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StockWatch: Regenxbio Tumbles Despite Positive Pivotal Data for DMD Gene Therapy Candidate

Regenxbio (NASDAQ: RGNX) shares nosedived 43% over two days late last week, reaching 52-week lows on consecutive days, despite generating positive pivotal Phase III data for its Duchenne muscular dystrophy (DMD) gene therapy candidate RGX-202.

While the data was encouraging enough to enable discussion of Regenxbio bringing a second DMD gene therapy to the market, investors and analysts concluded it was not encouraging enough to pose a competitive threat to the developer of the first marketed DMD gene therapy, Sarepta Therapeutics (NASDAQ: SRPT), or to Solid Biosciences (NASDAQ), whose DMD gene therapy candidate SGT-003 is in Phase III as well as Phase I/II trials.

Even worse, investors were jolted by Regenxbio’s disclosure that the FDA had recommended the company conduct a randomized controlled trial (RCT) to assess RGX-202 in DMD during talks with agency officials. Regenxbio sought to reassure investors in its first-quarter earnings press release by noting past FDA guidance that externally controlled trials “may be adequate for demonstrating substantial evidence of effectiveness, especially when the treatment effect is sufficiently large enough to overcome limitations of externally controlled trials.”

Regenxbio plans to discuss its data with FDA officials at a future meeting. The agency has offered to review the RGX-202 data and alternative proposals, according to the company.

“RGX-202 pivotal data point to potential entry of second DMD gene theory, but a possibility of RCT requirement makes market entry timing unclear,” Kostas Biliouris, PhD, a managing director on the biotechnology research team of Oppenheimer & Co., wrote in a research note.

If the FDA does not insist on an RCT, RGX-202 could gain accelerated approval in 2027, Biliouris noted. Otherwise, the gene therapy is looking at not reaching the market for at least three additional years.

“Completing an RCT study as a precursor to filing or a precursor to approval means that it’s very unlikely that any new gene therapy would be approved until 2030. And I think that scenario is really untenable for the [DMD] community,” Simpson said. “It’s the opposite of regulatory flexibility.”

These regulatory and competitive concerns sent investors scrambling to sell Regenxbio shares late last week. The shares tumbled 38% from $10.04 to $6.24 Thursday, then slid another 8% Friday, sinking to $5.72 at the closing bell.

Positive microdystrophin expression

Regenxbio’s stock woes came despite the company announcing positive results from its pivotal Phase III portion of the Phase I/II/III AFFINITY DUCHENNE® trial (NCT05693142) of RGX-202. The company said the trial met its primary endpoint as 93% of participants (28 of 30) reached at least 10% microdystrophin expression at Week 12. A 31st participant refused a muscle biopsy and, as a result, did not have a Week 12 biopsy available for evaluation.

Microdystrophin expression averaged 71.1% across all participants, and 41.6% in older boys, aged >8 years, with 80% of participants achieving >40% microdystrophin expression, Regenxbio said.

“High unmet need remains for Duchenne patients as current options face limitations related to efficacy, safety, and access. The untreated Duchenne population continues to grow in the United States and globally. Physicians and patients need new next-generation options,” Curran M. Simpson, president and CEO, told analysts on the company’s first quarter earnings call.

Regenxbio acknowledged two reports of treatment-related serious adverse events (~6.5% of treated patients): An 8-year-old patient developed subacute myocarditis, while a 10-year-old patient showed a case of asymptomatic liver injury.

“Both were easily managed and resolved within weeks without sequelae,” Simpson told analysts.

Biliouris acknowledged RGX-202’s positive microdystrophin but said it will not likely have a material impact on Sarepta and its marketed DMD gene therapy Elevidys® (delandistrogene moxeparvovec-rokl).

Limited likelihood

“RGX-202’s functional benefit remains unclear without RCT data, limiting the likelihood of AA [accelerated approval] given an already fully approved DMD gene therapy,” Biliouris said.

He added that RGX-202’s safety profile could deteriorate once the gene therapy reaches the market and is being administered to patients, as happened with Elevidys after some 800 had been treated with the therapy, prompting Sarepta to halt shipments of Elevidys for non-ambulatory patients and pause a Phase III trial.

The halt—plus a label update limiting Elevidys use to ambulatory patients—explains why the gene therapy’s net product revenue plunged 73% year-over-year in Q1, to $102 million from $375 million. Elevidys generated $898.7 million in 2025 revenue—it ranks second on GEN’s just published A-List of Top 10 Best-Selling Gene Therapies—which was 9.5% above 2024’s $820.8 million.

The Q1 sales decline has sent Sarepta’s stock into decline: From $23.06 on May 6, before releasing Q1 results after that day’s closing bell, Sarepta shares have slumped 22.5%, to $17.88 on Friday.

Elevidys sparked a showdown with the FDA last summer when the agency briefly demanded Sarepta also pause Elevidys shipments to ambulant patients following the second patient death tied to Elevidys, then reversed itself after, according to news reports, pleas to Congress, the FDA, and President Donald Trump by conservative leaders and DMD patient advocates—who launched a Change.org petition that garnered 1,900 signatures.

Competitive advantage

Despite the slumping sales and resulting stock decline, Biliouris noted that Sarepta and Elevidys have a significant competitive advantage over challengers: A 3-1/2 year first to market advantage, with statistically significant functional benefits reported from randomized trials, as well as what the analyst called “compelling” three-year positive topline follow-up data from ambulatory DMD patients in the 52-patient active arm in Part 1 of Sarepta’s EMBARK trial (Study SRP-9001-301, NCT05096221).

That data showed significant improvements in North Star Ambulatory Assessment (NSAA), Time to Rise (TTR), and 10-meter walk/run (10MWR).

“Even if RGNX secures AA, we expect minimal impact given the large DMD market size (can accommodate multiple companies) and potential Elevidys monopoly in the non-ambulatory market,” projected for 2027 and later, Biliouris wrote.

According to Sarepta, Duchenne affects approximately 1 in 3,500 to 5,000 males born worldwide—some 300,000 people worldwide, according to research and patient care group Cure Duchenne. In the United States, about 15,000 young men and a few young women live with DMD, according to Parent Project Muscular Dystrophy estimates. A 2019 study found that most people with DMD become non-ambulatory around ages 10–12 and need assisted ventilation at around 20 years of age.

Andrew Tsai, equity analyst with Jefferies, said Sarepta’s three-year data, including muscle MRI data, has only begun to be promoted by the company this year. Since it can take six months to go from “start form” initiating the treatment process to infusion with Elevidys, Tsai reasoned, “we expect momentum to rebuild progressively/steadily in Q3/Q4, restoring confidence in the ambulatory DMD oppty.”

Some ~80% of ambulatory DMD patients remain untreated, Tsai noted, while Sarepta told investors in its Q1 earnings presentation that more than 1,300 patients have been treated with Elevidys in commercial settings or clinical trials as of May 5.

“While Elevidys’ safety perception has changed, we think marketing efforts on muscle MRI data, long-term three-year EMBARK data, and no deaths in ambulatory DMD could entice patients/caregivers and physicians to adopt Elevidys more, widening the moat,” Tsai wrote.

Maury Raycroft, PhD, a colleague of Tsai and equity analyst with Jefferies, wrote that Regenxbio’s data “reinforces microdystrophin as a surrogate, which is constructive for SLDB [Solid Biosciences].”

Playing to strengths

However, Raycroft added that Regenxbio’s safety events (notwithstanding immunosuppression) and limited regulatory clarity absent a pivotal RCT “play into SLDB’s strengths,” such as its use of a steroid-only prophylactic immunomodulation regimen (no safety issues to date) and its ongoing Phase III IMPACT DUCHENNE trial (NCT07160634), which is an RCT, thus a derisking factor from a regulatory standpoint.

“We believe RGNX is relying on and will require reg[ulatory] flexibility, which incorporates add’l risk and limitations, especially w/ FDA leadership in flux,” Raycroft wrote. “We caught up w/ SLDB, who also pointed out that RGNX could run into challenges to run an RCT given their immunosuppressive regimen.”

That regimen consisted of sirolimus, eculizumab, and steroids that included prednisone, researchers from Regenxbio and clinical partners reported in a poster presented at the International Congress of the World Muscle Society, held October 7–11, 2025, in Vienna.

On May 7, Solid announced it had dosed the first patient in the IMPACT DUCHENNE trial in Australia, at the Children’s Hospital at Westmead. The multi-country, placebo-controlled, randomized, double-blind trial has a pre-specified primary endpoint of change from baseline at 18 months in time to rise from supine (TTR) velocity, based on a Type C meeting with the FDA.

“With the initiation of a randomized, placebo-controlled clinical trial, we are reinforcing our conviction in SGT-003 and our long-standing commitment to generating well-controlled, high-quality data,” Gabriel Brooks, MD, Solid’s chief medical officer, said in a statement.

Solid shares dipped 2% on news of the dosing, from $7.20 to $7.07. Since then, the shares have yo-oed, climbing 9% to $7.72 on May 12 but sliding 10% since then, to $6.92 on Friday.

In its Phase I/II INSPIRE DUCHENNE study (NCT06138639), SGT-003 has also been administered to 46 patients, with approximately 30 participants dosed as of year-end 2025, Solid said.

“Families living with Duchenne continue to face difficult treatment decisions in a setting of significant unmet medical need,” Brooks added. “Solid remains focused on helping inform the Duchenne community of potential additional treatment options through the responsible and rigorous clinical evaluation of SGT-003.”

uniQure, Replimune gain as Makary exits FDA

Two gene therapy developers saw their stocks enjoy significant gains after Martin A. Makary, MD, resigned as FDA commissioner.

Makary’s resignation on May 12 capped nearly a week of speculation that he was about to exit the agency after a turbulent 13-month tenure. That tenure was marked in part by the elimination of 3,500 FDA positions as part of the Elon Musk-led Department of Government Efficiency (DOGE)-directed federal job cuts—as well as more frequent rejections of biologics license applications (BLAs) for new therapies, particularly gene therapies in rare disease indications.

Those rejections were carried out by the agency’s Center for Biologics Evaluation and Research (CBER) during the two tenures of Vinayak (Vinay) Prasad, MD, as Center director. Prasad resigned the first time in August 2025 after less than three months at the CBER helm, after he led the FDA’s confrontation with Sarepta over patient deaths tied to Elevidys (see Regenxbio item, above). The second resignation was announced in March and took effect on April 30, after he led the FDA’s hardline stance and public criticism against uniQure (NASDAQ: QURE)’s Huntington’s disease (HD) gene therapy candidate AMT-130.

While uniQure stock roller-coastered after Prasad’s second resignation, the stock jumped 21% in the four trading days between May 8, when an unnamed-source report about Makary being fired first surfaced in The Wall Street Journal, and May 13, the day after he resigned. uniQure rose 14.5% from $24.15 to $27.66 the day of the WSJ report, plateaued on May 10, dipping two cents to $27.64, then resumed their climb, rising 5% to $29.10 the following day before inching up another 0.2% to $29.17 on Wednesday.

An even bigger winner among stocks, however, was Replimune Group (NASDAQ: REPL). The developer of oncolytic immunotherapies saw its shares rocket 59% after news surfaced of Makary exiting the FDA.

Replimune has found itself in the FDA’s crosshairs over its biologics license application (BLA) for its lead product candidate RP1 (vusolimogene oderparepvec) in combination with nivolumab to treat advanced melanoma, instead issuing two complete response letters (CRLs)—one in April 2025, the other last month.

On April 10, the FDA rejected Replimune’s BLA for a second time, issuing a complete response letter (CRL) contending that the data set upon which the agency’s breakthrough therapy designation was awarded was not sufficient to allow for RP1 approval—an assertion Replimune vehemently rejects.

Replimune responded to the second BLA by criticizing the FDA for an inconsistent review process, saying the agency contradicted earlier guidance to the company and assessed the resubmitted BLA through a different review team that replaced the team that previously interacted with the company.

Replimune also defended the combination therapy’s data in the Phase II IGNYTE trial (NCT03767348)—a 34% response rate with a median duration of 24.8 months and a favorable safety profile, the basis of the combo’s breakthrough therapy designation.

Following the first news report of a Makary firing in the works, Replimune shares jumped 22% from $3.34 to $4.07. After slipping 8% to $3.74 the following trading day (May 11), Replimune rose 9% to $4.09 the following day after Makary resigned—then vaulted 30% to $5.30 on Wednesday.

“Broadly, we see multiple options for experienced leaders who could help stabilize the Agency following the many leadership transitions, and believe the tendency toward the administration’s “Right to Try” could draw a next leader who is more permissive on drug approvals near-term positive on the space,” Brian Abrahams, MD, head of global healthcare research with RBC Capital Markets, wrote in a research note.

Abrahams put forward six possible permanent successors to Makary:

  • Kyle Diamantas, current interim FDA commissioner; previously FDA deputy commissioner for human foods and senior counselor to Health and Human Services Secretary Robert F. Kennedy Jr.
  • Stephen Hahn, MD, CEO of Nucleus RadioPharma and a former FDA commissioner in President Donald Trump’s first administration (December 2019–January 2021).
  • Brett Giroir, MD, CEO of Altesa Biosciences; previously assistant secretary for health in Trump’s first term and an acting FDA commissioner (2019).
  • Sara Brenner, MD, HHS senior counselor for public health as of April 16; previously FDA principal deputy commissioner and acting FDA commissioner (January–April 2025).
  • Houman Hemmati, MD, PhD, a board-certified ophthalmologist and co-founder of Optigo Biotherapeutics, who is under consideration for CBER director.
  • Richard Pazdur, MD, a 26-year FDA veteran who retired in December 2025 after serving three weeks as CDER director (November–December 2025); previously founding director, FDA Oncology Center of Excellence (2017–2025).

“If Makary’s ouster indeed stemmed from political disagreements (vapes, abortion), the next Commissioner could harbor more ideological views—which could compromise perceived Agency credibility—and just by virtue of having another change, this would likely exacerbate the mixed messages companies have been receiving around FDA’s bar for their drugs, one of the key regulatory challenges the sector has faced,” Abrahams cautioned.

Leaders and laggards

  • Innate Pharma (Euronext Paris: IPH) shares jumped 35% from €1.23 ($1.42) to €1.66 ($1.92) on Wednesday after the Marseille, France-based developer of cancer drugs based on innate immunity and antibody engineering reported first-quarter results that beat analyst expectations. Innate finished Q1 with earnings per share of -0.1522, vs. the consensus forecast of -0.1616, on revenue of €2.6 million ($3.022 million) that was more than double (117% above) the €1.2 million ($1.395 million) reported in Q1 2025, thanks to partial or entire recognition of the proceeds received under collaboration agreements with AstraZeneca (NYSE, London Stock Exchange, and NASDAQ Stockholm: AZN) and Sanofi (Euronext Paris: SAN). Innate’s American depositary shares (ADSs) (NASDAQ: IPHA) rocketed 64% from $1.32 to $2.17 Wednesday.
  • Reviva Pharmaceuticals Holdings (NASDAQ: RVPH) shares plummeted 56% from 80 cents to 35 cents on Wednesday after the central nervous system (CNS), inflammatory, and cardiometabolic disease drug developer disclosed in a regulatory filing that the Nasdaq Hearings Panel had delisted the company’s stock, suspending it from trading on the exchange as of Thursday. Reviva said its shares will instead begin trading that day on the OTCQB Venture Market under its existing symbol. The Panel told Reviva that it failed to comply with Nasdaq’s minimum bid price of $1 per share required for continued listing on the Nasdaq Capital Market. Reviva disclosed the delisting the same day it reported first quarter results: The company narrowed its quarterly net loss year-over-year, finished Q1 with a net loss of approximately $3.2 million ($0.46 per share) vs. approximately $6.4 million ($2.61 per share) in the year-ago quarter.

The post StockWatch: Regenxbio Tumbles Despite Positive Pivotal Data for DMD Gene Therapy Candidate appeared first on GEN – Genetic Engineering and Biotechnology News.

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STAT+: U.K. advocacy groups threaten court action over a key provision in the pharma trade deal with the U.S.

Two advocacy groups are demanding the United Kingdom revoke regulations at the heart of a new trade agreement with the U.S. over concerns the deal will allow outsiders to influence official decisions about the cost-effectiveness of medicines. And if the government does not comply, the groups are readying legal action.

Under the trade agreement that covers pharmaceuticals, which was finalized last month, the Trump administration committed to impose zero tariffs on medicines exported from the U.K. for at least three years. The deal is significant because it would make the U.K. the only country with tariff-free access for medicines to the U.S. market.

In return, the U.K. government took steps to appease the pharmaceutical industry, which is a key part of its economy, by pledging to increase spending on medicines from 0.3% of GDP to 0.35% by 2028 and then to 0.6% by 2035. At the same time, the U.K.’s National Health Service will increase the prices paid for by medicines by 25% and slash the maximum rebate it can claw back from drugmakers to 15%.

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In Ebola outbreak, a number of Americans in the Congo believed to have had exposure to suspected cases

A number of Americans who are in the Democratic Republic of the Congo are believed to have had exposure to suspected cases in the country’s latest Ebola outbreak, with several deemed to have had high-risk exposures, sources have told STAT. At least one of these individuals may have developed symptoms.  

One source said that there are not yet test results for any of the individuals, but the U.S. government is reportedly trying to arrange to transport them out of the DRC to somewhere they can be safely quarantined, and cared for, if they prove to have been infected. It’s not clear if that would be in the United States; there is some discussion of perhaps taking the individuals to an American military base in Germany, a source said.

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Trump blasts ‘disloyal’ Sen. Cassidy while pushing challenger in Louisiana Republican primary

NEW ORLEANS — U.S. Sen. Bill Cassidy is fighting for his political life in Louisiana’s Republican primary on Saturday as he faces a challenge backed by President Donald Trump, the latest attempt by the president to purge the party of politicians he views as disloyal.

Trump endorsed U.S. Rep. Julia Letlow over Cassidy, in an unusual attempt to dislodge an incumbent senator. Cassidy voted to convict Trump in his second impeachment trial, stemming from the attack on the U.S. Capitol on Jan. 6, 2021. Cassidy, a doctor, has also clashed with Health Secretary Robert F. Kennedy Jr. over vaccine policy, even though he provided crucial support to help Kennedy get confirmed.

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Evaluating Crowdsourced Data Collection for Carceral Death Surveillance: Pilot Study Using Amazon Mechanical Turk

Background: People who are incarcerated face significantly higher health risks than the general population, yet deaths in custody remain underreported and poorly monitored by public health systems. Although the federal Death in Custody Reporting Act requires reporting of all deaths in correctional facilities to the US Department of Justice, reporting has been inconsistent, delayed, and often publicly inaccessible. Consequently, researchers have turned to press releases issued by correctional agencies as one of the few timely sources of information on deaths in custody. However, these press releases vary widely in content and structure, making standardized data extraction difficult. Crowdsourcing platforms such as Amazon Mechanical Turk (MTurk) may offer a faster, low-cost method for gathering data, but their utility in this setting remains untested. Objective: This pilot study evaluated whether MTurk could be used to extract structured information from press releases about deaths in custody. Methods: We selected 144 press releases describing deaths between 2000 and 2023 from state prison systems and Immigration and Customs Enforcement. Each press release was assigned to 3 MTurk crowd workers (who were required to be English speaking and located in the United States), resulting in 432 individual responses. Workers were informed in advance that the task involved reviewing sensitive content related to deaths in custody. Crowd workers completed a 16-question form aligned with Death in Custody Reporting Act variables, including age, race and ethnicity, date of death, and facility location. Data quality was assessed using strict concordance (all 3 responses matched), 2-way concordance (2 of 3 responses matched), and qualitative review of common errors. Task completion time was also recorded. Sampling included complete subsets of selected press releases and a stratified subset from systems with more complex reporting formats. Results: All 144 entries were completed within 48 hours. However, agreement across crowd workers was low: strict concordance was 14.2% (20/144) for age, 12.3% (18/144) for race or ethnicity, and 11.4% (16/144) for date of birth. Qualitative review identified frequent errors, missing data, and inattentive or automated responses. Crowd workers often misinterpreted system-specific terminology or copied placeholders instead of extracting information from the source. The low agreement indicated that this baseline MTurk configuration produced insufficient data quality for more resource-intensive use. Conclusions: MTurk enabled rapid task completion but produced low-quality results when applied to extracting structured data from carceral press releases. These findings suggest that general crowdsourcing platforms are poorly suited to complex data abstraction tasks without additional training or oversight. With improved task design or support from artificial intelligence tools, crowdsourcing may help address gaps in the surveillance of deaths in custody. Long-term improvements will require consistent, transparent, and standardized reporting practices across correctional institutions.
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PPG-Derived Digital Biomarker Developed for Peripheral Artery Disease Detection

A research team at the University of California, San Diego has developed a machine learning-based screening approach for peripheral artery disease (PAD) that uses a light-based technology called photoplethysmography (PPG) that can measure changes in blood volume in tissue. The researchers reported that short-duration PPG recordings in a patient’s toe, analyzed by machine learning models, identified PAD with a high degree of accuracy and may provide the basis for a scalable digital screening tool that could eventually be deployed through smartphones, pulse oximeters, and wearable devices. The team’s findings are published in npj Digital Medicine.

“PPG works by shining a light into tissue, in our case, the toe,” said co-first author Ava J. Fascetti, a PhD student in the digital health technology lab of senior author Edward J. Wang, PhD. “A photosensor measures how much light is reflected back, allowing us to detect tiny changes in blood volume: what we call the PPG signal.”

PAD is caused by plaque buildup in arteries that restricts blood flow, particularly to the legs and lower extremities. The disease affects an estimated 12 million Americans and 200 million adults worldwide. PAD substantially increases the risk of limb loss and major cardiovascular events, yet many patients are not diagnosed until later stages of disease progression. The researchers noted that the condition disproportionately affects underserved populations and is underdiagnosed in part because the current standard diagnostic, ankle-brachial index (ABI), requires specialized equipment, staff, and clinic visits.

“There exists a glaring unmet clinical need to develop technology to meet the demands of modern practice,” the researchers wrote. Further, ABI testing, introduced about 60 years ago, has has remained largely unchanged and has long-standing barriers to widespread use in primary care and under-resourced settings.

The current study originated from discussions between co-first author Mattheus Ramsis, MD, and assistant professor of medicine and medical director of cardiology informatics, and co-author Elsie G. Ross, MD, an associate professor of surgery in vascular and endovascular surgery, who noted that vascular labs conducting ABI testing often also collected toe PPG waveforms.

“The light-bulb went off for me at that moment,” Ramsis said.

PPG works by shining light into tissue and measuring backscattered light associated with blood volume changes. PPG has previously been used to identify cardiovascular and metabolic conditions including diabetes and atrial fibrillation. Earlier research efforts to use PPG for PAD detection had relied on small datasets, long recordings and less interpretable deep-learning approaches.

For their approach, the UCSD team assembled a dataset containing more than 10,000 toe PPG recordings from more than 3,500 patients who underwent ABI testing at UC San Diego Health between 2020 and 2025. Using these data, the researchers extracted 78 waveform features from the PPG signals that correlated significantly with ABI measurements. Those features were then used to train an explainable support vector machine model designed to identify PAD from PPG data alone.

Ramsis said the model correctly distinguished PAD cases approximately 83% of the time using only PPG data, compared with roughly 60% to 65% performance typically achieved using clinical risk-factor assessments alone. Incorporating smoking status of the patients further improved the performance of the new method.

Importantly, the model performed consistently across Black, Hispanic, and White patient populations, and among patients with diabetes, coronary artery disease, and end-stage renal disease. The researchers also reported similar performance across two UC San Diego Health campuses that used different equipment and staff.

The investigators noted that the physiologic basis for their findings align with established vascular biology. In PAD, reduced blood flow and arterial stiffness alter the morphology of PPG waveforms. Healthier patients demonstrated steeper systolic upstrokes and narrower waveform widths, while patients with PAD showed more dampened signals.

“Our findings support the existence of a reproducible PPG-derived digital biomarker that captures peripheral vascular pathophysiology relevant to ABI-defined PAD,” the researchers wrote.

The researchers said they don’t think their new model should replace ABI testing. Instead, they envision PPG screening as a complementary tool that could serve to identify patients earlier that might need further vascular evaluation.

The team said prospective deployment studies are already underway to evaluate performance in clinical settings and across additional reference standards, including toe pressure measurements, ultrasound imaging, and angiography. Additional research will also gauge performance in consumer-grade environments, including smartphones and wearable devices, and assess how the screening tool functions in broader patient populations outside specialized vascular clinics.

“If we can catch PAD early enough to prevent a limb amputation, that would be the ultimate impact: preserving limb function, reducing mortality, and addressing barriers in underserved populations,” Ramsis said.

The post PPG-Derived Digital Biomarker Developed for Peripheral Artery Disease Detection appeared first on Inside Precision Medicine.

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Antibody-Drug Conjugate Shows Activity in Hard-to-Treat Uterine Cancer

A Phase II clinical trial led by researchers at Yale School of Medicine and Yale Cancer Center has found that the antibody-drug conjugate sacituzumab govitecan, also known as Trodelvy, demonstrated encouraging clinical activity in patients with recurrent uterine cancer who had already exhausted several standard treatment options.

The findings, published in Clinical Cancer Research, suggest the therapy could become an important new option for patients with advanced endometrial cancer after chemotherapy and immunotherapy stop working.

A difficult disease to treat after relapse

Endometrial cancer is the most common gynecologic cancer in the United States, and rates continue to rise worldwide. While recent advances in immunotherapy have improved treatment for some patients, options remain limited once the disease returns after platinum-based chemotherapy or checkpoint inhibitor therapy.

Patients with recurrent disease often face poor outcomes, particularly those with aggressive tumor types such as uterine serous carcinoma and carcinosarcoma. Standard second-line chemotherapies typically produce modest response rates and short-lived disease control.

Researchers therefore wanted to investigate whether sacituzumab govitecan could improve outcomes in this challenging setting.

How the drug works

Sacituzumab govitecan belongs to a newer class of targeted therapies known as antibody-drug conjugates, or ADCs. These drugs combine an antibody that recognizes cancer cells with a chemotherapy payload designed to destroy them.

In this case, the therapy targets Trop-2, a protein commonly overexpressed in several aggressive cancers, including many uterine tumors. Attached to the antibody is SN-38, the active metabolite of irinotecan, a well-known chemotherapy drug.

By delivering chemotherapy directly to Trop-2–expressing cancer cells, researchers hope to increase anti-tumor activity while limiting damage to healthy tissue.

The drug is already approved for metastatic breast cancer and urothelial cancer, but remains investigational in uterine cancer.

Trial included heavily pretreated patients

The study enrolled 50 patients with recurrent or persistent endometrial cancer between 2020 and 2024. All participants had previously received platinum-based chemotherapy, and many had also undergone treatment with immune checkpoint inhibitors such as pembrolizumab or dostarlimab.

The study population represented a particularly difficult-to-treat group. Most patients had aggressive tumor histologies, including serous carcinoma, carcinosarcoma, or grade 3 endometrioid disease. Patients had received a median of two prior treatment regimens, with some undergoing as many as four lines of therapy before entering the study.

Encouraging responses and survival data

The trial met its primary endpoint, achieving an objective response rate of 28%. Two patients experienced complete responses with no detectable cancer remaining, while another 12 patients achieved partial responses with substantial tumor shrinkage.

In total, more than 70% of evaluable patients experienced some degree of tumor reduction during treatment. The study also reported durable responses, with a median response duration of 9.3 months. Several patients were still responding at the time of analysis.

Median progression-free survival reached 5.5 months, while median overall survival was 17.5 months in this heavily pretreated population.

Researchers also noted that responses were observed across multiple tumor subtypes rather than being limited to one specific histology.

“The results of our Investigator Initiated Trial complement and extend the TROPiCS-03 Trial results by demonstrating significant clinical activity of SG not only against the most common histological types of uterine cancer (endometrioid tumors) but also in patients harboring biologically aggressive endometrial tumors such as uterine serous carcinoma and carcinosarcoma,” said Alessandro Santin, MD, the study’s lead author.

Side effects remained manageable

As expected with potent cancer therapies, treatment-related side effects were common. The most frequent severe toxicities included neutropenia, anemia, fatigue, diarrhea, and febrile neutropenia.

However, investigators reported that most adverse events were manageable using supportive care measures such as growth factor support, anti-diarrheal medications, hydration, and dose adjustments. No treatment-related deaths were reported during the trial.

Looking ahead

The researchers cautioned that the study was relatively small and lacked a randomized comparison arm. Nevertheless, the results add to growing evidence supporting sacituzumab govitecan in advanced endometrial cancer, particularly for patients who have limited options remaining after standard therapies fail.

A larger international Phase III study is already underway to compare the drug directly against standard chemotherapy in patients with recurrent endometrial cancer following platinum chemotherapy and immunotherapy.

The team also highlighted future possibilities for combining the therapy with immunotherapy approaches, particularly because the drug’s chemotherapy payload may help stimulate anti-tumor immune responses.

“This is a major bench-to-bedside accomplishment for patients with uterine cancer,” Santin said.

 

The post Antibody-Drug Conjugate Shows Activity in Hard-to-Treat Uterine Cancer appeared first on Inside Precision Medicine.

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Exchange Marketplace Launched to Help Stalled Cell and Gene Therapies

A type of exchange marketplace for cell and gene therapies has launched to try and reinvigorate and relaunch candidate therapies that are no longer being developed due to financial constraints, despite having good science behind them.

CGTxchange, an online platform enhanced by artificial intelligence, is the brainchild of the American Society of Gene and Cell Therapy (ASGCT) and the Orphan Therapeutics Accelerator. The latter was set up in 2024 as a patient‑centered, non‑profit biotech accelerator that acquires “shelved” clinical‑stage therapies for ultra‑rare diseases and completes their development and commercialization so patients can actually access them.

The new platform was launched at the ASGCT conference in Boston and is designed to help stalled assets to find a new home. This is designed to address funding pull‑backs, program terminations, and company exits, which are forcing hundreds of cell and gene therapy programs to be discontinued or indefinitely paused despite having good scientific promise.

CGTxchange will link up companies who want to develop their assets with funders and partners who are looking for new development opportunities in cell and gene therapy.

“The cell and gene therapy field has built an extraordinary base of clinical evidence, and yet too many of those programs sit on the shelf for reasons that have nothing to do with the science,” said Terry Flotte, MD, dean of University of Massachusetts Chan Medical School and president of the ASGCT, in a press statement.

“CGTxchange gives our community a structured way to bring those programs back into view, and to connect them with the partners and funders who can help reactivate them.”

The platform is built so that people who own assets they want to develop can securely upload information about them into a database that is then searchable by possible interested parties such as investors, nonprofits, biotechs, and academic groups. They will have to pay a fee for this service, but the press statement says, “up to 10 of the initial listings will be eligible for discounted onboarding.”

When a match is achieved then the asset owner and the interested party will come to some sort of agreement on further financing. This could include traditional or alternative development and financing models, including nonprofit and hybrid structures.

The platform can theoretically be used by people from around the world, but users must apply and be approved rather than simply signing up. According to the platform’s terms and conditions on its website, “access is restricted to authorized representatives of biotechnology companies, academic institutions, nonprofits, and accredited investors.”

The post Exchange Marketplace Launched to Help Stalled Cell and Gene Therapies appeared first on Inside Precision Medicine.

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STAT+: Takeda will pay $13.6 million to settle allegations it paid kickbacks to doctors

Takeda Pharmaceuticals agreed to pay $13.6 million to settle allegations that it paid kickbacks to doctors to prescribe an antidepressant and, consequently, violated federal law by causing Medicaid to pay false claims, the U.S. Department of Justice said in announcing the settlement.

From January 2014 to October 2020, the company allegedly offered speaking fees and paid for meals at “high-end” restaurants to persuade physicians to prescribe Trintellix. Moreover, certain doctors who attended multiple programs on the same topic and received meals and drinks did not gain any educational benefit from attending the programs.

“This settlement demonstrates the continued commitment of my office to ensure that patients’ best interests remain paramount,” Eric Grant, the U.S. attorney for the Eastern District of California, said in a statement. “Prescribing decisions should not be influenced by drug companies’ payments or side perks made available to physicians.”

Continue to STAT+ to read the full story…

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ASGCT Q1 Landscape Report Paints Positive Picture for Gene and RNA Therapy

BOSTON – The CEO of the American Society for Gene and Cell Therapy (ASGCT), David Barrett, JD, presented highlights from the Society’s latest Landscape Report on Cell, Gene and RNA Therapy for the first quarter (Q1) of 2026.

The ASGCT report is developed in conjunction with Citeline, a subsidiary of Norstella (a pharmaceutical intelligence provider covering drug development from preclinical to commercialization).

Barrett said there are currently 42 gene therapies approved worldwide, along with 38 RNA therapies and 76 (non-genetically modified) cell therapies, which are steadily growing the field. Two cell therapies were approved in Japan in Q1.

There was a small increase in deal-making, and a significant 30% increase in startup funding compared to the same period in 2025. “I think that signals and underscores a rebounding sector,” said Barrett.

Of the eight gene therapies approved over the past 12 months, half were in the United States, with three more in China. “The regulatory pace is starting to pick up, another strong indicator for the future of our field,” Barrett said. It is a similar picture in RNA therapies. “We see a steady uptick over the course of the last year,” he added.

Zooming out, Barrett estimated that there are more than 4,200 therapies currently in development, from preclinical through pre-registration. The vast majority of those (more than 4,130) are gene and genetically modified cell therapies, including about 1,300 RNA therapies.

In the field of gene-modified cell therapies, CAR T continues to lead the pipeline for ex vivo gene therapies, with natural killer (NK) and T-cell receptors gaining traction. Not surprisingly, genetically modified cell therapy overwhelmingly targets cancers, but Barrett noted growth in the percentage of these therapies targeting immunological diseases, including lupus, multiple sclerosis, and HIV.

Pipeline growth

Barrett also noted growth and “a promising future” in the clinical trials pipeline. There are currently 350 Phase I, 319 Phase II, and 41 Phase III trials in gene therapy (up from 35 a year ago). “Hopefully, we will see a number of completed trials and FDA decisions in the near term,” said Barrett. A growing proportion of gene therapy trials (exceeding 60 percent) is for non-oncology indications.

In the RNA space, “RNAi therapies are jumping,” said Barrett. The same cannot be said, however, for mRNA. “Unsurprisingly, mRNA therapies continue to slide quarter over quarter,” a symptom of “shaken confidence” in that space, he continued. RNA therapies are targeting primarily non-oncology indications, especially in rare diseases.

Upcoming catalysts

On the business front, Barrett noted there has been “a nice uptick” in Q1 in start-up funding compared to the same quarter last year, which he deemed “a really promising indication.” The number of start-ups historically has tended to hover between 5-20. For Q1, that number was 26.

The Q1 report tracks various business catalysts anticipated through the end of 2027, including increased interest and uptake in expedited review designations—fast track, RMAT, orphan drug breakthroughs and other accelerated approval pathways.

“FDA is getting a lot done… and hopefully we’ll see the same moving forward,” Barrett said.

 

The full Landscape Report is available online from the ASGCT website.

The post ASGCT Q1 Landscape Report Paints Positive Picture for Gene and RNA Therapy appeared first on GEN – Genetic Engineering and Biotechnology News.