Jul 17, 2023
  • DTx Pharma is a preclinical stage biotechnology company that focuses on developing siRNA therapies for neuroscience indications, leveraging its proprietary fatty acid ligand-conjugated oligonucleotide (FALCON) platform
  • Deal includes DTx-1252, a potential therapy for the neuromuscular disorder Charcot-Marie-Tooth disease type 1A (CMT1A), and two additional preclinical programs for other neuroscience indications
  • Acquisition expands Novartis capabilities in RNA-based therapeutics, adding DTx’s FALCON platform to the Novartis siRNA toolkit

Basel, July 17, 2023 — Novartis announced today that it has acquired DTx Pharma, a San-Diego based, preclinical stage biotechnology company focused on leveraging its proprietary FALCON platform to develop siRNA therapies for neuroscience indications.

DTx’s lead program, DTx-1252 targets the root cause of CMT1A—the overexpression of PMP22, a protein that causes the myelin sheath that supports and insulates nerves in the peripheral nervous system to function abnormally. DTx-1252, which was recently granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA),1 decreases the expression of this protein in Schwann cells, the target cell type for the development, maintenance, and function of peripheral nerves. The FALCON platform facilitates this targeted approach by conjugating siRNAs to naturally occurring fatty acids to improve the biodistribution and cellular uptake to tissues and cell types of interest.2

“We look forward to continuing the development of DTx’s therapeutic programs and bringing new hope to patients with neuromuscular and other neurological disorders for which there have historically been few treatment options,” said Fiona Marshall, President of the Novartis Institutes for BioMedical Research (NIBR). “We are also excited to bring DTx’s FALCON technology to Novartis and explore its potential to deliver drugs to extrahepatic tissues.”

Charcot-Marie Tooth disease (CMT) is a group of inherited disorders affecting the nervous system. CMT1A is the most prevalent subtype of CMT, impacting up to approximately 150,000 patients in the U.S. and Europe.3 A slow-progressing, degenerative disease of the peripheral nervous system, the disease causes progressive muscle wasting, neuropathic pain, and difficulty walking, and can have a significant impact on quality of life. DTx-1252 could potentially be a first-in-class treatment for the disease, as there are currently no approved treatments to address the underlying genetic cause of CMT1A.4

In addition to DTx-1252, the deal also brings two additional preclinical programs for other neuroscience indications to Novartis and expands the Novartis siRNA toolkit, building on its capabilities in xRNA, one of the Novartis technology platforms. The agreement underscores the Novartis commitment to Neuroscience, one of the company’s five core therapeutic areas, and to pursuing innovative therapies for patients with neuromuscular disorders.

“The growing arsenal of therapeutic platforms available to us continues to enable the development of novel, high-value medicines—particularly in cases where the underlying biology of disease is well defined,” said Robert Baloh, Global Head of Neuroscience for NIBR. “We look forward to continuing the development of DTx’s potential first-in-class medicine for CMT1A and are hopeful we can bring a therapeutic option to patients living with this debilitating neuromuscular condition.”

Under the terms of the agreement, Novartis will make an upfront payment of USD 500m and additional payments upon completion of pre-specified milestones.

This media update contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for any investigational or approved products resulting from the DTx-1252 program or the FALCON platform, the acquisition of DTx Pharma, or regarding potential future revenues from DTx-1252. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that any investigational or approved products resulting from the DTx-1252 program or the FALCON platform will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Neither can there be any guarantee expected benefits or synergies from this transaction will be achieved in the expected timeframe, or at all, nor can there be any guarantee that the DTx-1252 program will be commercially successful in the future. In particular, our expectations regarding DTx-1252 or the transaction described in this media update could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this media update as of this date and does not undertake any obligation to update any forward-looking statements contained in this media update as a result of new information, future events or otherwise.

About Novartis
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  1. U.S. Food & Drug Administration. "Orphan Drug Designations and Approvals: Double-stranded small-interfering ribonucleic acid (siRNA) comprised of an antisense strand complementary to a targeted sequence within human PMP22 messenger RNA and a nucleotide sense strand linked to a fatty acid motif." Available at: https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=940323. Accessed July 7, 2023.
  2. DTx Pharma. Available at: https://dtxpharma.com/platform/. Accessed April 19, 2023.
  3. Data on file.
  4. “Charcot-Marie-Tooth disease type 1A.” NIH Genetic and Rare Diseases (GARD) Information Center. Available at: https://rarediseases.info.nih.gov/diseases/1245/charcot-marie-tooth-disease-type-1a. Accessed April 19, 2023.

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