On January 5, 2024 Illumina Inc. (NASDAQ: ILMN), a global leader in DNA sequencing and array-based technologies, reported that it has signed an agreement with Janssen Research & Development, LLC (Janssen) (Press release, Illumina, JAN 5, 2024, View Source [SID1234639026]). This collaboration will be the first relating to the development of Illumina’s novel molecular residual disease (MRD) assay, a whole-genome sequencing (WGS) multi-cancer research solution that detects circulating tumor DNA (ctDNA) to better understand the persistence or recurrence of disease following clinical intervention.

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In oncology, MRD testing is increasingly being used as a prognostic indicator of disease recurrence after treatment by helping clinicians assess the effectiveness of a patient’s current course of clinical intervention and guide their decisions about precision therapy. MRD testing for solid tumors shows promise for improving the standard of care where current disease-monitoring tools fall short in accurately identifying patients’ response to treatment.

"This collaboration reflects the value of our unique MRD technology and the promise of Illumina’s whole-genome approach in oncology," said Joydeep Goswami, chief strategy and corporate development officer and chief financial officer of Illumina. "Working together with pharma partners like Janssen, we aim to deliver a sensitive, accurate, and easily accessible whole-genome sequencing MRD assay to advance clinical research in oncology."

The Illumina WGS MRD assay, which is currently in development, will detect ctDNA for MRD assessment in research settings that evaluate samples from patients previously diagnosed with cancer across multiple solid tumor indications. In contrast with existing MRD solutions with complex workflows, Illumina plans to develop a research solution that will provide a cost-effective, highly sensitive, and automated workflow, with the potential to achieve a turnaround time of five to seven days.

Illumina intends to collaborate with other leaders in pharma to help further develop and expand the utility of its WGS MRD assay.

On January 5, 2024 Orion Corporation ("Orion") and MSD (known as Merck & Co., Inc., Rahway, N.J., USA in the United States and Canada) reported to have initiated two pivotal Phase 3 clinical trials evaluating ODM-208/MK5684, an investigational CYP11A1 inhibitor, in combination with hormone replacement therapy (HRT), for the treatment of certain patients with metastatic castration-resistant prostate cancer (mCRPC) (Press release, Orion, JAN 5, 2024, View Source [SID1234639025]). Patients are now enrolling in the trials, named OMAHA1 (NCT06136624) and OMAHA2a (NCT06136650).

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"The start of our co-development Phase 3 program with MSD provides exciting opportunities to evaluate the potential of ODM-208/MK5684 as a novel treatment of mCRPC, both in front-line and late-line patients, including those with and without androgen receptor ligand binding domain (AR LBD) mutations," said Professor Outi Vaarala, Senior Vice President, Innovative Medicines and Research and Development, Orion. "Our ultimate aim is to bring innovative medicines to all patients with unmet need, and we look forward to the beginning of these studies with our partner, MSD."

"By inhibiting CYP11A1 enzyme activity, we believe ODM-208/MK5684 represents a compelling approach to suppress the production of steroid hormones, a key driver of prostate cancer," said Dr. Scot Ebbinghaus, Vice President, global clinical development, MSD Research Laboratories. "The initiation of these two Phase 3 trials of ODM-208/MK5684 in different stages of mCRPC, in collaboration with Orion, demonstrates our continued commitment to exploring innovative new approaches and pathways to treat this complex disease."

OMAHA1 is a randomized, open-label Phase 3 trial evaluating ODM-208/MK5684 in combination with HRT for the treatment of patients with later-line mCRPC who have failed one prior new hormonal agent (NHA) and one or two prior taxane-based chemotherapies compared to an alternative NHA (abiraterone or enzalutamide). The trial will enroll an estimated 1,200 patients around the world. The primary endpoints are overall survival (OS) and radiographic progression-free survival (rPFS) by AR LBD mutation status. Secondary endpoints include time to first subsequent therapy (TFST), objective response rate (ORR) and duration of response (DOR).

OMAHA2a is a randomized, open-label Phase 3 trial evaluating ODM-208/MK5684 in combination with HRT for the treatment of patients with front-line mCRPC who have failed one prior NHA compared to physician’s choice of NHA (abiraterone or enzalutamide). The trial will enroll an estimated 1,500 patients around the world. The primary endpoints are OS and rPFS by AR LBD mutation status. Secondary endpoints include TFST, ORR and DOR.

About ODM-208/MK-5684

ODM-208/MK5684 is an oral, non-steroidal and selective inhibitor of the CYP11A1 enzyme discovered and developed by Orion and is being investigated for the treatment of hormone-dependent cancers, such as prostate cancer. By inhibiting CYP11A1 enzyme activity, ODM-208/MK5684 is designed to suppress the production of all steroid hormones and their precursors that may activate the androgen receptor signaling pathway.

About metastatic castration-resistant prostate cancer

Development of prostate cancer is often driven by male sex hormones called androgens, including testosterone. In patients with mCRPC, their prostate cancer grows and spreads to other parts of the body, despite the use of androgen-deprivation therapy to block the action of male sex hormones. Approximately 10-20% of patients with prostate cancer are estimated to develop castration-resistant prostate cancer (CRPC) within five years, with at least 84% of these patients presenting with metastases at the time of CRPC diagnosis. Of patients with no metastases at CRPC diagnosis, 33% are likely to develop metastases within two years. mCRPC is associated with a significant mortality rate.

On January 5, 2024 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported preliminary, unaudited ORLADEYO (berotralstat) net revenue for the fourth quarter and full year 2023 (Press release, BioCryst Pharmaceuticals, JAN 5, 2024, View Source [SID1234639024]). The company also provided guidance for full year 2024 ORLADEYO net revenue, full year 2024 operating expenses, expected peak ORLADEYO sales and an accelerated path to profitability.

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"After three years on the market, ORLADEYO continues on a steady growth trajectory to achieve $1 billion at peak. This commercial success, alongside our proven discovery platform that is producing additional first-in-class or best-in-class molecules, uniquely positions BioCryst to achieve financial independence from the capital markets and accelerate our path to profitability," said Jon Stonehouse, president and chief executive officer of BioCryst.

The company also announced that, if its ongoing proof-of-concept trial produces best-in-class data, it plans to out-license late-stage development and commercialization of BCX10013, its potential once-daily, oral Factor D inhibitor, to a partner that can drive the speed and breadth of investment required to accelerate BCX10013 for patients across multiple complement-mediated diseases and maximize the commercial potential of the program. As a result, the company has reduced the size of its R&D organization and accelerated its timeline to profitability.

Preliminary Fourth Quarter and Full Year 2023 ORLADEYO Revenue and 2024 ORLADEYO Outlook
Preliminary, unaudited ORLADEYO net revenue in the fourth quarter of 2023 was $89.9 million (+27 percent y-o-y). Preliminary, unaudited ORLADEYO net revenue for full year 2023 was $325 million (+29 percent y-o-y).

The company expects full year 2024 global net ORLADEYO revenue to be between $380 million and $400 million. The general pattern of revenue throughout 2024 is expected to be similar to past years, with the seasonal impact of prescription reauthorizations and the potential impact of the Inflation Reduction Act in the first quarter driving a quarter-over-quarter revenue decline in the first quarter, followed by a strong return to growth in the second quarter.

"ORLADEYO growth remained strong in the fourth quarter of 2023 as hereditary angioedema patients gain the excellent attack control they expect. Comparing U.S. patient trends year over year (y-o-y), we had more new patient prescriptions and a lower average rate of monthly discontinuations in 2023 compared to 2022. U.S. performance combined with continued global expansion keep ORLADEYO on track for $1 billion in peak sales," said Charlie Gayer, chief commercial officer of BioCryst.

Operating Expense and Profitability Outlook
The company expects full year 2024 operating expenses to be between $365 million and $375 million, flat to expected full year 2023 operating expenses. The company now expects that R&D expenses in 2024 will be reduced by $20 million versus 2023. This represents a $45 million to $55 million reduction from the 2024 R&D expense guidance it provided at its R&D Day in November 2023, and reflects both the R&D restructuring and the postponement of previously planned capital expenditures at its Discovery Center in Alabama. SG&A expenses are expected to increase by $20 million in 2024, primarily to support the continued U.S. and global growth of ORLADEYO to $1 billion in peak sales.

This operating expense outlook does not reflect non-cash stock compensation expense, or one-time expenses related to the reduction of 59 jobs (10 percent of total organization) in the first quarter of 2024.

Based on the company’s disciplined approach to capital allocation, and the revenue expected from ORLADEYO, the company expects to achieve a full-year operating profit in 2024 (not including non-cash stock compensation), be approaching quarterly positive earnings per share (EPS) and positive cash flow in the second half of 2025 (not including non-cash stock compensation), and be profitable on an EPS basis, with positive cash flow, for full year 2026. The company expects it can achieve these financial milestones without raising additional funds and does not intend to draw the additional $150 million of debt available to it from Pharmakon.

Presentation Tuesday at 42nd Annual J.P. Morgan Healthcare Conference
On Tuesday, January 9, 2024 at 6:00 p.m. ET, the company will present at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco. Links to a live audio webcast and replay of the presentation may be accessed in the Investors section of BioCryst’s website at https://www.biocryst.com/.

On January 5, 2024 Vor Biopharma presented its corporate presentation (Presentation, Vor BioPharma, JAN 5, 2024, View Source [SID1234639023]).

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On January 5, 2024 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that Michael S. Weiss, the Company’s Chairman and Chief Executive Officer, will present at the 42nd Annual J.P. Morgan Healthcare Conference. The presentation is scheduled to take place on Tuesday, January 9, 2024, at 4:30 PM PT (Press release, TG Therapeutics, JAN 5, 2024, View Source [SID1234639022]).

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A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source A replay of the webcast will be available on TG’s website following the event.