Drug Discovery and Development Archives - Page 71 of 158

Novartis reveals positive Phase 3 data for tislelizumab plus chemotherapy in esophageal cancer

The Swiss Big Pharma Novartis (NYSE:NVS) announced that the anti-PD-1 immune checkpoint inhibitor tislelizumab with chemotherapy improved overall survival in patients with advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) in a Phase 3 study.

The RATIONALE 306 trial of tislelizumab and chemotherapy met its primary endpoint at interim analysis. The study pitted the combination therapy against chemotherapy as a monotherapy.

Novartis noted that the study focusing on first-line ESCC follows on the heels of FDA and EMA accepting tislelizumab in a second-line context.

The company is optimistic that the drug could treat a variety of solid tumors.

To date, tislelizumab has been the subject of more than 200 clinical studies.

Novartis licensed rights to the drug from BeiGene in February 2021 for $650 million upfront plus another $1.55 billion in potential milestone payments.

The Swiss company noted that it would collab…

Moderna seeks authorization for COVID-19 vaccine in children as young as 6 months old

While a portion of the public is now eligible to receive a second booster dose of COVID-19 vaccine, the FDA has not yet authorized a vaccine for children aged 6 months to 5 years old.

Moderna (Nasdaq:MRNA) is vying to be the first. The company announced today that it is filing a request for emergency use authorization (EUA) for its mRNA-1273 vaccine in young children. The company anticipates that it will complete the filing next week.

The request breaks young children into two groups, with the first covering the ages of 6 months to under 2 years and the second covering kids between 2 and 5 years old. The authorization request refers to a primary series consisting of two 25-μg doses of mRNA-1273.

Moderna also seeks similar authorizations for young children from other international regulatory bodies.

The company is basing the requests on positive interim data from the Phase 2/3 KidCOVE study, which met its primary endpoint.

The study analyzed…

How biologists of the future could displace some data scientists in drug development

[Photo courtesy of Pexels]

A decade ago, data scientist seemed like the sexiest job of the 21st century, to paraphrase an influential Harvard Business Review article.

In the pharmaceutical industry, data science certainly continues to have tremendous potential, but in years to come, data-savvy biologists could have as least as much of an impact on drug development as data scientists, according to David Harel, co-founder and president of Cytoreason, which has developed a computational disease model for drug developers.

“We call this the biologist of the future,” Harel said, referring to biologists with significant data science training received either in academia or on the job.

The consulting firm Gartner has espoused a similar idea, which it terms a citizen data science to refer to workers outside of statistics and analytics who create data science models based on predictive or prescrip…

AstraZeneca and Daiichi Sankyo win new breakthrough therapy designation for Enhertu

AstraZeneca/Daiichi-Sankyo

The HER2-directed antibody-drug conjugate Enertu (trastuzumab deruxtecan) has won its fifth breakthrough therapy designation from the FDA.

Enhertu was developed jointly by AstraZeneca (LON:AZN) and Daiichi Sankyo (OTCMKTS: DSNKY).

The latest designation relates to treating adults with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-negative) breast cancer.

Of the five breakthrough therapy designations for the drug, three are for breast cancer, while the other two are in lung and gastric cancers, respectively.

In particular, the designation covers adults who have received prior systemic therapy for metastatic breast cancer or who have experienced disease recurrence within six months of finishing adjuvant chemotherapy.

In addition, the designation specifies that patients with hormone receptor (HR) positive breast cancer have received or be inelig…

How Cellares aims to transform cell therapy manufacturing

The startup Cellares has a mission to enable industrial-scale cell therapy manufacturing with its Cell Shuttle, which it dubs a “factory in a box.”

The germ for the idea traces back to market research that South San Francisco, California-based Cellares co-founder and CEO Fabian Gerlinghaus focused on in a prior role. While attending industry conferences, Gerlinghaus stumbled upon traditional cell therapy manufacturing challenges and the opportunity to accelerate the process. “At these conferences, people were shouting from the rooftops, ‘We need cell therapy manufacturing technologies that are fully automated, fully closed and scalable to produce these life-saving therapeutics at a large scale for all the patients who need them,'” Gerlinghaus recounted.

Fabian Gerlinghaus

Cellares was born in April 2019 and has raised $100 million to date.

One of the chief objectiv…

Black Diamond Therapeutics slashes workforce by 30% while discontinuing development of BDTX-189

Black Diamond Therapeutics logo

Precision oncology firm Black Diamond Therapeutics (Nasdaq:BDTX) has announced a restructuring plan to free up cash to prioritize the development of BDTX-1535 and BDTX-4933.

The company plans on trimming nearly one-third of its workforce.

BDTX shares ticked up about 1% to $2.75 after announcing the news yesterday but fell 4% today to $2.64.

According to the company, the reorganization could provide sufficient cash runway to last until the third quarter of 2024.

“In order to increase our operational efficiency and execute on our mission, we have made the difficult decision to reduce our workforce by approximately 30%,” said David Epstein, Black Diamond Therapeutics CEO, in a press release.

The company is also halting development work on BDTX-189. This drug candidate would have potentially been indicated for advanced breast cancer, biliary cancer, mal…

Advances in RORγt inhibition: Selective targeting of IL-17 producing cells while maintaining proper thymocyte maturation

Retinoic acid receptor-related orphan receptor gamma t (RORγt) inhibitors are currently in development for various indications. RORγt plays a crucial role in driving T helper 17 (Th17) differentiation and interleukin 17 (IL-17) expression. In homeostasis, these cells play a vital role in the defense against bacteria and fungi. However, this cell type seems to be dysregulated in many autoimmune diseases such as psoriasis, multiple sclerosis and others. In recent years, the focus has been on psoriasis, but other autoimmune and inflammatory diseases such as chronic obstructive pulmonary disease (COPD), inflammatory bowel disease (IBD) and dry eye disease are also of interest. Finally, one study is being conducted on castration-resistant prostate cancer.

The nuclear receptor RORγt

RORγt is the shorter isoform of the retinoic acid receptor-related orphan receptor gamma (RORγ, NR1F3). It belongs to the nuclear receptor (NR) superfamily, discovered in 1998, about four years …

Gilead notches remdesivir indication to treat young children

FDA has announced that it has signed off on the use of COVID-19 therapy Veklury (remdesivir) from Gilead Sciences (Nasdaq:GILD) to include children at least 28 days old who weigh at least 3 kg (roughly 7 lb) and test positive results for COVID-19.

The indication is limited to children who are either hospitalized or have a high risk of developing severe COVID-19, including hospitalization or death.

In October 2020, remdesivir became the first FDA-approved COVID-19 treatment and is now the first drug to win approval for children under 12 years of age.

Remdesivir was formerly authorized for use in young children under emergency use authorization but was FDA-approved for COVID-19-infected individuals at least 12 years old who weigh at least 40 kg (roughly 88 lb).

“As COVID-19 can cause severe illness in children, some of whom do not currently have a vaccination option, there continues to be a need for safe and effective COVID-19 treatment options for …

FDA approves first microencapsulated benzoyl peroxide for rosacea

Image courtesy of Sol-Gel/Galderma

Sol-Gel (Nasdaq:SLGL) and Galderma have announced that it has won FDA approval for Epsolay cream, a proprietary topical formulation of benzoyl peroxide, 5%, for treating inflammatory rosacea lesions.

Rosacea is a common condition, but experts have struggled to estimate its global prevalence. A 2018 estimate in the British Journal of Dermatology concluded that the condition affects between 5% and 46% of the adult population.

Galderma and Sol-Gel report that Epsolay Cream is the only topical treatment shown to relieve bumps and blemishes associated with rosacea.

Galderma, the world’s largest independent dermatology company, will commercialize the cream in the U.S.

First prepared in 1958, benzoyl peroxide is widely used as an over-the-counter topical acne treatment.

Epsolay cream is encapsulated within patented silica shells that steadily release be…

A real-world data approach for bridging diversity disparities in clinical trials

[Image courtesy of Pixabay]

The lack of appropriate representation in clinical trials, particularly in terms of ethnicity and race, has been a long-standing issue that directly impacts health equity and treatment efficacy. In a 2020 analysis of the global participation in clinical trials, the Food and Drug Administration (FDA) highlighted the vast difference between enrolled participants and the global population. Of the more than 297,000 participants in clinical trials globally, 76% were white, 11% were Asian, and only 7% were Black. By comparison, 60% of the global population hails from Asia, 16% from Africa, 10% from Europe, 8% from Latin America, and just over 4% from the United States.1

The FDA has focused on addressing this issue, drafting new guidance in April 2022 aimed at increasing clinical trial enrollment from underrepresented racial and ethnic populations. This draft guidance, “Diversity Plans to Im…

10 leading pharma execs

Given the complexity of the pharmaceutical business, it can be challenging to gauge the performance of any chief executive in the space. That said, CEOs’ Annualized return over tenure as CEO over their term can provide some indication of performance. In addition, rating sites like Glassdoor also give an indication of CEOs’ popularity.

Here, we parsed a range of metrics, including CEOs’ approval ratings, their ability to navigate the pandemic and their firms’ stock performance over time, to identify ten leading pharma executives.

1. Pfizer CEO: Dr. Albert Bourla 2021 total compensation: $24.3 million Glassdoor approval rating: 89%

Pfizer veteran Dr. Albert Bourla has worked at the company for more than 25 years, becoming CEO in January 2019. The CEO shined during the pandemic, ramping up Pfizer’s focus on innovation while investing more than $2 billion at-risk to accelerate the development of a COVID-19 vaccine with partner B…

Biogen gives up seeking European approval for aducanumab

After winning FDA approval for the controversial Alzheimer’s drug Aduhelm (aducanumab) in June 2021, Biogen (Nasdaq:BIIB) has encountered a string of setbacks.

Most recently, the company has decided to yank its applications for market approval after the European Medicines Agency (EMA) recommended the refusal of the marketing authorization for Aduhelm in December 2021.

At that time, Biogen announced that it would fight the decision.

In April, the company notified EMA that its decision to withdraw its application was “based on interactions with the CHMP [Committee for Medicinal Products for Human Use] indicating that the data provided thus far would not be sufficient to support a positive opinion on the marketing authorization of Aduhelm (aducanumab).”

“We are thankful to the patients, caregivers and physicians that supported the re-examination process as part of the EMA review,” said Dr. Priya Singhal, head of global safety and regulatory sciences …