PharmaFocus Today – Page 246 – media

Sarepta Fails Confirmatory Trial for DMD Therapy, Still Eyes Label Expansion

By Tristan Manalac Sarepta Therapeutics on Monday posted topline data from the Phase III EMBARK study, showing that its Duchenne muscular dystrophy gene therapy Elevidys (delandistrogene moxeparvovec-rokl) fell short of its primary efficacy endpoint, unable to significantly improve functional mobility versus placebo. Nonetheless, based on what the company calls “robust evidence” of “clinically meaningful treatment benefit” across all EMBARK’s pre-specified key functional secondary endpoints, Sarepta will push through with filing for a label expansion for Elevidys. “We have shared the EMBARK topline results with FDA leadership and they have confirmed that, based on the totality of the evidence, they are open to such label expansion if supported by review of the data, and that they intend to proceed rapidly with consideration of the submission,” Sarepta CEO Doug Ingram said in a statement. In EMBARK, a randomized, two-part crossover and placebo-controlled study, Sarepta dosed 125 Duchenne muscular dystrophy (DMD) patients aged ...
- Source: drugdu
- 117
- November 2, 2023
Pfizer Continues COVID Cliff in Q3 with First Quarterly Loss Since 2019

By Tyler Patchen Pfizer reported third-quarter financial results Tuesday, pulling in $13.2 billion in revenues, a 42% drop compared to the prior-year period and the first quarterly loss since 2019. Third-quarter revenues for antiviral treatment Paxlovid dropped 97% operationally compared with the prior-year period, while COVID-19 vaccine Comirnaty revenues declined 70% in the quarter. However, Pfizer’s non-COVID products grew 10% operationally. Sales of its other vaccines, such as Prevnar 13 and 20 for pneumococcal conjugate vaccination, brought in over $1.8 billion in the third quarter while its RSV vaccine Abrysvo pulled in $375 million. “We are encouraged by the strong performance of Pfizer’s non-COVID products in the third quarter of 2023, including significant contributions from new launches and robust year-over-year growth for several key in-line brands,” CEO Albert Bourla said in a statement. Pfizer CFO David Denton noted in the release that new product launches will meet the company’s non-COVID ...
- Source: drugdu
- 116
- November 2, 2023
Merck KGaA and Hengrui Pharmaceuticals announce oncology partnership worth over €1.4bn

Merck KGaA has signed a licensing agreement worth over €1.4bn with Jiangsu Hengrui Pharmaceuticals for the rights to its next-generation PARP1 inhibitor HRS-1167 outside of China. The deal also includes an option for an exclusive licence for its Claudin-18.2 antibody drug conjugate (ADC), SHR-A1904. Compared to first-generation PARP inhibitors, HRS-1167 has higher selectivity and affinity for PARP1 and induces DNA trapping, Merck said, adding that the candidate is currently in phase 1 clinical development and “has the potential to be used as a monotherapy and as part of a combination therapy for treating a wider range of patients”. Danny Bar-Zohar, global head of research and development and chief medical officer for the Healthcare business of Merck KGaA, said: “This partnership with Hengrui fully aligns with both our external innovation ambition and our oncology research and development strategy by diversifying our robust internal pipeline in our focus areas of DNA damage ...
- Source: drugdu
- 114
- November 2, 2023
Shorla acquires methotrexate solution from Therakind in the US

Shorla Oncology has acquired the oncology and autoimmune drug Jylamvo (methotrexate) from Therakind, for commercialisation in the US market. Jylamvo was approved by the US Food and Drug Administration (FDA) approval in November 2022, to treat adults with acute lymphoblastic leukaemia, mycosis fungoides, relapsed or refractory non-Hodgkin lymphoma, rheumatoid arthritis and severe psoriasis. Shorla is headquartered in Ireland and has operations in the US. The company had raised $35m earlier this month to advance its oncology pipeline. Jylamvo is an oral solution, making the treatment accessible to patients who may struggle taking tablets or other dosage forms. Commercialisation is set to begin immediately in the US. Chief Technical Officer and founder of Shorla Oncology Orlaith Ryan said: “This acquisition will provide a much-needed treatment to patients in need. It brings a crucial oral treatment to a larger patient population who suffer from cancer and other debilitating illnesses.” In March, Shorla ...
- Source: drugdu
- 107
- November 2, 2023
Sarepta’s Elevidys fails on primary endpoint, but executives see path to wider DMD approval anyway

After Sarepta Therapeutics overcame several regulatory hurdles to finally win FDA approval for its Duchenne muscular dystrophy (DMD) gene therapy Elevidys, the company now faces the prospect of more FDA scrutiny because a pivotal study on the drug has failed on the primary endpoint. The endpoint, a measure of motor function called the North Star Ambulatory Assessment (NSAA), failed to reach statistical significance in the phase 3 EMBARK study, Sarepta said in a Monday release. Despite this result, Sarepta Chief Scientific Officer Louise Rodino-Klapac, Ph.D., touted “clinically meaningful” effects seen across the trial’s secondary endpoints. Speaking on a Monday conference call, Rodino-Klapac said Sarepta plans to file for a label expansion to treat “all DMD patients” and to convert the drug’s accelerated approval into a traditional nod. Sarepta CEO Doug Ingram said the study “met the standard” to show “evidence of effectiveness.” He added that the results show that the ...
- Source: drugdu
- 99
- November 2, 2023
Amid COVID slump, Pfizer turns attention to RSV launch and a potential future in obesity

As COVID revenues dwindle for pandemic stalwart Pfizer, the company is turning attention to its respiratory syncytial virus (RSV) vaccine launch and a potential entry into the hot obesity market. With only five months of sales under its belt, Pfizer’s RSV vaccine Abrysvo is “doing better than we thought,” Chief Commercial Officer Angela Hwang said on the company’s third-quarter earnings call, touting “very fast uptake” for the new shot. After a pair of approvals in late May and August, Abrysvo pulled in revenues of $375 million during the third quarter, Pfizer said Tuesday. Earlier this year, the shot won FDA nods to immunize older adults and then as a maternal vaccination to protect infants. In both settings, the Pfizer offering is facing off against Big Pharma competition. In the adult RSV vaccine marketplace, Pfizer is challenging GSK and its immunization Arexvy. GSK is set to report its third-quarter results Wednesday. ...
- Source: drugdu
- 106
- November 2, 2023
Intuitive stock drops on mixed Q3 financial results

BY JIM HAMMERAND The Da Vinci Xi robotic surgery system. Intuitive continues to dominate the space. [Image courtesy of Intuitive] Intuitive Surgical (Nasdaq: ISRG)+ stock declined after the surgical robotics developer missed on revenue for the third quarter but beat analysts’ expectations on profit. Shares of ISRG dropped 8% to $251 in after-hours trading when Intuitive released the results. MassDevice’s MedTech 100 Index — which includes stocks of the world’s largest medical device companies — was down slightly on the day. The Sunnyvale, California-based surgical robotics leader posted profits of $416 million, or $1.16 per share for the three months ended Sept. 30, 2023. That was a 28% bottom-line gain compared to Q3 2022. The company reported sales of $1.74 billion, up 12% from the same quarter last year. Intuitive attributed the gains to growth in da Vinci surgical robot procedures and an increase in the installed base of systems. Adjusted earnings per share ...
- Source: drugdu
- 140
- October 31, 2023
Roche Cuts Pipeline as COVID, Currency Headwinds Impact Q3 Results

Pictured: Roche tower in Switzerland/iStock, olli0815 Roche is eliminating four clinical programs amid drooping sales brought about by a sharp decline in demand for COVID-19 products and a strong Swiss franc, the pharma group announced Thursday morning in its third-quarter earnings results. According to a development pipeline document released alongside the quarterly report, Roche will end a Phase I solid tumor trial for its investigational CEA/CD3 bispecific antibody cibisatamab. The company will also stop Phase II trials of its antipsychotic drug candidate ralmitaront in schizophrenia and the developmental cannabinoid receptor agonist vicasinabin in diabetic retinopathy. Roche discontinued the mid-stage study of ralmitaront after it failed a Phase II trial in May 2023, unable to elicit significant improvements on negative symptoms in a preliminary analysis. The vicasinabin and cibisatamab programs were likewise scrapped following underwhelming reviews of their efficacy in their respective studies. However, in a media call Thursday morning, a Roche spokesperson said that these two ...
- Source: By Tristan Manalac
- 143
- October 31, 2023
Supply disruptions are delaying surgeries and compromising patient care, survey finds

Dive Brief Shortages of medical devices and therapies are compromising patient care by delaying treatment and driving unsafe practices, according to a survey of healthcare professionals conducted by the patient safety nonprofit ECRI and the Institute for Safe Medication Practices. The survey found that supply shortages have caused surgical cases to be rescheduled, postponed or canceled, and that a lack of endotracheal tubes and pulmonary artery catheters is on the cusp of impacting the ability to provide adequate clinical care. ECRI and the Institute for Safe Medication Practices used the findings to call for “long-term, nationally coordinated solutions” to stop persistent shortages. Dive Insight The Food and Drug Administration currently lists 11 medical devices that are in short supply. More than 120 drugs face shortages, according to another FDA list. The tendency for some products to become hard to source, for reasons such as manufacturing problems and rising demand, predates ...
- Source: https://www.medtechdive.com/news/supply-disruptions-delaying-surgeries-ecri/697140/
- 215
- October 31, 2023
Pupil size may predict response to TMS treatment for depression

New findings from researchers at UCLA Health suggest that measuring changes in how pupils react to light could help predict recovery from depression and personalize transcranial magnetic stimulation (TMS) treatment of major depressive disorder. TMS is a safe, non-invasive therapy that uses magnetic fields to stimulate parts of the brain involved in mood regulation. While TMS is proven effective, not all patients respond equally well to the therapy. The ability to predict who will benefit most could allow doctors to better customize and target treatments. In two recent studies, UCLA scientists found that the pupil’s response to light before treatment correlated with improvements in depression symptoms over the course of therapy. Pupil size reflects activation of the autonomic nervous system, which controls involuntary functions and is negatively impacted in people with depression. The first study, appearing in the Journal of Affective Disorders, reports on outcomes for 51 patients who underwent ...
- Source: https://www.news-medical.net/news/20231020/Pupil-size-may-predict-response-to-TMS-treatment-for-depression.aspx
- 119
- October 31, 2023

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