Last Week in Biomedicine

Part 2 covers three more standout stories from the same week: a Phase 3 trial that gives babies with a devastating enzyme disease a fighting chance, a first-of-its-kind cell therapy approved to make bone-marrow transplants safer, and a new lymphoma combination that could give aggressive-blood-cancer patients another option after other therapies fail.

In Part 2 we cover:

4. Sanofi — Nexviazyme (avalglucosidase alfa) for infantile-onset Pompe disease (Phase 3)

5. Orca Bio — Tregzi (allogeneic regulatory T-cell immunotherapy) for adults with blood cancers undergoing matched-donor bone-marrow transplant (FDA approval)

6. Genmab / AbbVie — Epkinly (epcoritamab) plus lenalidomide for relapsed or refractory diffuse large B-cell lymphoma (Phase 3)

What could this mean for patients? The Sanofi Baby-COMET result is the most emotionally consequential story of the week. Infantile-onset Pompe disease is usually fatal in a baby's first year without treatment, and a Phase 3 trial hitting every primary and secondary endpoint means more infants may reach toddlerhood breathing on their own — an outcome that was unimaginable a generation ago.

Catch Part 1 if you missed it. Subscribe at TrialBreaks.com.

#biomedicine #clinicaltrials #science #Pompedisease #enzymereplacement #rarediseases #genetherapy #celltherapy #bonemarrowtransplant #GVHD #hematology #lymphoma #DLBCL #bispecificantibody #immunotherapy #oncology

CRISPR gene editing crossed a major line this week: the FDA now allows Casgevy — the first CRISPR therapy of any kind — for children as young as 2 with sickle cell disease, meaning we can potentially prevent a lifetime of pain crises and organ damage instead of treating them after the fact. Part 1 also digs into a rare kind of trial where a new lung cancer drug takes on the already-approved competition, and a New England Journal of Medicine paper on the first oral pill for a form of childhood dwarfism.

In Part 1 we cover:

1. Vertex Pharmaceuticals / CRISPR Therapeutics — Casgevy (exagamglogene autotemcel) for sickle cell disease and transfusion-dependent beta thalassemia in children ages 2 and up (FDA approval — label expansion)

2. Roche / Genentech — divarasib for KRAS G12C-mutant non-small cell lung cancer (Phase 3)

3. BridgeBio Pharma / Kyowa Kirin — oral infigratinib for achondroplasia in children (Phase 3, published in NEJM)

What could this mean for patients? Casgevy in toddlers is the story with the biggest potential ripple effect. Sickle cell disease starts hurting kids in infancy, so treating a 2-year-old before repeated strokes and organ damage sets in could redefine what growing up with sickle cell looks like. It's also a proof point that CRISPR-based cures can be delivered safely in very young children — a green light for other pediatric gene-editing programs in development.

Part 2 drops Thursday morning. Subscribe at TrialBreaks.com.

#biomedicine #clinicaltrials #science #CRISPR #genetherapy #sicklecell #lungcancer #KRAS #achondroplasia #oncology #rarediseases #pediatrics #hematology #NEJM #immunotherapy

Part 2 covers a new drug for the bulging-eye autoimmune disease, the first Phase 3 ADHD trial designed for patients who also have anxiety, and a major pediatric expansion for a top-selling psoriasis biologic. In Part 2 we cover:

4. Viridian Therapeutics — Lumvoa (veligrotug) for thyroid eye disease, active and chronic (FDA approval)

5. Otsuka Pharmaceutical — centanafadine for adult ADHD with comorbid anxiety (Phase 3b)

6. AbbVie — Skyrizi (risankizumab) for pediatric plaque psoriasis and psoriatic arthritis (FDA approval)

The biggest story in Part 2: Lumvoa is only the second IGF-1 receptor antibody ever approved for thyroid eye disease, but it has a much broader label than its predecessor — covering both the active early form and the chronic long-standing form that affects far more patients. For people who've lived with bulging eyes and double vision for years and were told nothing could help, this approval is genuinely new ground.

Catch Part 1 if you missed it. Subscribe at TrialBreaks.com.

#biomedicine #clinicaltrials #science #ophthalmology #thyroideyedisease #ADHD #anxiety #mentalhealth #dermatology #psoriasis #pediatrics #immunology #FDAapproval #autoimmune #IL23

This week, an LSD-based therapy hit its main goal in a Phase 3 depression trial — and two huge breast cancer approvals landed at the FDA on the same week. In Part 1 we cover:
1. Definium Therapeutics — DT120 (LSD-based orally disintegrating tablet) for major depressive disorder (Phase 3)
2. Gilead Sciences / Merck — Trodelvy (sacituzumab govitecan) ± Keytruda for first-line PD-L1+ metastatic triple-negative breast cancer (FDA approval)
3. Pfizer — Ibrance (palbociclib) + trastuzumab ± pertuzumab for HR+/HER2+ metastatic breast cancer maintenance (FDA approval based on Phase 3 PATINA)
The biggest story: Definium's Emerge trial showed a single dose of DT120 produced rapid relief from major depression that lasted weeks — the first time an LSD-based therapy has cleared a pivotal Phase 3 in this disease. After years of mixed results for psilocybin and MDMA programs, this readout could reopenthe door for psychedelic medicines and give patients who don't respond to standard antidepressants a fundamentally new option.
Part 2 drops Thursday morning. Subscribe at TrialBreaks.com.
#biomedicine #clinicaltrials #science #psychiatry #depression #psychedelics#oncology #breastcancer #immunotherapy #ADC #FDAapproval #neuroscience#womenshealth #cancerresearch

Part 2 of our week of June 14–20, 2026 wrap-up covers a new molecular glue for relapsed multiple myeloma that cut disease progression by half, a heart drug that may finally work forboth forms of hypertrophic cardiomyopathy, and a new combination regimen poised to upgrade the first-line standard of care in myelofibrosis.
In Part 2 we cover:4. Bristol Myers Squibb — Mezigdomide for relapsed/refractory multiple myeloma (Phase 3)
5. Edgewise Therapeutics — EDG-7500 for obstructive and non-obstructive hypertrophic cardiomyopathy (Phase 2)
6. Karyopharm Therapeutics / Menarini — Selinexor plus ruxolitinib for first-line myelofibrosis (Phase 3)
The most patient-impactful of these three is probably the Edgewise heart drug. Hypertrophic cardiomyopathy — a too-thick heart muscle that makes it hard to pump blood — is the most common inherited heart disease, but today'smyosin-inhibitor drugs only help people whose blood flow is physically blocked. EDG-7500 showed clear improvements in both the obstructive and non-obstructive forms, potentially more than doubling the number of patients who could benefit.
Catch Part 1 if you missed it. Subscribe at TrialBreaks.com.
#biomedicine #clinicaltrials #science #oncology #cardiology #hematology#myeloma #HCM #cardiomyopathy #myelofibrosis #CELMoD #sarcomere#drugdevelopment #precisionmedicine

This week opened with one of the most significant gene-editing milestones in clinical medicine: a single CRISPR infusion cut hereditary angioedema attacks by 87 percent in a Phase 3 trial, published the same day in the New England Journal of Medicine. Plus a first-in-class drug that may reshape how we treat narcolepsy, and the first new oral antifungal class in over 20 years.

In Part 1 we cover:

1. Intellia Therapeutics — Lonvoguran ziclumeran (lonvo-z) for hereditary angioedema (Phase 3)

2. Takeda Pharmaceutical — Oveporexton (TAK-861) for narcolepsy type 1 (Phase 3)

3. F2G / Shionogi — Olorofim for invasive aspergillosis (Phase 3)

The Intellia readout is the headline of the week. Hereditary angioedema causes sudden, painful and sometimes life-threatening swelling attacks, and current treatments require regular injections to suppress them. Lonvo-z is given once — a tiny package of CRISPR machinery shuts down the gene responsible for the attacks inside liver cells permanently — and 81 percent of patients were completely attack-free at one year. If this holds up, an entire category of chronic disease management could be replaced by single-dose curative gene

editing.

Part 2 drops Thursday morning. Subscribe at TrialBreaks.com.

#biomedicine #clinicaltrials #science #CRISPR #genetherapy #raredisease

#neurology #narcolepsy #orexin #infectiousdisease #antifungal #immunology #HAE

#aspergillosis #drugdevelopment

Part 2 of our June 6–13 wrap-up: Novo Nordisk's CagriSema beats semaglutide alone in three Phase 3 diabetes trials, Takeda's zasocitinib pill outperforms Sotyktu head-to-head in psoriasis, and Agios's mitapivat headlines the EHA Plenary with breakthrough Phase 3 sickle cell data.

In Part 2 we cover:

4. Novo Nordisk — CagriSema (cagrilintide + semaglutide) for type 2 diabetes (Phase 3)

5. Takeda — Zasocitinib for moderate-to-severe plaque psoriasis (Phase 3)

6. Agios Pharmaceuticals — Mitapivat for sickle cell disease (Phase 3)

Mitapivat is a once-daily pill that flips on an enzyme inside red blood cells so they can make more energy and resist the sickling that causes excruciating pain crises. The Phase 3 RISE UP data, presented at the prestigious EHA 2026 Plenary, showed it both raises hemoglobin and cuts pain crises — a long-overdue option for a disease with very few approved therapies that disproportionately affects Black communities globally.

Catch Part 1 if you missed it. Subscribe at TrialBreaks.com.

#biomedicine #clinicaltrials #science #diabetes #GLP1 #obesity #sicklecell #hematology #psoriasis #dermatology #immunology #raredisease #metabolic

A blockbuster week in biomedicine — Eli Lilly's retatrutide produced weight loss approaching bariatric surgery in Phase 3, and Gilead and Merck's once-weekly oral HIV pill cleared two Phase 3 trials. Plus the first in-vivo CRISPR therapy keeps stacking up Phase 3 wins.
In Part 1 we cover:1. Eli Lilly — Retatrutide for obesity (Phase 3)
2. Gilead Sciences and Merck — Islatravir/Lenacapavir for HIV (Phase 3)
3. Intellia Therapeutics — Lonvoguran ziclumeran (lonvo-z) for hereditary angioedema (Phase 3)
Retatrutide is the standout. By activating three gut hormone receptors at once — GLP-1, GIP, and glucagon — it drove mean weight loss up to about 30% in the TRIUMPH-1 trial, while also improving blood sugar, knee osteoarthritis pain, and sleep apnea. If that holds up, the next generation of obesity medicineswon't just shrink waistlines, they'll treat the cluster of conditions tied to obesity.
Part 2 drops Thursday morning. Subscribe at TrialBreaks.com
#biomedicine #clinicaltrials #science #obesity #GLP1 #HIV #infectiousdisease #CRISPR #genetherapy #rarediseases #raredisease #metabolic #immunology

From a prostate cancer combo that uses your tumor's own broken DNA-repair against it, to the first drug ever to succeed in a rare autoimmune disease called IgG4-related disease — Part 2 of our weekly biomedicine recap covers three more major readouts from the week of May 30 – June 5, 2026. In Part 2 we cover:

4. Pfizer — Talazoparib plus enzalutamide for metastatic castration-sensitive prostate cancer with DNA-repair mutations (Phase 3)

5. Zenas BioPharma — Obexelimab for IgG4-related disease (Phase 3)

6. Karyopharm Therapeutics — Selinexor plus ruxolitinib for myelofibrosis (Phase 3)

The IgG4-RD result may be the most quietly historic of the week: IgG4-related disease is a rare autoimmune condition where misbehaving B cells form fibrotic masses in organs throughout the body, and there has never been an approved therapy — patients rely on steroids long-term. Obexelimab, a clever 'bifunctional' antibody that calms B cells without killing them, cut the risk of disease flare by 56% in a 52-week Phase 3 trial — the first positive Phase 3 ever in this disease, published in the New England Journal of Medicine. We explain what makes the antibody design unusual and why a positive trial here could change rare-disease drug development more broadly.

Catch Part 1 if you missed it. Subscribe at TrialBreaks.com

#oncology #prostatecancer #PARPinhibitor #autoimmune #IgG4 #rheumatology #rarediseases #myelofibrosis #hematology #targetedtherapy #precisionmedicine #biomedicine #clinicaltrials #science

The biggest week in cancer research of 2026 dropped at the ASCO meeting in Chicago, and one drug stole the show: a new RAS-blocking pill that nearly doubled survival in metastatic pancreatic cancer, one of the deadliest diseases in medicine. In Part 1 we cover:

1. Revolution Medicines — Daraxonrasib for metastatic pancreatic cancer (Phase 3)
2. Summit Therapeutics and Akeso — Ivonescimab for first-line squamous non-small cell lung cancer (Phase 3)
3. Eli Lilly — Selpercatinib for adjuvant RET fusion-positive non-small cell lung cancer (Phase 3)
Daraxonrasib targets RAS, the 'on switch' that drives roughly a third of all cancers and almost every pancreatic tumor. Unlike earlier RAS drugs that hit only one specific mutation, daraxonrasib blocks the active form of multiple RAS variants at once — and the result was a near-doubling of median overallsurvival from 6.6 months to 13.2 months in patients who had already failed first-line chemo. Doctors at the ASCO Plenary called the data 'transformative' and gave a standing ovation. We break down the science, explain why pancreatic cancer has been so untreatable, and walk through what RAS-ON inhibitors could mean for other tumors in the years ahead.
Part 2 drops Thursday morning. Subscribe at TrialBreaks.com
#oncology #pancreaticcancer #lungcancer #precisionmedicine #RAS #immunotherapy #bispecificantibody #PD1 #targetedtherapy #ASCO2026 #biomedicine #clinicaltrials #science