rare disease day 2022

Rafael Pharmaceuticals reports progress with rare cancer drug

By Jenni Spinner

- Last updated on GMT

(luismmolina/iStock via Getty Images Plus)
(luismmolina/iStock via Getty Images Plus)

Related tags Rare disease Drug development Oncology Cancer Phase II phase I Clinical trials COVID-19

The company (which specializes in developing therapies for rare cancers) currently is working on a number of candidates, including its lead drug CPI-613.

The pursuit of drugs aimed toward the treatment of rare cancers involves a unique set of challenges. Outsourcing-Pharma recently spoke with Sanjeev Luther, president and CEO of Rafael Pharmaceuticals (which specializes in the development of treatments for rare cancers) about its lead drug CPI-613 (devimistat) and other projects in process, as well as advice on dealing with challenges in the path.

OSP: Could you please tell us a bit about the work that Rafael Pharmaceuticals does, why it’s so important, and what sets your company apart?

SL: At Rafael Pharmaceuticals we’re on a mission to develop therapies for rare cancers that have few to no treatment options available. With science stemming from the world’s most renowned institutions and compassion that runs deep, Rafael strives to improve the lives of people with rare cancers, inaugurating a new era of hope.

We currently have Phase II trials for biliary tract cancer, Burkitt’s lymphoma, and Phase I/II for clear cell sarcoma. Patients and loved ones affected by these devastating, rare, and hard-to-treat cancers have little to no viable treatment options— a phenomenon we are committed to changing.

OSP: Please tell us about CPI-613 (devimistat) and the progress you’ve made in treating it, and why targeting the cancer it’s intended for is important.

SL: Our lead compound, CPI-613 is designed to target the mitochondria of cancer cells in order to disrupt their energy production, cutting off the fuel for disease growth. Devimistat is designed to selectively target cancer cell mitochondria to treat some of the most intractable forms of cancer.

Because it is designed to be highly specific, minimally toxic, and effective against a wide variety of cancers, devimistat can be used for recurrent and hard-to-treat cancers, including on a chronic basis; it may also enhance the sensitivity of cancer cells to other treatment modalities, including chemotherapy.

More than 800 patients to date have received devimistat or completed clinical trials on multiple solid tumors and hematological malignancies. Devimistat has demonstrated positive efficacy signals in several clinical trials including Burkitt’s lymphoma, biliary tract cancer, and clear cell sarcoma.

OSP: What are some of the obstacles you’ve faced in your research and trialing of this treatment to date?

Sanjeev Luther, president and CEO, Rafael Pharmaceuticals

SL: Typical with the nature of research and trialing, especially in the rare disease space, there have been moments where we’ve faced disappointment and uncertainty. However, the very reason this field is so difficult is what drives our passion, as it’s quite literally a matter of life and death for our patients.

At the end of the day, there is nothing more rewarding than knowing we can help save a life and provide hope to a community with such a high unmet need in quality care.

OSP: What are some of the issues your patient-participants have faced in trial participation to this point?

SL: Although we’ve seen a number of patients demonstrate very good signals of efficacy with excellent response rates and survival statistics in several tumor types, there’s a great deal that remains beyond our control.

As every patient is unique, responsiveness to a drug can differ vastly among patients with the same diagnosis depending on a variety of factors. However, the main problem we face in trial participation is simply getting patients enrolled in the first place. Patient awareness is critical, and educating eligible patients, clinicians, and caretakers of the available treatment options at hand is our primary goal. 

The site selection strategy in clinical trials is of utmost importance. Selecting the right location is critical to ensure healthy trial enrollment and retention.

In addition to the PIs and their respective institutions, it’s essential that the trial sites are equipped to properly administer the medications, compile and report the data, and ultimately execute trial operations in general. I make an effort to visit most sites myself and corroborate and create relationships with the PIs to make sure we are supporting their specific trial sites to the best of our ability.

Especially as we work with rare cancers, we must ensure that there is a patient population for the specific indication in the sites’ regions. When treating very sick patients, the more barriers we can eliminate in participating in the trials, the more likely patients are to respond positively to the treatment.

OSP: Specifically, how has the COVID-19 pandemic impacted your work in investigating this and other treatments?

SL: Despite the fact the COVID-19 pandemic has impacted every facet of healthcare, we’ve remained committed to our patients with multiple trials continuing to run. Since our patients must visit hospitals and clinics in person, the high peaks of the pandemic were very difficult as our patients are already very sick and at a high risk of developing more severe reactions to COVID-19. This, coupled with the healthcare worker shortages and the general public’s increasing mistrust in the healthcare system, has made enrollment and retention more difficult. In response, we increased outreach and communication to ensure our patients feel safe and supported during these unprecedented times.

OSP: Please talk about some of the trial recruitment and strategies you’ve implemented, and shifts you’ve made in recent months/years to deal with the pandemic and other obstacles.

SL: With deep roots in healthcare, we have leveraged our network to connect with leaders in rare cancers from around the world. In the most recent years, we have focused on opportunities in the US, initiating new trials, and improving and extending the lives of patients.

We pride ourselves on being a relationship-first organization. Finding and hiring PIs who are not only willing but who share our passion for catering towards patients with little to no efficacious treatment methods for their specific diagnosis, is of utmost importance to me.

Maintaining a consistent stream of communication between the medical staff at our selected trials increases the caliber of care and ultimately the level of patient success. At the end of the day, we are a company grounded in our patients, and everything we do must reflect back to how we can improve their quality of life.  

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