Drug Discovery and Development Archives - Page 67 of 158

Roche’s Evrysdi wins FDA approval in newborns with spinal muscular atrophy

Roche (SIX:RO, ROG; OTCQX:RHHBY) has announced that the FDA has approved a label extension to allow the use of Evrysdi (risdiplam) in babies younger than two months old with spinal muscular atrophy (SMA).

SMA is a genetic disease associated with progressive muscle wasting and weakness that affects roughly 1 in 11,000 babies.

The drug first won FDA approval in 2020 for adults and children at least two months old.

The label extension was based on interim efficacy and safety data from the RAINBOWFISH study in newborns.

That data demonstrated the majority of pre-symptomatic babies who received Evrysdi achieved developmental milestones related to sitting and standing. In addition, half of the babies were walking after 12 months of treatment.

“The approval of Evrysdi for pre-symptomatic babies is particularly important, as early treatment of SMA before symptoms start to arise can help babies to achieve motor milestones,” said Dr. Richard Finkel, …

Cybin files IND with FDA for early human study focused on major depressive disorder

The biopharma Cybin Inc. (NYSEAMERICAN:CYBN) has filed a submission of an Investigational New Drug (IND) application with the FDA for its Phase 1/2a first-in-human clinical trial focused on CYB003.

CYB003 is a proprietary psilocybin analog as a proposed treatment of major depressive disorder (MDD) as an adjunct to psychotherapy.

The study would enroll approximately 40 patients, according to a description on ClinicalTrials.gov. The trials would focus on studying the safety, pharmacokinetics and pharmacodynamics of multiple ascending doses of CYB003 in patients with MDD.

The proposed study would use the Montgomery-Asberg Depression Rating Scale to gauge improvements in mood and other parameters.

Psilocybin has received a growing amount of interest in recent years. The compound is the focus of more than 50 studies, including Phase 2 studies focused on depressive and post-traumatic stress disorders.

Cybin is also investigating the potential of …

FDA approves new Opdivo indications for upper gastroesophageal cancers

Bristol Myers Squibb (NYSE:BMY) has won FDA approval for Opdivo (nivolumab) in combination with fluoropyrimidine- and platinum-based chemotherapy as a first-line treatment for adults with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC).

The agency also approved Opdivo with Yervoy (ipilimumab) for ESCC.

Both approvals cover ESCC regardless of programmed death-ligand 1 (PD-L1) status.

First FDA approved in 2014 for advanced melanoma, Opdivo has won a score of oncology indications over the years.

The drug now has five indications covering upper gastroesophageal cancers.

The two recent approvals are based on data from the Phase 3 CheckMate -648 trial, which Bristol Myers Squibb notes is the largest Phase 3 trial of an immunotherapy in first-line ESCC. In that study, Opdivo with chemotherapy offered a superior overall survival rate than chemotherapy alone.

Opdivo with Yervoy also led to improved survivability…

U of Maryland scientist to set up NIH’s new Advanced Research Projects Agency for Health

U.S. Dept. of Health and Human Services (HHS) announced that it formed a new research project agency for health.

HHS Secretary Xavier Becerra announced the formal establishment of the Advanced Research Project Agency for Health (ARPA-H) as an independent entity within the National Institutes of Health (NIH). Adam H. Russell was appointed as acting deputy director, effective next month.

Russell currently serves as chief scientist at the University of Maryland’s Applied Research Laboratory for Intelligence and Security (ARLIS). He previously spent more than 10 years as a program manager, first at the Intelligence Advanced Research Projects Activity (IARPA) and then at the Defense Advanced Research Projects Agency (DARPA).

At Darpa, Russell focused on new experimental platforms and tools to facilitate discovery, quantification and “big validation” of fundamental measures in social science, behavioral science and human performance, accordin…

Janssen touts positive Tremfya and Stelara data for IBD at Digestive Disease Week

Johnson & Johnson’s (NYSE:JNJ) Janssen unit announced new Phase 2 GALAXI 1 data at Digestive Disease Week (DDW), showing substantial rates of clinical-biomarker and endoscopic response and clinical remission through 48 weeks of Tremfya (guselkumab) therapy for Crohn’s disease.

The company also announced long-term pooled safety analyses for Stelara (ustekinumab) at the DDW event, which is scheduled for May 21-24, in San Diego, California,

In the GALAXI 1 study focused on adults with moderately severe Crohn’s disease who were poor candidates for traditional therapies or biologics, guselkumab recipients had a clinical biomarker response between 47.5% and 66.7% at week 48 across dose groups. About 45% of such patients had an endoscopic response. In addition, between 39.3% and 66.7% achieved remission based on C-reactive protein (CRP) or fecal calprotectin levels in the same time interval.

At present, Tremfya is not FDA approved for Croh…

7 strategies to improve PROTACs’ oral bioavailability

[PROTAC image courtesy of Wikipedia]

PROteolysis TArgeting Chimera (PROTAC)* drugs emerged in 2001 as a novel therapeutic option for treating previously untreatable diseases. To date, no PROTAC drugs have earned U.S. FDA approval, but the technology has opened up a promising new chapter in drug discovery. PROTACs are being widely explored across both industry and academia and have expanded from treating cancer to immune disorders, neurodegenerative diseases, viral infections, etc.

A PROTAC molecule consists of three components: a ligand binding to a target protein, a ligand binding to E3 ligase and a linker. Unlike conventional small molecules that block target proteins via inhibition, PROTACs bind specifically to target proteins and induce their degradation by leveraging the ubiquitin-proteasome system (UPS). PROTACs’ binding process is referred to as a “chemical knockdown” approach.

PR…

How the CMS decision to limit Aduhelm coverage could impact Medicare pricing

Since winning approval for the Alzheimer’s drug Aduhelm (aducanumab) in June 2021, Biogen (Nasdaq: BIIB) has faced a punishing string of setbacks. As a result, the company’s share price is currently trading near $200 per share — less than half of its $414.71 peak on June 10, 2021.

In April 2022, the Centers for Medicare & Medicaid Services (CMS) dealt one of the most punishing blows to Aduhelm’s future by limiting coverage for the drug to clinical trial participants.

The CMS decision was “certainly an unusual action from CMS,” said Corey Metzman, chief operating officer and co-founder of Chapter, a data-driven digital Medicare advisor. Typically, CMS grants Medicare coverage to FDA-approved drugs. “What was unique about [Aduhelm] is that it went through the accelerated approval process.”

Another wrinkle is that the drug failed to win the backing of the FDA’s Peripheral and Central Nervous System Drugs A…

CytoDyn settles legal dispute with former chief medical officer

The biotech CytoDyn (OTCQB:CYDY) has resolved a legal dispute with Dr. Richard Pestell, the company’s former medical officer who had been “fired with cause” in July 2019.

Today, CytoDyn announced that it had reached a “non-cash settlement” with Dr. Pestell, agreeing to release 8.3 million shares of its stock held in escrow.

in August 2019. the Australian oncologist and endocrinologist Pestell filed a legal complaint with claims related to wage collection, breach of contract, declaratory judgment, wage collection and defamation.

Dr. Richard Pestell [Image courtesy of Wikipedia]

As part of its settlement with Pestell, CytoDyn will also transfer assets acquired from ProstaGene in 2018.

Pestell was the founder and CEO of ProstaGene from February 2011 to November 2018.

A 2018 SEC document cites Pestell as the “Stockholder” of ProstaGene, while Pestell served as vice chairman of the board, …

AbbVie touts positive cariprazine data in major depressive disorder

The schizophrenia and bipolar drug cariprazine (Vraylar) fared well as an adjunctive therapy in the Phase 3 3111-301-001 study involving patients with major depressive disorder (MDD).

AbbVie (NYSE:ABBV) is aiming to win a new FDA indication for the drug as an adjunctive treatment of MDD by the end of the year.

According to MedPage Today, 44% of patients in the 1.5-mg arm had at least a 50% reduction in the Montgomery-Åsberg Depression Rating Scale (MADRS) score by week 2 of treatment.

The company did not divulge significant data in a news release, stating that patients receiving a 1.5mg/day dose of the drug met the primary endpoint of statistically significant improvement based on the MADRS at week six compared to placebo. Patients receiving 3.0 mg/day per day had an improved MADRS total score, but the results were not statistically significant.

Patients in the 1.5 mg group also saw improvements in their scores on the Hamilton Depression Rating Sc…

U.S. secures option to purchase millions of monkeypox vaccine doses from Bavarian Nordic

As monkeypox cases are investigated in North America and Europe, Bavarian Nordic A/S (OMX:BAVA) has secured a contract worth potentially $119 million with Biomedical Advanced Research and Development Authority (BARDA) to convert bulk vaccine into freeze-dried doses of Jynneos vaccine.

Jynneos was FDA approved for both smallpox and monkeypox in 2019.

Smallpox and monkeypox are broadly similar with the latter causing lymph nodes swelling while the former does not. Smallpox and monkeypox are broadly similar with the latter causing lymph nodes swelling while the former does not. Monkeypox also tends to be milder.

BARDA has additional options to convert up to roughly 13 million freeze-dried doses of the Jynneos vaccine that would likely be manufactured in 2024 and 2025.

BAVA shares have surged in recent days. Today, it rose 18.4% to 216.60 DKK. In the past five days, the stock has increased 63.6%.

Monkeypox remains extremely rare in the U.S., CD…

How 4D Path and PathPresenter aim to further AI-enabled digital pathology for breast tumor profiling

The digital pathology software company PathPresenter (Montville, New Jersey) and the precision oncology platform firm 4D Path have entered into a partnership that could improve the diagnostic accuracy of breast tumor profiling.

PathPresenter has developed a digital workflow tool for clinicians, pathologists and pharma companies to aggregate medical data.

The platform can be used for medical education, patient care and management. In addition, pharma companies can use it to create models for drug development.

“For these workflow solutions to be effective, you need good AI models running in the background,” said Dr. Rajendra Singh, co-founder of PathPresenter. “And one of the best companies on the planet right now producing AI models is 4D Path.”

That observation ultimately led to the partnership between the two companies. “It was a natural fit to bring in their AI models into a workflow solution to provide a complete p…

Moderna’s first bivalent COVID-19 vaccine booster candidate shows promise

Moderna’s (NYSE:MRNA) bivalent booster COVID-19 vaccine candidate, mRNA-1273.211, bested its first-generation mRNA-1273 vaccine in a Phase 2/3 study.

The study compared the neutralizing antibody levels generated from both vaccine boosters. The mRNA-1273.211 vaccine booster candidate offered superior performance against all variants of concern, including omicron.

The company notes that the mRNA-1273.211 booster outperformed the first-generation vaccine for six months.

In particular, the next-gen products led to a 2.20-fold boost in neutralizing antibody titers against omicron with the mRNA-1273.211 booster dose relative to the mRNA-1273 booster dose at 1 month. At 6 months, the new booster candidate led to a 2.15-fold increase in neutralizing antibody titers compared to the current booster.

mRNA-1273.211 is based on the beta variant, which shares some mutations with the omicron variant, whose subvariants continue to dominate the world.