On July 15, 2025 Pierre Fabre Pharmaceuticals, Inc. reported the transfer of the Investigational New Drug Application for tabelecleucel from its partner, Atara Biotherapeutics, Inc. (Nasdaq: ATRA) (Press release, Pierre Fabre, JUL 15, 2025, View Source [SID1234654391]). "This announcement marks an important milestone as Pierre Fabre Laboratories assumes global responsibility for clinical development of tabelecleucel following transfer of manufacturing responsibilities earlier in the year," said Adriana Herrera, Chief Executive Officer of Pierre Fabre Pharmaceuticals., the Pierre Fabre Laboratories pharmaceutical subsidiary in the United States. "Ongoing recruitment into the ALLELE study demonstrates our commitment to clinical research and development as we await regulatory action on the BLA resubmitted by our partner Atara."

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Atara Biotherapeutics resubmitted the tabelecleucel BLA on July 11, having rapidly addressed the third-party manufacturing facility observations outlined in the January 2025 Complete Response Letter in collaboration with Pierre Fabre Pharmaceuticals (PFP). On March 31, PFP assumed global responsibility for tabelecleucel manufacturing, including commercial product supply for European markets where the innovative cell therapy is already approved, as well as for global clinical trial supply. On July 14, 2025, Atara Biotherapeutics transferred the tabelecleucel IND to Pierre Fabre Medicament, a subsidiary of Pierre Fabre Laboratories.

The two studies currently open for tabelecleucel access in the US are:

NCT03394365: Tabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Participants with Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease (EBV+ PTLD) After Failure of Rituximab or Rituximab and Chemotherapy (ALLELE)

The ALLELE study is investigating the effects of tabelecleucel, an off-the-shelf allogeneic Epstein-Barr virus (EBV)-specific cytotoxic T cell therapy, to treat relapsed/refractory EBV-associated post-transplant lymphoproliferative disease arising after SOT or HCT.

NCT04554914: A Study to Evaluate Tabelecleucel in Participants with Epstein-Barr Virus-associated Diseases

This study is a multicenter, multicohort, open-label, single-arm, Phase 2 study investigating the efficacy and safety of tabelecleucel for the treatment of EBV-associated diseases.

Tabelecleucel is an allogeneic, off-the-shelf, EBV-specific T-cell immunotherapy designed to selectively target and eliminate EBV-infected cells. Unlike autologous CAR-T therapies, allogeneic T-cells are derived from third party donors and are not genetically modified. Immune cells are collected from the blood of healthy donors and exposed to Epstein-Barr virus antigens to help enrich for T cells that recognize EBV. These EBV T cells are expanded, characterized, kept alive and stored for future use to treat patients.

Tabelecleucel was granted marketing authorization under the brand name EBVALLO in December 2022 by the European Commission (EC) as a monotherapy for the treatment of adult and pediatric patients two years of age and older with r/r EBV+ PTLD who have received at least one prior therapy.

In December 2023, Atara announced an expanded global partnership with Pierre Fabre Laboratories for the U.S. and remaining global commercial markets for tabelecleucel, building on an initial partnership covering Europe, Middle East, Africa, and other select emerging markets.

About EBV+PTLD

EBV+ PTLD is an ultra-rare, acute, and potentially deadly hematologic malignancy that occurs after transplantation when patent T-cell immune responses are compromised by immunosuppression. It can impact patients who have undergone solid organ transplant (SOT) or allogeneic HCT. Poor median survival of 3 weeks and 4.1 months for HCT and SOT, respectively, is reported in EBV+ PTLD patents for whom standard of care failed, underscoring the significant need for new therapeutic options.

On July 15, 2025 Adagene Inc. ("Adagene or the Company") (Nasdaq: ADAG), a company transforming the discovery and development of novel antibody-based therapies, reported outcomes from its Type B meeting with the United States Food and Drug Administration (FDA). Adagene has received written feedback from the FDA on its clinical development plan to evaluate muzastotug (ADG126) in combination with Merck’s (known as MSD outside of the US and Canada) anti-PD-1 therapy, KEYTRUDA (pembrolizumab: 200 mg, Q3W) in patients with microsatellite stable colorectal cancer (MSS CRC) (Press release, Adagene, JUL 15, 2025, View Source [SID1234654376]).

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ADG126 is a masked, anti-CTLA-4 SAFEbody that targets a unique epitope of CTLA-4 in regulatory T cells (Tregs) in the tumor microenvironment. ADG126 is currently in phase 1b/2 clinical studies in combination with anti-PD-1 therapy, particularly focused on MSS CRC. To date, over 150 patients have received ADG126, either as monotherapy or in combination with anti-PD-1 therapy. Adagene has completed patient enrollment for the MSS CRC dose expansion cohorts of the ADG126 + pembrolizumab combination (NCT05405595). The phase 2 trial will be conducted primarily to identify which of the two doses of ADG126 will be used in the phase 3 trial.

Key Outcomes from the FDA Type B Meeting

● Patient Population: Future trials will enroll late-line patients with MSS CRC without liver metastases, including those with peritoneal metastasis/involvement.

● Dose and Regimen: Phase 2 will randomize patients to either 10 mg/kg or 20 mg/kg of ADG126 in combination with pembrolizumab, using an induction-maintenance regimen, without cycle limitations of ADG126.

● Phase 2 Trial Design: Approximately 30 patients will be enrolled in each arm of the Phase 2 study, without a requirement for an ADG126 monotherapy arm.

● Phase 3 Trial Design: The FDA agreed with Adagene’s proposed standard-of-care (SOC) control arm for the Phase 3 clinical trial and confirmed that an ADG126 monotherapy arm is also not required in the pivotal study.

● Phase 2 Endpoints: The primary endpoint of the Phase 2 trial will be overall response rate (ORR). Secondary endpoints include duration of response (DOR), progression-free survival (PFS), and overall survival (OS).

● Phase 3 Endpoints: The primary endpoint of the Phase 3 trial will be OS. Secondary endpoints will include PFS, DOR and ORR.

"Receiving the FDA’s endorsement to explore ADG126 at doses 10 to 20 times higher than other anti-CTLA-4 antibodies, without limitations on treatment cycles, has demonstrated a nearly two-fold increase in ORR from 17% at 10 mg/kg to 29% at 20 mg/kg, while Grade 3 treatment-related adverse events (TRAEs) remain approximately 20% at comparable drug exposures for ADG126 at 10 mg/kg Q3W versus 20 mg/kg Q6W. These results highlight the wide therapeutic index and favorable safety profile we have seen to date in the clinic" said Dr. Marwan Fakih, Professor of Medical Oncology and Therapeutics Research at City of Hope.

Peter Luo, Ph.D., CEO and President of R&D at Adagene added, "Our Phase 1b/2 trial has produced robust dose-dependent efficacy data with a potential best-in-class safety profile, identifying two optimized dose levels for the Phase 2, consistent with Project Optimus. With the FDA’s endorsement to advance into the Phase 2 part of the clinical trial, we’d like to thank our strategic partner, Merck, for supplying pembrolizumab to support this exciting randomized dose optimization cohort to identify a single dose to take into Phase 3, a critical step towards registrational trial for FDA approval."

On July 15, 2025 GT Medical Technologies, Inc., a medical device company dedicated to improving the lives of patients with brain tumors, reported the company has completed the close of its oversubscribed $53 million Series D equity financing (Press release, GT Medical Technologies, JUL 15, 2025, View Source [SID1234654392]).

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The final close of the Series D comprised an additional $16 million from new investors including FemHealth Ventures, Warren Point Capital, an undisclosed strategic investor, and increased commitments from all existing institutional investors. Earlier this year, GT Medical Technologies announced the completion of a $37 million first close of the Series D equity financing led by Evidity Health Capital, alongside new investor Accelmed Partners and existing investors MVM Partners, Gilde Healthcare, and Medtech Venture Partners.

"Increasing the size of the Series D allows us to stay focused on driving results for patients into the foreseeable future," said Per Langoe, Chief Executive Officer at GT Medical Technologies. "We are thankful for our investors’ continued support as we expand our commercial footprint and clinical initiatives for GammaTile."

The company will use the funds to drive the expansion of its U.S. commercial activities for GammaTile. In addition, the financial resources will enable GT Medical Technologies to complete enrollment of its ROADS randomized controlled trial for newly diagnosed brain metastases and will fund an additional randomized control trial of GammaTile in newly diagnosed glioblastoma cases.

"FemHealth Ventures is excited to join GT Medical Technologies as an investor and support the commercial acceleration of GammaTile Therapy," stated Maneesha Ghiya, Managing Partner at FemHealth Ventures. "We are proud to back a company that is transforming the standard of care in oncology and shares our commitment to improving health outcomes, including meaningful applications in women’s health."

GammaTile is an innovative form of radiation therapy placed at the time of brain tumor removal surgery, delivering immediate, targeted radiation to the tumor site when cancer cells are at their lowest levels. Unlike conventional approaches, which often require a delay between surgery and radiation therapy to allow for wound healing, GammaTile eliminates this treatment gap. By delivering immediate, localized radiation directly at the tumor site, it is designed to maximize the treatment’s effectiveness against remaining cancer cells to reduce the risk of regrowth.1

"The completion of an oversubscribed Series D equity financing from premier medical device investors is a testament to the important work of our team and mission," added James Leech, Chief Financial & Strategy Officer of GT Medical Technologies.

Recent GT Medical Technologies Milestones

GT Medical Technologies recently celebrated important milestones, reflecting the growing adoption of GammaTile. To date, more than 1,900 patients have been treated with GammaTile Therapy, and over 800 patients have been enrolled in GammaTile clinical studies.

Looking Ahead

GT Medical Technologies anticipates completing enrollment in both the ROADS and GESTALT studies in Q3 2025.

On July 15, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (TSX: APS; OTC: APTOF), a clinical-stage precision oncology company developing a tuspetinib (TUS) based triple drug frontline therapy to treat patients with newly diagnosed acute myeloid leukemia (AML), reported that it has received an additional advance of US$2.0M from Hanmi Pharmaceutical Co. Ltd. ("Hanmi"), as part of a US$8.5M loan facility agreement with Hanmi (the "Loan Agreement") announced prior on June 20, 2025 (Press release, Aptose Biosciences, JUL 15, 2025, View Source [SID1234654377]). To date, Aptose has received an aggregate of US$4.5M under the Loan Agreement.

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"Tuspetinib in combination with venetoclax and azacitidine (the TUS+VEN+AZA triplet) continues to demonstrate exciting antileukemic activity and safety across genetically diverse populations of newly diagnosed AML patients – including TP53-mutated AML and wildtype AML, representing large AML populations for which there are few treatment options," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose. "We are very grateful to Hanmi for its continued support to help advance tuspetinib clinical development and further extend Aptose’s ability to fund this important study."

Tuspetinib is a convenient once daily oral agent that potently inhibits targets that drive excessive proliferation and anti-apoptotic mechanisms, including SYK, mutated and wild type forms of FLT3, mutated KIT, JAK1/2, and RSK2 kinases. Yet, TUS maintains a favorable safety profile by avoiding typical toxicity concerns observed with other agents. The ongoing TUSCANY triplet Phase 1/2 study is designed to test various doses and schedules of TUS in combination with standard dosing of azacitidine and venetoclax in newly diagnosed patients with AML who are ineligible to receive induction chemotherapy. At the European Hematology Association (EHA) (Free EHA Whitepaper) Congress in June, Aptose reported early data from the first two dose cohorts that have demonstrated safety, CRs and minimal residual disease (MRD) negativity across patients with diverse mutations.

On July 15, 2025 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a clinical-stage precision oncology company, reported it has entered into an exclusive worldwide licensing agreement with Debiopharm International S.A. ("Debiopharm"), a privately-owned, Swiss-based biopharmaceutical company aiming to establish tomorrow’s standards of care to cure cancer and infectious diseases, for lunresertib, a first-in-class precision oncology PKMYT1 inhibitor (Press release, Repare Therapeutics, JUL 15, 2025, View Source [SID1234654393]).

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"The exclusive worldwide licensing agreement with Debiopharm allows for the continued development of lunresertib, a novel PKMYT1 inhibitor, that has demonstrated encouraging results across multiple clinical trials in difficult-to-treat solid tumors. This agreement builds upon the success of Repare and Debiopharm’s existing collaboration studying the combination of lunresertib and Debio 0123," said Steve Forte, President, Chief Executive Officer and Chief Financial Officer of Repare. "Our recent business development efforts have continued to enable Repare to focus on the advancement of our clinical priorities and sustained value creation. We remain focused on two ongoing Phase 1 clinical trials with readouts expected in the second half of 2025: the LIONS trial evaluating our RP-1664 PLK4 inhibitor and the POLAR trial evaluating our RP-3467 Polθ ATPase inhibitor."

Under the terms of the agreement, Repare will receive a $10 million upfront payment, and is eligible to receive up to $257 million in potential clinical, regulatory, commercial and sales milestones, including up to $5 million in potential near-term payments, and single-digit royalties on global net sales. Repare and Debiopharm entered into a clinical study and collaboration agreement in January 2024 to explore the synergy between lunresertib and Debio 0123, a potential best-in-class, brain penetrant and highly selective WEE1 inhibitor. Debiopharm will assume sponsorship of the MYTHIC study and take over existing and future development activities related to lunresertib.

"We are excited to enter into this worldwide license agreement with Repare for lunresertib. Based on very promising Phase 1/1b clinical data, we believe the combination of lunresertib and Debio 0123 is highly synergistic and could potentially drive rapid and deep tumor regressions," said Bertrand Ducrey, CEO of Debiopharm. "We believe the synthetic lethality approach of lunresertib in combination with Debio 0123 will allow us to bring this innovative precision therapy to patients with difficult to treat cancers."

Continued Prioritization of RP-3467 and RP-1664

Moving forward, Repare will remain focused on the advancement of its two ongoing Phase 1 clinical trials, POLAR and LIONS. The POLAR clinical trial is a multicenter, open-label, dose-escalation Phase 1 clinical trial designed to investigate the safety, pharmacokinetics, pharmacodynamics, and preliminary clinical activity of RP-3467, a small molecule inhibitor of polymerase theta (Polθ) that is a synthetic lethality target associated with BRCA mutations and other genomic alterations, alone or in combination with olaparib in adults with locally advanced or metastatic epithelial ovarian cancer, metastatic breast cancer, metastatic castration-resistant prostate cancer, or pancreatic adenocarcinoma. Topline safety, tolerability and early efficacy data from the Phase 1 POLAR clinical trial of RP-3467 alone and in combination with olaparib is expected in the third quarter of 2025. The LIONS clinical trial is a first-in-human, multicenter, open-label Phase 1 clinical trial designed to investigate safety, pharmacokinetics, pharmacodynamics and the preliminary efficacy of RP-1664, a first-in-class, highly selective, oral inhibitor of Polo-like kinase 4 (PLK4) that is a synthetic lethality target associated with TRIM37 overexpression. Initial topline safety, tolerability and early efficacy data from the Phase 1 LIONS clinical trial of RP-1664 is expected in the fourth quarter of 2025.