Latest news with #clinical trial
Reuters
a day ago
- Business
- Reuters
Sarepta's licensing partner Arrowhead expects near-term payments despite setbacks
July 23 (Reuters) - Sarepta Therapeutics' (SRPT.O), opens new tab licensing partner Arrowhead Pharmaceuticals (ARWR.O), opens new tab said on Wednesday it expects to receive near-term milestone payments from the drugmaker despite recent setbacks, including the death of a trial patient reported last week. Shares of Arrowhead rose nearly 4% in premarket trading. Its stock has declined 11% since Sarepta disclosed that a 51-year-old man who received its experimental gene therapy SRP-9004 died of liver failure. Investors and analysts criticized Sarepta for reporting the patient's death a day after it disclosed cost cutting efforts, including its plans to halt the study and layoffs. Two teenage boys who received Elevidys, Sarepta's gene therapy approved for a rare muscular dystophy, have also died of liver toxicities this year. Sarepta's shares declined 25% in the last week, and have fallen nearly 90% so far this year. Stock fell 1.6% to $13.4 in premarket hours. The companies entered into an agreement in late 2024, and Sarepta gained licensing rights to four of Arrowhead's early-stage experimental therapies. "Sarepta has provided no indication of any intention to fail to fulfill any of its obligations," the Pasadena, California-based Arrowhead said. Arrowhead expects to receive $300 million by the end of this year, related to patient enrollment for its early-to-mid stage study of ARO-DM1, which is being tested for a genetic condition. But if Sarepta fails to make certain payments, Arrowhead would have the right to terminate the partnership, the drug developer said. Provisions of the deal allow both the parties to terminate the agreement under certain circumstances. Bernstein analyst William Pickering said earlier in the week that there was not much reason for Arrowhead to pull out of the deal unless they find another partner, as it does not have enough financial resources to launch the therapies on its own.
Bloomberg
3 days ago
- Health
- Bloomberg
Sarepta Announces Voluntary Pause of Elevidys Shipments in US
Sarepta Therapeutics Inc. announced that the company plans to temporarily pause all shipments of its gene therapy to treat Duchenne muscular dystrophy, Elevidys, in a reversal of its prior stance. The Food and Drug Administration asked Sarepta to pause shipments of the drug on Friday following news reports of a patient death in a clinical trial for another Sarepta therapy that works in a similar way. The company initially refused, which drew backlash.
Yahoo
6 days ago
- Business
- Yahoo
Third Fatal Liver Failure Hits Sarepta Gene Trial
Sarepta (NASDAQ:SRPT) has now seen a third fatal incident in its gene therapy trials due to acute liver failure. That happened last month in a limb girdle muscular dystrophy study and follows two teenage deaths in advanced Duchenne muscular dystrophy patients. The trial was halted and Elevidys distribution paused for non walking patients. Warning! GuruFocus has detected 3 Warning Signs with SRPT. The company reported the latest death promptly to regulators and agreed to alert doctors and patients about liver failure risks as the FDA requested. Talks are underway on how to protect non walking patients going forward. To steady its finances, Sarepta will cut more than a third of its workforce and pause several drug programs. This move is expected to save around four hundred million dollars a year. Investors will watch closely for signs that these steps can rebuild confidence. This article first appeared on GuruFocus.
Yahoo
6 days ago
- Business
- Yahoo
Sarepta share price drops again after third gene therapy death
Sarepta Therapeutics' share price has fallen again following the death of a third patient dosed with its gene therapy treatment. A 51-year-old man with limb-girdle muscular dystrophy (LGMD) type 2D/R3 died after receiving Sarepta's gene therapy candidate SRP-9004 in a Phase I trial, according to Bloomberg. Sarepta Therapeutics was approached for comment by Clinical Trials Arena but did not respond before publication. This comes after two deaths were announced following treatment with Sarepta's Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec). Both patient deaths, the first announced in March 2025 and the second in June 2025, were due to acute liver failure (ALF). Elevidys and SRP-9004 both use the same recombinant adeno-associated viral vector (AAV) in AAVrh74. Reports state that Sarepta has informed regulators and clinical investigators of the latest death after dosing with SRP-9004. Earlier this week, Sarepta said it was cutting 500 jobs as part of its restructuring and pipeline prioritisation plan, as well as updating the label for Elevidys. The company is engaging with the US Food and Drug Administration (FDA) regarding the label update for the gene therapy, which includes adding a black box warning for acute liver injury (ALI) and ALF. Nasdaq-listed Sarepta saw its share price jump 19.53%, rising from $18.38 at the close of 16 July to $21.97 on July 17, following news of the restructuring and new Elevidys label. However, shares plunged 22.62%, to open at $17.00 on 18 July after the company announced the death of the patient in the clinical trial. In May 2025, the FDA announced that American haematologist oncologist Vinay Prasad will lead its Center for Biologics Evaluation and Research (CBER), the division responsible for regulating gene therapies and vaccines. Prasad has openly criticised Elevidys, primarily about the clinical evidence and the FDA's decision to approve the therapy. CGT sector at a 'crossroads' GlobalData healthcare analyst Momna Ali said the recent setbacks for Sarepta put the cell and gene therapy (CGT) sector at a 'crossroads'. Ali said: 'Following Sarepta's announcement of a third patient death, a new label for Elevidys and them laying off 500 employees, or 36% of its workforce, shelving parts of its pipeline to save $420m, and shifting focus from gene therapy to siRNA programmes, this is going to put the CGT sector at a pivotal crossroad. "While the scientific potential of CGT therapies remains extraordinary, this moment serves as a reminder of the complexity, cost, and responsibility involved. There is a lot of buzz around therapies 'beyond the pill'; however, for the landscape to keep evolving at the pace it has been in the last 3-5 years, there has to be greater transparency, patient safety, and sustainable innovation - otherwise, it'll be met with more setbacks.' Pfizer pulled an investigational DMD therapy last year after the death of a patient in a Phase II trial and a Phase III trial failed to meet its primary endpoint. In May 2025, a patient in Rocket Pharmaceuticals' pivotal Phase II trial of gene therapy for Danon disease died after suffering a serious adverse event (AE). "Sarepta share price drops again after third gene therapy death" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
Bloomberg
7 days ago
- Health
- Bloomberg
Sarepta Says Another Patient Died After Receiving Gene Therapy
Sarepta Therapeutics Inc. said another patient has died from acute liver failure after receiving one of its gene therapies, putting additional pressure on the biotech company after the recent deaths of two teenage boys. A company spokesperson said a patient had died of acute liver failure last month in an early-stage trial of a gene therapy to treat limb-girdle muscular dystrophy, which often weakens muscles around the hips and shoulders. The company told regulators and investigators about the death 'in an appropriate and timely manner,' the spokesperson said in a statement.
