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Types of Mitochondrial Diseases and the Science Behind Them: A Journey from Dysfunction to Discovery
Notizia - Recent Pharma, Healthcare and Biotech Happenings
Jun 10, 2025
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Amylyx Pharmaceuticals announced that the FDA has granted Fast Track designation to its investigational antisense oligonucleotide (ASO), AMX0114, for the treatment of amyotrophic lateral sclerosis (ALS). The candidate targets calpain-2, an enzyme linked to axonal degeneration, a critical driver of ALS progression.
“Obtaining FDA Fast Track designation for AMX0114 is an important step forward in our mission to develop potential treatments for people living with ALS, a relentlessly progressive and fatal disease with limited therapeutic options,” said Dr. Camille L. Bedrosian, Chief Medical Officer, Amylyx. “The FDA’s recognition underscores both the seriousness of ALS and the promising preclinical evidence supporting AMX0114’s potential to target calpain-2.”
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The Fast Track designation facilitates closer interaction with the FDA, eligibility for Priority Review, and a potentially faster development pathway. AMX0114 showed improved neuronal survival and reduced extracellular neurofilament light (NfL) levels in preclinical studies across multiple models of neurodegeneration.
Amylyx began dosing patients in April 2025 in the Phase I LUMINA trial, a multinational, randomized, placebo-controlled study evaluating AMX0114’s safety and biological activity in ALS patients. Approximately 48 participants are being enrolled, with early cohort data expected later this year.
The FDA has awarded Breakthrough Therapy Designation to Cellectar Biosciences‘ investigational drug, iopofosine I 131, for the treatment of relapsed/refractory Waldenstrom macroglobulinemia (r/r WM). The agent, a novel phospholipid ether radioconjugate, is positioned as a potential first-in-class monotherapy targeting this life-threatening hematologic malignancy.
“The FDA’s Breakthrough Therapy Designation underscores the potential of iopofosine I 131, as it may offer substantial improvement on at least one clinically significant endpoint over available therapies,” said James Caruso, President and CEO, Cellectar. “With robust clinical data, expedited review designations, and compelling commercial potential, we believe iopofosine I 131 is an attractive candidate for collaboration and accelerated development.”
The designation is supported by data from the Phase II CLOVER WaM study (NCT02952508), where iopofosine I 131 demonstrated an overall response rate (ORR) of 83.6% and a major response rate (MRR) of 58.2%, far exceeding the trial’s primary endpoint. Results were presented at the 66th ASH Annual Meeting in December 2024 by Dr. Sikander Ailawadhi of Mayo Clinic.
In addition to Breakthrough status, iopofosine I 131 has already secured FDA Fast Track and Orphan Drug Designations and EMA PRIME and Orphan Designations. Cellectar recently submitted a data package to the EMA and expects a decision by late July 2025 on whether to proceed with a Conditional Marketing Authorization Application (MAA) for the European market.
The FDA has approved YolTech Therapeutics’ Investigational New Drug (IND) application for YOLT-101, an in vivo base editing therapy designed to target PCSK9 for the treatment of heterozygous familial hypercholesterolemia (HeFH), a rare genetic disorder leading to dangerously high cholesterol levels.
“The FDA IND clearance marks a significant milestone for YolTech,” said Dr. Yuxuan Wu, Co-founder and CEO of YolTech. “In vivo gene editing represents a new generation of therapeutics, offering one-time, durable solutions for chronic and genetic diseases. We are committed to advancing breakthrough gene editing solutions that offer transformative benefits for patients with severe genetic and cardiovascular diseases.”
YOLT-101 utilizes base editing to permanently modify the PCSK9 gene in vivo, aiming to deliver a long-lasting reduction in LDL cholesterol after a single administration. If successful, this approach could eliminate the need for chronic lipid-lowering treatments in HeFH patients.
With the IND now cleared, YolTech will proceed with its first-in-human clinical trial of YOLT-101 in HeFH patients, advancing a promising new gene editing approach for cardiovascular disease management.
Oncovita announced that the FDA has granted Orphan Drug Designation (ODD) to its lead investigational therapy, MVdeltaC, for the treatment of pleural mesothelioma, a rare, aggressive cancer with significant unmet medical need and limited therapeutic options.
“Receiving Orphan Drug Designation for MVdeltaC marks a major milestone for Oncovita and validates our approach of harnessing measles vaccine viruses to treat solid tumors,” said Stéphane Altaba, CEO of Oncovita. “This regulatory support strengthens our strategy to advance innovative immunotherapies as we prepare to enter clinical development with MVdeltaC by 2026.”
MVdeltaC is a novel oncolytic immunotherapy based on a genetically modified attenuated Schwarz strain measles virus, designed to selectively replicate in tumor cells, induce direct tumor lysis, and trigger a systemic anti-cancer immune response. The platform holds promise for improving outcomes in pleural mesothelioma and other difficult-to-treat solid tumors.
“With this designation, Oncovita is now well-positioned to advance MVdeltaC in the U.S. market,” added Dr. Stéphane Champiat, Head of Medical Affairs. The FDA’s ODD provides incentives such as tax credits, fee waivers, and up to seven years of market exclusivity upon approval, accelerating the path to market for therapies targeting rare diseases.
The FDA has granted approval to Merck’s ENFLONSIA (clesrovimab-cfor) for preventing respiratory syncytial virus (RSV) lower respiratory tract disease in newborns and infants entering their first RSV season. ENFLONSIA is a long-acting monoclonal antibody designed to provide durable protection through a typical 5-month RSV season with a single 105 mg dose regardless of weight.
“RSV disease is the leading cause of infant hospitalization in the U.S. and can lead to serious conditions like bronchiolitis and pneumonia,” said Dr. Octavio Ramilo of St. Jude Children’s Research Hospital and lead investigator for the CLEVER and SMART trials. “ENFLONSIA combines dosing convenience with strong clinical data showing significant reductions in RSV disease incidence and hospitalizations.”
FDA approval is based on results from the pivotal Phase IIb/III CLEVER trial, where ENFLONSIA reduced RSV-associated medically attended lower respiratory infections by 60.5% and hospitalizations by 84.3% compared to placebo. Additional supportive data came from the SMART trial, which evaluated ENFLONSIA versus palivizumab in high-risk infants.
“ENFLONSIA provides an important new preventive option with the same dose for all infants, helping reduce the burden of RSV on families and healthcare systems,” added Dr. Dean Y. Li, President of Merck Research Laboratories. CDC’s Advisory Committee on Immunization Practices will review use recommendations later this month, with U.S. availability targeted before the 2025–2026 RSV season.
Article in PDF
Jun 10, 2025
Table of Contents
Amylyx Pharmaceuticals announced that the FDA has granted Fast Track designation to its investigational antisense oligonucleotide (ASO), AMX0114, for the treatment of amyotrophic lateral sclerosis (ALS). The candidate targets calpain-2, an enzyme linked to axonal degeneration, a critical driver of ALS progression.
“Obtaining FDA Fast Track designation for AMX0114 is an important step forward in our mission to develop potential treatments for people living with ALS, a relentlessly progressive and fatal disease with limited therapeutic options,” said Dr. Camille L. Bedrosian, Chief Medical Officer, Amylyx. “The FDA’s recognition underscores both the seriousness of ALS and the promising preclinical evidence supporting AMX0114’s potential to target calpain-2.”
The Fast Track designation facilitates closer interaction with the FDA, eligibility for Priority Review, and a potentially faster development pathway. AMX0114 showed improved neuronal survival and reduced extracellular neurofilament light (NfL) levels in preclinical studies across multiple models of neurodegeneration.
Amylyx began dosing patients in April 2025 in the Phase I LUMINA trial, a multinational, randomized, placebo-controlled study evaluating AMX0114’s safety and biological activity in ALS patients. Approximately 48 participants are being enrolled, with early cohort data expected later this year.
The FDA has awarded Breakthrough Therapy Designation to Cellectar Biosciences‘ investigational drug, iopofosine I 131, for the treatment of relapsed/refractory Waldenstrom macroglobulinemia (r/r WM). The agent, a novel phospholipid ether radioconjugate, is positioned as a potential first-in-class monotherapy targeting this life-threatening hematologic malignancy.
“The FDA’s Breakthrough Therapy Designation underscores the potential of iopofosine I 131, as it may offer substantial improvement on at least one clinically significant endpoint over available therapies,” said James Caruso, President and CEO, Cellectar. “With robust clinical data, expedited review designations, and compelling commercial potential, we believe iopofosine I 131 is an attractive candidate for collaboration and accelerated development.”
The designation is supported by data from the Phase II CLOVER WaM study (NCT02952508), where iopofosine I 131 demonstrated an overall response rate (ORR) of 83.6% and a major response rate (MRR) of 58.2%, far exceeding the trial’s primary endpoint. Results were presented at the 66th ASH Annual Meeting in December 2024 by Dr. Sikander Ailawadhi of Mayo Clinic.
In addition to Breakthrough status, iopofosine I 131 has already secured FDA Fast Track and Orphan Drug Designations and EMA PRIME and Orphan Designations. Cellectar recently submitted a data package to the EMA and expects a decision by late July 2025 on whether to proceed with a Conditional Marketing Authorization Application (MAA) for the European market.
The FDA has approved YolTech Therapeutics’ Investigational New Drug (IND) application for YOLT-101, an in vivo base editing therapy designed to target PCSK9 for the treatment of heterozygous familial hypercholesterolemia (HeFH), a rare genetic disorder leading to dangerously high cholesterol levels.
“The FDA IND clearance marks a significant milestone for YolTech,” said Dr. Yuxuan Wu, Co-founder and CEO of YolTech. “In vivo gene editing represents a new generation of therapeutics, offering one-time, durable solutions for chronic and genetic diseases. We are committed to advancing breakthrough gene editing solutions that offer transformative benefits for patients with severe genetic and cardiovascular diseases.”
YOLT-101 utilizes base editing to permanently modify the PCSK9 gene in vivo, aiming to deliver a long-lasting reduction in LDL cholesterol after a single administration. If successful, this approach could eliminate the need for chronic lipid-lowering treatments in HeFH patients.
With the IND now cleared, YolTech will proceed with its first-in-human clinical trial of YOLT-101 in HeFH patients, advancing a promising new gene editing approach for cardiovascular disease management.
Oncovita announced that the FDA has granted Orphan Drug Designation (ODD) to its lead investigational therapy, MVdeltaC, for the treatment of pleural mesothelioma, a rare, aggressive cancer with significant unmet medical need and limited therapeutic options.
“Receiving Orphan Drug Designation for MVdeltaC marks a major milestone for Oncovita and validates our approach of harnessing measles vaccine viruses to treat solid tumors,” said Stéphane Altaba, CEO of Oncovita. “This regulatory support strengthens our strategy to advance innovative immunotherapies as we prepare to enter clinical development with MVdeltaC by 2026.”
MVdeltaC is a novel oncolytic immunotherapy based on a genetically modified attenuated Schwarz strain measles virus, designed to selectively replicate in tumor cells, induce direct tumor lysis, and trigger a systemic anti-cancer immune response. The platform holds promise for improving outcomes in pleural mesothelioma and other difficult-to-treat solid tumors.
“With this designation, Oncovita is now well-positioned to advance MVdeltaC in the U.S. market,” added Dr. Stéphane Champiat, Head of Medical Affairs. The FDA’s ODD provides incentives such as tax credits, fee waivers, and up to seven years of market exclusivity upon approval, accelerating the path to market for therapies targeting rare diseases.
The FDA has granted approval to Merck’s ENFLONSIA (clesrovimab-cfor) for preventing respiratory syncytial virus (RSV) lower respiratory tract disease in newborns and infants entering their first RSV season. ENFLONSIA is a long-acting monoclonal antibody designed to provide durable protection through a typical 5-month RSV season with a single 105 mg dose regardless of weight.
“RSV disease is the leading cause of infant hospitalization in the U.S. and can lead to serious conditions like bronchiolitis and pneumonia,” said Dr. Octavio Ramilo of St. Jude Children’s Research Hospital and lead investigator for the CLEVER and SMART trials. “ENFLONSIA combines dosing convenience with strong clinical data showing significant reductions in RSV disease incidence and hospitalizations.”
FDA approval is based on results from the pivotal Phase IIb/III CLEVER trial, where ENFLONSIA reduced RSV-associated medically attended lower respiratory infections by 60.5% and hospitalizations by 84.3% compared to placebo. Additional supportive data came from the SMART trial, which evaluated ENFLONSIA versus palivizumab in high-risk infants.
“ENFLONSIA provides an important new preventive option with the same dose for all infants, helping reduce the burden of RSV on families and healthcare systems,” added Dr. Dean Y. Li, President of Merck Research Laboratories. CDC’s Advisory Committee on Immunization Practices will review use recommendations later this month, with U.S. availability targeted before the 2025–2026 RSV season.
