Sanofi revealed that its investigational BTK inhibitor rilzabrutinib notched a significant win in the LUNA 3 phase 3 study, hitting the primary endpoint of durable platelet response in adults with persistent or chronic immune thrombocytopenia (ITP). The study showed a significantly higher proportion of rilzabrutinib-treated patients achieved the platelet response goal compared to placebo in…
HELIOS trial Q&A: Amylyx Pharmaceuticals’ AMX0035 as a potential treatment for Wolfram syndrome
The recently launched phase 2 HELIOS clinical trial from Amylyx Pharmaceuticals, aims to explore the potential of AMX0035 (sodium phenylbutyrate and taurursodiol) in treating Wolfram syndrome, a rare and complex genetic disorder. In a recent interview, Drs. Lahar Mehta and Fumihiko Urano discussed the AMX0035 Wolfram syndrome trial design, objectives and implications for future research…
FDA approves Bristol-Myers Squibb immunotherapy for gastric cancer
The FDA announced today that it approved Opdivo (nivolumab) in combination with chemotherapy to treat gastric cancer, among other diseases. Opdivo, developed by Bristol-Myers Squibb, when used in combination with certain types of chemotherapy is designed to treat advanced or metastatic gastric cancer, gastroesophageal junction cancer and esophageal adenocarcinoma. It is now the first FDA-approved…
Investigational drug gives sight to patient with inherited form of blindness
A patient with childhood blindness has gained durable vision improvement after a single injection of sepofarsen, an investigational RNA therapy. The patient had a rare genetic disorder known as Leber congenital amaurosis (LCA) that affects the retina and often causes severe visual impairment. Injection of sepofarsen led to measurable changes in the fovea, a depression…
Why RedHill Biopharma is expanding its focus on infectious disease
The specialty biopharmaceutical company Redhill Biopharma (NSDQ:RDHL) recently announced that the first patient had been dosed in a Phase 2/3 investigating the potential of RHB-107 (upamostat) for mild-to-moderate COVID-19. The company is also testing the potential of a drug known as opaganib for hospitalized patients. In the following interview, Gilead Raday, COO at Redhill Biopharma,…
OncoSec eyes next steps with its cancer immunotherapy candidate
OncoSec (NASDAQ: ONCS), a cancer immunotherapy company currently focused on R&D, is planning U.S. promotion of its tavokinogene telseplasmid (TAVO) drug for a type of metastatic melanoma — and eventually other cancers. “We don’t currently have a sales force. If successful, we’re going to need to have a sales and marketing team embedded in the…
FDA releases guidance to speed personalized drug development
The FDA today released draft guidance designed to make it easier for researchers to seek approvals for drugs designed for individuals with severe genetic diseases. The draft guidance focuses on investigational new drug submissions for antisense oligonucleotide (ASO) products because these are the most common drugs being developed for severely debilitating or life-threatening genetic diseases.…
How COVID-19 has affected Big Pharma and biotech
Biotechnology traces its roots to the Neolithic era when early civilizations discovered how to ferment grains to prepare alcoholic brews and bread. It wasn’t until genetic engineering became mainstream in the mid-1970s, however, that biotechnology, as we know it today, was launched. The field continues to evolve today. The advent of new technology has revolutionized the market…
Amylyx Pharmaceuticals wins orphan drug designation from FDA
The neurodegenerative-diseased-focused biotech Amylyx Pharmaceuticals announced that it had received orphan drug designation for AMX0035, a proprietary blend of sodium phenylbutyrate and tauroursodeoxycholic acid. The Cambridge, Mass.-based company won that designation to treat Wolfram syndrome, a rare genetic disease associated with diabetes, progressive optic atrophy, hearing loss and severe neurological disabilities. The prognosis for patients…
Scholar Rock sees stock soar after announcing positive SMA results
A monoclonal antibody that blocks the activation of the skeletal muscle protein myostatin could be novel muscle-directed therapy for spinal muscular atrophy patients. Cambridge, Mass.–based Scholar Rock (NSDQ:SRRK), which is developing the SRK-015 investigational antibody, saw its shares more than double to $30.02 apiece yesterday. SRK-015 could lead to the formation of a new treatment…
FDA approves first Ebola virus treatment
The FDA granted approval to Inmazep, a mixture of three monoclonal antibodies, making it the first FDA-approved treatment for Ebola virus. Regeneron Pharmaceuticals (NSDQ:REGN) received the approval, having recently been in the news for providing its investigational COVID-19 therapeutic to President Donald Trump. Inmazeb targets the glycoprotein that is on the surface of the Ebola virus.…
The most innovative pharmaceutical agents of 2020
The Galien Foundation recently announced nominees for most innovative pharmaceutical agents for its 14th Annual Prix Galien USA Awards. The foundation bestows the Prix Galien Award annually to examples of outstanding biomedical, pharmaceutical drugs and medical technology products that are designed to improve the human condition. “As we celebrate 50 years of the Prix Galien…
Genentech’s Rituxan Meets Endpoints in Phase III Study
Genentech announced positive top line results from the Roche-sponsored Phase III Pemphix study evaluating the efficacy and safety of Rituxan (rituximab) compared to mycophenolate mofetil (MMF) in adults with moderate to severe pemphigus vulgaris (PV). The study met the primary endpoint, and demonstrated that Rituxan is superior to MMF in achieving sustained complete remission. “The Pemphix study provides…
A Promising New Treatment for Infants with Noonan Syndrome
When Sequencing Fails to Pinpoint a Rare Disease
FDA Approves Treatment for Patients With Rare Type of Immune Disease
FDA Approves First Treatment Specifically for Patients With Rare Immune Disease
The FDA has approved Novimmune SA’s Gamifant (emapalumab) for the treatment of pediatric (newborn and above) and adult patients with primary hemophagocytic lymphohistiocytosis (HLH) who have refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. This FDA approval is the first for a drug specifically for HLH. “Primary HLH is a rare and…
CHDI Foundation and IRBM Extend Huntington’s Disease Therapeutics Collaboration
IRBM, a global contract research organization (CRO), has strengthened its position in neurodegenerative disease research following an extension of its long-standing collaboration with CHDI Foundation, a nonprofit biomedical research organization exclusively dedicated to collaboratively developing therapeutics that substantially improve the lives of those affected by Huntington’s disease (HD). HD is a genetic neurodegenerative disease for…
IntraBio Receives Niemann-Pick Disease European Orphan Drug Designation
Alizé Pharma 3 to Build Biopharmaceutical Company Focused on Rare Endocrine and Metabolic Diseases
Alizé Pharma 3, a company specialized in developing therapeutic peptides for the treatment of rare endocrine and metabolic diseases, announced a key addition to its leadership team and the structuring of its product portfolio in line with its ambition to become a leading global rare disease company. Alizé Pharma 3 is the third venture…
Tiny Pacemakers Aim to Make Infant Heart Surgeries Less Invasive
FDA Approves Jazz Pharmaceuticals’ Xyrem for Treatment of Pediatric Narcolepsy
The FDA approved Xyrem (sodium oxybate) for the treatment of cataplexy and excessive daytime sleepiness (EDS) in pediatric patients (seven to 17 years old) with narcolepsy. Xyrem is a central nervous system (CNS) depressant that was approved in 2002 for the treatment of cataplexy in adult patients with narcolepsy. Cataplexy is a sudden and transient episode…
Immune Therapy Generates Promising Results in Ankylosing Spondylitis
AstraZeneca, Merck Obtain Orphan Drug Designation for Lynparza for Pancreatic Cancer
AstraZeneca and Merck announced that the FDA granted orphan drug designation (ODD) for Lynparza for the treatment of pancreatic cancer. Lynparza is currently being investigated as maintenance therapy in patients with germline BRCA-mutated (gBRCAm) metastatic pancreatic cancer whose disease has not progressed following first-line platinum-based chemotherapy. Pancreatic cancer is a rare, life-threatening disease that accounts for about 3…
Akcea Therapeutics Genetic Disease Treatment Gets FDA Clearance
The FDA approved Akcea Therapeutics’ Tegsedi (inotersen) for the treatment of polyneuropathy caused by hereditary transthyretin (hATTR)-mediated amyloidosis in adults. The self-administered RNA-targeting therapeutic was approved by European agencies three months ago. Tegsedi’s approval has come shorty after the FDA clearance of Alnylam Pharmaceuticals’ Onpattro (patisiran) for the treatment of adults with polyneuropathy due to hATTR, marking the first…