Immunocore Reports 2022 Financial Results and Provides Business Update

On March 1, 2023 Immunocore Holdings plc (Nasdaq: IMCR), a commercial-stage biotechnology company pioneering the development of a novel class of T cell receptor (TCR) bispecific immunotherapies designed to treat a broad range of diseases, including cancer, infectious diseases and autoimmune conditions, reported its financial results for the fourth quarter and year ended December 31, 2022, and provided a business update (Press release, Immunocore, MAR 1, 2023, View Source [SID1234627978]).

"We are proud that KIMMTRAK, the world’s first commercialized TCR therapy, has been approved in over 30 countries for patients with metastatic uveal melanoma. In 2023, we will make it available to even more patients with launches planned in additional countries and through enrollment of cutaneous melanoma patients into our registrational tebentafusp trial," said Bahija Jallal, Chief Executive Officer of Immunocore. "We are also focused on progressing our PRAME clinical programs with ongoing expansion arms in multiple tumor types. Our platform continues to deliver first-in-class oncology programs, like PIWIL1, and several infectious disease candidates."

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Full Year and Fourth Quarter 2022 Highlights (including post-period)

Financial Results

Total net product and net pre-product revenue (or "net sales") arising from the sales of KIMMTRAK (tebentafusp) were £42.3 million (or $51.1 million) in the fourth quarter of 2022, of which £32.1 million (or $38.8 million) was in the United States, £10.1 million (or $12.2 million) in Europe and £0.1 million (or $0.1 million) in international regions. For the year ended December 31, 2022, the Company generated total revenue from the sale of KIMMTRAK and tebentafusp in the amount of £116.8 million (or $141.1 million), of which £80.4 million (or $97.2 million) was in the United States, £35.5 million (or $42.9 million) in Europe and £0.9 million (or $1.1 million) in international regions.

R&D expenses for the year 2022 were £89.2 million (or $107.9 million), compared to £73.2 million for the year 2021. Selling and Administrative expenses for the year 2022 were £93.7 million (or $113.2 million), compared to £88.4 million for the year 2021.

Net loss for the fourth quarter of 2022 was £25.2 million (or $30.4 million) compared to a net loss of £39.4 million in the same period in 2021, and full year net loss for 2022 was £41.24 million (or $49.8 million) compared to a full year net loss of £131.5 million in 2021.

The fourth quarter basic and diluted loss per share for was $0.63, compared to $1.21 on December 31, 2021. Basic and diluted loss per share for the year ended December 31, 2022 was $1.09, compared to $4.19 for 2021.

Cash and cash equivalents for the year ended December 31, 2022 were £332.5 million (or $401.6 million). The Company’s current cash position and projected KIMMTRAK revenue provides a projected cash runway into 2026.

KIMMTRAK (tebentafusp-tebn) for metastatic uveal melanoma

During the fourth quarter of 2022, the Company continued to add new accounts prescribing KIMMTRAK in the United States, Germany, and France. As of December 31, 2022 there were 240 new accounts prescribing KIMMTRAK in the United States, which brings the capture rate of these accounts, according to the Company’s internal estimates, to 50% of potentially eligible patients. There were 100 new accounts prescribing KIMMTRAK in Germany and France, which brings the capture rate to approximately 80% of the eligible patient population. In the United States, Germany, and France the commercial team is focused on treating patients closer to home. In the three countries where KIMMTRAK is commercially available the real-world mean duration of treatment for KIMMTRAK continues increasing to the 9 months observed in the Phase 2 and 3 clinical trials.

During the fourth quarter of 2022, the Company received numerous awards and positive recognitions for KIMMTRAK’s clinical benefit. In November, the Company was awarded the SCRIP award in the UK in the ‘Best New Drug’ category, and in December, the prestigious Prix Galien France 2022 award in the ‘Medicine in Innovative Therapeutics’ category, for KIMMTRAK. KIMMTRAK’s clinical benefit to patients was recognized with Germany’s G-BA (Gemeinsamer Bundesausschuss) granting the therapy a considerable added benefit rating, as well as in France, where the HAS transparency committee granted an ASMR III and a SMR rating of important. These recommendations build upon the positive recommendations by American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and National Comprehensive Cancer Network (NCCN) in the second quarter of 2022. KIMMTRAK was approved by the U.S. Food and Drug Administration; the European Commission; and health authorities in the United Kingdom, Australia and Canada in the first half of 2022.

The Company is undergoing reimbursement discussions in a number of countries, including Germany and France where the Company is currently receiving revenues. The Company expects reimbursement decisions in one additional major European country by mid-2023 and up to five smaller countries by end of 2023.

In November 2022, the Company and Medison Pharma Ltd. ("Medison") amended and restated their exclusive distribution agreement for KIMMTRAK originally entered into in September 2021. Medison is the exclusive distribution partner for KIMMTRAK in Canada, Australia, New Zealand, Israel, Central and Eastern Europe, and following this amendment South and Central America, and the Caribbean.

Tebentafusp Phase 2/3 trial in advanced melanoma

The Company is screening patients in its Phase 2/3 clinical trial of tebentafusp in patients with previously treated advanced melanoma. The trial will enroll patients with advanced melanoma, excluding uveal melanoma, who have progressed on an anti-PD1, received prior ipilimumab and, if applicable, received a tyrosine kinase inhibitor (TKI). Patients will be randomized to one of three arms including tebentafusp, as monotherapy or in combination with an anti-PD1, and a control arm. The Phase 2 portion of the trial will include 40 patients per arm and has a dual primary endpoint of overall survival (OS) and circulating tumor DNA (ctDNA) reduction.

IMC-F106C targeting PRAME-A02 in multiple solid tumors

The Company’s planned global expansion of the clinical trial footprint for PRAME-A02 studies is underway, with additional patients now being recruited into the Phase 1/2 monotherapy and combination expansion arms in order to characterize the breadth of clinical activity across multiple tumor types. Initial Phase 1 data with IMC-F106C targeting PRAME (PRAME-A02) was presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2022, and the Company initiated four expansion arms—cutaneous melanoma, ovarian, non-small cell lung cancer (NSCLC), and endometrial cancers. The combinations with standards-of-care (checkpoint inhibitors, chemotherapy, and tebentafusp) will position the Company to explore IMC-F106C in earlier lines of treatment. The Company expects to report data from the monotherapy and combination arms by the first half of 2024.

Expansion of PRAME franchise: IMC-T119C (PRAME-A24) & IMC-P115C (PRAME-A02 HLE)

In January 2023, the Company revealed the addition of two new PRAME ImmTAC candidates for solid tumors to the pipeline. Building on enthusiasm for IMC-F106C targeting PRAME HLA-A02, the Company has expanded its franchise targeting PRAME.

IMC-F106C is an ImmTAC targeting PRAME for patients with HLA-A02, which is expressed in approximately 40% of Western populations (United States, Canada, EU). In order to expand the potential of TCR therapy targeting PRAME, the Company is developing IMC-T119C, a first-in-class ImmTAC product candidate targeting a PRAME peptide presented by HLA-A24. HLA-24 is an HLA-type that is estimated to be present in 60% of people in Japan and 15-20% in Western populations.

In addition, the Company is developing IMC-P115C, a half-life extended (HLE) ImmTAC product candidate targeting PRAME-A02, with the aim of improving patient convenience. IMC-P115C targets the same PRAME-A02 peptide and uses the same CD3 end and TCR specificity as IMC-F106C.

The Company plans to submit investigational new drug applications (INDs) for these two ImmTAC candidates in 2024.

First-in-class ImmTAC candidate – IMC-R117C (PIWIL1)

In January 2023, the Company announced an ImmTAC targeting a novel protein for colorectal and other gastrointestinal cancers. The Company has leveraged its proprietary peptidomic 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 gastro-esophageal, and pancreatic cancer. PIWIL1 is also reported to be a negative prognostic marker. The Company believes IMC-R117C is the first PIWIL1 targeted immunotherapy and plans to submit an IND in the fourth quarter of 2023.

IMC-C103C targeting MAGE-A4

In December 2022, the Company presented Phase 1 data for IMC-C103C in a poster presentation at the ESMO (Free ESMO Whitepaper) Immuno-Oncology 2022 Congress.

In February 2023, Genentech accepted Immunocore’s proposal to cease co-funding the development of MAGE-A4 HLA-A02 targeted programs, except for Immunocore’s equal share of the wind-down costs of the IMC-C103C Phase 1 clinical trial. The clinical trial with IMC-C103C is nearing completion and Immunocore does not plan to enroll additional patients. Immunocore is eligible to receive development and commercial milestone payments plus royalties from Genentech on any sales of MAGE-A4 HLA-A02 targeted products arising under the Genentech agreement.

ImmTAV clinical programs

In February 2023, the Company presented initial safety and pharmacodynamic activity data with IMC-C113V, the first soluble TCR therapy for people living with Human Immunodeficiency Virus (HIV), at the Conference on Retroviruses and Opportunistic Infections (CROI) 2023. IMC-M113V is an immunotherapeutic approach designed to specifically eliminate CD4+ cells that are persistently infected with HIV (‘reservoirs’). All doses (1.6 mcg, 5 mcg, and 15 mcg) of IMC-M113V were well tolerated and not associated with cytokine release syndrome or neurotoxicity of any grade. There were no serious adverse events, nor significant changes in hematology or chemistry. Plasma viral load remained suppressed throughout dosing and follow-up. In addition, transient, dose-dependent increases in serum IL6 occurred 8-24 hours post-infusion. Five out of the ten participants who received the 15-mcg dose showed a >4-fold rise in IL6, which had been prespecified as indicative of pharmacodynamic activity based on prior experience from clinical trials with KIMMTRAK.

The Company has started enrolling people living with HIV in the multiple ascending dose (MAD) part of the trial, to identify a safe and tolerable dosing schedule that could lead to reduction in the viral reservoir and control of HIV after stopping antiretroviral therapies (ART), or functional cure. The MAD trial will enroll up to 28 participants.

In the second quarter of 2022, the Company presented data from the first three patients in the first-in-human clinical trial of IMC-I109V for chronic hepatitis B at the EASL International Liver Congress. In this first cohort, the three patients received a single dose of 0.8 mcg, based on the minimum anticipated biological effect level (MABEL). The dose in this initial cohort was well tolerated and was not associated with adverse events and resulted in a transient, small decrease in serum HBsAg with concomitant minor increase in alanine transaminase (ALT). The Company is enrolling patients in the single ascending dose portion of the trial.

Corporate and financial updates

In December 2022, the Company entered into an agreement with Gadeta B.V., to develop the first gamma delta (γδ) TCR ImmTAC for solid tumors, including colorectal cancer. Immunocore will collaborate with Gadeta on ‘201 γδ-TCR target discovery and has an option to develop ImmTAC therapies derived from the ‘201 TCR as part of the research collaboration.

Financial Results

Basic and diluted loss per share was £0.54 (or $0.63) and £0.90 (or $1.09) for the quarter and year ended December 31, 2022, respectively, as compared to a basic and diluted loss per share of £0.90 and £3.10, respectively, for the same periods in 2021. Total operating loss for the quarter and year ended December 31, 2022, was £22.3 million (or $27.0 million) and £39.6 million (or $47.8 million), respectively, as compared to £37.8 million and £135.2 million respectively for the same periods in 2021.

For the fourth quarter and year ended December 31, 2022, we generated total revenue from the sale of therapies of £42.3 million ($51.1 million) and £116.8 million ($141.1 million), respectively, due to the sale of KIMMTRAK and tebentafusp, of which £32.1 million ($38.8 million) and £80.4 million ($97.2 million), respectively was in the United States, £10.1 million ($12.2 million) and £35.5 million ($42.9 million), respectively, was in Europe, and £0.1 million ($0.1 million) and £0.9 million ($1.1 million), respectively, was in the rest of the world. We received marketing approval for KIMMTRAK in the United States, Europe and other territories in the year ended December 31, 2022, and did not have marketing approval for, and thus no product revenue from, KIMMTRAK in the year ended December 31, 2021.

For the fourth quarter and year ended December 31, 2022, our research and development ("R&D") expenses were £27.1 ($32.8 million) and £89.2 million (or $107.9 million), respectively as compared to £20.1 million and £73.2 million for the quarter and year ended December 31, 2021. These increases were due to increased expenses in connection with our IMC-F106C program and increased headcount and laboratory costs. 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, 2022, our selling and administrative expenses were £43.1 million ($52.1 million) and £93.7 million (or $113.2 million), respectively, compared to £24.4 million and £88.4 million for the quarter and year ended December 31, 2021. These increases were related to higher selling and distribution costs following regulatory approval of KIMMTRAK and an increase in headcount costs (excluding share-based payment charges, which reduced in 2022). Our administrative expenses also increased in the fourth quarter of 2022 due to approximately $15 million of non-cash foreign exchange losses, primarily on U.S. dollar balances.

Cash and cash equivalents were £332.5 million (or $401.6 million) as of December 31, 2022, as compared to £237.9 million (or $321.1 million) as of December 31, 2021. We expect that our existing cash, along with anticipated revenue from KIMMTRAK, will be sufficient to fund our planned operating expenses, financial commitments and other cash requirements into 2026.

We maintain our books and records in pounds sterling. For the convenience of the reader, we have translated pound sterling amounts as of and for the period ended December 31, 2022 into U.S. dollars at a rate of £1.00 to $1.2077.

Audio Webcast

Immunocore will host a conference call today, March 1, 2023 at 8:00 A.M. ET/ 1:00 PM GMT, to discuss the fourth quarter and full year 2022 financial results and provide a business update. The call will also be available via webcast by visiting the Events & Presentations section on Immunocore’s website. A replay of this webcast will be available for 30 days.

Conference Call Details:
U.S. (toll-free): 877-405-1239
International (toll): +1 201-389-0851

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About KIMMTRAK
KIMMTRAK is a novel bispecific protein comprised of a soluble T cell receptor fused to an anti-CD3 immune-effector function. KIMMTRAK specifically targets gp100, a lineage antigen expressed in melanocytes and melanoma. This is the first molecule developed using Immunocore’s ImmTAC technology platform designed to redirect and activate T cells to recognize and kill tumor cells. KIMMTRAK has been approved for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma in the United States, European Union, Canada, Australia, and the United Kingdom.

About Phase 3 IMCgp100-202 Trial
IMCgp100-202 (NCT03070392) is a randomized pivotal trial that evaluated overall survival (OS) of KIMMTRAK compared to investigator’s choice (either pembrolizumab, ipilimumab, or dacarbazine) in HLA-A*02:01-positive adult patients with previously untreated mUM. KIMMTRAK demonstrated an unprecedented OS benefit with a Hazard Ratio (HR) in the intent-to-treat population favoring KIMMTRAK, HR=0.51 (95% CI: 0.37, 0.71); p< 0.0001, over investigator’s choice (82% pembrolizumab; 13% ipilimumab; 6% dacarbazine).

IMPORTANT SAFETY INFORMATION

Cytokine Release Syndrome (CRS), which may be serious or life-threatening, occurred in patients receiving KIMMTRAK. Monitor for at least 16 hours following first three infusions and then as clinically indicated. Manifestations of CRS may include fever, hypotension, hypoxia, chills, nausea, vomiting, rash, elevated transaminases, fatigue, and headache. CRS occurred in 89% of patients who received KIMMTRAK with 0.8% being grade 3 or 4. Ensure immediate access to medications and resuscitative equipment to manage CRS. Ensure patients are euvolemic prior to initiating the infusions. Closely monitor patients for signs or symptoms of CRS following infusions of KIMMTRAK. Monitor fluid status, vital signs, and oxygenation level and provide appropriate therapy. Withhold or discontinue KIMMTRAK depending on persistence and severity of CRS.

Skin Reactions

Skin reactions, including rash, pruritus, and cutaneous edema occurred in 91% of patients treated with KIMMTRAK. Monitor patients for skin reactions. If skin reactions occur, treat with antihistamine and topical or systemic steroids based on persistence and severity of symptoms. Withhold or permanently discontinue KIMMTRAK depending on the severity of skin reactions.

Elevated Liver Enzymes

Elevations in liver enzymes occurred in 65% of patients treated with KIMMTRAK. Monitor alanine aminotransferase (ALT), aspartate aminotransferase (AST), and total blood bilirubin prior to the start of and during treatment with KIMMTRAK. Withhold KIMMTRAK according to severity.

Embryo-Fetal Toxicity
KIMMTRAK may cause fetal harm. Advise pregnant patients of potential risk to the fetus and patients of reproductive potential to use effective contraception during treatment with KIMMTRAK and 1 week after the last dose.

The most common adverse reactions (≥30%) in patients who received KIMMTRAK were cytokine release syndrome, rash, pyrexia, pruritus, fatigue, nausea, chills, abdominal pain, edema, hypotension, dry skin, headache, and vomiting. The most common (≥50%) laboratory abnormalities were decreased lymphocyte count, increased creatinine, increased glucose, increased AST, increased ALT, decreased hemoglobin, and decreased phosphate.

For more information, please see full Summary of Product Characteristics (SmPC) or full U.S. Prescribing Information (including BOXED WARNING for CRS).

About KIMMTRAKConnect
Immunocore is committed to helping patients who need KIMMTRAK obtain access via our KIMMTRAKConnect program. The program provides services with dedicated nurse case managers who provide personalized support, including educational resources, financial assistance, and site of care coordination. To learn more, visit KIMMTRAKConnect.com or call 844-775-2273.

Elevation Oncology to Participate at Upcoming Investor Conferences

On March 1, 2023 Elevation Oncology, Inc. (Nasdaq: ELEV), an innovative oncology company focused on the discovery and development of selective cancer therapies to treat patients across a range of solid tumors with significant unmet medical needs, reported that management will participate in the following investor conferences in March (Press release, Elevation Oncology, MAR 1, 2023, View Source;utm_medium=rss&utm_campaign=elevation-oncology-to-participate-at-upcoming-investor-conferences [SID1234627977]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Cowen 43rd Annual Health Care Conference
Date: Tuesday, March 7
Panel Title: GI/GU Oncology
Panel Time: 12:50 p.m. ET

Oppenheimer 33rd Annual Healthcare Conference
Date: Wednesday, March 15
Presentation Time: 8:00 a.m. ET

A live webcast and replay of the event will be available on the Events page of the Company’s Investor Relations website at View Source

Dynavax to Present at the Cowen 43rd Annual Health Care Conference

On March 1, 2023 Dynavax Technologies Corporation (Nasdaq: DVAX), a commercial-stage biopharmaceutical company developing and commercializing innovative vaccines, reported that Kelly MacDonald, Chief Financial Officer, will participate in a fireside chat at the Cowen 43rd Annual Health Care Conference on Wednesday, March 8, at 2:10 p.m. E.T (Press release, Dynavax Technologies, MAR 1, 2023, https://investors.dynavax.com/news-releases/news-release-details/dynavax-present-cowen-43rd-annual-health-care-conference [SID1234627976]).

Schedule your 30 min Free 1stOncology Demo!
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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The presentation will be webcast and may be accessed through the "Events & Presentations" page on the "investors" section of the Company’s website at View Source

CYTOKINETICS REPORTS FOURTH QUARTER 2022 FINANCIAL RESULTS

On March 1, 2023 Cytokinetics, Incorporated (Nasdaq: CYTK) reported financial results for the fourth quarter and full year 2022. Net loss for the fourth quarter was $137.4 million or $1.45 per share and the net loss for the year 2022 was $389.0 million or $4.33 per share (Press release, Cytokinetics, MAR 1, 2023, View Source [SID1234627975]). Net loss for the fourth quarter of 2021 was $30.6 million or $0.36 per share and net loss for the year 2021 was $215.3 million or $2.80 per share. Cash, cash equivalents and investments totaled $829.3 million at December 31, 2022.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"Aficamten remains our top priority and is advancing in a broad development program with emphasis on the conduct of SEQUOIA-HCM, our pivotal Phase 3 clinical trial in obstructive HCM, and the start of two additional Phase 3 trials." said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "At the same time, COURAGE-ALS, our Phase 3 clinical trial of reldesemtiv, is continuing to enroll with the planned second interim analysis expected to occur in the second quarter. These activities will continue as company priorities while we will also assess potential next steps for omecamtiv mecarbil. We entered 2023 with a strong balance sheet as we advance multiple late-stage programs as well as make progress in our early-stage pipeline, continuing our 25-year commitment to bringing forward potential new medicines for patients in need."

Q4 and Recent Highlights

Cardiac Muscle Programs

omecamtiv mecarbil (cardiac myosin activator)


Cytokinetics announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding the New Drug Application (NDA) for omecamtiv mecarbil, stating that GALACTIC-HF is not sufficiently persuasive to establish substantial evidence of effectiveness for reducing the risk of heart failure events and cardiovascular death in adults with chronic heart failure with
reduced ejection fraction, in lieu of evidence from at least two adequate and well-controlled clinical investigations.


The European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for omecamtiv mecarbil for the treatment of advanced or worsening HFrEF.


Ji Xing Pharmaceuticals announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of the People’s Republic of China has accepted the submission of the New Drug Application (NDA) for omecamtiv mecarbil for the treatment of heart failure with reduced ejection fraction (HFrEF) in China.


Presented results from two additional analyses from GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure) at the American Heart Association Scientific Sessions 2022, including one showing that the estimated cost reduction due to heart failure events avoided due to treatment with omecamtiv mecarbil averaged $6,052 per patient (26.9% reduction) at three years, and one that showed that women had lower quality of life at baseline and a lower rate of the primary composite outcome, but the treatment benefit of omecamtiv mecarbil did not differ between men and women (interaction p=0.68).

aficamten (cardiac myosin inhibitor)


Continued enrolling patients with obstructive hypertrophic cardiomyopathy (HCM) in SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), our first Phase 3 trial of aficamten in the U.S. and Europe.


Completed enrollment of patients with non-obstructive HCM in Cohort 4 of REDWOOD-HCM (Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM).


Continued preparations for the second Phase 3 clinical trial of aficamten as monotherapy in patients with obstructive HCM, MAPLE-HCM (Metoprolol vs Aficamten in Patients with LVOT Obstruction on Exercise Capacity in HCM).


Hired a U.S. marketing leader and continued to advance the go-to-market strategy for aficamten in the U.S.


Published a manuscript entitled "Phase 2 Study of Aficamten in Patients with Obstructive Hypertrophic Cardiomyopathy" in the Journal of the American College of Cardiology.


Published a manuscript entitled "Pharmacokinetics of a Single Dose of Aficamten (CK-274) on Cardiac Contractility in a A31P MYBPC3 Hypertrophic Cardiomyopathy Cat Model" in Journal of Veterinary Pharmacology and Therapeutics.

Skeletal Muscle Program

reldesemtiv (fast skeletal muscle troponin activator (FSTA))


Continued enrolling patients with ALS in COURAGE-ALS (Clinical Outcomes Using Reldesemtiv on ALSFRS-R in a Global Evaluation in ALS), the Phase 3 clinical trial of reldesemtiv.


Announced that the Data Monitoring Committee (DMC) for COURAGE-ALS (Clinical Outcomes Using Reldesemtiv on ALSFRS-R in a Global Evaluation in ALS) convened to review unblinded data from the clinical trial for the first interim analysis and recommended that conduct of the Phase 3 trial continue.


Presented results from an additional analysis from FORTITUDE-ALS (Functional Outcomes in a Randomized Trial of Investigational Treatment with CK-2127107 to Understand Decline in Endpoints – in ALS), the Phase 2 clinical trial of reldesemtiv showing that the ENCALS predicted risk scores are strongly correlated with the rate of decline in ALSFRS-R.


Published a manuscript entitled "MiToS and King’s staging as clinical outcome measures in ALS: a retrospective analysis of the FORTITUDE-ALS trial" in Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration.

Pre-Clinical Development and Ongoing Research


Continued to advance new muscle directed compounds and conduct IND-enabling studies with the expectation of our potentially moving 1-2 drug candidates into clinical development this year.


Continued research activities directed to our other muscle biology research programs.


Published a manuscript entitled "Distinct Mechanisms for Increased Cardiac Contraction Through Selective Alteration of Either Myosin or Troponin Activity" in the Journal of the American College of Cardiology: Basic to Translational Science.

Corporate


Joined with the European Organisation for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD) to recognize Rare Disease Day, an international campaign elevating the public understanding of rare diseases.


Awarded Cytokinetics Communications Grants to patient advocacy organizations serving the heart failure, HCM and ALS communities to support increased capacity in communications, awareness building and community engagement for nonprofit organizations serving the patient community.

2023 Corporate Milestones

Cardiac Muscle Programs

omecamtiv mecarbil (cardiac myosin activator)


Request meeting with FDA to understand what may be required to support potential approval of omecamtiv mecarbil in the United States.


Engage with EMA regarding the MAA for the treatment of HFrEF.

aficamten (cardiac myosin inhibitor)


Present data from Cohort 4 of REDWOOD-HCM at the American College of Cardiology’s 72nd Annual Scientific Session.


Present data from 48 weeks of treatment with aficamten in FOREST-HCM at the American College of Cardiology’s 72nd Annual Scientific Session.


Complete patient enrollment in SEQUOIA-HCM in Q2 2023, with results expected in Q4 2023.


Begin MAPLE-HCM, the second Phase 3 clinical trial of aficamten as monotherapy in patients with obstructive HCM in Q2 2023.


Begin a Phase 3 clinical trial of aficamten in non-obstructive HCM in 2H 2023.


Advance U.S. go-to-market strategy for aficamten.

CK-3828136 (CK-136) (cardiac troponin activator)


Expect data from the Phase 1 study of CK-136 in 2H 2023.

Skeletal Muscle Program

reldesemtiv (fast skeletal muscle troponin activator (FSTA))


Expect the Data Monitoring committee to conduct the second interim analysis from COURAGE-ALS in Q2 2023, which will assess for futility and allow for a fixed increase in total enrollment, if deemed necessary, to augment the statistical power of the trial.


Complete patient enrollment in COURAGE-ALS in Q2 2023, subject to second interim analysis.

Pre-Clinical Development and Ongoing Research


Expect to advance CK-4021586, an additional cardiac myosin inhibitor, into clinical development in 1H 2023.

Financials

Revenues for the three and twelve months ended December 31, 2022 were $2.0 million and $94.6 million, respectively, compared to $55.6 million and $70.4 million for the corresponding period in 2021. The increase in revenues for the prior year was primarily due to the recognition of $54.9 million of license revenue recognized for the transaction with Ji Xing.

Research and development expenses for the three and twelve months ended December 31, 2022 increased to $75.0 million and $240.8 million, respectively, compared to $43.5 million and $159.9 million for the same period in 2021.

The changes were primarily due to increases in current year activities for clinical development for COURAGE-ALS and our cardiac myosin inhibitor programs.

General and administrative expenses for the three and twelve months ended December 31, 2022 increased to $54.0 million and $178.0 million, respectively, from $33.8 million and $96.8 million for the same period in 2021 due to higher outside service spending in anticipation of the potential launch of omecamtiv mecarbil and an increase in personnel related cost including stock-based compensation.

2023 Financial Guidance

The company reported financial guidance for 2023. The company anticipates revenue will be up to $5 million, driven by Astellas reimbursement of the costs of COURAGE-ALS. In addition, we expect to receive $50 million in a milestone payment from Royalty Pharma upon the start of the pivotal Phase 3 clinical trial of aficamten in nHCM. Operating expenses will be in the range of $420 to $450 million, and net cash utilization will be approximately $350 to $375 million. Our current cash balance of approximately $830 million represents more than 2 years of forward cash based on our projected 2023 operating expenses and net cash utilization.

Conference Call and Webcast Information

Members of Cytokinetics’ senior management team will review the company’s fourth quarter results on a conference call today at 4:30 PM Eastern Time. The conference call will be simultaneously webcast and can be accessed from the homepage and in the Investors & Media section of Cytokinetics’ website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by registering in advance at the following link: Cytokinetics Q4 2022 Earnings Conference Call. Upon registration, participants will receive a dial-in number and a unique passcode to access the call. An archived replay of the webcast will be available via Cytokinetics’ website for twelve months.

CTI BioPharma to Present at the 43rd Annual Cowen Health Care Conference

On March 1, 2023 CTI BioPharma Corp. (Nasdaq: CTIC), a commercial biopharmaceutical company focused on the development and commercialization of novel targeted therapies for blood-related cancers, reported that the management team will present a corporate overview at the 43rd Annual Cowen Health Care Conference on Wednesday, March 8, 2023 at 10:30 a.m. ET (7:30 a.m. PT) (Press release, CTI BioPharma, MAR 1, 2023, View Source [SID1234627974]).

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A link to the live and archived webcast may be accessed on the CTI BioPharma website under the Investors & Media section: Events and Presentations.