On January 8, 2024 Incyte (Nasdaq:INCY) reported that it will highlight growth opportunities and provide key updates across its investigational pipeline and commercial portfolio during a presentation today at 7:30 a.m. PT at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Incyte, JAN 8, 2024, View Source [SID1234639131]).

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"As we enter 2024, we see great promise in our portfolio and its potential to drive long-term growth fueled by our strong R&D engine and broad commercial footprint across Oncology and Inflammation & Autoimmunity," said Hervé Hoppenot, Chief Executive Officer, Incyte. "Today, we provide further clarity into several potential high-impact product launches anticipated by 2030. This includes both indication expansion opportunities and the advancement of novel medicines that could broaden our ability to positively affect patients’ lives."

Notably, Incyte announces that the primary endpoint was met in its randomized, placebo-controlled, Phase 2 study evaluating the safety and efficacy of ruxolitinib cream (Opzelura) in adults with mild/moderate hidradenitis suppurative (HS). At Week 16, patients receiving ruxolitinib cream 1.5% twice daily (BID) had significantly greater decreases from baseline versus placebo in total abscess and inflammatory nodule (AN) count, the primary endpoint of the study. The overall safety profile of ruxolitinib cream is consistent with previous data, and no new safety signals were observed. The Phase 2 data will be submitted for presentation at an upcoming scientific meeting in 2024. A Phase 3 study is currently being evaluated.

Additionally, the Company will highlight progress across its Oncology pipeline and the research it is advancing in areas of high potential, including promising early clinical efficacy data for INCB123667, a potent and selective inhibitor of CDK2, demonstrating its potential use as monotherapy or combination therapy for late-stage cancers. In a Phase 1 study of INCB123667, early clinical activity was observed with several patients with amplified/overexpression of CCNE1, a cell cycle regulator and potential predictive biomarker, achieving partial response (PR). Tumor shrinkage was observed across multiple tumor types, including CCNE+ patients with ovarian cancer. The safety profile of for INCB123667 aligns with the mechanism of action.

In addition, Incyte will provide key updates on:

Expanding leadership in myeloproliferative neoplasms (MPNs) and graft-versus-host disease (GVHD) with a pipeline including axatilimab, mCALR and V617F that has disease-modifying potential for patients with graft-versus-host disease and the more than 200,000 patients with myelofibrosis, polycythemia vera and essential thrombocythemia.
Its emerging dermatology franchise, and the intent to maximize the potential of ruxolitinib cream (Opzelura) and expand povorcitinib into multiple indications with high unmet need.
The Company intends to provide additional financial guidance and updates on key clinical programs during its 2023 fourth quarter and year-end earnings conference call.

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.

On January 8, 2024 Nuvation Bio Inc. (NYSE: NUVB), a biopharmaceutical company tackling some of the greatest unmet needs in oncology by developing differentiated and novel therapeutic candidates, reported that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application to evaluate NUV-1511, the first clinical candidate from the company’s novel drug-drug conjugate (DDC) platform (Press release, Nuvation Bio, JAN 8, 2024, View Source [SID1234639129]).

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"This IND clearance expands our clinical pipeline and validates the approach of our proprietary DDC platform to design potent oncology-focused chimeric small molecules which combine tumor-targeting specificity with the anti-cancer activity of known oncology agents," said David Hung, M.D., Founder, President, and Chief Executive Officer of Nuvation Bio. "We believe NUV-1511 has the potential to provide favorable clinical benefit for patients with various solid tumors and we look forward to initiating the Phase 1/2 study."

The dose escalation portion of the Phase 1/2 study will initially evaluate safety and tolerability, and explore the potential for clinical efficacy, in patients with advanced solid tumors who previously received and progressed on or after treatment with Enhertu and/or Trodelvy per approved FDA labeling, human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer, metastatic castration-resistant prostate cancer (mCRPC), pancreatic cancer, and platinum-resistant ovarian cancer (PROC).

On January 8, 2024 Medison Pharma ("Medison"), a global pharma company focused on providing access to highly innovative therapies to patients in international markets, reported their exclusive multi-national agreement with Regeneron Ireland DAC, a wholly owned subsidiary of Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN), a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases, to commercialize Libtayo (cemiplimab), a fully human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T cells, in select European markets and additional markets around the world (Press release, Medison Pharma, JAN 8, 2024, View Source;agreement-with-regeneron-pharmaceuticals-to-commercialize-libtayo-cemiplimab-in-multiple-countries-302028359.html [SID1234639128]).

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Libtayo was invented in laboratories at Regeneron, which acquired exclusive worldwide development, commercialization, and manufacturing rights to the medicine from Sanofi in July 2022. Medison and Regeneron will work together with all stakeholders and regulatory authorities to facilitate a seamless transition of commercialization activities to Medison during the first half of 2024.

Libtayo, which was invented using Regeneron’s proprietary VelocImmune technology, is currently approved by regulatory authorities in more than two dozen countries, including by the European Medicines Agency (EMA). In select markets, it is approved as monotherapy treatment for certain patients with advanced basal cell carcinoma (BCC), as monotherapy treatment for certain patients with advanced cutaneous squamous cell carcinoma (CSCC), as both monotherapy or in combination with chemotherapy for certain patients with advanced non-small cell lung cancer (NSCLC) and/or as monotherapy treatment for certain patients with recurrent or metastatic cervical cancer. Libtayo is a leading and first-in-class PD-1 inhibitor approved for the treatment of two non-melanoma skin cancers and is considered standard of care in these indications.

"Our multi-regional commercial platform is a great fit for Regeneron’s go-to-market plan and another great opportunity for Medison to facilitate access to highly innovative therapies for people suffering from rare and severe diseases," said Meir Jakobsohn, Founder and Executive Chairman of Medison. "Regeneron’s selection of Medison is yet another testimony of the unique value that our multi-regional solution can generate for innovative biotech companies wishing to focus on selected markets."

"We are proud to partner with Regeneron," said Gil Gurfinkel, CEO of Medison. "Our one-of-a-kind centralized, unified commercial platform, with single alliance management across multiple markets and regions, is of great value to more and more leading biotech companies. Our platform enables biotech companies to transform numerous complex and fragmented markets and regions into a unified territory."

About Libtayo

Libtayo is a fully human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T cells and was invented using Regeneron’s proprietary VelocImmune technology. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation. In the U.S. and other countries Libtayo is indicated in certain patients with advanced BCC, advanced CSCC and advanced NSCLC, as well as in advanced cervical cancer in the European Union, Canada and Brazil. As of July 1, 2022, Libtayo is developed and marketed globally by Regeneron. Outside of the U.S., the generic name for Libtayo is cemiplimab.

The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. Libtayo is currently being investigated in trials as a monotherapy, as well as in combination with either conventional or novel therapeutic approaches for other solid tumors and blood cancers. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.

Select Approved Indications for Libtayo

Libtayo is a cancer medicine used in adults to treat:

a type of skin cancer called cutaneous squamous cell carcinoma (CSCC) when the cancer is locally advanced (has spread nearby) or metastatic (has spread to other parts of the body). It is used in patients who cannot have surgery or treatment with radiation to cure their disease;
a type of skin cancer called basal cell carcinoma (BCC) when the cancer is locally advanced or metastatic. It is used in patients who cannot tolerate treatment with a type of medicine called a ‘hedgehog pathway inhibitor (HHI)’ or whose disease has worsened after such treatment;
a type of lung cancer called non-small cell lung cancer (NSCLC) when the cancer is locally advanced and cannot be treated with chemotherapy (medicines to treat cancer) and radiation therapy, or when the cancer is metastatic. It is used either alone in patients whose tumours have a protein called PD-L1 in more than 50% of cells and no mutations in the genes EGFR, ALK and ROS1 involved in the development of NSCLC, or together with platinum-based chemotherapy in patients whose tumours have PD-L1 in at least 1% of the cells and no mutations in the EGFR, ALK and ROS1 genes;
cervical cancer that has come back (recurrent) or is metastatic. It is used in patients whose disease has progressed during or after treatment with platinum-based chemotherapy.

It is not known if Libtayo is safe and effective in children.

On January 8, 2024 Shuttle Pharmaceuticals Holdings, Inc. (Nasdaq: SHPH) ("Shuttle Pharma") reported that they have received the ‘Safe to Proceed’ letter from the U.S. Food and Drug Administration (FDA) for the Company’s investigational new drug (IND) application for its Phase II study of Ropidoxuridine (IPdR) as a radiation sensitizing agent during radiotherapy in patients with newly diagnosed IDH-wildtype glioblastoma with unmethylated MGMT promoter (Press release, Shuttle Pharmaceuticals, JAN 8, 2024, View Source [SID1234639127]). Receipt of the letter allows Shuttle to commence the Phase II study.

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Shuttle Pharma is currently finalizing site enrollment with ‘first patient, first dose’ expected in the coming months. Ropidoxuridine is Shuttle Pharma’s lead radiation sensitizer candidate for use in combination with radiation therapy (RT) to treat glioblastoma, a deadly malignancy of the brain with no known cure.

"We are excited to have been granted approval to commence Ropidoxuridine’s Phase II clinical trial following the receipt of the FDA’s ‘Safe to Proceed’ letter," stated Shuttle Pharma’s Chairman and CEO, Anatoly Dritschilo, M.D. "Radiation therapy is a proven modality for treating cancers. However, there is a significant need in the market to make radiation more effective. The results of this Phase II clinical trial will be important as we look to leverage radiation sensitizers to increase cancer cure rates, prolong patient survival and improve quality of life for patients suffering from glioblastoma."

An estimated 800,000 patients in the US are treated with radiation therapy for their cancers yearly. According to the American Cancer Society and the American Society of Radiation Oncologists, about 50% are treated for curative purposes and the balance for therapeutic care. The market opportunity for radiation sensitizers lies with the 400,000 patients treated for curative purposes, with this number expected to grow by more than 22% over the next five years.

Shuttle Pharma has received Orphan Drug Designation from the FDA, providing potential marketing exclusivity upon first FDA approval for the disease.

On January 8, 2024 GC Cell, a fully integrated cell therapy pioneer, reported the approval from both the Australian Human Research Ethics Committee (HREC) and the Korean Ministry of Food and Drug Safety (MFDS) for a Phase 1 Investigational New Drug (IND) trial for its AB-201 cancer treatment, a HER2 targeted chimeric antigen receptor-natural killer (CAR-NK) cell therapy that shows great promise (Press release, GC Cell, JAN 8, 2024, View Source [SID1234639126]). Additionally, GC Cell is excited to reveal a strategic partnership with Lunit, the medical AI leader, to enhance precision and clinical intelligence of AB-201’s efficacy evaluation by applying its advanced AI technology, in addition to traditional Immunohistochemistry (IHC) based assessments. After participating in the US White House’s Cancer Moonshot initiative this past October, both accomplishments represent major milestones in GC Cell’s efforts to revolutionize immunotherapy-based cancer treatment while harnessing AI and digital transformation to fight cancer.

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"Our preclinical studies suggest the possibility that AB-201 causes complete cancer remission and tumor suppression," remarked James Park, CEO of GC Cell. "We are thrilled to move forward to Phase 1 trials — the first Korean company to do so for CAR-NK cell therapy — and we are grateful to Lunit for joining hands with us in our digital transformation and the fight against cancer — now armed with the power of AI. This initiative is part of GC Cell’s broader strategy to integrate digital & AI technology into all aspects of healthcare, from research and development to patient care. I believe it will prepare our company for the upcoming era of digital healthcare."

Promising treatment to begin clinical trials

AB-201, a novel CAR-NK cell therapy targeting solid tumors, represents a breakthrough in cancer immunotherapy, capable of killing malignant cells. While existing NK cell treatments typically dissipate within a few weeks, AB-201 has demonstrated persistence for over three months in preclinical studies, highlighting its potential in managing long-term, advanced cancers. A multi-country study, the Phase 1 trial will evaluate safety and efficacy in 48 patients with HER2-overexpressing breast, gastric, and gastroesophageal junction cancers. GC Cell holds exclusive rights to AB-201 in the Asia Pacific region, while ex-Asia-Pacific rights were licensed to Artiva Biotherapeutics, Inc. in an exclusive partnership.

Lunit partnership for AI-powered pharma development

A recent McKinsey report found that AI has the potential to increase the value of the pharmaceutical R&D industry by six to eleven billion US dollars. Employing the technology, GC Cell anticipates that AI-driven imaging can analyze vast amounts of data with a high degree of precision and consistency while reducing variability by individual interpretation. This leads to a more accurate assessment of HER2 expression levels, which is vital for appropriate treatment decisions. With such quantitative analysis, AI can help standardize the assessment of cancer target expression across different laboratories and geographical locations. This standardization is essential for multicenter clinical trials and for ensuring that patients receive consistent care regardless of where they are treated. It’s a pivotal step in GC Cell’s journey towards a digital future, aligning with the broader healthcare industry’s shift towards technology-enhanced solutions.

About AB-201

AB-201 is an allogeneic HER2-targeted chimeric antigen receptor NK (CAR-NK) cell therapy for the treatment of solid tumors in the outpatient setting with the option for repeat dosing. A novel, high affinity anti-HER2 antibody converted to scFv structure confers highly specific tumor targeting and is coupled with a unique costimulatory structure and IL-15 expression for enhanced activity and persistence. AB-201 has demonstrated potent anti-tumor activity in multiple preclinical HER2-positive tumor model systems. The underlying NK cell is derived from umbilical cord blood donors preselected for advantageous attributes including the high affinity variant of the CD16 receptor and a KIR-B haplotype. AB-201 cell products maintain high expression of CD16, as well as other activating innate cell tumor engaging receptors, enabling the potential for dual targeting therapeutic approaches via monoclonal antibody combinations. The resulting CAR-NK is manufactured at very large scale and cryopreserved in infusion-ready media to enable repeat clinical administrations in the outpatient setting.

GC Cell holds exclusive rights to AB-201 in the Asia-Pacific region, while ex-Asia-Pacific rights were licensed to Artiva Biotherapeutics, Inc. in an exclusive partnership. The U.S. Food and Drug Administration previously cleared Artiva’s investigational new drug (IND) application for AB-201 in 2022.