On February 28, 2024 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, reported fourth quarter and full year 2023 financial results and corporate updates (Press release, Iovance Biotherapeutics, FEB 28, 2024, View Source [SID1234640587]).

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Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "Throughout 2023, we executed toward our first approval and commercial launch while advancing our pipeline. We are seeing healthy demand and momentum for Amtagvi following the recent U.S. FDA approval in advanced melanoma. To expand the launch globally, we plan to submit regulatory dossiers in the European Union in the first half of 2024 and in Canada and the United Kingdom in the second half of 2024. We are also excited about our robust development pipeline across solid tumor cancers. As a fully integrated company, Iovance is well positioned to execute on our regulatory, pipeline, manufacturing, and commercial launch activities to advance our mission to remain the global leader in TIL therapy."

Recent and Fourth Quarter 2023 Highlights and Corporate Updates

Amtagvi (lifileucel):

U.S. Approval and Launch Highlights in Advanced Melanoma

The U.S. FDA approved Amtagvi on February 16, 2024, as the first treatment option for advanced melanoma after anti-PD-1 and targeted therapy. Amtagvi is also the first FDA-approved T cell therapy for a solid tumor indication.
Onboarding is complete at approximately 30 U.S. authorized treatment centers (ATCs) and approximately 50 ATCs are expected to be onboard by the end of May 2024.
The Iovance Cell Therapy Center (iCTC) began commercial manufacturing for Amtagvi patients within a week of approval. The iCTC, and a nearby FDA-approved contract manufacturer, are built today for capacity for several thousands of patients annually.
The U.S. launch of Amtagvi, and additional sales of Proleukin used with the treatment regimen, are expected to drive significant revenue for Iovance in 2024.
Since approval, there are at least 20 Amtagvi patients in process, which includes 10 patients already registered in IovanceCares with scheduled or pending manufacturing slots.
Launch Expansion into New Markets and Indications

Iovance’s global expansion strategy can more than double the total addressable patient population for Amtagvi in advanced melanoma. Anticipated regulatory submissions include the following:
A marketing authorization application (MAA) in the European Union (EU) in the first half of 2024.
An MAA in the U.K. and a new drug submission (NDS) in Canada in the second half of 2024.
Regulatory submissions in Australia and additional countries with significant populations of advanced melanoma patients in 2025.
The registrational Phase 3 TILVANCE-301 trial is underway to support accelerated and full approvals of Amtagvi in combination with pembrolizumab in frontline advanced melanoma.
Global site activation and patient enrollment continue with strong momentum in the U.S., Europe, Australia, Canada, and additional countries.
Following the U.S. FDA’s recent accelerated approval of Amtagvi in post-anti-PD-1 advanced melanoma, TILVANCE-301 is the confirmatory trial to support full approval in this initial indication.
An updated data cut for Cohort 1A of the IOV-COM-202 trial, in a presentation on the efficacy and safety of lifileucel and pembrolizumab in patients with immune checkpoint inhibitor-naive advanced melanoma, is planned for a medical meeting this year and is supportive of the rationale for TILVANCE-301.
Manufacturing Highlights

More than 700 patients have been treated with Iovance TIL therapy manufactured using proprietary Iovance processes as of December 31, 2023.
Capacity expansion is underway at iCTC to supply TIL cell therapies for more than 5,000 patients annually in the next few years.
Iovance TIL Therapy Clinical Pipeline Highlights

Enrollment in the registrational cohorts in the Phase 2 Trial IOV-LUN-202 in post-anti-PD-1 NSCLC is estimated to complete in 2025. Iovance is working collaboratively with the U.S. FDA to resume new patient enrollment in IOV-LUN-202 following the partial clinical hold for new patients on December 22, 2023.
A Phase 2 study in endometrial cancer in mismatch repair (MMR) deficient and MMR proficient patient populations is on track to commence in the first half of 2024.
The first in human IOV-GM1-201 trial is investigating PD-1 inactivated TIL therapy (IOV-4001) in previously treated advanced melanoma and NSCLC.
Corporate Updates

As of February 22, 2024, Iovance’s unaudited cash position is approximately $485.2 million, which includes net proceeds of approximately $197.1 million from a follow-on equity financing in February of 2024. The current cash position and anticipated revenue from Amtagvi and Proleukin are expected to be sufficient to fund current and planned operations well into the second half of 2025.
Iovance currently owns more than 60 granted or allowed U.S. and international patents for TIL compositions and methods of treatment and manufacturing in a broad range of cancers, with Gen 2 patent rights expected to provide exclusivity into 2038 and additional patent rights expected to provide exclusivity into 2042. More information on Iovance’s patent portfolio is available on the Intellectual Property page on www.iovance.com.
Fourth Quarter and Full Year 2023 Financial Results

Iovance had $346.3 million in cash, cash equivalents, investments and restricted cash at December 31, 2023, compared to $478.3 million at December 31, 2022. With the net proceeds of approximately $197.1 million raised in the February 2024 follow-on stock offering and anticipated revenue from Amtagvi and Proleukin, the cash position is expected to be sufficient to fund current and planned operations well into the second half of 2025.

Net loss for the fourth quarter ended December 31, 2023, was $116.4 million, or $0.45 per share, compared to a net loss of $105.3 million, or $0.64 per share, for the fourth quarter ended December 31, 2022. Net loss for the year ended December 31, 2023 was $444.0 million, or $1.89 per share, compared to a net loss of $395.9 million, or $2.49 per share, for the year ended December 31, 2022. The net loss for the year ended December 31, 2023 includes amortization of intangible assets acquired as part of the Proleukin transaction.

Revenue for the fourth quarter and year ended December 31, 2023, was $0.5 million and $1.2 million, respectively, and comprised of product sales following the Proleukin acquisition in May 2023. There was no revenue for the fourth quarter and year ended December 31, 2022. Cost of sales for the fourth quarter and year ended December 31, 2023, was $4.4 million and $10.8 million, respectively, and comprised of cost of inventory associated with sales of Proleukin as well as $3.9 million and $9.7 million, respectively, of non-cash amortization expenses of the acquired intangible asset for developed technology. There was no cost of revenues for the fourth quarter and year ended December 31, 2022.

Research and development expenses were $87.5 million for the fourth quarter ended December 31, 2023, an increase of $6.9 million compared to $80.6 million for the same period ended December 31, 2022. Research and development expenses were $344.1 million for the year ended December 31, 2023, an increase of $49.3 million compared to $294.8 million for the same period ended December 31, 2022.

The increases in research and development expenses in the fourth quarter and the year ended December 31, 2023, over the prior year periods were primarily attributable to increases in headcount and related costs to support internal manufacturing and clinical development activities, manufacturing costs to support increased production and commercial manufacturing readiness, clinical trial costs driven primarily by the initiation of our Phase 3 TILVANCE-301 clinical trial, and facility and related costs to expand manufacturing capacity.

Selling, general and administrative expenses were $29.9 million for the fourth quarter ended December 31, 2023, an increase of $3.4 million compared to $26.5 million for the same period ended December 31, 2022. Selling, general and administrative expenses were $106.9 million for the year ended December 31, 2023, an increase of $2.8 million compared to $104.1 million for the same period ended December 31, 2022.

The increase in selling, general and administrative expenses in the fourth quarter and the year ended December 31, 2023, compared to prior year periods was primarily attributable to increases in headcount and related costs to support the growth in the overall business and related corporate infrastructure, professional fees and travel costs, including costs associated with Proleukin integration. These increases were partially offset by a decrease in stock-based compensation expenses, legal and other costs. For additional information, please see the Company’s Selected Condensed Consolidated Balance Sheet and Statement of Operations below.

Webcast and Conference Call

To participate in the live conference call Q&A, please register at https://register.vevent.com/register/BI289df7d30f474a72a72e1c4f7a754c92. To listen to the live or archived audio webcast, please register at View Source The live and archived webcast can be accessed in the Investors section of the Company’s website, IR.Iovance.com. The archived webcast will be available for one year.

On February 28, 2024 Inhibrx, Inc. (Nasdaq: INBX) ("Inhibrx" or the "Company"), a biopharmaceutical company with three clinical programs in development and a strong emerging pipeline, reported financial results for the fourth quarter and fiscal year 2023 (Filing, 3 mnth, DEC 31, Inhibrx, 2024, FEB 28, 2024, View Source [SID1234640586]).

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Key Highlights
•Sale of INBRX-101 to Sanofi: In January 2024, the Company announced that it entered into a definitive agreement with Aventis, Inc. ("Aventis"), a subsidiary of Sanofi, whereby Sanofi will indirectly acquire, through Aventis, all of the assets and liabilities associated with INBRX-101 ("the Merger"). Immediately prior to the closing of the Merger, all non-101 assets and liabilities will be spun out into a new publicly traded company, Inhibrx Biosciences, Inc. ("New Inhibrx"). Sanofi will acquire all outstanding shares of the Company and, in turn, each shareholder will receive (i) $30.00 per share in cash, (ii) one contingent value right per share, representing the right to receive a contingent payment of $5.00 in cash upon the achievement of a regulatory milestone, and (iii) one SEC-registered, publicly-traded, share of New Inhibrx per every four shares of Inhibrx common stock held. In addition, in connection with the transaction, Sanofi will assume and retire the Company’s outstanding third-party debt and fund New Inhibrx with $200.0 million in cash. Sanofi will also retain an equity interest in New Inhibrx of 8%. The Company expects the transaction to close in the second quarter of 2024.
•INBRX-105: The Company decided to terminate its INBRX-105 program after evaluation of the totality of data from the expansion cohorts, in which it determined the initial signal was not sufficiently validated to support the continuation of the program. The Company is in the process of winding down the clinical trial and expects it to be completed within the first half of 2024.
•All Company Employees and Directors are Currently Subject to a Company-wide Blackout Restricting the Trading of Inhibrx Stock: The Company plans to file the proxy statement related to the sale of INBRX-101 in the next few days. Shortly after public filing of the Company’s proxy statement, and in accordance with the Company’s insider trading policy, the Company expects to lift the Company-wide blackout. As of today, employees and board members hold approximately 3.5 million vested and in-the-money options in Inhibrx common stock. The shares issuable upon exercise of these options are generally freely tradable. Any in-the-money options still outstanding at the Merger close will be converted into the Merger Consideration, as defined in the Merger Agreement, and will not convert into New Inhibrx shares.

Financial Results
•Cash and Cash Equivalents. As of December 31, 2023, Inhibrx had cash and cash equivalents of $277.9 million, compared to $337.3 million as of September 30, 2023.
•R&D Expense. Research and development expenses were $82.1 million during the fourth quarter of 2023 as compared to $30.5 million during the fourth quarter of 2022. Research

Exhibit 99.1
and development expenses were $191.6 million during the fiscal year 2023 as compared to $110.2 million during the fiscal year 2022. The increase in research and development expenses during both periods was primarily due to the following factors:
◦an increase in contract manufacturing expenses due to the nature of the development and manufacturing activities performed during the current period at our CDMO and CRO partners supporting our clinical and preclinical therapeutic candidates, primarily due to large scale drug substance manufacturing services, including the utilization of raw materials during the fourth quarter of 2023, performed by one of our CDMO partners for INBRX-101, in addition to other activities performed with our CDMO partners which reflect the stage-specific needs of each of our programs, including early and late stage drug substance clinical manufacturing, drug product manufacturing, and selected BLA-enabling activities;
◦an increase in clinical trial expenses, primarily related to costs incurred following the initiation of the registration-enabling Phase 2 trial for INBRX-101 for the treatment of emphysema due to AATD, which was initiated during the current year, as well as the progression of the Company’s INBRX-109 registration-enabling Phase 2 trial for the treatment of unresectable or metastatic conventional chondrosarcoma. The Company also incurred increased costs associated with the utilization of Keytruda used in combination with INBRX-105 in our Phase 1/2 clinical trial; and
◦an increase in personnel-related costs, primarily related to an increase in headcount as a result of a significant expansion of the Company’s clinical team, as well as the issuance of additional stock options and the expansion of the bonus eligibility pool during the current year.
•G&A Expense. General and administrative expenses were $7.8 million during the fourth quarter of 2023, compared to $5.3 million during the fourth quarter of 2022. General and administrative expenses were $29.4 million during the fiscal year 2023, compared to $21.1 million during the fiscal year 2022. This increase in general and administrative expenses during both periods was primarily due to the following factors:
◦an increase in personnel-related costs, primarily related to an increase in headcount as the Company continues to build its commercial strategy and medical affairs team, as well as increased expense related to additional stock option grants to employees and the expansion of the bonus eligibility pool in the current year;
◦an increase in pre-commercialization expenses, primarily related to increases in consulting services to support the Company’s commercial operations business intelligence strategies and market research expenses related to INBRX-101 and INBRX-109; and
◦an increase in professional service expenses related to accounting and legal services which support the Company in its general corporate and intellectual property matters, including services performed during the fourth quarter of 2023 as related to the Company’s proposed Merger.

Exhibit 99.1
•Net Loss. Net loss was $93.6 million during the fourth quarter of 2023, or $1.73 per share, compared to $40.9 million during the fourth quarter of 2022, or $0.95 per share. Net loss was $241.4 million during the fiscal year 2023, or $5.12 per share, compared to $145.2 million during the fiscal year 2022, or $3.62 per share.

On February 28, 2024 Inhibikase Therapeutics, Inc. (Nasdaq: IKT) ("Inhibikase" or "Company"), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Parkinson’s disease, Parkinson’s-related disorders and other diseases of the Abelson Tyrosine Kinases, reported preliminary outcomes of the Company’s discussion with the U.S. Food and Drug Administration (FDA) on the path to approval of IkT-001Pro in blood and stomach cancers, the Company’s prodrug of the anticancer agent imatinib mesylate (Press release, Inhibikase Therapeutics, FEB 28, 2024, View Source [SID1234640585]).

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"With the full meeting minutes from the FDA Review Team in hand, we are confident in our approach as we develop the NDA for IkT-001Pro," said Dr. Milton Werner, President and Chief Executive Officer of Inhibikase. "Of note, we may not need to complete a formal risk analysis of the possibility of medication errors following discussion of alternative dosage forms with the FDA, but we will have to provide justification for the selected dosage forms to be proposed in the NDA. Further, we are not restricted from use in children nor restricted to just blood cancer-related approved indications. This opens-up the opportunity to seek all 11 approved indications for IkT-001Pro that were previously approved for imatinib mesylate. We are pleased with the discussions we’ve had so far with the FDA and look forward to the work ahead of us needed for the NDA submission."

On February 12, 2024, the FDA Review Team from the Division of Hematologic Malignancies issued final meeting minutes from the pre-NDA video conference that took place January 19, 2024. In the minutes, the FDA noted that it may not be necessary to conduct a formal use-related risk analysis of medication errors due to the Company proposing dosage forms that would not overlap with those of imatinib mesylate. In the NDA package, the Company will have to justify why its alternate dosage forms could overcome the risk of dosing errors by physicians and patients. Following the discussion with the Review Team about possible differences in the way IkT-001Pro and imatinib mesylate are absorbed in the gut, the FDA agreed with the Company’s assertion that a pre-clinical analysis of gut absorption should be performed to determine whether a food effect clinical study is warranted, since the Company’s clinical measures were all performed using an FDA-approved meal prior to taking IkT-001Pro or imatinib mesylate. While the Company will have to supply data and/or rationale for use of IkT-001Pro in any indication for which imatinib mesylate is approved, use in children will need to be accompanied by a statutory planning document related to dose adjustments and use in children of different ages and/or weights. This planning document is required for any product in which use in children is proposed. All other elements of the pre-NDA meeting remain unchanged from those reported on February 7, 2024.

The Company continues to explore additional indications for which imatinib delivered by IkT-001Pro could be useful and has an upcoming meeting with the FDA to discuss cardiopulmonary applications of IkT-001Pro in April, 2024.

About IkT-001Pro
IkT-001Pro is a prodrug formulation of imatinib mesylate and has been developed to improve the safety of the first FDA-approved Abelson (Abl) kinase inhibitor, imatinib (marketed as Gleevec). Imatinib is commonly taken for hematological and gastrointestinal cancers that arise from Abl kinase mutations found in the bone marrow or for gastrointestinal cancers that arise from c-Kit and/or PDGFRa/b mutations in the stomach; c-Kit, PDGFRa/b and Abl are all members of the Abelson Tyrosine Kinase protein family. IkT-001Pro has the potential to be a safer alternative for patients and may improve the number of patients that reach and sustain major and/or complete cytogenetic responses in Stable-Phase Chronic Myelogenous Leukemia ("Stable-Phase CML") and/or reduce the relapse rate for these patients. In preclinical studies, IkT-001Pro was shown to be as much as 3.4 times safer than imatinib in primates, reducing burdensome gastrointestinal side effects that occur following oral administration. Imatinib delivered as IkT-001Pro was granted Orphan Drug Designation for Stable-Phase CML in September, 2018.

On February 28, 2024 Immunocore Holdings plc (Nasdaq: IMCR) ("Immunocore" or the "Company"), a commercial-stage biotechnology company pioneering and delivering transformative immunomodulating medicines to radically improve outcomes for patients with cancer, infectious diseases and autoimmune diseases, reported its financial results for the fourth quarter and year ended December 31, 2023 and provided a business update (Filing, 3 mnth, DEC 31, Immunocore, 2024, FEB 28, 2024, View Source [SID1234640584]).

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"In the last 5 years, Immunocore has transformed from a research organization to a revenue-generating, sustainable company," said Bahija Jallal, CEO of Immunocore. "We look forward to the next 5 years, when we maximize the potential of KIMMTRAK, expect to launch our PRAME ImmTAC therapy, and advance our clinical candidates across oncology, infectious diseases and autoimmune diseases."

"Throughout the last year, we expanded the reach of KIMMTRAK in metastatic uveal melanoma with additional approvals, launches, and sales growth across all territories," said Ralph Torbay, Head of Commercial. "We believe KIMMTRAK, the world’s first approved TCR therapy, could benefit thousands more patients and look forward to broadening indications with ongoing late-stage clinical trials in cutaneous and adjuvant uveal melanoma."

Full Year and Fourth Quarter 2023 Highlights (including post-period)

Financial Results

Total net product revenue (or "net sales) arising from the sales of KIMMTRAK (tebentafusp) was $67.6 million in the fourth quarter of 2023, of which $49.1 million was generated in the United States, $18.3 million in Europe and $0.2 million in international regions. For the year ended December 31, 2023, the Company generated net sales from the sale of KIMMTRAK in the amount of $238.7 million, of which $169.8 million was in the United States, $67.6 million in Europe and $1.3 million in international regions.

Research & development expenses for the year 2023 were $163.5 million, compared to $101.9 million for the year 2022. Selling, general and administrative (SG&A) expenses for the year 2023 were $144.5 million, compared to $123.1 million for the year 2022.

Net loss for the fourth quarter of 2023 was $19.7 million compared to a net loss of $30.0 million in the same period in 2022, and full year net loss for 2023 was $55.3 million compared to a full year net loss of $52.5 million in 2022.

The fourth quarter basic and diluted loss per share was $0.40, compared to $0.64 for the fourth quarter of 2022. Basic and diluted loss per share for 2023 was $1.13, compared to $1.15 for 2022.

Cash and cash equivalents at December 31, 2023 were $442.6 million. In February 2024, the Company raised net cash proceeds of $389.3 million from an offering of convertible notes with a six-year term and 2.50% interest rate (Convertible Notes).

KIMMTRAK expansion strategy

KIMMTRAK (tebentafusp-tebn) for metastatic uveal melanoma

Since the start of the 2024, KIMMTRAK has been launched in two additional countries (Australia and Canada), for a total of 12 launched and 38 approved countries. KIMMTRAK continues to be the standard of care for HLA-A02+ patients with metastatic uveal melanoma (mUM) in all of the countries in which it has been launched. In 2024, the Company plans to reach more patients in the United States, Europe and globally, as it continues to drive global launches and focuses on supporting early patient identification and treatment.

KIMMTRAK Phase 2/3 clinical trial in 2L+ advanced cutaneous melanoma

The Company also continues to enroll patients into a Phase 2/3 clinical trial (TEBE-AM) to investigate the potential of KIMMTRAK in 2L+ advanced cutaneous melanoma. Topline data from the Phase 2 portion of the trial is expected to be available by the fourth quarter of 2024.

KIMMTRAK Phase 3 clinical trial in adjuvant uveal (or ocular) melanoma

In 2023, the Company signed an agreement for a European Organisation for Research and Treatment of Cancer (EORTC)-sponsored trial to study KIMMTRAK as adjuvant therapy for uveal (or ocular) melanoma (ATOM). The Company anticipates that the EORTC will randomize the first patient in the second half of 2024.

PRAME franchise

PRISM-MEL301 – First PRAME Phase 3 clinical trial with IMC-F106C in first-line advanced cutaneous melanoma

In February 2024, the Company entered into a clinical trial collaboration and supply agreement with Bristol Myers Squibb (NYSE:BMY) to investigate Immunocore’s ImmTAC bispecific TCR candidate targeting PRAME HLA-A02, IMC-F106C, in combination with Bristol Myers Squibb’s nivolumab, in first-line advanced cutaneous melanoma. Under the terms of the collaboration, Immunocore will sponsor and fund the registrational Phase 3 clinical trial of IMC-F106C in combination with nivolumab in first-line advanced cutaneous melanoma (PRISM-MEL-301), and Bristol Myers Squibb will provide nivolumab.

In August 2023, the Company announced plans to start a registrational Phase 3 clinical trial with IMC-F106C in first-line advanced cutaneous melanoma (CM). The Company decided to advance IMC-F106C into a Phase 3 first-line CM clinical trial in combination with nivolumab with a primary endpoint of progression-free survival (PFS), based on the Company’s analysis of the ongoing Phase 1 data in previously treated CM which demonstrated monotherapy clinical activity including partial responses (PR), durable tumor reduction, disease control (PR and SD), PFS and circulating tumor DNA (ctDNA) reduction (consistent with prior reported data for IMC-F106C and tebentafusp). Additional rationale includes safety in combination with checkpoints (from the ongoing Phase 1 data and prior experience with tebentafusp) and evidence from across the platform for increased clinical activity in earlier line patients compared to later line. As such, PRISM-MEL-301, the first PRAME Phase 3 clinical trial with IMC-F106C, will randomize patients with HLA-A*02:01-positive, first-line advanced CM to IMC-F106C + nivolumab versus a control arm of either nivolumab or nivolumab + relatlimab, depending on the country where the patient is enrolled. The Company plans to randomize the first patient in this trial in the first quarter of 2024.

Phase 1/2 clinical trial of IMC-F106C targeting PRAME-A02 in multiple solid tumors

In addition to progressing IMC-F106C into a registrational trial in cutaneous melanoma, the Company is continuing to enroll patients in the monotherapy and combination arms of the Phase 1/2 clinical trial across multiple tumor types, including expansion arms for patients with advanced ovarian, non-small cell lung, and endometrial carcinoma. The initial data from the Phase 1 clinical trial of IMC-F106C, the first PRAME x CD3 ImmTAC bispecific protein, was presented at the 2022 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in September 2022. Durable Response Evaluation Criteria in Solid Tumors (RECIST) responses and reduction in ctDNA were observed across multiple solid tumors. In August 2023, the Company provided an updated analysis of the original 18 melanoma patients (initially presented at ESMO (Free ESMO Whitepaper) in September 2022), which continued to show promising durability of the clinical activity (range of duration of partial response from 6 months to 17 months). The Company expects to report clinical data from the ongoing monotherapy and combination cohorts throughout 2024 including cutaneous melanoma (expected in Q2 2024), ovarian (expected by Q3 2024), and non-small cell lung carcinoma (expected by Q4 2024).

IMC-P115C (PRAME HLA-A02 Half-Life Extended) & IMC-T119C (PRAME HLA-A24)

The Company is expanding the PRAME franchise with two new PRAME ImmTAC candidates, IMC-P115C (PRAME HLA-A02 HLE) and IMC-T119C (PRAME HLA-A24) for solid tumors, which are both on track for investigational new drug (IND) or clinical trial application (CTA) submissions for IMC-P115C in the middle of 2024 and the fourth quarter of 2024 for IMC-T119C.

IMC-R117C (PIWIL1) for colorectal and other gastrointestinal cancers

The Company has leveraged its proprietary peptide (ImmSPECT) database to validate a novel target, PIWIL1. PIWIL1 is believed to play a role in tumor progression and is expressed across a range of tumors, including colorectal which is historically insensitive to immune checkpoints, as well as other gastrointestinal cancers. PIWIL1 is also reported to be a negative prognostic marker and the Company believes IMC-R117C is the first PIWIL1-targeted immunotherapy. The Company submitted a CTA to regulatory authorities in December 2023, and expects the trial to start in the second half of 2024.

Enrolling ImmTAV candidates for a functional cure in infectious diseases

The Company continues to enroll people living with HIV in the multiple ascending dose (MAD) part of a Phase 1 clinical trial with IMC-M113V, to identify a safe and tolerable dosing schedule. This trial will also test whether IMC-M113V could lead to reduction in the viral reservoir and, after stopping all therapies (antiretroviral therapies and IMC-M113V), delay or prevent HIV rebound (known as functional cure). The MAD part of the trial will enroll up to 28 participants. The Company expects to present a data update from the Phase 1 clinical trial in the second half of 2024.

In 2023, the Company amended the design of the ongoing Phase 1 clinical trial with IMC-I109V for people living with HBV to include HBV-positive hepatocellular carcinoma. The Company continues to enroll patients into the single ascending dose portion of the trial in 2024.

Tissue-specific down modulation of the immune system for autoimmune diseases

The Company is expanding its platform into autoimmune diseases with two first-in-class new bispecific candidates entering the Company’s pipeline. The key differentiator of the ImmTAAI platform is tissue-specific down modulation of the immune system. When tethered to the tissue of interest, the new candidates supress pathogenic T cells via PD1 receptor agonism.

The first candidate, IMC-S118AI (PPIxPD1), is targeted specifically to pancreatic beta cells and is intended for disease-modifying treatment in type 1 diabetes. IMC-S118AI recognizes a peptide from pre-proinsulin presented by HLA-A02 on beta cells coupled with a PD1 agonist effector arm. IMC-S118AI is advancing towards GMP manufacturing in 2024.

The second target is present in the skin and intended to treat inflammatory dermatological diseases. The candidate is an antigen presenting cell (APC) tethered ImmTAAI and is not HLA restricted (e.g. universal for all populations).

Financial Results

Basic and diluted loss per share was $0.40 and $1.13 for the quarter and year ended December 31, 2023, respectively, as compared to a basic and diluted loss per share of $0.64 and $1.15, respectively, for the same periods in 2022. Net loss for the quarter and year ended December 31, 2023, was $19.7 million and $55.3 million, respectively, as compared to $30.0 million and $52.5 million, respectively, for the same periods in 2022.

For the fourth quarter and year ended December 31, 2023, we generated net sales of $67.6 million and $238.7 million, respectively, due to the sale of KIMMTRAK and tebentafusp, of which $49.1 million and $169.8 million, respectively was in the United States, $18.3 million and $67.6 million, respectively, was in Europe, and $0.2 million and $1.3 million, respectively, was in the international regions. The increase in net sales was due primarily to increased volume in the United States and global country expansion, as we continued our commercialization efforts.

For the fourth quarter and year ended December 31, 2023, our research and development (R&D) expenses were $45.6 million and $163.5 million, respectively as compared to $31.1 million and $101.9 million for the quarter and year ended December 31, 2022. These increases were driven by expenses incurred for our PRAME programs, increases in headcount-related expenses as a result of higher number of employees and associated staff costs, increases related to consumables and facilities costs, and decreased R&D tax credits due to us no longer qualifying as a ‘small and medium enterprise’ (SME) under the UK R&D tax regulations. The Company expects R&D expenses to increase in future periods as the Company advances its trials and further develops clinical and preclinical pipeline candidates.

For the quarter and year ended December 31, 2023, our SG&A expenses were $41.4 million and $144.5 million, respectively, compared to $35.4 million and $123.1 million for the quarter and year ended December 31, 2022. These increases were related to an increase in headcount costs, higher selling and distribution costs following regulatory approval of KIMMTRAK, and costs associated with expansion as a growing publicly listed and commercial company.

Cash and cash equivalents were $442.6 million as of December 31, 2023, as compared to $402.5 million as of December 31, 2022. In February 2024, the Company raised net cash proceeds of $389.3 million from a Convertible Notes offering with a six-year term and 2.50% interest rate. The Company plans to use $50 million from the net proceeds to repay its existing Pharmakon loan by the end of 2024. The Company estimates Pro Forma for the net cash proceeds from the Convertible Notes offering in February 2024, and the Pharmakon loan repayment, its cash and cash equivalents at year-end 2023, were approximately $782 million.

As of December 31, 2023, the Company no longer qualified as a foreign private issuer for U.S. publicly company reporting purposes. Effective January 1, 2024, it now files periodic reports on U.S. domestic filer forms with the Securities and Exchange Commission (SEC) and comply with other rules as required, including but not limited to presenting its financial results in press releases and Annual Report on Form 10-K in accordance with U.S. GAAP, with such change being applied retrospectively. See the Company’s Annual Report on Form 10-K filed today with the SEC for more information.

On February 28, 2024 HUTCHMED (China) Limited, the innovative, commercial-stage biopharmaceutical company, reported its financial results for the year ended December 31, 2023 and provides updates on key clinical and commercial developments (Filing, 3 mnth, DEC 31, Hutchison China MediTech, 2024, FEB 28, 2024, View Source [SID1234640583]). HUTCHMED to host results call and webcasts today at 7:30 a.m. EST / 12:30 p.m. GMT / 8:30 p.m. HKT in English, and at 8:30 a.m. HKT in Chinese (Putonghua) on Thursday, February 29, 2024.

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All amounts are expressed in U.S. dollars unless otherwise stated.

Strategic: global vision, commitment to patients and path to self-sustainability

· Executed our global vision of bringing our innovative medicines worldwide, as demonstrated through the Takeda1 partnership which brought $435 million in upfront and milestone payments plus manufacturing income and royalties on net sales, setting a strategic example for the rest of our pipeline.

· On track to be self-sustaining with a disciplined approach to leveraging our R&D2 expertise and creating value through licensing and commercialization.

Pipeline: fruquintinib global and China expansion, sovleplenib China NDA3 review, savolitinib NSCLC4 enrolled

· Fruquintinib U.S. FDA5 approval three weeks ahead of PDUFA6 date for third-line CRC7, leading to a swift launch by Takeda, inclusion in NCCN8 guidelines and U.S. in-market sales9 of $15.1 million. Global regulatory progress with MAA10 filing to the EMA11 validated in June 2023 and NDA submitted to PMDA12 in September 2023.

· Fruquintinib NDA for second-line gastric cancer accepted for review in China. Registrations studies in China for 2L EMC13 and 2L RCC14 completed enrollment during 2023 for fruquintinib in combination with sintilimab, expecting NDA filing to the NMPA15 for EMC in early 2024 and topline results for RCC by end of 2024.

· NDA for sovleplenib, a novel Syk16 inhibitor, for primary ITP17 accepted and granted priority review in China, supported by data from Phase III trial (ESLIM-01), meeting all endpoints.

· SAVANNAH, the pivotal global Phase II trial for savolitinib in NSCLC, completed enrollment, to be followed by potential NDA filing to the U.S. FDA by AstraZeneca18 around the end of 2024.

Outlook and financial: expecting strong product revenue growth and reduced expenses; substantial cash

· Total revenue up 97% (102% at CER19) to $838.0 million for 2023, with Oncology/Immunology consolidated revenue up 223% (228% at CER) to $528.6 million at high end of guidance, including recognition of $280 million of the upfront payment from Takeda. Net income attributable to HUTCHMED of $100.8 million.

· 2024 Oncology/Immunology consolidated revenue guidance of $300 million to $400 million, driven by 30% to 50% growth target in marketed product sales and royalties.

· R&D expenses focused in line with strategy targeting key projects.

· Strengthened cash balance, with $886.3 million at year end (2022: $631.0m), ensures HUTCHMED is well placed to deliver on its objective of becoming a self-sustaining business.

2023 FULL YEAR RESULTS & BUSINESS UPDATES

Mr Simon To, Executive Chairman of HUTCHMED, said, "We have made significant progress throughout 2023. We executed against our commitment to bring our innovative medicines to patients worldwide with the U.S. FDA approval of FRUZAQLA in November 2023, while remaining dedicated to becoming a self-sustaining business. The Takeda partnership, which is one of the biggest small-molecule overseas licensing deals in the history of China biotech, strengthened our cash position by $435 million. Takeda delivered a successful U.S. launch within 48 hours of approval, and has subsequently seen strong early patient uptake."

"We will continue to deliver on our strategy in 2024. We will stay focused on our target of becoming sustainable through our balanced strategy of growing sales of our novel medicines in China, and advancing our medicines overseas with our partners. This, when combined with our other goals on pipeline progression and further business development, means that while the global macroeconomic environment remains uncertain. HUTCHMED is positioned to thrive and continue to deliver innovative medicines to ever more patients around the world."

Dr Weiguo Su, Chief Executive Officer and Chief Scientific Officer of HUTCHMED, said, "HUTCHMED delivered impressive financial results in 2023, with revenue up 97% to $838 million. This, alongside our significantly strengthened cash balance of $886 million, will enable us to continue advancing our pipeline and successfully executing our strategy."

"2023 was an important year for HUTCHMED, particularly for fruquintinib, for which we filed market authorization applications in the U.S., EU and Japan, based on the successful FRESCO-2 study. Following the U.S. FDA approval for third-line patients with advanced CRC, we continue to work together with Takeda continue to pursue additional launches in new markets worldwide. In China, we also filed an NDA for second-line gastric cancer based on the FRUTIGA study."

"Another milestone was the successful ESLIM-01 registration study in China in ITP patients for sovleplenib, our first potential novel medicine in immunological diseases. The NDA was accepted and granted priority review by the NMPA in January 2024. There are over 250,000 new and existing adult ITP patients in China.20 The treatment options are limited to steroids and TPO/TPO-RAs21, representing an unmet medical need that sovleplenib could help address, with its new mechanism of action and favorable safety profile. Syk inhibition has the potential to target other major diseases such as rheumatoid arthritis. We are also planning to initiate clinical development of sovleplenib outside China in 2024."

"For savolitinib, we completed the confirmatory trial in NSCLC patients with MET22 exon 14 skipping alterations. An NDA submission is expected in the first quarter of 2024, with potential to expand the label indication to include first-line patients in China. Outside China, we will continue our work with AstraZeneca on the pivotal global savolitinib lung cancer trial SAVANNAH, which, subject to favorable data, can support a filing to the U.S. FDA for approval. This study completed enrollment with a potential NDA submission towards the end of 2024 in EGFR23 mutant NSCLC patients who progressed on TAGRISSO treatment, which received U.S. FDA Fast Track designation in January 2023. We believe the convenient dosing, targeted efficacy and safety profile of savolitinib as an oral medicine in combination with TAGRISSO, the leading oral third-generation EGFR TKI24, should position it well in a competitive market and address the unmet needs of MET+ NSCLC patients."

"Our China commercialization efforts progressed well, as we successfully renewed NRDL25 coverage for both fruquintinib and surufatinib without further price reduction. Their in-market sales saw strong growth in 2023. Over the next two years, we plan to continue growth in China through expanded indications and the launch of new products together with revenue from FRUZAQLA overseas commercialization."