Voyager Therapeutics, Inc. (NASDAQ:VYGR) Q4 2023 Earnings Call Transcript February 28, 2024
Voyager Therapeutics, Inc. beats earnings expectations. Reported EPS is $1.25, expectations were $-0.59. Voyager Therapeutics, Inc. isn’t one of the 30 most popular stocks among hedge funds at the end of the third quarter (see the details here).
Operator: Good day. Thank you for standing by. Welcome to the Q4 2023 Voyager Therapeutics Earnings Conference Call. At this time, all participants are in a listen-only mode. [Operator Instructions]. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Peter Pfreundschuh, Chief Financial Officer. Please go ahead.
Peter Pfreundschuh: Thank you. And good afternoon. Joining me on the call today is Dr. Al Sandrock, our CEO, and Dr. Todd Carter, our Chief Scientific Officer. We issued our Q4 and year-end 2023 financial results press release this afternoon. The press release and 10-K are available on our website. In a moment, I will turn the call over to Al. Before I do this, I want to remind everyone that, during this call, Voyager representatives may make forward-looking statements as noted in slide 2 of today's deck. These forward-looking statements include future expectations, plans and prospects. All forward-looking statements are inherently uncertain and are subject to risks and uncertainties that may cause actual results to differ materially from those indicated by these forward-looking statements.
You are encouraged to review and understand the various material risks and uncertainties facing the company as described in the company's most recent annual report on Form 10-K filed with the SEC this afternoon. All SEC filings are available on the company's website. Now, it is my pleasure to turn the call over to Al.
Alfred Sandrock: Thank you, Pete. And good afternoon, everyone. Please turn to slide 3. I'd like to start by defining Voyager's position as an emerging leader in neurogenetic medicine. First, our pipeline. We anticipate having at least four wholly owned and partnered CNS programs in the clinic by the end of 2025, with the potential to generate clinical data in 2025 and 2026. Our most advanced programs are an anti-tau antibody for Alzheimer's disease and our SOD1 gene therapy program for amyotrophic lateral sclerosis, or ALS. I will talk more about both programs in a few minutes. Second, our platform. Voyager is working to solve the delivery challenges inherent to CNS gene therapies with our TRACER capsid discovery platform. We have demonstrated high transduction in multiple brain areas at relatively low doses with detargeting of the liver and dorsal root ganglia across multiple species.
We have also shown blood brain barrier penetrance across multiple animal species, and we have identified a receptor that is expressed in humans. Third, partnerships. In January of 2024, we received $100 million from Novartis in a combination of upfront payment and equity investment to develop gene therapies for Huntington's disease and spinal muscular atrophy. This brings our total of partnered programs to 13, with the potential to generate $8.2 billion in longer term milestone payments. Whereas this is "biobucks" number, it is not factored into our cash runway guidance. I will note that some of it is becoming real. Earlier this week, we triggered a $5 million milestone payment upon selection of a lead development candidate for our Neurocrine partnered Friedreich's ataxia program.
All this has given us a strong balance sheet, which we expect to provide runway into 2027, removing our financial overhang and enabling us to potentially generate value creating clinical data in 2025 and 2026. Finally, potential. We have already demonstrated our strength as a leader in CNS capsid technology. We now aim to expand from gene therapy and antibodies into other modalities of neurogenetic medicine, potentially broadening our impact. We continue to explore the potential to leverage Receptor X to shuttle non-viral genetic medicines across the blood brain barrier and look forward to sharing data on this in the future. On slide 4, I want to take a moment to acknowledge just how much Voyager has achieved recently. Following the Novartis collaboration, we closed a $100 million public offering.
We closed 2023 with approximately $231 million in cash. When you add the $100 million from Novartis and the $100 million from the offering, that brings us to a pro forma cash number of approximately $431 million as of December 31, 2023. We're also progressing our GLP toxicology work with our anti-tau antibody, VY-TAU01, for Alzheimer's disease and remain on track for an IND filing in the first half of this year. We achieved two development candidate selections with gene therapy programs. One, our wholly owned SOD1 ALS gene therapy and one with our Neurocrine partnered Friedreich's ataxia program. We also generated data with our wholly-owned tau silencing gene therapy program, showing robust reductions in human tau mRNA and protein in a mouse model, which Todd will share more on later.
A researcher handling a vial of adeno-associated virus gene therapy for a preclinical program.
All of these milestones are helping us build a robust pipeline, as you can see on slide 5. I do want to note that the wholly owned programs at the top of the slide denoted in orange are the only programs we fund. The rest of our pipeline is funded by our partners. While I won't go into detail on all of these today, I do want to dig into some of our wholly owned programs, particularly our anti-tau antibody, our tau knockdown gene therapy, and our SOD1 ALS gene therapy. Moving to slide 6, when I look at the Alzheimer's space, I'm encouraged by the progress, particularly the approval of two anti-amyloid antibodies. I view tau as the next exciting target in this field. Why? We've long known that the spread of pathological tau correlates to the progression of Alzheimer's disease.
In fact, Alzheimer's disease progression is characterized by Braak staging, which is based on the spread of pathological tau. Our anti-tau antibody, VY-TAU01, is differentiated from other approaches based on the epitope of targets, which is located in the C terminal rather than the N terminal or mid domain, and which has been shown to inhibit the spread of pathological tau by more than 70% preclinical study. We are currently progressing through IND-enabling studies and remain on track to file an IND in the first half of this year. We plan to initiate a single ascending dose study this year in healthy volunteers, and we plan to initiate a multiple ascending dose study next year in patients with early stages of Alzheimer's disease. We hope to generate proof of concept data for slowing the spread of pathological tau via PET imaging in 2026.
I'll now turn it over to Todd to talk about another approach we are developing to target tau.
Todd Carter : Thank you, Al. Please turn to slide 7. In addition to our anti-tau antibody approach, we are also working on a tau silencing gene therapy. This approach leverages a TRACER derived BBB-penetrant AAV capsid to deliver vectorized tau targeted siRNA. As shown, the single intravenous administration of our tau-silencing gene therapy in humanized tau mouse model caused robust reductions in human tau mRNA and protein. This was observed across multiple brain regions. And in some regions, we have seen up to a 90% reduction in tau mRNA. This work will be presented at the 2024 AD/PD conference. Given these promising data, Voyager has prioritized this program and advanced it into a research. And we anticipate an IND filing in 2026.
I'll just echo what Al said here. We see tau as an incredibly important target for Alzheimer's disease, so much so that we're pursuing multiple modalities, both an antibody and a gene therapy. We are encouraged by the early data for both of these programs and look forward to continuing to share at scientific conferences. In addition, I want to share a little data from our SOD1 ALS gene therapy program. We announced the selection of a development candidate for this program in December of 2023 and some of the data behind this decision are shown on slide 8. As you can see, a single IV dose of our development candidate in non-human primates resulted in 73% and 82% reductions in SOD1 mRNA in cervical and lumbar spinal cord motor neurons, respectively.
These are quite significant reductions in some of the key cell types affected by the disease. Additionally, we think an added benefit is that the effect is not limited to the spinal cord. When you look at approaches of injecting a medicine intrathecally, you often see steep gradients of effects dropping off once you move away from the injection site. But because our IV approach leverages the vascular system to deliver across the blood brain barrier, we see not only strong knockdown along the spinal cord, but also lowering of the brainstem and motor cortex, which we believe will be important in addressing ALS. This program continues to progress toward an IND filing in mid-2025. Now I'll turn the call back to Al.
Alfred Sandrock : Thank you, Todd. Turning to slide 9, you can see Voyager has started the year off strong, and we continue to execute on our milestones. We began the year with our second Novartis collaboration, this time to advance gene therapies for Huntington's disease and spinal muscular atrophy. We then raised $100 million through a public offering in January of 2024, providing runway into 2027. Just this week, we announced the selection of a development candidate for our Neurocrine partnered Friedreich's ataxia gene therapy program, which triggered a $5 million milestone. I want to close by acknowledging all of the hard work of the Voyager team, which has allowed us to progress as much as we have. Looking forward, Voyager is well capitalized to advance at least four wholly owned and partnered CNS programs into the clinic this year and next.
And with our financial overhang removed, we look forward to potentially generating value creating clinical data in 2025 and 2026. With that, we're happy to take any questions you may have. Operator?
