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October 2024

# of articles ... 62

as of October 31

First approval ... 5

Astellas’ VYLOY (zolbetuximab-clzb) Approved by U.S. FDA for Treatment of Advanced Gastric and GEJ Cancer - - VYLOY is the first and only CLDN18.2-targeted treatment < October 21, 2024

The U.S. FDA has approved VYLOYTM (zolbetuximab-clzb) in combination with fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of adults with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors are claudin (CLDN) 18.2 positive as determined by an FDA-approved test.

U.S. FDA Approves VYALEV™ (foscarbidopa and foslevodopa) for Adults Living with Advanced Parkinson's Disease < ABBV, October 17, 2024

The U.S. FDA has approved VYALEV™ (foscarbidopa and foslevodopa) as the first and only subcutaneous 24-hour infusion of levodopa-based therapy for the treatment of motor fluctuations in adults with advanced Parkinson's disease (PD). The approval was supported by the pivotal Phase 3, 12-week study evaluating the efficacy of continuous subcutaneous infusion of VYALEV in adult patients with advanced PD compared to oral immediate-release carbidopa/levodopa (CD/LD IR)1, along with a 52-week, open-label study which evaluated the long-term safety and efficacy of VYALEV.

FDA Approves Pfizer’s HYMPAVZI™ (marstacimab-hncq) for the Treatment of Adults and Adolescents with Hemophilia A or B Without Inhibitors < October 11, 2024

HYMPAVZI’s approval is based on Phase 3 study results demonstrating substantial bleed reduction compared to routine prophylaxis and on-demand treatment in eligible patients with hemophilia A or B without inhibitors. In the U.S., HYMPAVZI is the first once-weekly subcutaneous prophylactic treatment for eligible people living with hemophilia B, and the first to be administered via a pre-filled pen or syringe for eligible people living with hemophilia A or B.

FDA approves Roche’s Itovebi, a targeted treatment for advanced hormone receptor-positive, HER2-negative breast cancer with a PIK3CA mutation < October 11, 2024

The US FDA approved Itovebi™ (inavolisib), in combination with palbociclib (Ibrance®) and fulvestrant, for the treatment of adults with endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy. The PIK3CA mutation is found in approximately 40% of HR-positive metastatic breast cancers.

Roche obtains CE certification for the first companion diagnostic to identify patients with gastric and gastroesophageal junction cancer eligible for targeted treatment with VYLOY < Astellas, October 10, 2024

The new VENTANA CLDN18 (43-14A) RxDx Assay helps fulfil an unmet medical need by enabling clinicians to identify patients with gastric or gastroesophageal junction (GEJ) cancer who may benefit from a targeted treatment option. CLDN18.2 is an emerging biomarker in gastric and GEJ cancers and helps predict the likelihood of response to targeted therapy.

Supplemental approval ... 7

Novartis Scemblix® FDA approved in newly diagnosed CML, offering superior efficacy, and favorable safety and tolerability profile < October 29, 2024

Scemblix® (asciminib) was granted accelerated approval by the US FDA for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). The accelerated approval is based on major molecular response rate (MMR) at week 48 from the ASC4FIRST Phase III trial that compared once daily Scemblix to all other investigator-selected (IS) standard of care (SoC) tyrosine kinase inhibitors (TKIs) (imatinib, nilotinib, dasatinib, and bosutinib).

Novartis ribociclib (Kisqali®) recognized as Category 1 preferred breast cancer adjuvant treatment by NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) < October 24, 2024

Ribociclib (Kisqali) plus aromatase inhibitor (AI) recommended for HR+/HER2- early breast cancer (EBC) node-positive and high-risk node-negative patients, as studied in the NATALEE trial and indicated by the FDA. Recommendation comes after recent FDA approval and positive CHMP Opinion for ribociclib (Kisqali) to help reduce the risk of recurrence in EBC. Ribociclib (Kisqali) is also the only Category 1 preferred CDK4/6 inhibitor for first-line treatment of patients with HR+/HER2- metastatic breast cancer in combination with an AI.

DARZALEX® (daratumumab)-SC based quadruplet regimen approved by the European Commission for patients with newly diagnosed multiple myeloma who are transplant-eligible < JNJ, October 23, 2024

The European Commission (EC) approved the indication extension for DARZALEX® (daratumumab) subcutaneous (SC) formulation in combination with bortezomib, lenalidomide and dexamethasone (daratumumab-VRd) in patients with newly diagnosed multiple myeloma (NDMM) who are eligible for an autologous stem cell transplant (ASCT).3 Patients will have the opportunity to receive this daratumumab SC-based quadruplet therapy at initial diagnosis, providing them with a new treatment shown to significantly improve outcomes.

CDC’S ACIP Recommends Merck’s CAPVAXIVE™ (Pneumococcal 21-valent Conjugate Vaccine) for Pneumococcal Vaccination in Adults 50 Years of Age and Older < October 23, 2024

The U.S. Centers for Disease Control and Prevention’s (CDC’s) Advisory Committee on Immunization Practices (ACIP) voted to update the adult age-based pneumococcal vaccination guidelines and recommends CAPVAXIVE™ (Pneumococcal 21-valent Conjugate Vaccine) for pneumococcal vaccination in adults 50 years of age and older.

U.S. FDA Approves Pfizer’s RSV Vaccine ABRYSVO® for Adults Aged 18 to 59 at Increased Risk for Disease - First for adults younger than 50 < October 22, 2024

The U.S. FDA has approved ABRYSVO ®, the company’s bivalent RSV prefusion F (RSVpreF) vaccine, for the prevention of lower respiratory tract disease (LRTD) caused by RSV in individuals 18 through 59 years of age who are at increased risk for LRTD caused by RSV. ABRYSVO now offers the broadest RSV vaccine indication for adults, which previously included those 60 years and older. Additionally, it remains the only RSV immunization approved for pregnant individuals at 32 through 36 weeks of gestation to protect infants from birth up to 6 months of age.

UCB receives U.S. FDA approval for 320 mg single-injection device presentations of BIMZELX® (bimekizumab-bkzx) < October 14, 2024

The new pre-filled syringe and pre-filled autoinjector mean that patients requiring a 320 mg dose of BIMZELX® bimekizumab-bkzx) will have single-injection administration options. The U.S. FDA has approved a 2 mL pre-filled syringe and pre-filled autoinjector, each containing 320 mg of BIMZELX® (bimekizumab-bkzx). These new device presentations add to the currently available 1 mL administration options, each containing 160 mg of bimekizumab-bkzx, and mean that patients requiring a 320 mg dose of bimekizumab-bkzx will have options for single-injection administration.

U.S. Food and Drug Administration Approves Perioperative Treatment of Neoadjuvant Opdivo® (nivolumab) and Chemotherapy Followed by Surgery and Adjuvant Single-Agent Opdivo for Resectable Non-Small Cell Lung Cancer (NSCLC) < BMY, October 03, 2024

Approval is based on the CheckMate-77T trial, in which the Opdivo- based regimen demonstrated significantly longer event-free survival compared to the chemotherapy and placebo arm; a high pathologic complete response rate was also observed. Opdivo is the only approved PD-1 inhibitor for resectable NSCLC in both a neoadjuvant-only regimen and as part of a perioperative treatment regimen.

Regulatory ... 11

Eisai Completes Rolling Submission to US FDA for LEQEMBI® (lecanemab-irmb) Biologics License Application for Subcutaneous Maintenance Dosing for the Treatment of Early Alzheimer’s Disease Under the Fast Track Status < BIIB, October 31, 2024

The BLA is based on data from the Clarity AD (Study 301) open-label extension (OLE) and modeling of observed data. If approved by the FDA, the LEQEMBI autoinjector could be used to administer LEQEMBI at home or at medical facilities, and the injection process is expected on average to take about 15 seconds. As part of the subcutaneous autoinjector 360 mg weekly maintenance regimen under review, patients who have completed the biweekly intravenous (IV) initiation phase would receive weekly doses that maintain effective drug concentrations to sustain the clearance of highly toxic protofibrils* which can continue to cause neuronal injury even after the amyloid-beta (Aβ) plaque has been cleared from the brain.

Astellas Provides Update on Marketing Authorization Application for Avacincaptad Pegol (ACP) in the European Union < Setback, Ophthalmic, October 28, 2024

Avacincaptad pegol intravitreal solution (ACP) is an investigational synthetic aptamer that inhibits the complement C5 protein for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The company's decision to withdraw its application followed interactions with the EMA’s Committee for Medicinal Products for Human Use (CHMP).

Advisory Committee on Immunization Practices Recommends PREVNAR 20® (20-Valent Pneumococcal Conjugate Vaccine) for Adults Aged 50 and Older < Pfizer, October 23, 2024

The U.S. Centers for Disease Control and Prevention’s (CDC) Advisory Committee on Immunization Practice (ACIP) voted to expand its recommendation for the use of certain pneumococcal vaccines, including PREVNAR 20 ® (20-valent Pneumococcal Conjugate Vaccine) for all adults aged 50 and older. This recommendation is pending final approval by the director of the CDC and the Department of Health and Human Services.

Gepotidacin accepted for priority review by US FDA for treatment of uncomplicated urinary tract infections in female adults and adolescents < GSK, 16 October 2024

The US FDA has accepted the NDA for gepotidacin, an investigational, first-in-class oral antibiotic with a novel mechanism of action for the treatment of female adults (≥40 kg) and adolescents (≥12 years, ≥40 kg) with uncomplicated urinary tract infections (uUTIs). The NDA is supported by positive results from the pivotal phase III EAGLE-2 and EAGLE-3 trials. In these studies, gepotidacin demonstrated non-inferiority to nitrofurantoin, the current standard of care for uUTI, in female adults (≥40 kg) and adolescents (≥12 years, ≥40 kg) with a confirmed uUTI and a uropathogen susceptible to nitrofurantoin.

U.S. Food and Drug Administration (FDA) accepts New Drug Application for elinzanetant < VMS, Bayer, October 9, 2024

Bayer today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for the investigational compound elinzanetant seeking approval for the treatment of moderate-to-severe vasomotor symptoms (VMS, also known as hot flashes) associated with menopause. The NDA application is based on the positive results from the OASIS 1, 2 and 3 Phase III studies evaluating the efficacy and safety of the investigational compound elinzanetant versus placebo.

Alnylam Submits Supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration for Vutrisiran for the Treatment of Transthyretin Amyloidosis with Cardiomyopathy < October 09, 2024

Vutrisiran is the generic name for AMVUTTRA®, which is currently approved by the U.S. FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults. As part of the submission, the Company utilized a Priority Review Voucher, which obligates the FDA to an accelerated review timeline. The application to the FDA was based on positive results from HELIOS-B, a Phase 3, randomized, double-blind, placebo-controlled multicenter global study in patients with ATTR-CM with substantial background use of other effective therapies.

Biogen Receives U.S. FDA Breakthrough Therapy Designation for Felzartamab for the Treatment of Antibody-Mediated Rejection in Kidney Transplant Recipients < October 9, 2024

Felzartamab, an investigational anti-CD38 monoclonal antibody, has received Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) for the treatment of late antibody-mediated rejection (AMR) without T-cell mediated rejection in kidney transplant patients.

Boehringer receives U.S. FDA Breakthrough Therapy designation and initiates two phase III trials in MASH for survodutide < October 08, 2024

FDA has granted BTD for survodutide (BI 456906), a dual glucagon/GLP-1 receptor agonist for the treatment of adults living with non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) and moderate or advanced fibrosis (stages 2 or 3). In addition, Boehringer announced the initiation of two Phase III clinical trials for survodutide for the treatment of adults living with MASH and fibrosis (scarring).

Calquence granted Priority Review in the US for patients with untreated mantle cell lymphoma < AZN, 3 October 2024

AstraZeneca’s supplemental New Drug Application (sNDA) for Calquence (acalabrutinib) has been accepted and granted Priority Review in the US for the treatment of adult patients with previously untreated mantle cell lymphoma (MCL). ECHO Phase III trial demonstrated Calquence combination reduced risk of disease progression or death by 27% compared to standard-of-care chemoimmunotherapy,

Johnson & Johnson files for U.S. FDA approval of DARZALEX FASPRO®-based quadruplet regimen for newly diagnosed multiple myeloma patients for whom transplant is not planned < October 2, 2024

JNJ announced the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for approval of a new indication for DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) in combination with bortezomib, lenalidomide and dexamethasone (D-VRd) for the treatment of adult patients with newly diagnosed multiple myeloma (NDMM) for whom autologous stem cell transplant (ASCT) is deferred or who are ineligible for ASCT.

Enhertu granted Priority Review in the US for patients with HER2-low or HER2-ultralow metastatic breast cancer who have received at least one line of endocrine therapy < AZN, Daiichi Sankyo, 1 October 2024

AstraZeneca and Daiichi Sankyo’s supplemental Biologics License Application (sBLA) for Enhertu (trastuzumab deruxtecan) has been accepted and granted Priority Review in the US for the treatment of adult patients with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-) or HER2-ultralow (IHC 0 with membrane staining) breast cancer who have received at least one endocrine therapy in the metastatic setting based on the positive results from the DESTINY-Breast06 Phase III trial which compared Enhertu to chemotherapy.

Pivotal studies ... 17

New England Journal of Medicine publishes landmark phase III results for Roche’s Itovebi, showing more than doubling of progression-free survival in certain type of HR-positive advanced breast cancer < October 31, 2024

Itovebi (inavolisib)-based regimen demonstrated a statistically significant and clinically meaningful benefit, reducing the risk of disease worsening or death by 57% compared with palbociclib and fulvestrant alone in the INAVO120 study. A detailed analysis of the positive phase III INAVO120 results, evaluating Itovebi (inavolisib) in combination with palbociclib (Ibrance®) and fulvestrant were published in the New England Journal of Medicine.

TREMFYA® (guselkumab) is the first and only IL-23 inhibitor to demonstrate robust results with a fully subcutaneous regimen in both induction and maintenance in Crohn’s disease < JNJ, October 28, 2024

A greater number of patients treated with subcutaneous TREMFYA® induction and maintenance achieved clinical and endoscopic remission at 48 weeks in the Phase 3 GRAVITI study versus placebo. TREMFYA® could become the first IL-23 treatment to offer both a subcutaneous and intravenous (IV) induction regimen for patients living with Crohn’s disease (CD), pending FDA approval

Novartis oral Fabhalta® (iptacopan) sustained clinically meaningful results at one year in Phase III C3 glomerulopathy (C3G) trial < October 27, 2024

New APPEAR-C3G data show Fabhalta sustained proteinuria reduction at 12 months. Novartis presented 12-month data from the Phase III APPEAR-C3G study at American Society of Nephrology (ASN) Kidney Week 2024 showing that patients with C3 glomerulopathy (C3G) treated with oral Fabhalta® (iptacopan) in addition to supportive care experienced clinically meaningful, sustained results at one year.

Biogen Presents Positive Results from Phase 2 IGNAZ Study of Felzartamab in IgA Nephropathy at American Society of Nephrology (ASN) Kidney Week 2024 < October 26, 2024

The detailed study results confirmed interim findings, showing stable kidney function and sustained treatment effect more than 18 months after the last dose of felzartamab. Felzartamab, an investigational anti-CD38 monoclonal antibody, is a potential first-in-class therapeutic candidate for a range of rare immune-mediated indications with planning underway for Phase 3 development

New data for Arexvy, GSK’s respiratory syncytial virus vaccine, show potential to help protect a broader group of adults at increased risk for RSV disease < 24 October 2024

Single dose of vaccine elicited robust immune response with acceptable safety profile in adults aged 18-49 at increased risk for RSV-LRTD. Two doses of vaccine in immunocompromised adults aged 18 and above elicited immune responses similar to one dose in healthy adults aged 50+ and with an acceptable safety profile

Otsuka Announces Positive Interim Results from the Phase 3 Trial of Sibeprenlimab for the Treatment of Immunoglobulin A Nephropathy in Adults < October 22, 2024

Phase 3 VISIONARY study met its primary endpoint at prespecified interim analysis; sibeprenlimab demonstrated a statistically significant and clinically meaningful reduction in 24- hour uPCR after nine months of treatment; the favorable safety profile of sibeprenlimab was consistent with previously reported data. • Immunoglobulin A nephropathy is a progressive, autoimmune, chronic kidney disease that can lead to end-stage kidney disease (ESKD) over the lifetime of most patients under current optimized standard care. Otsuka plans to review interim results with the FDA to enable a potential regulatory submission for accelerated approval; study continues with final results expected in early 2026.

UCB Announces Bepranemab Phase 2a Study Results Accepted for Late-Breaking Presentation at Clinical Trials on Alzheimer’s Disease (CTAD) 2024 Meeting < 22 October 2024

Bepranemab - an investigational anti-tau antibody - targeting the mid-region of the tau protein is being studied to assess its potential to delay Alzheimer’s disease progression. Late-breaking Phase 2a study TOGETHER (AH0003) results include all relevant clinical and imaging data.

Oral semaglutide demonstrates a 14% reduction in risk of major adverse cardiovascular events in adults with type 2 diabetes in the SOUL trial < Novo, 21 October 2024

Novo Nordisk today announced the headline results from the SOUL cardiovascular outcomes trial. The double-blinded, randomised trial compared oral semaglutide to placebo as an adjunct to standard of care for the prevention of major adverse cardiovascular events (MACE). The trial enrolled 9,650 people with type 2 diabetes and established cardiovascular disease (CVD) and/or chronic kidney disease (CKD). As part of standard of care, 49% of patients received SGLT2i at some point during the trial.

Gilead and Merck Announce Phase 2 Data Showing a Treatment Switch to an Investigational Oral Once-Weekly Combination Regimen of Islatravir and Lenacapavir Maintained Viral Suppression in Adults at Week 48 < October 19, 2024

At 48 weeks, the novel investigational combination maintained a high rate (n=49; 94.2%) of viral suppression (HIV-1 RNA <50 copies/mL) in virologically suppressed adults, a secondary endpoint of the study. Zero participants had a viral load of ≥50 copies/mL at Week 48.

Merck’s Clesrovimab (MK-1654), an Investigational Respiratory Syncytial Virus (RSV) Preventative Monoclonal Antibody, Significantly Reduced Incidence of RSV Disease and Hospitalization in Healthy Preterm and Full-term Infants < October 17, 2024

Clesrovimab has the potential to become the first and only approved immunization designed to protect infants with the same single dose regardless of weight for the duration of their first RSV season. Results from MK-1654-004, a placebo-controlled Phase 2b/3 pivotal trial evaluating a single dose of clesrovimab administered to healthy preterm and full-term infants (birth to 1 year of age) met all prespecified endpoints, with consistent results through both the 5-month and 6-month time points.

Amgen Presents Positive Phase 3 Data For Uplizna® (Inebilizumab-cdon) In generalized Myasthenia Gravis (gMG) At AANEM 2024 < October 15, 2024

AMGN announced the presentation of positive top-line results from the Phase 3 MINT trial evaluating the efficacy and safety of UPLIZNA® (inebilizumab-cdon) for the treatment of adults with generalized myasthenia gravis (gMG), a rare autoimmune disorder. The trial met its primary endpoint, with a statistically significant change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score for UPLIZNA (-4.2) compared with placebo (-2.2) (difference: –1.9, p<0.0001) at Week 26 for the combined study population.

GSK announces positive phase III results from ANCHOR trials for depemokimab in chronic rhinosinusitis with nasal polyps < 14 October 2024

Primary endpoints met with statistically significant reduction in nasal polyp size and nasal obstruction versus placebo plus standard of care, at 52 weeks. Depemokimab is an ultra-long-acting biologic administered once every six months. Patients with chronic rhinosinusitis with nasal polyps (CRSwNP) experience a range of symptoms which are widely underestimated and often sub-optimally treated.

Pfizer’s TALZENNA® in Combination with XTANDI® Prolongs Overall Survival in Phase 3 TALAPRO-2 Trial < CRPC, October 10, 2024

PFE announced positive topline results from the final prespecified overall survival (OS) analysis of the TALAPRO-2 study of TALZENNA ® (talazoparib), an oral poly ADP-ribose polymerase (PARP) inhibitor, in combination with XTANDI ® (enzalutamide), an androgen receptor pathway inhibitor (ARPI), in patients with metastatic castration-resistant prostate cancer (mCRPC). Results showed a statistically significant and clinically meaningful improvement in the final OS in all-comers (cohort 1) as well as in those patients with homologous recombination repair (HRR) gene-mutated mCRPC (cohort 2), compared to XTANDI alone.

Merck’s KEYTRUDA® (pembrolizumab) Met Primary Endpoint of Event-Free Survival (EFS) as Perioperative Treatment Regimen in Patients With Head and Neck Squamous Cell Carcinoma < October 8, 2024

KEYNOTE-689 is the first Phase 3 trial to demonstrate statistically significant and clinically meaningful improvement in EFS in the intent-to-treat population in the neoadjuvant and adjuvant setting for an anti-PD-1 therapy in earlier stages of head and neck squamous cell carcinoma. The Phase 3 KEYNOTE-689 trial evaluating KEYTRUDA as a perioperative treatment for patients newly diagnosed with stage III or IVA, resected, locally advanced head and neck squamous cell carcinoma (LA-HNSCC) met its primary endpoint of event-free survival (EFS).

New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA < BIIB, October 8, 2024

Findings from Part B and Part C of the DEVOTE study support the clinical benefits of a higher dose regimen of nusinersen (50/28 mg) in both individuals previously treated and treatment-naïve to nusinersen. Investigational regimen also shows more rapid slowing of neurodegeneration, as measured by neurofilament. Biogen plans to submit regulatory applications around the world for approval of the nusinersen higher dose regimen

Johnson & Johnson Statement on the SunRISe-2 Study < TAR-200, Bladder cancer, Setback, October 7, 2024

The SunRISe-2 study in patients with muscle-invasive bladder cancer (MIBC) who are not receiving radical cystectomy was a bold approach to disrupt the established standard of care in chemoradiation in this difficult-to-treat population. Following an Independent Data Monitoring Committee recommendation and pre-specified interim analysis, SunRISe-2 was discontinued for not showing superiority versus chemoradiation.

Airsupra demonstrated statistically significant and clinically meaningful reduction in the risk of severe exacerbations in patients with intermittent or mild persistent asthma in BATURA Phase III trial < AZN, 7 October 2024

Independent Data Monitoring Committee recommended trial stop early due to overwhelming efficacy at pre-planned analysis. Positive high-level results from the BATURA Phase IIIb trial showed AstraZeneca’s Airsupra (albuterol/budesonide) met the primary endpoint, demonstrating a statistically significant and clinically meaningful reduction in the risk of a severe exacerbation when used as an as-needed rescue medication in response to symptoms compared to as-needed albuterol. The trial included patients with intermittent or mild persistent asthma, including those on short-acting beta2-agonist (SABA) alone, low-dose inhaled corticosteroid (ICS) maintenance therapy, or leukotriene receptor antagonist (LTRA) maintenance therapy.

Other R&D ... 17

Roche presents new data at CTAD, demonstrating its growing momentum in diagnostics for Alzheimer’s disease < October 31, 2024

New data highlight the potential of the Roche Elecsys® Amyloid Plasma Panel and Elecsys ptau181 for ruling out Alzheimer’s disease related amyloid pathology with very good accuracy. In the largest worldwide clinical trial of its kind, the blood-based test showed very good accuracy in ruling out Alzheimer's pathology in those being investigated for the disease, potentially avoiding the need for further invasive and unnecessary tests.

Bristol Myers Squibb Presents New Long-term Data from the EMERGENT Program Evaluating COBENFY™ (xanomeline and trospium chloride) in Adults with Schizophrenia at Psych Congress 2024 < October 31, 2024

Long-term treatment with COBENFY was associated with continued improvements in symptoms of schizophrenia, demonstrating maintenance of effect. BMY announced new topline results from the Phase 3 EMERGENT-4 and EMERGENT-5 open-label trials evaluating the long-term efficacy, safety, and tolerability of COBENFY™ (xanomeline and trospium chloride) in adults with schizophrenia over 52 weeks of treatment.

Eisai Presents Data on Benefits of Long-Term Administration of Dual-Acting Lecanemab at the 17th Clinical Trials for Alzheimer’s Disease (CTAD) Conference < BIIB, October 30, 2024

The open-label long-term extension study (OLE) following the core Phase 3 Clarity AD study showed that the mean change from baseline in CDR-SB (global cognitive and functional scale) in the lecanemab treated group relative to the placebo group was -0.45 at 18 months, and at 36 months, this expanded to -0.95 compared to a prespecified natural history cohort of AD. There was a 30% reduction in the relative risk of progressing to the next disease stage.

Modified Titration of Donanemab Demonstrated Reduction of ARIA-E in Early Symptomatic Alzheimer's Disease Patients in Phase 3b study < Lilly, October 29, 2024

LLY announced positive results from the TRAILBLAZER-ALZ 6 Phase 3b study, showing a reduction in amyloid-related imaging abnormalities with edema/effusion (ARIA-E) at the 24-week primary endpoint for people receiving a slightly modified titration of donanemab in adults with early symptomatic Alzheimer's disease (AD).

Merck and Moderna Initiate Phase 3 Trial Evaluating Adjuvant V940 (mRNA-4157) in Combination with KEYTRUDA® (pembrolizumab) After Neoadjuvant KEYTRUDA and Chemotherapy in Patients With Certain Types of Non-Small Cell Lung Cancer (NSCLC) < October 28, 2024

MRK and MRNA announced the initiation of INTerpath-009, a pivotal Phase 3 randomized clinical trial evaluating V940 (mRNA-4157), an investigational individualized neoantigen therapy (INT), in combination with KEYTRUDA ® (pembrolizumab) as adjuvant treatment for patients with resectable Stage II, IIIA or IIIB (N2) non-small cell lung cancer (NSCLC) who did not achieve a pathological complete response (pCR) after receiving neoadjuvant KEYTRUDA plus platinum-based chemotherapy.

Lilly's mirikizumab is first and only IL23p19 antagonist to report long-term, multi-year, sustained efficacy and safety data for both ulcerative colitis and Crohn's disease < October 28, 2024

Two multi-year, Phase 3 studies, LUCENT-3 in moderately to severely active UC and VIVID-2 in moderately to severely active Crohn's disease, showed patients treated with mirikizumab sustained stable, long-term remission across two types of inflammatory bowel diseases (IBD). At three years, over 80% of patients with moderately to severely active UC who were in remission with mirikizumab sustained long-term remission and relief from disruptive symptoms, including bowel urgency.

New SPECTREM study findings reveal TREMFYA® (guselkumab) effectively clears overlooked and undertreated plaque psoriasis < JNJ, October 25, 2024

First-of-its-kind Phase 3b study shows TREMFYA® achieved statistical significance across all primary and secondary endpoints in low body surface area psoriasis with special site involvement. Ttreatment with TREMFYA® (guselkumab) resulted in clear or almost clear skin in the majority of adults with low body surface area (BSA) moderate plaque psoriasis (PsO) with special site involvement who had failed topical treatment. Sensitive or highly visible areas affected by PsO, including the scalp, face, skin folds and genitals, are considered “special sites” and can have significant impact on patients’ daily lives, yet systemic treatment is infrequently provided and this group of patients remains largely undertreated.

Dupixent late-breaking positive phase 3 data in chronic spontaneous urticaria to be presented at ACAAI < Sanofi, October 24, 2024

Dupixent significantly reduced itch and hive activity from baseline; 41% of patients achieved well. The phase 3 LIBERTY-CUPID Study C evaluating the investigational use of Dupixent (dupilumab) in biologic-naive patients with uncontrolled chronic spontaneous urticaria (CSU) who receive background therapy with antihistamines will be presented in a late-breaking oral presentation at the American College of Allergy, Asthma and Immunology (ACAAI) 2024 Annual Scientific Meeting in Boston, Massachusetts.

Roche’s Vabysmo improved vision in underrepresented populations with diabetic macular edema (DME) < October 19, 2024

The ELEVATUM study showed clinically meaningful improvement in vision and reduction in retinal fluid in people with diabetic macular edema (DME) treated with Vabysmo who identify as African American, Black, Hispanic and Latino.
... positive topline one-year results from the open-label, single-arm phase IV ELEVATUM study evaluating Vabysmo® (faricimab) for the treatment of diabetic macular edema (DME) in people from racial and ethnic groups that are often underrepresented in clinical trials.

Nipocalimab demonstrates sustained disease control in adolescents living with generalized myasthenia gravis in Phase 2/3 study < JNJ, October 15, 2024

Johnson & Johnson today announced positive results from the Phase 2/3 Vibrance-MG study of nipocalimab in anti-AChRa positive adolescents (aged 12 – 17 years) living with generalized myasthenia gravis (gMG). Study participants who were treated with nipocalimab plus standard of care (SOC) achieved sustained disease control as measured by the primary endpoint of immunoglobulin G (IgG) reduction from baseline over 24 weeks, and secondary endpoints of improvement in MG-ADLb and QMGc scores.

Lilly reports one-year histologic outcomes in Phase 3 study of mirikizumab compared to ustekinumab for Crohn's disease < October 14, 2024

Data show more patients treated with mirikizumab achieved histologic response at Week 52 compared to ustekinumab. New data are first-of-its-kind analysis of microscopic mucosal resolution that go beyond endoscopy, setting a new potential standard for the evaluation of therapeutic response.

Majority of children with spinal muscular atrophy (SMA) treated with Roche’s Evrysdi are able to sit, stand and walk independently, two-year data demonstrate < October 14, 2024

Roche presented positive two-year data from the ongoing RAINBOWFISH study at the 29th World Muscle Society (WMS) Congress, 8-12 October, 2024, assessing the efficacy and safety of Evrysdi® (risdiplam) in children with SMA who were treated pre-symptomatically as infants before six weeks of age (n=23). The study found the majority of children achieved key motor milestones, were able to swallow and feed orally, and demonstrated cognitive skills typical of children without SMA, with none requiring permanent ventilation.

TREMFYA® (guselkumab) demonstrates impressive results across biologic-naïve and biologic-refractory patients in Crohn’s disease and ulcerative colitis < JNJ, October 10, 2024

JNJ announced TREMFYA® (guselkumab) data in both Crohn’s disease (CD) and ulcerative colitis (UC) showing high rates of endoscopic remission in both biologic-naïve and biologic-refractory patients (including UC patients refractory to JAK inhibitors), indicating a normal appearance of intestinal mucosa. These subgroup analyses are from pooled data from the Phase 3 GALAXI 2 & 3 studies of TREMFYA® in adults with moderately to severely active CD and the Phase 3 QUASAR maintenance study of TREMFYA® in adults with moderately to severely active UC.

Lundbeck initiates clinical trial in immunology for Lu AG22515 in Thyroid Eye Disease < October 03, 2024

H. Lundbeck A/S (Lundbeck) today announced taking one further step in developing treatments for indications in the neuroimmunology and neuroinflammatory space with the initiation of the first clinical trial of its CD40L blocker, Lu AG22515, in patients. Lundbeck’s proof-of-concept (PoC) trial will evaluate the efficacy, safety, and tolerability of Lu AG22515 as a potential treatment for Thyroid Eye Disease, an autoimmune disease causing a debilitating, disfiguring, and potentially blinding periocular condition.

GSK presents positive data for Arexvy, its respiratory syncytial virus (RSV) vaccine, indicating protection over three RSV seasons < 08 October 2024

GSK announced new data from the AReSVi-006 (Adult Respiratory Syncytial Virus) phase III trial evaluating the efficacy of a single dose of Arexvy (respiratory syncytial virus vaccine, recombinant adjuvanted) against lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in adults aged 60 years and older, including those at increased risk over three full RSV seasons (NCT04886596).

Johnson & Johnson to Discontinue Phase 2 Field Study Evaluating Investigational Antiviral for the Prevention of Dengue < October 4, 2024

Johnson & Johnson is discontinuing the Phase 2 field study (NCT05201794) evaluating the efficacy of investigational antiviral candidate mosnodenvir for the prevention of dengue virus in adults aged 18-65 years. The decision to discontinue this study is part of a strategic reprioritization of the Company’s Communicable Diseases R&D portfolio. No safety issues were identified.

ERLEADA® (apalutamide) demonstrates statistically significant and clinically meaningful improvement in overall survival compared to enzalutamide in patients with metastatic castration-sensitive prostate cancer < JNJ, October 2, 2024

Largest head-to-head real-world study in mCSPC demonstrated that ERLEADA® reduced risk of death by 23 percent at 24 months compared to enzalutamide. JNJ announced the results of a landmark real-world, head-to-head study showing that ERLEADA® (apalutamide) provided a statistically significant overall survival benefit at 24 months compared to enzalutamide in patients with metastatic castration-sensitive prostate cancer (mCSPC).

Business development ... 5

GSK enters agreement to acquire CMG1A46 from Chimagen Biosciences to expand immunology pipeline < SLE, BDL, 29 October 2024

GSK announced an agreement to acquire CMG1A46, a clinical-stage dual CD19 and CD20-targeted T cell-engager (TCE), from Chimagen for $300 million upfront. GSK plans to develop and commercialise CMG1A46 with a focus on B cell-driven autoimmune diseases, such as systemic lupus erythematosus (SLE) and lupus nephritis (LN), with potential to expand into related autoimmune diseases. CMG1A46 is currently in phase I clinical trials in leukaemia and lymphoma in both the US and China. GSK aims to begin a phase I trial in lupus in 2025.

Bayer and Dewpoint Therapeutics sign Licensing Agreement for Heart Disease Program < October 24, 2024

Bayer obtains global license to develop and commercialize disease-modifying treatment specifically for dilated cardiomyopathy (DCM) patients characterized by specific mutations. This is the first option exercise under the research collaboration between Bayer and Dewpoint Therapeutics, Inc., which began in November 2019. It leverages Dewpoint’s proprietary platform for biomolecular condensates and Bayer’s small molecule R&D capabilities to develop new treatments for cardiovascular and renal diseases.

Exelixis and Merck Sign Clinical Development Collaboration to Evaluate Investigational Zanzalintinib in Combination With KEYTRUDA® (pembrolizumab) in Head and Neck Cancer and in Combination With WELIREG® (belzutifan) in Renal Cell Carcinoma < October 14, 2024

EXEL and MRK announced that the companies have entered into a clinical development collaboration to evaluate the combination of Exelixis’ investigational tyrosine kinase inhibitor (TKI) zanzalintinib with Merck’s anti-PD-1 therapy KEYTRUDA® (pembrolizumab) in a Phase 3 pivotal trial for the treatment of patients with head and neck squamous cell carcinoma (HNSCC), and zanzalintinib with WELIREG® (belzutifan), Merck’s oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, in a Phase 1/2 trial and two Phase 3 pivotal trials for the treatment of patients with renal cell carcinoma (RCC).

Astellas and AviadoBio Announce Exclusive Option and License Agreement for Gene Therapy AVB-101 Targeting Frontotemporal Dementia and Other Indications < October 08, 2024

AviadoBio receives $20 million equity investment, up to $30 million in upfront payments and is eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option. AVB-101 is an investigational AAV-based gene therapy in Phase 1/2 development for patients with frontotemporal dementia with progranulin mutations (FTD-GRN).

AstraZeneca strengthens its cardiovascular pipeline with agreement for a pre-clinical novel lipid-lowering therapy < 7 October 2024

AstraZeneca has entered into an exclusive license agreement with CSPC Pharmaceutical Group Ltd (CSPC) to advance the development of an early stage, novel small molecule Lipoprotein (a) (Lp(a)) disruptor that has the potential to offer additional benefits for patients with dyslipidaemia. This asset further strengthens the company’s cardiovascular portfolio to help address the major risk factors driving chronic cardiovascular disease.

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about the Library

The Library integrates web-based information on pharmaceutical industry,

In the Reading Room at the entrance, visitors can find titles of about fifty new articles released by major pharmaceutical companies in a most recent month. By clicking linked keywords in a title, readers can find the full article at the top of previous articles in a binder of the relevant subject. Readers can also easily find other binders of similar subjects in adjacent shelves.

and offers complete overview of the companies, products, and their pharmacology and/or therapeutics.

The extensive, in-depth data are maintained and updated in inter-linked sections in the library: 1) PHARMACOLOGY CLASS, 2) THEAPEUTIC AREA, 3) INDUSTRY (Companies, Management, Market & Environment), and 4) Database. For ease and speed of access, these data have been categorized and stored under various key sub-titles, such as alliance, M&A, and diversification in the "INDUSTRY" section, and, for example, melanoma on the "THERAPEUTICS AREA, and PD-1 inhibitor in the "PHARMACOLOFY CLASS."

Readers will instantly grasp a change in a whole picture of the industry, which is necessary for making intelligent decisions in management and investment.

The New Drug Library, or Pharma Asset Library, is operated on Google Sites platform with easy-to-use navigation tools designed to help you instantly find the relevant data. The Google navigation system allows you to feel as if you are flying over a target freely and taking snapshots from any angle you wish. The service is available in smart phones and tablets as well as desktop computers.

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