On February 13, 2025 Nain Bio, a leading global biopharmaceutical company focused on addressing the challenge of tumor drug resistance , reported that preclinical research results from its independently developed small molecule modulator targeting the PDIA6/IRE1 protein interaction will be presented as a poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, held in Chicago, USA, April 25-30, 2025 (Press release, Nain Biotech, FEB 13, 2025, View Source [SID1234654626]). This groundbreaking research provides a new target and therapeutic strategy for selectively enhancing the sensitivity of tumor cells to endoplasmic reticulum stress (ER stress ) and inducing regulated cell death in tumors.

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Research Highlights:
Poster Topic: Development of a first-in-class PDIA6/IRE1 modulator for selectively sensitizing cancer cells to ER stress and regulated cell death.
Conference Section: Experimental and Molecular Therapeutics.
Panel Discussion: Novel Antitumor Agents 2.
Presentation Time: April 28, 2025, 2:00 PM–5:00 PM (Central Time).
Presentation Location: Booth 21, Poster #14 (Abstract #3045).

As the world’s oldest and largest academic oncology conference, the AACR (Free AACR Whitepaper) Annual Meeting has always been a touchstone for the development of innovative drugs. The inclusion of this research result not only validates Nain Bio’s original innovation capabilities in the field of tumor drug resistance , but also highlights the significant clinical translational value of this drug candidate. By precisely regulating the endoplasmic reticulum stress pathway , the company’s R&D team has opened up a new therapeutic dimension for overcoming tumor drug resistance .

On February 13, 2025 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer and obesity drugs, reported the receipt of newly issued patents in the United States and New Zealand, and updated investors on the extent of its global intellectual property portfolio (Press release, Bio-Path Holdings, FEB 13, 2025, View Source [SID1234650244]).

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Bio-Path received Notice of Allowance from the United States Patent and Trademark Office for U.S. Patent No. 17/339,366 titled, "P-ethoxy nucleic acids for STAT3 inhibition." The New Zealand Intellectual Property Office has granted Patent No. 741793 titled, "P-ethoxy nucleic acids for liposomal formulation." These new patents build on earlier patents granted that protect the platform technology for DNAbilize, the Company’s novel RNAi nanoparticle drugs.

"We are proud of our growing global intellectual property portfolio as it safeguards our technology, serves as a deterrent to would-be competitors and creates value around our core competencies," said Peter Nielsen, Chief Executive Officer of Bio-Path. "These efforts are designed to protect our investments and advance our clinical programs to bring new medicines to patients suffering with obesity and cancer. Importantly, our newly issued U.S. patent provides additional intellectual property protection around our STAT3 program, where we intend to advance into non-small cell lung cancer."

Bio-Path continues to expand its intellectual property portfolio by filing patent applications that are applicable to its technology and business strategy. Bio-Path’s patent portfolio currently includes seven issued patents in the U.S. and 61 issued patents in foreign jurisdictions, providing protection in 26 countries. The Company has three additional pending patent applications in the U.S. and five additional allowed patent applications in foreign jurisdictions.

On February 13, 2025 Oncotelic Therapeutics, Inc (OTCQB:OTLC). Oncotelic Therapeutics, a leader in RNA-based therapeutics, reported that Vuong Trieu, Ph.D., Chairman & CEO, will be delivering a featured presentation at the SWCR 2025 Conference on March 24th (Press release, Oncotelic, FEB 13, 2025, View Source [SID1234650261]). His talk will highlight the central role of Transforming Growth Factor Beta 2 (TGFB2) in the convergence of disease pathways spanning cancer, lupus, and aging, as well as its potential as a therapeutic target in immuno-oncology.

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Dr. Trieu’s presentation, titled "Transforming Growth Factor Beta 2 in Aging, Cancer, Lupus, and Immuno-Oncology: A Convergence of Disease Pathways and Therapeutic Potential," will delve into the immunosuppressive mechanisms of TGFB2 and its profound impact on solid tumors, autoimmune disorders, and age-related diseases. His research underscores how high TGFB2 expression fosters tumor-associated macrophage infiltration, disrupts antigen presentation, and drives resistance to immunotherapy-ultimately contributing to poor patient outcomes.

A key focus of the presentation will be the clinical development of OT-101, an antisense therapeutic targeting TGFB2, which is currently in Phase 3 clinical trials (STOP-PC study) for pancreatic ductal adenocarcinoma (PDAC). The talk will explore how TGFB2 inhibition-alone or in combination with immune checkpoint inhibitors or interferon-based strategies-may reshape the tumor microenvironment, boost immune response, and enhance therapeutic efficacy.

Dr. Vuong Trieu’s Vision for TGFB2 as a Therapeutic Target

"TGFB2 has emerged as a master regulator of immune suppression, not just in cancer but across multiple chronic diseases," said Dr. Trieu. "Our work on OT-101 aims to neutralize TGFB2-driven resistance, unlocking the full potential of immunotherapy and improving outcomes for patients with pancreatic cancer, gliomas, and beyond."

On February 13, 2025 (the "Closing Date"), Sarepta Therapeutics, Inc. (the "Company") and Sarepta Therapeutics Investments, Inc., a wholly owned subsidiary of the Company (together with the Company, the "Obligors") reported to have entered into a credit agreement (the "Credit Agreement") with JPMorgan Chase Bank, N.A., as administrative agent (in such capacity, the "Administrative Agent") and as collateral agent (in such capacity, the "Collateral Agent") and the lenders party thereto (Filing, 8-K, Sarepta Therapeutics, FEB 13, 2025, View Source [SID1234650287]). The Credit Agreement provides for a five-year, $600 million senior secured revolving credit facility (the "Revolving Credit Facility").

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Interest rates under the Revolving Credit Facility are variable and equal to the Secured Overnight Financing Rate plus a credit spread adjustment of 0.10% per annum ("Adjusted SOFR"), plus a margin of 1.125% to 1.75% per annum based on the Company’s total gross leverage ratio, or, at the Company’s option, at a base reference rate equal to the highest of (a) the federal funds rate plus 0.50%, (b) the rate of interest last quoted by the Administrative Agent as its "base rate" and (c) the one-month Adjusted SOFR rate plus 1.00%, plus a margin of 0.125% to 0.75% per annum based on the Company’s total gross leverage ratio.

The Company will pay customary agency fees and a commitment fee based on the daily unused portion of the Revolving Credit Facility at a rate of 0.20% to 0.35% per annum based on the Borrower’s total gross leverage ratio. The Revolving Credit Facility is not subject to amortization and will mature on the fifth anniversary of the Closing Date.

On the Closing Date, each of the Obligors and the Collateral Agent entered into a pledge and security agreement, pursuant to which the Obligors granted a security interest in substantially all of their respective assets, in each case, subject to customary exceptions and exclusions.

The Credit Agreement contains customary representations and warranties, affirmative covenants, negative covenants and events of default. The Credit Agreement also contains financial covenants that are tested on the last day of each of the Company’s fiscal quarters. These financial covenants include a (x) maximum secured net leverage ratio of 3.5:1.0, subject to a 4.0:1.0 covenant holiday following certain permitted acquisitions or permitted collaborations, and (y) minimum consolidated interest coverage ratio of 2.5:1.0.

The foregoing description of the Credit Agreement does not purport to be complete and is subject to, and qualified in its entirety by reference to, the full text of the Credit Agreement, attached hereto as Exhibit 10.1 and incorporated herein by reference.

On February 13, 2025 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2024 and provided financial guidance (Press release, Blueprint Medicines, FEB 13, 2025, View Source [SID1234650245]).

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"We are entering 2025 in the strongest position we have ever been in as a company with a focus on driving growth and innovation through operational excellence. AYVAKIT is a medicine that is changing patients’ lives, and the SM market is larger and growing faster than we originally expected. Our 2025 guidance of $680 to $710 million represents robust growth, putting AYVAKIT firmly on track to achieve $2 billion in revenue by 2030. And with the larger potential SM franchise peak opportunity of $4 billion, we have a plan to drive innovation in the treatment of SM with elenestinib as we move beyond symptom control to disease modification in the HARBOR registration study, building a durable SM franchise that we anticipate will grow throughout the next decade," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "Our next potential blockbuster opportunity, BLU-808, is also coming into focus with the healthy volunteer data reported last month. BLU-808 demonstrated a best-in-class profile for an oral wild-type KIT inhibitor enabling our differentiated approach to modulating mast cell activity across thousands of patients with mast-cell-mediated allergic and inflammatory diseases. BLU-808 offers us the opportunity to find the right balance of efficacy and tolerability to realize true pipeline-in-a-medicine potential. Altogether, our portfolio represents a unique set of assets, targeting fundamental mast cell biology, that will drive durable and diversified growth over the next decade with significant operating leverage."

Fourth Quarter 2024 Highlights and Recent Progress

Mast cell disorders

● Achieved AYVAKIT net product revenues of $479.0 million, including $421.8 million in the US and $57.1 million ex-US, representing 135 percent growth year-over-year. In the fourth quarter of 2024, AYVAKIT achieved $144.1 million, including $124.1 million in the US and $20.0 million ex-US.
● Blueprint estimates the peak revenue opportunity for the company’s SM franchise is $4 billion, with $2 billion in annual revenues expected to be achieved by AYVAKIT by 2030.
● Presented AYVAKIT data at the 2024 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, showing a significant survival benefit in treatment-naïve patients with advanced SM, and sustained improvement in bone density for advanced SM patients with low bone mass at baseline. View the presentations here.
● Reported in January 2025 results from the Phase 1 healthy volunteer study of BLU-808, a wild-type KIT inhibitor, showing a differentiated profile that enables the evaluation of tunable dosing strategies. BLU-808 was well-tolerated at all doses tested, showed consistent pharmacokinetics supporting once daily oral dosing, and achieved dose-dependent reductions in tryptase exceeding 80 percent. These positive data support initiation of multiple proof-of-concept studies planned in 2025.

Corporate

● Presented the company’s 2025 corporate outlook and strategy to drive continued growth at the 43rd Annual J.P. Morgan Healthcare Conference. View the press release here.
● Following closing of the IDRx acquisition by GSK, Blueprint anticipates approximately $80 million in gross proceeds from an equity stake in IDRx.
● Announced the appointment of Sherwin Sattarzadeh to Chief Business Officer, overseeing business development, portfolio leadership and project management, and alliance management. His previous roles over a decade of service at Blueprint include Head of Regulatory Affairs, Chief of Staff to the Chief Executive Officer, and Senior Vice President of Strategic Operations. He succeeds Helen Ho, Ph.D. who is departing the company to pursue another career opportunity.

2025 Financial Guidance

Blueprint reported it anticipates approximately $680 million to $710 million in global AYVAKIT net product revenues for all approved indications in 2025. The midpoint of this range represents 45 percent year-over-year revenue growth. In 2024, Blueprint reduced cash burn by more than 50 percent and expects further reduction in 2025 as the company continues to invest in advancing prioritized programs, balancing investing in innovation with financial discipline. The company continues to expect that its existing cash, cash equivalents and investments, together with anticipated future product revenues, will maintain a durable capital position to enable the company to achieve a self-sustainable financial profile.

Key Upcoming Milestones

The company plans to achieve the following milestones in the first half of 2025:

● Present 14 abstracts at the 2025 American Academy of Allergy, Asthma and Immunology (AAAAI) / World Allergy Organization (WAO) Joint Congress, including:
o Three-year safety and efficacy data from the PIONEER trial of AYVAKIT.
o Bone density improvements shown by AYVAKIT in the PIONEER trial.
o Phase 1 healthy volunteer data for BLU-808, a highly potent and selective oral inhibitor of wild-type KIT, consistent with top-line results reported in January 2025.
● Initiate two proof-of-concept studies of BLU-808 in patients with chronic spontaneous urticaria/chronic inducible urticaria, and allergic rhinitis/allergic conjunctivitis.

Fourth Quarter and Year End 2024 Results

● Revenues: Revenues were $146.4 million for the fourth quarter of 2024, including $144.1 million of net product revenues from sales of AYVAKIT/AYVAKYT and $2.2 million in collaboration revenues. Revenues for the year ended December 31, 2024 were $508.8 million, including $479.0 million of net product revenues from sales of AYVAKIT/AYVAKYT, and $29.9 million in collaboration and license revenues. Blueprint Medicines recorded $72.0 million and $249.4 million in revenues in the fourth quarter and year ended December 31, 2023, respectively.
● Cost of Sales: Cost of sales was $7.4 million for the fourth quarter of 2024 and $20.2 million for the year ended December 31, 2024, as compared to $0.3 million and $8.5 million for the fourth quarter and year ended December 31, 2023, respectively. This increase was primarily due to higher sales to our collaboration and other partners and an increase in product sales volume.
● R&D Expenses: Research and development expenses were $83.7 million for the fourth quarter of 2024 and $341.4 million for the year ended December 31, 2024, as compared to $97.5 million and $427.7 million for the fourth quarter and year ended December 31, 2023, respectively. This decrease was primarily due to operational efficiency across our portfolio as we executed across our top priority programs, and the timing of manufacturing of clinical trial materials. Research and development expenses also included $11.7 million in stock-based compensation expenses for the fourth quarter of 2024 and $47.5 million in stock-based compensation for the year ended December 31, 2024.
● SG&A Expenses: Selling, general and administrative expenses were $96.5 million for the fourth quarter of 2024 and $359.3 million for the year ended December 31, 2024, as compared to $79.3 million and $295.1 million for the fourth quarter and year ended December 31, 2023, respectively. This increase was primarily due to an increase in activities supporting the commercialization of AYVAKIT/AYVAKYT. Selling, general and administrative expenses included $16.6 million in stock-based compensation expenses for the fourth quarter of 2024 and $61.5 million in stock-based compensation for the year ended December 31, 2024.
● Net Loss: Net loss was $(50.0) million for the fourth quarter of 2024 and $(67.1) million for the year ended December 31, 2024, or a diluted net loss per share of $(0.79) and diluted net loss per share of $(1.07), respectively, as compared to a net loss of $(110.9) million for the fourth quarter of 2023 and a net loss of $(507.0) million for the year ended December 31, 2023, or a diluted net loss per share of $(1.82) and a diluted net loss per share of $(8.37), respectively.
● Cash Position: As of December 31, 2024, cash, cash equivalents and investments were $863.9 million, as compared to $767.2 million as of December 31, 2023.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:00 a.m. ET today to discuss fourth quarter and full year 2024 financial results and recent business activities. The conference call may be accessed by dialing 833-470-1428 (domestic) or 404-975-4839 (international), and referring to conference ID 349846. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

Upcoming Investor Conferences

Blueprint Medicines will participate in two upcoming investor conferences:

● 45th Annual Cowen Health Care Conference on Monday, March 3, 2025 at 1:10 p.m. ET.
● H.C. Wainwright 3rd Annual Autoimmune & Inflammatory Disease Virtual Conference on Thursday, March 27, 2025 at 12:00 p.m. ET.

Live webcasts of the presentations will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source Replays of the webcasts will be archived on the Blueprint Medicines website for 30 days following the presentations.