On June 4, 2025 TOLREMO therapeutics AG (TOLREMO)), a clinical stage biotechnology company pioneering non-oncogene addiction in cancer, reported data from its ongoing Phase I study of TT125-802, a novel, orally administered bromodomain inhibitor of CBP/p300, for patients with advanced solid tumors who have relapsed or are refractory to standard-of-care therapies, including an abstract published at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting (Press release, TOLREMO, JUN 4, 2025, View Source [SID1234653717]). Updated data from the study was discussed at TOLREMO’s SAB meeting during ASCO (Free ASCO Whitepaper) and recorded in a virtual ASCO (Free ASCO Whitepaper) data update.

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To date TT125-802 shows impressive anti-tumor activity in advanced solid tumors, including deep and durable responses in non-small cell lung cancer (NSCLC). In addition, TT125-802 demonstrates a best-in-class safety profile without thrombocytopenia, the primary toxicity associated with its class of inhibitors.

"Five out of seven NSCLC patients on this study experienced tumor shrinkage following progression on their prior therapy. The two patients with KRAS-G12C- or EGFR-mutant NSCLC – the two tumor types we had preclinically selected as target indications – each showed deep and durable responses to single agent TT125-802. There remains a large and urgent need for more effective and tolerable therapies, and TT125-802 has the potential to offer an improved therapeutic option through resistance-targeted combinations," said Florian Vogl, M.D., PhD, Chief Medical Officer at TOLREMO.

"EGFR- and KRAS-targeted therapies have historically been limited by intrinsic and acquired resistance. Inhibiting transcriptional mechanisms of resistance via CBP/p300 represents an exciting and much needed opportunity for more effective and tolerable therapies. TT125-802 has shown impressive activity in NSCLC even as a monotherapy, and I look forward to combining the drug with EGFR and KRAS-G12C inhibitors to provide better treatment options to my patients," added Pasi Jänne, M.D., PhD, Scientific Advisory Board member at TOLREMO and Senior Vice President for Translational Medicine and the Director of the Belfer Center for Applied Cancer Science at the Dana-Farber Cancer Institute and a Professor of Medicine at Harvard Medical School.

"The best-in-class safety and activity in solid tumors is particularly notable because we didn’t set out to develop a CBP/p300 inhibitor – initially, TT125-802 was developed phenotypically to inhibit non-oncogene addiction in cancer," said Stefanie Flückiger-Mangual, PhD, CEO and co-founder of TOLREMO. "By starting with the biology around transcriptional addiction, it led us to CBP/p300 as a novel target in this space and yielded best-in-class chemistry, enabling a wider therapeutic window and broadening the clinical potential of our asset both in hematological malignancies as well as in solid tumors."

Dr. Jänne and Dr. Omar Saveedra Santa Gadea, a medical oncologist at NEXT Oncology Hospital Quirónsalud in Barcelona, Spain also shared their perspective on the importance of these results, including specific review of clinical case studies as part of an ASCO (Free ASCO Whitepaper) video update at View Source

On June 4, 2025 RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage immunology-based biopharmaceutical company focused on discovering, developing and commercializing novel therapies for patients living with inflammatory and immunological diseases, reported that members of the RAPT management team will participate in the following investor conferences in June (Press release, RAPT Therapeutics, JUN 4, 2025, https://investors.rapt.com/news-releases/news-release-details/rapt-therapeutics-participate-upcoming-investor-conferences-1 [SID1234653715]):

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Goldman Sachs 46th Annual Global Healthcare Conference – Fireside chat on Tuesday, June 10, 2025 at 9:20 a.m. ET
UBS Spring Biotech Conference – Investor one-on-one meetings on Tuesday, June 24, 2025
To access the live webcast or subsequent archived recording of the Goldman Sachs fireside chat, please visit the RAPT Therapeutics website at https://investors.rapt.com/events-and-presentations.

On June 4, 2025 Oncovita, a biotechnology company developing innovative virus-based immunotherapies for cancer treatment, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead investigational therapy, MVdeltaC, for the treatment of pleural mesothelioma—a rare and aggressive cancer with high unmet medical need and limited therapeutic options (Press release, Oncovita, JUN 4, 2025, View Source [SID1234653714]).

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MVdeltaC is a novel immunotherapy based on a genetically modified attenuated Schwarz strain measles virus, engineered to selectively replicate in tumour cells and stimulate a potent anti-cancer immune response. This approach combines direct tumor cell lysis and immune system activation, with the goal of improving outcomes for patients with advanced solid tumors, including pleural mesothelioma.

"Receiving Orphan Drug Designation from the FDA for MVdeltaC marks a major milestone for Oncovita and validates our approach of harnessing the potential of measles vaccine viruses to treat solid tumors, particularly rare and devastating cancers such as pleural mesothelioma," said Stéphane Altaba, CEO of Oncovita. "This regulatory support strengthens our strategy to advance innovative immunotherapies as we prepare to enter clinical development with MVdeltaC by 2026."

"With this designation, Oncovita is now well-positioned to enter the U.S. market with its modified attenuated measles virus for the treatment of pleural mesothelioma. This recognition highlights the promise of this novel approach against one of the most aggressive cancers in medicine," added Dr. Stéphane Champiat, MD, PhD, Head of Medical Affairs at Oncovita.

The FDA’s Office of Orphan Products Development grants Orphan Drug Designation to support the development of therapies for rare diseases affecting fewer than 200,000 people per year in the United States. This designation provides several benefits for the development of such therapies, including tax credits for eligible clinical trials, waiver of FDA application fees, and up to seven years of market exclusivity following product approval.

On June 4, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viruses,reported positive topline efficacy results from its completed U.S. Phase 1B/2 clinical trial evaluating Annamycin for the treatment of soft tissue sarcoma lung metastases ("STS lung mets") (MB-107) (Press release, Moleculin, JUN 4, 2025, View Source [SID1234653713]).

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The MB-107 trial was a multi-center, open-label, single-arm monotherapy study that in Phase 1B determined the Maximum Tolerable Dose and Recommended Phase 2 Dose ("MTD", "RP2D" respectively) and safety of Annamycin and in Phase 2 explored the efficacy of Annamycin as a single agent for the treatment of subjects with STS lung mets for which chemotherapy was considered appropriate. For more information about the MB-107 trial visit clinicaltrials.gov and reference identifier NCT04887298.

"These positive topline results from MB-107 are incredibly encouraging. The impact Annamycin demonstrated on median overall survival, particularly with patients who received multiple prior chemotherapy regimens, exceeded expectations. Additionally, the improvement seen with PFS after two doses represents a real potential for Annamycin to provide a meaningful treatment option for the treatment of STS lung mets," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "Looking ahead, we believe these results strongly support further evaluations of Annamycin for the treatment of STS lung mets and we look forward to exploring opportunities to potentially bring this important treatment option to patients."

Topline Efficacy Results Summary

Clinical Benefit Rate ("CBR") was 59.4% (n=32), comprised of 18 subjects with stable disease and 1 subject with a partial response (no subjects achieved Complete Response or CR)
Progression Free Survival ("PFS") & Overall Survival ("OS"):
Dose and regimen optimized subjects demonstrated PFS of ~4 months and OS of ~20 months
Overall (N=36) Median PFS was 63 days, with a 95% Confidence Interval ("CI") between 43 and 105 days
Median OS was 411 days, with a 95% CI between 241 and 583 days
In Phase 2 (N=17) at 330 mg/m2:
Median PFS was 105 days and OS for all-comers (median 6 prior therapies) of 13.5 months exceeded typical results for 2nd line monotherapies (8-12 months)1
OS/PFS was higher (19.9 months/127 days) for subjects with fewer prior therapies ( 2) (n=7) and receiving doses of Annamycin 330 mg/m2 (the RP2D)
Results suggest overall disease control was better at 330 mg/m2, but once combined with Phase 1B data OS and PFS was the same as overall
Subjects achieving a Partial Response ("PR") or Stable Disease ("SD") with 2 cycles also experienced higher OS/PFS (19.7 months/122 days), demonstrating that achieving CBR from Annamycin resulted in better outcomes
No cardiotoxicity demonstrated in all subject data as noted by an independent Expert
Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

On-Demand Webcast Details

An on-demand Virtual STS Lung Mets KOL Webcast to discuss the MB-107 data will be available for investors, analysts, and other interested parties on Thursday, June 5, 2025 beginning at 8:30 AM ET.

For the event, Walter Klemp, Chairman and Chief Executive Officer, and Dr. Paul Waymack, Senior Chief Medical Officer of Moleculin will be joined by Key Opinion Leaders: Mohamad Cherry, MD, Medical Director of Hematology at Atlantic Health System; Prof. Bernd Kasper, Sarcoma Unit of the Mannheim Cancer Center (MCC) at the Mannheim University Medical Center, University of Heidelberg; and Sant P. Chawla, MD, Director, Sarcoma Oncology Center, Director, Cancer Center of Southern California.

Interested participants and investors may access the on-demand video webcast on the Events page of the Investors section of the Moleculin website, moleculin.com. The webcast will be accessible for 90 days.

About STS Lung Mets

Soft tissue sarcoma is a type of cancer that originates in the soft tissues of the body, including muscles, tendons, fat, blood vessels and nerves. Lung metastases are a common occurrence in STS, and can impact overall survival. According to The American Cancer Society approximately 13,500 individuals, both adults and children, will be diagnosed with soft tissue sarcoma in 20252. Soft tissue sarcomas account for 1% of all adult cancers, 7% of cancers in children up to age 15, and 3% of cancers in children under the age of 14. The Soft Tissue Sarcoma Market was valued at USD 1.58 Billion in 2024, and is expected to reach USD 2.57 Billion by 2030, rising at a CAGR of 8.43%. OS for standard of care for metastatic STS has been estimated at 8-12 months and certain experimental targeted and cytotoxic therapies (as monotherapies or some in combination) have yielded OS of 13.4 months as second line therapy.3

MB-107 Study Design

In Phase 2, Annamycin was administered as an intravenous (IV) infusion over 2 hours on Day 1, followed by 20 days off (1 cycle = 21 days). Subjects visit the study site every 21 days (±3 days) at which time safety monitoring – including for adverse events (AEs), as well as a physical examination, laboratory evaluations (clinical chemistry, complete blood count), vital signs, weight measurements, Eastern Cooperative Oncology Group (ECOG) performance status, and electrocardiograms (ECGs) – is performed, followed by an IV infusion of study drug. Cardiac function is followed by echocardiogram (ECHO) scans at screening, at the end of the first two cycles and then following every other cycle thereafter, at the End of Treatment visit, and if feasible, during follow up at 6 months (±1 month) and 1 year (±1 month) after study drug discontinuation. As long as the Investigator considers that the benefits of treatment with Annamycin continue to outweigh the risks, treatment will continue every 21 days until tumor progression is observed or unacceptable toxicity occurs.

Tumor response is monitored every 6 weeks (±1 week) from Cycle 1 Day 1 during treatment, at the End of Treatment visit, and then every 3 months (±1 month) until disease progression using RECIST 1.1 criteria. Those subjects who leave the study after a maximum response is achieved and who do not start another therapy will be followed every 3 months (±1 month) for progression-free survival (PFS). If a subject receives further therapy after discontinuing from the study, they will be followed only for overall survival (OS) and if feasible, follow-up ECHO scans at 6 months (±1 month) and 1 year (±1 month) were conducted after study drug discontinuation.

On June 4, 2025 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported that Robert M. Davis, chairman and chief executive officer, and Dr. Dean Y. Li, executive vice president and president, Merck Research Laboratories, are scheduled to participate in a fireside chat at the 46th Annual Goldman Sachs Global Healthcare Conference on Tuesday, June 10, 2025, at 10:00 a.m. EDT (Press release, Merck & Co, JUN 4, 2025, View Source [SID1234653712]).

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Investors, analysts, members of the media and the general public are invited to listen to a live audio webcast of the presentation at this weblink.