Drug News 02 Sept 2024, EMA, FDA approvals, new Phase 2 Phase 3 clinical data

September 2, 2024

European Medicine Agency (EMA)

The EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) held their monthly meeting this week from August 5th to 8th . The CHMP decisions are to be published.

The CHMP did not issue positive or negative opinions in their August 2024 meeting.

US Food and Drug Administration (FDA)

The FDA approved the following drugs:

Tecelra (afamitresgene autoleucel, Adaptimmune) accelerated approval for the treatment of adults with unresectable or metastatic synovial sarcoma. Tecelra becomes the first engineered cell therapy for a solid tumor and the first TCR-T therapy to enter the market. Adaptimmune press release.
Darzalex Faspro (daratumumab and hyaluronidase-fihj, Johnson and Johnson) in combination with bortezomib, lenalidomide, and dexamethasone for induction and consolidation in patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant (ASCT). J&J press release.
Xembify (immune globulin subcutaneous human-klhw, Grifols) approved for a label expansion to include treatment-naïve patients with primary humoral immunodeficiencies (PI). Grifols press release.
Jemperli (dostarlimab, GSK) received approval for a label expansion in combination with carboplatin and paclitaxel for the treatment of adult patients with primary advanced or recurrent endometrial cancer. GSK press release.
Zurnai (nalmefene hydrochloride Auto-Injector, Purdue Pharma) approved for the emergency treatment of known or suspected opioid overdose induced by natural or synthetic opioids in adults and pediatric patients aged 12 years and older. Purdue Pharma press release.
Voranigo (vorasidenib, Servier Pharmaceuticals) approved for the treatment of grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation. FDA press release.
Lymphir (denileukin diftitox-cxdl, Citius Pharma) was approved for the treatment of adults with relapsed or refractory cutaneous T-cell lymphoma. Citius Pharma press release.
neffy (epinephrine nasal spray, ARS Pharmaceuticals) was approved for the treatment of Type I allergic reactions, including anaphylaxis, in adults and children who weigh ≥30 kg (66 lbs.). ARS press release.
Crexont (carbidopa and levodopa, Amneal Pharma) extended-release capsules was approved for the treatment of Parkinson’s disease (PD). Amneal press release.
Fabhalta (iptacopan, Novartis) was granted FDA accelerated approval for the reduction of proteinuria in primary IgA nephropathy (IgAN). Novartis press release.
Livdelzi (Seladelpar, Gilead) was granted FDA Accelerated Approval for the treatment of primary biliary cholangitis. Gilead press release.
Imfinzi (durvalumab, AstraZeneca) in combination with chemotherapy was approved for the treatment of adult patients with resectable early-stage non-small cell lung cancer (NSCLC) and no known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements. AstraZeneca press release.
Yorvipath (palopegteriparatide, Ascendis Pharma) injection was approved for the treatment of adults with hypoparathyroidism. Ascendis Pharma press release.
Niktimvo (axatilimab-csfr, Incyte) was approved for the treatment of chronic graft-versus-host disease (GVHD). Incyte press release.
Nemluvio (nemolizumab, Galderma) was approved for the treatment of adults with prurigo nodularis. Galderma press release.
ACAM2000 (Smallpox and Mpox (Vaccinia) Vaccine, Live, Emergent BioSolutions) received a label expansion by the FDA to include prevention of mpox disease in individuals determined to be at high risk for mpox infection. Emergent press release.
Novavax COVID-19 Vaccine (Novavax) was granted Emergency Use Authorization (EUA) from the FDA for active immunization to prevent COVID-19 in individuals aged 12 and older. Novavax press release.

The FDA did reject the following applications:

The new drug application (NDA) for midomafetamine capsules (Lykos Therapeutics) for the treatment of post-traumatic stress disorder (PTSD) in adults was rejected. The Complete Response Letter (CRL) indicated the need for an additional Phase 3 study. Lykos press release.

The FDA did take the following decision:

Scemblix (asciminib, Novartis) was granted Priority Review status for the treatment of newly diagnosed adult patients with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP). Novartis 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 in August 2024 per therapeutic area:

Oncology

BioNTech announced positive data from the Phase 2 clinical trial of BNT111 in combination with Libtayo (cemiplimab, Regeneron) in patients with unresectable stage III or IV melanoma whose disease had progressed following anti-PD-(L)1-containing treatment. BioNTech press release.
AstraZeneca shared positive data from the Phase 3 trial of Calquence (acalabrutinib) for the treatment of chronic lymphocytic leukaemia (CLL). AstraZeneca press release.
Imunon shared positive data from the Phase 2 trial of IMNN-001 plus chemotherapy in patients with advanced ovarian cancer. Immunon press release.
FibroGen announced that two Phase 3 trials investigating pamrevlumab in patients with locally advanced, unresectable pancreatic cancer and patients with metastatic pancreatic ductal adenocarcinoma failed to meet their primary endpoints, triggering an immediate termination of the program and the related team. FibroGen press release.
ALX Oncology shared positive data from its Phase 2 trial of evorpacept in patients with HER2-positive gastric or gastroesophageal junction (GEJ) cancer. ALX Oncology press release.
Ultimovacs announced that the Phase 2 study of UV1 vaccine in combination with Keytruda (pembrolizumab, Merck) in patients with metastatic or recurrent head and neck cancer failed to meet its primary and secondary endpoints. Ultimovacs press release.
Sanofi reported positive data from the Phase 3 study of Sarclisa (isatuximab) in combination with standard-of-care lenalidomide, bortezomib, and dexamethasone (RVd) induction treatment in patients with multiple myeloma eligible for transplant. Sanofi press release.
Merck discontinued the Phase 3 trial evaluating a fixed-dose combination of vibostolimab and pembrolizumab with chemotherapy for extensive-stage small cell lung cancer (ES-SCLC). The company’s decision follows a recommendation from an independent Data Monitoring Committee (DMC). Merck press release.
Incyte reported positive results from the Phase 3 study of Monjuvi (tafasitamab) in patients with relapsed or refractory follicular lymphoma (FL). Incyte plans to file for a label extension by the end of the year. Incyte press release.
Syros Pharmaceuticals announced that it will discontinue enrollment in the Phase 2 clinical trial evaluating the triplet regimen of tamibarotene in combination with venetoclax and azacitidine in newly diagnosed, unfit patients with acute myeloid leukemia (AML) and RARA gene overexpression. This decision followed a prespecified interim analysis that indicated a low likelihood of success. Syros Pharmaceuticals press release.
Verrica Pharmaceuticals shared positive results from its Phase 2 trial of VP-315 for the treatment of basal cell carcinoma. Verrica press release.
Merck announced the early termination of two Phase 3 trials of Keytruda (pembrolizumab) as adjuvant therapy for lung and skin cancer. The trials were stopped following recommendations from independent data analysts. Merck press release.
NuCana announced the discontinuation of the Phase 2 study of NUC-3373 in patients with colorectal cancer. The decision was made after a pre-planned initial analysis indicated that the study was unlikely to achieve its primary objective. NuCana press release.

Cardio-metabolic disorders

vTv Therapeutics announced the FDA has placed a hold on their cadisegliatin clinical program which includes an ongoing Phase 3 trial in type 1 diabetes. The clinical hold was based on the discovery of a chromatographic signal in a recent human absorption, distribution, metabolism, and excretion (ADME) study of cadisegliatin. vTv press release.
Eli Lilly shared positive topline results from the Phase 3 trial of tirzepatide injection in adults with heart failure with preserved ejection fraction (HFpEF) and obesity. Lilly press release.
NewAmsterdam Pharma reported positive data from the Phase 3 trial of obicetrapib in adult patients with heterozygous familial hypercholesterolemia (HeFH). NewAmsterdam Pharma press release.
Diamyd Medical reported positive interim results from the Phase 3 trial of Diamyd in type 1 diabetes. Diamyd press release.
Bayer announced that the Phase 3 study of finerenone in patients with heart failure with mildly reduced or preserved ejection fraction met its primary endpoint. Bayer press release.
Rivus Pharmaceuticals announced that its Phase 2a trial of HU6 in patients with obesity-related heart failure with preserved ejection fraction (HFpEF) met both its primary and secondary endpoints. Rivus press release [pdf].
Novartis revealed that the Phase 3 trial of Leqvio (inclisiran) in patients at low or moderate risk of atherosclerotic cardiovascular disease (ASCVD) met its primary endpoints. Novartis press release.

Neurology

Longeveron announced that the Phase 2a trial of Lomecel-B for treating mild Alzheimer’s disease met its primary safety and secondary efficacy endpoints. Longeveron press release.
Anavex Life Sciences presented positive results from the Phase 2b/3 study of blarcamesine (ANAVEX 2-73) in people with early Alzheimer’s disease (AD). The company plans to submit an application to the EMA for approval before the end of 2024. Anavex press release.
PepGen reported positive results from the low dose cohort (5mg/kg) of the Phase 2 trial of PGN-EDO51 for the treatment of Duchenne muscular dystrophy (DMD). PepGen press release.
Vaccinex’s announced that the Phase 1b/2 trial evaluating its Alzheimer’s disease treatment pepinemabin met its primary endpoint. Vaccinex press release.
Neurocrine Biosciences reported positive news from the Phase 2 study of NBI-1117568 (NBI-‘568) in adults with schizophrenia. Neurocrine press release.

Other indications:

Ventyx Biosciences announced that the Phase 2 trial of VTX958 in patients with moderately to severely active Crohn’s disease did not meet its primary endpoint. The company will stop funding further trials of VTX958. Ventyx press release.
Celldex Therapeutics announced positive results from the Phase 2 trial of barzolvolimab in patients with chronic inducible urticaria (CIndU). Celldex press release.
Johnson & Johnson published positive results from the Phase 2 trial evaluating nipocalimab in pregnant women at high risk of developing haemolytic disease of the foetus and newborn (HDFN). J&J press release.
Sobi and Apellis Pharmaceuticals announced positive data from the Phase 3 study of pegcetacoplan in treating C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). Apellis plans to submit an approval application to the FDA early next year, while Sobi plans to do the same with the EMA sometime in 2025. Apellis press release.
Aldeyra Therapeutics announced that the Phase 3 trial of reproxalap for dry eye disease met its primary endpoint. Aldeyra press release.
Pfizer announced positive results from the Phase 3 trial evaluating two doses of Abrysvo vaccine (RSV bivalent vaccine) in immunocompromised adults aged 18 and older at risk of developing severe respiratory syncytial virus (RSV)-associated lower respiratory tract disease (LRTD). Pfizer press release.
Pfizer and BioNTech announced that a Phase 3 trial of their combination mRNA vaccine for the treatment of influenza and Covid-19 failed to meet one of its two primary endpoints. Pfizer press release.
Acelyrin announced that the Phase 3 trial of izokibep in Hidradenitis Suppurativa (HS) achieved its primary endpoint. Acelyrin press release.
The National Institutes of Health’s (NIH) National Institute of Allergy and Infectious Diseases (NIAID) announced that a study evaluating tecovirimat (SIGA Technologies) in children and adults with clade I monkeypox (mpox) in the Democratic Republic of the Congo (DRC) did not meet its primary endpoint. SIGA press release.
Invivyd announced positive data from the Phase 3 trial of pemivibart for the pre-exposure prophylaxis (PrEP) of COVID-19. Invivyd press release.