Drug news June 2024, approvals, clinical trials reported data

European Medicine Agency (EMA)

The CHMP meeting is planned this week, starting June 24th.

The PRAC, EMA’s Pharmacovigilance Risk Assessment Committee, had their monthly meeting this week from June 10thto June 13th. Here are the key takeaways:

1. The PRAC concluded that secondary malignancies of T-cell origin may occur after treatment with chimeric antigen receptor (CAR) T-cell medicines. Patients treated with CAR T-cell medicines should be monitored life-long for secondary malignancies.
2. The PRAC discussed a direct healthcare professional communication (DHPC) regarding CAR T-cell medicines. The DHPC will inform healthcare professionals of the PRAC’s review conclusion on secondary malignancies of T-cell origin, including CAR-positive malignancies.

The EMA has started a review of medicines containing the painkiller metamizole following concerns that the measures in place to minimize the known risk of agranulocytosis may not be effective enough. PRAC meeting highlights.

Stay tuned for the outcome of the CHMP meeting!

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US Food and Drug Administration (FDA)

In the last 2 weeks, the FDA did take the following decisions:

1. Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc, argenx) was approved for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP). Argenx press release.
2. Capvaxive (Pneumococcal 21-valent Conjugate Vaccine, Merck) approved for the prevention of invasive pneumococcal disease and pneumococcal pneumonia in adults. Merck press release.
3. Sofdra (sofpironium, Botanix Pharmaceuticals) topical gel was approved to treat primary axillary hyperhidrosis (excessive underarm sweating) in patients ages 9 and older. Botanix press release.
4. Skyrizi (risankizumab-rzaa, AbbVie) approved for adults with moderately to severely active ulcerative colitis. AbbVie press release.
5. Krazati (adagrasib) received Accelerated Approval in combination with Cetuximab for adult patients with previously treated KRAS G12C-mutated locally advanced or metastatic colorectal cancer (CRC). BMS press release.
6. Indication extension of Elevidys (delandistrogene moxeparvovec-rokl, Sarepta Therapeutics), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD). Sarepta press release.
7. Retevmo (selpercatinib, Eli Lilly) approved for adult and pediatric patients 2 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer. FDA press release.
8. Augtyro (repotrectinib, BMS) was granted accelerated approval for the treatment of patients with NTRK-positive locally advanced or metastatic solid tumors. BMS press release.
9. Farxiga (dapagliflozin, AstraZeneca) approved for the treatment of paediatric type-2 diabetes. AstraZeneca press release.
10. Kevzara (sarilumab, Regeneron) was approved for patients with active polyarticular juvenile idiopathic arthritis (pJIA). Regeneron press release.
11. Approval of Imfinzi (durvalumab, AstraZeneca) in combination with carboplatin plus paclitaxel for adult patients with primary advanced or recurrent endometrial cancer that is mismatch repair deficient (dMMR). FDA press release.
12. Accelerated approval for Iqirvo (elafibranor, Ipsen) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA). Ipsen press release.
13. Blincyto (blinatumomab, Amgen) was approved for the treatment of adult and pediatric patients one month or older with CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (B-ALL) in the consolidation phase. Amgen press release.
14. Another approval for Keytruda (pembrolizumab, Merck) with carboplatin and paclitaxel for adult patients with primary advanced or recurrent endometrial carcinoma. FDA press release.
15. Klisyri (tirbanibulin, Almirall) was granted a label extension by the FDA for the treatment of actinic keratosis on expanded area of face or scalp up to 100 cm2. Almirall press release.

In addition, Tagrisso (Osimertinib, AstraZeneca) was granted a priority review for an indication extension. And IBI343 (TOPO1i anti-CLDN18.2 ADC, Innovent) received a fast track designation.

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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 or updates in the last 2 weeks, between the 10th and 23rd of June 2024 in immunology / oncology:

1. AstraZeneca announced that the Phase 3 trial for Truqap (capivasertib) in combination with paclitaxel in patients with locally advanced (inoperable) or metastatic triple-negative breast cancer (TNBC) did not meet its primary endpoints. AstraZeneca press release.
2. Regeneron Pharmaceuticals reported positive results from the Phase 1/2 trial of linvoseltamab in patients with relapsed/refractory (R/R) multiple myeloma (MM). Regeneron press release.
3. Takeda shared positive long-term findings from the Phase 3 trial of Hyqvia (Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase) in treating chronic inflammatory demyelinating polyneuropathy (CIDP). Takeda press release.
4. Pfizer shared positive results from the Phase 2 study of Elrexfio (elranatamab-bcmm) in patients with heavily pretreated relapsed or refractory multiple myeloma (RRMM). Pfizer press release.
5. UroGen Pharma reported positive results from the Phase 3 study of UGN-102 (mitomycin) in patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). UroGen press release.
6. Roche shared positive results from its Phase 3 study of Columvi (glofitamab) in combination with gemcitabine and oxaliplatin (GemOx) for patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL). Roche press release.
7. Zentalis Pharmaceuticals announced that the FDA has placed a partial clinical hold on three Phase 1/2 studies of azenosertib following two patient deaths due to presumed sepsis. The drug is developed in several cancer indications. Zentalis press release.

In Neurology/Psychiatry, the following insights were reported:

1. Jazz Pharmaceuticals announced that the Phase 2b trial evaluating the efficacy and safety of suvecaltamide (JZP385) in patients with essential tremor did not meet its primary or secondary endpoints. The company will wait for results from a separate trial focused on Parkinson’s disease tremor before deciding on the next steps for the program. Jazz press release.
2. PTC Therapeutics announced that the FDA has lifted the partial clinical hold on the Phase 2 study of PTC518 in Huntington’s disease (HD) patients. This decision follows interim results showing the drug’s efficacy, safety, and tolerability. PTC press release.
3. Takeda announced that two Phase 3 studies of soticlestat for patients with the rare epilepsies Lennox-Gastaut syndrome (LGS) and Dravet syndrome have missed their primary endpoints. Takeda press release.
4. Marinus Pharmaceuticals reported mixed results from the Phase 3 trial of intravenous (IV) ganaxolone for treating Refractory Status Epilepticus (RSE). The trial met only one of two key endpoints. Marinus press release.
5. Intra-Cellular Therapies announced that the Phase 3 study evaluating Caplyta (lumateperone) as an adjunctive therapy in patients with major depressive disorder (MDD) met its primary and key secondary endpoints. Intra-Cellular press release.
6. Woolsey Pharmaceuticals announced positive results from its Phase 2a study of Bravyl in patients with Amyotrophic Lateral Sclerosis (ALS). Woolsey press release.
7. Pfizer reported that its Phase 3 trial of fordadistrogene movaparvovec, an investigational gene therapy, for the treatment of ambulatory boys aged four to seven years with Duchenne muscular dystrophy (DMD), failed to achieve its primary endpoint. Pfizer press release.

In other therapeutic areas, the following studies shared updates:

1. Gilead shared positive results from its Phase 3 trial of its HIV drug Sunlenca (lenacapavir). The drug prevented 100% of HIV cases in cisgender women. Gilead press release.
2. Aerovate Therapeutics announced that the Phase 2b/3 study of AV-101 in adults with pulmonary arterial hypertension (PAH) did not meet its primary or secondary endpoints. Aerovate press release.
3. Aphaia Pharma announced that its Phase 2 trial evaluating APHD-012 for prediabetes treatment met its primary endpoint. Aphaia press release.
4. Moderna announced that the two Phase 3 trials trial of mRNA-1083 (combination vaccine against influenza and COVID-19) and mRNA-1283 (next-generation Covid-19 vaccine) have met their primary endpoints. Moderna press releases [1],[2].
5. Ultragenyx Pharmaceutical and Mereo BioPharma announced positive results from the Phase 2 portion of the ongoing Phase 2/3 study of setrusumab (UX143) in patients with Osteogenesis Imperfecta (OI). Ultragenyx press release.
6. Skye Bioscience announced that its Phase 2a clinical trial of SBI-100 Ophthalmic Emulsion in patients with primary open-angle glaucoma or ocular hypertension did not meet its primary endpoint. Skye intends to discontinue clinical development and spending related to SBI-100. Skye press release.
7. Vertex Pharmaceuticals reported positive long-term data from several trials of its gene therapy Casgevy (exa-cel) in patients with sickle cell disease or transfusion-dependent beta-thalassemia. Vertex press release.
8. Cara Therapeutics announced that the Phase 2/3 study of oral difelikefalin in adult patients with moderate-to-severe pruritus with notalgia paresthetica (NP) did not meet its primary endpoint, resulting in the company’s decision to discontinue the clinical program in NP. Cara press release.
9. Oculis announced positive results from its Phase 2b trial of licaminlimab in patients with dry eye disease (DED). Oculus press release.