Drug Discovery and Development Archives - Page 32 of 158

Canadian company to explore psychedelic as a traumatic brain injury treatment

Algernon Pharmaceuticals is one of the few companies aiming to develop the classic psychedelic N,N-Dimethyltryptamine (DMT). Most recently, the company’s Algernon NeuroScience announced its plans to explore DMT as a treatment for traumatic brain injury (TBI). The company plans on launching a Phase 2 study in the fourth quarter of 2023 to test its potential in TBI. There are currently no drugs approved for the condition.

Preclinical studies have shown that DMT has promise for treating neurological disorders, including stroke and traumatic brain injury.

Early this year, Algernon launched a Phase 1 study of DMT in the Netherlands to find a safe and well-tolerated sub-psychedelic IV drug dose.

A Phase 1 study is underway

Algernon announced that investigators had completed dosing the first participant group in the Phase 1 study. They found no safety or tolerability problems. Additionally, the safety review committee overseeing the investigation has give…

Exploring the future of mRNA therapy: Where are we now?

Small interfering RNA bound to a messenger RNA. [Image courtesy of Adobe Stock]

Interest in mRNA therapy has exploded in recent years following the development of mRNA-based COVID-19 vaccines, but can the platform live up to the initial hype that surrounded it? More companies have announced clinical trial data from mRNA trials for conditions ranging from cardiovascular disease to cancer.

mRNA, or messenger RNA, is a type of genetic material involved in protein synthesis. The therapeutic use of mRNA has an array of applications, but a handful of factors are dampening enthusiasm in the platform, including the warning immunity of mRNA COVID-19 vaccines. Additionally, mRNA-based flu vaccine candidates have yet to prove their superiority over traditional vaccines in the clinic.

In addition, mRNA therapies are currently more expensive to manufacture than traditional small-molecule drugs. They…

Phase 3 study shows zavegepant can relieve migraine pain in 15 minutes

[Zavegepant image from PubChem]

The Lancet has published Phase 3 study results for zavegepant, an investigational nasal spray for the acute treatment of migraine. The study found that a single 10 mg intranasal dose of zavegepant was more effective than placebo for pain relief and freedom from the most bothersome symptom at two hours post-dose.

Zavegepant is a small molecule calcitonin gene-related peptide (CGRP) receptor antagonist. CGRP is a protein involved in transmitting pain signals in the brain.

Zavegepant’s nasal formulation could be particularly valuable for migraine patients who experience nausea and difficulty swallowing.

Zavegepant is now a Pfizer asset

Pfizer (NYSE:PFE) acquired the asset through last year’s purchase of Biohaven Pharmaceutical Holding Company (NYSE:BHVN), which had commercialized the dual-acting migraine therapy Nurtec ODT (rimegepant).

In the recent …

Ranking of most overpaid CEOs includes several pharma executives

[Image courtesy of Adobe Stock]

Determining the most overpaid CEOs is a controversial topic, but one notable list that has recently surfaced is the one published by the non-profit As You Sow. This list includes several pharmaceutical and life science executives on its 100 most overpaid CEOs ranking.

To determine its most recent ranking, As You Sow assessed compensation packages that were subject to voting in the year before June 30, 2022.

As You Sow identifies its ranking of 100 most overpaid CEOs through three rankings based on the S&P 500. The first assesses the financial performance of each company by comparing their CEO’s pay with their total shareholder return. The second dimension identifies companies with the highest percentage of institutional shareholders who voted against their CEO’s pay package. The third dimension gauges the ratio of CEO’s pay to that of an average employ…

San Francisco mayor hopes to woo biotech to fill office vacancies

London Breed [Image courtesy of Wikimedia Commons]

San Francisco Mayor London Breed has suggested that vacant downtown office spaces should be made available for biotech companies to expand, according to the San Francisco Business Times. The biotech industry could help breathe new life into the area, Breed argued.

She highlighted a 5% life sciences vacancy rate in the city in contrast to the 25% office vacancy rate. For context, the vacancy rate for the office market was about 5% before the pandemic.

Breen recently announced a 75,000-square-foot expansion of the Gladstone Institutes’ facility in Mission Bay. The organization is a non-profit biomedical research institution.

She proposed removing code barriers to help developers convert office space into biotech labs, saying that the city wants to make it easier so that it happens more quickly. Breed said that biotech is the future of San Franc…

Overcoming the challenges of metabolite identification and profiling for developing oligonucleotides

[Image courtesy of WuXi AppTec]

The use of oligonucleotides (i.e., “oligos”) was first proposed in the late 1970s, but has not achieved widespread popularity until recently. Developments in delivery method and target specificity for oligos have led to oligos’ resurgence in popularity. These therapies are particularly of interest because their mechanism of action allows for targeting virtually any nucleic acid sequence including previously “undruggable” disease targets. Therefore, the development of oligo drugs can help to address unmet medical needs.

Ten oligo-based drugs have been approved since 2018, with 16 approved drugs in total. Additionally, more than 100 phase II or phase III clinical trials are currently underway. However, many unique challenges remain for oligo drugs. The novelty, complexity and evolving regulatory guidance of these therapies can pose greater technical challenges than conve…

Genentech says new Oceanside biologics plant will be one of the world’s most modern

New Oceanside facility rendering. [Image courtesy of Genentech]

Roche (SWX:ROG) division Genentech recently announced that it would build a new biologics manufacturing facility in Oceanside, California. Optimizing for both speed and modularity, the facility will use the latest digital technologies and in-house process improvements to maximize production volumes. Genentech is also designing the facility with sustainability in mind. The plant will reduce energy use, generate renewable electricity and eliminate plastic waste sent to landfills.

The announcement of the new plant follows last year’s opening of a new clinical supply center at its South San Francisco birthplace.

In a recent interview, we spoke with Nazeli Dertsakian, general manager of Genentech’s Oceanside facility, about the technology and sustainability features of the new biologics manufacturing facility. Dertsakian shares insight…

Vir Biotechnology parts from GSK in its development of next-gen COVID-19 therapies

Vir Biotechnology (Nasdaq: VIR) has amended its research collaboration with GSK to reflect its ongoing efforts to develop next-generation COVID-19 therapies. The former company said it would continue developing COVID-19 therapies alone or in cooperation with other partners.

Vir and GSK will continue collaborating to ensure ongoing access to the monoclonal antibody sotrovimab, where it remains authorized.

Under the revised terms of the agreement, Vir retains exclusive rights to pursue the development of new therapies resulting from the joint coronavirus vaccine and antibody initiatives, with a tiered royalty payment ranging from low to mid-single digits to GSK.

The companies will continue working on sotrovimab (VIR-7831) and VIR-7832, a monoclonal antibody with a dual mechanism of action aimed at the spike glycoprotein of the SARS-CoV-2 virus.

Today, VIR shares held steady, ticking up 0.11% to $26.18. GSK shares were up about 1% to $36.23.

FDA …

Frequency Therapeutics shares plummet after FX-322 Phase 2b failure

Frequency Therapeutics (Nasdaq:FREQ) shares were down more than 80% on Monday afternoon after the company announced the failure of the Phase 2b study for FX-322, its lead candidate drug for hearing loss. As a result, the Lexington, Massachusetts-based biotech company will discontinue its hearing loss program and cut its workforce by 55%.

It says its cost savings measures will extend its runway into 2025.

In late 2022, Otonomy, another neurotology biopharma once listed on Nasdaq, said it had decided to dissolve its business and liquidate its assets.

FX-322 had no statistically significant difference over placebo

Frequency Therapeutics said the Phase 2b study showed that the drug FX-322 did not improve speech perception more than placebo. The company will also discontinue its second program for sudden or noise-induced sensorineural hearing loss, FX-345.

FX-322 did not meet its primary endpoint or any of the study’s secondary endpoints. The saf…

Breaking through the status quo: Moving beyond traditional safety signal detection

[Image courtesy of Adobe Stock]

Signal detection is an essential pharmacovigilance (PV) activity designed to support patient safety by actively uncovering potential safety risks in medical drugs and vaccines. For post-market products, the status quo for signal detection involves monitoring individual case safety reports (ICSR) and literature from multiple sources.

Since the turn of the century, organizations have relied on ICSR data for signal detection. Whether used to report product problems, adverse events or consumer complaints, ICSRs provide information integral to medicinal products’ safety.

When the system flags a disproportionate number of adverse events (AEs) for a product, highly trained safety scientists or risk physicians on the PV signal team analyze underlying cases and other available evidence. They use this information to determine a possible causal relationship between the AEs and related…

Japan scraps 140 million dose order of Novavax COVID vaccine, Takeda reveals

In 2021, Takeda (TYO:4502) announced that it would manufacture 150 million doses of COVID-19 vaccine from NovaVax (Nasdaq:NVAX). But the company has disclosed that the Japanese government has canceled the remaining order for the Nuvaxovid vaccine.

The Japanese government acquired 8.24 million vaccine doses but said it would cancel the remaining order for the remaining 141.76 million doses owing to low demand.

Takeda and Novavax are collaborating on developing a new vaccine optimized for future variants in 2023.

Novavax currently has an omicron vaccine candidate in a Phase 3 trial.

The company has data demonstrating that its adjuvanted NVX-CoV2373 vaccine prompts a robust immune response when used as a booster and is effective against forward drift variants of SARS-CoV-2.

Novavax plans to provide a monovalent or bivalent vaccine for the 2023–2024 vaccination season based on FDA feedback. The FDA’s Vaccines and Related Biological Produc…

FDA approves Jemperli to treat dMMR-related endometrial cancer

FDA has approved Jemperli from GSK (NYSE:GSK) for adult patients with mismatch repair-deficient or advanced endometrial carcinoma (dMMR).

GSK predicts Jemperli will generate peak sales of $1.4 billion to $2.8 million.

The drug is indicated for adults with dMMR-related or advanced endometrial carcinoma who have progressed after a previous platinum-containing regimen. This drug is not suitable for those who need curative surgery or radiation.

Approved based on a Phase 1 study

The FDA approved the drug based on data from GARNET Phase 1, in which it had a 45.4% overall response rate. The single-arm, multicenter, open-label study of Jemperli monotherapy showed that 85.9% of patients experienced a response within 12 months. 54.7% had a response lasting at least 24 months.

Data from the ongoing GARNET trial’s A1 cohort supported Jemperli’s approval. This trial evaluated Jemperli as a monotherapy in patients with advanced solid tumors. The dru…