Drug News 21 October to 18 November 2024

European Medicine Agency

The EMA’s Committee for Medicinal Products for Human Use (CHMP) held its monthly meeting from November 11-14. Here are the key takeaways:

1. Augtyro (repotrectinib, Bristol-Myers Squibb) received conditional marketing authorization for treating adults and adolescents with advanced solid tumors, as well as adults with locally advanced or metastatic non-small cell lung cancer (NSCLC).
2. Gohibic (vilobelimab, InflaRx) was recommended for a marketing authorization under exceptional circumstances for the treatment of adult patients with SARS‑CoV2‑induced acute respiratory distress syndrome.
3. Lazcluze (lazertinib, Johnson & Johnson) received a positive opinion for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLSC) in combination with amivantamab.
4. Baiama and Ahzantive (aflibercept, Klinge Biopharma) received positive opinions as biosimilars to Eylea (Bayer and Regeneron) for the treatment of neovascular age-related macular degeneration, and visual impairment due to macular edema.
5. Obodence and Xbryk (denosumab, Samsung Bioepis) received positive as biosimilars to Prolia and Xgeva (Amgen) for the treatment of osteoporosis and bone loss, and the prevention of skeletal-related events in adults with advanced malignancies involving bone.
6. Leqembi (lecanemab, Biogen and Eisai) received a positive opinion following re-examination for the treatment of mild cognitive impairment or mild dementia due to Alzheimer’s disease (early Alzheimer’s disease).
7. Cinainu (allium/citrus/paullinia/cacao, Legacy Healthcare) received a negative opinion for the treatment of moderate to severe alopecia areata.
8. Kizfizo (temozolomide, Orphelia Pharma) received a negative opinion for the treatment of neuroblastoma. CHMP meeting highlights.

US Food and Drug Administration (FDA)

The following drugs were approved by the FDA:

1. Danziten (nilotinib, Azurity Pharmaceuticals) was approved for adult patients with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase and adult patients with chronic phase (CP) and acute phase (AP) resistant or intolerant to prior therapy that included imatinib. Azurity press release.
2. Kebilidi (eladocagene exuparvovec-tneq, PTC Therapetics) was approved for the treatment of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency. PTC press release.
3. Revuforj (revumenib, Syndax Pharmaceuticals) was approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients one year and older. Syndax press release.
4. Abrysvo (Respiratory Syncytial Virus Vaccine, Pfizer) receiveda label expansion approval for the prevention of lower respiratory tract disease (LRTD) caused by RSV in individuals aged 18 to 59 who are at increased risk for LRTD caused by RSV. Pfizer press release.
5. Orlynvah (sulopenem etzadroxil/ probenecid, Iterum Therapeutics) was approved for the treatment of uncomplicated urinary tract infections (uUTIs). Iterum press release.

The application of Ocaliva (obeticholic acid, Intercept Pharmaceuticals) was rejected for the treatment of indicated patients with primary biliary cholangitis (PBC). The Complete Response Letter (CRL) cited safety data from Study 747-302, along with other safety information. Intercept press release.

The FDA has proposed removing oral phenylephrine as an active ingredient in over-the-counter (OTC) medications for temporary nasal congestion relief. This decision follows a review of existing data, which concluded that oral phenylephrine is ineffective for this purpose. FDA press release.

New data from clinical trials (Phase 2, Phase 3)

Here are the recent updates on Phase 2 and Phase 3 clinical trials with published data between the 21st October and 17th November 2024 by therapeutic area.

Onco-immunology:

1. AstraZeneca and Merck announced that a Phase 3 trial of Koselugo (selumetinib) in adults with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas met its primary endpoints. AstraZeneca press release.
2. Syndax Pharmaceuticals shared positive results from Phase 2 study of revumenib in patients with relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukemia (AML). Syndax press release.
3. Syros Pharmaceuticals announced that the Phase 3 trial evaluating tamibarotene in combination with azacitidine in newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) patients with RARA gene overexpression did not meet its primary endpoint. Syros press release.
4. Adaptimmune Therapeutics announced that the Phase 2 trial of lete-cel in patients with synovial sarcoma or myxoid/round cell liposarcoma (MRCLS) met its primary endpoint. Adaptimmune press release.
5. Johnson & Johnson reported positive results from the Phase 2 study of nipocalimab in adult patients with moderate-to-severe Sjögren’s disease (SjD). J&J press release.
6. GSK shared positive results from the Phase 3 trial of Blenrep (belantamab mafodotin) in combination with bortezomib plus dexamethasone (BorDex) as a second-line or later treatment for relapsed or refractory multiple myeloma. GSK press release.
7. Beam Therapeutics shared positive news from the Phase 1/2 trial of BEAM-101 in patients with Sickle Cell Disease. Beam press release.
8. Eli Lilly shared positive results from two Phase 3 studies of mirikizumab in patients with inflammatory bowel diseases (IBD), ulcerative colitis (UC) and Crohn’s disease. Eli Lilly press release.
9. Roche announced positive results from the Phase 3 trial evaluating 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. Roche press release.
10. Johnson & Johnson reported positive results from the Phase 3 study of Tremfya (guselkumab) in patients with Crohn’s disease. J&J press release.
11. Otsuka Pharmaceutical announced that the Phase 3 study of sibeprenlimab for the treatment of immunoglobulin A nephropathy (IgA nephropathy) in adults met its primary endpoint. Otsuka press release.
12. Intellia Therapeutics shared positive results from the Phase 2 study of NTLA-2002 in patients with hereditary angioedema (HAE). Intellia Therapeutics press release.
13. Novavax announced that the FDA lifted the clinical hold on its Investigational New Drug (IND) application for its COVID-19-Influenza Combination (CIC) and stand-alone influenza vaccine candidates. The FDA has cleared Novavax to begin enrolling the planned Phase 3 trial after the company satisfactorily addressed the FDA’s concerns. Novavax press release.

Neurology

1. AbbVie announced that its two Phase 2 trials investigating emraclidine in adults with schizophrenia experiencing an acute exacerbation of psychotic symptoms did not meet their primary endpoint. AbbVie press release.
2. Neurogene shared positive interim data from the Phase 1/2 trial of NGN-401 gene therapy for the treatment of female pediatric patients with Rett syndrome. Neurogene press release.
3. AC Immune announced positive data from the Phase 2 trial of ACI-7104.056 for the treatment of patients with early Parkinson’s disease (PD). AC Immune press release.
4. Intra-Cellular Therapies announced positive results from a Phase 3 study assessing the efficacy and safety of lumateperone in preventing relapse in adult patients with schizophrenia. Intra-Cellular press release.
5. Eli Lilly reported positive results from the Phase 3b study of donanemab in adults with early symptomatic Alzheimer’s disease (AD). Eli Lilly press release.
6. UCB reported data from the Phase 2a study of bepranemab in people living with prodromal to mild Alzheimer’s disease. The trail’s primary endpoint was not met, however in key secondary endpoints bepranemab slowed cognitive decline and rate of tau accumulation. UCB press release.
7. Zai Lab reported positive results from its Phase 3 trial of KarXT (xanomeline and trospium chloride) in China to treat schizophrenia. Zai Lab press release.
8. ESSA Pharma announced the termination of the Phase 2 study of masofaniten combined with enzalutamide in patients with metastatic castration-resistant prostate cancer. The decision was based on an interim analysis that determined a low likelihood of meeting the prespecified primary endpoint of the study. ESSA Pharma press release.
9. Alto Neuroscience announced that the Phase 2b study of ALTO-100 in patients with major depressive disorder (MDD) did not meet the primary endpoint. Alto press release.
10. Marinus Pharmaceuticals reported that the Phase 3 trial of oral ganaxolone in treating seizures linked to tuberous sclerosis complex (TSC) in children and adults did not meet its primary endpoint. Marinus press release.

Cardiovascular – metabolic

1. Eli Lilly reported positive news from the Phase 3 study of tirzepatide in treating pre-diabetic and obese individuals. Eli Lilly press release.
2. Novo Nordisk announced positive results from two Phase 3 trials of semaglutide: in adults with metabolic dysfunction-associated steatohepatitis (MASH) and moderate to advanced liver fibrosis (stage 2 or 3). Novo Nordisk press release. And oral semaglutide for the prevention of major adverse cardiovascular events (MACE). Novo Nordisk press release.

Other conditions

1. Microbion Corporation shared positive results from the Phase 2 study of topical pravibismane for diabetic foot ulcer infections (DFI). Microbion press release.
2. Amgen and AstraZeneca announced that the Phase 3 trial of Tezspire (tezepelumab-ekko) in patients with chronic rhinosinusitis with nasal polyps (CRSwNP) met both co-primary endpoints. Amgen press release.
3. Spero Therapeutics announced that the Phase 2a proof-of-concept study of SPR720 for the treatment of Nontuberculous Mycobacterial Pulmonary Disease (NTM-PD) did not meet its primary endpoint. The company suspend the development program for SPR720. Spero Therapeutics press release [pdf].