Karyopharm to Present at 41st Annual J.P. Morgan Healthcare Conference

On January 4, 2023 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that the Company’s senior management team will participate at the 41st Annual J.P. Morgan Healthcare Conference in a podium presentation on Wednesday, January 11 at 10:30 a.m. PT, followed by a question-and-answer breakout session at 10:50 a.m. PT (Press release, Karyopharm, JAN 4, 2023, View Source [SID1234625813]).

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A live webcast of the presentation, along with accompanying slides, can be accessed under "Events & Presentations" in the Investor section of the Company’s website, View Source, and will be available for replay for 30 days following the event.

ISA Pharmaceuticals and Cancer Focus Fund Announce $5 Million Investment to Support First-in-Human Trial of ISA’s Novel Immunotherapy ISA103 in Uveal Melanoma

On January 4, 2023 ISA Pharmaceuticals B.V., a clinical stage biotech company developing immunotherapies to treat cancers and infectious diseases, and Cancer Focus Fund, LP, a unique investment fund established in collaboration with The University of Texas MD Anderson Cancer Center to provide funding and clinical expertise to advance promising cancer therapies, reported that Cancer Focus Fund is investing $5 million in funding to support ISA103, ISA’s PRAME-targeting immunotherapy, in a first-in-human study for the treatment of uveal melanoma, a rare ocular cancer (Press release, ISA Pharmaceuticals, JAN 4, 2023, View Source [SID1234625811]).

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ISA103 immunotherapy targets PRAME (PReferentially expressed Antigen in MElanoma), a cancer testis antigen that is overexpressed in various solid and liquid cancers. It plays a crucial role in tumor survival and spread. ISA103 was developed using the company’s proprietary Synthetic Long Peptide (SLP) technology, designed to fully harness and direct the body’s defense mechanisms towards fighting the disease. It contains multiple long peptides spanning the most immunogenic regions of the PRAME protein.

"At ISA we believe that our rationally designed immunotherapies have the potential to produce improved outcomes for cancer patients," said Gerben Moolhuizen, Chief Executive Officer of ISA Pharmaceuticals. "We have already achieved proof-of-concept successes with our lead product ISA101b in HPV-induced cancers, and we view PRAME-positive tumors as another promising target for our unique approach. We believe this investment from the Cancer Focus Fund and the clinical expertise provided by MD Anderson will significantly advance ISA103 as an immunotherapy with the potential to combat this rare but debilitating malignancy."

"Cancer Focus Fund is committed to using its investments to support the clinical development of truly innovative cancer therapies," said Ross Barrett, a founder and Managing Partner of Cancer Focus Fund. "ISA’s multi-pronged approach to the design and development of fully synthetic novel immunotherapeutics has great potential and importantly, has already demonstrated early success in its initial programs. Our distinctive investment model is continuing to resonate globally, with another of our portfolio programs now based outside the U.S., and we are pleased to be collaborating in this effort with ISA and MD Anderson."

PRAME expression is common in uveal melanoma and is associated with poor disease outcome. In the two-part open label Phase 1b/2 trial, ISA103 will be tested in combination with standard of care checkpoint blocker immunotherapies. An initial dose-finding phase will be followed by an expansion cohort using the optimal dose. Study endpoints will include safety, immunogenicity and signs of efficacy (response rates and survival indicators). A total of 90 patients will be enrolled in the trial. The trial will be conducted at MD Anderson under the direction of Principal Investigator Sapna Patel, M.D., Associate Professor of Melanoma Medical Oncology and Director of MD Anderson’s Uveal Melanoma Program.

About ISA103 and PRAME

PRAME (PReferentially expressed Antigen in MElanoma) is a protein that is expressed on various types of tumors, including lung, breast, head & neck and kidney cancers, as well as melanoma and neuroblastoma, while there is minimal expression in heathy tissue. There appears to be a functional association of PRAME expression and tumorigenesis. Overexpression of PRAME is associated with poor disease prognosis. ISA103 is designed to create a strong and specific T cell immune response against PRAME. It consists of multiple Synthetic Long Peptides (SLP) spanning the most immunogenic regions of the PRAME protein. ISA103 is under investigation as a potential treatment for PRAME expressing cancers.

About ISA’s SLP Technology

ISA’s SLP immunotherapeutics are easy-to-manufacture synthetic peptides. They cover the most immunogenic regions of the therapeutic target and contain epitopes for the efficient induction of both CD4 and CD8 T cell responses by the patient, regardless of the individual’s HLA type. SLP-based immunotherapeutics overcome central tolerance, cover a broad range of HLA restricted epitopes to induce strong and lasting specific T cell responses, and are complementary to standard of care therapy, including, in the case of cancer, chemotherapy and immunomodulators like anti-PD1 antibodies.

About Cancer Focus Fund
The Cancer Focus Fund LP is a unique investment fund established in collaboration with The University of Texas MD Anderson Cancer Center. The fund provides investment support to advance promising cancer therapies that are close to being tested in humans or are in early clinical development, as well as the clinical trial expertise and infrastructure of MD Anderson and strategic partners Ochsner Health System Precision Cancer Therapies Program New Orleans and the LSU Feist Weiller Cancer Center Shreveport. The Fund’s objective is to leverage this unique combination to provide investors with superior risk-adjusted returns. In collaboration with partner MD Anderson, the Cancer Focus Fund provides both capital and translational research expertise with the goal of accelerating the development of novel cancer therapies that result in better outcomes for patients while generating returns for investors.

Disclosures

The University of Texas MD Anderson Cancer Center’s relationship with Cancer Focus Fund, and all research conducted at MD Anderson related to Cancer Focus Fund, has been identified as an institutional financial conflict of interest by MD Anderson’s Institutional Conflict of Interest Committee and therefore is managed under an Institutional Conflict of Interest Management and Monitoring Plan.

Invitae to Present at the 41st Annual J.P. Morgan Healthcare Conference

On January 4, 2023 Invitae (NYSE: NVTA), a leading medical genetics company, reported that Ken Knight, president & chief executive officer, will present at the 41st Annual J.P. Morgan Healthcare Conference on Wednesday, January 11, 2023, at 12:00 p.m. Eastern / 9:00 a.m. Pacific (Press release, Invitae, JAN 4, 2023, View Source [SID1234625810]).

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The live audio webcast of the presentation may be accessed by visiting the investors section of the company website at ir.invitae.com. A replay of the webcast will be available shortly after the conclusion of the presentation.

Invectys and CTMC Announce FDA Clearance of IND Application for Anti-HLA-G CAR-T Cell Therapy

On January 4, 2023 Invectys, Inc. and CTMC, a joint venture between MD Anderson Cancer Center and National Resilience, Inc., reported Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for a Phase 1/2a clinical study of IVS-3001, Invectys’s lead engineered human leukocyte antigen A (HLA-G) targeting chimeric antigen receptor (CAR) T cell therapy for the treatment of solid tumors (Press release, Invectys, JAN 4, 2023, View Source [SID1234625809]).

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IVS-3001 has been developed in collaboration with CTMC as part of an agreement announced in 2022, and it will now move forward through an MD Anderson-sponsored trial led by principal investigator (PI) Aung Naing, M.D., professor of Investigational Cancer Therapeutics at MD Anderson and co-PI Samer Srour, M.D., assistant professor of Stem Cell Transplantation and Cellular Therapy at MD Anderson. The planned first-in-human, single-arm, open-label, Phase 1/2a study will evaluate the safety, tolerability, pharmacokinetics, and clinical activity of IVS-3001 in patients with histologically or pathologically confirmed diagnosis of a locally advanced unresectable or metastatic HLA-G+ select solid tumor malignancy who failed or was intolerant to standard of care therapies known to confer clinical benefit per treating physician.

IVS-3001 may be administered to up to a total of 117 patients under approved protocol. Up to 24 may be treated in Phase 1 Dose Escalation and up to 93 may be treated in Phase 2a. All participants will be asked to enter the long-term follow up (LTFU) study as per FDA standard requirement for all gene and cell therapies. The IVS-3001 therapy for the Phase I trial will be manufactured by CTMC, which was launched to speed the development and manufacturing of innovative cell therapies for patients with cancer.

"CTMC was created to accelerate impactful cell therapies reaching patients, » said Jason Bock, CEO of CTMC. "We are excited to partner with Invectys to move the HLA-G CAR-T from contract execution to IND safe-to-proceed, in less than a year."

"The FDA clearance of the IND application for IVS-3001 represents an important milestone for Invectys and our colleagues at CTMC, and it is the result of years of commitment to developing a novel class of engineered CAR-T therapy," said Praveen Tyle, Ph.D., CEO of Invectys. "We believe IVS-3001 therapy has the potential to significantly transform the treatment landscape for cancer patients and the potential to achieve improved clinical outcomes."

About IVS–3001

IVS-3001 is an HLA-G-targeting chimeric antigen receptor (CAR) T cell therapy. HLA-G is not only an immune checkpoint but also a tumor-specific antigen. The CAR construct consists of the HLA-G antigen-targeting domain fused to intracellular signaling domains. The CAR allows IVS-3001 to recognize and kill target cells which express HLA-G on the cell surface. Antigen-specific activation of IVS-3001 results in CAR-T cell proliferation, cytokine secretion, and subsequent cytolytic killing of HLA-G-expressing cells.

IVS-3001 preclinical studies have generated adequate data to support efficacy and safety of the CAR-T therapy leading to IND clearance and initiation of First-in-Human clinical trial.

Immutep Achieves 50% Enrollment Milestone in Randomised Phase IIb TACTI-003 Trial for First Line Head & Neck Cancer

On January 4, 2023 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a clinical-stage biotechnology company developing novel LAG-3 immunotherapies for cancer and autoimmune disease, reported that it has enrolled and randomised over 50% of the planned 154 patients in the TACTI-003 Phase IIb trial (Press release, Immutep, JAN 4, 2023, View Source [SID1234625808]). TACTI-003 is evaluating Immutep’s first-in-class soluble LAG-3 protein eftilagimod alpha ("efti"), in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy KEYTRUDA (pembrolizumab) as 1st line treatment of recurrent or metastatic head and neck squamous cell carcinoma (1L HNSCC).

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Marc Voigt, CEO of Immutep stated: "We are pleased to reach this important milestone and extend our sincere appreciation to our investigators, clinical team, partners, and most importantly patients, that have participated in this study. As clinical evidence showing the compelling benefits of combining efti with immune checkpoint therapies such as pembrolizumab continues to grow, we are increasingly excited about efti’s potential to safely deliver superior clinical outcomes and meaningfully expand the population of cancer patients that respond to treatment."

The 1:1 randomised, controlled multinational TACTI-003 trial is currently accruing patients at over 25 centers in the United States, Australia, and Europe, and is expected to be fully recruited by mid-2023. Based largely on the promising data in 2nd line HNSCC from the Phase II TACTI-002 trial (KEYNOTE-798), including encouraging overall response rates regardless of PD-L1 expression and five complete responses (CR), eftilagimod alpha was granted Fast Track designation by the FDA in April 2021 for treatment of 1L HNSCC.

As recently announced, the Independent Data Monitoring Committee (IDMC) for the TACTI-003 trial reviewed initial safety data and recommended continuing the trial with no modifications. The IDMC also reviewed initial efficacy data, although this was not the primary focus of the analysis.

HNSCC is the sixth most common cancer by incidence worldwide, with 890,000 new cases and 450,000 deaths reported in 2018.1,2,3 HNSCC is an aggressive, genetically complex, and difficult to treat cancer.4 Furthermore, HNSCC is associated with high levels of psychological distress and compromised quality of life (QOL).5 As such, HNSCC patients are in need of improved treatment options.

1
Ferlay, J. et al. Estimating the global cancer incidence and mortality in 2018: GLOBOCAN sources and methods. Int. J. Cancer 144, 1941–1953 (2019).

2
Bray, F. et al. Global cancer statistics 2018: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J. Clin. 68, 394–424 (2018).

3
Ferlay, J. et al. Global Cancer Observatory: Cancer Today. Lyon, France: International Agency for Research on Cancer (accessed 18 September 2020). IARC View Source (2018).

4
Alsahafi, E., Begg, K., Amelio, I. et al. Clinical update on head and neck cancer: molecular biology and ongoing challenges. Cell Death Dis 10, 540 (2019).

5
Johnson, D.E., Burtness, B., Leemans, C.R. et al. Head and neck squamous cell carcinoma. Nat Rev Dis Primers 6, 92 (2020).

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About Eftilagimod Alpha (Efti)

Efti is Immutep’s proprietary soluble LAG-3 clinical stage candidate that is a first-in-class antigen presenting cell (APC) activator for the treatment of cancer, capitalising on LAG-3’s unique characteristics to stimulate both innate and adaptive immunity. Efti binds to and activates antigen presenting cells via MHC II molecules leading to expansion and proliferation of CD8+ (cytotoxic) T cells, CD4+ (helper) T cells, dendritic cells, NK cells, and monocytes. It also upregulates the expression of key biological molecules like CXCL10 that further boost the immune system’s ability to fight cancer.

Efti is under evaluation for a variety of solid tumours including non-small cell lung cancer (NSCLC), head and neck squamous cell carcinoma (HNSCC), and HER2–/HR+ metastatic breast cancer. Its favourable safety profile enables various combinations, including with anti-PD-[L]1 immunotherapy and/or chemotherapy. Efti has received Fast Track Designation in 1st line HNSCC and in 1st line NSCLC from the United States Food and Drug Administration (FDA).

About TACTI-003

TACTI-003 is a Phase IIb clinical trial in 1st line head and neck squamous cell carcinoma (HNSCC). The study will evaluate efti in combination with MSD’s KEYTRUDA (pembrolizumab) as a 1st line therapy in metastatic or recurrent HNSCC patients with PD-L1 negative and PD-L1 positive (CPS ≥1) tumours. It is a randomised, controlled clinical study in approximately 154 patients and will take place across Australia, Europe and the United States of America in up to 35 clinical sites.

The study will evaluate the safety and efficacy of efti in combination with pembrolizumab, compared to pembrolizumab alone in 1st line metastatic or recurrent HNSCC patients with PD-L1 positive (CPS ≥1) tumours (cohort A), and determine the efficacy and safety of efti plus pembrolizumab in patients with PD-L1 negative tumours (CPS <1) (cohort B). According to the current plans, about 130 patients in cohort A will be randomised 1:1 to receive either efti plus pembrolizumab or pembrolizumab alone. Subjects in cohort B (up to 24 patients) will receive a combination of efti and pembrolizumab. The primary endpoint of the study is Overall Response Rate (ORR) according to RECIST 1.1. Secondary endpoints include Overall Survival (OS) and Progression Free Survival (PFS). For more information about the Phase IIb trial, visit clinicaltrials.gov (NCT04811027).