Novartis will partner with Voyager Therapeutics to develop gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA) based on Voyager’s adeno-associated virus (AAV) capsid discovery platform, through an up to $1.3 billion expansion of a nearly two-year-old collaboration with the pharma giant.

The companies said today that they agreed to create HD and SMA gene therapies based on Voyager’s RNA-based AAV capsid platform, which the company calls Tropism Redirection of AAV by Cell-type-specific Expression of RNA (TRACER™). That expanded activity could raise the Lexington, MA, biotech’s potential proceeds from their alliance to roughly $2 billion.

TRACER is a broadly applicable, RNA-based screening platform designed to enable rapid discovery of AAV capsids with robust penetration of the blood-brain barrier and enhanced central nervous system (CNS) tropism in multiple species, including non-human primates (NHPs). In preclinical studies, according to Voyager, TRACER generated capsids have shown widespread gene expression in the CNS compared to conventional AAV capsids as well as cell- and tissue-specific transduction, including to areas of the brain that have been traditionally difficult to reach, while de-targeting the liver and dorsal root ganglia.

Voyager has agreed to license to Novartis target-exclusive access to its TRACER capsids related to SMA, as well as oversee all development and commercialization. Voyager also agreed to license to Novartis worldwide rights to Voyager’s AAV gene therapy for HD, including Voyager’s TRACER capsids and proprietary payloads. For the HD program, however, Voyager will oversee preclinical advancement while Novartis will be responsible for all clinical development and commercialization.

In this expanded collaboration, Novartis has agreed to pay Voyager $80 million upfront, plus purchase $20 million in Voyager stock—2,145,002 newly-issued shares priced at $9.324, about 10.5% above the company’s Friday closing share price of $8.44. Novartis also agreed to pay Voyager development, regulatory, and commercialization milestone payments of:

  • Up to $200 million for the SMA program and up to $225 million for the HD program, in each case for the first corresponding product to achieve the corresponding milestone.
  • Sales milestone payments of up to $400 million for the SMA program and up to $375 million for the HD program.
  • Tiered, escalating royalties in the high single-digit to low double-digit percentages of annual net sales of the SMA program products and the HD program products.

30% stock surge

Investors signaled their approval of the deal with a stock buying surge that sent Voyager shares jumping nearly 30% in early trading, to $10.96 as of 10:15 a.m. ET. Novartis shares stayed flat, inching up nearly 1% to $101.78 as of the same time.

The companies agreed in March 2022 to develop TRACER-based gene therapies for up to three undisclosed neurological disease targets, in return for $54 million upfront and more than $1.5 billion in potential payments tied to achieving development, regulatory, and commercial milestones.

A year later in March 2023, Novartis exercised options to license novel TRACER-generated capsids for two of the undisclosed targets, in return for paying Voyager a $25 million option exercise payment. At the time, Novartis scaled back its future commitment, agreeing to paying up to $600 million in associated potential development, regulatory, and commercial milestone payments, plus mid- to high-single-digit tiered royalties based on net sales.

Also at the time, Voyager and Novartis also disclosed the potential of expanding that agreement to include capsids for up to two additional targets over the subsequent 18 months—an expansion the companies realized today.

Based on the Novartis option exercise, plus an October 2022 opt-in by Pfizer for another Voyager capsid for an undisclosed rare neurological disease target, “we believe external validation for VYGR’s novel capsid platform is stronger than ever,” Sumant Kulkarni, a managing director covering biotechnology stocks for Canaccord Genuity, commented in a March 7, 2023, research note, adding: “We are now looking for execution on internal programs to lend further support to our BUY thesis, which we believe remain firmly on track. Clinical trials and meaningful data from those are still some time away.

“But, we continue to believe that VYGR affords significant potential for investors looking for exposure to neuro-focused gene therapy platforms that are showing very intriguing, albeit early signs of success with novel capsids,” Kulkarni added.

Baird senior research analyst Jack K. Allen, CFA, was similarly positive on Voyager last March, predicting: “we expect Voyager will remain active on the deal front moving forward, and note that the advancement of these partnered programs towards clinical studies should also help to validate the TRACER platform over the course of the coming years.”

Last July, Pfizer sold its licensing rights for the Voyager capsid to Alexion, AstraZeneca Rare Disease as part of a $1 billion selloff of its early-stage rare disease gene therapy portfolio.

Several partners

Alexion and Novartis are two of several biopharma partners with which Voyager has inked collaboration agreements. Voyager has also licensed its capsids to Sangamo Therapeutics for a prion disease gene therapy program, and is partnered with Neurocrine Biosciences on seven gene therapy programs—a glucocerebrosidase 1 (GBA1)-targeting gene therapy for Parkinson’s and other GBA1-mediated diseases; an FXN-targeting gene therapy for Friedreich’s ataxia; and five gene therapy programs for undisclosed diseases.

“We look forward to broadening our work with Voyager to help bring forward novel, high-impact gene therapies with the potential to improve the lives of patients affected by severe neurologic conditions,” said Fiona Marshall, president of Biomedical Research at Novartis. “We believe Voyager’s TRACER capsids hold promise for enabling next-generation gene therapies for diseases of the central nervous system, aligning well with our deep neuroscience expertise and gene therapy leadership at Novartis.”

Voyager CEO Alfred W. Sandrock, Jr., MD, PhD, said the upfront cash Voyager is receiving from Novartis will strengthen its balance sheet and extend its runway into mid-2026.

That’s a year’s additional runway from “mid-2025,” Voyager’s last runway guidance to investors based on the $252.9 million in cash, cash equivalents, and marketable securities the company reported as of September 30, 2023, as disclosed in its most recent Form 10-Q filing, covering the third quarter of 2023.

“We are thrilled to expand our existing relationship with Novartis, a global leader in the gene therapy field,” said Alfred W. Sandrock, Jr., MD, PhD, CEO of Voyager. “Combining the proven capabilities of Novartis in gene therapy development and commercialization with Voyager’s next-generation TRACER capsids and payloads could enable the advancement of important new therapies for patients.”


To listen to Alex Philippidis talk about Novartis and Voyager and J.P. Morgan’s 2024 meeting listen to Touching Base‘s episode 3: Novartis and Voyager, JPM Ahead, Epigenetic Engineering; Alex Aravanis.

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