On March 10, 2025 enGene Holdings Inc. (Nasdaq: ENGN, or "enGene" or the "Company"), a clinical-stage, non-viral genetic medicines company, reported its financial results for the first quarter ended January 31, 2025, and provided a business update (Press release, enGene, MAR 10, 2025, View Source [SID1234651054]).

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"With the expansion of LEGEND study sites into Europe and Asia, enrollment in our pivotal cohort continues to track in-line with our plan to file a BLA for detalimogene in mid-2026," said Ron Cooper, Chief Executive Officer of enGene. "We look forward to the remainder of 2025, during which we expect to announce enrollment completion of LEGEND’s pivotal cohort and provide additional updates on other LEGEND study cohorts. We continue to believe in detalimogene’s opportunity to transform the treatment landscape of NMIBC through a unique and differentiated overall profile that is highly attractive to both patients and physicians due to its potential for tolerability, ease-of-use, and efficacy."

Recent Corporate Updates

LEGEND study trial site expansion: Over the course of the first quarter of 2025, the Company expanded its clinical footprint for the LEGEND study with the addition of trial sites in Europe and Asia. In addition, all four of LEGEND’s cohorts are now open for enrollment, including the pivotal cohort evaluating detalimogene voraplasmid (also known as detalimogene, and previously EG-70) in high-risk, BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with CIS as well as Cohort 2a and 2b (i.e., NMIBC patients with CIS who are naïve to treatment with BCG and NMIBC patients with CIS who have been exposed to BCG, but have not received adequate BCG treatment), and Cohort 3 (BCG-unresponsive high-risk NMIBC patients with papillary-only disease). Global enrollment is taking place under an amended protocol that is designed to align with the American Urological Association’s current treatment guidelines and real-world practices.

LEGEND study now includes maintenance dosing: The amended LEGEND protocol allows patients from all cohorts who are in complete response at 12 months to continue receiving detalimogene on a dose-reduced maintenance schedule throughout their second year of treatment. At the end of year two, patients may elect to remain on maintenance therapy for another year, for a total of three years of therapy. Maintenance treatment consists of two instillations of detalimogene per three-month cycle, administered at week one and at week two of each cycle. The maintenance regimen is intended to evaluate the potential of longer-term detalimogene treatment to improve or maintain durability of response with the lower patient burden associated with a less frequent dosing regimen.

First Quarter 2025 Financial Results

As of January 31, 2025, cash, cash equivalents and marketable securities were $272.8 million. The Company expects that its existing cash, cash equivalents and marketable securities will fund operating expenses, debt obligations and capital expenditures into 2027.

Three Months ended January 31, 2025

Total operating expenses were $26.6 million for the three months ended January 31, 2025, compared to $10.8 million for the three months ended January 31, 2024. Research and development expenses increased by $14.3 million, mainly due to increasing manufacturing and clinical costs related to our pivotal LEGEND study and headcount costs. General and administrative expenses increased by $1.5 million, primarily driven by increased headcount intended to support the Company’s operation as a publicly traded company.

For the three months ended January 31, 2025, net loss attributable to common shareholders was approximately $24.6 million, or $0.48 per share, compared to approximately $10.7 million, or $0.46 per share, for the same period for the three months ended January 31, 2024. The increase in net loss is mainly attributed to the increase in operating expenses, partially offset by net interest income earned during the period.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Non-muscle invasive bladder cancer (NMIBC) is a disease with a significant patient burden, high clinical needs, and massive economic impact on our healthcare system. NMIBC occurs when cancer cells grow in the tissues that line the interior of the bladder, but the cancer has not yet penetrated deeper into the muscle of the bladder wall. About 75-80% of new bladder cancer diagnoses are NMIBC. Patients suffering from high-risk NMIBC who are unresponsive to the standard of care, Bacillus Calmette-Guérin (BCG), face high rates of disease recurrence (50-70%) and are subject to full removal of the bladder (cystectomy) as a curative but life-altering next step.

About Detalimogene

Detalimogene is a novel, investigational, non-viral genetic medicine for patients with high-risk, non-muscle invasive bladder cancer (NMIBC), including Bacillus Calmette-Guérin (BCG)-unresponsive disease. It is designed to be instilled in the bladder and elicit a powerful yet localized anti-tumor immune response.

Detalimogene was developed using the Company’s Dually Derivatized Oligochitosan (DDX) platform, a technology designed to transform how gene therapies are accessed by patients and utilized by clinicians. Medicines developed with the DDX platform can potentially overcome the limitations of viral-based gene therapies, simplify safe handling and cold storage complexities, and streamline both manufacturing processes and administration paradigms.

Detalimogene has received Fast Track designation from the U.S. Food and Drug Administration (FDA) based on its potential to address the high unmet medical need for patients with BCG-unresponsive carcinoma in situ (CIS) NMIBC with or without resected papillary tumors who are unable to undergo cystectomy. Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

About the Pivotal LEGEND Trial

Detalimogene is being evaluated in the ongoing, open-label, multi-cohort, Phase 2 LEGEND trial to establish its safety and efficacy in high-risk, non-muscle invasive bladder cancer (NMIBC). LEGEND’s pivotal cohort (Cohort 1) consists of approximately 100 patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) (with or without papillary disease) and is designed to serve as the basis of the Company’s planned Biologics License Application (BLA) filing. In addition to this pivotal cohort, three additional cohorts are actively enrolling patients, including NMIBC patients with CIS who are naïve to treatment with BCG (Cohort 2a); NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment (Cohort 2b); and BCG-unresponsive high-risk NMIBC patients with papillary-only disease (Cohort 3). The LEGEND trial is actively enrolling patients with sites participating in the USA, Canada, Europe, and the Asia-Pacific region

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.

On March 10, 2025 Agilent Technologies Inc. (NYSE: A) reported its PD-L1 IHC 28-8 pharmDx kit has received two new companion diagnostic indications approvals under EU IVDR1, expanding the eligibility of treatment to early-stage non-small cell lung cancer (NSCLC) and previously untreated advanced melanoma patients (Press release, Agilent Technologies, MAR 10, 2025, View Source [SID1234651055]). These two new indications bring the total indications launched in Europe for PD-L1 IHC 28-8 pharmDx to nine. PD-L1 IHC 28-8 pharmDx is approved for exclusive use with the Agilent Autostainer Link 48 advanced staining solution.

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Lung cancer and malignant melanoma are major healthcare concerns worldwide, with lung cancer accounting for over two million new cases in 2020 and an estimated 1.77 million deaths each year, and malignant melanoma accounting for over 324,000 new cases in 2020 and over 57,000 deaths each year2. PD-L1 is a critical biomarker for potential response to anti-PD-1 therapies, which are revolutionizing the treatment of cancer. Pathology labs play an important role in informing treatment decisions.

When used in conjunction with the PD-L1 IHC 28-8 pharmDx as a companion test, in the European Union: (a) resectable NSCLC patients with PD-L1 expression ≥1% and at high risk of recurrence may be eligible for treatment with Bristol Myers Squibb’s OPDIVO (nivolumab) in combination with platinum based chemotherapy; and (b) patients 12 years of age and older with tumor cell PD-L1 expression < 1% that have previously untreated advanced (metastatic or unresectable) melanoma may be eligible for treatment with Bristol Myers Squibb’s Opdualag (nivolumab and relatlimab).

PD-L1 IHC 28-8 pharmDx is the only clinically validated test for identifying patients for these treatments; these two new indications will aid pathologists, in conjunction with oncologists, in selecting appropriate treatment options, offering hope for patients diagnosed with these cancers. IVDR compliance certification further enhances the confidence of patients, consumers, and healthcare professionals in the EU by demonstrating that these medical devices can be safely relied upon as part of the diagnostic workflow.

Simon May, senior vice president of Agilent’s Life Sciences and Diagnostics Markets Group, remarked: "The two added indications of PD-L1 IHC 28-8 pharmDx will give physicians in Europe critical information to inform treatment decisions for patients with these common and potentially deadly cancers. This endorsement underscores Agilent’s leadership in the development of companion diagnostics for groundbreaking therapies containing anti-PD-1 antibodies."

An innovative industry leader with more than 50 years of experience, Agilent launched the first FDA-approved companion diagnostic and continues to deliver world-class CDx products in close collaboration with pharma partners.

OPDIVO is a registered trademark of Bristol-Myers Squibb Company; Opdualag is a trademark of Bristol-Myers Squibb Company.

In Vitro Diagnostic Medical Devices Regulation (europa.eu)
Sung. H., Ferlay. J., et al. Global Cancer Statistics 2020: GLOBOCAN Estimates of Incidence and Mortality Worldwide for 36 Cancers in 185 Countries, CA. Cancer J. Clin. 2021, 71, 209–249

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 9, 2025 Sun Pharmaceutical Industries Limited (Reuters: SUN.BO, Bloomberg: SUNP IN, NSE: SUNPHARMA, BSE: 524715 (together with its subsidiaries and/or associated companies, "Sun Pharma")) and Checkpoint Therapeutics, Inc. (Nasdaq: CKPT) ("Checkpoint") reported that they have entered into an agreement by which Sun Pharma will acquire Checkpoint, an immunotherapy and targeted oncology company (Press release, Sun Pharma, MAR 9, 2025, View Source [SID1234651025]).

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Checkpoint is a Nasdaq-listed commercial-stage company focused on developing novel treatments for patients with solid tumor cancers. Checkpoint has received approval from the U.S. Food & Drug Administration (FDA) for UNLOXCYT (cosibelimab-ipdl) for the treatment of adults with metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC who are not candidates for curative surgery or curative radiation.

Dilip Shanghvi, Chairman & Managing Director of Sun Pharma, said, "Combining UNLOXCYT, an FDA-approved anti-PD-L1 treatment for advanced cutaneous squamous cell carcinoma, with Sun Pharma’s global presence means patients with cSCC may soon have access to an important, new treatment option. The acquisition further bolsters our innovative portfolio in onco-derm therapy."

"I am proud of the dedication and passion of our team at Checkpoint that allowed us to achieve the first and only FDA-approved anti-PD-L1 treatment for patients with advanced cSCC, and we are excited to enter this transaction with Sun Pharma as the next step to bringing UNLOXCYT to cSCC patients in need of a differentiated immunotherapy treatment option," said James Oliviero, President and Chief Executive Officer of Checkpoint. "Sun Pharma is aligned with Checkpoint’s commitment to improving the lives of skin cancer patients, and I believe this transaction will maximize value for our stockholders and provide accelerated access to UNLOXCYT in the United States, Europe and other markets worldwide."

Transaction Summary

Upon completion of the transaction, Sun Pharma will acquire all outstanding shares of Checkpoint and Checkpoint stockholders will receive, for each share of common stock they hold, an upfront cash payment of $4.10, without interest, and a non-transferable contingent value right (CVR) entitling the stockholder to receive up to an additional $0.70 in cash, without interest, if cosibelimab is approved prior to certain deadlines in the European Union pursuant to the centralized approval procedure or in Germany, France, Italy, Spain or the United Kingdom, subject to the terms and conditions in the contingent value rights agreement.

The upfront cash payment of $4.10 per share of common stock represents a premium of approximately 66.0% to Checkpoint’s closing share price on March 7, 2025, the last trading day prior to today’s announcement.

In connection with the transaction, Checkpoint, Sun Pharma and Fortress Biotech, Inc., Checkpoint’s controlling stockholder ("Fortress"; Nasdaq: FBIO), have entered into a royalty agreement, under which following the closing of the transaction Fortress would be entitled to receive royalty payments based on future sales of cosibelimab during a specified term, in lieu of royalty rights that were granted to Fortress in connection with its founding of Checkpoint.

In connection with the evaluation of Checkpoint’s strategic alternatives, the Checkpoint board of directors (the "Board") formed a special committee of independent and disinterested directors (the "Special Committee"), which led the review and negotiations for this transaction. The Special Committee, with the assistance of its independent financial and legal advisors, conducted a comprehensive review of potential strategic alternatives available to Checkpoint and ultimately determined that the compelling and certain cash consideration and meaningful upside presented by the CVRs in this transaction provides superior risk-adjusted value relative to Checkpoint’s standalone prospects and other available alternatives. The Special Committee unanimously approved, and recommended that Checkpoint’s Board approve, the proposed transaction. After considering this recommendation, Checkpoint’s Board unanimously approved the proposed transaction. In arriving at its unanimous recommendation in favor of the transaction, the Special Committee considered several additional factors which will be outlined in public filings to be made by Checkpoint.

The transaction is expected to be completed in the second calendar quarter of 2025. The transaction is subject to customary closing conditions, including required regulatory approvals and approval by the holders of a majority of the voting power of outstanding shares of Checkpoint common stock, and by the holders of a majority of the shares of Checkpoint common stock that are not held by Fortress or by certain other affiliates of Checkpoint.

For the nine-month period ending September 2024, Checkpoint reported $0.04 million in revenue and a net loss of $27.3 million. The R&D expense for the nine-month period was $19.3 million. As of September 30, 2024, Checkpoint had a cash balance of $4.7 million, outstanding accounts payable and accrued expenses of $15.6 million, and outstanding accounts payable and accrued expenses – related party of $2.0 million.

In connection with the transaction, Fortress, which holds a majority of Checkpoint’s outstanding voting power, has agreed to vote in favor of the transaction.

Advisors

Barack Ferrazzano Kirschbaum & Nagelberg LLP and Allen Overy Shearman Sterling US LLP are serving as legal advisors to Sun Pharma.

Locust Walk is serving as the exclusive financial advisor to Checkpoint and lead financial advisor to Checkpoint on the transaction.

Cooley LLP and Morris, Nichols, Arsht & Tunnell LLP are serving as legal advisors to the Special Committee. Kroll, LLC is serving as financial advisor to the Special Committee.

Alston & Bird LLP is serving as legal advisor to Checkpoint.

About Cutaneous Squamous Cell Carcinoma

cSCC is the second-most common type of skin cancer in the United States, with an estimated annual incidence of approximately 1.8 million cases according to the Skin Cancer Foundation. Important risk factors for cSCC include chronic ultraviolet exposure and immunosuppressive conditions. While most cases are localized tumors amenable to curative resection, each year approximately 40,000 cases become advanced and an estimated 15,000 people in the United States die from this disease. In addition to being a life-threatening disease, cSCC causes significant functional morbidities and cosmetic deformities due to tumors that commonly arise in the head and neck region, and that invade blood vessels, nerves and vital organs, such as the eye or ear.