BioBuzz
By Steve Surdez

Leadiant Biosciences, Inc. formerly known as Sigma-Tau Pharmaceuticals, recently received FDA approval for Revcovi ™, which provides improved treatment for patients living with ADA-SCID, better known as “Bubble Boy Disease.” The journey that lead to Revcovi is a prime example of Leadiant’s steadfast dedication to their patients and their rare disease portfolio.

Revcovi will replace their legacy product, AdagenⓇ, which was approved in 1990, as the primary enzyme replacement therapy (ERT) available in the U.S. to ADA-SCID patients. However, the need for a new treatment quickly became crucial when Leadiant’s manufacturing partner notified the company that it had to discontinue future production.

Adagen is an animal-based enzyme therapy produced through a cumbersome and unreliable bovine source material. In fact, 5.5 metric tons or 12,000 pounds of material are required to yield 80 grams of Adagen. Production challenges were compounded by more stringent FDA regulations, essentially rendering Adagen obsolete due to cost inefficiencies.

Instead of dropping ADA-SCID from its product portfolio, Leadiant doubled-down, investing in a new way forward for the patients.

Leadiant developed Revcovi using recombinant technology, which eliminates the need to source Revcovi from animals. This new approach increases efficiencies, creates more reliable yields and lengthens product shelf life. Revcovi is administered by intramuscular injection and is a PEGylated recombinant adenosine deaminase (rADA) enzyme.

For more than three decades, iterations of the company now known as Leadiant has displayed an unwavering commitment to treating and helping ADA-SCID patients and their families. ADA-SCID–or adenosine deaminase deficiency-severe combined immunodeficiency–is an ultra-rare, inherited autosomal metabolic disorder that severely compromises the immune system and can be fatal if untreated. Approximately 1 in 200,000 to 1,000,000 newborns are impacted worldwide, representing about 15% of all SCID cases (Source: NIH). As an ultra-rare disease, the patient population is miniscule: Leadiant currently treats 35 ADA-SCID patients in the U.S. and seven in Canada.

Michael Minarich CEOCEO Michael Minarich, who joined Leadiant in 2015, came to the rare disease market from a Big Pharma background having worked with the likes of Pharmacia and Pfizer. He immediately noticed a marked difference between the world of Big Pharma and the rare disease space: “Your familiarity with your patients, their families and prescribers is so much greater. You’re able to more deeply understand the impacts of the disease on the patients and their families, while also getting to know the challenges faced by prescribers trying to identify and correctly treat a lesser known disease. You feel so much more a part of the whole therapeutic process and you become a conduit for communication and education on a much more personal and intimate level than in Big Pharma.”

Indeed, the Leadiant team remains on a first name basis with its very first ADA-SCID patient who has been treated with Adagen for nearly 30 years.

Headquartered in Gaithersburg, Maryland, Leadiant’s roots run deep in the treatment of rare and ultra-rare diseases. Leadiant Biosciences, Inc. is a subsidiary of Leadiant S.p.A., which traces its roots back to Sigma-Tau, a family-owned pharmaceutical company founded in Italy by Emilio and Dr. Claudio Cavazza in 1957. Sigma-Tau Pharmaceuticals was a rare disease treatment pioneer, receiving the fourth orphan drug designation ever in 1984. In 2015, as part of a company reorganization, Sigma-Tau’s North American operation was rebranded as Leadiant Biosciences, Inc. The Sigma-Tau name continues to be used in Europe and is focused on its other traditional businesses. Leadiant Biosciences has operations in North America (U.S. and Canada) and the European Union.

Today, Leadiant’s sole focus is on researching, developing and distributing novel treatments for patients afflicted with some of the rarest diseases in the world. Revcovi’s recent FDA approval for the treatment of ADA-SCID gives Leadiant eight rare disease products on the North American (U.S. and Canada) market.

The deployment of recombinant technology to produce Revcovi has been a game changer for all parties involved. For Leadiant, the production process has stabilized, improving cost effectiveness. And for patients, they get a purer and more accessible product.

“We now have a cell bank of these bacteria, used to make recombinant products, which will be available for the next 80 years. The recombinant process eliminates any chance of mad cow disease and provides consistent quality and supply and an improved shelf life,” added Minarich. “We’re administering the last batches of Adagen and we’re eager to help patients transition to Revcovi. We’re currently working on this transition with patients and prescribers through the Immune Deficiency Foundation. We’re in the process of educating and training everyone and are receiving prescriptions as we speak.”

Revcovi will join bone marrow stem cell transplants as the only other ADA-SCID treatment available in the U.S. Revcovi can be used pre and post bone marrow transplants to augment the transplant process and represents a less complex and invasive alternative to bone marrow procedures. If identified early, ADA-SCID patients have a strong prognosis and can lead fairly normal lives, though they will need to take Revcovi for life.

The company’s long-standing corporate value of “Rare Dedication” serves both as a reminder and a call-to-action for its staff. As Big Pharma has largely moved out of the ultra-rare disease space, passionate and dedicated companies like Leadiant have continued to step up to serve the rare disease patient population. Patient advocacy, education and support are hallmarks of Leadiant’s mission that go beyond the research and development of new therapies.

“One of our core values is ‘Rare Dedication’. We’re dedicated to those patients who are rare and ultra-rare. If we didn’t serve this population, I’m not sure another company would. We have a passion for whom we serve that makes us unique. We go where other companies won’t. For us, helping these patients is very satisfying. Once we commit to these patients we see it all the way through no matter what. You see the patient’s condition. These patients and families have few alternatives. You get to know these families, and you have an attachment and commitment that goes above and beyond. It’s personal,” stated Minarich.

With a number of research and development projects moving through Leadiant’s pipeline, including a recently approved treatment in Europe for ultra-rare CTX (Cerebrotendinous Xanthomatosis), the company is strongly positioned to uphold its robust legacy for alleviating the suffering of patients and families afflicted by the world’s rarest diseases.