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on May 13, 2025, approximately 2,281,724 shares of bluebird common stock have been validly tendered and not properly withdrawn pursuant to the Offer.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]).

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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.

On May 14, 2025 Azitra, Inc. (NYSE American: AZTR), a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, reported that it will present a poster describing the Phase 1/2 clinical trial of ATR04-484 for EGFR inhibitor ("EGFRi")-associated rash at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) (Press release, Azitra, MAY 14, 2025, View Source [SID1234653053]). ASGCT (Free ASGCT Whitepaper) is being held May 13-17, 2025, in New Orleans, Louisiana.

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Details of the presentation are as follows:

Event:

American Society of Gene and Cell Therapy

Location:

New Orleans Ernest N. Morial Convention Center, New Orleans, Louisiana

When:

May 15, 2025; 5:30-7:00PM CT

Title:

A Novel Staphylococcus epidermidis Compound for the Topical Treatment Epidermal
Growth Factor Receptor (EGFR) Inhibitor-Induced Dermal Toxicity

Presenter:

Mary Spellman, MD, Chief Medical Officer

Registration:

View Source

"The Annual Meeting of ASGCT (Free ASGCT Whitepaper) is an important conference for researchers and industry leaders who are seeking to develop next-generation cell and gene therapies for patients in need," said Mary Spellman, MD, Chief Medical Officer of Azitra. "We are excited to present supporting evidence for the clinical development of topical ATR04-484 and our Phase 1/2 clinical study design, in anticipation of initiating dosing for the first patient in the coming month."

On May 14, 2025 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported new positive data from its pivotal Phase 2 iMMagine-1 study of anitocabtagene autoleucel (anito-cel), in patients with relapsed or refractory multiple myeloma (RRMM) (Press release, Arcellx, MAY 14, 2025, View Source [SID1234653052]). These data will be presented during an oral presentation at the EHA (Free EHA Whitepaper)2025 Congress in Milan on June 14, 2025. Anito-cel is partnered with Kite, a Gilead Company.

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The Phase 2 iMMagine-1 data are from a May 1, 2025 data cutoff date, including all 117 patients with a median follow-up of 12.6 months and a minimum follow-up of four months after treatment with anito-cel. All patients received a single infusion of anito-cel (target dose of 115×106 CAR+ viable T cells). 101 of 117 patients (86%) were triple refractory, and 48 of 117 patients (41%) were penta refractory. Patients received a median of three prior lines of therapy, with 60 of 117 patients (51%) having received three prior lines.

Overall response rate (ORR) was 97% (114/117) with a complete response/stringent complete response (CR/sCR) rate of 68% (79/117) and a very good partial response or higher (≥VGPR) rate of 85% (100/117), per International Myeloma Working Group (IMWG) criteria as investigator-assessed. Of those evaluable for minimal residual disease (MRD) testing at the time of this data cut, 93.3% (70/75) achieved MRD negativity at a minimum of 10-5 sensitivity. Six-month progression-free survival (PFS) and overall survival (OS) rates were 91.9% and 96.6%, respectively, and 12-month PFS and OS rates were 78.8% and 95.2%, respectively. Median PFS and median OS have not been reached.

No delayed or non-immune effector cell-associated neurotoxicity syndrome (ICANS) neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome, and no immune-mediated enterocolitis have been observed to date with anito-cel. No additional treatment- or therapy-related deaths or Grade ≥3 cytokine release syndrome (CRS) or ICANS events have occurred since the previous data presentation in December 2024.

Conclusions

Preliminary results from the Phase 2 iMMagine-1 study demonstrate deep and durable responses with a predictable and manageable safety profile in a fourth-line or higher (4L+) RRMM population, including triple- and penta-class refractory disease. Notably, no delayed or non-ICANS neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome, and no immune-mediated enterocolitis have been observed with anito-cel to date.

"These clinical data from our registrational study continue to support our belief that anito-cel has the potential to address the needs of myeloma patients and the physicians who serve them," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. "There is no cure for multiple myeloma. We believe there remains an unmet medical need for CAR-T therapies that are efficacious, safe, and accessible. Anito-cel has the unique potential to address these needs thanks to our differentiated technology, our incredible and entrepreneurial team, the robust clinical data generated to date, and our strong partnership with Kite. Our 2026 commercial launch plans for anito-cel with our partners at Kite are well underway and we are excited for the opportunity to advance anito-cel in support of the myeloma community. We look forward to sharing these data with the clinical community at EHA (Free EHA Whitepaper) and are honored that the iMMagine-1 data will be presented during an oral presentation on Saturday, June 14."

EHA2025 Presentation Details

Phase 2 Registrational Study of Anitocabtagene Autoleucel for the Treatment of Patients with Relapsed and/or Refractory Multiple Myeloma: Preliminary Results from the iMMagine-1 Trial

Speaker: Gurbakhash Kaur, M.D., Assistant Professor of Internal Medicine, Mount Sinai Health System

Session Title: s431 Treatment of relapsed and/or refractory multiple myeloma (RRMM)

Session Date: June 14, 2025

Session Time: 17:00-18:15 CEST

Publication Number: S201

Presentation Title: S201 Phase 2 Registrational Study of Anitocabtagene Autoleucel for Relapsed and/or Refractory Multiple Myeloma (RRMM): Updated Results from iMMagine-1

About Multiple Myeloma

Multiple Myeloma (MM) is a type of hematological cancer in which diseased plasma cells proliferate and accumulate in the bone marrow, crowding out healthy blood cells and causing bone lesions, loss of bone density, and bone fractures. These abnormal plasma cells also produce excessive quantities of an abnormal immunoglobulin fragment, called a myeloma protein (M protein), causing kidney damage and impairing the patient’s immune function. MM is the third most common hematological malignancy in the United States and Europe, representing approximately 10% of all hematological cancer cases and 20% of deaths due to hematological malignancies. The median age of patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years. Because MM tends to afflict patients at an advanced stage of life, patients often have multiple co-morbidities and toxicities that can quickly escalate and become life-endangering.

About Anitocabtagene Autoleucel (anito-cel)

Anitocabtagene autoleucel (anito-cel, previously ddBCMA) is the first BCMA-directed CAR T-cell therapy to be investigated in multiple myeloma that utilizes Arcellx’s novel and compact binder known as the D-Domain. The small, stable D-Domain binder enables high CAR expression without tonic signaling and is designed to quickly release from the BCMA target. This combination may allow for the effective elimination of multiple myeloma cells without severe immunotoxicity. Anito-cel has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy Designations by the U.S. Food and Drug Administration.