Drug Discovery and Development Archives - Page 30 of 158

Harnessing the untapped potential of legacy data in pharma R&D

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Clinical trials for a new therapy cost a median of $41,117 per patient. Costs like this are no surprise to pharma leaders. But during an age of increasing budgetary pressures, drug developers are under pressure to do more with less money and staff. While there are no “simple” answers to this challenge, there is one strategy that offers research and development (R&D) teams a very powerful approach: better leveraging existing legacy data.

Pharmaceutical companies own petabytes of imaging data, generated by in-house research, investigator-initiated studies or clinical trials. This data is valuable and can yield insights that can help researchers better understand disease mechanisms and inform therapeutic approaches. But in many cases, researchers cannot access this important data, as it remains in silos with CROs, investigator labs, or within a specific research group…

Vericel’s unique biopharma focus: Tackling unmet needs in sports medicine and burn treatment

Vericel (Nasdaq:VCEL), a biopharma focused on sports medicine and severe burn care therapies, has a portfolio including the cartilage repair treatment MACI and the burn treatments Epicel and NexoBrid.

Focal cartilage defects, which occur in approximately 60% of arthroscopies, can lead to debilitating osteoarthritis if left untreated. Vericel’s flagship product, MACI, tackles this medical challenge head-on by enabling younger, active patients to avoid knee replacements. Harnessing the power of the patient’s own cells to repair cartilage damage, MACI halts the progression to osteoarthritis.

In the realm of burn care, Vericel’s Epicel serves as a permanent skin replacement for patients with severe burns, while NexoBrid is an enzymatic treatment designed to remove dead tissue from burn wounds. In a recent interview, Vericel CEO Nick Colangelo highlighted the company’s unique position in the market, noting the limited competition and signific…

Pfizer and BioNTech secure FDA nod for omicron-adapted COVID-19 booster for kids under 5

COVID-19 vaccine developers Pfizer (NYSE:PFE) and its partner BioNTech (Nasdaq:BNTX) have won emergency use authorization (EUA) for a single booster dose of their omicron BA.4/BA.5-adapted bivalent COVID-19 booster for kids between 6 months and 4 years of age.

The booster can be administered at least two months after completing the primary vaccination series with three doses of the original Pfizer-BioNTech COVID-19 vaccine. Additionally, FDA has authorized the bivalent vaccine for use as the third dose in a primary series for the same age group. The agency, however, has not yet authorized a fourth booster dose.

Trial data Supports bivalent vaccine safety and efficacy

The EUA is based on data from substudies within the companies’ Phase 1/2/3 trial (NCT05543616) evaluating the safety, tolerability and immunogenicity of a fourth bivalent vaccine dose in 300 children aged 6 months to 4 years. A subset of 60 study participants within the same age group demonst…

Meet Suzette DiMascio: Executive vice president At FFF Enterprises and advocate for women in pharma

Suzette DiMascio is a prominent figure in the specialty pharmacy industry with nearly three decades of experience and a relentless commitment to improving patient access to care. Currently serving as Executive Vice President at the biopharmaceutical and plasma product supplier FFF Enterprises (Temecula, California), she also sits on several boards, including ICON International, Zensights, and OncoSpark.

Throughout her illustrious career, she has won numerous awards, including the prestigious Dorothy Brothers Scholarship Award from the Women’s Business Enterprise National Council. While CEO of the company she founded, Suzette received the Commonwealth’s Top Women-Led Business for three consecutive years.

Beyond her professional achievements, DiMascio is an advocate for diversity and equity in the workforce and serves as a mentor to many women across the globe through her volunteer work with HBA’s Global Ambassador Program.

In the followi…

Prominent cell and gene therapy vendors in 2023

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In recent years, the cell and gene therapy market has grown at a rapid clip. The momentum is likely to continue. According to Vision Research Reports, the global cell and gene therapy market size could hit around $42.56 billion by 2030, growing at a CAGR of 39% from 2022 to 2030.

The burgeoning demand in the industry, along with an increasing number of FDA-approved therapies, is also fueling the growth of cell and gene therapy vendors offering tools and services for product development and manufacturing.

The following table includes notable vendors and suppliers active in the call and gene therapy marketplace. This curated selection of companies includes a brief overview of their focus areas.

We have a separate feature focused on companies with prominent cell and gene therapy development efforts.

Company Name Description Notable partnerships Founded He…

Emory and Pfizer partner to develop new COVID-19 treatments

Emory University has partnered with Pfizer (NYSE:PFE) to create new drugs and therapies aimed at stopping serious disease from COVID-19. The Emory and Pfizer collaboration will take place in Emory University’s Schinazi Laboratory, led by scientist Raymond Schinazi, with the goal of producing novel antiviral compounds to combat COVID, according to The Atlanta Journal-Constitution.

Although the threat of COVID has waned over the past year, the virus remains a leading cause of death. CDC data shows 1,862 COVID-related deaths in the U.S.

Pfizer, the developer of Paxlovid and collaborator with BioNTech (Nasdaq:BNTX) on the Comirnaty COVID-19 vaccine, is arguably the most visible COVID drug developer.

For Pfizer, this partnership represents an opportunity to maintain its leadership position in the COVID-19 drug market and potentially increase revenue. The company, however, expects its revenue to slide by as much as one-third this year as a result of ebbi…

Prominent pharma M&A deals to watch in Q1 2023: Pfizer, Sanofi and others on the hunt

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After a relatively quiet 2022, 2023 is anticipated to be a more robust year for pharma M&A activity. In recent months, players such as Pfizer (NYSE:PFE), Sanofi (NYSE:SNY), Chiesi, AstraZeneca (LON:AZN) and Amgen (Nasdaq:AMGN) have unveiled acquisition plans that could reshape the landscape of the sector. This article delves into the details of prominent recent M&A deals and explores their potential impact on the future of the pharma sector.

Pfizer plans to acquire Seagen

New York City–based Pfizer has recently been on a shopping spree, scooping up Trillium Therapeutics, Biohaven, Global Blood Therapeutics, and Arena Pharmaceuticals. Now, the company has unveiled its plans to acquire Seagen (Nasdaq:SGEN), a Seattle biotech firm focused on oncology. The proposed acquisition, valued at $43 billion, awaits regulatory approval. Pfizer plans to offer $229 per share…

Cybin updates on its psychedelic-based therapeutics at R&D Day

Psychedelic therapy company Cybin (NEO:CYBN/NYSE American:CYBN) hosted a virtual R&D Day on February 28, 2023. In the event, the company’s leadership team provided an update on its development pipeline of deuterated psychedelic-based therapeutics, including CYB003 and CYB004.

CYB003 is synthetic psilocybin analog, for the potential treatment of major depressive disorder. Cybin provided an interim readout from the ongoing Phase 1/2a study of CYB003.

Additionally, Cybin offered an update from the ongoing Phase 1 exploratory CYB004-E trial evaluating IV N,N-dimethyltryptamine (DMT). The company is also evaluating CYB004, a deuterated form of DMT that has a longer duration of action than traditional DMT that holds potential for generalized anxiety disorder.

Phase 2a study of CYB003

Cybin’s ongoing Phase 1/2a clinical trial of CYB003 demonstrated positive interim findings, including rapid and short-acting psychedelic responses in participants.

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PathAI launches AISight digital pathology platform and NSCLC algorithm

PathAI has launched a new digital pathology platform, AISight. Supporting AI-driven research, the digital pathology platform was trained on a real-world dataset of over 5,000 samples with inputs from more than 350,000 cell and tissue-level annotations from more than 50 pathologists. The company has also released its AIM-PD-L1 NSCLC RUO algorithm, which calculates the percentage of PD-L1 positive tumor and immune cells in NSCLC samples. The AIM-PD-L1 NSCLC RUO algorithm calculates the percentage of PD-L1 positive tumor and immune cells in NSCLC samples over the entire slide image (WSI).

Validating the AIM-PD-L1 NSCLC algorithm

PathAI previously shared validation data for the AIM-PD-L1 NSCLC algorithm at the AACR Annual Meeting in 2022. The AISight Early Access Network laboratories will build on this work by testing the algorithm’s performance on real-world data. Other IHC quantitation algorithms for various types of cancer are also available to these labs. PathAI…

100 top cell and gene therapy companies to watch in 2023

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The cell and gene therapy sector is poised to deliver a wave of new therapies that could potentially cure rare and common diseases. As many as 13 new cell or gene therapies could be approved for use in the U.S., Europe, or both by the end of 2023.

While manufacturing and regulatory challenges remain, the cell and gene therapy industry continues to offer significant therapeutic potential and commercial opportunities.

This year, we have beefed up our list of leading cell and gene therapy companies, doubling the number of organizations from 50 to 100.

In selecting the companies for this list, we weighed public companies’ degree of involvement in cell and gene therapy, including drug developers, equipment vendors and CDMOs specializing in the area. For private companies, we factored in their funding levels, pipeline and degree of history for the list.

B…

This T-cell vaccine could be the future of COVID-19 protection

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The current mRNA vaccines from Moderna (Nasdaq:MRNA) and Pfizer (NYSE:PFE)/BioNTech (Nasdaq:BNTX) stimulate the production of neutralizing antibodies that bind to the spike protein of SARS-CoV-2.

But there is a novel mRNA vaccine that activates T cells to attack cells infected with the virus. Developed by researchers at MIT and other institutions, the new vaccine could sidestep the need for updated boosters based on currently circulating variants.

In a preclinical study published in Frontiers in Immunology, the T-cell vaccine successfully attacked cells infected with the virus rather than activating the part of the immune system that releases neutralizing antibodies. In addition, the researchers tested the vaccine in humanized mice and successfully generated both CD8+ (cytotoxic) and CD4+ (helper) T-cell responses.

This new MIT-T-COVID vaccine could …

QurAlis to tap $88M funding to develop ALS product candidates QRL-201 and QRL-101

QurAlis, a clinical-stage biotech focused on amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, announced that it has closed an oversubscribed $88 million Series B financing.

According to Kasper Roet, CEO and co-founder of QurAlis, the funding round recognizes the company’s scientific track record. “We are breaking down scientific barriers in our quest to bring much-needed precision therapies to patients,” Roet said in a statement.

In an interview at the JP Morgan Healthcare Conference earlier this year, Roet said he has long wanted to develop therapies for neurodegenerative diseases. AI-based advances have helped shine a light on the genetic underpinnings of ALS. “There’s about 40 different genes that have been now identified that contribute to development of the disease,” Roet said. The continued breakthroughs helped convince Roet that the disease was “starting to become a tractable problem,” he said. “I think we can actuall…