Houston-based CNS Pharmaceuticals is combining an unusual financing method with some interesting chemistry to expand the therapeutic choices for glioblastoma patients. The company is using crowdfunding to bridge the gap between an early friends and family round and their IPO, which is slated for summer. The ultimate goal is move their blood-brain barrier-penetrating chemotherapy, berubicin, into a phase 2 glioblastoma trial.
Glioblastoma has become famous both for the lives it’s taken – including Senator Ted Kennedy and former Vice President Joe Biden’s son Beau – and the grimness of the prognosis. According to the American Brain Tumor Association, median survival is just 14 months.
Behavioral Health, Interoperability and eConsent: Meeting the Demands of CMS Final Rule Compliance
In a webinar on April 16 at 1pm ET, Aneesh Chopra will moderate a discussion with executives from DocuSign, Velatura, and behavioral health providers on eConsent, health information exchange and compliance with the CMS Final Rule on interoperability.
“You do temozolomide and radiation to shrink the tumor, and then you do surgery, and then you radiate the rest of the tumor, giving temozolomide,” said CNS Pharma’s CMO Sandra Silberman in a phone interview. “Then you just wait for the tumor to come back, unfortunately, and most tumors do.”
Glioblastomas are hard targets because of their aggression and location – most therapies can’t get through the blood-brain barrier, which acts like a bouncer, keeping toxic molecules (including chemotherapy) out of the club.
Researchers have spent decades trying to overcome this hurdle. San Diego-based Tocagen is testing a therapy that combines an antifungal drug, which penetrates the barrier, with gene therapy that converts it into chemo.
CNS has taken a simpler approach. Company founder and MD Anderson medicinal chemist Waldemar Priebe wanted to take a powerful class of chemotherapy drugs called anthracyclines and disguise them to get past the barrier.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
“It’s a chemical change to the molecule, changing some of the ionic properties so it’s no longer recognized,” said Silberman.
Early preclinical results were promising and the phase I study showed the drug was well-tolerated and even hinted at efficacy.
“It showed incredible activity against these tumors,” said Silberman. “Not only did some patients have shrinkage of their tumors but the shrinkage lasted and one patient had a complete response.”
CNS now wants to build on these results in a phase II trial and is using the crowdfunding campaign in place of the traditional venture route. They felt this approach could engage patients and families while avoiding the dilution and other issues associated with venture capital.
“We were never under any illusions that crowdfunding was going to generate a sufficient raise to get the company where it needed to go,” said CEO John Climaco in a phone interview. “But we did recognize an opportunity to bring a wide group of people in who would diversify our shareholder base, give people who have been affected by this disease, or cancer in general, a stake in the company and help us get where we’re going.”
The crowdfunding effort has exceeded its $100,000 goal, raising nearly $150,000, with a couple of months left on the clock. The IPO is planned for $15 million.
Once cash is in-hand, CNS hopes to move rapidly into phase II. Given glioblastoma’s orphan status and low bar for efficacy, the drug has the potential to move relatively quickly through the regulatory process.
“As soon as we are public and have some funding,” said Silberman, “we can hit the ground running with this and determine if it’s effective or not.”
Photo: Abscent84, Getty Images
