On May 5, 2025 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the first quarter ended March 31, 2025, and provided a corporate update (Press release, BioCryst Pharmaceuticals, MAY 5, 2025, View Source [SID1234652498]).

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"We started 2025 with another quarter of outstanding performance. ORLADEYO revenue growth was driven by moving ORLADEYO patients from free drug to paid at a much faster rate than we expected, resulting in a substantial increase to our annual guidance as we also move closer to peak sales of $1 billion. This increased financial strength accelerates our path to profitability and enables us to start paying down our debt, while continuing to invest in and advance our pipeline," said Jon Stonehouse, president and chief executive officer of BioCryst.

ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks

ORLADEYO net revenue in the first quarter of 2025 was $134.2 million (+51 percent year-over-year (y-o-y)).

The total percentage of all ORLADEYO patients on paid drug has increased to approximately 84 percent (compared to 73.5 percent at end of 2024), generating higher than expected ORLADEYO revenue that the company expects will continue through the full year.

First quarter prescriptions were strong, above the quarterly average in 2024, and the percentage of U.S. HAE patients who describe a strong preference for an oral prophylaxis therapy increased to 70 percent, up from 50 percent in 2023, in the company’s latest market survey of HAE patients.

Sales from the U.S. contributed 89.5 percent of global ORLADEYO net revenues in the first quarter. The number of patients treated with ORLADEYO outside the U.S. continued to grow in new and existing markets.
"By driving a dramatic increase in the rate of paid ORLADEYO patients through the prescription reauthorization period, on top of continued very strong new patient demand, our team has achieved in four months what we had expected would take three years, getting the rate of paid patients nearly to our long-term goal of 85 percent. As a result, we will capture significantly more revenue opportunity in 2025 and subsequent years and our path to peak sales of $1 billion is even more profitable than we had projected," said Charlie Gayer, chief commercial officer of BioCryst.

Rare Disease Pipeline

The goal with our pipeline is to build on our success with ORLADEYO by bringing additional selected, highly differentiated rare disease products to patients.

The company has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) to expand the ORLADEYO label to children with HAE aged 2 to 11 using an oral granule formulation. The company also expects to submit regulatory filings in 2025 in global territories, including Europe, Japan and Canada. ORLADEYO would be the first targeted oral prophylactic therapy for children with HAE.

The FDA has cleared the company’s investigational new drug application (IND) which will enable its clinical trial of BCX17725, an investigational KLK5 inhibitor for the treatment of Netherton syndrome, to enroll patients in the United States. This phase 1 trial is also open in Australia. The company expects initial data from the program in 2025.

Netherton syndrome is a serious, rare, lifelong genetic disorder causing disruption of the skin barrier with premature separation of the skin layers, chronic inflammation and vulnerability to serious infections, caused by lack of normal function of a natural inhibitor of KLK5. People with Netherton syndrome often have itchy, red, scaly, inflamed skin, fragile hair, and are more likely to develop severe food allergies, asthma and eczema. Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections. Currently, there are no approved treatments that target the underlying cause of Netherton syndrome. BCX17725 is designed to replace missing functions of the natural KLK5 inhibitor, which could restore the normal skin barrier and result in improved skin function, including protection from severe inflammatory and infectious complications of the disease.

The first clinical trial with suprachoroidal delivery of avoralstat, the company’s investigational plasma kallikrein inhibitor for the treatment of diabetic macular edema (DME), has been granted authorization to proceed in Australia. The company expects initial data from DME patients in 2025.

DME is an important cause of vision loss in diabetes and is due to leakage of fluid from the blood vessels in the retina. While current treatments focus on vascular endothelial growth factor (VEGF) inhibition, DME can develop from other mechanisms, such as the kallikrein-bradykinin pathway. This is supported by observations that many DME patients have an incomplete response to intravitreal anti-VEGF therapies that are administered every four to eight weeks. Avoralstat targets the kallikrein-bradykinin system on the retinal vascular endothelial cells and may result in less vascular leakage and less edema. Avoralstat, delivered to the suprachoroidal space, is designed to provide long-lasting exposure to the retinal vessels, which could result in less frequent injections and a reduced burden on patients and the healthcare system.
"Today we are reporting significant milestones for each of the next three programs in our pipeline, demonstrating our focus on advancing medicines with the potential to provide differentiated outcomes for patients of all ages. As we move closer to delivering an urgently needed oral prophylactic therapy to children with HAE, we are simultaneously progressing two clinical-stage programs in Netherton syndrome and DME towards the first patient data later this year," said Dr. Helen Thackray, chief research and development officer of BioCryst.

First Quarter 2025 Financial Results

For the three months ended March 31, 2025, total revenues were $145.5 million (+$52.7 million y-o-y), compared to $92.8 million in the first quarter of 2024 (+56.8 percent y-o-y). The increase was primarily due to $134.2 million (+$45.3 million y-o-y) in ORLADEYO net revenue in the first quarter of 2025, compared to $88.9 million in ORLADEYO net revenue in the first quarter of 2024 (+51.0 percent y-o-y).

Research and development expenses for the first quarter of 2025 decreased to $37.3 million from $46.5 million in the first quarter of 2024 (-19.8 percent y-o-y), primarily due to decreased expenses driven by the discontinuation and close-out of the Factor D programs, BCX10013 and BCX9930. These reductions were partially offset by an increase in other research, preclinical and development costs, comprised of avoralstat and other early-phase pipeline programs, and a change in general and administrative expense allocations.

Selling, general and administrative expenses for the first quarter of 2025 increased to $82.5 million, compared to $59.5 million in the first quarter of 2024 (+38.7 percent y-o-y). The increase was primarily due to increased commercial investment to support our growing ORLADEYO revenue, our newly launched regions, expanded international operations and global commercial support activities. Additionally, there was an increase to general and administrative expenses, and an offsetting reduction to research and development expenses, due to a change in the general and administrative expense allocations in 2025.

Operating income for the first quarter of 2025 was $21.2 million, compared to an operating loss of $14.5 million for the first quarter of 2024. Non-GAAP operating income, excluding stock-based compensation expense, was $42.6 million for the first quarter of 2025, compared to a non-GAAP operating loss of $0.8 million for the first quarter of 2024.

Interest expense was $23.5 million in the first quarter of 2025, compared to $24.5 million in the first quarter of 2024 (-4.1 percent y-o-y). The decrease was primarily due to a decrease in interest expense associated with the interest accrued under the Pharmakon Loan Agreement.

Net income for the first quarter of 2025 was $32 thousand, or $0.00 per share, compared to a net loss of $35.4 million, or $0.17 per share, for the first quarter of 2024.

Cash, cash equivalents, restricted cash and investments totaled $317.3 million at March 31, 2025, compared to $338.4 million at March 31, 2024. Net cash utilization for the first quarter of 2025 was $25.5 million, which was driven by debt service, royalty payments and one-time first quarter compensation expense.

Early in the second quarter, the company paid down $75 million of the outstanding Pharmakon debt, which will result in approximately $23.5 million in interest savings over the life of the loan.

Financial Outlook for 2025
The company is increasing its outlook for full year 2025 global net ORLADEYO revenue to be between $580 million and $600 million (previously $535 million to $550 million).

The company now expects full year 2025 operating expenses will be $440 million to $450 million (previously $425 million to $435 million). This operating expense outlook does not reflect non-cash stock compensation expense.

The company is accelerating its expectation for sustainable profitability and positive cash flows by a year. The company now expects to deliver net income and positive cash flows for full year 2025 (previously expected both for full year 2026). Positive cash flow refers to the improvement in cash, cash equivalents, restricted cash and investments from year end 2024 to year end 2025, not including the impact of the $75 million Pharmakon prepayment made in April 2025.

Conference Call and Webcast
BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 1-844-481-2942 for domestic callers and 1-412-317-1866 for international callers. A live webcast and replay of the call will be available online in the investors section of the company website at www.biocryst.com.

On May 5, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (TSX: APS; OTC: APTOF), a clinical-stage precision oncology company, reported updated and new data from Aptose’s Phase 1/2 TUSCANY trial in newly diagnosed acute myeloid leukemia (AML) patients dosed with a 40 mg or 80 mg dose of tuspetinib (TUS) in combination with standard of care dosing of venetoclax and azacitidine (TUS+VEN+AZA triplet) (Press release, Aptose Biosciences, MAY 5, 2025, View Source [SID1234652497]). The TUS+VEN+AZA triplet is being developed as a safe and mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy.

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Earlier this year, Aptose announced the initiation of the TUSCANY trial and dosing in newly diagnosed AML patients at an initial dose of 40 mg TUS in the first cohort of four patients. The second cohort of patients is now receiving 80 mg TUS. Data from the first two cohorts, with a 40 mg or 80 mg dose of tuspetinib in the TUS+VEN+AZA combination, reveal promising clinical safety and antileukemic activity.

To date, four newly diagnosed AML patients received the initial dose of TUS (40 mg) as part of the (TUS+VEN+AZA) combination.
Notably, a patient with biallelic TP53 mutations and a complex karyotype (TP53-mutated/CK) and FLT3-wildtype achieved a complete remission (CR) and the clinical site reported no measurable residual disease (MRD-negative status) in this patient.
One FLT3-wildtype patient having an IDH-2 mutation achieved a CR and MRD-negative status.
Another FLT3-wildtype patient achieved a CRi during Cycle 1 and MRD-negative status.
The first three patients continue on treatment, while a fourth patient did not respond at this 40 mg dose level of TUS and was discontinued.
The 40 mg dose of the TUS+VEN+AZA triplet remains safe, and no dose-limiting toxicities (DLTs) have been reported.
To date, three newly diagnosed AML patients having diverse mutation profiles have received the 80 mg of TUS, as part of the TUS+VEN+AZA combination. The 80 mg TUS dose has been considered the optimal dose that has demonstrated safety and consistent blood exposure levels that exert potent antileukemic activity.
All patients achieved blast reductions in Cycle 1 that met the criteria for complete remissions (CR or CRi) and continue on treatment.
Notably, another TP53-mutated/CK and FLT3-wildtype patient achieved blast reductions that met CRi criteria in Cycle 1 and is now receiving additional therapy in Cycle 2.
The second patient, having FLT3-wildtype status, achieved a CR.
The third patient, having FLT3-ITD and NPM1 mutations and entering the trial with a 75% bone marrow blast count, achieved a CRi.
All three patients are early in their course of treatment and are expected to show further improvements in their disease status as they are all continuing with treatment, and MRD status will be monitored as the patients move through their courses of therapy.
The 80 mg dose of TUS, as part of the TUS+VEN+AZA triplet, continues to show favorable safety with no dose-limiting toxicities (DLTs) having been reported.
"The treatment paradigm for AML is shifting to triplet combination therapy," said Rafael Bejar, M.D., Ph.D., Chief Medical Officer of Aptose. "We have always maintained that tuspetinib, with its notable safety profile and ability to treat the larger, difficult-to-treat AML populations with high-risk mutations, could be an ideal drug for a triplet combination therapy in the frontline setting. With the majority of patients already achieving complete responses — including early responses in patients with adverse mutations — the clinical findings to date are bearing that out."

William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose, will review the data at a presentation today, Monday, May 5th, 2025, 3:00 p.m. EDT, at the 2025 Bloom Burton & Co. Healthcare Investor Conference. The presentation and webcast can be accessed here and will be available on the Aptose website.

TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study

Tuspetinib based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 trial with the goal of creating an improved frontline therapy for newly diagnosed AML patients that is active across diverse AML populations, durable, and well tolerated. Earlier APTIVATE trials of TUS as a single agent and in combination as TUS+VEN demonstrated favorable safety and broad activity in diverse relapsed or refractory (R/R) AML populations that went beyond the more prognostically favorable NPM1 and IDH mutant subgroups. Responses to TUS were also observed in those with prior-VEN and prior-FLT3 inhibitor (FLT3i) therapies, those with highly adverse TP53 and RAS mutations, and those with mutated or unmutated (wildtype) FLT3 genes.

The TUSCANY triplet Phase 1/2 study is designed to test various doses and schedules of TUS in combination with standard dosing of AZA and VEN for patients with AML who are ineligible to receive induction chemotherapy. A convenient, once daily oral agent, TUS will be administered in 28-day cycles, beginning at 40mg once daily, with dose escalations planned after a safety review of each dose level. Multiple U.S. sites are enrolling in the TUSCANY trial with anticipated enrollment of 18-24 patients by mid-late 2025. Data will be released as it becomes available.

More information on the TUSCANY Phase 1/2 study can be found on www.clinicaltrials.gov (here).

On May 5, 2025 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported financial results for the first quarter ended March 31, 2025, and provided a business update (Press release, AnaptysBio, MAY 5, 2025, View Source [SID1234652496]).

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"Our lead program, rosnilimab, delivered impressive three-month Phase 2b efficacy, safety and tolerability data in rheumatoid arthritis (RA), with data through six months surpassing those of competitor all-active, head-to-head trials. We will report updated clinical and translational RA data in the first week of June, as well as initial Phase 2 ulcerative colitis (UC) data in Q4 2025, further defining rosnilimab’s game-changing potential," said Daniel Faga, president and chief executive officer of Anaptys. "With ANB033 and ANB101 progressing through Phase 1 trials, our autoimmune portfolio promises multiple catalysts over the next couple of years. We remain well-capitalized as we execute on our broad development plan for all three programs, while concurrently executing our $75 million stock repurchase program which are both further supported by substantial royalties and milestone payments anticipated from our GSK financial collaboration."

PORTFOLIO UPDATES

Rosnilimab (PD-1 depleter and agonist)

Announced in February that subcutaneously administered rosnilimab, including two once-monthly doses, achieved positive results in 424-patient Phase 2b RA trial and highest-ever reported clinical disease activity index (CDAI) low disease activity (LDA) response over 6 months
Full press release can be found here
Anaptys to host an investor call featuring Anaptys management and key opinion leaders in the first week of June to present rosnilimab’s updated Phase 2b clinical and translational data

Enrollment ongoing for global Phase 2 trial in moderate-to-severe UC
132-patient trial assessing two dose levels of subcutaneously administered rosnilimab vs. placebo (randomized 1:1:1)
Primary statistical analysis at Week 12 on well-established endpoints, including the primary endpoint of change from baseline in modified Mayo score (mMS) and supportive secondary endpoints of clinical response on mMS, clinical remission on mMS and endoscopic remission
All patients in all three study arms treat-through to Week 24 and remain blinded to treatment arm. Placebo-treated patients who achieved clinical response on partial modified Mayo score (pmMS) at Week 12 remain on placebo, while placebo-treated patients who are non-responders are crossed over to the high-dose rosnilimab treatment arm
Patients who are in clinical response on pmMS at Week 24 are eligible for an additional 26-week (50 weeks of total treatment) blinded treatment extension period (TEP)
Initial Phase 2 data anticipated in Q4 2025
ANB033 (CD122 antagonist)

Enrollment ongoing for Phase 1a trial in healthy volunteers
Phase 1b indication to be disclosed at H2 2025 R&D event
ANB101 (BDCA2 modulator)

Enrollment initiated for Phase 1a trial in healthy volunteers
COLLABORATION UPDATES

GSK Immuno-Oncology Financial Collaboration

GSK announced strong commercial performance for Jemperli ($220 million in Q1 2025 sales) with >15% quarter-over-quarter growth
GSK announced the EMA approval of Jemperli plus chemotherapy for all adult patients with primary advanced or recurrent endometrial cancer in January 2025

Anticipate receipt of a $75 million commercial sales milestone payment from GSK in either 2025 or 2026 once Jemperli achieves $1 billion in worldwide net sales in a calendar year

GSK anticipates top-line data in mid-2025 from COSTAR Lung Phase 3 trial in patients with advanced NSCLC who have progressed on prior anti-PD-(L)1 therapy and platinum-based chemotherapy comparing docetaxel alone to cobolimab, a TIM-3 antagonist, plus dostarlimab, a PD-1 antagonist, plus docetaxel and to dostarlimab plus docetaxel

Recent data published in The New England Journal of Medicine (NEJM) and presented at American Association for Cancer Research (AACR) (Free AACR Whitepaper) demonstrated neoadjuvant treatment with dostarlimab resulted in organ preservation in a high proportion of patients (80% of 103 patients), including 100% (rectal; n=49), 100% (bladder; n=6), and 82% (colon; n=22) complete responses in April 2025
GSK anticipates top-line data in 2026 from AZUR-1 pivotal Phase 2 trial of dostarlimab monotherapy in patients with untreated stage II/III dMMR/MSI-H locally advanced rectal cancer
Jemperli received U.S. FDA Breakthrough Therapy Designation for this indication in December 2024
Vanda Imsidolimab Collaboration

Announced an exclusive $15 million global out-license agreement with Vanda Pharmaceuticals to develop and commercialize imsidolimab (IL-36R antagonist), with Anaptys eligible to receive up to $35 million for future regulatory approvals and sales milestones, in addition to 10% royalty on global net sales
FDA BLA submission for generalized pustular psoriasis (GPP) expected in 2025
Full press release can be found here
FINANCIAL UPDATES

Stock Repurchase Program and Cash Runway

Authorized a Stock Repurchase Program in March 2025 of $75.0 million of the Company’s outstanding common stock

Cash and investments of $383.0 million as of March 31, 2025, and reiterating cash runway through year-end 2027
First Quarter 2025 Financial Results

Cash, cash equivalents and investments totaled $383.0 million as of March 31, 2025, compared to $420.8 million as of December 31, 2024, for a decrease of $37.8 million due primarily to operating activities and $4.4 million in shares repurchased offset by $15.0 million received from Vanda Pharmaceuticals for the license of imsidolimab.

Collaboration revenue was $27.8 million for the three months ended March 31, 2025, compared to $7.2 million for the three months ended March 31, 2024. The increase is due to a $11.0 million increase in royalties recognized for sales of Jemperli and $9.6 million in revenue recognized for the Vanda license agreement.

Research and development expenses were $41.2 million for the three months ended March 31, 2025, compared to $37.0 million for the three months ended March 31, 2024. The increase was due primarily to development costs relating to the Phase 2 trials in RA and UC for rosnilimab, and the Phase 1 trials for ANB033 and ANB101, offset by a decrease in development costs for imsidolimab and ANB032. The R&D non-cash, stock-based compensation expense was $4.4 for the three months ended March 31, 2025 as compared to $3.5 million in the same period in 2024.

General and administrative expenses were $14.1 million for the three months ended March 31, 2025, compared to $12.3 million for the three months ended March 31, 2024. The increase was due primarily to transaction costs associated with the Vanda Pharmaceuticals license agreement. The G&A non-cash, stock-based compensation expense was $4.8 million for the three months ended March 31, 2025 as compared to $6.7 million in the same period in 2024.

Net loss was $39.3 million for the three months ended March 31, 2025, or a net loss per share of $1.28, compared to a net loss of $43.9 million for the three months ended March 31, 2024, or a net loss per share of $1.64.

On May 4, 2025 Nutshell Therapeutics ( Shanghai ) Co., LTD. reported to have received IND clearance from the FDA to initiate Phase 1 clinical trial in the United States for its NTS071, a novel small molecule allosteric reactivator targeting p53 Y220C mutation (Press release, Nutshell Therapeutics, MAY 4, 2025, View Source [SID1234652483]).

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NTS071 is an oral small molecule allosteric reactivator targeting p53 Y220C with a novel scaffold. It selectively binds to the p53 Y220C mutant protein, improving its thermal stability, thereby enhancing the mutant protein’s ability to bind with DNA and restoring its transcriptional activity as well as tumor-suppressing function.

NTS071 was discovered by leveraging Nutshell’s proprietary AI driven allosteric small molecule drug discovery platform ALLOSTAR. NTS071 demonstrated potential Best-in-Class preclinical properties for its target. NTS071 achieved a picomolar-level biochemical activity which is 20 folds more potent than the competitive compound PC14586. Additionally, NTS071 shows better stability in both liver microsomes and hepatocytes across different species and exhibits lower in vivo clearance rates and higher oral exposure s in preclinical PK studies across all tested species compared to PC14586. NTS071 has relatively lower plasma protein binding and higher free fraction than PC14586, which is beneficial for in vivo efficacy. NTS071 also addresses the CYP3A4 inhibition issue of PC14586, presenting lower risks for potential drug – drug interactions. NTS071 further displays a large safety window by exhibiting an overall good safety profile in non-clinical toxicology studies.

NTS071 exhibited dose-dependent in vivo anti-tumor activity in multiple CDX and PDX models harboring p53 Y220C mutation, spanning a number of different cancer types, including ovarian cancer, lung cancer, gastric cancer, breast cancer, head and neck cancer, esophageal cancer, pancreatic cancer, and bladder cancer, etc. Therefore, NTS071 has the potential to be a tumor-agnostic therapy for patients carrying p53 Y220C mutation. Compared to PC14586, NTS071 has shown significantly lower effective doses or better efficacy at the same dose level in all comparative preclinical in vivo studies, implying that NTS071 may overcome the limitation of its competitor that has a higher dose requirement, thereby potentially achieving better therapeutic effects. NTS071 is anticipated to initiate Phase 1 clinical trial in second half of 2025 and expected to benefit patients with solid tumors harboring this mutation.

p53 Y220C mutation is widespread in various solid tumors. Studies show that there are 125,000 to 150,000 new cases worldwide annually, representing a significant market potential. Enriched with years of accumulated experience with its proprietary computation-based allosteric drug development technology and breakthrough innovation capabilities at Nutshell Therapeutics, NTS071 has the potential to stand out among peer products and become the most competitive drug molecule for this target.

The NTS071 Poster presented at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) (ENA) 2024 conference can be accessed here: View Source

On May 3, 2025 TAE Life Sciences (TLS) reported the signing of a Letter of Intent to collaborate with The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) in the development and evaluation of novel boron-based drug compounds—an essential component of Boron Neutron Capture Therapy (BNCT) (Press release, TAE Life Sciences, MAY 3, 2025, View Source [SID1234652484]).

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This partnership marks the first-of-its-kind U.S.-based academic-industry alliance dedicated to accelerating BNCT drug innovation and translational research. By optimizing boron delivery agents, TLS and OSUCCC aim to unlock BNCT’s full potential as a powerful, highly selective, and relatively non-toxic therapy and positioning it as a new modality in the next era of precision oncology.

"We are entering a pivotal moment in cancer therapy," said Dr. Arnab Chakravarti, Chair and Professor of Radiation Oncology, Klotz Family Chair of Cancer Research and Director of the Brain Tumor Program at Ohio State. "BNCT offers an unprecedented opportunity to treat malignancies that are otherwise unresponsive to conventional therapies, while sparing healthy tissue. Our laboratory is uniquely poised to lead this next wave of boron drug innovation, and we are excited to collaborate with TAE Life Sciences in accelerating this effort on a global scale."

The initial focus of the collaboration will be the preclinical evaluation of TLS’s proprietary boron-10 drugs in cellular and animal models, leveraging Ohio State’s specialized neutron source optimized for BNCT research. In parallel, the teams will pursue joint development of next-generation boron drugs for BNCT and adjacent applications—unlocking new possibilities in precision cancer treatment.

"This partnership marks a major leap forward for the future of BNCT," said Robert Hill, CEO of TAE Life Sciences. "We are deeply honored to collaborate with Dr. Chakravarti and his world-class team of research and clinical experts. Their pioneering track record of leadership and innovation will be instrumental not only in advancing the science behind novel boron drug compounds but also in paving the way toward potential regulatory approval. Together, we’re creating a dynamic force for innovation that has the potential to redefine cancer care."

Hill notes that this partnership establishes a key pillar for accelerator-based BNCT and significantly accelerate the path to breakthrough cancer therapies, positioning the United States to have a leading position in the next wave of innovation in precision oncology. Hill emphasized that the growing body of international clinical evidence—particularly from Japan, Taiwan, and Europe validates BNCT’s ability to deliver durable responses in patients with otherwise untreatable cancers. This collaboration will help position the United States in having a leading position in advanced drug development and establishing the next wave in precision medicine.