo-controlled, multicenter Phase 3 trial conducted in adults with NF1 who have symptomatic, inoperable plexiform neurofibromas. Trial results indicate that Koselugo has the potential to reduce the size of plexiform neurofibromas, positively impacting patient care. Its safety profile was consistent with previous clinical trials in children and adolescents, with no new safety signals identified. AstraZeneca Rare Disease Alexion will share data with regulatory authorities and present findings at medical conferences. AstraZeneca and Merck & Co., Inc. are jointly developing and commercializing Koselugo globally, offering new hope for adult patients.

5. AstraZeneca Invests $3.5 Billion in the U.S. to Expand New Drug Research and Manufacturing

On November 12 local time, AstraZeneca announced a $3.5 billion investment in the United States to expand research and manufacturing of new drugs. This includes $2 billion in new investments, which will create over 1,000 high-skilled jobs. Its U.S. presence spans the R&D center in Cambridge, Massachusetts, the biologics production facility in Maryland, cell therapy manufacturing on both coasts, and specialty manufacturing in Texas. The CEO of AstraZeneca stated that this investment reflects the appeal of the U.S. business environment and its talent advantages, aiming to strengthen therapy development, support U.S. leadership in healthcare innovation, and is a crucial step toward achieving the company’s target of $80 billion in total revenue by 2030.

6. Bayer to Showcase Multiple Research Achievements at the 2024 American Heart Association Scientific Sessions

On November 13 local time, Bayer announced that data from three new analyses of the FINEARTS-HF study and the FIDELITY pooled analysis will be presented at the 2024 American Heart Association Scientific Sessions. This phase III trial will include analyses assessing the efficacy of finerenone in heart failure patients, the risk of hyperkalemia, its impact on different genders, as well as the effects of air pollution exposure and finerenone treatment on patients with type 2 diabetes-related chronic kidney disease. Finerenone, a non-steroidal selective mineralocorticoid receptor antagonist marketed under the names Kerendia™ or Firialta™, has been approved in over 90 countries/regions worldwide for the treatment of adult patients with chronic kidney disease associated with type 2 diabetes. As a global enterprise, Bayer is committed to providing innovative therapies in the cardiovascular and renal disease fields, contributing to human health and the prosperity of the planet.

7. Regeneron to Highlight Groundbreaking Advances in Blood Cancer Treatment at ASH 2024

On November 13 local time, Regeneron Pharmaceuticals announced that new and updated data from its hematology pipeline will be presented at the 2024 American Society of Hematology (ASH) Annual Meeting, to be held from December 7 to 10. This includes 23 abstracts covering applications of odronextamab in various blood cancers, findings on linvoseltamab in multiple myeloma, and more. Notably, oral presentations will share head-to-head results of pozelimab combined with cemdisiran versus ravulizumab combination therapy for treating paroxysmal nocturnal hemoglobinuria. Preliminary results of odronextamab in frontline follicular lymphoma also show significant potential. Regeneron is committed to transforming scientific breakthroughs into differentiated medicines for hematology patients, bringing new hope to those with blood cancers and rare blood disorders.

8. Lilly's Tirzepatide Shows Significant Three-Year Research Results

On November 13 local time, Eli Lilly announced the three-year research results of the phase 3 SURMOUNT-1 trial for tirzepatide. Compared with placebo, tirzepatide significantly reduced the risk of prediabetes patients with obesity (overweight) developing type 2 diabetes by 94%, and nearly 99% of patients treated with tirzepatide did not develop diabetes at week 176. At a dose of 15 mg, participants experienced a 22.9% weight loss that was maintained for over three years. The study also showed that tirzepatide can control blood glucose, reduce cardiometabolic risk factors, and improve quality of life. Post-hoc analysis indicated that about half of the delayed onset effect is related to weight loss. The safety and tolerability profile at week 193 was consistent with previous findings, with common adverse events related to the gastrointestinal tract. Tirzepatide has been approved for use in adults with type 2 diabetes and obesity, and this year, Eli Lilly also submitted data to regulators for obstructive sleep apnea and plans to submit data for heart failure with preserved ejection fraction and obesity.

9. CHMP Recommends EU Approval of Sarclisa for Newly Diagnosed Multiple Myeloma

On November 14, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency recommended the approval of Sarclisa in combination with VRd for the treatment of newly diagnosed adult patients with multiple myeloma who are not eligible for autologous stem cell transplantation. If approved, Sarclisa will become the first anti-CD38 therapy in the EU that can be used in combination with VRd for such patients. The positive opinion of CHMP is based on data from the Phase 3 IMROZ study, which showed that Sarclisa combined with VRd significantly improved progression-free survival compared to VRd alone. In September 2024, Sarclisa was approved by the U.S. FDA for the same indication. Currently, Sarclisa is approved in more than 50 countries/regions for two indications in certain adult patients with relapsed or refractory multiple myeloma. Sanofi is continuing to advance its patient-centered clinical development program for Sarclisa.

10. Amgen to Present New Data on Rare Diseases at ACR 2024

On November 14 local time, Amgen announced new data for its rare disease products at the American College of Rheumatology Convergence 2024 meeting held in Washington, D.C. UPLIZNA (inebilizumab-cdon) demonstrated reduced disease activity in Immunoglobulin G4-Related Disease (IgG4-RD), with Phase 3 study results showing a decreased risk of flares and improved complete remission rates compared to placebo. In August, UPLIZNA received FDA Breakthrough Therapy Designation, and regulatory filings are underway. KRYSTEXXA (pegloticase) showed positive safety and efficacy data when administered with a shorter infusion time in patients with uncontrolled gout, and regulatory submissions are ongoing.

11. GSK Announces Blenrep Shows Overall Survival Benefit in Multiple Myeloma Trial

On November 14 local time, GlaxoSmithKline announced positive interim analysis results from the DREAMM-7 head-to-head Phase III trial. Blenrep (belantamab mafodotin) in combination with bortezomib and dexamethasone (BorDex) significantly reduced the risk of death compared to the standard of care, daratumumab combined with BorDex, in the treatment of relapsed/refractory multiple myeloma, achieving a key secondary endpoint of overall survival. The interim analysis results will be presented at the American Society of Hematology Annual Meeting in December. The DREAMM clinical development program also includes the ongoing DREAMM-8 trial and a new study expected to launch by the end of 2024. In 2024, the belantamab mafodotin combination therapy has been submitted for marketing approval or received designations in multiple countries and regions. This drug, an antibody-drug conjugate, is expected to bring new hope to patients with multiple myeloma.

12. ACR Convergence 2024: UCB Announces New Data on BIMZELX

On November 14 local time, UCB presented new two-year data at ACR Convergence 2024, confirming sustained clinical responses with BIMZELX (bimekizumab-bkzx) in patients with psoriatic arthritis, non-radiographic axial spondyloarthritis, and ankylosing spondylitis. In September, bimekizumab-bkzx received approval from the U.S. FDA for these indications. The new data shows that the drug maintains robust clinical responses, including skin clearance, reduced disease activity, and improved joint pain. For patients with axial spondyloarthritis, there was a significant reduction in inflammation and structural damage, with minimal progression on spinal imaging. Additionally, the drug demonstrated a long-term safety profile consistent with previous studies in two pooled safety analyses of active patients. The results reinforce the potential of bimekizumab-bkzx as an effective treatment option, reflecting UCB’s efforts to provide differentiated therapies for patients with chronic inflammatory diseases.

In China

1. Strategic Cooperation Signing between Lunan Pharmaceutical Group and China Association of Traditional Chinese Medicine

On November 12 local time, Lunan Pharmaceutical Group Corporation and the China Association of Chinese Medicine held a strategic cooperation signing ceremony. Chen Junfeng, Deputy Secretary-General of the China Association of Chinese Medicine, attended the event, introduced the association's work and goals in promoting the development of traditional Chinese medicine (TCM), highly praised Lunan Pharmaceutical’s innovative achievements in the TCM field, and expressed hopes for deeper collaboration in TCM scientific research and academic exchanges to advance high-quality growth in the TCM industry. Zhang Guimin, Party Secretary, Chairman, and General Manager of Lunan Pharmaceutical Group Corporation, presented an overview of the company’s development, core strengths, cultural philosophy, and analyzed the dose-effect relationship and health considerations of TCM using key products as examples. He emphasized the company’s commitment to inheriting and innovating TCM, expressing hopes that this signing will serve as an opportunity for collaboration in clinical research, achievement transformation, talent cultivation, and creating a model for academia-enterprise cooperation. Relevant department heads attended the meeting.

Abroad

1. Boehringer Ingelheim Collaborates with Mary Tyler Moore Vision Initiative to Combat Diabetes-Induced Vision Loss

On November 12 local time, Boehringer Ingelheim and the Mary Tyler Moore Vision Initiative (MTM Vision) announced the start of a long-term collaboration. More than 530 million diabetes patients worldwide are at risk of developing diabetic retinal disease (DRD), and with the rising incidence of diabetes, this epidemic is expected to grow. This partnership aims to address the need for early detection of DRD and the development of new therapies. Alliance members will work together to improve diagnosis and staging of DRD, validate clinical trial endpoints and biomarkers, and accelerate the development of breakthrough treatments. The Global Head of Retinal Health at Boehringer Ingelheim stated that the collaboration will enhance understanding of diabetic retinopathy, support portfolio development, and openly share evidence-generation efforts. The founder of MTM Vision noted that this is a critical moment that will promote the development of new approaches to protect the vision of millions of patients.

2. Primus Sells for $9.5 Billion to BioNTech

On November 13, BioNTech will acquire Promab for an upfront payment of $800 million, with potential milestone payments of up to $1.5 billion. The acquisition is expected to be completed in the first quarter of 2025. Through this acquisition, BioNTech will gain full rights to Promab’s pipeline of drug candidates and its bispecific antibody drug development platform, with the centerpiece being PM8002, a bispecific antibody targeting the PD-L1/VEGF-A pathway. At the end of last year, PM8002 demonstrated anti-tumor activity in first-line treatment for triple-negative breast cancer. The two parties had previously entered into a collaboration agreement, and following this acquisition, BioNTech will hold complete global rights to the asset. Founded six years ago, Promab has advanced over 20 novel drug projects. This "sale" may serve as an effective exit strategy for Promab, but new drug development remains fraught with uncertainties, and whether PM8002 will successfully reach the market is still unknown.

III. Merck Obtains Global Exclusive License for LM-299 from Lixun Pharmaceuticals

On November 14, Merck & Co. and Limin Pharmaceuticals announced that Merck & Co. has obtained the exclusive global license for the development, manufacturing, and commercialization of Limin's investigational PD-1/VEGF bispecific antibody LM-299. LM-299 employs a differentiated molecular design capable of blocking immune checkpoint and anti-angiogenic signaling pathways. Limin Pharmaceuticals will receive an upfront payment of $588 million and may also obtain up to $2.7 billion in milestone payments based on various developments. The transaction is expected to be completed in the fourth quarter of 2024, after which Merck will incur a pre-tax charge of $588 million. The Phase 1 clinical trial of LM-299 is currently recruiting patients in China. Limin Pharmaceuticals focuses on developing novel ADC and immuno-oncology biotherapies, while Merck is committed to advancing innovative drug research and development to meet patient needs.