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.

On March 10, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported Walter Klemp, Chairman and Chief Executive Officer of Moleculin will participate in a fireside chat on Tuesday, March 18, 2025 at 11:00 AM PT at the 37th Annual ROTH Conference being held in Dana Point, CA (Press release, Moleculin, MAR 10, 2025, View Source [SID1234651042]).

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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 in the Investors section of the Company’s website (moleculin.com).

On March 10, 2025 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for serious neurological conditions, reported its fourth quarter and full year 2024 financial and operating results and will host a conference call today at 4:30 p.m. Eastern Time to discuss these results and provide a business update (Press release, Lineage Cell Therapeutics, MAR 10, 2025, View Source [SID1234651041]).

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"Throughout 2024, we made substantial progress across multiple fronts, advancing our programs, expanding collaborations, and strengthening our balance sheet to support significant anticipated milestones," stated Brian M. Culley, Lineage’s CEO. "Our lead asset OpRegen continues to make progress in the ongoing GAlette Study, and we are encouraged by Roche and Genentech’s commitment to the OpRegen program. Our partner’s efforts to advance OpRegen for the treatment of dry AMD with GA include adding clinical sites and seeking and obtaining RMAT designation. We view these actions as positive indicators for this pioneering cell transplant and look forward to further updates on the program."

"We anticipate our internal programs will similarly advance through important milestones during 2025," added Mr. Culley. "We will focus on conducting the recently-initiated DOSED clinical study of a novel OPC1 delivery system, advancing ReSonance for the treatment of sensorineural hearing loss, and advancing other carefully selected early-stage initiatives. In parallel, we will continue our efforts to demonstrate an in-house manufacturing capability that supports Lineage having an industry-leading position in allogeneic cell banking and production. As the power of our cell differentiation and manufacturing platforms are further demonstrated and validated, we believe that our pipeline of internally-owned assets and cell-based know-how will make us a desirable development partner and an attractive opportunity for investors."

Select Business Highlights

– RG6501 (OpRegen)

Ongoing execution of Lineage’s contributions to our collaboration with Roche and Genentech, a member of the Roche Group, across multiple functional areas, including support for the ongoing Phase 2a clinical study (the "GAlette Study") in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD), and expansion of the clinical study to sites in Israel.
Roche announced receipt of RMAT designation from the U.S. FDA for OpRegen, for the treatment of GA secondary to AMD.
Entered into a separate services agreement with Genentech to further support development of OpRegen, including: (i) activities to support the ongoing Phase 1/2a study and currently enrolling GAlette Study; and (ii) additional technical training and materials related to our cell therapy technology platform to support commercial manufacturing strategies.
Positive clinical data from long-term follow-up of patients from the Phase 1/2a clinical study of OpRegen featured at the 2024 Retinal Cell & Gene Therapy Innovation Summit.
– OPC1

Submitted an Investigational New Drug Amendment (INDa) for OPC1 to enable initiation of DOSED (Delivery of Oligodendrocyte Progenitor Cells for Spinal Cord Injury: Evaluation of a Novel Device) clinical study in subacute and chronic spinal cord patients.
DOSED study initiated in February 2025; UC San Diego Health named as the first participating study site.
Lineage and OPC1 program featured on CNN: "He was paralyzed his last day of high school. How an experimental trial is showing ‘unexpected improvement."
Created and hosted the 2nd Annual Spinal Cord Injury Investor Symposium in partnership with the Christopher & Dana Reeve Foundation, with additional grant support from CIRM.
The goals of this collaborative effort include increasing disease awareness, improving the probability of success in product development, and supporting clinical trial participation. Presenting companies have included AbbVie, Mitsubishi Tanabe, Neuralink, NervGen Pharma and ONWARD.
– ReSonance (ANP1)

Preclinical results presented at 59th Annual Inner Ear Biology Workshop.
ReSonance manufactured by a proprietary process, developed in-house, at clinical scale, with relevant in-vitro functional activity.
Immediate-use, thaw-and-inject formulation durably engrafted in multiple preclinical hearing loss models.
ReSonance is currently being evaluated in a functional model of hearing loss through a collaboration with the University of Michigan Kresge Hearing Research Institute.
– Corporate

Closed two financings totaling $44 million in gross proceeds; potential to receive an additional $36 million in gross proceeds upon the full cash exercise of OpRegen clinical milestone-linked warrants.
Balance Sheet Highlights

Cash, cash equivalents, and marketable securities of $47.8 million as of December 31, 2024, together with the approximate $5.5 million in net proceeds from the second closing under our November 2024 registered direct offering completed in January 2025, is expected to support planned operations into Q1 2027.

Fourth Quarter Operating Results

Revenues: Revenue is generated primarily from collaboration revenues, royalties, and other revenues. Total revenues for the three months ended December 31, 2024 were approximately $2.9 million, a net increase of $0.8 million as compared to $2.1 million for the same period in 2023. The increase was primarily driven by more collaboration revenue recognized from deferred revenues under the collaboration and license agreement with Roche.

Operating Expenses: Operating expenses are comprised of research and development ("R&D") expenses and general and administrative ("G&A") expenses. Total operating expenses for the three months ended December 31, 2024 were $7.8 million, a decrease of $0.4 million as compared to $8.2 million for the same period in 2023.

R&D Expenses: R&D expenses for the three months ended December 31, 2024 were $3.4 million, a decrease of $0.5 million as compared to $3.9 million for the same period in 2023. The net decrease was primarily driven by $1.2 million for our OPC1 program expenses and $0.2 million for other research and development expense programs, partially offset by $0.4 million for our OpRegen program expenses and $0.5 million for our preclinical programs.

G&A Expenses: G&A expenses for the three months ended December 31, 2024 of $4.4 million were primarily in line with expenses for the same period in 2023.

Loss from Operations: Loss from operations for the three months ended December 31, 2024 was $5.1 million, a decrease of $1.3 million as compared to $6.4 million for the same period in 2023.

Other Income/(Expenses): Other income/(expenses) for the three months ended December 31, 2024 reflected other income of $1.8 million, compared to other income of $1.6 million for the same period in 2023. The net increase was primarily driven by changes in fair value of warrant liability, largely offset by exchange rate fluctuations related to Lineage’s international subsidiaries and certain warrant-related transaction costs incurred as part of the November 2024 financing.

Net Loss Attributable to Lineage: The net loss attributable to Lineage for the three months ended December 31, 2024 was $3.3 million, or $0.02 per share (basic and diluted), compared to a net loss of $4.8 million, or $0.03 per share (basic and diluted), for the same period in 2023.

Full Year Operating Results

Revenues: Revenue is generated primarily from collaboration revenues, royalties, and other revenues. Total revenues for the year ended December 31, 2024 were $9.5 million, a net increase of $0.6 million as compared to $8.9 million for the same period in 2023. The increase was primarily driven by more collaboration revenue recognized from deferred revenues under the collaboration and license agreement with Roche.

Operating Expenses: Operating expenses are comprised of R&D expenses and G&A expenses. Total operating expenses for the year ended December 31, 2024 were $31.0 million, a decrease of $2.7 million as compared to $33.7 million for the same period in 2023.

R&D Expenses: R&D expenses for the year ended December 31, 2024 were $12.5 million, a decrease of $3.2 million as compared to $15.7 million for the same period in 2023. The decrease was primarily driven by $2.7 million for our OPC1 program expenses, $1.1 million for our preclinical and other research and development programs. These decreases were partially offset by $0.6 million for our OpRegen program.

G&A Expenses: G&A expenses for the year ended December 31, 2024 were $18.2 million, an increase of approximately $0.9 million as compared to $17.3 million for the same period in 2023. The net increase was primarily driven by $0.6 million for stock-based compensation expense and $0.4 million for personnel costs, partially offset by an overall decrease in costs incurred for services provided by third parties.

Loss from Operations: Loss from operations for the year ended December 31, 2024 was $21.5 million, a decrease of $3.2 million as compared to $24.7 million for the same period in 2023.

Other Income/(Expenses): Other income (expenses) for the year ended December 31, 2024 reflected other income of $2.9 million, compared to other income of $1.5 million for the same period in 2023. The net increase of $1.4 million was primarily driven by changes in the fair value of warrant liability, exchange rate fluctuations related to Lineage’s international subsidiaries, and fair market value changes in marketable equity securities. This increase in other income was partially offset by certain warrant-related transaction costs incurred as part of the November 2024 financing, as well as the non-recurring prior year employee retention credit.

Net Loss Attributable to Lineage: The net loss attributable to Lineage for the year ended December 31, 2024 was $18.6 million, or $0.09 per share (basic and diluted), compared to a net loss of $21.5 million, or $0.12 per share (basic and diluted), for 2023.

Conference Call and Webcast

Interested parties may access the conference call on March 10, 2025, by dialing (800) 715-9871 from the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through March 17th, 2025, by dialing (800) 770-2030 from the U.S. and Canada and entering conference ID number 6707203.

On March 10, 2025 Genmab A/S (Nasdaq: GMAB) reported that Johnson & Johnson (J&J) has decided that it will not exercise its option to receive a worldwide license to develop, manufacture and commercialize HexaBody-CD38 (GEN3014) (Press release, Genmab, MAR 10, 2025, View Source [SID1234651040]). While the initial HexaBody-CD38 clinical data is promising and showed robust clinical efficacy, following a thorough evaluation of the data, the market landscape, and Genmab’s rigorous portfolio prioritization, Genmab will not pursue further clinical development of HexaBody-CD38.

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"While we are disappointed that J&J has decided not to advance HexaBody-CD38, the data confirms the clinical potential of the HexaBody platform, reinforcing its value for future applications," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab. "With EPKINLY (epcoritamab) moving from strength to strength and two wholly owned assets, rinatabart sesutecan (Rina-S) and acasunlimab in Phase 3 development, we are confident in the potential of our existing pipeline of innovative antibody therapeutics. Genmab intends to maintain its laser sharp focus on and disciplined investments in our promising late-stage proprietary clinical pipeline and continues to execute against our capital allocation framework ensuring future growth."

As stipulated by the development and option agreement between Genmab and J&J for HexaBody-CD38, Genmab provided J&J with data from a clinical proof-of-concept study in multiple myeloma, including a head-to-head comparison with DARZALEX FASPRO (daratumumab and hyaluronidase fihj).

The Phase 2 expansion Part B of the study assessed the objective response rate (primary endpoint) of intravenous HexaBody-CD38 versus subcutaneous daratumumab in patients with anti-CD38 antibody- naïve relapsed or refractory multiple myeloma.

Preliminary data submitted by Genmab to J&J, inclusive of 88 patients who received a study treatment and 84 patients who were response evaluable (42 in each arm), demonstrated an overall response rate (ORR) of 55% (95% CI: 39%, 70%) in the HexaBoby-CD38 IV arm vs. 52% in the daratumumab SC arm (95% CI: 36%, 68%); very good partial response (VGPR) or better rate was 29% vs. 17%; and complete response (CR) or better rate was 7% vs. 2%.

Due to the relatively short follow-up time, secondary efficacy endpoints including duration of response, progression-free survival, and overall survival were not mature yet. Treatment emergent Adverse events (TEAEs) above 20% in the Hexabody-CD38 arm were neutropenia, infusion related reactions, anemia, and thrombocytopenia. No new safety findings were observed in the daratumumab arm of the study. TEAEs leading to death included one patient in the HexaBody-CD38 IV arm and two patients in the daratumumab SC arm; none of these deaths was related to the study treatment. Follow-up is ongoing and more mature data will be presented at a future medical conference.

This news does not impact Genmab’s 2025 Financial Guidance.

Conference Call Details
Genmab will host a conference call to discuss this event today at 5:00 PM CET / 4:00 PM GMT / 12:00 PM EDT. To join the call or listen to the webcast, please register using the following link: View Source

An archived webcast of the call will be available at View Source

About the 3014-01 Trial
3014-01 is a Phase 1/2, open-label, multi-center trial to evaluate the safety and preliminary efficacy of HexaBody-CD38 as a monotherapy in patients with relapsed or refractory multiple myeloma and other blood cancers. The trial consists of three parts: a dose-escalation phase (Phase 1) and an expansion phase (Part A and Part B). The primary objective of Phase 1 is to determine the recommended Phase 2 dose and the maximum tolerated dose as well as establish the safety profile of HexaBody-CD38 monotherapy. The purpose of Phase 2 Expansion Part A is to assess the objective response rate of HexaBody-CD38 for patients with relapsed or refractory multiple myeloma and other blood cancers. The purpose of Phase 2 Expansion Part B is to assess the objective response rate of HexaBody-CD38 versus subcutaneous daratumumab in patients with CD38 antibody naïve relapsed or refractory multiple myeloma. More information on this trial can be found at View Source (NCT04824794).

On March 10, 2025 Fennec Pharmaceuticals Inc. (NASDAQ:FENC; TSX: FRX), a specialty pharmaceutical company, reported its financial results for the fiscal year ended December 31, 2024 and provided a business update (Press release, Fennec Pharmaceuticals, MAR 10, 2025, View Source [SID1234651038]).

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"2024 marked the beginning of a foundational transformation for Fennec, setting the stage for the PEDMARK strategy that we are utilizing throughout 2025 to realize our next phase of growth. With key management and commercial hires in Q3 and Q4, we strengthened our leadership team and with this enhanced expertise, we are now well-positioned to drive execution and excellence in the field. We are seeing encouraging momentum in early 2025, particularly with adoption by academic institutions and new patient segments, reinforcing the value and need for PEDMARK," said Jeff Hackman, chief executive officer of Fennec Pharmaceuticals. "Global access to PEDMARK has also expanded meaningfully, with recent PEDMARQSI commercial launches in the United Kingdom and Germany in 2025. With the right foundational strategies now in place, we are confident that our strong and focused execution will translate into significant shareholder value in 2025 and beyond."

Business Highlights:

● U.S. Clinical Compendia Update: All medical compendia have incorporated Fennec’s clinical updates, and AHFS, the largest online platform for pharmacists, has updated its content to reflect and differentiate PEDMARK in accordance with its labeling. We also continue to advance our efforts to have PEDMARK added to the NCCN Drug and Biologics Compendium, a key step in further expanding access and reimbursement pathways.

● PEDMARQSIÒ Commercial Launch in Europe: In December 2024, Norgine received positive guidance from National Institute for Health and Care Excellence (NICE) recommending PEDMARQSI for the prevention of cisplatin-induced hearing loss in patients (aged 1 month to 17 years) with localized, non-metastatic, solid tumors and PEDMARQSI is currently available in the U.K. In February 2025, Norgine announced that it has commercially launched PEDMARQSI in Germany. Both milestones mark an important step in achieving Fennec’s mission of expanding access to PEDMARQSI to cancer patients across the globe at risk of hearing loss.

● Ex-U.S. Opportunities for PEDMARKÒ: In Japan, the investigator-initiated clinical trial (STS-J01) in Japan evaluating PEDMARKÒ fully enrolled in Q4 2024 and the results of the trial are expected in the second half of 2025 with the potential evaluation for registration of PEDMARK in Japan thereafter. Further, Fennec has partnered with Inpharmus, formerly named TRPharm İlaç Sanayi Ticaret A.Ş. and TRPharm FZ-LLC, for the distribution of PEDMARK in Turkey and Gulf Cooperation Council Countries.

●Early Repayment of $13 Million of the Company’s Approximately $32 Million Outstanding Convertible Debt Facility: In December 2024, Fennec announced the early partial repayment of a significant portion of its debt to Petrichor in a financial and strategic action that optimizes the Company’s balance sheet and overall capital structure, while effectively saving approximately $1.5 million in future annual interest payments and eliminating potential dilutive shares.

Financial Results for the Fourth Quarter and Full Fiscal Year Ended December 31, 2024

● Net Product Sales – For the fourth quarter of 2024, the Company recorded net product sales of $7.9 million compared to $7.0 million in the third quarter of 2024, representing an increase of approximately 13%. For the full fiscal year (FY) 2024, the Company recorded $29.6 million compared to $21.3 million in 2023, representing an increase of approximately 40%. The increase in sales in both the quarter and year reflects strong growth in accounts and increased penetration in the AYA market opportunity.

● Cash Position – Cash and cash equivalents were $26.6 million as of December 31, 2024. Of note, for the fourth quarter of 2024, our cash flow from operations decreased by $0.6 million. For the FY 2024, there was a $13.4 million increase in cash and cash equivalents between December 31, 2023 and December 31, 2024. The increase in cash was primarily due to the $43 million in upfront cash from the Norgine transaction and cash collected from product sales offset by operating expenses and the $13 million convertible debt paydown in December 2024.
● Selling and Marketing Expenses – The Company recorded $3.9 million in selling and marketing expenses in the fourth quarter of 2024 compared to $4.6 million in the third quarter of 2024. For the FY 2024, the Company recorded $18.4 million in selling and marketing compared to $12.1 million in fiscal year 2023. The increase is largely related to increased payroll and additional marketing expenses in the comparable periods as we focused on expanding our outreach to community oncology centers and the adolescent and young adult (AYA) population.

● General and Administrative (G&A) Expenses – The Company recorded $4.1 million in G&A expenses fourth quarter of 2024 compared to $7.0 million in the third quarter of 2024. For the FY 2024, the Company recorded $23.1 million in G&A expenses compared to $20.6 million in fiscal year 2023. For the fourth quarter of 2024, G&A expenses decreased due largely to lower cash based stock compensation and a one-time severance payment related to the previous CEO in the third quarter. For the full year G&A expenses were higher both due to European pre-commercialization related expenses, expenses associated with the Norgine transaction and intellectual property expenses related to ongoing litigation.

Fourth Quarter and Full-Year 2024 Conference Call Information

Date: Monday, March 10, 2025

Time: 8:30 a.m. ETWebcast Link: View Source

Participant Link: https://register.vevent.com/register/BIeb244773eed644bd83882935e4272e91

To access the live webcast link, log onto www.fennecpharma.com and proceed to the News & Events/Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the conference call to ensure adequate time for any software download that may be required to listen to the webcast. A webcast replay of the conference call will also be archived on www.fennecpharma.com for thirty days.

Financial Update

The selected financial data presented below is derived from our unaudited condensed consolidated financial statements, which were prepared in accordance with U.S. generally accepted accounting principles. The complete audited condensed consolidated financial statements for the period ended December 31, 2024 and management’s discussion and analysis of financial condition and results of operations will be available via www.sec.gov and www.sedar.com. All values are presented in thousands unless otherwise noted.

Unaudited Consolidated

Statements of Operations:

(U.S. Dollars in thousands except per share amounts

Three Months Ended

Twelve Months Ended

December 31,

December 31,

December 31,

December 31,

2024

2023

2024

2023

Revenue

Product sales, net

$

7,925

$

9,735

$

29,580

$

21,252

Licensing revenue

—

—

17,958

—

Total revenue

7,925

9,735

47,538

21,252

Operating expenses:

Cost of product sales

669

685

3,184

1,259

Research and development

50

32

307

56

Selling and marketing

3,944

3,868

18,426

12,123

General and administrative

4,196

6,968

23,053

20,585

Total operating expenses

8,859

11,553

44,970

34,023

Loss from operations

(934)

(1,818)

2,568

(12,771)

Other (expense)/income

Realized foreign exchange (loss)/gain

(27)

2

(82)

5

Amortization expense

(25)

(70)

(89)

(287)

Unrealized loss on securities

(66)

4

(80)

(39)

Interest income

399

115

1,682

441

Interest expense

(966)

(915)

(4,069)

(3,394)

Total other (expense)/income

(685)

(864)

(2,638)

(3,274)

Net (loss) / income

$

(1,619)

$

(2,682)

$

(70)

$

(16,045)

Basic net loss per common share

$

(0.06)

$

(0.10)

$

(0.00)

$

(0.60)

Diluted net loss per common share

$

(0.06)

$

(0.10)

$

(0.00)

$

(0.60)

Weighted-average number of common shares outstanding basic

27,460

26,833

27,294

26,574

Weighted-average number of common shares outstanding diluted

27,460

26,833

27,294

26,574

December 31,

December 31,

2024

2023

Assets

Current assets

Cash and cash equivalents

$

26,634

$

13,269

Accounts receivable, net

12,884

8,814

Prepaid expenses

3,080

583

Inventory

1,060

2,156

Other current assets

364

21

Total current assets

44,022

24,843

Non-current assets

Other non-current assets, net of amortization

924

2,021

Total non-current assets

924

2,021

Total assets

$

44,946

$

26,864

Liabilities and shareholders’ (deficit) equity

Current liabilities:

Accounts payable

$

2,875

$

3,778

Accrued liabilities

3,428

3,754

Operating lease liability – current

248

21

Contract liability – current

2

—

Total current liabilities

6,553

7,553

Long term liabilities

Term loan

18,206

30,000

PIK interest

1,271

1,219

Debt discount

(139)

(288)

Contract liability – long-term

24,561

2

Total long term liabilities

43,899

30,933

Total liabilities

50,452

38,486

Commitments and Contingencies

Shareholders’(deficit) equity:

Common stock, no par value; unlimited shares authorized; 27,292 shares issued and outstanding (2023 -26,361)

145,608

144,307

Additional paid-in capital

66,958

62,073

Accumulated deficit

(219,315)

(219,245)

Accumulated other comprehensive income

1,243

1,243

Total shareholders’ (deficit) equity

(5,506)

(11,622)

Total liabilities and shareholders’ (deficit) equity

$

44,946

$

26,864

Working capital

Fiscal Year Ended

Selected Asset and Liability Data:

December 31, 2024

December 31, 2023

(U.S. Dollars in thousands)

Cash and equivalents

$

26,634

$

13,269

Other current assets

17,388

11,574

Current liabilities

6,553

7,553

Working capital

$

37,469

$

17,290

Selected Equity:

Common stock and additional paid in capital

212,566

206,380

Accumulated deficit

(219,315)

(219,245)

Shareholders’ equity

(5,506)

(11,622)

About Cisplatin-Induced Ototoxicity

Cisplatin and other platinum compounds are essential chemotherapeutic agents for the treatment of many malignancies. Unfortunately, platinum-based therapies can cause ototoxicity, or hearing loss, which is permanent, irreversible, and particularly harmful to the survivors of pediatric cancer.i

The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids or cochlear implants, which can be helpful for some, but do not reverse the hearing loss and can be costly over time.ii Infants and young children that are affected by ototoxicity at critical stages of development lack speech and language development and literacy, and older children and adolescents often lack social-emotional development and educational achievement.iii

PEDMARK (sodium thiosulfate injection)

PEDMARK is the first and only U.S. Food and Drug Administration (FDA) approved therapy indicated to reduce the risk of ototoxicity associated with cisplatin treatment in pediatric patients with localized, non-metastatic, solid tumors. It is a unique formulation of sodium thiosulfate in single-dose, ready-to-use vials for intravenous use in pediatric patients. PEDMARK is also the first and only therapeutic agent with proven efficacy and safety data with an established dosing regimen, across two open-label, randomized Phase 3 clinical studies, the Children’s Oncology Group (COG) Protocol ACCL0431 and SIOPEL 6.

As a reminder, PEDMARK is indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors. PEDMARK is recommended for the AYA population by the National Comprehensive Cancer Network, or NCCN, with a 2A endorsement.

In the U.S. and Europe, it is estimated that, annually, more than 10,000 children may receive platinum-based chemotherapy. The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids. There is currently no established preventive agent for this hearing loss and only expensive, technically difficult, and sub-optimal cochlear (inner ear) implants have been shown to provide some benefit. Infants and young children that suffer ototoxicity at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement.

PEDMARK has been studied by co-operative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, COG ACCL0431 and SIOPEL 6. Both studies have been completed. The COG ACCL0431 protocol enrolled childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, medulloblastoma, and other solid tumors. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors.

Indications and Usage

PEDMARK (sodium thiosulfate injection) is indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors.

Limitations of Use

The safety and efficacy of PEDMARK have not been established when administered following cisplatin infusions longer than 6 hours. PEDMARK may not reduce the risk of ototoxicity when administered following longer cisplatin infusions, because irreversible ototoxicity may have already occurred.

Important Safety Information

PEDMARK is contraindicated in patients with history of a severe hypersensitivity to sodium thiosulfate or any of its components.

Hypersensitivity reactions occurred in 8% to 13% of patients in clinical trials. Monitor patients for hypersensitivity reactions. Immediately discontinue PEDMARK and institute appropriate care if a hypersensitivity reaction occurs. Administer antihistamines or glucocorticoids (if appropriate) before each subsequent administration of PEDMARK. PEDMARK may contain sodium sulfite; patients with sulfite sensitivity may have hypersensitivity reactions, including anaphylactic symptoms and life-threatening or severe asthma episodes. Sulfite sensitivity is seen more frequently in people with asthma.

PEDMARK is not indicated for use in pediatric patients less than 1 month of age due to the increased risk of hypernatremia or in pediatric patients with metastatic cancers.

Hypernatremia occurred in 12% to 26% of patients in clinical trials, including a single Grade 3 case. Hypokalemia occurred in 15% to 27% of patients in clinical trials, with Grade 3 or 4 occurring in 9% to 27% of patients. Monitor serum sodium and potassium levels at baseline and as clinically indicated. Withhold PEDMARK in patients with baseline serum sodium greater than 145 mmol/L.

Monitor for signs and symptoms of hypernatremia and hypokalemia more closely if the glomerular filtration rate (GFR) falls below 60 mL/min/1.73m2.

Administer antiemetics prior to each PEDMARK administration. Provide additional antiemetics and supportive care as appropriate.

The most common adverse reactions (≥25% with difference between arms of >5% compared to cisplatin alone) in SIOPEL 6 were vomiting, nausea, decreased hemoglobin, and hypernatremia. The most common adverse reaction (≥25% with difference between arms of >5% compared to cisplatin alone) in COG ACCL0431 was hypokalemia.

Please see full Prescribing Information for PEDMARK at: www.PEDMARK.com.