Tom Equels earned a chest full of ribbons, including two Distinguished Flying Crosses and a Purple Heart, flying Cobra Gunships in more than 300 combat missions in the Vietnam war. Today, he finds himself in the pilot's seat in the battle against cancer as CEO of Hemispherx Biopharma Inc (NYSEAMERICAN:HEB).
At the core of Hemispherx’s business is its flagship drug Ampligen (rintatolimod), recently approved in Argentina as the world’s only approved therapeutic for the treatment of Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS). The drug, which is an experimental therapeutic in the US, is currently in clinical trials for the treatment of multiple cancers.
CEO steers new course
When Equels took over as CEO in March 2016, the Ocala, Florida-based company had almost no money and no supplies of clinical-grade Ampligen. Its facility for the second drug in its pipeline, FDA-approved Alferon, was shut down after major flood damage at the New Jersey manufacturing facility.
Equels immediately got to work engineering a turnaround. He is now steering a course for the company, which involves not only developing Ampligen as an ME/CFS treatment but also a series of clinical trials at major cancer research centers. These trials are designed to evaluate whether the combination of Ampligen with checkpoint blockade therapies — powerful immunotherapy drugs that block proteins that restrain the body’s immune system from fighting cancer — will improve clinical tumor responses, time to progression, and survival rates.
“We believe Ampligen is an agent that holds tremendous promise — not only for pancreatic cancer, but also for a variety of other cancers,” said Dr Michael Hollingsworth, head of pancreatic cancer research at the University of Nebraska Medical Center’s Buffett Cancer Center. Hollingsworth conducted the preclinical pancreatic cancer proof-of-concept studies and is preparing one of the clinical trials.
Hopeful Progress of Ampligen in cancer trials
The company is supporting the clinical trial evaluation of Ampligen’s use in fighting eight solid tumor types: urothelial, renal cell, triple-negative breast, colorectal, ovarian, prostate, and pancreatic carcinomas as well as melanosarcoma (melanoma).
“The big cancer institutions like Roswell Park in Buffalo, the Buffett Cancer Center in Nebraska, the University of Pittsburgh and Erasmus in the Netherlands all share our excitement about what Ampligen can do in oncology,” points out Equels. “They share it to the point that they are undertaking this work based on grant funding.”
A key advantage is that Ampligen shows considerable promise priming the environment for tumor eradication by converting so-called “cold” tumors, which are more difficult to treat, into “hot” tumors, which have a better chance of being treated by both the immune system and checkpoint blockade therapies.
In more scientific terms, the findings of ovarian carcinoma tissue explant studies and a clinical trial in colorectal cancer have provided fact-based evidence that Ampligen is able to reprogram the tumor microenvironment by inducing the anti-tumor beneficial aspects of inflammation by attracting killer T cells into the tumor microenvironment without amplifying immune suppressive elements such as regulatory T cells.
“Ampligen on a standalone basis has demonstrated effects in a number of solid tumors,” says Equels. “If we’re able to establish in clinical trials that Ampligen has a place as the primer for tumors being treated by checkpoint blockade therapies — creating a synergy for enhanced effectiveness — then you’re looking at an important advancement in human cancer treatment as well as a potential disruptive pharmaceutical business opportunity.”
Backers on Wall Street
Hemispherx is not only generating considerable attention in the scientific community — it’s attracting supporters on Wall Street.
In a note to investors sent last October, Edward Woo, an analyst with Ascendiant Capital Markets, kicked off coverage of the stock. While Hemispherx operates in a “highly competitive environment,” such concerns are outweighed by Ampligen’s growth prospects and valuation.
“Though we acknowledge that Hemispherx’s oncology drugs still have long development roads left, we believe the approximately billion-dollar market potentials present a high reward for the risks,” Woo wrote.
Like many biopharma R&D companies of its size, Hemispherx is looking for a partner with adequate capital and expertise, or a buyout. “We don’t intend to try to invent ourselves into a major pharma company,” says Equels. “It’s our intention to deliver an immuno-oncology combination therapy product so that a big pharma company can take the ball and run with it to tremendous financial success.”
While the Food and Drug Administration has already granted Ampligen orphan drug status and authorized a compassionate care program using Ampligen for ME/CFS, the drug’s new drug application for ME/CFS is still pending approval in the United States.
“The FDA has asked us to do a number of things, but primarily a confirmatory Phase 3 clinical trial on top of the existing Phase 3 trial,” explains Equels.
Second drug in focus
While it catches the most attention, Ampligen is not the only drug in Hemispherx’s pipeline.
The company’s second product is Alferon N Injection, its injectable form of natural alpha interferon, which is already FDA-approved for commercial sales in the US for the treatment of genital warts and is also approved by the National Administration of Drugs, Foods and Medical Devices (ANMAT) in Argentina for commercial sales for the treatment of genital warts and in patients refractory to treatment with recombinant interferons. The company’s New Brunswick, New Jersey plant is set up to manufacture alpha interferon from start to finish out of a 600-liter bioreactor.
“The engineering is done. The installation is done. All of the equipment is there,” Equels reports. While the Hemispherx facility in New Brunswick is set up and approved by the FDA under the Biologic License Application (BLA) for Alferon, this status will need to be reaffirmed by an FDA pre-approval inspection which will not occur until Hemispherx is prepared to re-start the validation and manufacturing process, submit a supplement to the BLA, and produce new batches of commercial filled and finished product.
Having raised more than $8.5 million in the six months ended March 31, 2018, from a rights offering and issuance of convertible debt, according to a regulatory filing, Equels is eager to continue to woo investors.
Equels’ leadership experience has come in handy in righting the course at Hemispherx. And the future now looks promising for Ampligen as further headway on the clinical trial front is made and money continues to be raised to fund development.
“We are knocking out milestone after milestone,” Equels said. “There is no other NYSE American company out there in our range that has these kinds of clinical activities going on in oncology.”
— Ellen Kelleher contributed to this article —
Contact Uttara Choudhury at [email protected]