PeptideJournal
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2025-2026 Peptide Research Highlights

The past eighteen months have been among the most consequential in peptide science. A triple-agonist injectable posted the largest weight-loss numbers ever recorded in a Phase 3 trial. The FDA approved the first-ever drug targeting mitochondria.

The past eighteen months have been among the most consequential in peptide science. A triple-agonist injectable posted the largest weight-loss numbers ever recorded in a Phase 3 trial. The FDA approved the first-ever drug targeting mitochondria. An oral GLP-1 pill moved to the doorstep of regulatory approval, promising to replace weekly injections for millions. And artificial intelligence began designing antimicrobial peptides that outperform decades-old antibiotics against superbugs.

At the same time, regulators tightened the screws on compounded peptides, effectively banning several popular compounds from pharmacy shelves and ending the era of compounded semaglutide and tirzepatide.

This article walks through the most important peptide research developments from 2025 into early 2026 -- what the data actually showed, what it means for patients, and where the field is headed next.

Table of Contents

  1. Next-Generation Obesity Drugs: Retatrutide, Orforglipron, and Beyond
  2. Tirzepatide Expands Its Reach
  3. Semaglutide's Cardiovascular Story Gets Stronger
  4. GLP-1 Agonists and the Brain: Alzheimer's Trials Disappoint, Addiction Research Advances
  5. Elamipretide: The First Mitochondria-Targeted Drug
  6. AI-Designed Peptides Enter the Lab
  7. Antimicrobial Peptides vs. Superbugs
  8. Peptide Cancer Vaccines Show Real Promise
  9. A Tiny Peptide for Traumatic Brain Injury
  10. FDA Compounding Crackdown: What Changed
  11. Timeline: Key Events, 2025-2026
  12. The Bottom Line
  13. References

Next-Generation Obesity Drugs: Retatrutide, Orforglipron, and Beyond {#next-generation-obesity-drugs}

Retatrutide: The Triple Agonist That Broke Records

If 2023 belonged to tirzepatide and 2024 to semaglutide's expanding indications, then 2025 was the year retatrutide announced itself as the next frontier.

Retatrutide is a first-in-class triple hormone receptor agonist -- it activates GIP, GLP-1, and glucagon receptors simultaneously. In December 2025, Eli Lilly released topline results from the Phase 3 TRIUMPH-4 trial in adults with obesity and knee osteoarthritis. The numbers were staggering:

  • 28.7% mean body weight reduction at the 12 mg dose (adjusted for placebo), translating to an average of 71.2 pounds lost
  • The 9 mg dose produced 20.0% weight loss; the 12 mg dose hit 23.7% before placebo adjustment
  • Pain scores on the WOMAC scale dropped by up to 75.8%, with 12-14% of participants reporting zero knee pain at 68 weeks
  • Systolic blood pressure fell by 14.0 mmHg at the highest dose, alongside improvements in non-HDL cholesterol, hsCRP, and triglycerides

These are the highest weight-loss figures ever reported in a Phase 3 obesity trial. For context, tirzepatide's SURMOUNT-1 trial produced 22.5% weight loss at its top dose. Retatrutide appears to be pushing the ceiling higher.

The trial also flagged a new safety signal: dysesthesia (abnormal touch sensation) occurred in 20.9% of patients on the 12 mg dose compared to 0.7% on placebo. Most cases were mild and did not lead to treatment discontinuation. Gastrointestinal side effects -- nausea, diarrhea, constipation -- remained the most common adverse events, consistent with the GLP-1 drug class.

Seven more Phase 3 readouts are expected in 2026, covering type 2 diabetes, sleep apnea, chronic low back pain, liver disease, and cardiovascular outcomes. Analysts project a 2027 FDA approval and potential peak sales of $15.6 billion by 2031.

Orforglipron: The Pill That Could Replace the Needle

The other blockbuster in Lilly's pipeline is orforglipron, a once-daily oral GLP-1 receptor agonist. Unlike oral semaglutide (which is still a peptide requiring careful absorption engineering), orforglipron is a small-molecule non-peptide compound -- a fundamentally different chemistry that could be cheaper and easier to manufacture at scale.

Orforglipron racked up positive Phase 3 results throughout 2025:

  • ATTAIN-1 (obesity): Participants lost an average of 22.9 pounds
  • ATTAIN-2 (obesity + type 2 diabetes): All three doses met primary and key secondary endpoints for weight loss, A1C reduction, and cardiometabolic risk factors at 72 weeks
  • ACHIEVE-2 and ACHIEVE-5 (type 2 diabetes): Significant A1C reduction and weight loss at 40 weeks
  • ATTAIN-MAINTAIN: Orforglipron helped patients maintain weight loss after switching from injectable Zepbound or Wegovy -- the first trial to demonstrate injectable-to-oral transition

Lilly submitted a New Drug Application to the FDA in late 2025 and received one of the agency's new "Commissioner's National Priority" vouchers, which accelerates review. The company has already stockpiled $1.5 billion worth of orforglipron inventory in anticipation of an approval decision, expected around April 2026.

Self-pay pricing will start at $149 per month for the lowest dose and up to $399 for higher doses -- a fraction of the list price for injectable GLP-1 drugs.

Meanwhile, Novo Nordisk's oral Wegovy (oral semaglutide for weight loss) received FDA approval in December 2025, making it the first oral GLP-1 approved specifically for obesity. The race for oral incretin dominance is fully underway.

Tirzepatide Expands Its Reach {#tirzepatide-expands-its-reach}

Tirzepatide (marketed as Mounjaro for diabetes and Zepbound for obesity) continued to broaden its clinical footprint in 2025.

Sleep Apnea: A New Indication

The SURMOUNT-OSA trials, published in the New England Journal of Medicine, tested tirzepatide in adults with moderate-to-severe obstructive sleep apnea (OSA) and obesity. Results showed:

  • Up to 62.8% reduction in the apnea-hypopnea index (AHI) -- roughly 30 fewer breathing disruptions per hour
  • Up to 50.2% of participants met criteria for disease resolution or near-resolution
  • Significant reductions in hypoxic burden, hsCRP, and systolic blood pressure

The FDA approved Zepbound for moderate-to-severe OSA in adults with obesity in December 2024, making it the first and only prescription drug approved for this indication.

Heart Failure and Cardiovascular Outcomes

Data from the SUMMIT trial showed tirzepatide reduced the risk of cardiovascular death or worsening heart failure by 38% in patients with heart failure with preserved ejection fraction (HFpEF) and obesity. The SURPASS-CVOT trial confirmed cardiovascular safety, with tirzepatide performing at least as well as dulaglutide over four years of follow-up.

The massive SURMOUNT-MMO trial -- enrolling over 15,000 people with obesity and high cardiovascular risk -- aims to determine whether tirzepatide can reduce actual cardiovascular events (heart attacks, strokes, death). Results are expected in 2027.

Semaglutide's Cardiovascular Story Gets Stronger {#semaglutides-cardiovascular-story-gets-stronger}

The SELECT trial had already established that semaglutide 2.4 mg reduced major adverse cardiovascular events (MACE) by 20% in 17,604 patients with obesity and cardiovascular disease but without diabetes. In 2025, a series of prespecified analyses and real-world studies deepened that story.

A prespecified analysis published in The Lancet found that semaglutide's cardiovascular benefit was consistent across all baseline weight and waist circumference categories. An estimated 33% of the MACE reduction could be attributed to waist circumference reduction -- meaning roughly two-thirds of the heart protection came from mechanisms beyond fat loss alone. Benefits appeared within three months, before patients had lost meaningful weight, suggesting direct anti-inflammatory or vascular effects.

The real-world SCORE study -- analyzing 27,963 U.S. patients -- confirmed the signal: semaglutide 2.4 mg was associated with a 45% reduction in three-point MACE over a mean follow-up of 200 days.

For a deeper dive into the cardiovascular data, see our GLP-1 cardiovascular outcomes analysis.

GLP-1 Agonists and the Brain: Alzheimer's Trials Disappoint, Addiction Research Advances {#glp-1-agonists-and-the-brain}

Alzheimer's: A Setback

The most anticipated GLP-1 neuroscience data of 2025 came back negative. Novo Nordisk's EVOKE and EVOKE+ trials tested oral semaglutide (14 mg daily) in 3,808 people aged 55-85 with early-stage Alzheimer's disease over 156 weeks. The drug failed to slow cognitive decline on the CDR-SB scale compared to placebo. Novo Nordisk cancelled its planned trial extensions and ended its Alzheimer's semaglutide program entirely.

The Phase 2b ELAD trial of liraglutide in mild-to-moderate Alzheimer's told a similar story: safe and tolerable, but no significant slowing of brain metabolism decline.

Researchers believe the problem may be brain penetration. Semaglutide's fatty-acid structure may prevent it from reaching the hippocampus in sufficient concentrations. Notably, real-world observational data still show that GLP-1 use is associated with 40-70% reduced Alzheimer's risk -- suggesting these drugs may work better for prevention than treatment.

For more on this evolving story, see our coverage of GLP-1 agonists and neurodegeneration research.

Addiction: A Surprising New Frontier

While the Alzheimer's data disappointed, GLP-1 research in addiction quietly built momentum. GLP-1 receptors sit in brain regions that govern reward processing and craving, and preclinical studies have consistently shown that GLP-1 agonists reduce alcohol intake, nicotine self-administration, and cocaine-seeking behavior in animal models.

In 2025, a Phase 2 trial published in JAMA Psychiatry tested weekly semaglutide in 48 adults with alcohol use disorder. Early clinical evidence indicated that semaglutide may reduce alcohol consumption, though larger trials are needed.

A pilot study from the Fralin Biomedical Research Institute at Virginia Tech found a novel mechanism: GLP-1 agonists appear to slow the speed at which alcohol enters the bloodstream, dampening its effects on the brain through a pathway different from existing medications like naltrexone.

Multiple randomized controlled trials are now underway testing GLP-1 agonists, tirzepatide, and other dual agonists in alcohol use disorder, tobacco dependence, and opioid use disorder. As NIH researcher Lorenzo Leggio stated: "This research is very important because alcohol and drug addiction are major causes of illness and death, yet there are still only a few effective treatment options."

Read more in our report on GLP-1 agonists and addiction.

Elamipretide: The First Mitochondria-Targeted Drug {#elamipretide-the-first-mitochondria-targeted-drug}

In September 2025, the FDA granted accelerated approval to FORZINITY (elamipretide) for Barth syndrome -- an ultra-rare mitochondrial disease affecting approximately 150 people in the United States. This approval represented two firsts: the first FDA-approved treatment for Barth syndrome, and the first FDA-approved drug designed to target mitochondria.

Elamipretide is a cell-permeable synthetic peptide that binds to cardiolipin on the inner mitochondrial membrane, stabilizing the organelle's structure and improving energy production. The approval was based on the TAZPOWER trial at Johns Hopkins Hospital, where patients showed improved knee extensor muscle strength over a 168-week open-label extension.

The drug's journey from bench to approval took over two decades. Pharmacologist Hazel Szeto at Weill Cornell Medicine discovered the compound, making it the first molecule discovered at that institution to receive FDA approval. Stealth BioTherapeutics, the company behind elamipretide, is now studying the peptide in dry age-related macular degeneration and primary mitochondrial myopathy, and developing a second-generation candidate, bevemipretide (SBT-272), for neurological and ophthalmic conditions.

AI-Designed Peptides Enter the Lab {#ai-designed-peptides-enter-the-lab}

The 2024 Nobel Prize in Chemistry -- awarded for breakthroughs in AI and de novo protein design -- signaled that computational biology had arrived. In 2025, that signal became action.

CycloPepper: Automating Cyclic Peptide Synthesis

Cyclic peptides are among the most promising drug candidates because of their stability, membrane permeability, and strong binding. But synthesizing them is notoriously fickle -- cyclization site selection can make or break a yield. A team published in Nature Communications introduced CycloPepper, a machine learning platform that predicts cyclization outcomes and guides automated synthesis. They trained the model on a standardized dataset of 306 cyclic peptides produced by CycloBot, their fully automated synthesis platform, creating a closed loop from prediction to production.

AI-Powered Antimicrobial Discovery

In a landmark 2025 study published in Nature Microbiology, researchers used ProteoGPT -- a protein language model -- to screen hundreds of millions of peptide sequences for antimicrobial activity. From 50,000 AI-generated candidates, 46 were synthesized and experimentally validated, showing broad-spectrum activity against carbapenem-resistant Acinetobacter baumannii (CRAB) and methicillin-resistant Staphylococcus aureus (MRSA). Critically, these peptides showed reduced susceptibility to resistance development compared to conventional antibiotics, and they did not disrupt gut microbiota in mouse models -- a persistent problem with traditional antibiotics.

De Novo Design for "Undruggable" Targets

AI systems can now predict receptor-binding hotspots with atomic precision, enabling the design of cyclic peptides targeting oncoproteins like KRAS -- long considered "undruggable." Deep learning frameworks like RFdiffusion generate cell-targeting peptides with 60% higher tumor affinity than those found through traditional phage-display methods.

The throughput gains are real. Traditional peptide discovery required multiple rounds of screening, extensive synthesis, and functional assays over months or years. AI-guided pipelines are compressing that timeline from years to weeks. By 2030, most novel peptides entering clinical trials are expected to originate from computational platforms that did not exist a decade ago.

Antimicrobial Peptides vs. Superbugs {#antimicrobial-peptides-vs-superbugs}

Antimicrobial resistance will kill an estimated 39 million people directly over the next 25 years. Antimicrobial peptides (AMPs) -- short chains of 10-50 amino acids found in nearly every living organism -- offer a fundamentally different attack strategy than conventional antibiotics. Instead of targeting a single bacterial pathway (which bacteria can evolve around), AMPs disrupt cell membranes through multiple mechanisms simultaneously, making resistance much harder to develop.

In 2025, the AMP field hit several milestones:

  • The APD3 database now catalogs 5,099 peptides, including 3,306 natural AMPs from six biological kingdoms
  • Only an estimated 3% of bacterial natural product biosynthetic pathways have been characterized, suggesting a vast untapped reservoir
  • Several AMPs are already in clinical use, including nisin, polymyxins, daptomycin, and vancomycin
  • Novel engineered peptides like PEW300 showed strong antibiofilm activity against Pseudomonas aeruginosa through multiple mechanisms: membrane disruption, reactive oxygen species induction, and virulence reduction

The biggest challenge remains clinical translation. AMPs can be susceptible to protein degradation in the body, and some show toxicity at therapeutic concentrations. Nanoparticle delivery systems and structural modifications (cyclization, stapling, non-natural amino acids) are being explored to overcome these limitations. Several candidates are in Phase I/II clinical trials for wound healing, melanoma, and oral mucositis.

Peptide Cancer Vaccines Show Real Promise {#peptide-cancer-vaccines-show-real-promise}

Personalized neoantigen peptide vaccines -- treatments designed from each patient's unique tumor mutations -- posted their strongest clinical data yet in 2025.

At Mount Sinai, the Phase 1 trial of PGV001 reported five-year follow-up data: of 13 patients with various advanced cancers who received the personalized multi-peptide vaccine, six survived, and three of those six were tumor-free. The results were published in Cancer Discovery.

A Phase 1 trial in kidney cancer, published in Nature, showed that none of nine patients with high-risk, fully resected clear cell renal cell carcinoma had a cancer recurrence at a median follow-up of 40.2 months. All nine generated T-cell immune responses against the vaccine antigens, including responses to known kidney cancer driver mutations.

Dana-Farber Cancer Institute reported that its updated NeoVaxMI formulation triggered T-cell responses in all nine patients tested, with cytotoxic CD8+ T-cell responses confirmed in six of nine. Tumor biopsies confirmed that vaccine-induced immune cells had infiltrated the tumors.

The broader field is accelerating. More than 30 peptide vaccine candidates reached Phase II clinical trials by mid-2025 -- a record. Merck and Moderna are running a trial testing a personalized neoantigen vaccine as adjuvant therapy in operable kidney cancer, while Genentech and BioNTech are testing mRNA-based approaches in pancreatic cancer. Peptide-based approaches account for 40% of all personalized cancer vaccine trials, with most activity concentrated in the U.S. (44%) and China (24%).

A Tiny Peptide for Traumatic Brain Injury {#a-tiny-peptide-for-traumatic-brain-injury}

There are currently no FDA-approved drugs to halt brain damage or its secondary effects after traumatic brain injury (TBI). Treatment is limited to stabilizing the patient and managing intracranial pressure.

In October 2025, researchers from Aivocode (a Sanford Burnham Prebys spin-off), IQAC-CSIC, and UC Davis published a study in EMBO Molecular Medicine showing that a four-amino acid peptide called CAQK has intrinsic neuroprotective effects when delivered intravenously. Originally developed as a drug-delivery vehicle -- it was identified through phage display for its ability to home in on injured brain tissue by binding glycoproteins in the extracellular matrix -- CAQK turned out to be therapeutic on its own.

In both mouse and pig models of TBI, CAQK reduced neuroinflammation and cell death (apoptosis) and partially restored motor and cognitive function. The researchers emphasized that CAQK showed no toxicity and effectively targeted injuries in large animal models, bringing it closer to human relevance. Aivocode plans to apply for FDA approval to begin Phase I clinical trials.

FDA Compounding Crackdown: What Changed {#fda-compounding-crackdown-what-changed}

The FDA's regulatory actions in 2025 reshaped the peptide market in ways that will be felt for years.

BPC-157 and Other Research Peptides: Banned From Compounding

The FDA now classifies BPC-157 as a "Substance with Safety Concerns" (Category 2 Bulk Drug Substance), citing immunogenicity and impurity risks. Licensed pharmacies cannot legally compound it for human use. TB-500 received the same classification.

Enforcement has teeth. The Department of Justice prosecuted Tailor Made Compounding LLC for distributing unapproved peptides including BPC-157, forcing the company to forfeit $1.79 million. The FDA has also been targeting vendors that use "Not for Human Consumption" labels as workarounds -- if the marketing includes dosing information, healing testimonials, or anti-aging claims, the research label is legally void.

For a complete breakdown, see our guide to the FDA peptide compounding crackdown.

Compounded GLP-1 Drugs: The Window Closed

The FDA declared the tirzepatide shortage over in December 2024 and the semaglutide shortage over in February 2025. This triggered the wind-down of temporary compounding flexibilities:

  • Tirzepatide: Enforcement discretion for state-licensed pharmacies ended February 18, 2025; for outsourcing facilities, March 19, 2025
  • Semaglutide: All compounded versions became unavailable by May 22, 2025

The Outsourcing Facilities Association challenged both decisions in court, but judges ruled in the FDA's favor. The practical effect: patients who had been using lower-cost compounded versions of these drugs were pushed back to brand-name products or forced to discontinue treatment. As one endocrinologist summarized the situation: "The shortage is much better; insurance coverage is much worse."

State attorneys general have also begun using consumer protection statutes to go after GLP-1 distribution channels that continued operating after the enforcement deadlines.

Timeline: Key Events, 2025-2026 {#timeline}

DateEvent
Dec 2024FDA approves Zepbound (tirzepatide) for obstructive sleep apnea
Dec 2024FDA declares tirzepatide shortage resolved
Feb 2025FDA declares semaglutide shortage resolved
Feb 2025Tirzepatide compounding enforcement begins (503A pharmacies)
Mar 2025Tirzepatide compounding enforcement begins (503B outsourcing facilities)
May 2025All compounded semaglutide and tirzepatide effectively banned
Aug 2025Orforglipron ATTAIN-1 Phase 3 results reported (obesity)
Sep 2025FDA approves elamipretide (Forzinity) for Barth syndrome
Oct 2025CAQK peptide TBI study published in EMBO Molecular Medicine
Oct 2025Orforglipron ACHIEVE Phase 3 results reported (type 2 diabetes)
Oct 2025AI-generated antimicrobial peptides published in Nature Microbiology
Dec 2025Retatrutide TRIUMPH-4 Phase 3 results: 28.7% weight loss
Dec 2025FDA approves oral Wegovy (oral semaglutide for obesity)
Dec 2025EVOKE/EVOKE+ Alzheimer's trial results reported (negative)
Late 2025Lilly submits orforglipron NDA with National Priority Voucher
Q1 2026Orforglipron ACHIEVE-4 results expected (type 2 diabetes)
Apr 2026 (est.)FDA decision expected on orforglipron for obesity
2026Seven additional retatrutide Phase 3 readouts expected
2026Lilly plans orforglipron T2D regulatory submission

The Bottom Line {#the-bottom-line}

The peptide field in 2025-2026 is defined by a clear pattern: established drugs are proving they can do more than we thought, while entirely new classes of peptide therapeutics are moving from concept to clinic faster than ever before.

Semaglutide's cardiovascular protection appears to work through mechanisms beyond weight loss. Tirzepatide now treats sleep apnea and may protect against heart failure. Retatrutide has posted weight-loss numbers that would have seemed implausible five years ago. And orforglipron could soon eliminate the injection barrier that has kept millions of patients from accessing GLP-1 therapy.

Beyond the incretin drugs, the approval of elamipretide proves that peptides can target subcellular organelles. AI platforms are designing antimicrobial peptides that work against bacteria resistant to every conventional antibiotic. Personalized cancer vaccines built from peptides are keeping patients tumor-free years after treatment.

The regulatory picture is more complicated. The FDA's compounding crackdown has real consequences for patients who relied on compounded peptides, whether for research compounds like BPC-157 or for affordable versions of GLP-1 drugs. The tension between safety, access, and pharmaceutical economics will not resolve easily.

What is not in question: peptides are no longer a niche therapeutic class. They are at the center of obesity medicine, cardiovascular prevention, oncology, neuroscience, infectious disease, and regenerative medicine. The next twelve months -- with retatrutide Phase 3 data, the orforglipron FDA decision, and ongoing AI-driven discovery -- will likely bring even more.

References {#references}

  1. Eli Lilly. "Lilly's triple agonist, retatrutide, delivered weight loss of up to an average of 71.2 lbs along with substantial relief from osteoarthritis pain in first successful Phase 3 trial." Press Release, December 2025. investor.lilly.com

  2. Eli Lilly. "Lilly's oral GLP-1, orforglipron, is successful in third Phase 3 trial, triggering global regulatory submissions this year for the treatment of obesity." Press Release, 2025. investor.lilly.com

  3. Lincoff AM, et al. "Semaglutide and Cardiovascular Outcomes in Obesity without Diabetes." New England Journal of Medicine. 2023;389:2221-2232. nejm.org

  4. Semaglutide and cardiovascular outcomes by baseline and changes in adiposity measurements: a prespecified analysis of the SELECT trial. The Lancet. 2025. thelancet.com

  5. Smolderen KG, et al. "Lower risk of cardiovascular events in patients initiated on semaglutide 2.4 mg in the real-world: Results from the SCORE study." Diabetes, Obesity and Metabolism. 2025. dom-pubs.onlinelibrary.wiley.com

  6. Malhotra A, et al. "Tirzepatide for the Treatment of Obstructive Sleep Apnea and Obesity." New England Journal of Medicine. 2024. nejm.org

  7. Novo Nordisk. EVOKE and EVOKE+ Phase 3 trial results presented at CTAD conference, December 2025. science.org

  8. Edison C, et al. "GLP-1 Receptor Agonists: Promising Therapeutic Targets for Alcohol Use Disorder." Endocrinology. 2025;166(4). academic.oup.com

  9. Stealth BioTherapeutics. "Stealth BioTherapeutics Announces FDA Accelerated Approval of FORZINITY (elamipretide) injection." Press Release, September 2025. stealthbt.com

  10. CycloPepper: a machine learning platform for predicting cyclization outcomes and optimizing synthesis of therapeutic cyclopeptides. Nature Communications. 2026. nature.com

  11. A generative artificial intelligence approach for the discovery of antimicrobial peptides against multidrug-resistant bacteria. Nature Microbiology. 2025. nature.com

  12. Mount Sinai Newsroom. "Personalized Cancer Vaccine Proves Promising in a Phase 1 Trial at Mount Sinai." 2025. mountsinai.org

  13. A neoantigen vaccine generates antitumour immunity in renal cell carcinoma. Nature. 2025. nature.com

  14. Mann AP, et al. "A neuroprotective tetrapeptide for treatment of acute traumatic brain injury." EMBO Molecular Medicine. 2025. springer.com

  15. Holt Law. "Deep Dive: Regulatory Status of Popular Compounded Peptides." 2025. djholtlaw.com

  16. CNN. "FDA crackdown on off-brand Ozempic products set to take effect." May 2025. cnn.com

  17. BioSpace. "Lilly's Retatrutide Scores Triple Trial Triumph With 26% Weight Loss, But New Safety Signal Emerges." December 2025. biospace.com

  18. Liraglutide in mild to moderate Alzheimer's disease: a phase 2b clinical trial. Nature Medicine. 2025. nature.com

  19. UMDF. "FDA Approves First Mitochondrial Disease Therapy." 2025. umdf.org

  20. Weill Cornell Medicine Newsroom. "The Winding Road from Bench to FDA Approval for First Mitochondria-Targeting Drug." December 2025. news.weill.cornell.edu