Retatrutide, a possible advance on GLP-1 drugs like Zepbound, significantly cut blood sugar and body weight in the study, a sign it might be similarly impactful against obesity.

Original title: “Lilly’s three-pronged obesity drug hits goal in large diabetes trial”

Sarepta Therapeutics says the FDA has agreed to review a regulatory package for Amondys 45 and Vyondys 53 after they failed a confirmatory trial, but whether the agency will agree to approve them is still unknown.

Original title: “Sarepta Plans FDA Run for Duchenne Exon Skippers Despite Confirmatory Trial Failure”

S-606001 is currently under investigation in clinical trials for the treatment of late-onset Pompe disease. The safety and effectiveness of S-606001 have not been established, nor has it been approved by FDA or any health authority. About Shionogi in Rare Disease Shionogi is committed to the ... S-606001 is currently under investigation in clinical trials for the treatment of late-onset Pompe disease. The safety and effectiveness of S-606001 have not been established, nor has it been approved by FDA or any health authority. About Shionogi in Rare Disease Shionogi is committed to the research and development of innovative medicines that address unmet medical needs for people worldwide. There is a significant unmet need for new treatment approaches that can be complementary to existing treatments to further slow disease progression,” said Juan Carlos Gomez, M.D., Chief Medical Officer, Shionogi & Co., Ltd. “This is an important milestone for Shionogi, as we continue to expand our work in rare disease, and we hope it will prove to be an important step forward for the Pompe community.” · Shionogi acquired exclusive worldwide rights for S-606001 (previously known as MZE001) from Maze Therapeutics, Inc. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter “Shionogi”) announced the first patients were enrolled in Esprit, a global Phase 2 clinical trial evaluating S-606001, an investigational drug for the treatment of late-onset Pompe disease (LOPD). Esprit is a multicenter, randomized, placebo-controlled, double-blind study evaluating the safety, pharmacodynamics and preliminary efficacy of S-606001 as an oral substrate reduction therapy (SRT) in addition to standard of care enzyme replacement therapy (ERT) in adults with a confirmed diagnosis of LOPD.1 This 52-week study will enroll participants across the U.S., European Union and United Kingdom.1 In 2025, S-606001 received a rare pediatric disease designation from the U.S. Food and Drug Administration (FDA) for the treatment of Pompe disease, a designation granted for serious and life-threatening diseases that primarily affect children ages 18 years or younger with fewer than 200,000 people in the U.S. The FDA also granted Orphan Drug Designation to the compound in 2022. Additional details about Esprit are available at ClinicalTrials.gov ID: NCT07123155 and on www.espritstudy.com.

Original title: “Shionogi Announces First Patients Enrolled in Global Phase 2 Clinical Trial in Adults with Late-Onset Pompe Disease - BioSpace”

Eli Lilly’s retatrutide could present a “differentiated option” for patients with type 2 diabetes who want to control their blood sugar and achieve maximal weight loss, according to analysts at BMO Capital Markets.

Original title: “Lilly Builds Differentiated Profile for Triple-G Agonist With Late-Stage Diabetes Data”

Early trial shows more than 50% LRRK2 reduction and supports expansion into other neurodegenerative conditions

Original title: “Arvinas reports strong phase 1 results for ARV-102 in Parkinson’s disease”

Lead Alzheimer's candidate shows clear biological effects and antidepressant potential

Original title: “Alzecure shares new preclinical data on NeuroRestore ACD856 at AD/PD conference”

S-606001 is currently under investigation in clinical trials for the treatment of late-onset Pompe disease. The safety and effectiveness of S-606001 have not been established, nor has it been approved by FDA or any health authority. ... Shionogi is committed to the research and development of innovative medicines that address unmet medical needs for people worldwide. Rare ... S-606001 is currently under investigation in clinical trials for the treatment of late-onset Pompe disease. The safety and effectiveness of S-606001 have not been established, nor has it been approved by FDA or any health authority. ... Shionogi is committed to the research and development of innovative medicines that address unmet medical needs for people worldwide. Rare diseases affect individuals and families around the world and treatment options for rare diseases are often limited. There is a significant unmet need for new treatment approaches that can be complementary to existing treatments to further slow disease progression,” said Juan Carlos Gomez, M.D., Chief Medical Officer, Shionogi & Co., Ltd. “This is an important milestone for Shionogi, as we continue to expand our work in rare disease, and we hope it will prove to be an important step forward for the Pompe community.” · Shionogi acquired exclusive worldwide rights for S-606001 (previously known as MZE001) from Maze Therapeutics, Inc. ・S-606001 has the potential to become the first oral substrate reduction therapy for Pompe disease · OSAKA, Japan, March 19, 2026 – Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter “Shionogi”) announced the first patients were enrolled in Esprit, a global Phase 2 clinical trial evaluating S-606001, an investigational drug for the treatment of late-onset Pompe disease (LOPD). In 2025, S-606001 received a rare pediatric disease designation from the U.S. Food and Drug Administration (FDA) for the treatment of Pompe disease, a designation granted for serious and life-threatening diseases that primarily affect children ages 18 years or younger with fewer than 200,000 people in the U.S. The FDA also granted Orphan Drug Designation to the compound in 2022. Additional details about Esprit are available at ClinicalTrials.gov ID: NCT07123155 and on www.espritstudy.com.

Original title: “Shionogi Announces First Patients Enrolled in Global Phase 2 Clinical Trial in Adults with Late-Onset Pompe Disease｜ 塩野義製薬”

S-606001 is currently under investigation in clinical trials for the treatment of late-onset Pompe disease. The safety and effectiveness of S-606001 have not been established, nor has it been approved by FDA or any health authority. ... Shionogi is committed to the research and development of innovative medicines that address unmet medical needs for people worldwide. Rare ... S-606001 is currently under investigation in clinical trials for the treatment of late-onset Pompe disease. The safety and effectiveness of S-606001 have not been established, nor has it been approved by FDA or any health authority. ... Shionogi is committed to the research and development of innovative medicines that address unmet medical needs for people worldwide. Rare diseases affect individuals and families around the world and treatment options for rare diseases are often limited. There is a significant unmet need for new treatment approaches that can be complementary to existing treatments to further slow disease progression,” said Juan Carlos Gomez, M.D., Chief Medical Officer, Shionogi & Co., Ltd. “This is an important milestone for Shionogi, as we continue to expand our work in rare disease, and we hope it will prove to be an important step forward for the Pompe community.” · Shionogi acquired exclusive worldwide rights for S-606001 (previously known as MZE001) from Maze Therapeutics, Inc. ・S-606001 has the potential to become the first oral substrate reduction therapy for Pompe disease · OSAKA, Japan, March 19, 2026 – Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter “Shionogi”) announced the first patients were enrolled in Esprit, a global Phase 2 clinical trial evaluating S-606001, an investigational drug for the treatment of late-onset Pompe disease (LOPD). In 2025, S-606001 received a rare pediatric disease designation from the U.S. Food and Drug Administration (FDA) for the treatment of Pompe disease, a designation granted for serious and life-threatening diseases that primarily affect children ages 18 years or younger with fewer than 200,000 people in the U.S. The FDA also granted Orphan Drug Designation to the compound in 2022. Additional details about Esprit are available at ClinicalTrials.gov ID: NCT07123155 and on www.espritstudy.com.

Original title: “Shionogi Announces First Patients Enrolled in Global Phase 2 Clinical Trial in Adults with Late-Onset Pompe Disease｜ 塩野義製薬”

The clearance is the first for an oral peptide that blocks IL-23, the target of popular medicines like Skyrizi and Tremfya. J&J has said the new drug, Icotyde, has the potential to top $5 billion in sales.

Original title: “J&J wins FDA nod for psoriasis pill that aims to compete with top-selling injections”

Aspen Neuroscience presented positive Phase 1/2 data for its stem cell-derived therapy. Elsewhere, Aldeyra lost over half its market value following a rejection from the FDA.

Original title: “Aspen’s fresh Parkinson’s data; Aldeyra tanks following FDA rejection”

Icotyde, co-developed by Johnson & Johnson and Protagonist Therapeutics, is backed by data from the Phase 3 ICONIC program, which, among other advantages, showed significant superiority over Bristol Myers Squibb’s Sotyktu.

Original title: “J&J Wins FDA Nod for First Targeted Oral Anti-IL-23 Therapy for Plaque Psoriasis”

Study to explore mechanism behind novel oral therapy for muscle‑sparing weight loss

Original title: “Atrogi begins dosing first subjects in human trial of ATR-258”

Closer to Cures: Cory R. Nicholas, ... in clinical trials for patients with epilepsy that does not respond to drugs. Alex on CIRM-funded treatment for cancer granted FDA breakthrough therapy designation · Kiara on Winner of Beast Games becomes champion for his son’s rare disease... Closer to Cures: Cory R. Nicholas, CEO and co-founder of Neurona Therapeutics, discusses progress in clinical trials for patients with epilepsy that does not respond to drugs. Alex on CIRM-funded treatment for cancer granted FDA breakthrough therapy designation · Kiara on Winner of Beast Games becomes champion for his son’s rare disease Closer to Cures: Cory R. Nicholas, CEO and co-founder of Neurona Therapeutics, discusses progress in clinical trials for patients with epilepsy that does not respond to drugs. CIRM board supports a rare disease funding plan, administrative changes The Langenhop children, diagnosed with a rare, life-threatening immune disease, had their immune function successfully restored through a CIRM-funded gene therapy trial by Rocket Pharmaceuticals. Adult stem cells Alpha Stem Cell Clinic network ALS Alzheimer's disease Asterias Biotherapeutics Autism Blindness Blood stem cells Bone marrow transplant Brain Breast cancer Bridges Program California Institute for Regenerative Medicine cancer cancer stem cells Cedars-Sinai Cell Stem Cell CIRM CIRM Board CIRM funding City of Hope clinical trial clinical trials Coronavirus COVID-19 CRISPR diabetes Discovery Research Dr. Maria Millan Duchenne Muscular Dystrophy embryonic stem cells FDA Food and Drug Administration Gene editing gene therapy Gladstone Institutes heart disease HIV/AIDS induced plur

Original title: “CIRM-funded clinical trial gives siblings a second chance at life”

Rhythm said that the failed trial of Imcrivee suffered a high dropout rate, which impacted the primary endpoints. No new safety signals were observed with Imcrivee, and the safety profile was consistent with prior clinical studies and commercial experience. The most common treatment-emergent adverse events (TEAEs) included skin hyperpigmentation, injection site reactions, nausea, vomiting, and headache. There had been a very high drop out rate in the trial, the company confirmed during an investor call. As a result, when using post hoc analysis using last observation carried forward for missing values, Imcrivee achieved statistically significant and clinically meaningful BMI reductions in the modified intent-to-treat patient populations in the POMC/PCSK1 Hets (5.5%) and SRC1 (6.2%). Dr David Meeker, CEO of Rhythm Pharmaceuticals, said: “While we are disappointed the EMANATE substudies did not meet their primary endpoint, we are encouraged by compelling signals from additional analyses of the heterozygous POMC/PCSK1 and SRC1 substudies and learnings that sharpen our ability to identify true loss-of-function variants and inform the development of our next-generation MC4R agonists in rare genetically driven obesity indications.” ... Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strat A Phase III trial of Rhythm Pharmaceuticals’ Imcivree (setmelanotide) failed to meet its primary endpoints in four rare, genetically driven obesities of the MC4R pathway.

Original title: “Rhythm’s Imcivree fails Phase III rare disease obesity trial - Clinical Trials Arena”

The drug, which one analyst described as a “critical” part of Pfizer’s oncology strategy, represents a possible improvement upon Ibrance and other, similar breast cancer medicines from Eli Lilly and Novartis.

Original title: “Pfizer’s Ibrance successor moves forward with new study data”

In its complete response letter, the FDA said Aldeyra had failed to demonstrate reproxalap’s efficacy in adequate and well-controlled studies. The FDA previously turned the candidate away in November 2023 and April 2025.

Original title: “Third FDA Rejection for Aldeyra’s Dry Eye Disease Drug Sends Shares Plummeting”

Pfizer has a lofty goal for the CDK4 inhibitor atirmociclib, the New York pharma’s answer to Ibrance’s loss of patent protection next year. In 2025, Ibrance led Pfizer’s oncology portfolio with $1.04 billion in sales.

Original title: “Pfizer’s Ibrance Heir Reduces Risk of Death in Phase 2 Breast Cancer Test”

The Phase 3 EMANATE study is a basket trial looking at the efficacy of Rhythm Pharmaceuticals’ injectable obesity drug across four types of obesities driven by specific genetic mutations.

Original title: “Rhythm’s Imcivree Fails Late-Stage Weight Loss Trial in Rare Obesities”

ATALANTA-1 data presented at ASH 2025 showed remarkable response rates and rapid manufacturing with an investigational autologous CAR T-cell therapy. ATALANTA-1 data presented at ASH 2025 showed remarkable response rates and rapid manufacturing with an investigational autologous CAR T-cell therapy. ... ASH 2025 data on the phase 2 TrAVeRse study evaluating acalabrutinib, venetoclax, and rituximab (AVR) in treatment-naive disease. He views AVR as a clear example of how frontline, chemo-free therapy may offer patients durable disease control with substantially reduced toxicity, positioning such regimens as a new standard of care for treatment-naive MCL. Stay ahead of policy, cost, and value—subscribe to AJMC for expert insights at the intersection of clinical care and health economics. Wang traces the field’s progress and how the advent of BTK inhibitors—beginning with ibrutinib and followed by newer-generation agents such as acalabrutinib—marked a turning point by delivering high response rates with oral therapy and substantially less toxicity. The addition of venetoclax further deepened responses, laying the groundwork for combination strategies capable of rivaling, and in some cases surpassing, outcomes historically achieved with chemotherapy. The TrAVeRse trial confirmed earlier single-center and smaller studies, demonstrating nearly universal overall response rates and complete response rates approaching 80%. These outcomes were consistent across risk groups, reinforcing the robustness of the regimen.

Original title: “Moving From Chemotherapy to Chemo-Free Frontline Care in MCL | AJMC”

Company shares spiked more than 60% after the therapy surpassed Wall Street benchmarks in Phase 1 testing against late-line colorectal tumors.

Original title: “CytomX surges on positive data for ‘masked’ ADC in colorectal cancer”