FDA Grants Orphan Drug Designation for Tinostamustine in Malignant Glioma

On November 10, 2025 Purdue Pharma L.P. ("Purdue") reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its pipeline drug tinostamustine for the treatment of malignant gliomas, a broad category of brain and spinal cord cancers that affect both adults and children and includes rapidly growing, invasive tumors like glioblastoma. As many as 22,000 people are diagnosed with malignant gliomas annually in the United States1. FDA grants ODD status to encourage development of promising medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

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Purdue is currently investigating tinostamustine in patients with glioblastoma, the most common malignant glioma, that is aggressive, very challenging to treat and for which there is no cure.2 Most patients do not survive more than 15 months with current treatment approaches.3 The ODD request for tinostamustine was supported by robust epidemiologic, clinical and pre-clinical evidence, as well as mechanistic rationale supporting its potential to address a critical unmet medical need for glioblastoma.

"As many as 15,000 people in the U.S. are diagnosed with glioblastoma each year.3 Unfortunately, there is limited survival benefit with existing treatment options," said Julie Ducharme, Vice President and Chief Scientific Officer, Purdue. "This recognition from FDA is an important milestone in our mission of advancing innovative science in areas of serious, unmet medical need. We look forward to further investigating tinostamustine, which has shown promise in early trials."

Orphan drug designation is intended to facilitate drug development for rare diseases and may provide certain incentives to drug developers.4,5 These benefits include tax credits for qualified clinical trials, exemption from user fees including New Drug Application (NDA), and a potential for seven years of market exclusivity following approval.

Tinostamustine is a potential first-in-class, new chemical entity that combines two potentially synergistic mechanisms of action, bifunctional alkylating activity and pan histone deacetylase inhibition (or HDAC inhibition). Tinostamustine has the potential to be a first-line treatment for glioblastoma.

"Behind every designation like this are real people, patients and families, facing the devastating reality of malignant gliomas, especially glioblastoma," said Craig Landau, MD, President and CEO, Purdue Pharma. "We are deeply committed to pursuing this medicine that has the potential to bring hope where few options exist today. Tinostamustine represents a step forward in our efforts to help address the urgent and unmet needs of those affected by these aggressive cancers."

The Company also recently entered into an agreement with the Global Coalition for Adaptive Research (GCAR) to pursue the Phase 2/3 clinical development of tinostamustine in GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment – NCT03970447), a global adaptive platform trial for glioblastoma.

This press release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that tinostamustine will successfully complete development or gain FDA approval.

(Press release, Purdue Pharma, NOV 10, 2025, View Source [SID1234659749])

Genmab Announces Proposed Private Offering of Senior Secured Notes and Senior Unsecured Notes and Syndication of New Senior Secured Term Loan Facility

On November 10, 2025 Genmab A/S (Nasdaq: GMAB) ("Genmab") reported that it and its wholly owned subsidiary Genmab Finance LLC ("Genmab Finance") intend to offer, subject to market and other conditions, $1.5 billion of senior secured notes due 2032 (the "Secured Notes") and $1.0 billion of senior unsecured notes due 2033 (the "Unsecured Notes," and together with the Secured Notes, the "Notes").

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Genmab also launched the syndication of a new $2.0 billion senior secured term loan "B" facility, which term loan "B" facility is in addition to a $1.0 billion senior secured term loan "A" facility and $500 million senior secured revolving credit facility (collectively, the "New Credit Facilities") that Genmab previously syndicated to certain lenders as part of the financing for the pending acquisition (the "Acquisition") of Merus N.V. ("Merus").

Genmab intends to use the net proceeds from this offering of the Notes, together with borrowings under the New Credit Facilities and cash on hand, to fund the consideration payable in connection with the Acquisition of Merus and related fees and expenses in connection with the Acquisition, the borrowings under the New Credit Facilities and the issuance of the Notes.

Prior to the Acquisition closing, the Notes and the related guarantees from Genmab subsidiaries will be secured solely by segregated securities accounts of Genmab in which the gross proceeds of the Notes will be held. Following the purchase of all Merus common shares tendered in the previously announced tender offer by Genmab, the Secured Notes will be secured by a first priority security interest in certain assets of Genmab and its subsidiaries that will guarantee the obligations under the New Credit Facilities, in accordance with certain customary practices in the relevant jurisdictions, and subject to certain thresholds, exceptions and permitted liens. The Secured Notes will be unconditionally guaranteed on a senior secured basis and the Unsecured Notes will be unconditionally guaranteed on a senior unsecured basis by certain subsidiaries of Genmab that will guarantee the obligations under the New Credit Facilities.

The indentures governing the Notes are expected to contain customary covenants that, among other things, restrict, with certain exceptions, the ability of each of Genmab and its subsidiaries to incur additional debt, pay dividends, make certain other restricted payments, incur debt secured by liens, dispose of assets, engage in consolidations and mergers or sell or transfer all or substantially all of its assets.

The Notes will not be registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state or other securities laws and may not be offered or sold in the United States absent an effective registration statement or an applicable exemption from the registration requirements of or in a transaction not subject to the Securities Act and any state or other applicable securities laws. Accordingly, the offering of the Notes is available only to persons who are either (1) reasonably believed to be "qualified institutional buyers" as defined in Rule 144A under the Securities Act or (2) non-U.S. persons outside the United States pursuant to Regulation S under the Securities Act. The Notes will be subject to restrictions on transferability and resale and may not be transferred or resold except in compliance with the registration requirements of the Securities Act or pursuant to an exemption therefrom and in compliance with any state or other applicable securities laws.

This announcement shall not constitute an offer to sell or a solicitation of an offer to purchase any securities and shall not constitute an offer, solicitation or sale in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful. The offering of the Notes may be made only by means of an offering memorandum.

(Press release, Genmab, NOV 10, 2025, View Source [SID1234659714])

Xencor to Participate at Upcoming Investor Conferences

On November 10, 2025 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered antibodies for the treatment of cancer and autoimmune diseases, reported that company management will participate at multiple upcoming investor conferences:

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TD Cowen Immunology and Inflammation Virtual Summit
Date: Wednesday, November 12, 2025
Presentation Time: 3:00 p.m. ET / 12:00 p.m. PT
Piper Sandler 37th Annual Healthcare Conference
Date: Tuesday, December 2, 2025
Presentation Time: 2:00 p.m. ET / 11:00 a.m. PT

Live webcasts of the presentations will be available under "Events & Presentations" in the Investors section of the Company’s website located at www.xencor.com. Replays of the events will be available on the Xencor website for at least 30 days following the presentations.

(Press release, Xencor, NOV 10, 2025, View Source [SID1234659730])

Iambic Raises Over $100 Million in an Oversubscribed Round to Advance Its Portfolio of AI-Discovered Therapeutics and Leading Platform Technologies

On November 10, 2025 Iambic, a clinical-stage life science and technology company developing novel medicines using its AI-driven discovery and development platform, reported raising over $100 million in an oversubscribed financing round with balanced support from new and existing investors, including Abingworth, Alexandria Venture Investments, Alumni Ventures, ARK, Ascenta, Catalio, Everbright Biofund, Freeflow Ventures, Illumina Ventures, Mubadala, Pegasus Tech Ventures, Qatar Investment Authority, Regeneron Ventures, Sequoia, Tao Capital Partners, Terra Magnum Capital Partners, Wilson Sonsini Goodrich & Rosati, and others.

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"We are thrilled to have the support of many outstanding and committed investors who are partnering with Iambic to advance our mission of creating technologies to bring better medicines to patients," said Tom Miller, PhD, Iambic Co-Founder and CEO. "We are proud of the scientific and business progress Iambic has made across its pipeline, partnerships, and platform over this past year and view this fundraise as a testament to the exceptional work of the Iambic team. We look forward to continuing Iambic’s progress and anticipate our KIF18A and CDK2/4 programs entering the clinic as well as additional discovery and technology enablement collaborations in the near term."

The announcement of this financing closely follows Iambic’s presentation of clinical data for IAM1363 at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress, where IAM1363 demonstrated anti-tumor activity and a favorable safety profile across HER2-wild-type and HER2-mutated cancers, as well as in multiple disease indications. Subsequently, Iambic announced a research collaboration with Jazz Pharmaceuticals to evaluate combination therapy with zanidatamab with IAM1363 in patients with HER2-positive breast cancer previously treated with Enhertu.

Earlier this year, Iambic announced a novel technology-enablement collaboration with Revolution Medicines which is providing access to proprietary data and Iambic is providing access to its NeuralPLexer technology for protein-ligand structure prediction. Iambic also reported industry-leading benchmarks for Enchant, its multimodal transformer model that makes high-confidence predictions of clinical and preclinical endpoints.

(Press release, Iambic Therapeutics, NOV 10, 2025, View Source [SID1234659750])

Kelun-Biotech Presented TROP2 ADC Sacituzumab Tirumotecan Results From Multiple Clinical Studies at the 2025 CCHIO Congress

On November 9, 2025 Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd. (the "Company," 6990.HK) reported results from multiple clinical studies on its TROP2 ADC sacituzumab tirumotecan (sac-TMT) as oral presentations. From November 6 to 9, the 2025 Chinese Congress on Holistic Integrative Oncology (CCHIO) was held in Kunming, Yunnan. This conference was jointly organized by the Chinese Anti-Cancer Association (CACA), the Tengchong Science Forum Organizing Committee Office, the World Association for Integrative Oncology (WAIO), and the China Institute for Integrative Medicine Development Strategy. Academicians, experts, and scholars from across the nation gathered to focus on innovations in cancer diagnosis and treatment while sharing scientific breakthroughs.

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KL264-01/MK-2870-001

On November 7, Professor Sheng Jindong from Tianjin Cancer Hospital presented oral results on the efficacy and safety of sacituzumab tirumotecan (sac-TMT) monotherapy for locally advanced or metastatic endometrial cancer (EC) in a Phase II study at the conference. The endometrial carcinoma (EC) cohort in this study enrolled a total of 158 patients, with 114 patients assigned to the sac-TMT 4 mg/kg group and 44 patients to the sac-TMT 5 mg/kg group.

At data cutoff (May 21, 2025), median (range) follow-up was 11.7 months in the 4 mg/kg group and 21.8 months in the 5 mg/kg group. Confirmed objective response rate (ORR) was 30.7% and 34.1% in the 4 mg/kg and 5 mg/kg groups; all responses were partial response (PR). Confirmed and unconfirmed ORR was 35.1% and 36.4% in the 4 mg/kg and 5 mg/kg groups. Median duration of response (DOR) was 9.3 months and 8.7 months in the 4 mg/kg and 5 mg/kg groups. Median progression free survival (PFS) was 6.0 months and 7.3 months in the 4 mg/kg and 5 mg/kg groups.

Grade ≥3 treatment-related AEs (TRAEs) occurred in 59 patients (51.8%) and 34 patients (77.3%) in the 4 mg/kg and 5 mg/kg groups respectively; Treatment-related AEs that led to treatment discontinuation was 2 and 1 patient in each group, respectively.

In patients with advanced EC, sac-TMT monotherapy showed promising antitumor activity in the ≥2L advanced/metastatic setting and manageable safety. Both the 4 mg/kg and 5 mg/kg groups exhibited manageable safety and tolerability profiles consistent with the known safety characteristics of sac-TMT. These data led to initiation of two ongoing global Phase 3 studies of sac-TMT 4 mg/kg Q2W in advanced EC, which were sponsored and led by MSD.

OptiTROP-Lung03

On November 8, Professor Fang Wenfeng from the Cancer Center of Sun Yat-sen University delivered an oral presentation at the conference on preliminary results from a Phase II study evaluating sac-TMT in treating locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring uncommon EGFR mutations in previously treated patients. As of Dec 01, 2024, 42 patients were enrolled, including 23 patients with EGFR uncommon non-ex20ins mutations and 19 patients with EGFR ex20ins. Patients received sac-TMT 5 mg/kg Q2W until disease progression or unacceptable toxicity. After a median follow-up of 9.9 months, the ORR was 35.7% (15/42, 3 pending confirmation). The disease control rate (DCR) was 85.7%. Responses were durable with the median duration of response (mDoR) not yet reached, and the 6-month DoR rate was 90.9%. The median progression-free survival (mPFS) was 9.5 months (95% CI: 5.6, 10.9).

In the subset of patients with uncommon non-ex20ins, the ORR was 34.8% (8/23, 1 pending confirmation); the mPFS was 10.9 months (95% CI: 5.6, NE). In the subset of patients with ex20ins, the ORR was 36.8% (7/19, 2 pending confirmation); the mPFS was 9.0 months (95% CI: 2.4, NE). Grade ≥3 treatment-related adverse events occurred in 52.4% of pts. No TRAE led to treatment discontinuation or death. No cases of interstitial lung disease/pneumonitis were reported.

Sac-TMT monotherapy demonstrated promising clinical activity with a manageable safety profile in previously treated advanced NSCLC patients with uncommon EGFR mutations. These findings warrant further investigation of sac-TMT as a potential therapy for this population.

OptiTROP-Lung01

Professor Fang Wenfeng also presented results from the non-squamous cohort in the Phase II study evaluating the combination of sac-TMT and tagitanlimab (anti-PD-L1) as first-line therapy for advanced NSCLC during the oral presentation session. As of Dec 30, 2024, 81 patients with non-squamous histology were enrolled. Patients receive sac-TMT (5 mg/kg Q3W or Q2W) plus tagitanlimab (1200 mg Q3W or 900 mg Q2W) until disease progression or unacceptable toxicity. After median follow-up of 17.1 months, the confirmed ORR was 59.3%; The DCR was 91.4%; mDOR was 16.5 months (95% CI: 11.7, 22.1); mPFS was 15.0 months (95% CI: 10.8, 24.8). Among pts with PD-L1 TPS< 1%, the confirmed ORR was 47.1%; mPFS was 12.4 months (95%CI: 7.6, 15.4); while for patients with PD-L1 TPS≥ 1%, the confirmed ORR was 68.1%; mPFS was 17.8 months (95%CI: 14.5, NE). Among patients with PD-L1 TPS≥ 50%, the confirmed ORR was 77.8%; mPFS was 17.8 months (95% CI: 10.8, NE). No TRAE led to treatment discontinuation or death.

Sac-TMT in combination with tagitanlimab demonstrated promising antitumor activity in treatment-naive advanced non-squamous NSCLC. The durable clinical activities were observed regardless of PD-L1 expression. This combination therapy showed a tolerable safety profile based on known profiles of the individual agents, with no new safety signals observed.

Currently, two Phase 3 registrational studies of sac-TMT, namely (i) sac-TMT in combination with pembrolizumab (KEYTRUDA1) versus pembrolizumab for first-line treatment of patients with PD-L1 positive locally advanced or metastatic NSCLC, and (ii) sac-TMT in combination with pembrolizumab versus chemotherapy combined with pembrolizumab as first-line treatment for patients with PD-L1 negative locally advanced or metastatic non-squamous NSCLC are in progress.

With the goal of addressing patients’ urgent clinical needs, Kelun-Biotech has initiated over 30 clinical studies across multiple disease areas including lung cancer, breast cancer, and gynecological tumors. Leveraging its proprietary ADC and novel DC platform, OptiDC, the Company will continue developing innovative drugs with significant clinical value. This commitment aims to further enhance patient outcomes and contribute corporate strength to advancing the Healthy China initiative.

About Sac-TMT

Sac-TMT, a core product of the Company, is a novel human TROP2 ADC in which the Company has proprietary intellectual property rights, targeting advanced solid tumors such as NSCLC, breast cancer (BC), gastric cancer (GC), gynecological tumors, among others. Sac-TMT is developed with a novel linker to conjugate the payload, a belotecan-derivative topoisomerase I inhibitor with a drug-to-antibody-ratio (DAR) of 7.4. Sac-TMT specifically recognizes TROP2 on the surface of tumor cells by recombinant anti-TROP2 humanized monoclonal antibodies, which is then endocytosed by tumor cells and releases the payload KL610023 intracellularly. KL610023, as a topoisomerase I inhibitor, induces DNA damage to tumor cells, which in turn leads to cell-cycle arrest and apoptosis. In addition, it also releases KL610023 in the tumor microenvironment. Given that KL610023 is membrane permeable, it can enable a bystander effect, or in other words kill adjacent tumor cells.

In May 2022, the Company licensed the exclusive rights to MSD (the tradename of Merck & Co., Inc., Rahway, NJ, USA) to develop, use, manufacture and commercialize sac-TMT in all territories outside of Greater China (includes Mainland China, Hong Kong, Macau, and Taiwan).

To date, three indications for sac-TMT have been approved and marketed in China for the treatment of adult patients with unresectable locally advanced or metastatic triple negative breast cancer (TNBC) who have received at least two prior systemic therapies (at least one of them for advanced or metastatic setting), EGFR mutation-positive locally advanced or metastatic non-squamous NSCLC following progression on EGFR-TKI therapy and platinum-based chemotherapy and EGFR mutant-positive locally advanced or metastatic non-squamous NSCLC who progressed after treatment with EGFR-TKI therapy. Sac-TMT is the first TROP2 ADC drug approved for marketing in lung cancer globally. In addition, the new indication application for sac-TMT for the treatment of adult patients with unresectable locally advanced, metastatic hormone receptor positive (HR+) and human epidermal growth factor receptor 2-negative (HER2-) BC who have received prior endocrine therapy and other systemic treatments in the advanced or metastatic setting was accepted by the Center for Drug Evaluation of the NMPA, and was included in the priority review and approval process.

As of today, the Company has initiated 9 registrational clinical studies in China. MSD has initiated 15 ongoing Phase Ⅲ global clinical studies of sac-TMT as a monotherapy or with pembrolizumab or other anti-cancer agents for several types of cancer. These studies are sponsored and led by MSD.

(Press release, Kelun, NOV 9, 2025, View Source [SID1234659674])