▎WuXiEdited by Kant Content Team

Recently, IDEA Pharma released a ranking list evaluating pharmaceutical companies' ability to successfully transform innovative scientific research achievements and technologies into early-stage products.In this Pharmaceutical Invention Index, Johnson & Johnson, Merck & Co., Inc., and AstraZeneca rank in the top three, indicating a significant increase in the number of novel drugs in their R&D pipelines.It is worth mentioning that,Jiangsu Hengrui Pharmaceuticals Co., Ltd. also made it to the TOP 10 list this year.In today's article, the WuXi AppTec content team will share with readers the highlights of the list and provide an introduction to the latest R&D progress of the companies featured. Click on "Read More", you can visit the IDEA Pharma website to browse the detailed content of the list.

WuXi AppTec content team created the chart based on reference [2].

Johnson & Johnson

Johnson & Johnson has recently achieved multiple R&D and pipeline expansion advancements. Earlier this month, Johnson & Johnson announced its investigational intravesical targeted-release therapy.TAR-210Positive Results from Phase 1 Trial for the Treatment of High-Risk (HR) Non-Muscle-Invasive Bladder Cancer (NMIBC) Patients.Among 21 HR-NMIBC patients, the 12-month recurrence-free survival rate was as high as 90%.In addition, Johnson & Johnson also reached an $850 million agreement this month with Proteologix, a biotechnology company specializing in treatments for immune-mediated diseases.Acquisition Agreement, which includes its bispecific antibody PX128 targeting IL-13 and TSLP for the treatment of moderate to severe atopic dermatitis. In April this year, the U.S. FDA approved the CAR-T therapy Carvykti (ciltacabtagene autoleucel), jointly developed by Johnson & Johnson and Legend Biotech, for second-line treatment of adult patients with relapsed or refractory (R/R) multiple myeloma (MM). In March this year, Tryvio (aprocitentan) oral tablets, co-developed by the company and Idorsia, were approved by the FDA for use in combination with other antihypertensive drugs to treat adult patients with hypertension.According to the press release, this drug is the first new mechanism-based antihypertensive medication approved in 30 years.In addition, Johnson & Johnson also acquired several clinical-stage Antibody-Drug Conjugates (ADCs) in January this year through the acquisition of Ambrx Biopharma.

MSD

In May this year, MSD's重磅 PD-1 inhibitor Keytruda once again delivered good news, with the therapyPhase 3 Clinical TrialIn China, the primary endpoint was reached. When used in combination with the anti-HER2 antibody trastuzumab and chemotherapy as a first-line therapy for patients with HER2-positive, unresectable locally advanced or metastatic gastric cancer or gastroesophageal junction (GEJ) adenocarcinoma, it significantly extended patients' overall survival. In addition to continuously advancing the development of Keytruda,MSD also welcomed its "first-in-class" therapy in March this year.WinrevairApproval of Sotatercept for the Treatment of Pulmonary Arterial Hypertension (PAH).This therapy was rated by the industry media Evaluate as one of the potential blockbuster therapies of 2024. The personalized neoantigen vaccine mRNA-4157, jointly developed by the company and Moderna, currently has three Phase III clinical trials underway, aimed at treating patients with surgically removed melanoma, non-small cell lung cancer, and cutaneous squamous cell carcinoma respectively. At this year's American Association for Cancer Research conference,MSD and Kelun-Biotech Announce Jointly Developed Trop2-Targeted ADCSKB264The disease control rate (DCR) for treating previously treated advanced gastric cancer or GEJ cancer reached 80.5%.

AstraZeneca

One of AstraZeneca's most notable recent advancements is the approval of Enhertu (trastuzumab deruxtecan), a significant antibody-drug conjugate jointly developed with Daiichi Sankyo.Accelerated ApprovalFor the treatment of adult patients with unresectable or metastatic HER2-positive solid tumors.According to the press release, Enhertu is the first HER2-targeted ADC therapy with a tumor-agnostic indication.Currently, an ADC therapy targeting Trop2, datopotamab deruxtecan, co-developed by the company and Daiichi Sankyo, has been submitted to the FDA for marketing approval to treat adult patients with unresectable or metastatic HR-positive, HER2-negative breast cancer. Additionally, the company's "first-in-class" oral small molecule Voydeya (danicopan) was approved in March this year for treating PNH patients. Besides actively advancing its internal pipeline,AstraZeneca also announced in November 2023 thatCellectisThe companies have reached a strategic cooperation and will jointly accelerate the development of up to 10 next-generation cell and gene therapies.

Bristol-Myers Squibb

Bristol-Myers Squibb received expanded approval for its CAR-T therapy Breyanzi (lisocabtagene maraleucel) in March and May this year, respectively, for the treatment of R/R chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), and follicular lymphoma (FL).Another CAR-T Cell Therapy from the CompanyAbecma(Ide-cel, idecabtagene vicleucel) has also been recently approved for the treatment of R/R multiple myeloma. In addition, in February this year, the U.S. FDA accepted the combination of KRAS G12C inhibitor Krazati (adagrasib) and cetuximab for the treatment of tumors with...KRAS The supplemental New Drug Application (sNDA) for G12C-mutant pretreated colorectal cancer has been granted priority review status. In the same month, the sNDA for its ROS1 and NTRK-targeted inhibitor Augtyro (repotrectinib) for the treatment of solid tumors also received priority review designation. Both sNDAs are expected to receive review outcomes by June this year. KarXT, a potential "first-in-class" therapy acquired through the acquisition of Karuna Therapeutics, is also anticipated to gain approval in September this year, becoming the first new mechanism drug for the treatment of schizophrenia in decades. Additionally,Bristol-Myers Squibb also extended its existing agreement with Editas Medicine earlier this month.R&D Collaboration, jointly developing autologous and allogeneic α-β T cell therapies for the treatment of cancer and autoimmune diseases.

Moderna

In April this year, Moderna published its investigational mRNA therapy for the treatment of propionic acidemia (PA) in the journal *Nature*.mRNA-3927The first clinical trial results of mRNA-3927 showed that it demonstrated good safety and tolerability, reducing the risk of disease onset by 70% in patients treated with mRNA-3927. Researchers noted,This is the first clinical trial to use mRNA to express intracellular proteins as a protein replacement therapy in patients with rare diseases.Moderna and MSD also launched a Phase 2/3 clinical trial in March this year to evaluate the efficacy and safety of their jointly developed personalized neoantigen vaccine mRNA-4157 (V940) in patients with resectable locally advanced cutaneous squamous cell carcinoma. This is the third pivotal clinical trial planned by the two companies following the launch of pivotal trials using mRNA cancer vaccines to treat melanoma and non-small cell lung cancer.

Pfizer

Pfizer Recently Welcomes Several Important Regulatory Developments.Its one-time gene therapyBeqvez(Fidanacogene elaparvovec) was approved by the U.S. FDA in April this year for the treatment of adult patients aged 18 years or older with moderate to severe hemophilia B.In the same month, the European Commission (EC) approved its joint development with AbbVie.Emblaveo(Aztreonam-avibactam) has been launched for the treatment of complicated intra-abdominal infections, hospital-acquired pneumonia (including ventilator-associated pneumonia), and complicated urinary tract infections (including pyelonephritis) in adults. Additionally, Pfizer and Arvinas have jointly reviewed the latest clinical data on the combination of their CDK4/6 inhibitor Ibrance (palbociclib) and the estrogen receptor (ER) PROTAC protein degrader vepdegestrant (ARV-471) for the treatment of patients with locally advanced or metastatic estrogen receptor-positive/human epidermal growth factor 2-negative (ER+/HER2-) breast cancer.Publish。The overall response rate for breast cancer patients receiving combination therapy was 41.9%, with a median progression-free survival of 11.2 months, demonstrating the potential of vepdegestrant combined with palbociclib in treating breast cancer.

Eli Lilly

Recently,Eli Lilly Announces Its Ultra-Long-Acting Once-a-Week Insulinefsitora alfa(Efsitora) Achieved Primary Endpoints in Both Phase III Clinical Trials.Compared with the globally commonly used daily basal insulin, efsitora demonstrated non-inferiority in reducing HbA1c levels in patients with type 2 diabetes. Additionally, Eli Lilly had two innovative therapies approved by the U.S. FDA in 2023. In October last year, its "first-in-class" antibody therapyOmvoh(Mirikizumab) has been approved for marketing to treat adult patients with moderate to severe active ulcerative colitis (UC).The press release noted that this is the first IL-23p19 antagonist for treating this patient population.The company's non-covalent selective Bruton's tyrosine kinase (BTK) inhibitorJaypirca(Pirtobrutinib) received accelerated FDA approval in January of last year for the treatment of patients with relapsed/refractory mantle cell lymphoma (MCL). In December of the same year, this therapy received another accelerated FDA approval for the treatment of adult patients with chronic lymphocytic leukemia or small lymphocytic lymphoma.

Vertex Pharmaceuticals

Vertex recently announced that the company has submitted its next-generation triple combination therapy (vanzacaftor/tezacaftor/deutivacaftor, referred to as vanza triple therapy) for the treatment of cystic fibrosis (CF) to the U.S. FDA and the European Medicines Agency (EMA).Marketing Application, for the treatment of CF patients aged 6 years and above. In April this year, Vertex initiated a rolling submission to the U.S. FDA for its investigational selective NaV1.8 inhibitor.suzetrigineNew Drug Application for (VX-548) for the Treatment of Moderate to Severe Acute Pain.According to the press release, suzetrigine is expected to become the first new drug for treating acute and neuropathic pain in over 20 years.The company reached an agreement in the same month to invest approximately US$4.9 billion.AcquisitionImmunotherapy company Alpine Immune Sciences acquires its lead product povetacicept (ALPN-303). This therapy is expected to enter a pivotal Phase 3 clinical trial for the treatment of IgA nephropathy (IgAN) in the second half of 2024.

Regeneron

Regeneron announced the Phase 1/2 CHORD trial this monthPreliminary Positive Results。A patient with hereditary deafness who received the investigational gene therapy DB-OTO at 11 months old recovered to normal hearing levels within 24 weeks, while another patient who received the therapy at age 4 also showed initial improvement in hearing 6 weeks after treatment.The重磅 therapy Dupixent (dupilumab), co-developed by the company and Sanofi, was granted Priority Review designation by the FDA in February and May this year for the treatment of chronic obstructive pulmonary disease and chronic rhinosinusitis with nasal polyps. In addition, Regeneron reached a nearly $100 million agreement with Mammoth Biosciences last month.Cooperation Agreement, will jointly develop in vivo CRISPR gene editing therapies for various tissues and cell types.

Jiangsu Hengrui Pharmaceuticals Co., Ltd.

Hengrui Pharma boasts a rich pipeline.Hengrui Pharma recently received clinical trial implied permission from the Center for Drug Evaluation (CDE) of the China National Medical Products Administration for three Class 1 new drugs, and will soon commence clinical trials.Including 1) NK-1 receptor antagonist for injection, HRS5580, intended for the prevention of postoperative nausea and vomiting; 2) Complement factor B inhibitor, HRS-5965 capsules, intended for the treatment of primary or secondary glomerular diseases mediated by complement, as well as hemolytic anemia; 3) ATR inhibitor, HRS2398 sustained-release tablets, under development for the treatment of patients with advanced malignant tumors. This month, Hengrui Pharma announced that it had licensed its self-developed GLP-1 product portfolio to U.S.-based Hercules Company, with the upfront payment and various milestone payments for this collaboration potentially exceeding $6 billion.

In addition to ranking the innovation indices of pharmaceutical companies, IDEA Pharma also released the Pharmaceutical Innovation Index list, which examines a company's ability to efficiently advance early-stage pipeline products to market approval, thereby benefiting patients with innovative and effective drugs. Due to space limitations, we will not provide a detailed introduction here. For the complete list, click on “Read More", you can visit the IDEA Pharma website to browse the detailed content of the list.