On January 13, 2025 Cytokinetics, Inc. (Nasdaq: CYTK) reported guidance for corporate milestones expected to occur in 2025 and outlined its aspirational Vision 2030, five-year strategic objectives designed to propel Cytokinetics to becoming the leading muscle-focused specialty biopharmaceutical company intent on meaningfully improving the lives of patients through global access to innovative medicines (Press release, Cytokinetics, JAN 13, 2025, View Source [SID1234649715]).

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"In 2025 we are poised for a defining year with principal focus to the potential approval and commercial launch of aficamten for obstructive HCM in the United States. At the same time, we are executing on a robust clinical trials development program for aficamten inclusive of MAPLE-HCM, with results expected in the first half of this year as may potentially support the use of aficamten as monotherapy," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "In addition, we are advancing our later-stage development programs for omecamtiv mecarbil and CK-586 in adjacent specialty cardiology indications with intention to further augment our pipeline over time. Cytokinetics is well positioned to prudently and meaningfully deliver increased shareholder value as we continue to execute well on key milestones and position the company to achieve longer-term aspirations defining of our Vision 2030."

Expected 2025 Milestones

Cardiac Muscle Programs

Aficamten (cardiac myosin inhibitor)

Advance go-to-market strategies and prepare to commercially launch aficamten in the U.S. in 2H 2025, subject to approval by the U.S. Food & Drug Administration (FDA).

Continue go-to-market plans in Germany and expand commercial readiness activities in Europe in 2025, in preparation for potential approval by the European Medicines Agency (EMA) in 1H 2026.

Coordinate with Sanofi to support the potential approval of aficamten in China in 2H 2025, pending approval by the National Medical Products Administration (NMPA).

Report topline results from MAPLE-HCM (Metoprolol vs Aficamten in Patients with LVOT Obstruction on Exercise Capacity in HCM), the Phase 3 clinical trial comparing aficamten as monotherapy to metoprolol as monotherapy in patients with symptomatic obstructive HCM, in 1H 2025.

Complete patient enrollment of ACACIA-HCM (Assessment Comparing Aficamten to Placebo on Cardiac Endpoints In Adults with Non-Obstructive HCM), the pivotal Phase 3 clinical trial of aficamten in patients with non-obstructive HCM, in 2H 2025.

Complete enrollment of the adolescent cohort in CEDAR-HCM (Clinical Evaluation of Dosing with Aficamten to Reduce Obstruction in a Pediatric Population in HCM), a clinical trial of aficamten in a pediatric population with symptomatic obstructive HCM, in 2H 2025.
Omecamtiv mecarbil (cardiac myosin activator)

Continue patient enrollment in COMET-HF (Confirmation of Omecamtiv Mecarbil Efficacy Trial in Heart Failure), a confirmatory Phase 3 clinical of omecamtiv mecarbil in patients with symptomatic heart failure with severely reduced ejection fraction (HFrEF) through 2025 to enable completion of enrollment in 2026.
CK-586 (cardiac myosin inhibitor)

Complete enrollment of the first two patient cohorts in AMBER-HFpEF, (Assessment of CK-586 in a Multi-Center, Blinded Evaluation of Safety and Tolerability Results in HFpEF), a Phase 2 clinical trial of CK-586 in patients with symptomatic heart failure with preserved ejection fraction (HFpEF) in 2H 2025.
Skeletal Muscle Program

CK-089 (fast skeletal muscle troponin activator)

Complete the Phase 1 study of CK-089 in healthy human participants in 2025.
Ongoing Research

Continue ongoing pre-clinical development and research activities directed to additional muscle biology focused programs.
Vision 2030

The Company also outlined its Vision 2030: "Empowering Muscle, Empowering Lives" with the following objectives:

Innovation: Advance two approved products across three indications and ten novel molecular entities (NMEs) in our pipeline.

Ignition: Achieve broad access and rapid use of our medicines in >15 countries throughout North America and Europe.

Impact: Reach >100,000 patients globally with our medicines.

Inspiration: Foster a patient-centric culture with emphasis on equitable access.

Ingenuity: Extend our leadership in muscle biology deploying multiple therapeutic modalities.
"Our Vision 2030 provides the aspirational roadmap, aligned with our corporate five-year strategic plan, that will propel us forward as a fully integrated and leading specialty biopharma company intent on delivering innovative medicines to patients around the world," said Mr. Blum. "Vision 2030 articulates ambitious company goals to deliver product approvals, achieve broad access to our medicines, promote equitable access and advance our pioneering research to benefit patients, shareholders and employees."

On January 13, 2025 First Ascent Biomedical, a pioneer in personalized cancer treatment, reported a $6 million investment from Vidal Duart Enterprises, Inc., with an additional co-investment from Techstars (Press release, First Ascent Biomedical, JAN 13, 2025, View Source [SID1234649694]). The funding will accelerate First Ascent Biomedical’s mission to transform medical oncology with highly individualized, prospective treatment planning technologies that improve patient survival.

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First Ascent Biomedical’s technology represents a shift from a one-size-fits-all approach to a tailored strategy, offering hope to cancer patients who have exhausted traditional options. The company’s functional precision medicine platform integrates drug testing, DNA/RNA sequencing and AI-guided tumor weakness mapping to test hundreds of FDA-approved drugs against a patient’s unique cancer profile. The platform delivers a highly personalized, actionable plan to a patient’s physician in an average of 10 days. Results show that First Ascent Biomedical’s technology has significantly improved patient outcomes. Patients treated with decisions guided by the platform demonstrated an 83% greater benefit than those treated with the standard of care of a doctor’s choice.

The platform’s technology was developed by Dr. Noah Berlow, co-founder and CTO, and Dr. Diana Azzam, First Ascent Biomedical’s co-founder and scientific advisor. Dr. Berlow designed the company’s AI framework. Dr. Azzan brings years of cancer research and a wealth of functional precision medicine expertise to First Ascent Biomedical, currently serving as an assistant professor of environmental health sciences at the Florida International University Robert Stempel College of Public Health and Social Work.

The capital raised will support the expansion of First Ascent Biomedical’s operations, including the establishment of a state-of-the-art lab in Florida, where the company will continue its mission to transform cancer care for all patients, especially those in underserved communities.

"The funding we have raised is a defining moment for First Ascent Biomedical and for cancer care," said Jim Foote, Co-founder and CEO of First Ascent Biomedical. "As someone who tragically lost my son to cancer, I know the devastating toll this disease takes on families. With this fresh capital, we are not just advancing technology—we are giving patients and their families hope. Florida is where our technology was invented, so we are excited to open our first commercial lab here as we charge forward in making personalized cancer care widely accessible."

"First Ascent Biomedical is at the forefront of a new era in cancer treatment. Their groundbreaking technology has already proven its potential to save lives and change the trajectory of this disease," said Carlos Duart, Owner of Vidal Duart Enterprises. "This investment is not just a business decision—it’s a commitment to improving outcomes for cancer patients everywhere."

Tina Vidal Duart, Co-owner of Vidal Duart Enterprises, added: "We are proud to support a company that combines innovation with compassion. The work First Ascent Biomedical is doing will have a ripple effect far beyond Florida, giving patients personalized care that improves their chances of survival. We are honored to help bring this vision to life."

"First Ascent Biomedical represents the future of cancer care, where innovation and personalization come together to save lives," said Andrew Cleland, Chief Investment Officer at Techstars. "The opportunity to transform medical oncology through precision medicine is immense, and First Ascent Biomedical is paving the way to make customized cancer treatment a reality with its drug prediction technologies."

On January 13, 2025 Immunome, Inc. (Nasdaq: IMNM), a biotechnology company focused on developing first-in-class and best-in-class targeted cancer therapies, reported that Clay Siegall, Ph.D., President and CEO of Immunome, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025, at 9:45 a.m. PT (Press release, Immunome, JAN 13, 2025, View Source [SID1234649693]).

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Interested parties can access the live audio webcast for this conference from the Investor Relations section of the company’s website at www.immunome.com. The webcast replay will be available after the conclusion of the live presentation for approximately 30 days.

The presentation will cover Immunome’s pipeline of targeted therapeutics, including varegacestat (formerly known as AL102), a once-daily oral gamma secretase inhibitor in the Phase 3 RINGSIDE clinical trial for the treatment of desmoid tumors. Immunome expects to report topline data in the second half of 2025.

Immunome also announced the FDA clearance of its IND submission for IM-1021, a ROR1-targeted ADC. Immunome anticipates submitting an IND for IM-3050, its FAP-targeted radioligand therapy, by the end of the first quarter of 2025.

Immunome additionally disclosed three novel ADCs for solid tumor indications that are in preclinical development: IM-1617, IM-1340 and IM-1335.

On January 13, 2025 Incyte (Nasdaq:INCY) reported it will provide an update on commercial growth, clinical progress and significant 2025 catalysts during a presentation today at 10:30 a.m. PT at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Incyte, JAN 13, 2025, View Source [SID1234649692]).

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"With significant advancements being made across our portfolio, 2025 will be a transformational year not only for Incyte, but also for the patients we serve," said Hervé Hoppenot, Chief Executive Officer, Incyte. "We have several novel medicines in development that can potentially address significant patient needs, and we remain on track to deliver numerous first- or best-in-class high impact launches from our pipeline by 2030."

Today’s presentation will highlight:

The launch of Niktimvo (axatilimab-csfr) for patients living with chronic graft-versus-host disease (GVHD) and ongoing studies of axatilimab in additional indications.
Near-term opportunities for tafasitamab (Monjuvi) following positive results from the Phase 3 inMIND trial in patients with relapsed/refractory follicular lymphoma (FL), as well as the anticipated data evaluating tafasitamab in first line diffuse large B-cell lymphoma (DLBCL).
The potential of povorcitinib (INCB54707), an investigational oral JAK1 inhibitor, to establish best-in-class efficacy across numerous indications with high unmet needs, including hidradenitis suppurativa (HS).
Incyte’s mCALR-targeting molecule (INCA33989) and its potential to be a first-in-class targeted therapy for mCALR positive myelofibrosis (MF) and essential thrombocythemia (ET) patients.
The development path for Incyte’s CDK2 inhibitor (INCB123667) and its potential to become a foundational treatment for patients with ovarian cancer.
The J.P. Morgan Healthcare Conference presentation and Q&A session can be accessed at investor.incyte.com. A replay will be archived on the Company’s website for 30 days following the presentation.

About Niktimvo (axatilimab-csfr)

Niktimvo (axatilimab-csfr) is a first-in-class anti-CSF-1R antibody approved for use in the U.S. for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).

In 2016, Syndax licensed exclusive worldwide rights to develop and commercialize axatilimab from UCB. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab in cGVHD and any future indications.

Axatilimab is being studied in frontline combination trials in chronic GVHD – a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids are expected to initiate by year end. Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).

Niktimvo is a trademark of Incyte.

About Tafasitamab (Monjuvi)

Tafasitamab (Monjuvi) is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP). MorphoSys and Incyte entered into: (a) in January 2020, a collaboration and licensing agreement to develop and commercialize tafasitamab globally; and (b) in February 2024, an agreement whereby Incyte obtained exclusive rights to develop and commercialize tafasitamab globally.

In the United States, Monjuvi (tafasitamab-cxix) received accelerated approval by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). In Europe, Minjuvi (tafasitamab) received conditional Marketing Authorization from the European Medicines Agency in combination with lenalidomide, followed by Minjuvi monotherapy, for the treatment of adult patients with relapsed or refractory DLBCL who are not eligible for ASCT.

XmAb is a registered trademark of Xencor, Inc.

Monjuvi, Minjuvi, the Minjuvi and Monjuvi logos and the "triangle" design are registered trademarks of Incyte.

About Povorcitinib (INCB54707)

Povorcitinib (INCB54707) is an oral small-molecule JAK1 inhibitor currently in Phase 3 clinical trials for hidradenitis suppurativa (HS) and vitiligo. A Phase 3 trial is being planned for prurigo nodularis (PN). Phase 2 studies of povorcitinib in PN, asthma and chronic spontaneous urticaria are also ongoing.

On January 13, 2025 Daiichi Sankyo Company, Ltd (TSE: 4568) reported that it will pay Glycotope $132.5 million to acquire intellectual property rights of the anti-tumor-associated mucin-1 (TA-MUC1) antibody, gatipotuzumab (Press release, Daiichi Sankyo, JAN 13, 2025, https://www.businesswire.com/news/home/20250113621008/en/Daiichi-Sankyo-Acquires-Intellectual-Property-Rights-for-Anti-TA-MUC1-Antibody-in-DS-3939-from-Glycotope-GmbH [SID1234649691]). Such payment by Daiichi Sankyo satisfies all potential clinical, regulatory and sales milestone payments, as well as royalties of products that include gatipotuzumab as part of a 2018 licensing agreement between the parties. In 2018, Daiichi Sankyo in-licensed exclusive rights to develop and commercialize gatipotuzumab worldwide as an antibody drug conjugate (ADC) from Glycotope.

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The anti-TA-MUC1 is the antibody contained in DS-3939, an ADC being developed by Daiichi Sankyo. DS-3939 is a specifically engineered potential first-in-class TA-MUC1 directed medicine designed using Daiichi Sankyo’s proprietary DXd ADC technology. DS-3939 is currently being evaluated in a phase 1/2 clinical trial in patients with several types of advanced solid tumors including non-small cell lung, breast, urothelial, ovarian, biliary tract and pancreatic cancer.

About TA-MUC1
TA-MUC1 is a tumor-specific transmembrane glycoprotein with aberrant glycosylation due to changes of the expression patterns of some sialyltransferases.1 Based on the overexpression of TA-MUC1 in most human epithelial cancers, it is an attractive target for cancer therapy.2 Currently, there are no TA-MUC1 directed therapies approved for any type of cancer.

About DS-3939
DS-3939 is an investigational potential first-in-class TA-MUC1 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, DS-3939 is comprised of a humanized anti-TA-MUC1 antibody, attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.