On October 16, 2025 Enterome, a clinical-stage company developing first-in-class OncoMimics immunotherapies to treat cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for follicular lymphoma in the low tumor burden "watch-and-wait" setting for its lead OncoMimics immunotherapy, EO2463. The decision highlights EO2463’s efficacy, excellent safety and tolerability as a first-in-class monotherapy in clinical testing to date in patients who currently do not normally receive any treatment as long as they do not show clinical symptoms, despite having been diagnosed with a cancer that progresses in the vast majority of cases.

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"The FDA’s decision is an important validation of the unique potential of Enterome’s OncoMimics program," said Pierre Belichard, CEO of Enterome. "It will expedite the clinical development and the regulatory pathways for EO2463, which is ready to enter registrational testing as early as next year after this Fast Track designation and a recent positive type-C meeting with the FDA."

The Fast Track designation provides increased opportunities for interaction with the FDA, rolling review and potential eligibility for priority review. EO2463 is ready for Phase 3 testing in watch-and-wait patients after showing marked efficacy as a monotherapy in interim data from the watch-and-wait population in the ongoing Phase 2 SIDNEY trial. The treatment was well tolerated, suggesting Enterome’s EO2463 immunotherapy may offer a safe and effective treatment option for patients in this setting, who have been diagnosed with a type of cancer they know is likely to progress, but show no troublesome symptoms and do not usually receive treatment.

Follicular Lymphoma (FL), one of several types of indolent Non-Hodgkin Lymphoma, is an incurable chronic condition with frequent relapses, characterized by slow progression and few symptoms, yet reduced life expectancy. It is usually diagnosed by the appearance of swollen lymph nodes, and the early stages of the disease can be characterized by a lack of troublesome symptoms such as night sweats, fever or weight loss. There is a widespread consensus among leading investigators of the need for a well-tolerated and effective monotherapy to stop or slow progression for watch-and-wait patients.

EO2463 is an innovative, off-the-shelf OncoMimics active immunotherapy that combines four synthetic peptides. These non-self, microbial-derived peptides correspond to CD8 HLA-A2 epitopes that exhibit molecular mimicry with the B lymphocyte-specific lineage markers CD20, CD22, CD37, and CD268 (BAFF receptor). It also includes the helper peptide (CD4+ epitope) universal cancer peptide 2 (UCP2). The unique ability of EO2463 to selectively target multiple B cell markers enables the destruction of malignant B lymphocytes. By ensuring broad target coverage across malignant B cells, this novel approach aims to simultaneously improve safety and maximize efficacy, reducing the tumor cells’ capacity to develop immune-resistance mechanisms such as antigen escape.

OncoMimics are bacteria-derived peptide antigens that closely mimic tumor-associated antigens or lineage markers. These synthetically produced peptides are designed in silico using AI and machine learning to mine Enterome’s extensive proprietary database of 23 million commensal bacteria genes. Because these peptides are "non-self," they tap into pre-existing pools of effector-memory CD8 T cells primed by gut bacteria, enabling rapid, strong, and durable anti-tumor responses while avoiding the self-tolerance that limits many cancer immunotherapies. Each product combines multiple high-affinity peptides to broaden target coverage and mitigate tumor heterogeneity.

OncoMimics are easy to manufacture, store, distribute and administer as an "off-the-shelf" subcutaneous injection. In clinical testing to date they have been shown to be extremely well tolerated, especially compared to other potent immunotherapies.

(Press release, Enterome, OCT 16, 2025, View Source [SID1234656700])

On October 16, 2025 Cycle Pharmaceuticals reported the launch of PHYRAGO, its first oncology product in the US. PHYRAGO is launched in partnership with Handa Therapeutics, LLC, and will be exclusively available through specialty pharmacy Onco360 with a specialty distribution network from McKesson, Cencora, and Cardinal Health.

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PHYRAGO is a tyrosine kinase inhibitor indicated for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML), adult patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), and pediatric patients 1 year of age and older with Ph+ CML in chronic phase, and newly diagnosed Ph+ ALL.1

PHYRAGO is bioequivalent to Sprycel (dasatinib) tablets,2 and has the unique clinical benefit of allowing patients to take proton pump inhibitors (PPIs) and H2 receptor antagonists (H2RAs) concomitantly to help manage gastric acid disorders.1 To help patients with medication access, financial savings, and clinical support, Cycle Vita will be available to eligible* patients taking PHYRAGO.†

"We are honored to introduce PHYRAGO as a new treatment option for people living with Ph+ leukemia," said Steve Fuller, Chief Strategy Officer at Cycle Pharmaceuticals.

"We understand the daily challenges that come with managing Ph+ CML and Ph+ ALL, and our goal is to ease that burden, not just with an improved formulation of dasatinib, but through the individualized care and hands on support offered by our Cycle Vita program. By enabling more flexibility in co-medication and providing a dedicated support team, we’re committed to helping patients and their families navigate treatment with greater confidence and peace of mind."

Cycle Pharmaceuticals has been empowering patients with rare diseases through its products and support hub since 2017.

NITYR (nitisinone) Tablets in 2017
SAJAZIR (icatibant) Injection in 2021
JAVYGTOR (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution in 2022
TASCENSO ODT (fingolimod) in 2023
ORMALVI (dichlorphenamide) tablets in 2024
VENXXIVA (tiopronin) Delayed-Release Tablets in 2025
BAFIERTAM (monomethyl fumarate) Delayed-Release Capsules in 2025
HARLIKU (nitisinone) Tablets in 2025
Indications

PHYRAGO is a kinase inhibitor indicated for the treatment of:

Newly diagnosed adults with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in chronic phase.
Adults with chronic, accelerated, or myeloid or lymphoid blast phase Ph+ CML with resistance / intolerance to prior therapy including imatinib.
Adults with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL) with resistance / intolerance to prior therapy.
Pediatric patients 1 year of age and older with Ph+ CML in chronic phase.
Pediatric patients 1 year of age and older with newly diagnosed Ph+ ALL in combination with chemotherapy.
Important Safety Information

Warnings and Precautions:

Myelosuppression and Bleeding Events: Severe thrombocytopenia, neutropenia and anemia may occur. Use caution if used concomitantly with medication that inhibits platelet function or anticoagulants. Monitor blood counts. Transfuse and interrupt PHYRAGO when indicated.

Fluid Retention: Fluid retention, including pleural effusions. Manage with supportive care and/or dose modification.

Cardiovascular Toxicity: Monitor for signs or symptoms and treat appropriately.

Pulmonary Arterial Hypertension (PAH): PHYRAGO may increase the risk of developing PAH which may be reversible on discontinuation. Consider baseline risk and evaluate patients for signs and symptoms of PAH during treatment. Stop PHYRAGO if PAH is confirmed.

QT Prolongation: Use PHYRAGO with caution in patients who have / may develop prolongation of the QT interval.

Severe Dermatologic Reactions: Severe mucocutaneous dermatologic reactions have been reported.

Tumor Lysis Syndrome: Tumor lysis syndrome has been reported. Maintain hydration and correct uric acid levels prior to initiating treatment.

Embryo-Fetal Toxicity: Can cause fetal harm. Advise patients of reproductive potential, of potential risk to fetus and to use effective contraception.

Effects on Growth and Development in Pediatric Patients: Epiphyses delayed fusion, osteopenia, growth retardation, and gynecomastia have been reported. Monitor bone growth and development.

Hepatotoxicity: Assess liver function before treatment, monthly thereafter or as clinically indicated. Monitor liver function when combined with chemotherapy.

Adverse Reactions:

Common adverse reactions in adults include myelosuppression, fluid retention events, diarrhea, headache, skin rash, hemorrhage, dyspnea, fatigue, nausea, and musculoskeletal pain.

Common adverse reactions in pediatric patients include mucositis, febrile neutropenia, pyrexia, diarrhea, nausea, vomiting, musculoskeletal pain, abdominal pain, cough, headache, rash, fatigue, constipation, arrhythmia, hypertension, edema, infections (bacterial, viral and fungal), hypotension, decreased appetite, hypersensitivity, dyspnea, epistaxis, peripheral neuropathy, and altered state of consciousness.

Postmarketing Experience:

Post approval adverse reactions:

Hepatitis B virus reactivation, atrial fibrillation/atrial flutter, interstitial lung disease, chylothorax, Stevens-Johnson syndrome, nephrotic syndrome, thrombotic microangiopathy, hepatotoxicity.

Drug Interactions:

Strong CYP3A4 Inhibitors and Strong CYP3A4 Inducers: Dose reduction may be necessary.

Antacids: Avoid concomitant use.

Use in Specific Populations:

Pregnancy: PHYRAGO can cause fetal harm. Advise a pregnant woman of the potential risk to a fetus.

Contraception: Should be used during treatment and for 30 days after the last dose.

Lactation: Breastfeeding is not recommended during treatment and for 2 weeks after the last dose.

Pediatric Use: There is no data in children under 1 year of age. Adverse reactions associated with bone growth and development were reported and should be monitored closely. Refer to the full USPI for pediatric patients with difficulty swallowing tablets.

Geriatric Use: Patients over 65 and older may experience: fatigue, pleural effusion, diarrhea, dyspnea, cough, lower gastrointestinal hemorrhage, appetite disturbance, abdominal distention, dizziness, pericardial effusion, congestive heart failure, hypertension, pulmonary Cinzia, and weight decrease. Monitor closely.

For more detailed information, please refer to the full Prescribing Information at phyrago.com/PI/.

To report SUSPECTED ADVERSE REACTIONS, contact Cycle Pharmaceuticals at
1-844-784-1807, or the FDA at: 1-800-FDA-1088 or www.fda.gov/medwatch.

US-DAS-2500114 | October 2025

(Press release, Cycle Pharmaceuticals, OCT 16, 2025, View Source [SID1234656699])

On October 16, 2025 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, reported to host a R&D Day in New York City. The Company’s leadership team and key opinion leaders will present the full Phase 1 dataset and outline the pivotal Phase 2 trial design and commercial opportunity for lasme-cel in r/r B-ALL.

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Details of the Event:

Date: Today, October 16, 2025
Time: 08:30 – 10:30 a.m. ET
Format: In-person and live webcast
Webcast: Join live via View Source

A replay will be available after the event on the Cellectis website.

(Press release, Cellectis, OCT 16, 2025, View Source [SID1234656698])

On October 16, 2025 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, reported the pricing of an underwritten offering to a select group of institutional investors consisting of 21,895,000 shares of its common stock and, in lieu of common stock to certain investors, pre-funded warrants to purchase an aggregate of up to 28,713,500 shares of its common stock. Each share of common stock and each pre-funded warrant is accompanied by a Class A Warrant and a Class B Warrant, each to purchase one share of common stock (or pre-funded warrant in lieu thereof) at an exercise price of $2.22 per share.

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The offering was led by RA Capital Management with participation from existing shareholders including OrbiMed, Soleus Capital, Lynx1 Capital Management, and Bain Capital Life Sciences.

The Class A Warrants are exercisable at any time through the earlier of (i) 30 days after the achievement of a trigger tied to the clinical results from the planned Phase 1b trial of cemsidomide with elranatamab, or (ii) five years from the date of issuance. The Class B Warrants are exercisable at any time prior to the fifth anniversary of the date of issuance and, under certain circumstances based on stock appreciation, C4T may require the mandatory exercise of the warrants. The shares of common stock (with accompanying Class A and Class B Warrants) are being sold at a combined price of $2.47 per share of common stock and accompanying warrants, and the pre-funded warrants (with accompanying Class A and Class B Warrants) are being sold at a combined price of $2.4699 per pre-funded warrant and accompanying warrants, which represents the per share price of the common stock less the $0.0001 per share exercise price for each pre-funded warrant.

All securities in the offering are being sold by C4T. The aggregate gross proceeds to C4T from the offering, before deducting underwriting discounts and commissions and offering expenses and excluding any proceeds from potential exercise of the Class A and Class B Warrants and nominal proceeds from potential exercise of the pre-funded warrants, are expected to be $125.0 million. If all Class A and Class B Warrants and pre-funded warrants are exercised, the aggregate gross proceeds to C4T from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $349.7 million. The offering is expected to close on or about October 17, 2025, subject to the satisfaction of customary closing conditions.

Together with its existing cash and cash equivalents and marketable securities, C4T intends to use the net proceeds of the offering to primarily fund its ongoing and planned clinical trials of cemsidomide, other research and development activities, and for working capital and general corporate purposes.

Jefferies, TD Cowen and Evercore ISI are acting as book-running managers for the offering.

The securities were offered by C4T pursuant to an effective shelf registration statement that was previously filed with the U.S. Securities and Exchange Commission (SEC) on October 31, 2024 and declared effective by the SEC on November 13, 2024 (File No. 333-282933). The prospectus supplement, accompanying prospectus and any free writing prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov.

When available, copies of the prospectus supplement, accompanying prospectus and any free writing prospectus relating to the offering may also be obtained from Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, New York 10022, by telephone at (877) 821-7388 or by email at [email protected]; TD Securities (USA) LLC, 1 Vanderbilt Avenue, New York, New York 10017, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717 or by email at [email protected]; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, New York 10055, by telephone at (888) 474-0200 or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction. The offering will be made only by means of the prospectus supplement and the accompanying prospectus.

(Press release, C4 Therapeutics, OCT 16, 2025, View Source [SID1234656697])

On October 16, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (OTC: APTOF, TSX: APS), a clinical-stage precision oncology company developing the tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed AML, reported that data from the ongoing TUSCANY trial of tuspetinib in combination with venetoclax and azacitidine (TUS+VEN+AZA) are being presented in a poster presentation, "TUSCANY Study of Safety and Efficacy of Tuspetinib plus Standard of Care Venetoclax and Azacitidine in Study Participants with Newly Diagnosed AML Ineligible for Induction Chemotherapy," at the European School of Haematology (ESH) 7th International Conference on Acute Myeloid Leukemia "Molecular and Translational": Advances in Biology and Treatment, being held from October 16-18, 2025 in Estoril, Portugal. Data to date from 10 patients in the TUSCANY trial across all three cohorts, 40 mg, 80 mg or 120 mg TUS dose in TUS+VEN+AZA, reveal promising clinical safety and antileukemic activity and support the use of TUS with standard of care treatment across a broad range of AML populations, including those carrying adverse mutations regardless of FLT3 mutation status.

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The TUS+VEN+AZA triplet is being developed as a safe and well-tolerated, mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy. Across all dose cohorts to date, no significant safety concerns or dose limiting toxicities (DLTs) have been observed in the TUSCANY trial, including no prolonged myelosuppression in Cycle 1 of subjects in remission, no reports of drug-related QTc prolongation or differentiation syndrome (DS), no CPK elevation and no treatment-related deaths. Dosing has begun at the 160 mg TUS dose level.

"We have observed that TUS can be safely added to a backbone VEN+AZA without needing to reduce the dose of these standard-of-care drugs. The activity we have observed with the TUS triplet in the first 10 patients has exceeded our expectations with 9 achieving complete remissions and 7 demonstrating MRD-negativity by central flow cytometry," said Rafael Bejar, M.D., Ph.D., Chief Medical Officer of Aptose. "In addition, these remissions are happening in diverse genetic subtypes including those with unmutated FLT3, FLT3-ITD, NPM1c, biallelic TP53 with complex karyotype, RAS, or myelodysplasia related mutations, making this a truly mutation agnostic therapy."

Data highlights:

TUS in combination with standard dosing of VEN+AZA has been well tolerated with no DLT, no treatment-related deaths, no differentiation syndrome, no QTc prolongation, no prolonged myelosuppression after remission in Cycle 1, and no CPK elevations reported at any dose levels to date in these newly diagnosed AML patients.
Addition of TUS to VEN+AZA achieved CR/CRh responses in 6/6 (100%) patients treated at the higher dose levels of 80 mg and 120 mg TUS, exceeding the 66% rate expected from VEN+AZA alone.
Overall, TUS+VEN+AZA CR/CRh responses were observed in 9/10 (90%) patients.
7 of 8 (88%) CR/CRh responses in FLT3 wildtype AML, representing 70% of AML population.
TUS+VEN+AZA MRD-negativity noted in 7/9 (78%) responding patients by central flow cytometry.
CR/CRh responses achieved across diverse mutational subtypes including: unmutated FLT3, FLT3-ITD, NPM1c, biallelic TP53 with complex karyotype, RAS, and myelodysplasia related mutations.
Dosing at the TUS 160 mg dose level is now ongoing.
See the ESH poster presentation here.

TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study

The tuspetinib-based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 trial with the goal of creating an improved frontline therapy for newly diagnosed AML patients that is active across diverse AML populations, durable, and well tolerated.

The TUSCANY triplet Phase 1/2 study, being conducted at 10 leading U.S. clinical sites by elite clinical investigators, is designed to test various doses and schedules of TUS in combination with standard dosing of AZA and VEN for patients with AML who are ineligible to receive induction chemotherapy. A convenient, once daily oral agent, TUS is being administered in 28-day cycles. Multiple U.S. sites are enrolling in the TUSCANY trial with anticipated enrollment of 18-24 patients by the end of 2025. Data will be released as it becomes available.

More information on the TUSCANY Phase 1/2 study can be found on www.clinicaltrials.gov.

(Press release, Aptose Biosciences, OCT 16, 2025, View Source [SID1234656696])