On May 14, 2025 Citius Pharmaceuticals, Inc. ("Citius Pharma" or the "Company") (Nasdaq: CTXR), a biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products reported business and financial results for the fiscal quarter ended March 31, 2025 (Press release, Citius Pharmaceuticals, MAY 14, 2025, View Source [SID1234653057]).

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"As we continue to focus on the planned launch of our first FDA-approved product, LYMPHIR, through Citius Oncology, we are actively engaged in securing the necessary financing to advance our launch strategy in the coming months, as well as exploring strategic partners for Citius Oncology," said Leonard Mazur, Chairman and CEO of Citius Pharmaceuticals and Citius Oncology.

"We are also in the process of preparing a submission to the FDA that reflects the valuable feedback we received from the agency concerning clinical efficacy, safety data, and in-vitro data. This submission is a key step toward supporting a future New Drug Application (NDA) for our Mino-Lok program. As a reminder, our Phase 3 Trial, which was completed last year, met its primary endpoints," added Mazur.

"During the quarter, we took deliberate steps to strengthen our financial position, including completing a registered direct offering and leveraging our existing at-the-market sales agreement to ensure capital flexibility. We also amended our license agreement with Eisai to align our payment obligations with our commercialization timeline. With these efforts underway, we believe we are positioned to deliver long-term value to patients and shareholders alike," concluded Mazur.

FISCAL SECOND QUARTER 2025 Financial Results:

Liquidity

During the six months ended March 31, 2025, the Company received net proceeds of $6 million from the issuance of equity. On April 2, 2025, the Company closed on a registered direct offering to an institutional investor of our common stock and pre-funded warrants to purchase common stock. The net proceeds to the Company from the offering were approximately $1.735 million, after deducting placement agent fees and other offering expenses payable by the Company.

As of March 31, 2025, the Company had $26,410 in cash and cash equivalents and 8,760,649 common shares outstanding excluding the April 2, 2025 financing. Citius Pharma will need to secure additional capital to support operations beyond May 2025.

Until Citius Oncology raises adequate capital through equity financings from outside investors and/or generates revenue from the future sales of LYMPHIR, Citius Pharma plans to continue to fund Citius Oncology. Citius Oncology has also retained Jefferies LLC as its exclusive financial advisor to evaluate strategic alternatives aimed at maximizing stockholder value.

Research and Development (R&D) Expenses

R&D expenses were $3.8 million for the quarter ended March 31, 2025, as compared to $3.6 million for the quarter ended March 31, 2024. For the six months ended March 31, 2025, R&D expenses were $5.9 million, as compared to $6.3 million during the six months ended March 31, 2024. R&D expenses primarily reflect LYMPHIR-related costs.

Research and development costs for LYMPHIR were $5.3 during the six months ended March 31, 2025, as compared to $3.2 million for the six months ended March 31, 2024. The $2.1 million increase in expenses was primarily due to costs associated with the expense of a drug substance batch needed for the pre license inspection of the manufacturer.

R&D expenses related to Mino-Lok decreased due to completion of the Phase 3 trial. There were no Halo Lido R&D expenses during the quarter, and $11 thousand was recorded for the six months ended March 31, 2025.

We expect that research and development expenses will continue to decrease in fiscal 2025 as we continue to focus on the commercialization of LYMPHIR and because we have completed the Phase 3 trial for Mino-Lok.

General and Administrative (G&A) Expenses

G&A expenses were $4.8 million for the quarter ended March 31, 2025, as compared to $4.3 million for the quarter ended March 31, 2024. For the six months ended March 31, 2025, G&A expenses were $10.2 million, as compared to $7.9 million for the six months ended March 31, 2024. The increase was primarily due to higher costs for pre-launch commercial activities associated with LYMPHIR. General and administrative expenses consist primarily of compensation costs, professional fees for legal, regulatory, accounting, and corporate development services, and investor relations expenses.

Stock-based Compensation Expense

For the quarter ended March 31, 2025, stock-based compensation expense was $2.7 million, as compared to $3.1 million for the quarter ended March 31, 2024. For the six months ended March 31, 2025, stock-based compensation expense was $5.2 million, as compared to $6.1 million for the six months ended March 31, 2024. Stock-based compensation expense is primarily related to the Citius Oncology stock plans. The decrease compared to the prior year is due to lower costs associated with the Citius Pharma stock plans.

Net loss

Net loss was $11.5 million, or ($1.27) per share, for the quarter ended March 31, 2025, as compared to a net loss of $8.5 million, or ($1.34) per share, for the quarter ended March 31, 2024, as adjusted for the reverse stock split. The increase in net loss was due to a $2.6 million decrease in other income, offset by the increase in general and administrative expenses and research and development expenses.

For the six months ended March 31, 2025, we incurred a net loss of $21.8 million, as compared to a net loss of $17.8 million for the six months ended March 31, 2024. The $4.0 million increase in the net loss was primarily due to the increase of $2.2 million in general and administrative expenses and the decrease in other income of $2.9 million, partially offset by lower research and development expense and lower stock-based compensation expense.

On May 14, 2025 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, reported financial results for the first quarter ended March 31, 2025 and other recent business developments (Press release, Celcuity, MAY 14, 2025, View Source [SID1234653056]).

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"We continue to make steady progress across our pipeline with several critical data catalysts anticipated this year," said Brian Sullivan, CEO and co-founder of Celcuity. "We now expect to report topline data for the PIK3CA wild-type cohort of the VIKTORIA-1 trial in the third quarter of this year and to report topline data for the PIK3CA mutant cohort in the fourth quarter of 2025. If our topline data from the WT cohort is positive, we expect the data will support the filing of our first new drug application and, if approved, our transition to a commercial stage company."

First Quarter 2025 Business Highlights and Other Recent Developments

● Based on evaluation of blinded event rates in the ongoing VIKTORIA-1 Phase 3 clinical trial, the primary completion date for the PIK3CA wild-type patient cohort is projected to occur in June 2025 with topline data now anticipated to be available in the third quarter of 2025.

○ Enrollment is ongoing in the PIK3CA mutant cohort of the VIKTORIA-1 trial and remains on track to report topline data in the fourth quarter of 2025.
○ VIKTORIA-1 is a global Phase 3 study evaluating gedatolisib in combination with fulvestrant with and without palbociclib in adults with HR+, HER2- advanced breast cancer who have received prior treatment with a CDK4/6 inhibitor.

● Site activation activities are underway globally for the VIKTORIA-2 Phase 3 clinical trial and dosing of the first patient is anticipated to occur in the second quarter of 2025.

○ VIKTORIA-2 is a global, Phase 3 open-label randomized study evaluating the efficacy and safety of gedatolisib in combination with fulvestrant plus a CDK4/6 inhibitor, either ribociclib or palbociclib, in comparison to fulvestrant plus a CDK4/6 inhibitor as a first-line treatment for patients with HR+/HER2- advanced breast cancer who are endocrine therapy resistant.
○ Prior to initiating the Phase 3 portion of the study, a safety run-in will be conducted in 12-36 participants to assess the safety of gedatolisib in combination with ribociclib and fulvestrant.

● The CELC-G-201 study is on track to report topline data for the Phase 1b portion of the trial late in the second quarter of 2025.

○ CELC-G-201 is a Phase 1b/2 evaluating gedatolisib in combination with darolutamide for the treatment of patients with metastatic castration resistant prostate cancer (mCRPC) whose disease progressed while on or after treatment with an androgen receptor signaling inhibitor.
○ The Phase 1b portion of the trial will assess the safety and tolerability of gedatolisib in combination with darolutamide and is expected to identify the recommended phase 2 dose regimen.

● Initiating a clinical trial collaboration with the Dana Farber Cancer Institute and Massachusetts General Hospital to evaluate gedatolisib in combination with abemaciclib and letrozole in patients with endometrial cancer

○ In a prior Phase 2 study, gedatolisib was evaluated as a monotherapy in patients with endometrial cancer.

First Quarter 2025 Financial Results

Unless otherwise stated, all comparisons are for the first quarter ended March 31, 2025, compared to the first quarter ended March 31, 2024.

Total operating expenses were $36.1 million for the first quarter of 2025, compared to $22.5 million for the first quarter of 2024.

Research and development ("R&D") expenses were $32.2 million for the first quarter of 2025, compared to $20.6 million for the prior-year period. Of the approximately $11.6 million increase in R&D expenses, $5.9 million primarily related to increased employee and consulting expenses. The remaining $5.7 million primarily related to activities supporting our ongoing clinical trials.

General and administrative ("G&A") expenses were $3.9 million for the first quarter of 2025, compared to $1.8 million for the prior-year period. Increased employee and consulting expenses accounted for $1.6 million of the increase. Professional fees, expanding infrastructure and other administrative expenses accounted for the remaining increase of approximately $0.5 million.

Net loss for the first quarter of 2025 was $37.0 million, or $0.86 loss per share, compared to a net loss of $21.6 million, or $0.64 loss per share, for the first quarter of 2024. Non-GAAP adjusted net loss for the first quarter of 2025 was $34.7 million, or $0.81 loss per share, compared to non-GAAP adjusted net loss of $19.9 million, or $0.59 loss per share, for the first quarter of 2024. Non-GAAP adjusted net loss excludes stock-based compensation expense, non-cash interest expense, and non-cash interest income. Because these items have no impact on Celcuity’s cash position, management believes non-GAAP adjusted net loss better enables Celcuity to focus on cash used in operations. For a reconciliation of financial measures calculated in accordance with generally accepted accounting principles in the United States ("GAAP") to non-GAAP financial measures, please see the financial tables at the end of this press release.

Net cash used in operating activities for the first quarter of 2025 was $35.9 million, compared to $17.1 million for the first quarter of 2024.

At March 31, 2025, Celcuity reported cash, cash equivalents and short-term investments of $205.7 million. We expect cash, cash equivalents, investments and drawdowns on our debt facility to fund current clinical development program activities through 2026.

Webcast and Conference Call Information

The Celcuity management team will host a webcast/conference call at 4:30 p.m. ET today to discuss the first quarter 2025 financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-800-717-1738 and international callers should dial 1-646-307-1865. A live webcast presentation can also be accessed using this weblink: View Source;tp_key=61a8c66165. A replay of the webcast will be available on the Celcuity website following the live event.

On May 14, 2025 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird"), Carlyle (NASDAQ: CG) ("Carlyle") and SK Capital Partners, LP ("SK Capital") reported they have amended their definitive agreement pursuant to which Carlyle and SK Capital will purchase all of the outstanding shares of bluebird (Press release, bluebird bio, MAY 14, 2025, View Source [SID1234653055]). Under the terms of the amended agreement bluebird stockholders can elect to receive either (x) the original offer of $3.00 per share in cash plus a contingent value right ("CVR") of $6.84 per share in cash payable upon achievement of a net sales milestone or (y) $5.00 per share in cash. The amended offer price provides an alternative for stockholders who would prefer greater upfront cash consideration instead of the potential upside of the CVR. Any shares tendered for which no election is made will receive the original consideration of $3.00 per share in cash and a contingent value right per share.

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The bluebird board of directors unanimously approved the amended agreement and recommends that all stockholders immediately tender their shares in support of the transaction. The bluebird board of directors continues to believe that the transaction with Carlyle and SK Capital, as amended, represents the only viable option for stockholders to receive consideration for their shares. Absent a majority of stockholders tendering, bluebird is at significant risk of defaulting on its loan agreements with Hercules Capital, and it is extremely unlikely that stockholders would receive any consideration for their shares in a bankruptcy or liquidation.

In connection with the amended agreement, the expiration date of the tender offer has been extended to expire at one minute after 11:59 p.m., New York City time, on May 29, 2025. Equiniti Trust Company, LLC, the depositary for the Offer, has advised that as of the close of business on May 13, 2025, approximately 2,281,724 shares of bluebird common stock have been validly tendered and not properly withdrawn pursuant to the Offer.

Instructions for Stockholders:

Stockholders that have previously tendered their shares and elect to receive the original offer of $3.00 per share plus a CVR do not need to re-tender their shares or take any other action in response to this extension
Stockholders that have previously tendered their shares and wish to elect to receive $5.00 per share in cash must withdraw and re-tender their shares and complete and sign the letter of election and transmittal attached to the Offer to Purchase. Detailed instructions are available in the Offer to Purchase.
Stockholders that hold shares of bluebird through a broker or other nominee may be subject to a processing cutoff that is prior to the tender deadline, so it is important to act now.
Stockholders who need assistance with tendering their shares of bluebird may contact the Information Agent, Innisfree M&A Incorporated, by calling toll-free at (877) 825-8793.
As previously announced on May 5, 2025, Carlyle and SK Capital have received all required regulatory approvals to complete the transaction, and all parties expect the transaction to be consummated promptly following the successful completion of the ongoing tender offer.

On May 15, 2025 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), a clinical-stage diabetes and obesity medicines company, reported that preliminary clinical data from the Phase I COVALENT-103 trial of BMF-500 in adults with acute leukemia (AL) were selected for a poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress, taking place June 12–15 in Milan, Italy (Press release, Biomea Fusion, MAY 14, 2025, View Source [SID1234653054]).

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The presentation will highlight emerging safety, pharmacokinetics/pharmacodynamics (PK/PD), and clinical activity of BMF-500, a covalent FLT3 inhibitor, in patients with relapsed or refractory (R/R) AL, including those with FLT3 mutations (FLT3m) who have previously received FLT3 inhibitors such as gilteritinib (gilt).

"While we have strategically shifted our internal focus to metabolic disease, the preliminary results from the COVALENT-103 study underscore the strong potential of BMF-500 in relapsed or refractory acute leukemia. Despite having received and failed multiple prior lines of therapy, the majority of treated patients experienced reductions in bone marrow blasts. Early signs of overall survival already exceed historical benchmarks, even at non-optimized dose levels," said Mick Hitchcock, Ph.D., Interim Chief Executive Officer of Biomea Fusion. "We are actively advancing partnership discussions for this very selective and active covalent binding molecule which was developed in-house for patients with very limited treatment options."

Abstract and Poster Presentation Details

Date/Time: Saturday, June 14 (18:30-19:30 CEST)
Title: Covalent FLT3 Inhibitor BMF-500 in Relapsed or Refractory (R/R) Acute Leukemia (AL): Preliminary Phase 1 Data from the COVALENT-103 Study (NCT05918692)
Poster Number: PS1520
Presenter: Farhad Ravandi-Kashani, M.D., University of Texas MD Anderson Cancer Center
Background
R/R FLT3m AL post-failure with gilteritinib (gilt) has a poor prognosis. BMF-500 is a covalent FLT3 inhibitor, potent against ITD, TKD, and resistance mutations like the gatekeeper F691. BMF-500 lacks cKIT inhibition, exhibits cytotoxicity even after washout, and elicits improved survival in FLT3m AML xenografts.

Aims
Here we update the ongoing COVALENT-103 study, an open-label Phase I study evaluating escalating doses of BMF-500 in R/R AML with or without FLT3m.

Methods
Eligible pts are adults with R/R AL ineligible for standard of care. Pts with FLT3m AL must have failed gilt in the R/R setting. Up to 33% pts may have WT FLT3. BMF-500 is dosed BID in 28-day cycles until progression/intolerability in two arms: pts not taking (Arm A) or taking (Arm B) CYP3A4 inhibitors. Expansion cohorts will obtain further safety and efficacy data at the OBD/RP2D.

Results
As of 03Feb25, 24 R/R AL pts enrolled; 4 remain on treatment. Baseline features: 8 (33.3%) females, 5 (20.8%) non-whites, mean 57 yrs (23,80), median therapies 4 (1,10), HSCT 10 (41.7%), 24 (100%) with prior venetoclax (ven). Fifteen (62.5%) had FLT3m AL; all had prior FLT3 inhibitors including gilt.

BMF-500 was well tolerated with no DLTs or discontinuations due to treatment-related toxicities, and no related QTc prolongation or cytopenias. Twenty-three pts comprised the safety population. Common TEAEs (>20%): febrile neutropenia, nausea, peripheral edema, hypokalemia, hypocalcemia, dyspnea, pleural effusion, hypoxia, hypotension (range 5-7 pts). TRAEs were Gr 1-2: vomiting, hypotension, AST elevation, hypomagnesemia, hand cellulitis, oropharyngeal pain (each 4.3%) and Gr 3-4: leukocytosis, low WBC, ALT elevation (each 4.3%). Median treatment duration was 48 d (1,170). Eleven (45.8%) pts had at least one disease assessment and were efficacy evaluable. Nine (81.8%) pts showed clinical activity: decreased BM blasts (77.8%; 1 normalized blasts, 1 >50% reduction, 5 <50% reduction), decreased peripheral blasts (33.3%; 2 complete clearance, 1 >50% reduction), 4 decreased hydroxyurea use, 4 decreased transfusions. Objective response (ELN 2017) occurred as early as end of C1, and best response as CRi by end of C2. At 100 mg BID/DL2 (Arm A), 1 of 2 FLT3m pts achieved CRi and completed six cycles; the other achieved >60% reduced BM blasts. mOS for the 23 pts is 3.48 mos (3.25, NE; 95% CI; see figure) whereas mOS for the 7 efficacy evaluable FLT3m pts has not been reached (not shown); 9 pts continue in survival follow up. The historical mOS for pts R/R to gilt/ven is 2.1 mos.

The highest levels cleared are 100 mg BID (Arm A /DL2) and 50 mg BID (Arm B /DL2), with 3 of 3 (100%) in Arm A and 4 of 5 (80%) in Arm B showing reduced BM blasts. Escalation continues at 150 mg BID/DL3 (Arm A) and 75 mg BID (Arm B/DL3).

Based on exposures surpassing the preclinical target AUC, the study pivoted from single-patient cohorts to 3+3 at 100 mg BID (Arm A) and 25 mg BID (Arm B). Exposures were comparable at these two dose levels and Plasma Inhibitory Assay showed near complete FLT3 inhibition at steady state. PK/PD showed an EC90 of 500 ng/mL, with most pts at 100 mg BID and 25 mg BID surpassing it. BMF-500 and its metabolites had similar concentrations in BM and plasma.

Summary/Conclusion
BMF-500 was well-tolerated. The majority of efficacy-evaluable pts showed reduced BM blasts, with 1 pt achieving CRi. mOS of the efficacy-evaluable FLT3m pts has not yet been reached. Enrollment is ongoing
to identify the OBD/RP2D.

Following completion of the dose escalation phase in relapsed/refractory acute leukemia patients with FLT3 mutations, Biomea plans to conclude its internal development of BMF-500 in oncology and is actively exploring strategic partnerships to advance the program.

About COVALENT-103
COVALENT-103 is a multicenter, open-label, non-randomized trial seeking to evaluate the safety and efficacy of BMF-500, a twice daily oral treatment, in adult patients with relapsed or refractory acute leukemia with FMS-like tyrosine kinase 3 (FLT3) wild-type and FLT3 mutations. The Phase I COVALENT-103 study aims to evaluate the safety and tolerability of BMF-500, determine the optimal biologic dose and recommended Phase II dose. Additional information about the Phase I clinical trial of BMF-500 can be found at ClinicalTrials.gov using the identifier, NCT05918692.

About BMF-500
BMF-500 is an investigational, orally bioavailable, covalent small molecule inhibitor of FLT3, discovered and developed in-house at Biomea using the company’s proprietary FUSION System. Designed to be highly potent and selective, BMF-500 has demonstrated encouraging potential in extensive preclinical studies. Its kinase inhibitory profile indicates strong target selectivity, which may translate to a reduced risk of off-target effects.

On May 15, 2025 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), a clinical-stage diabetes and obesity medicines company, reported that preliminary clinical data from the Phase I COVALENT-103 trial of BMF-500 in adults with acute leukemia (AL) were selected for a poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress, taking place June 12–15 in Milan, Italy (Press release, Biomea Fusion, MAY 14, 2025, View Source [SID1234653054]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The presentation will highlight emerging safety, pharmacokinetics/pharmacodynamics (PK/PD), and clinical activity of BMF-500, a covalent FLT3 inhibitor, in patients with relapsed or refractory (R/R) AL, including those with FLT3 mutations (FLT3m) who have previously received FLT3 inhibitors such as gilteritinib (gilt).

"While we have strategically shifted our internal focus to metabolic disease, the preliminary results from the COVALENT-103 study underscore the strong potential of BMF-500 in relapsed or refractory acute leukemia. Despite having received and failed multiple prior lines of therapy, the majority of treated patients experienced reductions in bone marrow blasts. Early signs of overall survival already exceed historical benchmarks, even at non-optimized dose levels," said Mick Hitchcock, Ph.D., Interim Chief Executive Officer of Biomea Fusion. "We are actively advancing partnership discussions for this very selective and active covalent binding molecule which was developed in-house for patients with very limited treatment options."

Abstract and Poster Presentation Details

Date/Time: Saturday, June 14 (18:30-19:30 CEST)
Title: Covalent FLT3 Inhibitor BMF-500 in Relapsed or Refractory (R/R) Acute Leukemia (AL): Preliminary Phase 1 Data from the COVALENT-103 Study (NCT05918692)
Poster Number: PS1520
Presenter: Farhad Ravandi-Kashani, M.D., University of Texas MD Anderson Cancer Center
Background
R/R FLT3m AL post-failure with gilteritinib (gilt) has a poor prognosis. BMF-500 is a covalent FLT3 inhibitor, potent against ITD, TKD, and resistance mutations like the gatekeeper F691. BMF-500 lacks cKIT inhibition, exhibits cytotoxicity even after washout, and elicits improved survival in FLT3m AML xenografts.

Aims
Here we update the ongoing COVALENT-103 study, an open-label Phase I study evaluating escalating doses of BMF-500 in R/R AML with or without FLT3m.

Methods
Eligible pts are adults with R/R AL ineligible for standard of care. Pts with FLT3m AL must have failed gilt in the R/R setting. Up to 33% pts may have WT FLT3. BMF-500 is dosed BID in 28-day cycles until progression/intolerability in two arms: pts not taking (Arm A) or taking (Arm B) CYP3A4 inhibitors. Expansion cohorts will obtain further safety and efficacy data at the OBD/RP2D.

Results
As of 03Feb25, 24 R/R AL pts enrolled; 4 remain on treatment. Baseline features: 8 (33.3%) females, 5 (20.8%) non-whites, mean 57 yrs (23,80), median therapies 4 (1,10), HSCT 10 (41.7%), 24 (100%) with prior venetoclax (ven). Fifteen (62.5%) had FLT3m AL; all had prior FLT3 inhibitors including gilt.

BMF-500 was well tolerated with no DLTs or discontinuations due to treatment-related toxicities, and no related QTc prolongation or cytopenias. Twenty-three pts comprised the safety population. Common TEAEs (>20%): febrile neutropenia, nausea, peripheral edema, hypokalemia, hypocalcemia, dyspnea, pleural effusion, hypoxia, hypotension (range 5-7 pts). TRAEs were Gr 1-2: vomiting, hypotension, AST elevation, hypomagnesemia, hand cellulitis, oropharyngeal pain (each 4.3%) and Gr 3-4: leukocytosis, low WBC, ALT elevation (each 4.3%). Median treatment duration was 48 d (1,170). Eleven (45.8%) pts had at least one disease assessment and were efficacy evaluable. Nine (81.8%) pts showed clinical activity: decreased BM blasts (77.8%; 1 normalized blasts, 1 >50% reduction, 5 <50% reduction), decreased peripheral blasts (33.3%; 2 complete clearance, 1 >50% reduction), 4 decreased hydroxyurea use, 4 decreased transfusions. Objective response (ELN 2017) occurred as early as end of C1, and best response as CRi by end of C2. At 100 mg BID/DL2 (Arm A), 1 of 2 FLT3m pts achieved CRi and completed six cycles; the other achieved >60% reduced BM blasts. mOS for the 23 pts is 3.48 mos (3.25, NE; 95% CI; see figure) whereas mOS for the 7 efficacy evaluable FLT3m pts has not been reached (not shown); 9 pts continue in survival follow up. The historical mOS for pts R/R to gilt/ven is 2.1 mos.

The highest levels cleared are 100 mg BID (Arm A /DL2) and 50 mg BID (Arm B /DL2), with 3 of 3 (100%) in Arm A and 4 of 5 (80%) in Arm B showing reduced BM blasts. Escalation continues at 150 mg BID/DL3 (Arm A) and 75 mg BID (Arm B/DL3).

Based on exposures surpassing the preclinical target AUC, the study pivoted from single-patient cohorts to 3+3 at 100 mg BID (Arm A) and 25 mg BID (Arm B). Exposures were comparable at these two dose levels and Plasma Inhibitory Assay showed near complete FLT3 inhibition at steady state. PK/PD showed an EC90 of 500 ng/mL, with most pts at 100 mg BID and 25 mg BID surpassing it. BMF-500 and its metabolites had similar concentrations in BM and plasma.

Summary/Conclusion
BMF-500 was well-tolerated. The majority of efficacy-evaluable pts showed reduced BM blasts, with 1 pt achieving CRi. mOS of the efficacy-evaluable FLT3m pts has not yet been reached. Enrollment is ongoing
to identify the OBD/RP2D.

Following completion of the dose escalation phase in relapsed/refractory acute leukemia patients with FLT3 mutations, Biomea plans to conclude its internal development of BMF-500 in oncology and is actively exploring strategic partnerships to advance the program.

About COVALENT-103
COVALENT-103 is a multicenter, open-label, non-randomized trial seeking to evaluate the safety and efficacy of BMF-500, a twice daily oral treatment, in adult patients with relapsed or refractory acute leukemia with FMS-like tyrosine kinase 3 (FLT3) wild-type and FLT3 mutations. The Phase I COVALENT-103 study aims to evaluate the safety and tolerability of BMF-500, determine the optimal biologic dose and recommended Phase II dose. Additional information about the Phase I clinical trial of BMF-500 can be found at ClinicalTrials.gov using the identifier, NCT05918692.

About BMF-500
BMF-500 is an investigational, orally bioavailable, covalent small molecule inhibitor of FLT3, discovered and developed in-house at Biomea using the company’s proprietary FUSION System. Designed to be highly potent and selective, BMF-500 has demonstrated encouraging potential in extensive preclinical studies. Its kinase inhibitory profile indicates strong target selectivity, which may translate to a reduced risk of off-target effects.