On November 10, 2025 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs) to profoundly improve people’s lives, reported financial results for the third quarter ended September 30, 2025, and highlighted recent progress.

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"In October, we announced that Avidity entered into a definitive merger agreement with Novartis, which we believe maximizes value for our investors, accelerates the global reach of our innovative neuroscience pipeline, and advances even more possibilities for our innovative science," said Sarah Boyce, president and chief executive officer of Avidity. "This important transaction, alongside compelling del-zota data and a successful pre-BLA meeting with the FDA in the third quarter, underscores the remarkable consistency of our AOC platform and the significant potential of del-zota, del-desiran, and del-brax to transform outcomes for people living with serious rare diseases. These achievements are possible because of our incredibly talented Avidity team and the close collaboration of the dedicated patient and clinical communities we serve."

Company Announcements, Highlights and Upcoming Milestones

▪Definitive Merger Agreement with Novartis AG

•In October 2025, Avidity announced it had entered into a definitive merger agreement with Novartis AG ("Novartis") which was unanimously approved by the Boards of Directors of both Avidity and Novartis. The closing of the acquisition will follow the separation of Avidity’s early-stage precision cardiology programs into SpinCo, which is expected to be a publicly traded company. SpinCo will be led by Kathleen Gallagher, currently Avidity’s chief program officer, as chief executive officer. Sarah Boyce, currently Avidity’s chief executive officer, will serve as chair of the board.
•Novartis will acquire Avidity’s programs and pipeline in neuroscience and gain access to its differentiated RNA-targeting delivery platform, which includes three late-stage clinical development programs: delpacibart zotadirsen (del-
zota) for the treatment of Duchenne muscular dystrophy (DMD), delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1) and delpacibart braxlosiran (del-brax) for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
•Expected closing is in the first half of 2026, subject to completion of the separation of SpinCo from Avidity and other customary closing conditions.
▪Delpacibart zotadirsen (del-zota) for the treatment of people living with Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44):
•Clear path forward aligned with FDA following October 2025 pre-BLA meeting. The BLA submission is planned for 2026 for accelerated approval.
•In September 2025, Avidity shared positive topline and functional del-zota data from EXPLORE44 and EXPLORE44-OLE trials demonstrating consistent, clinically meaningful improvements across functional endpoints at approximately one year of treatment. Data demonstrated reversal of disease progression and unprecedented improvement compared to baseline and natural history across multiple functional measures. Del-zota continued to demonstrate a favorable long-term safety and tolerability profile.
•In July 2025, Avidity announced the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to del-zota.
▪Delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1):
•In July 2025, Avidity announced completion of enrollment for the Phase 3 HARBOR trial, the first global Ph3 trial of del-desiran for the treatment of DM1.
•Expected publication of data analyses from the completed Phase 1/2 MARINA trial in the fourth quarter of 2025.
•54-week topline data readout from global Phase 3 HARBOR study expected in the second half of 2026.
▪Delpacibart braxlosiran (del-brax) for the treatment of facioscapulohumeral muscular dystrophy (FSHD):
•Topline data from FORTITUDE biomarker cohort expected in the second quarter of 2026.
•Alignment with FDA on accelerated and full approval pathways for del-brax, and initiated global, confirmatory Phase 3 study, FORTITUDE-3, intended to support global approval strategy for del-brax.
•Phase 3 FORTITUDE-3 readout and global regulatory submissions expected in 2028.

▪Collaboration Progress:
•In the third quarter of 2025, Avidity received collaboration revenue of a $10.0 million clinical development milestone under Avidity’s research collaboration and license agreement with Eli Lilly and Company.
Third Quarter 2025 Financial Results

▪Cash, cash equivalents and marketable securities totaled approximately $1.9 billion as of September 30, 2025, which reflects net proceeds of $651.4 million from a public offering and $185.5 million from the sale of common stock under the Company’s sales agreement.

•The Company expects that its cash, cash equivalents and marketable securities as of September 30, 2025, will be sufficient to fund its operations to mid-2028.

▪Collaboration revenues of $12.5 million for the third quarter of 2025 and $17.9 million for the first nine months of 2025, primarily relate to a $10.0 million clinical development milestone under Avidity’s research collaboration and license agreement with Eli Lilly and Company, as well as additional revenues under Avidity’s research collaboration and license agreement with Bristol Myers Squibb. Collaboration revenues of $2.3 million for the third quarter of 2024 and $7.9 million for the first nine months of 2024, primarily relate to revenues under Avidity’s research collaboration and license agreement with Bristol Myers Squibb.

▪Research and development expenses for the third quarter of 2025 were $154.9 million, compared to $77.2 million for the same period of 2024. Research and development expenses for the nine months ended September 30, 2025 were $392.6 million, compared to $208.0 million for the same period of 2024. The increases were primarily driven by increased costs associated with the advancement of del-desiran, del-brax and del-zota, higher manufacturing costs, and higher personnel costs.

▪General and administrative expenses for the third quarter of 2025 were $46.3 million, compared to $23.3 million for the same period of 2024. General and administrative expenses for the nine months ended September 30, 2025 were $116.8 million, compared to $57.9 million for the same period of 2024. The increases were primarily due to higher personnel and commercial infrastructure costs to support the company’s expanded operations.

(Press release, Avidity Biosciences, NOV 10, 2025, View Source [SID1234659735])

On November 10, 2025 Daiichi Sankyo reported that the first patient has been dosed in a first-in-human phase 1 trial evaluating DS3610 in patients with advanced, metastatic or unresectable solid tumors.

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DS3610 is an investigational STING agonist antibody drug conjugate (ADC) containing an immunomodulatory payload discovered by Daiichi Sankyo (TSE:4568).

Despite the availability of various cancer immunotherapies, there remains an unmet need for novel treatment options with distinct mechanisms of action that may overcome resistance to current immunotherapy, enhance tumor responses and delay disease progression in solid tumors.

"By combining precise tumor targeting with an immunotherapy payload, Daiichi Sankyo is exploring a new way to harness the body’s own defenses to attack cancer," said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. "The initiation of this first-in-human trial of DS3610 represents an important step forward in advancing the next wave of our antibody drug conjugate portfolio and reaffirms our commitment to creating transformative medicines for patients with cancer."

About the Phase 1 Trial
The multicenter, open-label, first-in-human phase 1 trial will assess the safety, tolerability, preliminary efficacy and pharmacokinetics of DS3610 in patients with advanced, metastatic or unresectable solid tumors for which no additional standard therapy is available. The dose escalation trial will assess the safety and tolerability of increasing doses of DS3610 to determine the recommended doses for expansion in patients with advanced, metastatic or unresectable solid tumors.

The trial will evaluate safety endpoints including dose-limiting toxicities and adverse events. Pharmacokinetic and immunogenicity endpoints will also be assessed, as well as exploratory efficacy endpoints including objective response rate, disease control rate, duration of response, time to response, progression-free survival and overall survival.

The trial is expected to enroll patients across multiple sites globally, including Asia, Europe and North America. For more information, please visit ClinicalTrials.gov.

About DS3610
DS3610 is an investigational STING agonist ADC consisting of a monoclonal antibody with novel Fc modifications attached to an immunomodulatory payload that acts as an agonist of a stimulator of interferon genes (STING). DS3610 delivers a STING agonist payload directly to tumor environments and induces the immune system in the body to target cancer cells.

(Press release, Daiichi Sankyo, NOV 10, 2025, https://www.businesswire.com/news/home/20251110993494/en/DS3610-Enters-Clinical-Development-in-Patients-with-Advanced-Solid-Tumors-as-First-STING-Agonist-ADC-in-Industry-Leading-ADC-Portfolio-of-Daiichi-Sankyo [SID1234659752])

On November 10, 2025 AIM ImmunoTech Inc. (NYSE American: AIM) ("AIM" or the "Company") reported a recent abstract containing data from the completed Phase 2 advanced recurrent ovarian cancer clinical study utilizing Ampligen (rintatolimod), which was presented at the 40th Annual SITC (Free SITC Whitepaper) Meeting on November 7, 2025, at National Harbor, MD.

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Platinum-sensitive patients with measurable peritoneal disease were eligible for combination therapy with up to six treatment cycles at 3-week intervals of intraperitoneal cisplatin, intravenous pembrolizumab (also known as Merck’s Keytruda) and intraperitoneal Ampligen. Of the 27 patients included in the trial, 24 were evaluable for response and of those 24 there were 5 patients with complete response and 7 patients with partial response, for an Objective Response Rate ("ORR") of 50%. In contrast, a previous study titled Keynote-100 found ORRs of 7.4% and 9.9% in two arms of a pembrolizumab-only study in advanced recurrent ovarian cancer.

Read the full abstract titled "A Phase II Trial of Combination Locoregional Chemoimmunotherapy in Recurrent Platinum-Sensitive Ovarian Cancer Triggers a T Lymphotactic Response Correlating with Clinical Outcomes."

Read more about the study at ClinicalTrials.gov: NCT03734692

These new ovarian cancer study results are consistent with AIM’s belief that Ampligen can act as a synergistic agent when combined with checkpoint inhibitors. This could be especially encouraging for refractory patients whose cancers do not respond to checkpoint inhibitors alone, such as in the Keynote-189 study in non-small cell lung cancer, which found that approximately 52% of study subjects did not respond in the pembrolizumab-combination group.

"Ampligen’s demonstrated ability to enhance the effectiveness of traditional cancer treatments could make it a difference maker that dramatically improves tumor immune responses and significantly prolongs the lives of people who have otherwise few effective options for prolonged remission and even possible cure," said Robert Edwards, MD, Milton Lawrence McCall Professor and Chair, Department of Obstetrics, Gynecology and Reproductive Sciences at the University of Pittsburgh School of Medicine.

AIM’s "synergistic" patents include a U.S. patent (expires August 9, 2039) for methods involving use of Ampligen as part of a combination oncology therapy when paired with an anti-PD-L1 antibody; a patent in Japan (expires December 20, 2039) for the use of Ampligen in combination with checkpoint inhibitors (anti-PD-1 or anti-PD-L1 antibodies) for the treatment of cancer; and a patent in the Netherlands (expires December 19, 2039) for the use of Ampligen as a combination cancer therapy with checkpoint blockade inhibitors, such as Keytruda (pembrolizumab), Opdivo (nivolumab) and Imfinzi (durvalumab).

For more information about the SITC (Free SITC Whitepaper) Annual Meeting, please visit sitcancer.org.

(Press release, AIM ImmunoTech, NOV 10, 2025, View Source [SID1234659701])

On November 10, 2025 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported that the U.S. Food and Drug Administration (FDA) has completed its review of the confirmatory Phase 3 protocol for lacutamab in cutaneous T-cell lymphomas (CTCL), with no further comments, clearing the trial to proceed.

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The planned confirmatory Phase 3 trial, TELLOMAK 3, is an open-label, randomized study designed to demonstrate the efficacy of lacutamab in patients with Sézary syndrome and Mycosis fungoides, who failed at least one prior line of systemic therapy. The trial will include two independent cohorts: one enrolling patients with Sézary syndrome post-mogamulizumab treatment randomized 1:1 to receive lacutamab or romidepsin, and one enrolling patients with Mycosis fungoides randomized 1:1 to receive lacutamab or mogamulizumab. The primary endpoint of the study for both cohorts is progression-free survival (PFS) evaluated by blinded central review.

Data from the Phase 2 TELLOMAK trial in CTCL demonstrated durable activity, a favorable safety profile, and improvements in patients’ quality of life. With this feedback from FDA, the Company is progressing towards the initiation of the confirmatory Phase 3 TELLOMAK 3 trial in H1 2026. FDA provided encouraging initial feedback on Innate Pharma’s proposed regulatory pathway, which could potentially include Accelerated Approval for Sézary syndrome, once the Phase 3 trial is underway.

"This important regulatory milestone with the FDA marks a key step forward for the lacutamab program," said Jonathan Dickinson, Chief Executive Officer of Innate Pharma. "Building on robust Phase 2 data from TELLOMAK, this milestone brings us one step closer to our next goal, submitting for accelerated approval in Sézary syndrome once the Phase 3 trial is underway. We remain deeply committed to advancing this differentiated therapy for patients with CTCL, while creating meaningful value for our shareholders."

"We are pleased to reach this important milestone for the lacutamab program as we prepare to initiate the confirmatory Phase 3 study, TELLOMAK 3," said Sonia Quaratino, Chief Medical Officer of Innate Pharma. "The efficacy and safety data from the TELLOMAK Phase 2 trial suggest that lacutamab has the potential to be a game changer in the treatment of CTCL, an orphan disease with a high unmet medical need. Our expert clinical team looks forward to collaborating with CTCL investigators and regulators to start the Phase 3 trial in due time."

About lacutamab

Lacutamab is a first-in-class anti-KIR3DL2 antibody, currently developed in cutaneous T-cell lymphoma (CTCL) and peripheral T-cell lymphoma (PTCL). CTCL is a group of rare non-Hodgkin’s lymphomas that develop in the skin and severely affect patients’ quality of life. Sezary syndrome (SS) is a rare and aggressive leukemic form with poor survival, while mycosis fungoides (MF) is the most common subtype, with advanced stages associated with poor outcomes.

Data from the Phase 2 TELLOMAK trial in CTCL demonstrated durable activity, a favorable safety profile, and improvements in patients’ quality of life. FDA provided encouraging initial feedback on Innate Pharma’s proposed regulatory pathway, which could potentially include Accelerated Approval for Sézary syndrome.

(Press release, Innate Pharma, NOV 10, 2025, View Source [SID1234659717])

On November 10, 2025 Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, reported financial results for the third quarter ended September 30, 2025, and provided a review of recent accomplishments and anticipated upcoming developments.

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Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "Biohaven remains energized and focused on our mission to advance innovative medicines to patients who are waiting every day for new treatments. Our pipeline consists of multiple novel approaches for unmet medical needs and has the potential to deliver paradigm shifting treatments for conditions such as epilepsy, autoimmune disease, obesity, depression and cancer. We are particularly excited about the continued progress across our key programs and clinical-stage assets including: MoDE and TRAP degrader programs, where our two lead assets, BHV-1300 and BHV-1400, show compelling evidence to change the treatment paradigm in immune-mediated diseases; opakalim, a novel Kv7 ion channel activator, for the treatment of epilepsy and depression; and taldefgrobep alfa, myostatin-activin targeting therapy for obesity and SMA."

Dr. Coric continued, "Our late-stage clinical programs are poised to transform their respective treatment paradigms, given their novel mechanistic foundations and the body of clinical and non-clinical data generated to date. Our redirected approach to ‘right-sizing’ innovation is an important step we have undertaken to ultimately drive growth and resources to the most critical areas of our business. With this thoughtful approach to rebalancing our portfolio, we believe Biohaven remains well-positioned to execute on our commitment to transforming the treatment landscape for patients with serious and underserved diseases and we remain unwaveringly committed to delivering on our promise to advance our programs for patients, families, and shareholders in the balance of the year and in the years ahead. We will also continue to provide updates on any progress determining a path forward in SCA."

Third Quarter 2025 and Recent Business Updates

Initiated strategic cost optimization efforts across portfolio to focus forward-looking spend on three value-driving, late-stage clinical programs that will prioritize resources:
Opakalim, Kv7 ion channel activator, in pivotal studies for focal epilepsy and depression;
Lead TRAP and MoDE extracellular degraders for IgA nephropathy (BHV-1400) and Graves’ disease (BHV-1300);
Taldefgrobep alfa, myostatin-activin pathway inhibitor, for obesity and spinal muscular atrophy.
Restructuring of business priorities and optimizing resource allocation may result in either pause, delay or halting of non-priority programs.
The cost optimization efforts are expected to achieve an approximately 60% reduction in annual direct R&D spend (which excludes personnel and share-based compensation).
Expected Upcoming Milestones:

We believe Biohaven is well positioned to achieve significant, value-creating milestones in 2025 and 2026 across numerous programs:

Kv7 Activator (Opakalim):

Deliver top-line results from Phase 2 study in major depressive disorder study in 4Q 2025.
Continue two Phase 2/3 studies in focal epilepsy with initial top-line results expected in 1H 2026.
Lead TRAP and MoDE Extracellular Degraders (BHV-1400 and BHV-1300)

Continue enrollment of patients with IgAN and Graves’s disease in expanded Phase 1b and advance to pivotal studies in IgAN and Graves’ disease.
Myostatin-Activin Pathway Inhibitor (Taldefgrobep alfa):

Initiate Phase 2 clinical trial in obesity in 4Q 2025.Continue ongoing Health Authority interactions to discuss SMA registrational path in the US and Europe.
Glutamate Modulator (VYGLXIA):

We requested a Type A meeting with FDA as part of initiating an appeal process for the SCA CRL and plan to meet with the FDA to discuss potential next steps.
Capital Position:

Cash, cash equivalents, marketable securities and restricted cash as of September 30, 2025, totaled approximately $263.8 million.

Third Quarter 2024 Financial Highlights:

Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $141.2 million for the three months ended September 30, 2025, compared to $157.6 million for the three months ended September 30, 2024. The decrease of $16.4 million was primarily due to decreases in direct program spend, largely related to BHV-2000 and opakalim, which were partially offset by increased personnel costs including non-cash share-based compensation, as compared to the same period in the prior year. Non-cash share-based compensation expense was $13.9 million for the three months ended September 30, 2025, an increase of $6.8 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.

General and Administrative (G&A) Expenses: G&A expenses, including non-cash share-based compensation costs, were $28.2 million for the three months ended September 30, 2025, compared to $20.6 million for the three months ended September 30, 2024. The increase of $7.7 million was primarily due to increased non-cash share-based compensation expense and increased legal costs. Non-cash share-based compensation expense was $7.6 million for the three months ended September 30, 2025, an increase of $2.6 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.

Other (Expense) Income, Net: Other (expense) income, net was other expense, net of $3.8 million for the three months ended September 30, 2025, compared to other income, net of $17.8 million for the three months ended September 30, 2024. The decrease of $21.6 million was primarily due to an increase in non-cash losses related to changes in fair value of our notes payable liability under the Note Purchase Agreement with Beetlejuice SA LLC, an affiliate of Oberland Capital Management LLC, entered into during the second quarter of 2025 (the Note Purchase Agreement), a decrease in gains recorded for the non-cash changes in the fair value of our forward contracts and derivative liabilities recorded in connection with the amendment to our Membership Interest Purchase Agreement with Knopp Biosciences LLC in May 2024 (the Knopp Amendment), and decreased investment income.

Net Loss: Biohaven reported a net loss for the three months ended September 30, 2025 of $173.4 million, or $1.64 per share, compared to $160.3 million, or $1.70 per share, for the same period in 2024. Non-GAAP adjusted net loss for the three months ended September 30, 2025 was $155.9 million, or $1.47 per share, compared to $164.1 million, or $1.74 per share, for the same period in 2024. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges and losses from the change in fair value of derivatives. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.

(Press release, Biohaven Pharmaceutical, NOV 10, 2025, View Source [SID1234659737])