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JP Morgan Healthcare Conference Day 3: Voyager, Precigen, Nykode, Grail, Myriad Genetics, Quanterix


NEW YORK – On the third day of the 42nd annual JP Morgan Healthcare Conference in San Francisco, highlights included an update from Voyager Therapeutics on the treatments it is developing for Alzheimer's disease and SOD1-mutated amyotrophic lateral sclerosis; Precigen's plans for its off-the-shelf and autologous CAR T-cell therapies; Nykode's efforts to move its HPV16-targeted vaccine into a registrational study; and the increasing uptake of Grail's Galleri multi-cancer early detection test amid ongoing research. 

Below are brief reports on individual presentations from the conference. A recap of news from the first and second day of the meeting is available here and here

Voyager Therapeutics 

Voyager Therapeutics expects to file at least four investigational new drug applications with the US Food and Drug Administration between this year and next, company CEO Alfred Sandrock said. 

The Lexington, Massachusetts-based firm plans to file an IND for its lead program, VY-TAU01, an anti-tau monoclonal antibody treatment for Alzheimer's disease, in the first half of this year. The company is planning to launch a single ascending-dose Phase Ia trial of healthy volunteers later in 2024 and a multiple ascending-dose Phase Ib study of Alzheimer's patients in 2025. VY-TAU01 is wholly owned by Voyager and currently in an IND enabling phase. 

Next year, Voyager plans to file an IND for its wholly owned small interfering RNA treatment for SOD1-mutated amyotrophic lateral sclerosis (ALS), which is designed to decrease expression of SOD1. The company also expects to file INDs for at least two of the gene therapy programs being codeveloped with Neurocrine Biosciences, which include candidates for Parkinson's disease, Friedreich's ataxia, and a third undisclosed disease. 

Voyager has a cash runway into 2027 to support these clinical trials, Sandrock said, not including $8.2 billion in potential long-term milestone payments from partnerships. 

That includes a public offering to raise $100 million, which Voyager unveiled just days after a capsid license and collaboration deal with Novartis that Voyager announced earlier this month, in which Novartis paid the company an upfront consideration of $100 million, including $20 million in equity, with the potential for up to $1.2 billion in various milestone payments. The $100 million from Novartis was expected to extend the runway through mid-2026. 

Financing from the public offering is needed to ensure the company can "get to some critical data readouts that we're expecting to happen in the second half of 2026," including PET imaging and other data on VY-TAU01 and six months to potentially a year of follow-up data for various gene therapy programs, Sandrock said. "We wanted to ensure runway to get to those inflection points." 


Precigen shared a series of pipeline updates and anticipated milestones for both its off-the-shelf and autologous cell therapies

The Germantown, Maryland-based firm is planning to begin evaluating its off-the-shelf adenovirus immunotherapy candidate, PRGN-2009, plus Merck's Keytruda (pembrolizumab) versus Keytruda alone in recurrent or metastatic cervical cancer patients whose tumors are PD-L1- and human papillomavirus-positive. That randomized Phase II trial is expected to begin in Q1 2024. 

In the autologous arena, Precigen is anticipating an interim data readout from its Phase Ib dose-expansion trial of PRGN-3006 in previously treated, relapsed or refractory acute myeloid leukemia during the second half of 2024. The therapy, which is one of several UltraCAR-T pipeline candidates that uses Precigen's point-of-care, semi-closed manufacturing system, is designed to express a chimeric antigen receptor that targets CD33 as well as membrane-bound interleukin-15 that allows for in vivo expansion. The treatment also incorporates a so-called safety switch, which can be activated by administering a specific agent should patients experience debilitating toxicities. The US Food and Drug Administration has granted PRGN-3006 orphan drug designation and fast-track designation for treating AML. 

By the end of 2024, Precigen CEO Helen Sabzevari said the firm also plans to report out preliminary dose-escalation data from its Phase I clinical trial of another autologous cell therapy, PRGN-3007, in advanced, ROR1-positive hematological and solid cancers. In addition to the ROR1-targeting CAR, the IL-15, and a safety switch, this treatment also includes a novel mechanism meant to block PD-1 gene expression. 

Finally, the firm said it is gearing up to bring its CD19-directed cell therapy, PRGN-3008, to the clinic with a Phase I clinical trial in B-cell malignancies. "PRGN-3008 has all the bells and whistles of our UltraCAR-T," Sabzevari said. "Although we might not be the first in the oncology market [with a CD19-targeting CAR T-cell therapy], we believe we can be the best in the market." 

In the future, the autologous CD19-targeting UltraCAR-T could also be developed as a treatment for autoimmune diseases, and indeed, Sabzevari said Precigen is pushing toward trying to launch a clinical trial of PRGN-3008 in autoimmune conditions by the end of 2024. 


Nykode aims in 2024 to advance its human papilloma virus (HPV)16-targeted vaccine VB10.16 within a potential registrational study in late-stage cervical and head and neck cancer, while also exploring its efficacy in early-stage and adjuvant settings. 

The company is conducting a Phase II trial, dubbed VB-C-04, to test the activity of VB10.16 alone and with Roche's immunotherapy Tecentriq (atezolizumab) in patients with HPV16- and PD-L1-positive recurrent or metastatic cervical cancer. Nykode has completed another Phase II trial, dubbed VB-C-02, of VB10.16-Tecentriq in the same cervical cancer setting, but that study included patients with PD-L1-positive and -negative tumors. Both trials focus on patients who have progressed on or after standard therapy. 

In final results from the completed VB-C-02 trial, PD-L1-positive patients receiving VB10.16-Tecentriq appeared to have an advantage over those who tested negative for PD-L1. The median overall survival in the entire study population of 52 patients was 16.9 months, and the median progression-free survival was 4.1 months. In the PD-L1-positive subset, median overall survival and progression-free survival was 25 months and 6.3 months, respectively. The overall response rate in the PD-L1-positive group was 29 percent versus 19 percent in the overall trial population, and the disease control rate was 75 percent versus 60 percent, respectively. 

Nykode CEO Michael Engsig said the company is encouraged by these results, particularly when compared to data from other trials in which patients receive just immunotherapy. In particular, the data from the VB-C-02 trial looks favorable when considered against the InnovaTV 301 trial of Genmab and Seagen's antibody-drug conjugate Tivdak (tisotumab), which Nykode views as VB10.16's primary competitor. 

In InnovaTV 301, researchers compared Tivdak to chemotherapy in patients with recurrent or metastatic cervical cancer who had progressed during or after standard treatments including Genentech's anti-angiogenic treatment Avastin (bevacizumab) and an anti-PD-L1 agent. Patients who received Tivdak had a median overall survival of 11.5 months, a median progression-free survival of 4.2 months, and an overall response rate of 18 percent. 

"We see a near-doubling of the numbers that you expect to see with a checkpoint inhibitor monotherapy," Engsig said. "Compared to Tivdak, we also see a very strong competitive profile, which makes us very comfortable as we move forward." 

Now, in the new, potentially registrational VB-C-04 trial, Nykode hopes to confirm the benefits seen in VB-C-02 and accelerate the product to market as quickly as possible. Engsig said Nykode expects to finalize enrollment in VB-C-04 in 2024 and report interim data by mid-2025. 

The Oslo, Norway-based firm in November also began a Phase I/IIa trial of VB10.16 as a first-line therapy in patients with HPV16-positive, PD-L1-positive recurrent or metastatic head and neck cancer. The company plans to do another trial, dubbed VB-C-05, of VB10.16 in locally advanced HPV16-positive cervical cancer in the adjuvant setting, which Engsig said "represents an even larger commercial opportunity" than recurrent and metastatic cervical cancer. 


Grail CEO Bob Ragusa said the company generated full-year 2023 revenues of $93 million, up 68 percent year over year and in line with revenue guidance provided by Illumina, which plans to divest the firm by the end of the second quarter of this year. Grail notched $30 million in revenue in Q4 2023. 

Ragusa said that to date, the company has performed 150,000 of its Galleri multi-cancer early detection (MCED) tests in the commercial setting and counts more than 9,000 ordering providers. He added that Grail has performed roughly 350,000 Galleri tests across both the commercial and research settings. 

Ragusa highlighted several near-term growth drivers for the company, including plans by the UK's National Health Service this year to review early data from the NHS Galleri trial. If the results are favorable, the NHS "may initiate a pilot of up to 1 million tests over a two-year period starting in 2024," he said, noting that this would mark "the first national [health] system adoption," which could help drive adoption in other single-payor systems. 

He said that the NHS Galleri study is on track to complete the final round of subject visits in Q3 2024 and anticipates having final results in 2026. 

Ragusa said that Grail has enrolled 25,000 participants in its Pathfinder 2 study, which he noted will be the primary study supporting its US Food and Drug Administration premarket approval submission for Galleri. 

He also highlighted the recently announced REACH/Galleri-Medicare study, in which the company plans to compare 50,000 Medicare beneficiaries receiving usual care plus an annual Galleri test to a similarly sized synthetic cohort. 

Ragusa also pointed to Grail's activities beyond MCED, noting that he sees opportunities for its methylation-based technology in areas including minimal residual disease detection, patient stratification, and monitoring of patient response to treatment. 

He did not comment on Illumina's planned divestment of the company except to note that Illumina has publicly announced the divestment and to say that Grail has operated as a standalone company since Illumina announced its acquisition of the firm three years ago. 

Myriad Genetics 

Myriad Genetics has completed its turnaround after years of struggle, but "the work is never done," said CEO Paul Diaz. After describing the growth of each of its product lines in 2023 and laying out its product roadmap for the following year, Diaz said he is "quite confident that the market's going to see … the opportunity here that we have to grow and expand multiple and create shareholder and patient value." 

Diaz also provided preliminary financial results during his presentation. Preliminary Q4 revenue is expected to be between $196 million and $197 million, slightly above analysts' consensus estimate of $194.4 million and putting Myriad at the high end of its fiscal year 2023 guidance of between $747 million and $753 million. Adjusted earnings per share is expected to be between $.02 and $.03 for Q4, beating analysts' consensus estimate of $.01. 

Diaz said he sees a large market opportunity across all of its products and expects to gain more market share as smaller players struggle with the infrastructure investments needed in the space, such as integrating with electronic medical records and dealing with reimbursement and regulatory challenges. 

In pharmacogenomics, although payor coverage for its GeneSight test has been "slow going in some regards," it has been an "underappreciated growth engine for the company, and we see a lot of opportunity to improve payor coverage and improve [average selling price]," Diaz said. 

Its oncology franchise is poised for additional growth as it prepares for the Precise Liquid launch in Q3 2024, a liquid-based version of its Precise tumor profiling test. 

A commercial launch of the firm's minimal residual disease monitoring test, Precise MRD, is expected in the second half of 2025. The test is currently undergoing studies with researchers at MD Anderson Cancer Center and Memorial Sloan Kettering Cancer Center in renal cancer and breast cancer, respectively. The whole-genome sequencing-based assay is built on Myriad's proprietary technology, and Diaz said the firm is "quite confident in our freedom to operate" regarding intellectual property. The MRD space has seen a spate of litigation in recent years, including a recent preliminary injunction against NeoGenomics' MRD test and permanent injunctions against Invitae and ArcherDx in litigation brought by Natera. 

"The IP related to this and the chemistry and the proprietary workflows on this whole-genome assay are differentiated from some of the other assays that are in controversy in the marketplace," he said. 

The company is also working on expanding its MyChoice CDx test for ovarian cancer to other indications, such as breast and prostate cancer, Diaz said. 

In its women's health business, Diaz said the Prequel prenatal screening test and the Foresight carrier screening test "see a lot of runway," and the firm expects strong growth this quarter in the prenatal business. The company plans to launch in Q1 2024 the Foresight Universal Plus assay, an expanded carrier screening panel of 274 genes. 

The FirstGene multiple prenatal screening test, meantime, is expected to have a soft launch in Q3 2024, with a full commercial launch the following quarter. The test is an integrated assay for noninvasive prenatal screening, carrier screening, fetal recessive status, and fetal-maternal blood compatibility. 


Quanterix CEO Masoud Toloue said that with the company's corporate reorganization and assay redevelopment program complete, it is now in a position to accelerate its assay development and expects to produce on the order of tens of new assays in 2024 and beyond. 

Citing the impact of the reorganization plan, he noted that Quanterix has reduced its cash burn by threefold since 2022 and delivered double-digit revenue growth for full-year 2023. The firm also expects to report fourth quarter revenue of $30 million, up 16 percent year over year and above analysts' consensus estimate of $28.6 million. 

Neurology remains central to the company's plans, with Toloue saying Quanterix believes the space is poised to grow over the next decade in much the way areas like oncology and immunology have in recent years. 

He pointed to the large number of central nervous system (CNS) drug programs ongoing, noting that there are currently more than 120 CNS programs and that Quanterix is involved in a substantial percentage of these programs. 

Alzheimer's disease is at the center of Quanterix's work within neurology. In October, the company launched its LucentAD p-Tau 217 lab-developed test for assessing brain amyloid pathology in patients suspected of having Alzheimer's disease. 

Toloue said Quanterix is currently running a prospective clinical trial for the assay with the goal of submitting it to the US Food and Drug Administration for regulatory approval. He said readout from that trial is expected in 2024. 

Quanterix is also working to develop the LucentAD p-Tau 217 assay for use with dried blood spot samples, which Toloue said could help improve accessibility to the test globally. 

Toloue said that in addition to assay development, the company is also investing in the development of a next-generation platform with a focus on combining single-molecule sensitivity with higher levels of multiplexing and throughput. Key to that process will be reducing the size of the instrument in order to meet what Toloue said is the company's goal of having Simoa, the firm's digital biomarker platform, not just in specialty labs but in all laboratories. 

He said the company expects to provide more details on this instrument development work in 2024.