Ofirnoflast (HT-6184) Receives Orphan Drug Designation from U.S. FDA for Myelodysplastic Syndromes

On October 23, 2025 Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company pioneering therapies that target the root causes of inflammation-driven diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its investigational medicine ofirnoflast (HT-6184) for the treatment of Myelodysplastic Syndromes (MDS) — a group of bone marrow disorders characterized by ineffective blood cell production and a risk of progression to acute myeloid leukemia (AML).

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The FDA grants Orphan Drug Designation to therapies intended for the treatment, prevention, or diagnosis of rare diseases or conditions that affect fewer than 200,000 people in the United States at the time of designation.

"This designation underscores the potential of our approach in Myelodysplastic Syndromes and supports our commitment to developing new treatment options for patients living with MDS," said David Bearss, PhD, Chief Executive Officer of Halia Therapeutics. "Ofirnoflast represents a first-in-class approach to modulating inflammasome biology, an upstream driver of inflammation, with the goal of restoring healthy bone marrow function."

Ofirnoflast is a selective NEK7 allosteric modulator designed to prevent the formation and promote the disassembly of the NLRP3 inflammasome, a central driver of chronic inflammation in multiple diseases. In MDS, inflammasome activation is increasingly recognized as a key contributor to ineffective hematopoiesis and bone marrow failure. By modulating NEK7, ofirnoflast aims to restore immune balance and improve blood-cell production without broad immunosuppression.

"Inflammasome biology represents a promising frontier for hematologic innovation," said Alan F. List, MD, member of Halia Therapeutics’ Scientific Advisory Board and former President and CEO of Moffitt Cancer Center. "Ofirnoflast’s approach is distinctive in that it seeks to modulate the underlying inflammatory drivers of MDS rather than just its downstream effects. This strategy has the potential to redefine how inflammation-linked bone marrow failure is treated."

Under the FDA’s Orphan Drug Act, orphan-drug status provides several incentives, including tax credits for qualified clinical testing, exemption from FDA user fees, and potential for seven years of U.S. market exclusivity upon approval. The FDA also administers grant programs to support clinical research and advance the development of therapies for rare diseases.

About Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes are a group of bone marrow disorders characterized by defective blood-cell formation, leading to anemia, infection risk, and bleeding complications. MDS primarily affects older adults and can progress to acute myeloid leukemia (AML). Current therapies, including hypomethylating agents and growth factors, often provide limited benefit and do not address the underlying inflammatory biology of the disease.

About Ofirnoflast (HT-6184)

Ofirnoflast (HT-6184) is Halia Therapeutics’ lead investigational compound and a first-in-class NEK7 modulator that regulates activation of the NLRP3 inflammasome — an upstream molecular complex involved in chronic inflammation. The drug is currently being evaluated across multiple disease areas, including:

Myelodysplastic Syndromes (MDS) – completed Phase 2 study evaluating safety and hematologic outcomes
Obesity (in combination with semaglutide) – ongoing Phase 2 study targeting adipose inflammation and metabolic dysregulation
Alzheimer’s Disease – early-stage program focused on genetically at-risk populations

(Press release, Halia Therapeutics, OCT 23, 2025, View Source [SID1234656964])

Tango Therapeutics Reports Positive Data from Ongoing Phase 1/2 Study with Vopimetostat (TNG462) in Patients with MTAP-deleted Cancers

On October 23, 2025 Tango Therapeutics, Inc. (NASDAQ: TNGX), a clinical-stage biotechnology company committed to discovering and delivering the next generation of precision cancer medicines, reported positive data from its ongoing Phase 1/2 study of vopimetostat (TNG462) in patients with MTAP-deleted cancers.

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"Today, we are presenting a substantive dataset on our lead PRMT5 inhibitor vopimetostat, which has the potential to be a turning point for treatment of multiple difficult-to-treat MTAP-del cancers, beginning with pancreatic cancer," said Barbara Weber, M.D., President and CEO of Tango Therapeutics. "Across the 16 cancer types enrolled in the trial, the ORR is 27% in patients with a median follow-up of 9.4 months, which supports the potential for vopimetostat as the best-in-class PRMT5 inhibitor. In 2L MTAP-deleted pancreatic cancer, the median PFS is 7.2 months and the ORR is 25%, more than double that observed in historical control studies, supporting our decision to initiate a pivotal trial in this patient population in 2026. With FDA alignment on the go-forward dose of 250 mg QD, we anticipate that this study will enroll rapidly, underscoring the potential for vopimetostat to be the first MTAP-selective PRMT5 inhibitor to market."

Dr. Weber continued, "Our ongoing study of vopimetostat in combination with two Revolution Medicines (RVMD) RAS(ON) inhibitors is enrolling rapidly and we anticipate sharing safety and efficacy data from that study in 2026. Today, we also announced data from the histology-agnostic cohort of the vopimetostat phase 1/2 trial, where we observed a 49% ORR and mPFS of 9.1 months (excluding sarcoma). These data from the histology agnostic cohort demonstrate the potential of vopimetostat in multiple cancers in addition to pancreatic and lung cancer and provide further optionality for clinical development in a large patient population with high unmet need. In summary, the efficacy data we have provided, supported by a favorable tolerability profile to date, reinforce the potential for vopimetostat to be the first- and best-in-class PRMT5 inhibitor for patients with a wide range of MTAP-deleted cancer types."

"Current standard of care for patients with advanced pancreatic cancer is multi-agent chemotherapy that confers modest benefit along with side effects that can adversely affect patient quality of life," said Brian Wolpin, M.D., M.P.H., Director, Gastrointestinal Cancer Center and Hale Family Center for Pancreatic Cancer Research, Dana-Farber Cancer Institute. "These initial data support the potential of vopimetostat to meaningfully change the treatment of patients with advanced pancreatic cancer by providing more durable benefit, along with a better safety and tolerability profile, and the convenience of a once-daily pill as opposed to intravenous chemotherapy."

Efficacy Results Across Study Indications:

As of September 1, 2025, 179 patients were enrolled across all histologies, with 154 at active doses (200 mg and above). Ninety-four tumor evaluable patients were enrolled more than 6 months prior to the efficacy analysis and included regardless of outcome. Across cancer types:
ORR: 27%
DCR: 78%
mPFS: 6.4 months
37/94 patients remained on treatment as of September 1, 2025.
Efficacy Results in Pancreatic Cancer Patients

As of September 1, 2025, 64 patients with pancreatic cancer were enrolled. 39 of these patients received active doses and were enrolled more than 6 months prior to the analysis; in these patients:
ORR in 2L pancreatic cancer patients: 25%
ORR for all pancreatic cancer patients: 15%
DCR for all pancreatic cancer patients: 71%
mPFS in 2L patients: 7.2 months
mPFS in 3L+ patients: 4.1 months
Median follow up: 7.8 months
Development Strategy in Pancreatic Cancer

Following consultation with the FDA on the trial design scheduled later this quarter, the company plans to start a global, randomized, pivotal study in patients with MTAP-del pancreatic cancer who have received one prior line of therapy, comparing patients treated with 250 mg QD vopimetostat to patients treated with one of four standard chemotherapy regimens. The company anticipates this study will enroll approximately 300 patients and is intended to begin enrollment in 2026.
The phase 1/2 combination study of vopimetostat with RAS(ON) multi-selective inhibitor daraxonrasib, and RAS(ON) G12D-selective inhibitor zoldonrasib (both from Revolution Medicines, RVMD), is ongoing with robust enrollment in previously treated MTAP-del/RAS mut pancreatic and lung cancer. The first dose escalation cohort of this study has been fully enrolled (n=7) and backfill is ongoing. Vopimetostat in combination with both daraxonrasib and zoldonrasib have been well-tolerated to date with exposures in the active range for each compound. The second cohort was initiated in early October. A cohort of 1L patients is expected to begin enrolling after go-forward doses have been selected. The data from this study have the potential to support a pivotal study in 1L MTAP-del/RAS mut pancreatic cancer. Initial data from the Phase 1/2 study are anticipated in 2026.
Enrollment in Lung Cancer

As of September 1, 2025, 41 patients with 2L+ lung cancer were enrolled, 12 of whom had received active doses and were enrolled more than 6 months prior to the analysis.
Emerging data are consistent with expectations, and the company anticipates providing a safety and efficacy update in 2026.
Efficacy Results in the Histology Agnostic Cohort:

As of September 1, 2025, 41 patients with 13 different cancers were enrolled at active doses and had received a first dose more than 6 months prior to the analysis. This cohort excludes pancreatic and lung cancers (being evaluated in histology-specific cohorts) and sarcomas (where no activity was observed, n=0/9 responses). In this histology agnostic cohort:
ORR: 49%
DCR: 89%
mPFS: 9.1 months
Safety and Tolerability
Consistent with previously reported data, vopimetostat is generally well tolerated at 250 mg QD, the go-forward dose agreed with the FDA, with a potentially best-in-class safety profile. The most common treatment-related adverse events (TRAEs), predominantly grade 1, were nausea (26%), anemia (20%), fatigue (19%), dysgeusia (19%) and thrombocytopenia (13%). No treatment-related Grade 4 or 5 events occurred. Grade 3 events were rare, with the exception of anemia which was observed in 13% of patients. No drug related dose discontinuations have occurred as of September 1, 2025, and only 8% of patients treated at 250 mg QD required dose reduction to 200 mg QD.

Study Design
The Phase 1/2 study (NCT05732831) is a multicenter, open-label, first in human study in solid tumor patients whose tumor has a confirmed homozygous MTAP deletion. The first part of the study was an open-label, dose escalation and the second part is an open label dose expansion/optimization in specific MTAP-deleted tumor types.

Investor Webcast and Conference Call Information
The company will host a conference call to discuss these data at 8:30 a.m. ET today, October 23, 2025. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.tangotx.com. The webcast will be available for replay for at least 30 days on the company website. Analysts who wish to join the teleconference and participate in Q&A should register here.

About Vopimetostat
Vopimetostat is a potentially best-in-class oral, once-daily, MTA-cooperative PRMT5 inhibitor that works selectively in cancer cells with MTAP (methylthioadenosine phosphorylase) deletion. MTAP deletions occur in 10-15% of all human cancers, including approximately 35% of pancreatic cancer and 15% of lung cancer. Vopimetostat is being evaluated as a monotherapy and in combination clinical studies. In ongoing clinical studies, vopimetostat has demonstrated a favorable safety and tolerability profile to date and shown durable activity in multiple tumor types.

(Press release, Tango Therapeutics, OCT 23, 2025, View Source [SID1234662278])

HUTCHMED Highlights HMPL-A251 Data Presented at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics

On October 23, 2025 HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM:​HCM; HKEX:​13) reported preclinical data for HMPL-A251 at the AACR (Free AACR Whitepaper)‑NCI‑EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), held October 22–26, 2025, in Boston, USA. HMPL-A251 is a first-in-class PI3K/AKT/mTOR ("PAM")-HER2 Antibody-Targeted Therapy Conjugate ("ATTC") comprising of a highly selective and potent PI3K/PIKK inhibitor payload linked to a humanized anti-HER2 IgG1 antibody, via a cleavable linker.

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HER2 is a well-established therapeutic target. HER2 overexpression is found in a variety of cancer types and often associated with poor prognosis. As a key downstream signaling pathway of HER2, the PAM pathway contributes significantly to the resistance against HER2-targeting treatments when altered. HMPL-A251 is innovatively designed to leverage the synergy between HER2 targeting and PAM pathway inhibition to address limitations of traditional toxin-based antibody-drug conjugates ("ADCs") and standalone PAM inhibitors.

In vitro, the PI3K/PIKK inhibitor payload exhibited high potency, selectivity, and broad anti-tumor activity across a panel of 130 tumor cell lines. By conjugating this potent payload with an anti-HER2 antibody via a hydrophilic linker, the ATTC compound HMPL-A251, upon binding to the HER2-positive target cells, undergoes rapid internalization, lysosomal trafficking, payload release, and inhibition of PAM and PIKK signaling, inducing tumor cell apoptosis. HMPL-A251 demonstrated HER2-dependent antitumor activity in vitro, potently inhibiting HER2-positive tumor cell growth regardless of PAM pathway alterations, with moderately reduced activity in HER2-low, PAM-altered cell lines. HMPL-A251 also demonstrated a bystander effect on HER2-null cells when co-cultured with HER2-positive cells.

Unlike toxin-based ADCs, which often face challenges with toxicity related to their cytotoxic payloads, ATTCs are designed to prioritize tumor-specific delivery of a pathway-modulating payload, enhancing safety for long‑term use and enabling potential frontline combinations with chemotherapy. In vivo, HMPL-A251 demonstrated superior anti-tumor efficacy and tolerability as compared to the naked antibody and payload administered together. A single intravenous dose of HMPL-A251 induced tumor regression across multiple models including HER2-positive and HER2-low models with or without PAM alteration. Efficacy correlated strongly with payload concentration and target inhibition in tumor tissue. Notably, when benchmarked against T-DXd (trastuzumab deruxtecan, a HER2‑directed ADC), HMPL-A251 achieved superior or comparable efficacy at equivalent doses in most tested models. Moreover, payload-based toxicities are expected to be low, as the plasma exposure of free payload was much lower than for HMPL-251, with a mass ratio of less than 1:500,000.

"We are excited to share the progress of HMPL-A251, the first candidate from our ATTC platform. It represents a potentially significant leap forward in addressing the limitations of toxin-based ADCs and narrow therapeutic window of systemic PAM inhibitors. By combining selective PI3K/PIKK inhibition with precise HER2 targeting, HMPL-A251 achieves potent antitumor effects while maintaining a favorable safety profile," said Dr Michael Shi, Head of R&D and Chief Medical Officer of HUTCHMED. "The compelling preclinical data presented underscore its potential to redefine treatment for a wide spectrum of cancers, and we are excited to advance HMPL-A251 as well as more ATTC drug candidates toward clinical trials."

HUTCHMED plans to initiate global clinical trials for HMPL-A251 around the end of 2025, followed by multiple global Investigational New Drug (IND) filings for more ATTC candidates in 2026.

About the ATTC platform
HUTCHMED’s Antibody-Targeted Therapy Conjugate platform represents a next-generation approach to precision oncology, combining monoclonal antibodies with proprietary small-molecule inhibitor payloads to deliver dual mechanisms of action. Unlike traditional cytotoxin-based ADCs, ATTCs combine targeted therapies to achieve synergistic anti-tumor activity and durable responses in preclinical models, outperforming standalone antibody or small-molecule inhibitor components in efficacy and safety.

Built on over 20 years of targeted therapy expertise, the platform enables development of drug candidates for diverse cancer types. By leveraging antibody-guided delivery and tumor-specific payload release, ATTCs improve the accessibility to tumors and reduce off-tumor toxicity. This overcomes challenges of traditional small-molecule inhibitors, ensures safer long-term use, and supports combinations with chemotherapy and immunotherapy, unlocking potential for early-line treatments.

(Press release, Hutchison China MediTech, OCT 23, 2025, View Source [SID1234656897])

Alligator Bioscience AB reports financial results for Q3 2025 and provides a business update

On October 23, 2025 Alligator Bioscience (Nasdaq Stockholm: ATORX), a clinical-stage biotechnology company developing tumor-directed immuno-oncology antibody drugs, reported its interim results for the third quarter of 2025 and provided a business update.

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"This quarter represented several important steps forward for Alligator, both scientifically and strategically. The final 30-month OPTIMIZE-1 results confirm mitazalimab’s unprecedented long-term survival benefit in pancreatic cancer and strengthen our conviction in its transformative potential. In parallel, we secured strong shareholder support through the successful TO 13 warrant program. These achievements position us well as we prepare for a Phase 3 trial and continue discussions with potential partners to fully realize the potential of mitazalimab and our broader pipeline."
Søren Bregenholt, CEO of Alligator Bioscience
BUSINESS UPDATE
Mitazalimab

Final OPTIMIZE-1 data: Reported on 22 September, confirming durable clinical benefit and long-term survival beyond two years — an unprecedented outcome in metastatic pancreatic cancer.
Scientific validation: 24-month results presented at ESMO (Free ESMO Whitepaper) GI; following the quarter, Cell Reports Medicine publication identified biomarkers linked to response and survival, supporting Phase 3 design.
Investigator-initiated studies: CROCOBIL (France) and APHRODITE (Italy) will further explore mitazalimab in biliary tract cancer and oral potentially malignant disorders, respectively.
HLX22

Phase 3 trial with HLX22: Henlius dosed the first U.S. patient in a global trial in HER2-positive gastric cancer — a key milestone for the program, which remains linked to potential milestone and royalty revenues for Alligator.
Partnering and innovation

RUBY agreement: Evaluation and option deal with a company in infectious diseases highlights platform potential beyond oncology.
ATOR-4066 progress: Preclinical data published in Cancer Immunology Research and presented at CICON25 confirmed its immune-modulating activity.
Conference recognition: Two abstracts for mitazalimab and ATOR-4066 accepted at SITC (Free SITC Whitepaper) 2025, underlining scientific momentum.
Company

TO 13 completed: 91.7% exercised, raising SEK 28.1 million (gross) to strengthen near-term liquidity.
Loan renegotiation: Updated agreement with Fenja Capital increases flexibility.
Enhanced visibility: Alligator hosted an R&D Day in August and presented at BioStock’s Investing in Life Science event in September.
Upcoming rights issue announced 22 October: Rights issue of approx. SEK 120 million gross (65% secured) will provide 6–9 months of financial runway during 2026, enabling Phase 3 preparations for mitazalimab and advancing strategic partnering discussions.
FINANCIAL SUMMARY FOR Q3 2025 AND YEAR-TO-DATE 2025
The financial summaries for the quarterly periods ending 30 September 2025 and 30 September 2024 are presented below.

All amounts in MSEK,
unless specified July – September
2025 July – September
2024
Net sales 0.47 1.4
Operating profit/loss -17.3 -62.0
Profit/loss for the period -12.3 -66.5
Cash flow for the period -8.8 -29.5
Cash and cash equivalents 25.1 47.8
Earnings per share before and after dilution, SEK -0.34 -87.74
The financial summaries for the year-to-date periods ending 30 September 2025 and 30 September 2024 are presented below.

All amounts in MSEK,
unless specified January – September
2025 January – September
2024
Net sales 0.47 16.0
Operating profit/loss -83.3 -169.1
Profit/loss for the period -22.3 -178.5
Cash flow for the period -38.4 -18.3
Cash and cash equivalents 25.1 47.8
Earnings per share before and after dilution, SEK -1.01 -246.01
The full report is attached as a PDF, and is also available on the company’s website: View Source

Alligator will host a webinar on Thursday, 23 October 2025, at 3 p.m. CEST/ 9 a.m. EDT for investors, analysts and media, where CEO Søren Bregenholt and CFO Johan Giléus will present and comment on the interim report, which will be followed by a Q&A session.

(Press release, Alligator Bioscience, OCT 23, 2025, View Source [SID1234656932])

INOVIO to Participate in the Stephens Biotechnology Virtual Fireside Chat Conference

On October 23, 2025 INOVIO (NASDAQ: INO), a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-associated diseases, cancer and infectious diseases, reported that it will participate in a virtual fireside chat at the upcoming Stephens Biotechnology Virtual Fireside Chat Conference. During the conference, members of INOVIO’s management team will also be conducting one-on-one meetings with investors.

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Date: November 4, 2025
Time: 3:00-3:50pm ET
Format: Virtual fireside chat

There will not be a replay or transcript of the call(s). To join the meetings or to obtain more information, please contact your Stephens representative.

(Press release, Inovio, OCT 23, 2025, View Source [SID1234656948])