Drug Discovery and Development Archives - Page 33 of 158

Psychedelic research firm Lucy Scientific Discovery misses IPO target

Lucy Scientific Discovery Inc. (Nasdaq:LSDI), a Canadian firm that focuses on developing and manufacturing psychotropics-based medicines, had its initial public offering (IPO) today. The company had priced 1,875,000 common shares at a public offering price of $4.00 per share.

On its first day of trading, however, the stock dropped by 25.25%, reaching a closing price of $2.99, a decline of 25.25%.

While the company had hoped to raise approximately $7.5 million before accounting for expenses related to the offering, it ultimately generated $5.6 million.

A different type of psychedelic company

Lucy Scientific Discovery differs from other psychedelic companies in that it is a licensed manufacturer of controlled substances. It aims to bolster its presence in the growing psychotropics-based medicines industry with growing contract research, development and manufacturing offerings.

The company has permission to manufacture psilocybin, psilocin, N,N-DMT, …

FDA approves Pfizer’s Cibinqo for adolescent atopic dermatitis

Pfizer (NYSE:PFE) has announced that the FDA has approved its supplemental New Drug Application (sNDA) for Cibinqo (abrocitinib) for moderate-to-severe atopic dermatitis. The approval expands the indication for the drug, which was previously only approved for adults with the skin condition in early 2022.

The expanded indication includes adolescents between the ages of 12 and 18 whose disease is not adequately controlled with other systemic drugs or when their use is inadvisable.

Cibinqo is a potential blockbuster

In 2020, Pfizer had estimated that abrocitinib would eventually garner $3 billion in peak annual sales.

The company is also investigating its potential in prurigo nodularis and pruritus.

The systemic JAK inhibitor is in the same class as baricitinib from Incyte and Lilly and upadacitinib from AbbVie.

The FDA updated the prescribing information for Cibinqo to include data from the JADE TEEN clinical trial, which evaluated the 100 mg…

Startup Evozyne identifies ‘supernatural’ proteins with Nvidia’s pretrained AI model

[Protein image courtesy of Nvidia]

AI computing firm Nvidia (Nasdaq:NVDA) and the Chicago-based biotech startup Evozyne have teamed up to develop an AI model for designing therapeutic proteins. Using NVIDIA’s BioNeMo language model service, the companies developed the Protein Transformer Variational AutoEncoder (ProT-VAE) model.

The initial focus of the Evozyne research team was on the PAH gene, which governs the production of the phenylalanine-hydroxylase enzyme. A mutation in the PAH gene has been linked to phenylketonuria, a rare condition characterized by elevated levels of phenylalanine that can lead to neurological problems.

With the goal of developing a novel treatment for phenylketonuria, ProT-VAE created new synthetic PAH variants that they predicted would have “supernatural” functionality. Lab tests eventually demonstrated that some of the protein variants were up to 2.5 times more effecti…

Zuranolone, novel oral medication for depression and postpartum depression, could be on the horizon

[Zuranolone image courtesy of PubChem]

Biogen (Nasdaq:BIIB) and Sage Therapeutics (Nasdaq:SAGE) have announced that the FDA accepted the New Drug Application (NDA) for zuranolone, a neuroactive steroid.

The drug candidate could be a novel 14-day oral medication to treat Major Depressive Disorders (MDD) or postpartum depression (PPD). Priority review has been granted and the application is slated to be processed by August 5, 2023.

LANDSCAPE and NEST programs

In their FDA filing, Biogen and Sage have shared data from the LANDSCAPE and NEST clinical development programs and a Phase 2 study conducted by Shionogi Japan.

The LANDSCAPE program contains five studies on zuranolone for adults with MDD, while the NEST program has two studies investigating the drug for adult women with PPD.

In clinical trials, patients with MDD and PPD showed efficacy with zuranolone in as few as three days. In contrast…

Regeneron’s Eylea approved for preterm infants with ROP, first drug treatment for condition

Regeneron Pharmaceuticals (Nasdaq:REGN) has received an indication for Eylea (aflibercept) injection for preterm infants with retinopathy of prematurity (ROP).

The approval is the first pharmacologic treatment for the condition, which affects roughly 50% to 70% of infants with a weight less than 1250 grams (2.76 pounds) at birth. The condition is more common in infants born before 31 weeks of pregnancy.

The condition is a common cause of childhood blindness.

A growing number of indications

FDA first approved Eylea for wet age-related macular degeneration in 2011. Indications followed for diabetic macular edema, macular edema following retinal vein occlusion and diabetic retinopathy in patients with diabetic macular edema.

The approval is based on data from two global Phase 3 trials, FIREFLEYE and BUTTERFLEYE. The studies compared EYLEA to laser photocoagulation in infants with ROP. The data showed that approximately 80% of EYLEA-treated infants…

Positive results for oral difelikefalin in Phase 2 nostalgia paresthetica study

NEJM has published positive results from the KOMFORT Phase 2 trial of oral difelikefalin for nostalgia paresthetica, a condition involving itching, burning or tingly sensations on the back.

Cara Therapeutics (Nasdaq:CARA) is the developer of the drug, which won FDA approval in an injectable form for moderate-to-severe pruritus in hemodialysis patients in 2021. That form of the drug has the trade name Korsuva. Difelikefalin is a selective kappa-opioid receptor agonist.

The company’s stock dipped about 2% to $11.67 after the company announced the data.

Drug appeared effective but associated with adverse events

The KOMFORT study’s primary endpoint was a change in the weekly mean of the 24-hour Worst Itch Numeric Rating Scale (WI-NRS) score. At 8 weeks, more patients achieved an at least 4-point improvement with oral difelikefalin than with placebo.

The study recruited 126 patients total.

The NEJM article noted that, while the drug supported mo…

Scleroderma treatment could leap forward with FT011 drug candidate

The story of the scleroderma drug candidate FT011 is one of “tenacity and persistence,” said Darren Kelly, CEO and managing director of Certa Therapeutics, which is developing the drug.

The drug was discovered around 2006 by Kelly’s laboratory at the University of Melbourne and Spencer Williams of the Bio21 Institute.

“It was really [Williams] being the chemist and me being the biologist working together on a problem and developing FT011,” Kelly said.

The drug was the lead asset of a company known as Fibrotech, where Kelly was CEO. Shire acquired the company in 2014. At that time, the drug had finished a Phase 1A study and was in a Phase 1B diabetic nephropathy trial.

In 2019, Takeda completed the acquisition of Shire, shelving several programs.

“We worked with Shire to take back FT011 and all the technology that was around that, and then set up Certa Therapeutics in 2018,” said Kelly

FT011 off…

Dual defense: FluGen’s M2SR vaccine shields against influenza and COVID-19

[Image courtesy of Adobe Stock]

For many years, vaccine developers have known that several problems with current influenza vaccines – short-lived serum antibodies, lack of immunity at the site of infection, and lack of robust protection against drifted strains – contribute to their modest efficacy. Although new approaches to influenza vaccine design, manufacture and administration have been explored, none has yielded an approved vaccine that addresses all of these problems.

mRNA COVID-19 vaccines have saved millions of lives worldwide since their approval, and their importance in combatting the pandemic cannot be overemphasized. However, these vaccines are already facing the same problems as influenza vaccines. Because they protect against illness but not infection, vaccinated individuals who contract the SARS-CoV-2 virus can still shed and transmit it.

To better control both viruses and the illnesses th…

Neuron23 launches clinical trial for NEU-723 in Parkinson’s with companion diagnostic in development

South San Francisco–based Neuron23 has started the first-in-human Phase 1 trial of NEU-723 for the treatment of Parkinson’s disease. NEU-723 is a highly potent and selective leucine-rich repeat kinase 2 (LRRK2) inhibitor. This clinical trial will evaluate the safety, tolerability and pharmacokinetics of NEU-723 in healthy volunteers.

A targeted approach

The company has also collaborated with the diagnostics firm Qiagen to develop a companion diagnostic to identify a sub-population of Parkinson’s disease patients likely to respond to LRRK2 inhibitors.

“We wanted to try to narrow the population to those who would benefit and respond,” said Nancy Stagliano, chief executive officer of Neuron23. “So we built a machine learning model that can predict if patients respond to this drug or not.”

Stagliano says the targeted approach is “a big differentiator.” “To our knowledge, no one else has done this in neurodegenerative disease,” she said.

Th…

FT011 yielded significant clinical improvements in more than 60% of scleroderma patients

Certa Therapeutics, a Melbourne-based biotech company, announced the results of its Phase 2 clinical trial for FT011, a novel oral therapy for chronic fibrosis.

The trial showed clinically meaningful improvements in more than 60% of scleroderma patients after 12 weeks of treatment.

In the Phase 2 study, 60% of patients treated with a 400 mg dose of FT011 showed a significant clinical improvement at 12 weeks while 20% of patients in the 200 mg did.

Several dimensions of improvement

Clinical trial investigators noted significant advances in lung function, patient self-assessment, and physician evaluation as measured by American College of Rheumatology Combined Response Index in diffuse cutaneous Systemic Sclerosis (CRISS) score, skin thickness, lung function, physician-reported assessment and quality of life evaluations.

The drug candidate was well tolerated in the study.

FT011 targets a G protein-coupled receptor (GPCR), which is inactive in…

Nipocalimab gets positive results in Phase 2 HDFN clinical trial

Johnson & Johnson subsidiary Janssen (NYSE:JNJ) has revealed positive results from the proof-of-concept Phase 2 UNITY clinical trial of nipocalimab for pregnant adults with a high risk of developing severe hemolytic disease of the fetus and newborn (HDFN).

HDFN is a rare condition affecting between 3 and 80 out of 100,000 newborns or fetuses each year. The serious autoimmune disease can lead to life-threatening anemia in the fetus.

The UNITY trial met its primary endpoint with most pregnant patients who received nipocalimab giving birth to a live baby at or after 32 weeks of gestation, without the need for intrauterine transfusions.

The treatment was administered as weekly intravenous infusions to 14 participants and was well-tolerated over a 20-week period.

FDA granted nipocalimab Fast Track designation in July 2019 and orphan drug status in June 2020.

Janssen acquired nipocalimab as part of its $6.5 billion acquisition of Momenta Phar…

Psychogenics CEO explores CNS drug development trends

In the late 2000s, many companies, particularly Big Pharma, exited the field of central nervous system (CNS) drug development and, particularly psychiatry. “Big Pharma exited the space because they were facing their patent cliff at the time and had to make some critical decisions about where to invest their resources,” said Emer Leahy, CEO of the contract research organization Psychogenics. “Oncology won out over CNS,” she added.

In a recent interview, Leahy opened up on the current renaissance in CNS drug development, including the role of AI while also touching on the impact of the Inflation Reduction Act on the field.

Innovation in next-generation medicines for neuropsychiatric disorders continues

Emer Leahy

Interest in ketamine for mood disorders has been a game-changer for CNS drug development. “Ketamine has shown to be effective in populations where they have been resistant to all therapies to d…