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Corporate presentation

On January 12, 2026 Tscan therapeutics presented its corporate presentation.

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(Presentation, TScan Therapeutics, JAN 12, 2026, View Source [SID1234661972])

Ferring Pharmaceuticals and Theralase® Technologies Announce Clinical Development Agreement to Investigate Combination Therapy in Bladder Cancer

On January 12, 2026 Ferring Pharmaceuticals and Theralase Technologies Inc. (TSXV:TLT) (OTCQB:TLTFF) reported that they have entered into a collaborative clinical development agreement ("Agreement"), on January 9, 2026. The Agreement builds on Theralase’s existing clinical program (NCT03945162) with a new cohort investigating Theralase’s investigational light-activated small molecule Ruvidar (TLD-1433) in combination with Ferring’s intravesical non-replicating gene therapy ADSTILADRIN(nadofaragene firadenovec-vncg) for adult patients diagnosed with high-risk Bacillus Calmette-Guérin ("BCG")-unresponsive Non-Muscle Invasive Bladder Cancer ("NMIBC") Carcinoma In-Situ ("CIS") with or without papillary tumors (±Ta/T1).

Under the terms of the Agreement, Theralase will remain the sponsor of the study, with both parties providing clinical overview of the new cohort through a joint development committee. The new cohort will be enrolled and treated in the United States and then subject to written agreement may expand into Canada or other countries.

"The introduction of ADSTILADRIN, as the first intravesical gene therapy, filled a critical unmet need for patients with BCG-unresponsive NMIBC who faced limited treatment options and needed an alternative to invasive bladder removal surgery," said Ashish Kamat, MD, MBBS, Endowed Professor of Urologic Oncology (Surgery) and Cancer Research at University of Texas MD Anderson Cancer Center, Houston, Texas and President of the International Bladder Cancer Group. "As the BCG-unresponsive treatment paradigm continues to evolve, the combination of complementary therapies may offer additional treatment options to definitive therapy. This new study offers an opportunity to explore the possibility of enhancing efficacy and durability of response with a combination of a promising, investigational light-activated small molecule with the leading gene therapy in NMIBC."

The collaboration between Ferring and Theralase underscores their shared commitment to advancing innovative therapies for patients with BCG-unresponsive bladder cancer. The new cohort of patients will explore the potential benefit of combining two innovative and complementary mechanisms of action. ADSTILADRIN is the first and only intravesical gene therapy approved by the U.S. Food and Drug Administration ("FDA") for high-risk BCG-unresponsive NMIBC CIS with or without papillary tumors (±Ta/T1) that delivers the human interferon alpha-2b gene directly to bladder cells, prompting local secretion of interferon that exert multiple anticancer and immunomodulatory effects. Ruvidar is an investigational small molecule that penetrates cancer cell walls and, upon light activation, delivers targeted cytotoxic effects to destroy cancer cells, while inducing immunogenic cell death, stimulating both innate and adaptive immune responses.

"Underscoring our ambition to establish ADSTILADRIN as the backbone therapy in NMIBC, this collaborative clinical study with Theralase aims to explore whether combination approaches can further enhance patient outcomes, building on the strong foundation ADSTILADRIN has established in treating this challenging disease." said Daniel Shoskes, M.D., M.Sc, FRCS (C), Vice President and Global Medical Director for Uro-Oncology, Ferring Pharmaceuticals.

Roger DuMoulin-White, B.Sc, P.Eng, Pro.Dir, President, Chief Executive Officer and Chairman of the Board of Theralase, added, "We are honored to collaborate with Ferring, a worldwide leader in uro-oncology with deep expertise in bladder cancer treatment. As we approach completion of our clinical study and regulatory submissions for Ruvidar, this partnership provides the perfect opportunity to investigate whether we can deliver even greater benefits to patients through innovative combinational approaches."

Study Details

In Study II NCT03945162, Theralase is clinically investigating light-activated Ruvidar in the treatment of patients diagnosed with BCG-unresponsive NMIBC CIS (Study II). Theralase has enrolled and successfully treated 88 out of a planned 90 patients in Study II and plans to complete enrollment and treatment of the remaining 2 patients by 1Q2026 with follow-up completed by 2Q2027.

In the Collaborative Clinical Study, Theralasewill apply to the FDA to add a new cohort to Study II. This cohort will be treated with Theralase’s lead small molecule, light-activated Ruvidar, followed by treatment with Ferring’s FDA-approved ADSTILADRIN.

About NMIBC

NMIBC is a form of bladder cancer that is found in the inner layer cells of the bladder and does not invade into or beyond the muscle wall.1 In the United States, bladder cancer is the sixth most common cancer,2 fourth among men,3 and it is estimated that there will be approximately 84,870 new cases of bladder cancer in the U.S. in 2025.3 Historically, 75% of bladder cancer presents as NMIBC.4 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care; however, approximately one third of patients with NMIBC will not respond to BCG therapy and 50% of those with an initial response will experience recurrence or progression of their disease.5 Current treatment options for BCG-unresponsive patients are very limited and National Comprehensive Cancer Network ("NCCN") guidelines recommend cystectomy (partial or complete removal of the bladder).6

About Ruvidar

Ruvidar (TLD-1433) is a small molecule, able to be activated by light, radiation, sound and/or other drugs, intended for the safe and effective destruction of various cancers, bacteria and viruses.

About ADSTILADRIN

ADSTILADRIN (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical non-replicating gene-therapy for the treatment of adult patients with high-risk BCG-unresponsive NMIBC CIS with or without papillary tumors (±Ta/T1). It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high and transient local expression of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defenses against the cancer.

ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-risk, BCG-unresponsive NMIBC, who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849 and final five-year follow-up analysis published in The Journal of Urology).7-8

INDICATION

ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1).

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product.

WARNINGS AND PRECAUTIONS:

Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy.
Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use or oral administration.

USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose.

ADVERSE REACTIONS: The most common (>10%) adverse reactions; including, laboratory abnormalities (>15%), were increased glucose, instillation site discharge, increased triglycerides, fatigue, bladder spasm, micturition (urination urgency), increased creatinine, hematuria (blood in urine), decreased phosphate, chills, pyrexia (fever) and dysuria (painful urination).

You are encouraged to report negative side effects of prescription drugs to FDA. Visit View Source or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING.

(Press release, Theralase, JAN 12, 2026, View Source [SID1234661971])

Rigel Provides Business Update and 2026 Outlook

On January 12, 2026 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported a business update including preliminary total revenue and net product sales for the fourth quarter of 2025, ongoing activity from the commercial business and development pipeline, and its financial outlook for 2026.

"2025 was an excellent year for Rigel, marked by strong commercial execution with record net product sales; significant financial progress, including continued profitability and generation of $77 million in cash; and meaningful clinical advancement of R289 in lower-risk MDS, where we shared encouraging Phase 1b data," said Raul Rodriguez, Rigel’s president and CEO. "In 2026, we plan to continue to execute across all areas of the business — driving sustained commercial performance, supporting our expanding clinical programs with a solid financial foundation, delivering preliminary results from the dose expansion phase of our R289 study and continuing to explore opportunities to add new products to our portfolio."

Preliminary 2025 Financial Results and Business Update

Preliminary Financial Results

While Rigel is still determining final results for the fourth quarter of 2025, the company expects to report fourth quarter total revenue of $69.8 million, compared to $57.6 million for the same period of 2024.
Rigel expects to report fourth quarter of 2025 gross product sales of $84.5 million. Net product sales are expected to be $65.4 million, compared to $46.5 million for the same period of 2024, including:
TAVALISSE (fostamatinib disodium hexahydrate) net product sales of $45.6 million compared to $31.0 million for the same period of 2024.
GAVRETO (pralsetinib) net product sales of $10.2 million compared to $8.1 million for the same period of 2024.
REZLIDHIA (olutasidenib) net product sales of $9.6 million compared to $7.4 million for the same period of 2024.
Contract revenues for the fourth quarter of 2025 are expected to be approximately $4.4 million, consisting of $4.1 million in contract revenues from collaborations and $0.3 million in government contract revenue. Contract revenue from collaborations is expected to include $3.4 million of revenue from Grifols S.A. related to delivery of drug supplies and earned royalties, $0.3 million of revenue from Kissei Pharmaceutical Co., Ltd. related to delivery of drug supplies and $0.2 million of revenue from Medison Pharma Trading AG related to earned royalties.
For the full year, Rigel expects to report total revenue of $294.3 million, including net product sales of $232.0 million and contract revenues of $62.3 million, compared to total revenue of $179.3 million in 2024, which included net product sales of $144.9 million and contract revenues of $34.4 million. Expected 2025 contract revenues and total revenue include $40.0 million in non-cash revenue recognized in the second quarter of 2025 resulting from the release of the remaining cost share liability related to the agreement with Lilly for the development and commercialization of ocadusertib.
Rigel expects to report cash, cash equivalents, and short-term investments of approximately $154.6 million as of December 31, 2025, compared to $77.3 million as of December 31, 2024.
The above information is preliminary, has not been audited, and is subject to change upon the audit of Rigel’s financial statements for the fourth quarter and year ended December 31, 2025. Rigel expects to provide complete fourth quarter and full year 2025 financial results in March 2026.

Commercial

Rigel’s commercial portfolio is expected to report preliminary full-year net product sales of $232.0 million, growth of 60% compared to 2024.
Clinical Development and Regulatory

Rigel continues to advance its Phase 1b clinical study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of R2891, a potent and selective dual inhibitor of interleukin receptor-associated kinases 1 and 4 (IRAK1/4) in patients with relapsed or refractory (R/R) lower-risk myelodysplastic syndrome (MDS). In October 2025, Rigel announced enrollment of the first patient in the dose expansion phase of the study, where up to 40 patients will be randomized to receive either 500 mg once daily (QD) or twice daily (BID) to determine the recommended Phase 2 dose for future clinical trials.
Rigel is on track to complete enrollment of the dose expansion phase of the Phase 1b study and select the recommended Phase 2 dose for future clinical studies in the second half of 2026. The company anticipates sharing preliminary data from the dose expansion phase of the study by the end of 2026.
Rigel announced updated data from the dose escalation phase of the ongoing Phase 1b clinical study of R289 at the 2025 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December, indicating that R289 continued to be generally well tolerated in a heavily pretreated R/R lower-risk MDS patient population, the majority of whom were high transfusion burden (HTB) at baseline. Furthermore, red blood cell transfusion independence (RBC-TI ≥8 weeks) was achieved by 33% (6/18) of evaluable transfusion dependent patients receiving R289 doses of at least 500 mg QD and higher.
Also at the ASH (Free ASH Whitepaper) Annual Meeting, four posters were presented on olutasidenib, which included data that add to the growing body of evidence supporting the benefits of its use in patients with R/R mutated isocitrate dehydrogenase-1 (mIDH1) acute myeloid leukemia (AML).
In October 2025, the first patient was enrolled in the CONNECT Phase 2 TarGeT-D study evaluating olutasidenib in combination with temozolomide, followed by olutasidenib monotherapy as a maintenance regimen for newly-diagnosed adolescent and young adult patients with a high-grade glioma harboring an IDH1 mutation (NCT06161974).
Rigel presented sub-analysis data from the ARROW study evaluating pralsetinib for the treatment of metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) at the 2025 North America Conference on Lung Cancer (NACLC) in December. The two poster presentations included efficacy and safety data for patients previously treated with immunotherapy and for patients from the United States. In the trial, median overall survival (OS) was 44.3 months for the overall patient population. Longer median OS was seen in patients treated in the United States (62.4 months).
Rigel presented the first data release for pralsetinib from the TAPISTRY study (NCT04589845) in a poster presentation at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) – Gastrointestinal Cancers Symposium (ASCO-GI) in January. The analysis reported results from the Phase 2 global, open-label, multicohort study, in which the efficacy and safety of pralsetinib was evaluated in a cohort of patients with RET fusion-positive solid tumors, including pancreatic, colorectal, and hepatobiliary cancers. Pralsetinib demonstrated robust and durable activity against RET fusion-positive solid tumors, including gastrointestinal (GI) tumors, and in the efficacy evaluable population showed an overall response rate (ORR) of 67% (26/39). These data validate RET fusions as a tissue-agnostic target with sensitivity to RET inhibition, suggesting the potential therapeutic utility of pralsetinib in these patients.
On December 22, 2025, the U.S. Food and Drug Administration (FDA) notified Rigel of the approval of a Prior Approval supplemental New Drug Application, which updated the U.S. Prescribing Information to add a boxed warning regarding serious infections, including opportunistic infections. Rigel previously communicated this risk information to healthcare providers via a Dear Healthcare Provider letter in October 2024. The FDA also notified Rigel that it has met its postmarketing commitment for GAVRETO from its September 2020 accelerated approval to submit the final report for the AcceleRET-Lung study.
Publication

A paper titled "Olutasidenib for Mutated IDH1 Acute Myeloid Leukemia: Final Five-Year Results from the Phase 2 Pivotal Cohort" was published in November 2025 by Jorge E. Cortes, M.D., Phase 2 trial investigator and Director, Georgia Cancer Center, Cecil F. Whitaker Jr., GRA Eminent Scholar Chair in Cancer, in the Journal of Hematology & Oncology. The publication reports the final 5-year data from the pivotal cohort of the registrational trial evaluating olutasidenib for the treatment of patients with R/R mIDH1 AML, which includes an additional two years of efficacy and safety data. These 5-year data further support the durable responses and manageable safety profile observed with olutasidenib in patients with R/R mIDH1 AML, including those previously treated with venetoclax-based regimens.
2026 Outlook

Rigel anticipates 2026 total revenue of approximately $275 to $290 million, including:

Net product sales of approximately $255 to $265 million.
Contract revenues of approximately $20 to $25 million.
The company anticipates it will report positive net income for the full year 2026, while funding existing and new clinical development programs.

Upcoming Investor Event
Raul Rodriguez, Rigel’s president and CEO, will present a company overview at the 44th Annual J.P. Morgan Healthcare Conference in San Francisco, CA on Wednesday, January 14, 2026 at 3:00 p.m. PT (6:00 p.m. ET). To access the live webcast or archived recording, visit the Investor Relations section of the company’s website at www.rigel.com.

About ITP
In patients with immune thrombocytopenia (ITP), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. Patients suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs), and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About NSCLC
It is estimated that over 226,000 adults in the U.S. will be diagnosed with lung cancer in 2025. Lung cancer is the leading cause of cancer death in the U.S, with non-small cell lung cancer (NSCLC) being the most common type accounting for 85-90% of all lung cancer diagnoses.2 RET fusions are implicated in approximately 1-2% of patients with NSCLC.3

About AML
Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that there will be about 22,010 new cases in the United States, most in adults, in 2025.4

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.5,6 Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.7 Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need.

About TAVALISSE
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Please click here for Important Safety Information and Full Prescribing Information for TAVALISSE.

About GAVRETO
GAVRETO is indicated for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).*

*Thyroid indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

Please click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING, for GAVRETO.

About REZLIDHIA
REZLIDHIA is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.

Please click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING, for REZLIDHIA.

To report side effects of prescription drugs to the FDA, visit www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).

TAVALISSE, GAVRETO and REZLIDHIA are registered trademarks of Rigel Pharmaceuticals, Inc.

(Press release, Rigel, JAN 12, 2026, View Source [SID1234661970])

JPM Healthcare conference

On January 12, 2026 Replimune presented its corporate presentation.

(Presentation, Replimune, JAN 12, 2026, View Source [SID1234661969])

Relmada Therapeutics Provides Regulatory Update Confirming FDA Alignment on Registrational Studies Design for NDV-01 for Two Separate Indications

On January 12, 2026 Relmada Therapeutics, Inc. (Nasdaq: RLMD, "Relmada" or the "Company"), a clinical-stage biotechnology company advancing innovative therapies for oncology and central nervous system disorders, reported that it has received written feedback from the U.S. Food and Drug Administration (FDA) regarding the registrational development pathway for NDV-01 in 2nd-line refractory, high-grade BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) — one of the highest-risk and most treatment-resistant NMIBC populations — and in intermediate risk NMIBC in the adjuvant setting, where there are currently no approved therapies.

In its written response to Relmada’s Type B pre-IND submission, the FDA indicated that a single-arm, open-label clinical trial in this high-grade, BCG-unresponsive with CIS population is an appropriate registrational approach for NDV-01. This feedback provides a clear and efficient development path toward a potential NDA submission for NDV-01 as a bladder-sparing therapeutic option in a patient population with significant unmet need.

The FDA also provided separate, supportive written feedback on the Company’s planned single registrational study in intermediate-risk NMIBC in the adjuvant setting, which is expected to follow an open-label, randomized-to-observation design.

Relmada continues to anticipate initiating both registrational trials in the first half of 2026.

"We are very pleased with the FDA’s alignment on the registrational design for NDV-01 in high-grade BCG-unresponsive NMIBC," said Raj S. Pruthi, MD, Chief Medical Officer – Oncology at Relmada Therapeutics. "A single-arm pivotal study in this setting represents a meaningful opportunity to advance an in-office, bladder-sparing therapy for patients who have few if any effective alternatives. This study represents the fastest path to approval for NDV-01."

Dr. Pruthi continued, "We are also encouraged by the FDA’s feedback on our intermediate-risk registration plans, where we believe NDV-01 could potentially provide meaningful clinical benefit to patients where no approved treatments currently exist."

About the Planned High-Grade Registrational Study

The planned pivotal Phase 3 study in 2nd-line, refractory, high-grade BCG-unresponsive NMIBC with carcinoma in situ (CIS) will be an open-label, single-arm trial evaluating:

Primary endpoint: Complete response (CR) rate at any time
Key secondary endpoint: Duration of response (DOR)
Assessments: Cystoscopy, cytology, and biopsy per protocol

The design reflects FDA’s written guidance on the study population, endpoint selection, and evaluation methodology and is consistent with prior FDA precedents for single-arm registrational trials in NMIBC.

About the Planned Intermediate-Risk Registrational Study

The planned pivotal Phase 3 study in intermediate-risk NMIBC in the adjuvant setting will be an open label randomized-to-observation study:

Primary endpoint: Disease Free Survival (DFS)
Key secondary endpoint: Duration of response (DOR)
Assessments: Cystoscopy, cytology, and biopsy per protocol

The design reflects FDA’s written guidance on the study population, endpoint selection, and evaluation methodology.

About NDV-01

NDV-01 is a sustained-release, intravesical formulation of gemcitabine and docetaxel (Gem/Doce), in development for the treatment of non-muscle invasive bladder cancer. It is designed to enable Gem/Doce bladder retention and gradual drug release over ten days. The formulation creates a soft matrix that enhances local exposure while minimizing systemic toxicity. The NDV-01 formulation is a ready to use, convenient to administer in-office in less than five minutes, and does not require anesthesia or specialized equipment. It is protected by patents through 2038.

About NMIBC

NMIBC represents 75-80% of all bladder cancer cases and is associated with high recurrence (50 – 80% over 5 years). With over 744,000 prevalent cases in the U.S. and limited treatment options, the market opportunity is significant. High-grade BCG-unresponsive disease represents one of the most difficult-to-treat NMIBC subtypes, with limited bladder-sparing options. Intermediate-risk NMIBC in the adjuvant setting has no currently approved therapies. NDV-01 has the potential to serve as a frontline or salvage therapy and could be applicable across multiple NMIBC subtypes.