On May 11, 2026 Ranok Therapeutics, a clinical-stage biotechnology company developing innovative therapies, reported the publication of preliminary clinical results from its Phase 1a study of RNK08954 in the peer-reviewed journal Cancer Discovery. RNK08954 is a proprietary, highly selective, oral small-molecule inhibitor targeting KRAS G12D mutation in patients with advanced solid tumors.

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The study evaluated the safety, tolerability, and clinical activity of RNK08954 in patients harboring KRAS G12D mutation across multiple sites in China. The study enrolled patients with advanced solid tumors, primarily focusing on safety and the determination of the Recommended Dose for Expansion. A total of 36 patients were evaluable for clinical activity. The overall objective response rate (ORR) was 28%, with a disease control rate (DCR) of 86%. Notably, patients with non-small cell lung cancer (NSCLC) achieved an ORR of 58.33% and a DCR of 100%. RNK08954 was generally well tolerated, with treatment-related adverse events consisting predominantly of Grade 1-2 gastrointestinal adverse events and decreased appetite. No dose-limiting toxicities were observed during the dose-escalation phase. [1]

"I am grateful that the editors of Cancer Discovery selected our study for publication," said Professor Song Zhengbo, Director of Phase I Clinical Trial Unit at Zhejiang Cancer Hospital, and the study’s Principal Investigator. "RNK08954 represents a critical step in advancing KRAS G12D-targeted therapy from concept to clinical validation. These findings not only accelerate the clinical translation of precision oncology but also lay a solid data foundation for subsequent pivotal clinical studies."

"The publication of these data underscores the potential for RNK08954 to provide a meaningful option for KRAS G12D-mutant cancers," commented Dr. Iman Elhariry, Chief Medical Officer at Ranok Therapeutics and co-author of the article. "Seeing a 58.33% objective response rate in the NSCLC cohort is particularly encouraging as it validates our approach of targeting the Switch II pocket with high selectivity. Based on these strong signals of clinical activity and the clean safety profile, we have already initiated our Phase 1b expansion study to further explore the drug’s potential as a monotherapy and in combination regimens across NSCLC, pancreatic and other indications."

Dr. Weiwen, Founder and CEO of Ranok Therapeutics, added: "The significant progress in KRAS inhibitors has been made possible by over a decade of accumulated knowledge in structural biology and medicinal chemistry. These clinical research achievements will inspire our team to further explore the clinical potential of RNK08954, fully unlocking its value for patients who currently lack effective targeted therapies."

About RNK08954 and KRAS G12D

KRAS G12D is one of the most prevalent oncogenic drivers in solid tumors, including pancreatic ductal adenocarcinoma (PDAC), colorectal cancer (CRC), and non-small cell lung cancer (NSCLC). For decades, this mutation was considered "undruggable". RNK08954 is designed to bind directly to the Switch II pocket of the KRAS G12D protein in its active and inactive states, effectively blocking downstream signaling pathways that drive tumor growth.

(Press release, Ranok Therapeutics, MAY 11, 2026, View Source [SID1234665456])

On May 11, 2026 Aktis Oncology, Inc. (NASDAQ:AKTS) (Aktis or the Company), a clinical-stage oncology company focused on expanding the breakthrough potential of targeted radiopharmaceuticals to large populations, including those not addressed by existing platform technologies, reported financial results and business highlights for the first quarter ended March 31, 2026.

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"We continue to build momentum toward delivering a new class of radiopharmaceuticals targeting tumor types with large patient populations, leveraging our differentiated miniprotein radioconjugate platform and patient-first end-to-end supply chain," said Matthew Roden, Ph.D., President and Chief Executive Officer of Aktis Oncology. "Last week, we announced the initiation of our Phase 1b clinical trial of AKY-2519 in patients with mCRPC. This is the first of two trials in our clinical development strategy designed to expand the breadth of tumors studied and augment speed to data, with preliminary data from the mCRPC trial anticipated in 2027. We plan to initiate a second Phase 1b basket trial of AKY-2519 in additional solid tumors in the second half of this year. We also look forward to presenting our first AKY-2519 clinical imaging and dosimetry data for AKY-2519 at ASCO (Free ASCO Whitepaper), which informed our clinical development strategy."

Dr. Roden continued, "AKY-2519 marks the second program we have advanced from our proprietary miniprotein radioconjugate platform to the clinic in the last twelve months. In parallel, we continue to enroll patients in our ongoing Phase 1b trial of AKY-1189 targeting Nectin-4 expressing tumors, with preliminary data expected in the first quarter of 2027. We remain focused on generating clinical data intended to support advancement of both programs and maximizing the potential clinical benefit for patients."

Q1 and recent business highlights

AKY-1189 highlights

AKY-1189 is a novel, clinical-stage miniprotein radioconjugate designed to selectively deliver actinium-225 (225Ac) to Nectin-4 expressing tumors.

Received Fast Track designation from the U.S. Food and Drug Administration (FDA) for AKY-1189 for the treatment of adult patients with locally advanced or metastatic urothelial cancer (mUC) who have progressed on or after prior systemic therapies.
Enrolling patients in the ongoing Phase 1b clinical trial of AKY-1189 in patients with locally advanced or mUC, breast cancer, non-small cell lung cancer, colorectal cancer, cervical cancer, and head and neck cancer.
Expect to report preliminary data from the Phase 1b trial in the first quarter of 2027.

AKY-2519 highlights

AKY-2519 is a novel, clinical-stage miniprotein radioconjugate designed to selectively deliver 225Ac to B7-H3 expressing tumors, including prostate, lung, and other solid tumors.

Received FDA clearance for the Company’s Investigational New Drug (IND) applications to proceed to a Phase 1b clinical trial with AKY-25191.
Announced two-trial clinical development strategy for AKY-2519, which was informed by insights from the Company’s clinical advisory boards and reflects the distinct treatment patterns and clinical needs of patients with mCRPC compared to other solid tumor patients. This strategy is designed to enable the broad evaluation of AKY-2519 in multiple patient segments while efficiently generating indication-relevant data.
Initiated Phase 1b, multicenter, open label clinical trial of AKY-2519 in PLUVICTO-naïve and PLUVICTO-experienced patients with mCRPC.
Plan to initiate a Phase 1b basket trial in patients with lung, colorectal, and other B7-H3 expressing solid tumors in the second half of 2026. The protocol for this trial has been finalized and is currently under regulatory review.
Announced clinical imaging and dosimetry data for AKY-2519 has been accepted for presentation at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held May 29 – June 2, 2026, in Chicago. The data facilitate initial understanding of AKY-2519 biodistribution and predicted absorbed doses in tumors and normal tissues in patients with B7-H3 expressing solid tumors.
1 IND applications were cleared for [64Cu]Cu-AKY-2519 for imaging and [225Ac]Ac-AKY-2519 for therapeutic use.

General corporate highlights

Appointed industry veteran Glenn Gormley, MD, PhD as an independent director to the Company’s Board of Directors (Board) and as co-chair of the Board’s newly established Science and Technology Committee.
In January 2026, completed an initial public offering of 20,297,500 shares of common stock, including the exercise in full by the underwriters of their option to purchase an additional 2,647,500 shares of common stock, at $18.00 per share, resulting in gross proceeds of approximately $365.4 million, before deducting underwriting discounts and commissions and other offering expenses.

Anticipated pipeline and corporate milestones for the next 12 months

AKY-1189: Expect preliminary data from the ongoing Phase 1b clinical trial in the first quarter of 2027.
AKY-2519:
Will present clinical imaging and dosimetry data of AKY-2519 in patients with mCRPC and various solid tumors at the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting.
Expect to commence Phase 1b basket trial in lung, colorectal, and other solid tumor cancers in the second half of 2026.
Expect preliminary data from the Phase 1b mCRPC clinical trial in 2027.
Early pipeline: Two programs are tracking toward development candidate nomination and commencement of IND-enabling activities in the first quarter of 2027.
Corporate: In-house Good Manufacturing Practices (GMP) facility expected to be operational in the second half of 2026 as part of the Company’s hybrid manufacturing strategy to expand capabilities and support clinical supply demand.

First quarter 2026 financial results

Cash position: Cash, cash equivalents and marketable securities were $538.5 million as of March 31, 2026, compared to $226.8 million as of December 31, 2025. The increase of $311.7 million primarily reflects net proceeds from the Company’s initial public offering in January 2026, primarily offset by cash used in operations. The Company’s cash, cash equivalents and marketable securities as of March 31, 2026 are expected to fund its operations into 2029.
Collaboration revenue: Collaboration revenue was $3.2 million for the quarter ended March 31, 2026, compared to $1.4 million for the comparable prior year period. The increase of $1.8 million was attributable to continued advancement of the Company’s research collaboration with Eli Lilly and Company, with revenue recognized over time using the cost incurred input method.
R&D expenses: Research and development expenses were $20.0 million for the quarter ended March 31, 2026, compared to $15.9 million for the comparable prior year period. The increase of $4.1 million was primarily driven by Phase 1b clinical trial expenses for AKY-1189, which initiated in the second quarter of 2025, increased expenses with advancing AKY-2519 through IND-enabling studies, and increased employee-related costs (including stock-based compensation) associated with increased hiring to support the advancing clinical pipeline.
G&A expenses: General and administrative expenses were $5.9 million for the quarter ended March 31, 2026, compared to $3.7 million for the comparable prior year period. The increase of $2.2 million was primarily due to higher employee-related costs (including stock-based compensation) related to increased hiring to support the Company’s growth, and increased expenses associated with operating as a public company.
Net loss: Net loss was $18.3 million for the quarter ended March 31, 2026, compared to $15.0 million for the comparable prior year period. The increase in net loss of $3.3 million was primarily driven by increased operating expenses.

About Aktis’ miniprotein radioconjugate platform
Aktis has developed a proprietary, isotope-agnostic miniprotein radioconjugate platform to selectively deliver the tumor-killing properties of radioisotopes to targeted tumors. Aktis’ therapeutic miniprotein radioconjugates are designed to maximize anti-cancer activity through high tumor penetration coupled with internalization and retention in cancer cells, while rapidly clearing from normal organs and tissues. The Aktis platform further enables clinicians to visualize and verify target engagement with imaging isotopes prior to exposure to therapeutic radioisotopes. Leveraging this platform, and its patient-first end-to-end supply chain, Aktis is advancing a pipeline of next-generation targeted radiopharmaceuticals to address the unmet needs of patients across a broad spectrum of solid tumors.

(Press release, Aktis Oncology, MAY 11, 2026, View Source [SID1234665455])

On May 11, 2026 Biomea Fusion, Inc. ("Biomea" or "Biomea Fusion" or "the Company") (Nasdaq: BMEA), a clinical-stage diabetes and obesity company, reported its financial results for the first quarter ended March 31, 2026, and provided a business update.

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"Across our portfolio, we continue to execute with focus and discipline, with all of our key clinical programs progressing on track toward important upcoming milestones, while maintaining a disciplined approach to managing our cash burn," said Mick Hitchcock, Ph.D., Interim Chief Executive Officer and Board Member of Biomea Fusion. "The recent 52-week data from the Phase II COVALENT-112 clinical trial in type 1 diabetes further support targeting menin as a novel approach across both type 1 and type 2 diabetes, which offers a paradigm shift and differs materially from existing therapeutic approaches. We are building on these findings with plans to initiate an investigator-sponsored Phase II clinical trial in collaboration with leading academic institutions specializing in T1D. We believe this collaboration, alongside the continued advancement of our type 2 diabetes and obesity programs, positions Biomea to deliver meaningful data across multiple indications in 2026."

Recent Corporate Highlights:

Icovamenib
Potential First-in-Class Oral Small Molecule Product Candidate Targeting Menin for Diabetes

Chronic toxicology studies in two species were successfully completed for icovamenib, providing nonclinical support for chronic clinical dosing beyond the 12-week duration used to date; findings demonstrated a favorable safety profile consistent with previously reported preclinical and clinical data.
With more than 400 subjects dosed to date, icovamenib was generally well tolerated and demonstrated a favorable safety profile throughout the observation periods.
Two Phase II clinical trials evaluating icovamenib in T2D have been initiated and enrollment is ongoing:
COVALENT-211, a Phase II, randomized, double-blind, placebo-controlled trial in patients with insulin-deficient T2D not achieving glycemic targets despite standard of care therapy.
COVALENT-212, a Phase II, randomized, double-blind, placebo-controlled trial in patients with T2D not achieving glycemic targets while on a GLP-1 RA-based therapy.
Both trials include a 26-week primary endpoint, with topline data anticipated in the fourth quarter of 2026.
Topline data from the Phase II COVALENT-112 clinical trial evaluating icovamenib in T1D patients were reported from the 52-week follow-up:
A 52% increase from baseline in mean C-peptide AUC at Week 12, after completion of the dosing period, in patients diagnosed within 0-3 years (n=5) receiving icovamenib 200 mg, with a dose response observed vs 100 mg (n=6). Persistence observed through Week 52, with mean C-peptide AUC largely preserved in the 200 mg group (~7% decline from baseline).
Preservation of C-peptide also observed in patients diagnosed between 3-15 years (n=9).
Icovamenib was generally well tolerated across all dosing arms and demonstrated a favorable safety and tolerability profile through Week 52.
Comprehensive dataset to be presented at the American Diabetes Association’s (ADA) Scientific Sessions (abstract is preliminary until time of presentation; full release scheduled for June 5, 2026 at 6:30 pm CDT).
Planning a Phase II trial in recently diagnosed T1D patients (≤ 3 years since diagnosis), in collaboration with four U.S. academic centers, to evaluate extended dosing (6–12 months) of icovamenib 200 mg and assess potential combination with an immunosuppressive agent; trial initiation expected in the second half of the year at leading centers including the Barbara Davis Center for Diabetes, Joslin Diabetes Center, University of Texas Health Science Center at San Antonio Diabetes Division, and the University of Miami Diabetes Research Institute.

BMF-650
Next-generation Oral Small Molecule GLP-1 RA Product Candidate for Obesity

GLP-131, a Phase I randomized, double-blind, placebo-controlled clinical trial evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of BMF-650 in otherwise healthy overweight or obese participants is ongoing.
Initial 28-day clinical weight reduction data from the Phase I GLP-131 clinical trial is anticipated in the second quarter of 2026.

First Quarter 2026 Financial Results

Cash, Cash Equivalents, and Restricted Cash: As of March 31, 2026, the Company had cash, cash equivalents and restricted cash of $45.1 million.

Net Loss: The Company reported a net loss attributable to common stockholders of $12.4 million for the three months ended March 31, 2026, which included $1.6 million of stock-based compensation, compared to a net loss of $29.3 million for the same period in 2025, which included $3.2 million of stock-based compensation.
Research and Development ("R&D") Expenses: R&D expenses were $9.1 million for the three months ended March 31, 2026, compared to $22.9 million for the same period in 2025. The decrease of approximately $13.8 million was primarily driven by a decrease of $7.6 million in external costs primarily driven by a decrease of $3.8 million related to clinical activities, a decrease of $1.9 million related to preclinical and exploratory programs, and a decrease of $1.9 million in other external costs related to consultants, advisors and other professional services to support our clinical trials. Personnel-related expenses decreased by $4.5 million, including stock-based compensation, due to a decrease in headcount. Facilities and other allocated expenses decreased by $1.7 million due to a decrease in rent and facilities-related costs.

General and Administrative ("G&A") Expenses: G&A expenses were $3.7 million for the three months ended March 31, 2026 compared to $6.8 million for the same period in 2025. The decrease of $3.2 million was primarily driven by a decrease of $1.9 million related to personnel-related expenses, including stock-based compensation, due to a decrease in headcount and a decrease of $1.1 million of corporate-related expenses. Facilities and other allocated expenses decreased by $0.2 million.

(Press release, Biomea Fusion, MAY 11, 2026, View Source [SID1234665452])

On May 11, 2026 Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing precision therapies for solid tumors designed to preserve organ function, reported financial results for the first quarter ended March 31, 2026, and provided recent business highlights.

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"We are entering a pivotal period for Aura as we advance our Phase 3 CoMpass trial toward enrollment completion and potential registration," said Natalie Holles, Chief Executive Officer of Aura Biosciences. "Bel-sar has the potential to become the first frontline, vision-preserving therapy for patients with early choroidal melanoma, and we are on track to deliver topline data from the CoMpass trial in the second half of 2027. Supported by our strengthened balance sheet, we are focused on executing against these priorities and translating this progress into long-term value for patients and shareholders."

Recent Pipeline Developments

Early Choroidal Melanoma

The ongoing Phase 3 CoMpass trial is the first registration-enabling study in early choroidal melanoma. This global, randomized Phase 3 trial is evaluating bel-sar versus a sham control. The trial is advancing toward enrollment completion, which is expected by mid-2026, with topline data for the 15-month primary endpoint anticipated in the second half of 2027.

Bel-sar has the potential to become the first frontline vision-preserving therapy in this setting. The Company previously received Orphan Drug Designation from the United States Food and Drug Administration (FDA) and the European Medicines Agency and Fast Track designation from the FDA for the treatment of early choroidal melanoma. The CoMpass trial is under a Special Protocol Assessment agreement with the FDA.

Metastases to the Choroid

The ongoing Phase 2 clinical trial of bel-sar in metastases to the choroid continues to enroll patients. The study is designed to include patients with choroidal metastases arising from a range of primary solid tumors and to evaluate early proof-of-concept based on a four-week efficacy endpoint. The Company remains on track to report early data from this trial in 2026.

Cancers of the Ocular Surface

The Company is initiating a Phase 1 proof-of-concept trial in Australia to assess safety, feasibility and tumor response through histopathologic evaluation at a 2–4-week time point. Development activities for this program are ongoing, with early proof-of-concept data expected in 2026.

Bladder Cancer

The ongoing Phase 1b/2 trial evaluating additional doses and cycles of bel-sar across intermediate- and high-risk non-muscle invasive bladder cancer (NMIBC) patients continues to advance, with initial 3-month clinical data expected mid-2026.

The trial is evaluating two approaches: immune ablative and neoadjuvant. In the immune ablative arm, bel-sar is given in two cycles without a transurethral resection of the bladder tumor (TURBT). In the neoadjuvant arm, bel-sar is given in two cycles before TURBT. Patients are monitored for response, recurrence (at 3, 6, 9, and 12 months), and safety.

Corporate Updates

As previously announced on May 4, 2026, the Company’s Board of Directors appointed Natalie Holles as Chief Executive Officer and President and member of the Board of Directors, effective April 30, 2026. Ms. Holles succeeds Elisabet de los Pinos, Ph.D., the Company’s founder, who stepped down from her roles as Chief Executive Officer and President and a member of the Board of Directors, effective on the same date.

First Quarter 2026 Financial Results

•
As of March 31, 2026, the Company had cash and cash equivalents and marketable securities totaling $114.7 million. On May 5, 2026, the Company completed an underwritten public offering of common stock and pre-funded warrants, which included the underwriters’ full exercise of their option to purchase additional shares of common stock. After deducting the underwriting discounts and commissions and estimated offering expenses, the Company received approximately $280.8 million in net proceeds from the offering, of which it subsequently used approximately $39.0 million to repurchase all the shares of its common stock held by Matrix Capital Management Master Fund, LP in the previously announced stock repurchase. The Company believes its current cash and cash equivalents and marketable securities, together with the net proceeds of the offering to the Company, are sufficient to fund its operations into the second half of 2028.

•
Research and development expenses increased to $28.0 million for the three months ended March 31, 2026 from $23.3 million for the three months ended March 31, 2025, primarily due to ongoing clinical and clinical research organization (CRO) costs associated with the progression of our global Phase 3 trial of bel-sar in early choroidal melanoma and manufacturing and development costs for bel-sar.

•
General and administrative expenses increased to $6.9 million for the three months ended March 31, 2026 from $5.7 million for the three months ended March 31, 2025. General and administrative expenses include $1.6 million of stock-based compensation for each of the three months ended March 31, 2026 and 2025. The increase in general and administrative expenses was primarily driven by higher personnel expenses related to growth of the Company and increased professional fees.

•
Net loss for the three months ended March 31, 2026, was $33.7 million, compared to $27.5 million for the three months ended March 31, 2025.

(Press release, Aura Biosciences, MAY 11, 2026, View Source [SID1234665451])

On May 11, 2026 Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) ("Tonix" or the "Company"), a fully integrated, commercial biotechnology company, reported financial results for the quarter ended March 31, 2026, and provided an overview of recent operational highlights.

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"TONMYA is the first new fibromyalgia medicine in 15 years," said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. "TONMYA is a non-opioid analgesic designed for bedtime administration and long-term use by adults. Since launch in November 2025, TONMYA has shown growth in prescriptions, new writers, refills, and patient access. Our first managed care partnership was announced in May, providing access to approximately 35 million U.S. commercial lives. We will continue engagement with commercial and government payers to expand patient access. Our focus remains on operational excellence across sales, marketing, medical affairs, and market access to educate and deliver on TONMYA’s differentiated potential."

Dr. Lederman continued, "We also continue to meaningfully advance our mid-stage clinical programs and our earlier-stage pipeline. For TNX-4800, our investigational long-acting borreliacidal, human monoclonal antibody targeting OspA on Borrelia burgdorferi, which causes the majority of Lyme disease in the U.S., we announced positive Phase 1 data and plans for an adaptive Phase 2 field study in 2027, pending FDA agreement. We look forward to our scheduled Type C meeting with the FDA early in the third quarter of 2026 to discuss the study. We believe TNX-4800 offers several advantages over vaccines in development, including onset of protection within two days and a simpler two-dose regimen with a second booster dose two months after the first. We also expect to begin our Phase 2 study of TONMYA for the treatment of Major Depressive Disorder (MDD) mid-year. Our other programs across CNS, infectious disease, immunology, and rare disease remain well positioned for near-term milestones."

Commercial Updates

TONMYA (cyclobenzaprine HCl sublingual tablets): a centrally acting, non-opioid analgesic for the treatment of fibromyalgia in adults

• On November 17, 2025, TONMYA became commercially available, following U.S. FDA approval in August 2025 for the treatment of fibromyalgia in adults. TONMYA is the first new prescription medicine approved for fibromyalgia in more than 15 years. The approval was based on two double-blind, randomized, placebo-controlled Phase 3 clinical studies of nearly 1,000 patients that demonstrated durable and statistically significant reduction in daily pain scores compared to placebo. There are now approximately 100 TONMYA sales reps in the field.
• In the first quarter of 2026, the first full quarter since launch, key metrics include:
o 2,145 unique healthcare providers prescribed TONMYA to patients.
o 3,588 unique patients initiated treatment with TONMYA.
o Approximately 5,400 prescriptions were filled. This includes bridge prescriptions that are facilitated through the Company’s specialty pharmacy channel. Bridge prescriptions represent initial patient fills provided while coverage determinations are pending and do not immediately generate net product revenue.
• For the period beginning November 17, 2025, through April 24, 2026, cumulative key metrics include:
o More than 2,700 unique healthcare providers have prescribed TONMYA to patients.
o Approximately 5,618 unique patients have initiated treatment with TONMYA.
• For the period beginning November 17, 2025, through May 1, 2026, cumulative key metrics include:
o Approximately 11,016 prescriptions were filled. This includes bridge prescriptions that are facilitated through the Company’s specialty pharmacy channel.
• Repeat prescriber and patient refill trends are encouraging.
• The Company is prioritizing engagement with commercial payers, Medicare, and Medicaid to increase access:
o In May 2026, Tonix secured commercial payer coverage with its first managed care partnership agreement with a leading GPO, which will provide access for approximately 35 million U.S. patients (20% of ~177 million commercial lives in the U.S.)
o To date, TONMYA is covered under Medicaid in 38 states, for approximately 55 million lives, representing 73% of the roughly 75 million Medicaid lives.
• Tonix has a robust patient access program and support services in place, including a TONMYA savings card, copay assistance, and prior authorization support, intended to reduce access barriers during early commercialization.
• To educate healthcare providers (HCPs), the Company held a multidisciplinary dialogue about TONMYA via a national webcast. Tonix also launched a national speaker training program with approximately 100 HCPs to maximize peer-to-peer speaker programs expected to occur across target specialties and regions this year.
• As part of a commitment to continued clinical evidence generation and education, Tonix presented clinical data on TONMYA at the 8th International Congress on Controversies in Fibromyalgia, 2026 American Academy of Pain Medicine (AAPM) PainConnect Annual Meeting, and 2026 Non-Opioid Pain Therapeutics Summit. The Company also published two articles in the peer-reviewed journal, Clinical Pharmacology in Drug Development.

Key Product Pipeline Candidates: Recent Highlights

Central Nervous System (CNS) Pipeline

TNX-102 SL (cyclobenzaprine HCl sublingual tablets): in Phase 2 development for MDD; remains on track to initiate mid-year 2026

• In November 2025, the FDA cleared the IND for TNX-102 SL 5.6 mg for the treatment of MDD in adults. The IND clearance enables Tonix to proceed with the HORIZON study, a potentially pivotal Phase 2, 6-week, randomized, double-blind, placebo-controlled study of TNX-102 SL as a first-line monotherapy in adults with MDD. About 360 patients will be enrolled at approximately 30 U.S. sites, with the primary endpoint being the MADRS total score change from baseline at Week 6. Tonix plans to initiate enrollment in mid-2026.

TNX-102 SL in Phase 2 development for the treatment of acute stress disorder (ASD) and acute stress reaction (ASR)

• The U.S. Department of Defense-funded Optimizing Acute Stress Reaction Interventions (OASIS) study is being conducted by the University of North Carolina under an investigator-initiated IND. The OASIS study examines the safety and efficacy of TNX-102 SL to reduce adverse posttraumatic neuropsychiatric sequelae among patients in the emergency department after a motor vehicle collision. Topline data is expected to be reported in the second half of 2026.

TNX-1300 (double-mutant cocaine esterase) for cocaine intoxication; Phase 2-program has Breakthrough Therapy designation from the FDA, with no products on the market for this indication

• The Company plans to meet with the FDA in 2026 to inform the clinical design of the next Phase 2 study (a Phase 2a study has been completed).

TNX-1900 (intranasal potentiated oxytocin): in development for several CNS disorders

• TNX-1900 is currently being studied in four Phase 2 and one Phase 1 investigator-initiated studies. The Phase 2 investigator-initiated studies include binge-eating disorder (Massachusetts General Hospital, "MGH"), adolescent obesity (MGH), bone health in autism (MGH and University of Virgina), and arginine vasopressin deficiency (MGH).
• In March 2026, Tonix announced the dosing of the first participant in a Phase 1 investigator-initiated pharmacodynamic study with Erasmus University of TNX-1900 in healthy female volunteers, using capsaicin and electrical stimulation to model trigeminal neurovascular reactivity.

Infectious Disease Pipeline

TNX-4800 (anti-OspA mAb): Phase 2-ready long-acting human monoclonal antibody in development for the seasonal prevention of Lyme disease in the U.S., which has no FDA-approved vaccines or prophylactics

• In March 2026, Tonix presented Phase 1 data at the World Vaccine Congress Washington 2026 and announced plans to initiate an adaptive Phase 2 field study in the first half of 2027, pending FDA agreement. The Company also presented Phase 1 data in April 2026 at the 4th Annual Ticks and Tickborne Diseases Symposium at Johns Hopkins University.
o TNX-4800 demonstrated encouraging safety, tolerability, pharmacokinetics, and immunogenicity, with serum TNX-4800 measurable at the earlier sampling time of 48 hours and no significant clinical or laboratory safety signals. The Phase 1 study was conducted by a team at UMass Chan Medical School led by Mark S. Klempner, MD, Professor of Medicine at UMass Chan and an inventor of TNX-4800.
• In April 2026, the Company announced it expects to lead a randomized, double-blind, placebo-controlled, adaptive Phase 2 field study to evaluate the efficacy of a two-dose regimen of TNX-4800 subcutaneous (SC) in preventing the first occurrence of confirmed Lyme disease during the primary efficacy surveillance period (Day 3 through Month 6 following administration). Each fixed dose is expected to provide exposures comparable to the 5 mg/kg dose evaluated in Phase 1. The first dose will be administered in the Spring and the second booster dose will be administered two months later. Participants will include adolescents and adults 16 years of age and older in Lyme-endemic areas in the U.S. The primary endpoint will be the prevention of Lyme disease for six months (comparison of TNX-4800 group and placebo group) following the initial dose.
• In April 2026, the Company announced it has scheduled a Type C meeting with the FDA early in the third quarter of 2026 to discuss the planned adaptive Phase 2 field study design.
• The Company expects to have GMP investigational product available for clinical testing in early 2027.

TNX-801 (recombinant horsepox virus): attenuated, pre-clinical live orthopoxvirus vaccine candidate for the prevention of smallpox and mpox

• In March 2026, Tonix presented animal and in vitro data on TNX-801 at the World Vaccine Congress Washington 2026. TNX-801 is expected to enter a Phase 1 study in 2027 pending FDA clearance of the Investigational New Drug (IND) application.

TNX-4200 (small molecule): broad spectrum anti-viral to protect against viral diseases

• TNX-4200 is a small molecule broad-spectrum antiviral agent targeting CD45 for the prevention or treatment of high lethality infections to improve the medical readiness of military personnel in biological threat environments.
• The TNX-4200 program is supported by an up to $34 million contract over five years from the Department of Defense’s Defense Threat Reduction Agency (DTRA). In the first quarter of 2026, the Company received confirmation that the project was cleared to enter the next budgetary and developmental phase.

Immunology Pipeline

TNX-1500 (dimeric Fc modified anti-CD40L, humanized mAb): Phase 2-ready third generation anti-CD40L for prophylaxis of kidney transplant rejection and treatment of autoimmune disorders

• In November 2025, Tonix announced a collaboration with MGH to advance a Phase 2, open-label, investigator-initiated clinical study of TNX-1500 in kidney transplant recipients, planned for initiation mid-year 2026, pending FDA clearance of the IND. The study is expected to enroll five adult kidney transplant recipients.

Rare Disease Pipeline

TNX-2900 (intranasal potentiated oxytocin): in development for Prader-Willi syndrome, with Orphan Drug designation as well as Rare Pediatric Disease designation that could make Tonix eligible for a Priority Review Voucher upon approval

• In September 2025, Tonix announced plans to initiate a Phase 2, randomized, double-blind, placebo-controlled study in children and adolescents with Prader-Willi syndrome. The study is expected to initiate in the first quarter of 2027.

Immuno-oncology Pipeline

TNX-1700 (TFF2-albumin fusion protein): in preclinical development for gastric and colorectal cancer

• In March 2026, Tonix presented preclinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2026. Data presented in an oral presentation showed how TNX-1700 reversed aging-associated gastric inflammation and significantly attenuated tumor progression in an aged gastric microenvironment in preclinical models. Data in a poster presentation demonstrated TNX-1700 exhibited dose-independent, linear pharmacokinetics in animals.

TNX-4700 (human anti-BTLA mAb): in preclinical development for immuno-oncology indications

• In March 2026, Tonix presented preclinical data in a poster presentation at the AACR (Free AACR Whitepaper) Annual Meeting 2026 demonstrating TNX-4700 demonstrated potent, high-affinity binding and functional antagonism. The mAb technology was licensed from Curia.

Financial: Recent Highlights

Tonix had approximately $185.5 million of cash and cash equivalents as of March 31, 2026, compared to approximately $207.6 million as of March 31, 2025. Net cash used in operations was approximately $42.3 million for the first quarter ended March 31, 2026, compared to $16.6 million for the same period in 2025.

Subsequent to quarter-end, the Company has raised $22.6 million proceeds using its at-the-market (ATM) facility.

The Company believes that its cash resources as of March 31, 2026, together with the net proceeds that it raised from equity offerings in the second quarter of 2026, will fund its planned operating and capital expenditure requirements into early second quarter of 2027.

As of May 8, 2026, the Company had 15,940,601 shares of common stock outstanding.

First Quarter 2026 Financial Results

Net product revenue for the first quarter 2026 was approximately $6.9 million, compared to $2.4 million for the same period in 2025, and consisted of combined net sales of TONMYA, Zembrace SymTouch, and Tosymra. Net revenue from sales of TONMYA for the first quarter was approximately $3.7 million. TONMYA was launched in November 2025. Net revenue from sales of TONMYA for the period from November 17, 2025, to December 31, 2025, was approximately $1.4 million. Net revenue from sales of Zembrace SymTouch and Tosymra for the was approximately $3.2 million compared to $2.4 million for the same quarter in 2025. Cost of sales for the first quarter 2026 was approximately $1.6 million, compared to $0.9 million for the same period in 2025.

Research and development expenses for the first quarter 2026 were approximately $18.2 million, compared to $7.4 million for the same period in 2025. This increase is predominately due to pipeline prioritization period over period, and increased headcount.

Selling, general, and administrative expenses for the first quarter 2026 were $28.6 million, compared to $10.1 million for the same period in 2025. The increase is predominately due to spending on sales and marketing related to TONMYA, as well as increased headcount.

Net loss available to common stockholders was $40.2 million, or $2.93 per basic and diluted share, for the first quarter 2026, compared to net loss available to common stockholders of $16.8 million, or $2.84 per basic and diluted share, for the same period in 2025. The basic and diluted weighted average common shares outstanding for the first quarter 2026 was 13,707,104 compared to 5,927,231 shares for the same period in 2025.

(Press release, TONIX Pharmaceuticals, MAY 11, 2026, View Source [SID1234665444])