Michael Hercz – Sentynl Therapeutics Inc.

While the rarest disease might afflict fewer than several hundred people a year, there are around 7,000 rare diseases. That’s more than enough, Michael Hercz laments, for one in 50 babies to be stricken by a malady for which a biopharmaceutical company has little commercial incentive to research and develop a remedy.

But some companies, including Sentynl Therapeutics Inc., concentrate on a select few diseases and make an impact for patients and their caregivers. As general counsel, senior vice president and chief compliance officer at this Southern California-based biopharma, Hercz has got his hands on just about every process outside the laboratory. That includes arranging critical partnerships, ensuring compliance in a very regulated industry, weighing in on personnel as head of HR and simply being available for whatever issue may arise. 

Michael Hercz | Senior Vice President, General Counsel, Chief Compliance Officer | Sentynl Therapeutics Inc.

“The incidence of the diseases we treat is very low, some having fewer than 100 patients worldwide,” he tells Vanguard in September. “Thankfully these diseases are very uncommon. Nonetheless, taking rare disease medications from discovery to approval is often more challenging and time-consuming than developing and commercializing widely used medications.” 

Logistical matters notwithstanding, Hercz has helped make one such medication available in the United States, Europe and Israel. It’s the intravenous infusion therapy, fosdenopterin, that is sold here under the brand name Nulibry® for treatment of a rare genetic disease known as molybdenum cofactor deficiency Type A—MoCD Type A—which might affect no more than a few hundred babies and young children worldwide annually, many of whom go undiagnosed.  

“No disease is too rare for us to want to treat,” Hercz is quick to say. “Diseases like MoCD Type A can have a high mortality rate and untreated children often won’t reach the age of 4.”   

Partnering in pediatrics  

Healthy newborns have a complex mechanism to prevent toxic sulfite accumulation in their brains, whereas babies with MoCD Type A do not. In 2021, another company, BridgeBio Pharma Inc., received FDA approval for Nulibry as a substrate replacement therapy. While the medication doesn’t cure MoCD Type A, it increases the likelihood of a child surviving toddlerhood and, hopefully, lays the groundwork for next-generation drugs.  

BridgeBio sought a partner that was focused on rare pediatric disease and Sentynl seeing a strategic fit, the brass tracked down Hercz in Iceland where he was vacationing with his family last year. He negotiated from afar the arrangements for Sentynl to acquire global rights to Nulibry as well as the responsibility for its continued development and commercialization.  More recently Hercz has been working behind the scenes on another  medication, CUTX-101, for the treatment of the rare Menkes disease.

Here, impaired copper metabolism results in abnormally low levels of copper in the liver and brain of mostly male infants who, without early intervention, rarely survive their third birthday. 

As is the case with Nulibry, CUTX-101 isn’t a cure-all, but Hercz says clinical data suggest that it can extend a child’s life. Though CUTX-101 is currently being presented to the FDA for review in a “rolling submission,” Hercz says the process has encountered delays outside of Sentynl’s control.   

Medications for spasticity and Lennox-Gastaut Syndrome—two more rare diseases affecting children—are in earlier stages of the regulatory pipeline, and Hercz reminds of the lengthy process that awaits any drug, particularly one for the rarest of conditions.  

Transition to rare 

Hercz has seen Sentynl transform since joining in 2015, all the while leading its legal and compliance functions. Sentynl was acquired in 2017 by the India-headquartered generics company Zydus Group, which rebranded as Zydus Lifesciences Ltd.  

Hercz played a pivotal role in the transaction, with Zydus keen on acquiring the company and retaining its entrepreneurial spirit. Zydus has been a phenomenal parent, he says, explaining how it respects Sentynl’s autonomy and experience in building portfolios. To wit, Zydus supports Sentynl’s focus on rare disease and ensures its operations remain robust, funding new acquisitions for its growing subsidiary.   

He sees Sentynl’s legal and compliance team growing alongside the company. For now, the team is “small, yet nimble and outsized relative to peers” with full-time compliance director and senior compliance counsel, Zarah Meyer, having joined in January 2022.  

Hercz can’t stress enough the need to adhere to rules and regulations everywhere. “Sentynl springs into action when prescribers anywhere in the world ask for access to its medications, evaluating if and how we can make them available to patients who might benefit from them,” he says. So sensitive are these drugs that they must be transported frozen and stored in a mini freezer.  

He’d also like there to be more testing for rare diseases despite practical constraints. Each state screens babies for around 30 diseases, but thousands of rare ones can go undetected. Hercz sees hope in genetic testing to screen for many more diseases and helped forge an agreement with Rady Children’s Institute for Genomic Medicine to collaborate on a project that uses rapid whole genome sequencing to identify genetic conditions in newborns before they get sick.  For now, it’s just a small pilot program, and thus a sobering reminder of Sentynl’s mission to ensure “no patient is overlooked.”  

In Hercz, there’s a measure of both optimism and realism. A University of Pennsylvania psychology major prior to USC Gould School of Law, his interest in healthcare law was spurred by an externship with a judge who oversaw a mental health court. He practiced healthcare law with a boutique firm, Hooper, Lundy & Bookman, from 1997 to 1999 and then with global heavyweight Akin, Gump, Strauss, Hauer & Feld for the next three years.   

The multinational biopharma Amgen Inc. recruited him in 2002 for an eight-year stretch and afterward came shorter stints with Victory Pharma and Deloitte and finally to Sentynl, which he calls “my most meaningful role.”  

He’s proud his wife has one too, she being Judge Nicole Heeseman of Los Angeles County Superior Court. The couple met in law school and have a 12-year-old son, Alex, and several pets. The family enjoys traveling, and Hercz seems the most adventurous, he being an advanced scuba diver and motorcycle enthusiast.   

Hercz is probably exaggerating when he says how he and his judge and lawyer buddies assemble to ride Hells Angels style. At any rate, Sentynl has been cutting into his playtime.  

“The last two years have been the most invigorating,” he says. “It brings out my passion—this role to play in ensuring medications are available for devastating neonatal and pediatric diseases. I’m more invested than I was at the firms. I’m part of everything whereas from the outside, you just see a slice.”  

View this feature in the Vanguard Fall II 2023 Edition here.