On July 30, 2024 Tempus AI, Inc. (NASDAQ: TEM), a leader in artificial intelligence and precision medicine, reported an expanded collaboration with Remix Therapeutics, a clinical-stage biotechnology company developing small molecule therapies to modulate RNA processing and address underlying drivers of disease (Press release, Tempus, JUL 30, 2024, View Source [SID1234645188]). The collaboration between Remix and Tempus began with the licensing of specific, de-identified data cohorts and has since expanded into a broader, strategic alliance.

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Remix is leveraging Tempus’ multimodal data to interrogate specific cohorts, including Adenoid Cystic Carcinoma (ACC) and Acute Myeloid Leukemia (AML) with Tempus’ data analytics platform, Lens. The newly expanded scope of work also includes next-generation sequencing support for Remix’s Phase I trial for REM-422, the company’s potent, selective, oral small molecule messenger RNA (mRNA) degrader.

To support its research, Remix is utilizing Tempus’ xT and xR assays to capture DNA and RNA data, as well as tracking treatment response on an exploratory research basis with xM Monitor, the company’s circulating tumor DNA (ctDNA) assay, which detects and monitors changes in circulating tumor fraction to determine response to therapy for patients with advanced cancers.

"We’re excited to work with a biotech like Remix that understands and embraces the value that Tempus’ multimodal data can bring to their important work," said Ryan Fukushima, Chief Operating Officer of Tempus. "We were able to quickly expand our collaboration and provide Remix with an array of our offerings that are uniquely positioned to support them in achieving their research and development goals."

"Tempus’ multimodal data, analytics, and sequencing support will be invaluable tools as we advance our lead candidate, REM-422, into the clinic," said Dominic Reynolds, Ph.D., Chief Scientific Officer of Remix. "This collaboration provides us with robust resources and data to propel our research forward, ultimately advancing our goal of creating meaningful new treatment options for patients."

On July 30, 2024 Day One Biopharmaceuticals, Inc. (Nasdaq: DAWN) ("Day One" or the "Company"), a biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, reported its second quarter 2024 financial results and highlighted recent corporate achievements (Press release, Day One, JUL 30, 2024, View Source [SID1234645152]).

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"We had an outstanding quarter across all facets of our business," said Jeremy Bender, Ph.D., chief executive officer of Day One. "Demand for OJEMDA led to strong early launch performance following our first approval, and we made significant progress advancing our programs and pipeline, including the addition of DAY301, a potential first-in-class ADC targeting PTK7 that we expect to be in the clinic in the coming months."

Program Highlights

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OJEMDA received U.S. Food and Drug Administration (FDA) accelerated approval in April 2024. It is the first and only FDA approved therapy for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.

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Day One provided updated duration of treatment data from the registrational Phase 2 FIREFLY-1 trial investigating tovorafenib in patients with BRAF-altered, relapsed or progressive pLGG. For the 77 patients enrolled on Arm 1, which was the dataset used to assess OJEMDA’s efficacy, the median duration of treatment is now 23.7 months, with some patients being on treatment out to 32 months. Additional analyses will be presented at future medical conferences.

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Day One and Ipsen entered into an exclusive licensing agreement to commercialize tovorafenib outside of the U.S. in July 2024. Under the agreement, Day One will receive approximately $111 million upfront in cash and equity investment at a premium with up to approximately $350 million in additional launch and sales milestone payments as well as tiered double-digit royalties starting in mid-teens percentage on net sales. Ipsen secured commercialization rights to tovorafenib outside of the U.S.

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Day One entered into an exclusive licensing agreement with MabCare Therapeutics for its novel ADC targeting protein-tyrosine kinase 7 (PTK7) in June 2024. The Company expects to dose the first patient in the Phase I portion of the Phase 1/2a clinical trial of DAY301 in the fourth quarter of 2024 or first quarter of 2025.

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Day One presented a poster on tovorafenib demonstrating reversibility of changes in growth velocity observed in the Phase 2 FIREFLY-1 clinical trial at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. These data were also shared at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO).

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Day One made the decision to close the pimasertib program in July 2024, including the FIRELIGHT-1 trial evaluating it in combination with tovorafenib. Resources will be redirected to the DAY301 program and results will be shared at a future medical meeting or publication.

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The pivotal Phase 3 FIREFLY-2/LOGGIC clinical trial evaluating tovorafenib as a front-line therapy in patients aged 6 months to 25 years with pLGG continues to enroll patients in the United States, Canada, Europe, Australia and Asia, with more than 100 sites activated.

Corporate Highlights and Upcoming Milestones

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Day One announced it entered into a definitive agreement for an oversubscribed private placement of its securities for total gross proceeds of approximately $175.0 million in July 2024.

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The Company sold the rare pediatric disease Priority Review Voucher awarded by the FDA upon OJEMDA’s approval for total cash proceeds of $108.0 million in May 2024, representing a gain on sale.

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Commercial operations veteran John Stubenrauch joined Day One in July 2024 as Chief Technology Officer. Dr. Stubenrauch, PhD, MBA, was most recently Chief Operating Officer at Nutcracker Therapeutics and brings more than 25 years of experience developing and commercializing medicines, including ADCs, as well as a broad range of product modalities.

Second Quarter 2024 Financial Highlights

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Cash Position: The Company’s cash, cash equivalents and short-term investments totaled $361.9 million as of June 30, 2024.

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Product Revenue, Net: OJEMDA net product revenues were $8.2 million for the second quarter of 2024, the first partial quarter of the U.S. launch.

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R&D Expenses: Research and development expenses were $92.1 million for the second quarter of 2024 compared to $32.2 million for the second quarter of 2023. The increase was primarily due to the MabCare Therapeutics license agreement upfront payment of $55.0 million, increased clinical trial activities related to tovorafenib, and additional employee compensation costs.

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SG&A Expenses: Selling, general and administrative expenses were $30.2 million for the second quarter of 2024 compared to $17.1 million for the second quarter of 2023. The increase was primarily due to additional employee compensation costs, commercial launch activities, and increased professional service expenses to support company growth.

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Net Loss: Net loss totaled $4.4 million for the second quarter of 2024 with non-cash stock-based compensation expense of $13.0 million, compared to $45.9 million for the second quarter of 2023 with non-cash stock-based compensation expense of $9.5 million.

Upcoming Events

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2024 Wedbush PacGrow Healthcare Conference, August 12-14, 2024

Conference Call

Day One will host a conference call and webcast today, July 30 at 8:00 a.m. Eastern Time. To access the live conference call by phone, dial 877-704-4453 (domestic) or 201-389-0920 (international), and provide the access code 13745150. Live audio webcast will be accessible from the Day One Investors & Media page. To ensure a timely connection to the webcast, it is recommended that participants register at least 15 minutes prior to the scheduled start time. An archived version of the webcast will be available for replay on the Events & Presentations section of the Day One Investors & Media page for 30 days following the event.

About OJEMDA

OJEMDA (tovorafenib) is a Type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases.

OJEMDA is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma and from the European Commission for the treatment of glioma.

For more information, please visit www.ojemda.com.

On July 30, 2024 Agendia, Inc. reported that the NSABP B-42 study evaluating the MammaPrint assay in predicting the benefit of extended endocrine therapy (EET) in early-stage breast cancer patients was published in the July issue of Journal of Clinical Oncology (Press release, Agendia, JUL 30, 2024, View Source [SID1234645189]).

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The published study, Utility of the 70-gene MammaPrint Assay for Prediction of Benefit from Extended Letrozole Therapy in the NRG Oncology/NSABP B-42 Trial, found that MammaPrint was predictive of EET benefit in early-stage hormone receptor positive (HR+) breast cancer patients who were classified by MammaPrint (MP) as Low Risk (0.000 to +0.355), having exhibited improved outcomes with EET.

NSABP B-42 (NCT00382070) was a prospective randomized, double-blind, placebo-controlled, phase III trial that enrolled 3,966 postmenopausal women with HR+ early-stage breast cancer who were disease-free after 5 years of endocrine therapy (with an Aromatase inhibitor (AI) or ≥3 years of tamoxifen followed by an AI). Patients were then randomized to receive 5 additional years of either letrozole or placebo. Results showed EET modestly reduced risk of recurrence in postmenopausal women and established a need to further stratify patients who could benefit from EET.

In a subset of 1,866 randomized patients, which were representative of the parent trial, MammaPrint classified tumors as either MammaPrint High Risk (n=706) or MammaPrint Low Risk (n=1160). MammaPrint Low Risk tumors were further stratified into UltraLow Risk (+0.356 to +1.000) (n=252) or Low (non-UltraLow) Risk (n=908). The data revealed that only MammaPrint Low (non-UltraLow) Risk tumors showed a statistically significant 10-year EET benefit of 9.5% for disease-free survival (DFS) and 7.9% for breast cancer-free interval (BCFI). Conversely, MammaPrint UltraLow and High Risk tumors did not derive statistically significant EET benefit. This highlights that MammaPrint Low (non-UltraLow) Risk is a predictive biomarker for improved outcomes with EET. These findings underscore MammaPrint’s utility in accurately predicting a subset of patients who benefit from the addition of EET to their treatment regimens, establishing MammaPrint as the most comprehensive standalone genomic test for short and long-term treatment planning.

"The work published by the NSABP B-42 group confirms the utility of MammaPrint for identifying women with early-stage HR+ breast cancer who will or will not benefit from extended endocrine therapy," said William Audeh, MD, MS, Chief Medical Officer at Agendia. "While we know that clinical factors are associated with the risk of developing a late recurrence in ER+ breast cancer, they do not indicate whether those late recurrences are preventable. These data support the utility of MammaPrint in predicting which women have a risk of late recurrence which is preventable by extended endocrine therapy. We are pleased to be able to add this study to the growing body of literature supporting the use of MammaPrint for answering many important questions in early breast cancer management with the goal of improving the standard of care in breast cancer."

On July 30, 2024 Leidos Holdings, Inc., a FORTUNE 500 innovation company, reported financial results for the second quarter of fiscal year 2024 (Press release, Leidos, JUL 30, 2024, View Source [SID1234647129]).

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Thomas Bell, Leidos Chief Executive Officer, commented, "In the second quarter, Leidos continued its industry-leading profitable growth and strong cash generation, showcasing the progress we’ve made towards our key operational priorities and the dedication to our "Promises Made, Promises Kept" philosophy. Our collaborative innovation strategy and capabilities-based organization position us well to deliver on our commitments for the remainder of 2024 and beyond."

On July 30, 2024 Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported that management will be attending the following upcoming investor conferences (Press release, Delcath Systems, JUL 30, 2024, View Source [SID1234645153]):

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BTIG Virtual Biotechnology Conference
Date: Tuesday, August 6, 2024
Location: Virtual

Canaccord Genuity’s 44th Annual Growth Conference
Date: Wednesday, August 14, 2024
Presentation Time: 9:30 am Eastern Time
Location: Boston, MA