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Novartis Nabs DTx in $1B Acquisition Deal

PHARMA: FDA Granted Lead Candidate Orphan Drug Status in June

DTx Pharma CEO Artie Suckow has brought his company a long way since he and two co-founders started it in 2017 with $200 and support from San Diego’s unique network of research institutions, venture groups, accelerators and a collaborative environment perfectly suited for innovative biotech companies.

Artie Suckow, Ph.D.
Co-founder & CEO
DTx Pharma

“Through those networks I was able to get some traction,” he said of DTx’s beginnings, adding “this was the only place I could start a company that could potentially have this kind of exit.”

That kind of exit is DTx’s recent acquisition by pharmaceutical giant Novartis in a deal worth a potential $1 billion, which was announced July 17.

The Novartis deal – a $500 million up-front payment, with a potential $500 million more for meeting certain milestones – is commercial validation of DTx’s unique FALCON platform (Fatty Acid Ligand Conjugated OligoNucleotides) technology which utilizes fatty acids attached to small interfering RNA therapeutics (siRNAs) – “the interfering RNA that binds to the message and leads to the chopping up of the message,” Suckow said.

In June, the technology was given regulatory validation when the FDA granted orphan drug status to DTx’s lead candidate DTx-1252, a treatment for Charcot-Marie-Tooth Disease 1A (CMT1A). In addition to the CMT1A program, Novartis has acquired full rights to the FALCON platform and two other early-stage programs in neuromuscular and central nervous system (CNS) indications.

“As the initial founder of the technology and initial IP, I couldn’t be more excited to have Novartis deploy their chemistry resources to continue to understand how one might leverage fatty acids to further enable this class of therapeutics,” Suckow said.

A Mutual Understanding

“One of the key aspects of the discussion with Novartis that drove the deal was how impressed we were with their understanding of the disease, the burden of the disease, the physicians and patients that are suffering from and treating the disease,” said DTx Pharma Chief Business Officer Peter Condon.

Peter Condon
Chief Business Officer
DTx Pharma

DTx was especially impressed with Global Head of Neuroscience for the Novartis Institutes for BioMedical Research Robert Baloh, who “founded a Southern California CMT research clinic as part of Cedar Sinai back in the day,” Suckow said. “He was as passionate as we were about treating the disease and I think that differentiated Novartis from of the other players that were around the table.”

Robert Baloh, M.D., Ph.D.
Global Head, Neuroscience
Novartis Institutes for BioMedical Research

Baloh said the DTx acquisition “underscores the Novartis commitment to bringing life-changing medicines forward for patients with neuromuscular diseases and other disorders of the nervous system. We are excited to drive forward these promising preclinical programs and explore the potential of the FALCON platform.”

Closer to a Treatment

Currently, there are no approved therapeutics addressing the underlying genetic cause of CMT1A, a disease with an estimated 150,000 patients in the U.S.  Europe. The DTx-1252 program has the potential to be the first.

“We had strong preclinical data on a model of disease showing that we can not just halt progression but actually reverse the disease,” Condon said, adding that as the program moved forward through IND enabling studies, “pharma became really impressed with how the technology was evolving and maturing. Novartis saw enough of what they wanted to see and decided to put forward an offer that was very compelling to our investors.”

The deal is not only compelling to investors, but also to the CMT Research Foundation – an early supporter of DTx that funded a project to validate the DTx FALCON platform’s relevance in overcoming the challenge of delivering drugs to Schwann cells.

Cleary Simpson
CMT Research Foundation

“The success of that work earned  DTx  a grant from the NIH and supported their raising more than $115 million based on the development of DTx-1252,” said CMT Research Foundation CEO Cleary Simpson.

Cleary described the acquisition as “a seminal moment in Charcot-Marie-Tooth disease drug development.”

“Novartis brings substantial resources and drug development experience to help accelerate the process of bringing DTx-1252 through clinical trials and into the marketplace to benefit an estimated 150,000 patients living with CMT1A in the U.S. and  Europe,” she added.

Katherine Forsey, Ph.D.
Chief Research Officer
Charcot-Marie-Tooth Association

The acquisition was also welcomed by the Charcot-Marie-Tooth Association (CMTA).

According to CMTA Chief Research Officer Katherine Forsey, the acquisition highlights the progress made in positioning CMT as an attractive proposition to pharma, the founding principle of the CMTA’s Strategy to Accelerate Research (STAR). The CMTA has been increasing its collaborative work with DTx Pharma team members as they prepare for the commencement of first-in-human clinical trials of DTx-1252, Forsey said.

“We are excited by the prospect of continuing this work with the Novartis team and applaud their commitment to bringing life-changing medicines forward for patients with neuromuscular diseases like CMT,” she added.

DTx Pharma

Founded: 2017
CEO: Artie Suckow, Ph.D
Headquarters: San Diego
Business: Biotech addressing challenges associated with delivering RNA-based therapeutics
Employees: 50
Website: dtxpharma.com
Notable: DTx’s FALCON platform conjugates combinations of naturally occurring fatty acids to siRNAs to improve their cellular uptake and biodistribution.


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