MedNess: bite-size biopharma and medtech news

Breakthrough Therapy Designation Application Submitted to FDA for Leronlimab as a Treatment for mTNBC
Nader Pourhassan, Ph.D., CytoDyn’s President and Chief Executive Officer, commented, “In our view, these findings provide strong support for our decision to submit an application for Breakthrough Therapy designation for leronlimab as a treatment for mTNBC. Patients with this horrific disease currently have few, if any, treatment options, and we hope that leronlimab can provide a therapeutic choice for substantially improved clinically relevant endpoints, quality of life, tolerability and safety over currently available SOC. We hope to receive a response from the FDA before the end of the year. We are grateful to have already received Fast Track designation for leronlimab for this indication (mTNBC) and congratulate Dr. Nitya Ray and his team for preparing the data analysis needed to file this BTD application so quickly.”

New, single-arm Ph 3 pivotal study of UGN-102 for the treatment of low-grade, intermediate-risk NMIBC planned
“We have worked diligently with the FDA over the past several years to define the unmet need in low-grade NMIBC, with a particular focus on the intermediate risk population that typically experiences multiple recurrences,” said Liz Barrett, President and Chief Executive Officer of UroGen. “We are preparing to initiate a single-arm pivotal study of UGN-102 to form the basis for a New Drug Application for UGN-102 in the treatment of low-grade, intermediate-risk NMIBC. We believe this new study increases the probability of regulatory success for UGN-102 given its streamlined design in addition to the encouraging results observed from our Phase 2 OPTIMA II study.”

Genomic analysis of circulating DNA leads to personalized medicine
A new study led by researchers in Indiana University is focusing on developing personalized therapy for patients with triple-negative breast cancer based on genomic data. Currently in phase 2 trial, PERSEVERE aims to stratify cancer patients based on risk of relapse, after they have gone through a primary round of treatments, e.g., surgery and chemotherapy and achieved remission. Studies show that two thirds of those patients may still harbor cancer in them which may cause relapse. According to the study leader Bryan P. Schneider, MD, “Recurrence and death rates are still too high, and novel strategies to improve that are markedly needed. We feel PERSEVERE is an innovative trial to try to help meet those needs.” The study takes advantage of past observations that suggest that patients who test positive for circulating tumor DNA (ctDNA) after surgery are at higher risk of relapse. Using ctDNA as markers, the study then stratifies patients in groups where ctDNA positive populations will receive targeted or standard therapy, whereas ctDNA negative population may opt out for the treatment while remaining within the trial for observation. According to Schneider, “In this trial, we’ll capitalize on that powerful ability to better focus our attention on novel therapies for those patients at extraordinarily high risk who have ctDNA positivity. Equally importantly, it will provide the opportunity for us to focus on those who do not test positive for ctDNA, a group that may be expected to do quite well in terms of de-escalating therapy and improving quality of life.” The study, which comprises of 200 patients at this point, will follow up with participants for up to five years to check for side effects as well as cancer recurrence.

Notable Launches Therapeutic Pipeline by Acquiring Worldwide Development and Commercialization Rights to Volasertib
“Volasertib represents an exciting milestone towards fulfilling Notable’s mission to deliver life-changing therapies precisely to those who will benefit, and leads the way in a new era of predictive precision medicine,” said Thomas Bock, M.D., Chief Executive Officer of Notable. “We targeted and licensed volasertib because of its compelling performance on our Predictive Precision Medicine Platform and as an important addition to our growing clinical pipeline. We plan to fast-track volasertib’s development with phase 2/3 clinical trials in AML and other cancers, selectively enrolling patients who are predicted to respond.”

Genentech teams up with Novome on a multi-target IBD collaboration focusing on seaweed-controlled oral cell therapies
Novome, a clinical stage Biotech company, has entered into a multi-year research collaboration with Genentech to discover and develop bacterial strains for targeted therapeutic delivery for inflammatory bowel disease treatment (IBD). The company will receive $15 million upfront to initiate IND-enabling preclinical studies and can get up to $590 million payments in the future depending on prespecified development achievement and commercial milestones. According to the CEO of Novome, Blake Lively, “Our unique platform for colonizing the gut with engineered microbes capable of delivering novel and validated cargo has great potential to help people suffering from IBD. It is our hope that, with access to the deep research expertise of Genentech’s scientific teams, we can advance this exciting science more rapidly and deliver a truly needed therapy to people living with IBD.”