How Lilly Oncology is aiming to improve clinical trial diversity

Minorities tend to participate in clinical trials at far lower rates than their real-world demographics and prevalence of the disease, according to the American Society of Clinical Oncology. In addition, minorities often have worse outcomes for certain cancers than the broader public. For instance, the mortality rate for Black women with breast cancer is 40% higher than that of white women.

The pandemic, however, has highlighted persistent health disparities while offering potential strategies for improvement, according to Amy Davis, senior director of clinical development at Lilly Oncology.

The pandemic has popularized the use of decentralized trials. For instance, Lilly debuted decentralized capabilities in a recent breast cancer trial and will continue doing so for upcoming trials. “We are building in decentralized capabilities from the get-go — decreasing the number of in-person visits by half,” Davis said. The company is using remote data capture and offer…

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What is Human AB Serum?

By Trevor Smith, MS MBA, Product Manager, Immune Cells

Human AB Serum is a staple in the biological research field, providing nutrients, vitamins and necessary growth factors in cellular culture and reliable controls in in vitro diagnostics. To truly understand serum, it is important to learn about its different production and processing methods.

Starting material for Human AB Serum is collected from healthy male donors with an AB blood type. A single gendered pool is used to reduce variation in the final product. The AB blood type means that donors have both A and B antigens on their red blood cells and therefore lack blood group antibodies. This guarantees that the serum will not react with other blood types, further minimizing immunoreactivity for researchers. Typical lots of Human AB Serum are produced from 150-250 donors and pooled together, but custom batches with fewer donors are available. BioIVT can produce Human AB Serum lots of less than 10 i…

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An inside look at Insmed

Many large drug developers employ individual groups or teams independently working on discrete steps in the drug development process.

Rare disease biopharma Insmed (NSDQ:INSM) differentiates itself through its focus on patients with rare diseases. To that end, the company works to ensure collaboration between various departments to consider questions related to the applicability of a given molecule and the potential impact it could have on patients.

The company has won FDA approval for Arikayce (amikacin liposome inhalation suspension), a novel treatment of Mycobacterium avium complex (MAC) lung disease in conjunction with an antibacterial drug regimen. 

Dr. Martina Flammer

“Many companies speak about patient focus and patient-centricity,” acknowledged Dr. Martina Flammer, the company’s chief medical officer. But often, that focus can be intermittent or can begin midway through development.

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Amylyx Pharmaceuticals preps NDA filing for ALS drug AMX0035  

Cambridge, Massachusetts–based Amylyx Pharmaceuticals intends to submit a New Drug Application (NDA) to FDA for AMX0035 (sodium phenylbutyrate and taurursodiol) to treat amyotrophic lateral sclerosis (ALS), which is also known as Lou Gehrig’s disease. 

The company decided to file after a series of meetings with the FDA, including a pre-NDA meeting on July 15.  

AMX0035 was the subject of the Phase 2 CENTAUR trial, which involved 137 participants with ALS. In that trial, the drug met its primary efficacy endpoint of slowing ALS based on the ALS Functional Rating Scale-Revised (ALSFRS-R). 

A forthcoming Phase 3 trial will evaluate the safety and efficacy of the drug over 48 weeks. 

Before reversing course, FDA had asked Amylyx to submit positive Phase 3 trial results before filing a New Drug Application.

To date, there are limited treatment options available for ALS. A 2020 paper in Neuropharmacology. 

Concluded that the two drugs with…

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A closer look at Urovant’s vibegron blood pressure study

In recent years, regulators including FDA have increased their focus on the potential of drugs to cause blood pressure increases. To that end, the agency asked Urovant (Irvine, California) to study the impact of the beta-3 adrenergic receptor agonist vibegron on blood pressure.

The study, recently published in the Journal of Urology, found no significant change in blood pressure among either vibegron or placebo recipients. There were 96 patients in the vibegron group. Another 101 received placebo. “The good news was that the changes in blood pressure were trivial — on average less than one millimeter of mercury,” said Dr. Michael A. Weber, a professor of medicine at SUNY Downstate College of Medicine in New York, who was involved in the study. 

Weber recently summarized the results at the Annual Meeting of the American Urological Association. 

FDA approved vibegron in late 2020. In addition, the agency approved another beta-3 adrenergic receptor agonist…

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Hookipa Pharma announces oncology partnership with Merck

Immunotherapy-specialist Hookipa Pharma (NSDQ:HOOK) has announced a clinical collaboration and supply agreement with Merck (NYSE:MRK). Hookipa will test its immunotherapeutic HB-200 in conjunction with Merck’s blockbuster anti-PD-1 therapy, Keytruda (pembrolizumab), as first-line treatment for patients with advanced head and neck squamous cell carcinoma (HNSCC).

Last year, Merck raked in $14.5 billion in sales revenue from Keytruda.

“There remains considerable unmet treatment need for people with metastatic head and neck cancers, and we believe the combination of HB-200 and Keytruda may offer hope,” said Joern Aldag, Hookipa CEO, in a statement.

Hookipa reports that the company has seen early success with combining Keytruda and HB-200 in heavily pre-treated patients.

The company plans on launching a Phase 2 trialing the combination of HB-200 with Keytruda in 2022.

The company is developing a range of novel arenaviral immunotherapies for onc…

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Theravance cuts 75% of staff after ampreloxetine failure

Theravance Biopharma (NSDQ:TBPH) has announced today that it is laying off 75%, or roughly 270, of its employees.

The South San Francisco, California–based company employed 359 people in 2020, according to its most recent annual report.

The company plans on making the bulk of the cuts in November, with more to follow in early 2022.

Theravance also revealed that a Phase 3 study of the orepinephrine reuptake inhibitor ampreloxetine did not meet its primary endpoint of improving treating neurogenic orthostatic hypotension (OH). Specifically, the trial focused on a symptom assessment based on the Orthostatic Hypotension Questionnaire, which evaluates symptoms such as dizziness, weakness, fatigue and difficulty concentrating.

“These are not the results we had hoped to achieve, especially given the clear unmet need for patients suffering from symptomatic nOH and the positive top-line four-week results from the Phase 2 study announced in 2018. We will co…

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Urovant shares positive data at the annual meeting of the American Urological Association

Urovant Sciences (Irvine, California) is upbeat about the prospects of beta-3 adrenergic receptor agonist Gemtesa (vibegron) in treating overactive bladder (OAB). This week, the drug was featured in two presentations at the virtual American Urological Association (AUA) Annual Meeting.

Historically, the primary treatment for OAB involved a type of anticholinergics known as muscarinic receptor antagonists. 

In 2012, Astellas Pharma (TYO: 4503) won FDA approval for Myrbetriq (mirabegron), the first beta-3 adrenergic receptor agonist for OAB to hit the market. More recently, Urovant, a subsidiary of Sumitovant Biopharma Ltd., won FDA approval for Gemtesa (vibegron), another beta-3 adrenergic receptor agonist, which became commercially available in April. 

Anticholinergics used for OAB can have bothersome side effects such as dry mouth and constipation, said Dr. Cornelia Haag-Molkenteller, chief medical officer at Urovant. Such side effects lead many patients…

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COVID-19 vaccine booster tensions mount between White House, CDC and FDA

Photo from Pexels

Facing the second-biggest wave of COVID-19 infections and waning popularity, President Biden has promised to launch an initiative to begin providing vaccine boosters starting September 20. 

But the plan hinges upon FDA and CDC support, and some officials at the agencies remain unconvinced that boosters are immediately necessary. 

Two departing senior FDA vaccine officials recently co-wrote an article published in The Lancet arguing that insufficient evidence exists to justify the case for boosters. “Even if boosting were eventually shown to decrease the medium-term risk of serious disease, current vaccine supplies could save more lives if used in previously unvaccinated populations than if used as boosters in vaccinated populations,” the paper argues. In addition, while the public could ultimately benefit from boosting, administering boosters before they are required could lead to an…

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QurAlis selects development candidate for ALS

Privately-held biotech QurAlis plans to begin clinical development for the first-in-class molecule QRL-201 for amyotrophic lateral sclerosis (ALS) in the second half of 2022.

QRL-201 is now the subject of IND-enabling studies.

The drug candidate targets the restoration of STATHMIN-2 (STMN2) expression in ALS. STMN2 is a protein encoded by the STMN2 gene that is involved in neural repair. ALS is often associated with decreased expression of STMN2.

In ALS patient-derived motor neuron disease models with TDP43 pathology, QRL-201 restored STMN2 loss of function.

A 2008 study in Current Opinion in Neurology found that TDP43 is involved in ALS and frontotemporal dementia (FTD) pathogenesis.

TDP43 pathology is also present in some Alzheimer’s and Parkinson’s disease patients.

“QRL-201 could potentially benefit ALS patients who have a loss of STMN2 due to TDP43 pathology, which could, in turn, slow disease progression,” explained Kasper Roet…

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Why pulmonary fibrosis deserves more attention

Idiopathic pulmonary fibrosis x-ray courtesy of Wikimedia Commons.

This September is the inaugural Pulmonary Fibrosis Awareness Month, which the Pulmonary Fibrosis Foundation and allies have launched to educate the public about the disease family. Involving scarring of the lungs, pulmonary fibrosis gradually robs the breath from patients it affects. 

Some 200,000 Americans live with idiopathic pulmonary fibrosis.  

At present, two drugs are available to treat the condition in the U.S., but pulmonary fibrosis remains an active focus area for drug developers.

Given that September is Pulmonary Fibrosis Awareness Month, the anniversary of the September 11 attacks also can serve as a reminder of the impact of interstitial lung disease. “Lots of lung disease has occurred in the first responders of the World Trade Center,” said Dr. Joyce Lee, a senior medical advisor for the Pulmonary Fibrosis Foundat…

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Sanofi completes Translate Bio acquisition

Sanofi (NSDQ:SNY) announced today that it has completed the purchase of its mRNA partner Translate Bio.

The previously announced deal, according to Sanofi, accelerates the French pharma giant’s efforts to develop transformative vaccines and therapies using mRNA technology.

The deal comes during a year in which mRNA-based Moderna (NSDQ:MRNA) and Pfizer-BioNTech (NYSE:PFE/NSDQ:BNTX) COVID-19 vaccines are generating tens of billions of dollars.

Sanofi generated $41 billion in revenue last year.

As of today, Translate Bio is now an indirect, wholly-owned subsidiary of Sanofi. All Translate Bio shares not validly tendered in the tender offer have been converted into the right to receive the same $38 per share in cash.

 

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