On May 15, 2025 Incyte (Nasdaq:INCY) reported that the U.S. Food and Drug Administration (FDA) has approved Zynyz (retifanlimab-dlwr), a humanized monoclonal antibody targeting programmed death receptor-1 (PD-1), in combination with carboplatin and paclitaxel (platinum-based chemotherapy) for the first-line treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC) (Press release, Incyte, MAY 15, 2025, View Source [SID1234653197]). In addition, the FDA granted approval for Zynyz as a single agent for the treatment of adult patients with locally recurrent or with metastatic SCAC with disease progression on or intolerance to platinum-based chemotherapy.

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"The FDA approval of Zynyz marks a pivotal moment, bringing effective combination and monotherapy treatment options to patients with advanced anal cancer after decades of limited innovation," said Hervé Hoppenot, Chief Executive Officer, Incyte. "At Incyte, we focus our efforts where we can make the biggest impact for patients. I am proud of our scientists and development teams for their perseverance in delivering the first approved PD-1 inhibitor to U.S. patients with SCAC."

The Priority Review and FDA approval of the supplemental Biologics License Application (sBLA) for Zynyz was based on data from two trials: the Phase 3 POD1UM-303/InterAACT2 trial evaluating Zynyz in combination with platinum-based chemotherapy (carboplatin-paclitaxel) in adult patients with metastatic or inoperable locally recurrent SCAC not previously treated with systemic chemotherapy, and the Phase 2 POD1UM-202 trial evaluating Zynyz monotherapy in previously treated patients with locally advanced or metastatic SCAC who have progressed on or were intolerant of platinum-based chemotherapy.

Results from POD1UM-303/InterAACT2, featured at a Presidential Symposium on Practice-Changing Trials at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) in 2024, showed a clinically meaningful and statistically significant 37% reduction in the risk of progression or death (P=0.0006). Patients in the Zynyz and chemotherapy combination group achieved a median progression-free survival (PFS) of 9.3 months compared to 7.4 months for patients in the placebo combination group. Additionally, a 6.2-month improvement in median overall survival (OS) was observed (P=0.0273) at an interim analysis; OS follow-up is ongoing. No new safety signals were observed. Serious adverse reactions occurred in 47% of patients receiving Zynyz in combination with chemotherapy. The most frequent serious adverse reactions (≥ 2% of patients) were sepsis (3.2%), pulmonary embolism (3.2%), diarrhea (2.6%) and vomiting (2.6%).

"Patients with inoperable locally recurrent or metastatic anal cancer have historically faced poor five-year survival rates and limited treatment options.1 The POD1UM data highlight the potential of Zynyz to be a meaningful new option, and notably demonstrate that the addition of Zynyz to platinum-based chemotherapy significantly improves progression-free survival," said Marwan Fakih, M.D., Professor, Medical Oncology & Therapeutics Research, Associate Director, Clinical Sciences, Medical Director, Briskin Center for Clinical Research, Division Chief, GI Medical Oncology, Co-Director, Gastrointestinal Cancer Program, City of Hope. "This approval marks an important advancement as it makes a new treatment approach available for this challenging cancer."

The Zynyz monotherapy approval is based on results from the POD1UM-202 study which demonstrated that treatment with Zynyz monotherapy produced an objective response rate (ORR) of 14% and disease control rate of 49%. Zynyz demonstrated a safety profile as expected of a PD-1 inhibitor with no loss of human immunodeficiency virus (HIV) infection control.2 Serious adverse reactions occurred in 40% of patients receiving Zynyz. The most frequent serious adverse reactions (≥ 2% of patients) were non-urinary tract infection, perineal pain, abdominal pain, anemia, hemorrhage, diarrhea, pyrexia, urinary tract infection, musculoskeletal pain and dyspnea.

"Patients with anal cancer often face a troubling lack of public awareness and understanding when it comes to risk factors, symptoms and their overall cancer journey," said David Winterflood, Chief Executive Officer of the Anal Cancer Foundation. "The approval of Zynyz marks a step forward for advanced SCAC treatment, brings attention to a long-overlooked condition with limited treatment options and offers patients whose anal cancer has returned or spread an option to treat their disease."

In addition to the sBLA in the U.S., Incyte submitted a Type II variation Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for retifanlimab in advanced SCAC and a Japanese New Drug Application (J-NDA) which was accepted by the Pharmaceuticals and Medical Devices Agency (PMDA) for retifanlimab in advanced SCAC.

Incyte is committed to supporting patients and removing barriers to access medicines. Eligible patients in the U.S. who are prescribed Zynyz have access to IncyteCARES (Connecting to Access, Reimbursement, Education and Support), a comprehensive program offering personalized patient support, including financial assistance and ongoing education and additional resources. More information about IncyteCARES is available by visiting www.incytecares.com or calling 1-855-452-5234, Monday through Friday, from 8 a.m. to 8 p.m. ET.

About Squamous Cell Carcinoma of the Anal Canal (SCAC)

SCAC is the most common type of anal cancer, making up 85% of cases.3 It is a rare disease for which the incidence is increasing approximately 3% per year.4 About 90% of cases are associated with human papillomavirus (HPV) infection—the number one risk factor for anal cancer.5 HIV is an important amplifier of anal cancer, as people with HIV are 25 to 35 times more likely to develop it.6,7 Anal cancer shares many of the same symptoms as non-cancerous conditions, such as hemorrhoids—including pain, itching, a lump or mass and changes in bowel movements—and as a result can go undetected leading to the majority of patients presenting with locally advanced disease.2,8 More information about SCAC is available by visiting www.analcancer.com.

About POD1UM

The POD1UM (PD1 Clinical Program in Multiple Malignancies) clinical trial program for retifanlimab includes POD1UM-303, POD1UM-202 and several other Phase 1, 2 and 3 studies for patients with solid tumors, including a registration-directed trial evaluating retifanlimab in combination with platinum-based chemotherapy for patients with non-small cell lung cancer.

About Zynyz (retifanlimab-dlwr)

Zynyz (retifanlimab-dlwr) is a humanized monoclonal antibody targeting programmed death receptor-1 (PD-1), indicated in combination with carboplatin and paclitaxel (platinum-based chemotherapy) for the first-line treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC) and as a single agent for the treatment of adult patients with locally recurrent or metastatic SCAC with disease progression or intolerance to platinum-based chemotherapy in the U.S.

Zynyz is also indicated for the treatment of adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC) in the U.S. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Zynyz is marketed by Incyte in the United States. In 2017, Incyte entered into an exclusive collaboration and license agreement with MacroGenics, Inc. for global rights to retifanlimab.

Zynyz is a registered trademark of Incyte.

Important Safety Information

What is the most important information I should know about Zynyz?

Zynyz is a medicine that may treat certain types of cancers by working with your immune system. Zynyz can cause your immune system to attack normal organs and tissues in any area of your body and can affect the way they work. These problems can sometimes become severe or life-threatening and can lead to death. You can have more than one of these problems at the same time. These problems may happen anytime during treatment or even after your treatment has ended.

Call or see your doctor right away if you develop any new or worsening signs or symptoms, including:

Lung problems: cough, shortness of breath, chest pain

Intestinal problems: diarrhea (loose stools) or more frequent bowel movements than usual; stools that are black, tarry, sticky, or have blood or mucus; severe stomach-area (abdomen) pain or tenderness

Liver problems: yellowing of your skin or the whites of your eyes; severe nausea or vomiting; pain on the right side of your stomach area (abdomen); dark urine (tea colored); bleeding or bruising more easily than normal

Hormone gland problems: headaches that will not go away or unusual headaches; eye sensitivity to light; eye problems; rapid heartbeat; increased sweating; extreme tiredness; weight gain or weight loss; feeling more hungry or thirsty than usual; urinating more often than usual; hair loss; feeling cold; constipation; your voice gets deeper; dizziness or fainting; changes in mood or behavior, such as decreased sex drive, irritability, or forgetfulness

Kidney problems: decrease in your amount of urine, blood in your urine, swelling of your ankles, loss of appetite

Skin problems: rash; itching; skin blistering or peeling; painful sores or ulcers in your mouth or nose, throat, or genital area; fever or flu-like symptoms; swollen lymph nodes

Problems can also happen in other organs and tissues. These are not all of the signs and symptoms of immune system problems that can happen with Zynyz. Call or see your doctor right away for any new or worsening signs or symptoms, which may include:

chest pain, irregular heartbeat, shortness of breath, or swelling of ankles
confusion, sleepiness, memory problems, changes in mood or behavior, stiff neck, balance problems, tingling or numbness of the arms or legs
double vision, blurry vision, sensitivity to light, eye pain, changes in eyesight
persistent or severe muscle pain or weakness, muscle cramps
low red blood cells, bruising
Infusion reactions that can sometimes be severe. Signs and symptoms of infusion reactions may include: chills or shaking, itching or rash, flushing, shortness of breath or wheezing, dizziness, feel like passing out, fever, back or neck pain.

Rejection of a transplanted organ or tissue. Your doctor should tell you what signs and symptoms you should report and monitor you, depending on the type of organ or tissue transplant that you have had.

Complications, including graft-versus-host disease, in people who have received a bone marrow (stem cell) transplant that uses donor stem cells (allogeneic). These complications can be serious and can lead to death. These complications may happen if you underwent transplantation either before or after being treated with Zynyz. Your doctor will monitor you for these complications.

Getting medical treatment right away may help keep these problems from becoming more serious. Your doctor will check you for these problems during your treatment. Your doctor may treat you with corticosteroid or hormone replacement medicines and may also need to delay or completely stop treatment if you have severe side effects.

Before you receive Zynyz, tell your doctor about all of your medical conditions, including if you:

have immune system problems such as Crohn’s disease, ulcerative colitis, or lupus
have received an organ transplant or tissue transplant, including corneal transplant
have received or plan to receive a stem cell transplant that uses donor stem cells (allogeneic)
have received radiation treatment to your chest area
have a condition that affects your nervous system, such as myasthenia gravis or Guillain-Barré syndrome
are pregnant or plan to become pregnant. Zynyz can harm your unborn baby
Females who are able to become pregnant:

Your doctor should do a pregnancy test before you start treatment.
You should use an effective method of birth control during your treatment and for 4 months after your last dose. Talk to your doctor about birth control methods that you can use during this time.
Tell your doctor right away if you become pregnant or think you may be pregnant during treatment.
are breastfeeding or plan to breastfeed. It is not known if Zynyz passes into your breast milk. Do not breastfeed during treatment and for 4 months after your last dose
Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Zynyz when given with the chemotherapy medicines carboplatin and paclitaxel in people with SCAC include tiredness, numbness, pain, tingling, or burning in your hands or feet; nausea; hair loss; diarrhea; muscle and bone pain; constipation; bleeding; rash; vomiting; decreased appetite; itching; stomach-area pain.

The most common side effects of Zynyz when used alone in people with SCAC include tiredness, muscle and bone pain, diarrhea, infection, rectal or genital-area pain, bleeding, urinary tract infection (UTI), rash, nausea, loss of appetite, constipation, stomach-area pain, shortness of breath, fever, vomiting, cough, itching, low levels of thyroid hormone, headache, decreased weight.

The most common side effects of Zynyz when used alone in people with MCC include tiredness, muscle and bone pain, itching, diarrhea, rash, fever, nausea.

These are not all the possible side effects of Zynyz. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Incyte Corporation at 1-855-463-3463.

General information about the safe and effective use of Zynyz

Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. If you would like more information about Zynyz, talk with your doctor. You can ask your doctor for information about Zynyz that is written for health professionals.

Please see the full Prescribing Information, including the Medication Guide, for Zynyz.

You may also report side effects to the FDA View Source or to Incyte Corporation at 1-855-463-3463.

On May 15, 2025 Pulmatrix ("Pulmatrix" or the "Company") (Nasdaq: PULM), a biopharmaceutical company that has focused on the development of novel inhaled therapeutic products intended to prevent and treat migraine and respiratory diseases with important unmet medical needs using its patented iSPERSE technology, reported first quarter financial results for 2025 and provided a corporate update (Press release, Cullgen, MAY 15, 2025, View Source [SID1234653162]).

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Peter Ludlum, Interim Chief Executive Officer of Pulmatrix, commented, "Our focus in the first quarter has been to advance steps to complete the proposed merger with Cullgen, a privately held, clinical-stage biopharmaceutical company applying its proprietary targeted protein degradation uSMITE platform to discover and advance therapeutics for the treatment of cancer and other diseases. If successful, the proposed merger would create a Nasdaq-listed company focusing on targeted protein degradation technology with three degrader programs in Phase 1 clinical trials – two for the treatment of cancer and one for the treatment of acute and chronic pain. As part of the proposed merger, Pulmatrix is currently in a process to divest its clinical assets, including our Phase 2-ready acute migraine product candidate PUR3100, along with our iSPERSE technology."

Proposed Merger with Cullgen

As previously reported, on November 13, 2024, the Company entered into an agreement and plan of merger with Cullgen Inc. ("Cullgen"), as amended by Amendment No. 1 thereto on April 7, 2025 (the "Merger Agreement" and such transaction, the "Merger"). The proposed Merger is anticipated to close in June 2025, subject to the satisfaction of certain closing conditions, among others, however the exact timing of the consummation of the proposed Merger cannot be predicted.

Additional information about the Merger Agreement and proposed Merger was previously disclosed in a registration statement on Form S-4 (File No. 333-284993) initially filed with the Securities and Exchange Commission (the "SEC") on February 14, 2025, as amended on April 17, 2025, and May 7, 2025.

Pulmatrix Currently Seeking Divestment of Clinical Assets and Proprietary iSPERSE Technology

PUR3100

PUR3100 is an orally inhaled dihydroergotamine ("DHE") engineered with Pulmatrix’s iSPERSE dry powder inhalation technology for the treatment of acute migraine.
In 2023, Pulmatrix announced the Food and Drug Administration’s acceptance of an Investigational New Drug ("IND") application for PUR3100 and receipt of a "study may proceed" letter to proceed with a Phase 2 study, positioning PUR3100 as a Phase 2-ready asset. The IND includes a Phase 2 clinical protocol where safety and preliminary efficacy of PUR3100 will be investigated in patients with acute migraine.
The Phase 2 IND builds on the Phase 1 trial results of PUR3100, which were published in 2024 in a peer-reviewed publication, Headache: The Journal of Head and Face Pain.
The study showed that PUR3100 achieved peak exposures in the targeted therapeutic range and time to maximum concentration occurred at five minutes after dosing at all dosing levels. The PUR3100 dose groups also showed a lower incidence of nausea and no vomiting compared to observations of nausea and vomiting in the intravenously ("IV") administered DHE dose group.
PUR1800

PUR1800 is a Narrow Spectrum Kinase Inhibitor ("NSKI"), engineered with our iSPERSE technology, for the treatment of acute exacerbations in chronic obstructive pulmonary disease ("AECOPD"). In 2023, Pulmatrix presented complete results from a Phase 1b study of PUR1800 for AECOPD, indicating PUR1800 was safe and well tolerated with no observed safety signals. The topline data, along with the results from chronic toxicology studies, support the continued development of PUR1800 for the treatment of AECOPD and other inflammatory respiratory diseases.
PUR1900

PUR1900 is the Company’s inhaled iSPERSE formulation of the antifungal drug itraconazole for indications where an orally inhaled antifungal may provide a therapeutic benefit or fulfill an unmet medical need.
The Company completed all Phase 2b wind down activities in the third quarter of 2024. With the study wind down complete, Pulmatrix bears no further financial responsibility for the development of PUR1900.
The Company’s partner Cipla has continued clinical development outside the United States and has advised us that they have completed their Phase 2 study in India and have been approved by India’s Central Drug Standard Control Organization to proceed with a Phase 3 clinical trial.
Should Cipla successfully market PUR1900 outside the United States, Pulmatrix will receive 2% royalties on any potential future net sales by Cipla outside the United States. Within the United States, the Company and Cipla will seek to monetize PUR1900 for indications where an orally inhaled antifungal may provide a therapeutic benefit or fulfill an unmet medical need.
iSPERSE Technology

iSPERSE particles are engineered with a small, dense and dispersible profile to exceed the performance of traditional dry powder particles as the iSPERSE particles have the dispersibility advantages of porous engineered particles. Pulmatrix believes this results in superior drug delivery compared to traditional oral and injectable forms of treatment for certain diseases.
As of March 31, 2025, Pulmatrix’s patent portfolio related to iSPERSE included approximately 146 granted patents, 18 of which are granted U.S. patents, with expiration dates from 2026 to 2037, and approximately 48 additional pending patent applications in the U.S. and other jurisdictions.
First Quarter 2025 Financial Results

Revenues decreased approximately $5.9 million to $0 for the three months ended March 31, 2025, compared to $5.9 million for the three months ended March 31, 2024. The decrease is primarily related to completion of the wind down of the PUR1900 Phase 2b clinical trial during the year ended December 31, 2024.

Research and development expenses decreased approximately $3.5 million to less than $0.1 million for the three months ended March 31, 2025, compared to $3.5 million for the three months ended March 31, 2024. The decrease was primarily due to winding down the PUR1900 Phase 2b clinical trial, disposal of the Company’s lab and facilities lease and employee terminations.

General and administrative expenses increased approximately $0.2 million to $1.8 million for the three months ended March 31, 2025, compared to $1.6 million for the three months ended March 31, 2024. The increase was primarily due to incurred costs related to the proposed Merger, partially offset by decreased employment and other operating costs.

The Company’s total cash and cash equivalents balance as of March 31, 2025, was $7.7 million. The Company anticipates that its cash position, based on operational efficiencies and prioritization of spending, is sufficient to fund its operations at least through the anticipated closing of the proposed Merger with Cullgen.

On May 15, 2025 Pyxis Oncology, Inc. (Nasdaq: PYXS), a clinical-stage company developing next-generation ADC therapeutics for difficult-to-treat cancers, reported financial results for the quarter ended March 31, 2025, and provided a business update (Press release, Pyxis Oncology, MAY 15, 2025, View Source [SID1234653180]).

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"We are enthusiastic about the progress we are making with micvotabart pelidotin, particularly our recent preclinical data that validate its unique three-pronged mechanism of action and indicate that MICVO monotherapy may be eliciting immune responses in previously unresponsive tumors," said Lara S. Sullivan, M.D., President, Chief Executive Officer and Chief Medical Officer of Pyxis Oncology. "Our focus remains on delivering durable and efficacious therapies for patients with recurrent or metastatic head and neck squamous cell carcinoma and other advanced solid tumors, building on the tremendous potential of our next generation ADC. We look forward to reporting data from our ongoing Phase 1 trials evaluating MICVO as a monotherapy and in combination with pembrolizumab later this year."

Pipeline Updates

•
Pyxis Oncology presented promising preclinical data at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting supporting the unique three-pronged mechanism of action of MICVO driving anti-tumor activity via direct tumor killing, bystander effect and immunogenic cell death. These data further reinforce the potential patient benefit of MICVO as a monotherapy and in combination with an anti-PD-1 therapy.
o
MICVO demonstrated broad anti-tumor activity across ten solid tumor indications in PDX models, attributed to EDB+FN target expression, proteolytic activity for MICVO linker cleavage and tumor responsiveness to the cytotoxic Auristatin0101 payload.
o
Differential gene expression analysis enabled identification of gene signatures associated with anti-tumor activity.
o
Upregulation of certain proteases may contribute to increased linker cleavage and subsequent increased MICVO activity, supporting hypothesis for extracellular mechanism.

o
Combining a mouse analog of MICVO with anti-PD-1 therapy inhibited EMT6 tumor growth and improved survival.

•
The Company expects to report preliminary data from the Part 2 monotherapy expansion cohorts of the ongoing Phase 1 clinical trial evaluating MICVO in 2L and 3L R/M HNSCC patients who have received prior platinum and PD-1 inhibitor therapy in the second half of 2025 and 2L and 3L R/M HNSCC patients who have received prior EGFRi and PD-1 inhibitor therapy in the first half of 2026. R/M HNSCC continues to be an area of high medical need despite improvements in treatment options.

•
Pyxis Oncology anticipates selecting a dose of MICVO in the ongoing Phase 1/2 combination study of micvotabart pelidotin and Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), in patients with R/M HNSCC and other advanced solid tumors by mid-year 2025 and reporting preliminary data from the trial in the second half of 2025.

First Quarter 2025 Financial Results

•
As of March 31, 2025, Pyxis Oncology had cash and cash equivalents, including restricted cash, and short-term investments, of $106.9 million. The Company believes that its current cash, cash equivalents, and short-term investments will be sufficient to fund its operations into the second half of 2026.

•
Research and development expenses were $17.0 million for the quarter ended March 31, 2025, compared to $13.0 million for the quarter ended March 31, 2024. The increase was primarily due to increased clinical trial-related expenses related to monotherapy and combination therapy of micvotabart pelidotin, increase in preclinical and translation work to support clinical development of micvotabart pelidotin and increased manufacturing of drug product and drug substance.

•
General and administrative expenses were $5.9 million for the quarter ended March 31, 2025, compared to $8.2 million for the quarter ended March 31, 2024. The decrease was primarily due to lower stock-based compensation costs, lower corporate insurance costs and decrease in legal, professional and consulting fees.

•
Net loss was $21.2 million, or ($0.35) per common share, for the quarter ended March 31, 2025, compared to $3.3 million, or ($0.06) per common share, for the quarter ended March 31, 2024. Net loss for the quarter ended March 31, 2024 included total revenues of $16.1 million related to the settlement agreement with Novartis for sale of royalty rights of Beovu for a one-time amount of $8.0 million and Novartis agreed to forgo its right to reclaim royalties previously paid of $8.1 million to us and Apexigen. Excluding non-cash stock-based compensation expense, the net loss for the quarter ended March 31, 2025 was $17.5 million, compared to net income of $1.1 million for the quarter ended March 31, 2024.

•
As of May 14, 2025, the outstanding number of shares of Common Stock of Pyxis Oncology was 61,947,665.

On May 15, 2025 MAIA Biotechnology, Inc. (NYSE American: MAIA), a clinical-stage biopharmaceutical company focused on developing targeted immunotherapies for cancer, reported that an abstract about the efficacy data from the Phase 2 THIO-101 clinical trial was accepted for poster presentation at the 2025 Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), taking place May 30 to June 3, 2025, in Chicago, Illinois (Press release, MAIA Biotechnology, MAY 15, 2025, View Source [SID1234653198]). The poster is scheduled for presentation on May 31, 2025, from 01:30pm to 04:30pm CDT in the Lung Cancer track.

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"We continue to believe that our telomere targeting agent ateganosine (THIO) could become a best-in-class anticancer treatment with the potential to challenge the standard of care for NSCLC," said MAIA CEO Vlad Vitoc, M.D. "Treatment with ateganosine has shown excellent efficacy in third-line NSCLC to date and we look forward to presenting our findings at ASCO (Free ASCO Whitepaper) 2025 on May 31st."

Poster Presentation Details

Poster title: "Phase 2 Study of Telomere-Targeting Agent THIO Sequenced With Cemiplimab in Third-Line Immune Checkpoint Inhibitor–Resistant Advanced NSCLC: Evaluation of Overall Survival"

Session date and time: May 31, 2025, 01:30pm to 04:30pm CDT

Presenter: Tomasz Jankowski, M.D.

MAIA’s poster will be available on the Publications page of MAIA’s website on the day of the presentation.

About ASCO (Free ASCO Whitepaper) 2025

The 2025 ASCO (Free ASCO Whitepaper) Annual Meeting will feature over 200 sessions and more than 5,000 posters complementing the theme, "Driving Knowledge to Action: Building a Better Future," reflecting the meeting’s tradition of inspiring and advancing the oncology field with the power of the latest knowledge in cancer care.

About Ateganosine

Ateganosine (THIO, 6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in non-small cell lung cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. Ateganosine-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with ateganosine followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. Ateganosine is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.

About THIO-101, a Phase 2 Clinical Trial

THIO-101 is a multicenter, open-label, dose finding Phase 2 clinical trial. It is the first trial designed to evaluate ateganosine’s anti-tumor activity when followed by PD-(L)1 inhibition. The trial is testing the hypothesis that low doses of ateganosine administered prior to cemiplimab (Libtayo) will enhance and prolong immune response in patients with advanced NSCLC who previously did not respond or developed resistance and progressed after first-line treatment regimen containing another checkpoint inhibitor. The trial design has two primary objectives: (1) to evaluate the safety and tolerability of ateganosine administered as an anticancer compound and a priming immune activator (2) to assess the clinical efficacy of ateganosine using overall response rate (ORR) as the primary clinical endpoint. The expansion of the study will assess ORR in advanced NSCLC patients receiving third line (3L) therapy who were resistant to previous checkpoint inhibitor treatments (CPI) and chemotherapy. Treatment with ateganosine followed by cemiplimab (Libtayo) has been generally well-tolerated to date in a heavily pre-treated population. For more information on this Phase 2 trial, please visit ClinicalTrials.gov using the identifier NCT05208944.

On May 15, 2025 Rakovina Therapeutics Inc. (TSX-V: RKV) (FSE: 7JO) ("Rakovina" or the "Company"), a biopharmaceutical company advancing innovative cancer therapies through artificial intelligence (AI)-powered drug discovery, reported a strategic financing of approximately $4 million (the "Offering") consisting of concurrent private placements of (i) convertible debenture units ("Debenture Units") for aggregate gross proceeds of approximately $1.1 million, and (ii) equity units ("Units" and, together with the Debenture Units, the "Offered Units") for aggregate gross proceeds of approximately $2.9 million (Press release, Rakovina Therapeutics, MAY 15, 2025, https://www.rakovinatherapeutics.com/rakovina-therapeutics-announces-strategic-private-placement-convertible-debt-financing-and-share-consolidation-to-accelerate-us-focused-growth-and-ai-powered-oncology-innovation-2/?utm_source=rss&utm_medium=rss&utm_campaign=rakovina-therapeutics-announces-strategic-private-placement-convertible-debt-financing-and-share-consolidation-to-accelerate-us-focused-growth-and-ai-powered-oncology-innovation-2 [SID1234653181]). The Offering is anchored by a $3 million indication of interest from strategic investors for $1.1 million of Debenture Units and $1.9 million of Units.

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The Company also announces that it will seek TSX Venture Exchange (the "TSXV") approval to implement a 10-for-1 share consolidation (the "Consolidation") to enhance its capital structure and position Rakovina for accelerated growth in U.S. capital markets, to be completed following closing of the Offering.

Offering

Pursuant to the Offering, Rakovina will issue approximately 58 million Units at an offering price of $0.05 per Unit, with each Unit consisting of one Pre-Consolidation Share (as defined herein) and one share purchase warrant (a "Warrant"). Each Warrant will entitle the holder to purchase one additional Pre-Consolidation Share at a price of $0.10 per Pre-Consolidation Share (or $1.00 per Post-Consolidation Share, following completion of the Consolidation), exercisable for a period of 24 months from issuance, subject to customary adjustments, including adjustment upon completion of the proposed Consolidation. If the closing price for the Company’s common shares on the TSXV is $0.25 or greater per Pre-Consolidation Share (or $2.50 per Post-Consolidation Share, following completion of the Consolidation) for five consecutive trading days, the expiry of the Warrants shall be accelerated to the date that is 30 days following the last day of the 5-day trading period.

Rakovina will also issue approximately 22 Debenture Units to a select group of investors at an offering price of $50,000 per Debenture Unit, for aggregate gross proceeds of approximately $1.1 million. Each Debenture Unit will be comprised of one unsecured convertible debenture (a "Debenture") in the principal amount of $50,000 and 100,000 share purchase warrants ("Debenture Warrants"). Each Debenture Warrant will entitle the holder to purchase one additional Pre-Consolidation Share at a price of $0.15 per Pre-Consolidation Share (or $1.50 per Post-Consolidation Share, following completion of the Consolidation), exercisable for a period of 24 months from issuance, subject to customary adjustments, including adjustment upon completion of the proposed Consolidation.

The principal amount of each Debenture shall be repayable in 36 months (unless earlier converted or redeemed) and will accrue interest at a rate of 12% per annum. Until the principal amount is repaid, a Debenture holder shall have the option to convert the principal amount of the Debenture into common shares of the Company at a conversion price of $0.10 per Pre-Consolidation Share (or $1.00 per Post-Consolidation Share, following completion of the Consolidation), subject to customary adjustments, including adjustment upon completion of the proposed Consolidation. Rakovina shall be entitled to redeem all or a portion of the principal amount of each Debenture at any time commencing 12 months after issuance of such Debenture, in cash and without premium.

The Offering may include one or more subscriptions by directors or other insiders of the Company. Subscriptions completed by insiders in the Offering may constitute a "related party transaction" as defined in Multilateral Instrument 61-101 – Protection of Minority Security Holders in Special Transactions ("MI 61-101") and Policy 5.9 of the TSXV. The Company is relying on exemptions from the formal valuation and minority shareholder approval requirements under MI 61-101 on the basis that neither the fair market value of the Offered Units issued to interested parties (as defined in MI 61-101), nor the consideration received for those Offered Units, will exceed 25% of the Company’s market capitalization.

Closing of the Offering is subject to the Company obtaining all necessary corporate and regulatory approvals, including approval of the TSXV. Pursuant to applicable Canadian securities laws, all securities issued in connection with the Offering will be subject to a statutory hold period of four months plus a day from the date of issuance. The Company may pay finders’ fees in connection with the Offering and in accordance with the policies of the TSXV.

Share Consolidation

Subject to the approval of the TSXV, Rakovina will consolidate all of its issued and outstanding common shares on the basis of 10:1, with each 10 Pre-Consolidation Shares (as defined below) being consolidated into one Post-Consolidation Share (as defined below). In accordance with the Company’s articles, shareholder approval of the proposed Consolidation will not be required.

The 140,042,575 common shares currently issued and outstanding (the "Pre-Consolidation Shares") will be reduced to approximately 14,004,257 common shares on a post-Consolidation basis (the "Post-Consolidation Shares"), assuming no additional Pre-Consolidation Shares are issued prior to completion of the Consolidation. Assuming 58,000,000 Pre-Consolidation Shares are issued pursuant to the Offering, there will be approximately 19,804,257 Post-Consolidation Shares issued and outstanding upon completion of the Consolidation. No fractional shares will be issued as a result of the Consolidation. Any fractional interest in shares that would otherwise result from the Consolidation will be rounded down to the nearest whole share, if the fractional interest is less than one-half of a share, and rounded up to the nearest whole share, if the fractional interest is equal to or greater than one-half of a share. No cash consideration will be paid in respect of fractional shares. The Company will not be changing its name in connection with the Consolidation and the Post-Consolidation Shares will continue to trade on the TSXV under the existing trading symbol.

The exercise or conversion price, and the number of Post-Consolidation Shares issuable under any of the Company’s outstanding convertible securities, will be proportionately adjusted upon the effective date of the Consolidation.

The effective date of the Consolidation, and new CUSIP and ISIN numbers for the Post-Consolidation Shares, if applicable, will be disclosed in a subsequent news release.

"This is more than a financing, it’s a pivotal inflection point," said Jeffrey Bacha, Executive Chairman of Rakovina Therapeutics. "We’re not just adding capital, we’re building capacity. The share consolidation strengthens our market position, while the continued integration of an advanced AI platform accelerates our ability to monetize our pipeline through high-value collaborations."

Rakovina’s proprietary DNA Damage Response (DDR) platform continues to show promise in targeting tumors with impaired DNA repair pathways—a hallmark of many treatment-resistant cancers. With a stronger capital structure, strategic investment, and embedded AI expertise, the Company is well-positioned to deliver lasting value to patients, partners, and shareholders alike.