Drug News: 21 April 2025

Intriguing science

Treating Parkinson’s disease remains a challenge.

However, there is light at the horizon: Two recent studies, published in Nature on 15 and 16 April 2025, reported on promising phase 1/2a data where patients with Parkinson disease received stem cells injected into the putamen. These stem cells aimed to replace some of the missing dopamine neurons. No significant safety or tolerability issues were identified after the completion of a monitoring period of up to 2 years post-injection. The collected data suggest an increase in dopaminergic activity, while the impact on motor activity was not consistently measured across these 2 studies. Now, both of these groups are working to initiate pivotal clinical trials to collect confirmation of efficacy and safety. And before that, they will probably attempt to increase the proportion of surviving cells.

Tabar, V. et al. Nature https://doi.org/10.1038/s41586-025-08845-y (2025).

Sawamoto, N. et al. Nature https://doi.org/10.1038/s41586-025-08700-0 (2025)

European Medicine Agency (EMA)

The EMA’s Committee for Medicinal Products for Human Use (CHMP) had its monthly meeting from March 24-27, 2025. Here are the key takeaways:

1. Xoanacyl (ferric citrate coordination complex, Averoa) received a positive recommendation for marketing authorization to treat concomitant hyperphosphatemia and iron deficiency in adults with chronic kidney disease.
2. Ryjunea (atropine, Santen Oy) received a positive opinion for slowing the progression of myopia in children aged 3 to 14 years.
3. Kisunla (donanemab, Eli Lilly) received a negative opinion for the treatment of early Alzheimer’s disease. Lilly will seek a re-examination of the decision.
4. Jubereq (denosumab, Accord Healthcare), Qoyvolma (Ustekinumab, Celltrion), and Osvyrti (denosumab, Accord Healthcare) received positive opinions as biosimilar medicines. CHMP meeting highlights.

The European Commission (EC) did take the following decisions:

1. PTC Therapeutics announced that the EC will not renew the conditional marketing authorization for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy, following the CHMP negative opinion. PTC Therapeutics press release.
2. Leqembi (lecanemab, Biogen and Eisai) was approved by the European Commission (EC) for the treatment of adult patients with a clinical diagnosis of mild cognitive impairment (MCI) and mild dementia due to Alzheimer’s disease (early AD). Biogen press release.
3. Columvi (glofitamab, Roche) was approved by the EC for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL). Roche press release.

US Food and Drug Administration (FDA)

Pluvicto (lutetium Lu 177 vipivotide tetraxetan, Novartis) was approved for patients with prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC). Novartis press release.

Qfitlia (fitusiran, Sanofi) was approved to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B, with or without inhibitors. Sanofi press release.

Cardamyst (etripamil, Milestone Pharmaceuticals) nasal spray was rejected for the treatment of acute episodes of paroxysmal supraventricular tachycardia PSVT in adults. The Complete Response Letter (CRL) cited two chemistry, manufacturing and control (CMC) issues. Milestone press release.

Vykat XR (diazoxide choline, Soleno Therapeutics) was approved for the treatment of hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome (PWS). Soleno press release.

Cabometyx (cabozantinib, Exelixis) was approved for adult and pediatric patients 12 years of age and older with previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic neuroendocrine tumors (pNET) and well-differentiated extra-pancreatic neuroendocrine tumors (epNET). Exelixis press release.

Blujepa (gepotidacin, GSK) was approved for the treatment of female adults (≥40 kg) and pediatric patients (≥12 years, ≥40 kg) with uncomplicated urinary tract infections (uUTIs). GSK press release.

Fabhalta (iptacopan, Novartis) was approved for the treatment of adults with C3 glomerulopathy (C3G). Novartis press release.

Tremfya (guselkumab, Johnson & Johnson) was approved for the treatment of adults with moderately to severely active Crohn’s disease. J&J press release.

Amvuttra (vutrisiran, Alnylam Pharmaceuticals) was approved for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults. Alnylam press release.

Uplizna (inebilizumab-cdon, Amgen) was approved for the treatment of adults living with Immunoglobulin G4-related disease (IgG4-RD). Amgen press release.

Vanrafia (atrasentan, Novartis) received FDA accelerated approval for reducing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. Novartis press release.

Imfinzi (durvalumab, AstraZeneca) received a label expansion for use in combination with gemcitabine and cisplatin for the treatment of adult patients with muscle-invasive bladder cancer (MIBC). AstraZeneca press release.

Opdivo (nivolumab, BMS) in combination with Yervoy (ipilimumab, BMS) received two approvals:

• For the treatment of adult and pediatric patients 12 years of age and older with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC).
• For the first-line treatment of adult patients with unresectable or metastatic hepatocellular carcinoma (HCC). BMS press releases [1], [2]

Dupixent (dupilumab, Sanofi and Regeneron) was approved for the treatment of adults and adolescents (12 years and older) with chronic spontaneous urticaria (CSU). Sanofi press release.

Vyvgart Hytrulo prefilled syringe (efgartigimod alfa and hyaluronidase-qvfc, argenx) was approved for the treatment of adult patients with generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP). argenx press release.

Jobevne (bevacizumab-nwgd, Biocon Biologics) was approved as a biosimilar to Avastin (bevacizumab, Roche). Biocon Biologics 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 March 17 and April 20, 2025 per major therapeutic area:

Onco-immunology

1. Immunovant shared positive results from its Phase 3 and Phase 2b studies of batoclimab in patients with myasthenia gravis and chronic inflammatory demyelinating polyneuropathy (CIDP). Immunovant press release.
2. Incyte announced that two Phase 3 trials of povorcitinib (INCB054707) in adult patients with moderate to severe hidradenitis suppurativa (HS) met their primary endpoints. Incyte press release.
3. Mural Oncology announced that the Phase 3 trial of nemvaleukin alfa in combination with Keytruda (pembrolizumab, Merck) in patients with platinum-resistant ovarian cancer (PROC) did not meet its primary endpoint. Mural Oncology press release.
4. BeiGene announced the discontinuation of the clinical development program for ociperlimab as a treatment for lung cancer, after an independent committee ruled that it was unlikely to meet its primary endpoint in a Phase 3 trial. BeiGene press release.
5. BioMarin Pharmaceutical announced that the Phase 3 trial evaluating Palynziq (pegvaliase-pqpz) in adolescents with phenylketonuria (PKU) met its primary efficacy endpoint. BioMarin press release.
6. Rallybio announced the discontinuation of its RLYB212 program for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT), following disappointing results from a Phase 2 trial. Rallybio press release.
7. Johnson & Johnson reported positive results from a Phase 3 study of icotrokinra (JNJ-2113) for the treatment of adolescents with moderate-to-severe plaque psoriasis. J&J press release.
8. Amgen reported that a Phase 3 trial evaluating Imdelltra (tarlatamab-dlle) for the treatment of patients with small cell lung cancer (SCLC) who progressed on or after a single line of platinum-based chemotherapy met its primary endpoint. Amgen press release.

Neurology and Psychiatry

1. Novartis reported positive results from the Phase 3 study of intrathecal onasemnogene abeparvovec (OAV101 IT) in patients aged two to <18 years with spinal muscular atrophy (SMA). Novartis press release.
2. Neurocrine Biosciences shared positive data from Phase 3 study of Ingrezza (valbenazine) for the treatment of tardive dyskinesia. Neurocrine Biosciences press release.
3. Cassava Sciences announced that the Phase 3 REFOCUS-ALZ study of simufilam in mild-to-moderate Alzheimer’s disease (AD) did not meet its primary or secondary endpoints. Cassava press release.
4. Axsome Therapeutics announced that the Phase 3 trial of solriamfetol in the treatment of attention deficit hyperactivity disorder (ADHD) achieved its primary and key secondary endpoints. Axsome press release.
5. Roche announced that the Phase 3 trial comparing a high dose of Ocrevus (ocrelizumab) intravenous (IV) infusion to the currently approved Ocrevus IV 600 mg dose in patients        with relapsing multiple sclerosis (RMS) did not meet its primary endpoint. Roche press release.
6. Transcend Therapeutics announced that the Phase 2 trial evaluating TSND-201 (methylone) for the treatment of PTSD met its primary endpoint. Transcend press release.
7. Alzheon reported that the Phase 3 trial of valiltramiprosate / ALZ-801 in patients with early AD—comprising mild cognitive impairment (MCI) and mild Alzheimer’s dementia—did not meet its primary endpoint. Alzheon press release.

Metabolic and Cardio-vascular

1. vTv Therapeutics announced that the FDA has lifted the clinical hold on the cadisegliatin clinical program. The Phase 3 trial in type 1 diabetes (T1D) is expected to have a reduced duration from 12 to 6 months. vTv Therapeutics press release.
2. Eli Lilly announced positive results from the Phase 2 study of lepodisiran in adults with elevated levels of lipoprotein(a). Eli Lilly press release. And shared positive results from a Phase 3 trial evaluating the safety and efficacy of orforglipron compared to placebo in adults with type 2 diabetes inadequately controlled with diet and exercise alone. Eli Lilly press release.
3. Palatin Technologies announced that the Phase 2 obesity trial of BMT-801 met its primary endpoint. Palatin press release.
4. Rhythm Pharmaceuticals shared positive results from the Phase 3 trial of setmelanotide for the treatment of acquired hypothalamic obesity. Rhythm press release.
5. Pfizer announced the decision to discontinue development of danuglipron (PF-06882961), which was being investigated for chronic weight management, after a case of possible liver damage was identified in one study participant. Pfizer press release.
6. Bristol Myers Squibb (BMS) announced that the Phase 3 trial of Camzyos (mavacamten) for the treatment of adults with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM) did not meet its dual primary endpoints. BMS press release.

Other conditions:

1. Wave Life Sciences shared positive data from the Phase 2 trial of WVE-N531 in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping. Wave Life Sciences press release.
2. AstraZeneca announced that the Phase 3 trial of eneboparatide (AZP-3601) in adults with chronic hypoparathyroidism met its primary endpoint. AstraZeneca press release.
3. Opthea announced that the Phase 3 trial of OPT-302 with aflibercept in patients with wet age-related macular degeneration (AMD) failed to meet its primary endpoint. Opthea press release.
4. UNITY Biotechnology reported positive results from the Phase 2b trial of intravitreal UBX1325 in patients with diabetic macular edema (DME). UNITY Biotechnology press release.
5. Vaxcyte shared positive results from its Phase 2 study of VAX-24 (24-valent pneumococcal conjugate vaccine (PCV)) in healthy infants. Vaxcyte press release.

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