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on May 13, 2025, approximately 2,281,724 shares of bluebird common stock have been validly tendered and not properly withdrawn pursuant to the Offer.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 14, 2025 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor reported initiation of its Phase 1/1b open label study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of PAS-004, in adult participants with neurofibromatosis type 1 (NF1) with symptomatic and inoperable, incompletely resected, or recurrent plexiform neurofibromas (Press release, Pasithea Therapeutics, MAY 14, 2025, View Source [SID1234653071]). The study will also assess preliminary anti-tumor activity and help determine a recommended dose for subsequent Phase 2 trials. Exploratory objectives include assessing the effects of PAS-004 on cutaneous neurofibromas.

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The first active clinical trial site is the Royal North Shore Hospital in Sydney, Australia, which is expected to begin patient enrollment in Q2 2025. Additional clinical trial sites in Australia, South Korea, and the United States are expected to be opened in the coming months.

Pasithea has selected Novotech (Australia) Pty Limited as its clinical research organization (CRO) for this trial. The Company is conducting the study through its wholly owned subsidiary in Australia, Pasithea MacroMEK Pty Ltd, and anticipates eligibility for an Australian R&D Tax Incentive with a cash refund of up to 48.5% of the amount spent annually on eligible R&D activities (trial costs) in Australia.

Dr. Rebecca Brown, M.D., Ph.D. a member of Pasithea’s Scientific Advisory Board and Associate Professor of Neuro Oncology at The University of Alabama at Birmingham commented, "I am pleased to have collaborated with the Pasithea team on the design of a comprehensive dose exploration and expansion study to assess the safety and tolerability of PAS-004 in adult NF1 patients. In addition to testing the effects of PAS-004 on plexiform neurofibromas, exploratory endpoints will also examine the effects of PAS-004 on cutaneous neurofibromas. The safety profile observed to date in advanced cancer patients is encouraging, and I look forward to seeing that profile translate to the NF1 population." Dr. Brown added, "One of the biggest challenges in treating plexiform neurofibromas associated with NF1 is ensuring that patients remain on MEK inhibitor therapy over the long-term. Real-world data shows that a significant proportion of NF1 patients discontinue treatment due to poor tolerability, including high rates of rash and gastrointestinal side effects. PAS-004 is also given as a once daily dose that offers a more convenient regimen than current FDA-approved therapies that are dosed twice a day and which could improve patient compliance."

Dr. Tiago Reis Marques, Pasithea’s Chief Executive Officer, said, "Following our recent financing, including the exercise of certain warrants, Pasithea is now funded to produce initial interim patient data in NF1. The initiation of this clinical trial in NF1, the initial indication we seek FDA marketing approval for, marks an important milestone for Pasithea and for patients living with NF1-related plexiform neurofibromas. Activating our first clinical trial site underscores our commitment to advancing PAS-004 as a potential best-in-class next-generation MEK inhibitor. We are encouraged by the safety and clinical data observed to date in oncology patients and are optimistic that PAS-004’s tolerability profile will extend to the NF1 population. Importantly, our existing cancer data has enabled us to begin the NF1 trial at a higher dose than originally contemplated. In addition, we anticipate meaningful cash rebates of eligible trial costs through the Australian R&D Tax Incentive, further enhancing the efficiency of this program."

About the Phase 1/1b Clinical Trial in Adult NF1 Patients

The primary objective of the Phase 1/1b study (NCT06961565) is to evaluate the safety and tolerability of PAS-004 when administered for one 28-day treatment cycle in adult NF1 participants with at least one and up to two additional target plexiform neurofibromas (PNs) that are symptomatic and inoperable, incompletely resected, or recurrent. Secondary objectives are (i) to identify the recommended Part B dose ("RPBD") or Maximum Tolerated Dose (MTD) of PAS-004, (ii) to characterize the PK and PD profile of PAS-004, (iii) to evaluate the preliminary efficacy of PAS-004 on target PN volume, (iv) to evaluate the preliminary efficacy of PAS-004 on the size, appearance, and associated symptoms of cutaneous neurofibromas (CNs), and (v) to evaluate the impact of PAS-004 on quality of life ("QOL") and any physical symptoms attributed to the target PN. Experimental objectives are (i) to evaluate the impact of PAS-004 on QOL and any physical symptoms attributed to CNs, (ii) to evaluate the impact of PAS-004 on pain and function attributed to PNs, and (iii) to investigate PAS-004 effects on CN tumor cellular and molecular biology.

The trial will be conducted in two parts. In Part A, following a screening period of up to 28 days, up to 24 eligible participants will be enrolled sequentially to receive one of four planned dose levels of PAS-004 tablets (4mg, 8mg, 12 mg, 18mg) in a modified 3+3 design. Part A will identify the recommended RPBD. During Part B, up to 24 eligible participants will be enrolled in parallel to receive one of two planned dose levels of PAS-004 tablets. Participants will be dosed at the RPBD level and at a dose level below the RPBD for up to six continuous 28-day treatment cycles. Part B will identify the recommended phase 2 dose (RP2D).

The study is planned to be conducted at five clinical trial sites in Australia, South Korea and the U.S.

To learn more about the PAS-004 clinical trial in adults with NF1-associated plexiform neurofibromas, please visit www.clinicaltrials.gov.

On May 14, 2025 Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, reported that preclinical data will be presented on its lead program, RGT-61159, at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress, which will be held from June 12-15, 2025, in Milan, Italy (Press release, Rgenta Therapeutics, MAY 14, 2025, View Source [SID1234653094]).

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Presentation Details:

Title: RGT-61159, a Potent and Selective Small Molecule MYB RNA Inhibitor, Showed Significant Anti-Tumor Activity as Monotherapy or in Combination with Standards of Care in Several Leukemia Disease Models Harboring AML Most Common Genetic Lesions.
Presenting Author: Patricia Soulard
Session title: Poster Session 2
Session date and time: Saturday, June 14 (18:30 – 19:30 CEST)
Abstract #: PS1424

About RGT-61159
RGT-61159 is an orally available small molecule designed to specifically modulate splicing of the transcription factor MYB resulting in the inhibition of the oncogenic MYB protein and potential cell death of the cancer cells that overexpress the MYB protein. MYB acts as a master regulator of cell proliferation, self-renewal, and differentiation processes and its aberrant expression has been demonstrated in multiple forms of human cancer including adenoid cystic carcinoma (ACC), acute myeloid leukemia (AML), T-cell acute lymphoblastic leukemia (T-ALL), colorectal cancer (CRC), small cell lung cancer (SCLC) and breast cancer. Rgenta is evaluating RGT-61159 in an ongoing multi-center, open-label Phase 1a/b clinical trial in patients with advanced relapsed or refractory ACC or CRC. The Phase 1a/b study is designed to evaluate safety, tolerability, pharmacokinetics, target engagement, and clinical efficacy of RGT-61159 in patients with ACC or CRC. Additional information about the Phase 1a/b clinical trial can be accessed at ClinicalTrials.gov (NCT06462183).

On May 13, 2025 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development, and commercialization of drugs for the treatment of cancer, reported financial results for the quarter ended March 31, 2025, and provided a corporate update on its promising portfolio of clinical and pre-clinical radiopharmaceutical therapeutics (Press release, Cellectar Biosciences, MAY 13, 2025, View Source [SID1234652958]).

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"Notwithstanding the need to gather additional clinical data for iopofosine I 131, as previously announced, we believe that the Phase 2 CLOVER WaM clinical trial data for this product candidate are impressive. We plan to present these data to the EMA during the second quarter of 2025 as part of the registration package seeking conditional marketing approval. We anticipate a response regarding the regulatory pathway from the European agency before the end of the third quarter of this year," said James Caruso, president and CEO of Cellectar. "In addition to iopofosine I 131, we have developed a pipeline of radiotherapeutic candidates, including our alpha- and Auger-emitting radioconjugates, with observed preclinical activity in solid tumor models. With cash into the fourth quarter of this year we are evaluating a variety of funding pathways to successfully advance our novel pipeline assets."

Corporate Update

· Announced plans to explore full range of strategic alternatives including, but not limited to mergers, acquisitions, partnerships, joint ventures, licensing arrangements or other strategic transactions. The company’s board of directors has engaged of Oppenheimer & Co. Inc. to serve as exclusive financial advisor to assist in the strategic evaluation process.

· Determined that the Phase 3 study for iopofosine I 131, for the treatment of relapsed/refractory Waldenstrom macroglobulinemia, would be a comparator, randomized controlled study with 100 patients per arm. Study initiation is dependent upon the company obtaining additional funding or a strategic collaboration.

· Funding dependent, the company is prepared to initiate a Phase 1b/2a dose-finding study for CLR 121125, the company’s lead Auger-emitting (iodine-125) PRC, in triple-negative breast cancer. CLR 121125 is designed to provide highly precise radiotherapeutic targeting as emissions only travel a few nanometers.

· In a series of pre-clinical studies evaluating CLR 121225, the company’s lead alpha-emitting actinium-225 PRC in refractory pancreatic models, desired pharmacokinetics, biodistribution and activity were observed, further supporting future clinical development.

First Quarter 2025 Financial Highlights

· Cash and Cash Equivalents: As of March 31, 2025, the company had cash and cash equivalents of $13.9 million, compared to $23.3 million as of December 31, 2024. The company believes its cash balance as of March 31, 2025, is adequate to fund its basic budgeted operations into the fourth quarter of 2025.

· Research and Development Expenses: R&D expenses for the three months ended March 31, 2025, were approximately $3.4 million, compared to approximately $7.1 million for the three months ended March 31, 2024. The overall decrease was primarily a result of the reduction in patient follow-up activities for our CLOVER WaM Phase 2 clinical study in WM and a reduction in personnel costs.

· General and Administrative Expenses: G&A expenses for the three months ended March 31, 2025, were approximately $3.0 million, compared to approximately $4.9 million for the same period in 2024. The decrease was primarily driven by a reduction in pre-commercialization and personnel costs.

· Net Loss: The net loss attributable to common stockholders for the three months ended March 31, 2025, was $6.6 million, or $0.14 per share, compared to $26.6 million, or $0.91 per share in the three months ended March 31, 2024.

Conference Call & Webcast Details

Cellectar management will host a conference call and webcast today, May 13, 2025, at 8:30 AM Eastern Time to discuss these results and answer questions. Stockholders and other interested parties may participate in the conference call by dialing 1-800-717-1738. A live webcast of the conference call can be accessed in the "Events & Presentations" section of Cellectar’s website at www.cellectar.com. A recording of the webcast will be available and archived on the company’s website for approximately 90 days.

On May 13, 2025 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported financial results for the first quarter of 2025 (Press release, Leap Therapeutics, MAY 13, 2025, View Source [SID1234652975]).

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Leap Highlights:

· Reported positive data from the randomized, controlled Part B of the Phase 2 DeFianCe study of sirexatamab (DKN-01) plus bevacizumab and chemotherapy in second-line colorectal cancer (CRC) patients demonstrating significantly higher overall response rate (ORR) and longer progression-free survival (PFS) across DKK1-high and VEGF-naïve subgroups

· Hosted virtual key opinion leader (KOL) event featuring Zev A. Wainberg, MD, to further discuss the positive data and patient benefit from the DeFianCe study

· Presented preclinical data of FL-501, a novel GDF-15 neutralizing antibody, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2025 Annual Meeting

· Strategic restructuring to prioritize clinical development of sirexatamab in CRC and advancing FL-501 in preclinical development, resulting in an approximately 50% reduction in workforce

"In the first quarter of 2025, sirexatamab, our novel anti-DKK1 antibody, continued to demonstrate encouraging efficacy, including statistically significant higher ORR and longer PFS in both DKK1-high and VEGF-naïve second-line CRC patients. With 42 patients currently remaining on study drug, 25 in the sirexatamab arm and 17 in the control arm, we continue to be optimistic about the maturing dataset in the full population and look forward to upcoming data updates this quarter," said Douglas E. Onsi, President and Chief Executive Officer of Leap. "In this difficult market environment, we focused our resources to position Leap to advance sirexatamab in CRC and FL-501 preclinically in order to provide the greatest value for our shareholders. I would like to personally thank all of our colleagues who have been impacted by this decision and express my appreciation for their contributions and dedication to provide meaningful new treatment options to cancer patients."

DKN-01 Development Update

· Reported updated clinical data from Part B of the DeFianCe Study of sirexatamab plus bevacizumab and chemotherapy in CRC patients. In March 2025, the Company announced updated preliminary data from Part B of the DeFianCe study (NCT05480306), a Phase 2, open-label, global study of sirexatamab in combination with bevacizumab and chemotherapy (Sirexatamab Arm) compared to bevacizumab and chemotherapy (Control Arm) in patients with MSS CRC who have received one prior systemic therapy for advanced disease. Updated data from the study is expected this quarter.

o In patients with high DKK1 levels (upper quartile, n=44), the Sierxatamab Arm had a statistically significant 32% higher ORR, 3.5 month longer PFS, and OS compared to the Control Arm

o In patients who had not received prior anti-VEGF therapy (n=95), the Sirexatamab Arm had a statistically significant 22% higher ORR and 2.6 month longer PFS compared to the Control Arm, with OS not mature but favoring the Sirexatamab Arm

o With a higher number of patients remaining on study drug in the Sirexatamab Arm compared to the Control Arm (25 vs. 17), PFS in the full intent-to-treat population continues to mature with a tail population advantage for the Sirexatamab Arm

o The strong signal from the DeFianCe study supports a registrational Phase 3 clinical trial to evaluate sirexatamab plus bevacizumab and chemotherapy in second-line MSS CRC patients with high DKK1 levels or in patients who have not received prior anti-VEGF therapy

· Hosted a virtual KOL event featuring Dr. Zev Wainberg, Co-Director of the UCLA GI Oncology Program and a globally recognized leader in gastrointestinal cancer research, to discuss sirexatamab in second-line patients with advanced MSS CRC. Dr. Wainberg discussed the unmet need and how sirexatamab may improve upon the current treatment landscape for previously treated patients with advanced MSS CRC and reviewed the positive data from Part A and Part B of the Phase 2 DeFianCe study.

Pipeline Updates

· Presented new preclinical data of FL-501 at the AACR (Free AACR Whitepaper) 2025 Annual Meeting. FL-501 is a first-in-class GDF-15 neutralizing antibody targeting the cachexia pathway, a condition commonly associated with poor outcomes in cancer patients.

o In humanized FcRn mouse studies, FL-501 demonstrated a 2-3-fold longer half-life and 50% reduced clearance compared to its wild-type precursor and ponsegromab

o In mouse cachexia models using GDF-15-overexpressing colorectal cancer cells, FL-501 fully restored body composition, comparably or better than clinical-stage antibodies visugromab and ponsegromab

o In a non-small cell lung cancer patient-derived xenograft model, FL-501 effectively countered cisplatin-induced weight loss, restoring body weight, composition, and condition scores

o These findings, as well as a favorable safety profile and strong pharmacodynamic activity, support advancing FL-501 as a potentially best-in-class molecule

Business Updates

· Engaged leading financial advisor to explore business development opportunities to further the development of sirexatamab. Strong signals from the DeFianCe study supports a registrational Phase 3 clinical trial in second-line CRC, which represents a significant potential global market opportunity.

· Announced strategic restructuring to prioritize clinical focus on the development of sirexatamab in CRC. Leap is realigning its resources in order to prioritize the clinical development of sirexatamab in CRC and advancing FL-501 preclinically. As part of the strategic restructuring, the Company has reduced its workforce by approximately 50%.

Selected First Quarter 2025 Financial Results

Net Loss was $15.4 million for the first quarter 2025, compared to $13.8 million for the first quarter 2024. The increase was primarily due to an increase in research and development expenses.

Research and development expenses were $12.9 million for the first quarter of 2025, compared to $11.3 million for the same period in 2024. The increase was primarily due to an increase of $1.4 million in clinical trial costs due to the expansion of the size of Part B of the DeFianCe study and the increase in activity associated with the end of Part C of the DisTinGuish study. There was also an increase of $0.1 million in manufacturing costs related to clinical trial material and manufacturing campaigns and an increase of $0.1 million in consulting fees associated with research and development activities.

General and administrative expenses were $3.0 million for the first quarter 2025, compared to $3.5 million for the same period in 2024. The decrease was primarily due to a $0.4 million decrease in professional fees and a $0.1 million decrease in stock based compensation expense.

Cash and cash equivalents totaled $32.7 million at March 31, 2025.