Hoth Therapeutics Announces Plans to Submit Expanded Access Application for HT-001 to Support Cancer Patients in Need

On March 10, 2025 Hoth therapeutics reported that HT-001 is being developed to alleviate the adverse dermatological effects experienced by cancer patients undergoing epidermal growth factor receptor (EGFR) inhibitor treatments (Press release, Hoth Therapeutics, MAR 10, 2025, View Source [SID1234651048]). With positive progress in ongoing clinical studies, Hoth Therapeutics aims to provide patients access to HT-001 outside of traditional clinical trials through the Expanded Access Program (EAP), commonly known as "compassionate use."

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NEW YORK, March 10, 2025 /PRNewswire/ — Hoth Therapeutics, Inc. (NASDAQ: HOTH), a clinical-stage biopharmaceutical company dedicated to developing innovative therapies, reported its intention to submit an Expanded Access application for HT-001, its novel therapeutic candidate for the treatment of dermatological conditions associated with cancer therapy.

HT-001 is being developed to alleviate the adverse dermatological effects experienced by cancer patients undergoing epidermal growth factor receptor (EGFR) inhibitor treatments. With positive progress in ongoing clinical studies, Hoth Therapeutics aims to provide patients access to HT-001 outside of traditional clinical trials through the Expanded Access Program (EAP), commonly known as "compassionate use."

"Submitting an Expanded Access application is an important step in our commitment to ensuring patients who may benefit from HT-001 have the opportunity to receive this promising treatment," said Robb Knie, CEO of Hoth Therapeutics. "We remain dedicated to advancing solutions that address critical unmet medical needs and improve quality of life for patients undergoing cancer treatment."

The Expanded Access Program is designed to provide investigational treatments to patients with serious or life-threatening conditions who lack comparable treatment options. Through this program, Hoth Therapeutics seeks to offer HT-001 to eligible patients while continuing to gather valuable data on its safety and efficacy.

For more information about HT-001, ongoing clinical trials, or the Expanded Access Program, please visit www.hoththerapeutics.com

Kelun-Biotech’s TROP2 ADC Sacituzumab Tirumotecan (sac-TMT) Approved for Marketing in Second Indication by NMPA for EGFRm NSCLC

On March 10, 2025 Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd. (the "Company") reported that the Company’s trophoblast cell-surface antigen 2 (TROP2)-directed antibody-drug conjugate (ADC) sacituzumab tirumotecan (sac-TMT, formerly SKB264/MK-2870) was approved for marketing by the National Medical Products Administration (NMPA) for the second indication which is for the treatment of adult patients with epidermal growth factor receptor (EGFR) mutant-positive locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) following progression on EGFR-tyrosine kinase inhibitor (TKI) therapy and platinum-based chemotherapy (Press release, Kelun, MAR 10, 2025, View Source [SID1234651047]). This is the first TROP2 ADC drug approved for marketing in lung cancer globally. Compared with current standard of care, sac-TMT significantly extents the overall survival benefits of these patients.

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The approval is based on a multi-center, randomized, controlled, pivotal study (OptiTROP-Lung03) that evaluates the efficacy and safety profile of sac-TMT monotherapy 5mg/kg every other week (Q2W) as an intravenous injection versus docetaxel for the treatment of patients with locally advanced or metastatic EGFR-mutant NSCLC who have failed after treatment with an EGFR-TKI and platinum-based chemotherapy (used sequentially or in combination). As demonstrated in a pre-specified interim analysis, sac-TMT monotherapy demonstrated a statistically significant and clinically meaningful improvement in objective response rate (ORR), progression-free survival (PFS) and overall survival (OS) compared with docetaxel.

Sac-TMT is also the first ADC drug to demonstrate a statistically and clinically significant improvement in overall survival benefit versus standard of care in patients with EGFR-mutant NSCLC after failure of EGFR-TKI and platinum-based chemotherapy.

Previously, the NMPA has approved the marketing of sac-TMT in China for adult patients with unresectable locally advanced or metastatic triple negative breast cancer (TNBC) who have received at least two prior systemic therapies (at least one of them for advanced or metastatic setting) and accepted a supplemental new drug application (sNDA) seeking the approval of sac-TMT monotherapy for patients with EGFR-mutant NSCLC who have failed after EGFR-TKI therapy.

Currently, sac-TMT is fully deployed in the NSCLC field, and Merck Sharp & Dohme and the company are conducting a total of 10 registrational Phase Ⅲ clinical studies in NSCLC globally and in China, with the threads covering from the late backline to early postoperative adjuvant, including monotherapy and combination studies. Among them, a registrational Phase Ⅲ clinical study has been conducted in China in combination with osimertinib for the first-line treatment of locally advanced or metastatic NSCLC with EGFR mutations (OptiTROP-Lung07).

Dr. Michael Ge, CEO of Kelun-Biotech said, "We are very pleased that the second indication of the Company’s TROP2 ADC sacituzumab tirumotecan has been approved for marketing, which is exciting progress for us in the field of lung cancer. The successful approval of this new indication will help to address an unmet treatment need for patients with later-stage EGFRm NSCLC in China. Kelun-Biotech has always been committed to promoting innovation and leadership, and we look forward to continuing our research in the field of ADCs in partnership with MSD."

About NSCLC

Lung cancer, the second most common cancer in the world, includes two main types of cancer: non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC). NSCLC is the most common pathological type, accounting for about 80 to 85% of all lung cancers. The molecular typing of NSCLC patients in China is different from that of Western populations, and EGFR mutation is a common variant gene type, accounting for about 40 to 50% of lung adenocarcinoma patients in China. According to the 2024 Chinese Journal of Cancer Research (CSCO) guidelines, EGFR-TKIs are the preferred treatment for stage IV EGFR-mutant NSCLC.1 Platinum-containing chemotherapy is the main first-line chemotherapy regimen after resistance to EGFR-TKIs; and existing treatment regimens are ineffective in those who have failed EGFR-TKIs and platinum-containing chemotherapy. New drugs are urgently needed to improve patient survival.

About sac-TMT (佳泰莱)

Sac-TMT, a core product of the Company, is a novel human TROP2 ADC in which the Company has proprietary intellectual property rights, targeting advanced solid tumors such as NSCLC, breast cancer (BC), gastric cancer (GC), gynecological tumors, among others. Sac-TMT is developed with a novel linker to conjugate the payload, a belotecan-derivative topoisomerase I inhibitor with a drug-to-antibody-ratio (DAR) of 7.4. Sac-TMT specifically recognizes TROP2 on the surface of tumor cells by recombinant anti-TROP2 humanized monoclonal antibodies, which is then endocytosed by tumor cells and releases KL610023 intracellularly. KL610023, as a topoisomerase I inhibitor, induces DNA damage to tumor cells, which in turn leads to cell-cycle arrest and apoptosis. In addition, it also releases KL610023 in the tumor microenvironment. Given that KL610023 is membrane permeable, it can enable a bystander effect, or in other words kill adjacent tumor cells.

In May 2022, the Company licensed the exclusive rights to MSD (the tradename of Merck & Co., Inc., Rahway, NJ, USA) to develop, use, manufacture and commercialize sac-TMT in all territories outside of Greater China (includes Mainland China, Hong Kong, Macau, and Taiwan).

UroGen Pharma Reports Fourth Quarter and Full Year 2024 Financial Results and Announces Updated 18-Month Duration of Response (DOR) of 80.6% from the Phase 3 ENVISION Trial of UGN-102

On March 10, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported financial results for the fourth quarter and full year ended December 31, 2024, and provided an overview of recent developments (Press release, UroGen Pharma, MAR 10, 2025, View Source [SID1234651045]).

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"2024 was a pivotal year for UroGen with achievements across our commercial business and pipeline," said Liz Barrett, President and Chief Executive Officer of UroGen. "The progress with UGN-102 for low-grade intermediate-risk non-muscle invasive bladder cancer, including completion of the submission of our NDA ahead of schedule and compelling DOR data from the Phase 3 ENVISION trial, further positions us to launch a product that we believe will represent a paradigm shift in care, if approved. UGN-102 is supported by a compelling body of clinical data, and we are very pleased to report today the 18-month DOR of 80.6% from the ENVISION trial, maintaining duration of response consistent with 12-month DOR of 82.5% after a three-month CR was achieved. If approved, UGN-102 will address an estimated market opportunity of over $5 billion. We continue to advance our early-stage pipeline, including through the recent purchase of ICVB-1042, a next generation investigational oncolytic virus therapy with potential applications in bladder and other cancers."

2024 and Recent Business Highlights:

UGN-102 (mitomycin) for intravesical solution:

UroGen’s NDA for investigational drug UGN-102 (mitomycin) for intravesical solution as a treatment for low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC), is currently under review by the FDA. The FDA assigned a PDUFA target action date of June 13, 2025.
The Company reported updated 18-month DOR data from the Phase 3 ENVISION trial that evaluated UGN-102 in patients with LG-IR-NMIBC. The 18-month DOR data is consistent with prior Kaplan-Meier estimates, with a DOR rate of 80.6% (95% CI: 74.0, 85.7) at 18-months (N=101) compared to 82.5% (95% CI: 76.1, 87.3) at 12-months (N=146). Median follow-up time at 18 months was 18.7 months after the three-month CR.
Results from a subgroup analysis of the ENVISION trial were featured in a poster at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Genitourinary Cancers Symposium (ASCO-GU 2025) in February 2025. The analysis of pre-specified subgroups (presented by Sandip M Prasad, MD, MPhil et al) suggests that tumor burden and the number of tumors at baseline did not significantly affect the CR rate or DOR for patients treated with UGN-102.
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC):

Generated net product revenue of $90.4 million and $82.7 million for the years ended December 31, 2024 and 2023, respectively. Underlying demand revenue increased 12% year-over-year, partially offset by a decrease in CREATES Act sales, which totaled $3.0 million in 2024 compared to $4.4 million in 2023. "New prescribers" and "new patient" starts increased 33% and 13%, respectively, in 2024.
A long-term study on JELMYTO titled, "Durability of Response of UGN-101: Longitudinal Follow-Up of Multicenter Study," was published online in Urologic Oncology: Seminars and Investigations in January 2025. The results showed 68% recurrence-free survival rate at three years across a broad patient population with LG-UTUC.
A long-term follow-up study to the OLYMPUS trial evaluating JELMYTO was presented at the Society for Urologic Oncology annual meeting in December 2024. The study reported a median DOR of 47.8 months in patients who achieved a CR after primary chemoablation with JELMYTO. These results were also published online in the Journal of Urology.
Next-generation novel mitomycin-based formulation for urothelial cancers

Enrollment is ongoing in the Phase 3 UTOPIA trial of investigational drug UGN-103 (mitomycin) for intravesical solution in patients with LG-IR-NMIBC. UGN-103 is a next generation investigational medicine that combines UroGen’s RTGel technology with a novel mitomycin formulation licensed from medac GmbH. UGN-103 is planned to follow the potential FDA approval and launch of UGN-102 for LG-IR-NMIBC. To learn more about the UTOPIA trial, refer to clinicaltrials.gov/NCT06331299.
UroGen plans to initiate a Phase 3 trial to explore the safety and efficacy of UGN-104, its next generation product for LG-UTUC in the first half of 2025.
UGN-301 (zalifrelimab), an anti-CTLA4 antibody for use in high-grade non-muscle invasive bladder cancer

Completed enrollment of the dose escalation phase of UGN-301, alone and in combination with fixed dose UGN-201 or gemcitabine. The treatments demonstrated an acceptable safety profile and were generally well tolerated across dose levels. Responses were observed in both monotherapy and combination therapy arms, and patient follow-up is ongoing to further assess the durability of these responses.
Next-generation investigational oncolytic virus ICVB-1042

In February 2025, UroGen expanded its pre-clinical oncology portfolio with the purchase of the product candidate ICVB-1042 and certain related assets from IconOVir Bio, Inc. ICVB-1042 is being developed to be a potent, fast-replicating, next-generation oncolytic virus therapy that can be administered locally to treat bladder cancer and potentially other specialty cancers. UroGen hosted a webinar to discuss ICVB-1042 on February 20, 2025 and a replay can be accessed on the Company’s website here.
Fourth Quarter and Full Year 2024 Financial Results

JELMYTO Revenue: JELMYTO net product revenue was $24.6 million in the fourth quarter of 2024, compared to $23.5 million for the same period in 2023. Underlying demand revenue increased 15% year-over-year, partially offset by a decrease in CREATES Act sales, which totaled $0.2 million in the fourth quarter of 2024, compared to $2.4 million in the same period in 2023. JELMYTO net product revenue for the full year ended December 31, 2024 was $90.4 million, compared with $82.7 million for the full year ended December 31, 2023.

R&D Expense: Research and development expenses for the fourth quarter of 2024 were $14.9 million, as compared to $11.3 million for the same period in 2023. Research and development expenses for the full year 2024 were $57.1 million compared with $45.6 million for the full year 2023.

SG&A Expense: Selling, general and administrative expenses for the fourth quarter of 2024 were $34.9 million compared to $24.6 million in the same period in 2023. Selling, general and administrative expenses were $121.2 million for the full year 2024 compared with $93.3 million for the full year 2023.

Financing on Prepaid Forward Obligation: UroGen reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $6.1 million in the fourth quarter of 2024, compared to $5.5 million in the same period in 2023. Non-cash financing expense related to RTW Investments were $23.4 million in the full year 2024 compared with $21.6 million in the full year 2023.

Interest Expense on Long-Term Debt: Interest expense related to the outstanding $125 million term loan facility with funds managed by Pharmakon Advisors was $3.9 million and $12.5 million, respectively, for the fourth quarter and full year 2024, compared with $3.6 million and $14.7 million, respectively for the fourth quarter and full year 2023.

Net Loss: UroGen reported a net loss of $37.5 million or ($0.80) per basic and diluted share in the fourth quarter of 2024 compared with a net loss of $26.0 million or ($0.72) per basic and diluted share in the same period in 2023. Net loss was $126.9 million or ($2.96) per basic and diluted share in 2024 compared with a net loss of $102.2 million or ($3.55) per basic and diluted share in 2023.

Cash & Cash Equivalents: As of December 31, 2024, cash, cash equivalents and marketable securities totaled $241.7 million.

For further details on the Company’s financials, refer to Form 10-K, filed with the SEC.

2025 JELMYTO Revenue and Company Operating Expense Guidance: The Company expects full-year 2025 net product revenues from JELMYTO to be in the range of $94 to $98 million. This implies a year-over-year growth rate of approximately 8% to 12% over the $87.4 million in demand driven JELMYTO sales in 2024, which excludes the $3.0 million in CREATES Act sales reported in 2024. Full-year 2025 operating expenses are expected to be in the range of $215 to $225 million, including non-cash share-based compensation expense of $11 million to $14 million.

Conference Call & Webcast Information: Members of UroGen’s management team will host a live conference call and webcast today at 10:00 AM Eastern Time to review UroGen’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at investors.UroGen.com. Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast.

About JELMYTO

JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated for the treatment of adult patients with LG-UTUC. It is recommended for primary treatment of biopsy-proven LG-UTUC in patients deemed appropriate candidates for renal-sparing therapy. JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for administration in a retrograde manner via ureteral catheter or antegrade through nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.

APPROVED USE FOR JELMYTO

JELMYTO is a prescription medicine used to treat adults with a type of cancer of the lining of the upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO. Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose. Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?

Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
JELMYTO is given to your kidney through a tube called a catheter.
During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:

JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:

Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: urinary tract infection, blood in your urine, side pain, nausea, trouble with urination, kidney problems, vomiting, tiredness, stomach (abdomen) pain.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1‑800‑FDA‑1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

Please see JELMYTO Full Prescribing Information, including the Patient Information, for additional information.

About Upper Tract Urothelial Cancer (UTUC)

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as UTUC. In the U.S., there are approximately 6,000 – 7,000 new or recurrent low-grade UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). These treatments can be associated with a high rate of recurrence and relapse.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the NDA submission in August, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a PDUFA goal date of June 13, 2025.

TuHURA Biosciences, Inc. to Present at the 37th Annual ROTH Conference

On March 10, 2025 TuHURA Biosciences, Inc. (NASDAQ:HURA) ("TuHURA"), a Phase 3 registration-stage immuno-oncology company developing novel technologies to overcome resistance to cancer immunotherapy, reported that James A. Bianco, M.D., President and Chief Executive Officer of TuHURA, will participate in a fireside chat on Tuesday, March 18, 2025 at 8:30 AM PT at the 37th Annual ROTH Conference being held in Dana Point, CA (Press release, TuHURA Biosciences, MAR 10, 2025, View Source [SID1234651044]).

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In addition to the presentation, management will be available to participate in one-on-one in-person meetings with qualified members of the investor community who are registered to attend the conference. For more information about the event, please visit the conference website.

A live webcast of the fireside chat will be available on the Events page of the Investors section of the Company’s website (tuhurabio.com).

Purple Biotech Reports Fourth Quarter and Full-Year 2024 Financial Results

On March 10, 2025 Purple Biotech Ltd. ("Purple Biotech" or "the Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class therapies that seek to overcome tumor immune evasion and drug resistance, reported financial results for the three and twelve months ended December 31, 2024 (Press release, Purple Biotech, MAR 10, 2025, View Source [SID1234651043]).

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"Our major value-driving milestones in 2024 included completing successful trials for both of our clinical-stage programs, CM24 and NT219, as well as expanding the body of exciting preclinical data for our CAPTN-3 tri-specific platform, supporting its differentiated benefit," stated Gil Efron, Purple Biotech CEO.

"CM24 met all of its efficacy endpoints in the randomized Phase 2 second-line pancreatic cancer trial, which also generated significant biomarker data that is now informing the design of our Phase 2b study for CM24, which we plan to initiate in the second half of 2025. As a biomarker-driven study, the Phase 2b study may evaluate CM24 across multiple oncology indications.

"Earlier in 2024, we concluded the dose escalation study for NT219, demonstrating activity in combination with cetuximab, good safety profile, and determining the recommended Phase 2 dose. This enabled us to move forward with a Phase 2 study in head and neck cancer in collaboration with the University of Colorado, which we expect will commence patient enrollment in the first half of 2025.

"We believe that our CAPTN-3 platform is well-positioned and differentiated in the T cell engagers (TCE) and multi-specific space, uniquely unleashes both innate and adaptive immune cells against the tumor, demonstrating synergistic effect of the T cell and NK cell activating arms. The unique NKG2A arm in the lead compound acts as a checkpoint inhibitor, enabling simultaneous NK and T cell activation, including effector subsets with high antitumor activity. We are excited about our CAPTN-3 collaboration with the Icahn School of Medicine at Mount Sinai. Our cash runway is expected to extend into mid-2026, providing us with the time to potentially deliver on more catalysts this year, in order to achieve our ambitious programs across all three assets.," Mr. Efron concluded.

Q4 2024 and Recent Clinical & Corporate Highlights:

● CM24 Planned to Advance into Phase 2b Study Supported by Biomarker Data

o Positive final results from randomized Phase 2 study of CM24 in second line pancreatic cancer

o Serum CEACAM1 biomarker associated with 79% reduction in risk of death

Purple Biotech reported positive final results from the randomized Phase 2 study of CM24, a humanized monoclonal antibody that blocks CEACAM1, in patients with second-line pancreatic ductal adenocarcinoma (PDAC). CM24, in combination with nivolumab and Nal-IRI/5FU/LV chemotherapy, demonstrated consistent improvements across all efficacy endpoints. The enhanced results in patients with elevated CEACAM1 and other serum markers suggest that selecting a biomarker-enriched patient population could further enhance CM24’s efficacy, potentially positioning it as a treatment for multiple CEACAM1-expressing malignancies in line with its mechanism of action. A biomarker-enriched patient population analysis based on pretreatment serum CEACAM1 levels demonstrated a significant improvement in the treatment arm over the control arm, with a 79% reduction in risk of death (HR 0.21, p = 0.04), a median overall survival (OS) improvement of 5.1 months, and over 90% reduction in the risk of progression or death (HR < 0.1, p = 0.003), with a median progression-free survival (PFS) improvement of 2.9 months and improvement in the objective response rate (ORR) of 50% in the treatment arm compared to 0% in the control arm.

Additional biomarker analysis revealed statistically significant results for 80% of the patients (24 out of 30) with serum CEACAM1 (5-16K pg/mL) or serum NET marker myeloperoxidase (MPO) (200-600 ng/mL) demonstrating 61% reduction in the risk of death (HR 0.39, p= 0.039) and 72% reduction in the risk of progression or death (PFS HR 0.28, P 0.006) following treatment with CM24 and nivolumab in combination with Nal-IRI/5FU/LV chemotherapy compared to same chemotherapy alone. In addition, median PFS increased by 2.2 months, and median OS increased by 2.4 months, from 5.5 months with chemotherapy alone to 7.9 months with the combination therapy.

● NT219 Advances into Phase 2 Head and Neck Cancer Trial

o Includes treatment arm of NT219 which we combine for the first time with pembrolizumab (Keytruda) immunotherapy, as well as expansion arm of NT219 combined with cetuximab

o Collaboration with University of Colorado

o Latest patent in U.S. enhances global IP protection

The Phase 2 study will evaluate the efficacy and safety of NT219 which we combine for the first time with standard-of-care checkpoint inhibitors, such as pembrolizumab (Keytruda), for the treatment of recurrent/metastatic squamous cell carcinoma of the head and neck (R/M SCCHN) and in combination with epidermal growth factor receptor (EGFR) blockers, such as cetuximab (Erbitux), which demonstrated activity in Purple Biotech’s Phase 1 dose escalation study. The Phase 2 study, expected to begin in the first half of 2025, is designed with two single-arm cohorts: one will evaluate NT219 in combination with pembrolizumab, and the other will evaluate NT219 in combination with cetuximab for the treatment of R/M SCCHN. Additionally, the study will explore potential biomarkers identified in a previous clinical study of NT219. The investigator-initiated Phase 2 trial is led by Dr. Antonio Jimeno, Professor and Director of the Head and Neck Cancer Program at the University of Colorado Anschutz Medical Campus.

The U.S. Patent and Trademark Office issued a patent for NT219 used in combination with EGFR antibodies for treating cancer patients who have acquired resistance to EGFR therapies. This latest U.S. patent completes the geographic patent protection for NT219 used in combination with cetuximab in major markets, such as the United States, Europe, China and Japan. We believe this additional patent positions the Company well for the potential future commercialization of NT219.

● CAPTN-3 Tri-Specific Antibody Platform Preclinical Studies Advance Toward First-in-Human Clinical Trials

o New data presented at EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics

o Research collaboration with Icahn School of Medicine at Mount Sinai

New data on CAPTN-3 were presented at the 36th European Organization for Research and Treatment of Cancer, National Cancer Institute, American Association for Cancer Research (AACR) (Free AACR Whitepaper) (EORTC-NCI-AACR) (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics. CAPTN-3 demonstrated sustained tumor regression in a triple-negative breast cancer in-vivo model, as well as dose-dependent activity and a synergistic effect of the engager arms in non-small cell lung cancer patient-derived explants. IM1240, Purple’s lead tribody candidate, demonstrated that cytokine release is 5T4-dependent and suppressed by the conditionally activated capping technology, suggesting a potentially beneficial safety profile.

Purple Biotech entered into a Research Collaboration Agreement with the Icahn School of Medicine at Mount Sinai in New York to explore the immunoregulation of NK and T cells within the tumor microenvironment by CAPTN-3 multi-specific engagers, designed with the purpose of enhancing tumor-specific immunity against various cancer types. This collaboration offers an opportunity to deepen the understanding of tumor immune evasion mechanisms that CAPTN-3 uniquely addresses, with the goal of paving the way for effective treatments for many challenging tumor indications. Purple Biotech is working with Principal Investigator Amir Horowitz, PhD, and his team at Mount Sinai to validate the unique aspects of the CAPTN-3 design in a wide screen of patient-derived tumors, potentially providing new insights for overcoming resistance to standard frontline immunotherapies.

Financial Results for the Three Months Ended December 31, 2024

Research and Development Expenses were $0.5 million, a decrease of $4.7 million, or 90.4%, compared to $5.2 million in the same period of 2023, primarily due to reduced clinical trials expenses.

Sales, General and Administrative Expenses were $0.6 million, compared to $1.0 million in the same period of 2023, a decrease of $0.4 million, or 40%, primarily due to a decrease in salary and salary related expenses and share based payment expenses.

Operating Loss was $1 million, a decrease of $5.3 million, or 84.1%, compared to $6.3 million in the same period of 2023, primarily due to the decrease in research and development expenses.

Adjusted Operating Loss (as reconciled below) was $1 million, a decrease of $5 million, compared to $6.0 million in the same period of 2023, primarily due to the decrease in research and development expenses.

Net Loss for the three months ended December 31, 2024, was $0.4 million, or $XX loss per basic ADS and $XX loss per diluted ADS, compared to a net loss of $4.9 million, or $3.8 per basic and diluted ADS, in the same period of 2023. The decrease in net loss was primarily due to the decrease in research and development expenses.

As of December 31, 2024, Purple Biotech had cash and cash equivalents and short-term deposits of $8.2 million, providing the Company a cash runway into mid-2026.

During the three months ended December 31, 2024, the Company raised $2.8 million through a registered direct offering. In addition, during the period the Company sold approximately 298 thousand ADSs, at an average price of $3.5 per ADS, under the Open Market Sale Agreement with Jefferies LLC, resulting in net proceeds to the Company of approximately $1.5 million, net of issuance expenses.

Financial Results for the Twelve Months Ended December 31, 2024

Research and Development Expenses were $7.6 million, a decrease of $9.4 million, or 55.3%, compared to $17 million in the same period of 2023. The decrease was primarily due to reduced clinical trials expenses.

Sales, General and Administrative Expenses were $3.2 million, a decrease of $2.0, or 38.5%, compared to $5.2 million in the same period of 2023, primarily due to a decrease in salary and salary related expenses and share based payment expenses.

Operating Loss was $11 million, a decrease of $11.3 million, or 50.7%, compared to $22.3 million in the same period of 2023, primarily due to the decrease in research and development expenses.

Adjusted Operating Loss (as reconciled below) was $10.4 million, a decrease of $10 million, compared to $20.4 million in the same period of 2023, primarily due to the decrease in research and development expenses.

Net Loss for the year ended December 31, 2024 was $7.3 million, or $4.44 loss per basic ADS and $4.99 loss per diluted ADS, compared to a net loss of $20 million, or $17.96 loss per basic and diluted ADS, in the same period of 2023. The decrease in net loss was primarily due to a $11.3 million decrease in operating expenses and a $1.4 million increase in financial income net.

Non-IFRS Financial Measures.

This press release includes information about certain financial measures that are not prepared in accordance with International Financial Reporting Standards ("IFRS"), including adjusted operating loss. This non-IFRS measure is not based on any standardized methodology prescribed by IFRS and is not necessarily comparable to similar measures presented by other companies. Adjusted operating loss adjusts for non-cash share-based compensation expenses. The Company’s management and board of directors utilize this non-IFRS financial measure to evaluate the Company’s performance. The Company provides this non-IFRS measure of the Company’s performance to investors because management believes that this non-IFRS financial measure, when viewed with the Company’s results under IFRS and the accompanying reconciliations, is useful in identifying underlying trends in ongoing operations. However, this non-IFRS measure is not a measure of financial performance under IFRS and, accordingly, should not be considered as an alternative to IFRS measures as indicators of operating performance. Further, this non-IFRS measure should not be considered a measure of the Company’s liquidity. A reconciliation of certain IFRS to non-IFRS financial measures has been provided in the tables included in this press release.