On September 9, 2024 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a leader in the field of masked, conditionally activated biologic therapeutics, reported that the first patient has been dosed with CX-801 monotherapy in a Phase 1 study (NCT06462794) in patients with solid tumors (Press release, CytomX Therapeutics, SEP 9, 2024, View Source [SID1234646428]). CX-801 is a dually-masked interferon alpha-2b PROBODY cytokine with potential broad applicability in both traditionally immune-oncology sensitive as well as insensitive (cold) tumors.

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The CX-801 Phase 1 dose escalation study is designed to evaluate safety and signs of clinical activity for CX-801 as monotherapy and in combination with Merck’s (known as MSD outside of the US and Canada) anti-PD-1 therapy KEYTRUDA (pembrolizumab). In dose escalation, the Phase 1 study will enroll patients with select solid tumors including advanced melanoma, renal cell carcinoma, and head and neck squamous cell carcinoma to inform a potential decision to move into Phase 1b indication-specific dose expansion cohorts.

"Interferon-alpha-2b is a powerful immune-modulating cytokine that has demonstrated clinical activity in multiple cancer types such as metastatic melanoma, renal cancer and bladder cancer but its clinical benefit has been limited by significant toxicities when administered systemically. CX-801 utilizes CytomX’s industry leading conditional activation platform to maintain potency and expand interferon’s therapeutic index to potentially become a foundational component of immuno-oncology combination regimens," said Wayne Chu, M.D., chief medical officer of CytomX Therapeutics.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA

On September 9, 2024 Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, reported it has completed a successful pre-Biologics License Application (pre-BLA) meeting with the U.S. Food and Drug Administration (FDA) that supports the Company’s plans to submit a BLA for RP1 (vusolimogene oderparepvec) for the treatment of anti-PD1 failed melanoma via the accelerated approval pathway in 2H 2024 (Press release, Replimune, SEP 9, 2024, View Source [SID1234646443]).

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"This successful pre-BLA meeting confirmed that the accelerated approval path is available for RP1 in anti-PD1 failed melanoma," said Sushil Patel, Ph.D., Chief Executive Officer at Replimune. "With the confirmatory IGNYTE-3 trial underway, we remain on track to submit the BLA in 2H 2024 and continue our preparations to bring RP1 to patients with advanced melanoma."

Topline results from the primary analysis of the IGNYTE clinical trial of RP1 plus nivolumab shared earlier this year showed an overall response rate of 33%. Independently reviewed data from the IGNYTE clinical trial, including key secondary endpoints and subgroup analyses will be presented as a late-breaking abstract during an oral session at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Congress 2024 in Barcelona on Sunday, September 15, 2024, at 3:45pm CEST.

About RP1
RP1 (vusolimogene oderparepvec) is Replimune’s lead product candidate and is based on a proprietary strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF, intended to maximize tumor killing potency, the immunogenicity of tumor cell death, and the activation of a systemic anti-tumor immune response.

On September 9, 2024 Bayer reported encouraging results from the expansion part of the ongoing Phase I/II SOHO-01 study evaluating the safety and preliminary efficacy of BAY 2927088 in advanced HER2-mutant non-small cell lung cancer (NSCLC). Data were presented during the Presidential Symposium at the IASLC 2024 World Conference on Lung Cancer (WCLC) hosted by the International Association for the Study of Lung Cancer, taking place in San Diego, United States, from September 7-10, 2024 (Press release, Bayer, SEP 9, 2024, View Source [SID1234646459]).

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"The encouraging results from the expansion phase of the SOHO-01 trial demonstrate the potential of this targeted therapy to advance outcomes for patients with HER2-mutant NSCLC, a type of lung cancer with limited treatment options and poor prognosis," said Christian Rommel, Ph.D., Head of Research and Development at Bayer’s Pharmaceuticals Division. "The development of BAY 2927088 underscores our ongoing commitment in lung cancer and our continued drive to develop precise and personalized healthcare solutions in disease areas with the highest unmet needs."

"HER2-mutant NSCLC is a challenging disease that can predominantly affect individuals who are young and never smoked a cigarette. Given the current lack of therapeutic options, there is an immediate need for effective, manageable treatments," said SOHO-01 lead trialist, Xiuning Le, MD, PhD, The University of Texas MD Anderson Cancer Center, Houston, TX. "BAY 2927088 showed promising preliminary efficacy in both response rate and duration, and a manageable safety profile, which emphasizes the importance of ongoing innovation to elevate the standard of care."

The objective of the expansion phase of the study was to determine the safety, tolerability, and pharmacokinetics of BAY 2927088 in patients with HER2-mutant NSCLC. These data support the ongoing investigation of BAY 2927088 in patients with advanced NSCLC harboring HER2 mutations. Just recently Bayer announced that the first patient has been enrolled in the global Phase III SOHO-02 trial, an open-label, randomized, multicenter clinical trial, assessing the efficacy and safety of investigational agent BAY 2927088 as first-line therapy in patients with advanced non-small cell lung cancer (NSCLC), whose tumors have activating HER2 mutations.

Detailed Results from SOHO-01
SOHO-01 is an ongoing, open-label, multicenter Phase I/II study. Results from patients with advanced NSCLC harboring a HER2-activating mutation with disease progression after ≥1 systemic therapies for advanced disease, and who are naïve to HER2-targeted therapy were presented. In the trial, patients received oral BAY 2927088 20 mg twice daily. Forty-three out of forty-four patients enrolled were evaluable for efficacy, with a confirmed objective response rate (ORR) of 72.1% (n=31; 95% CI 56.3, 84.7) including 1 complete response (2.3%). Median duration of response (DOR) and progression-free survival (PFS) were 8.7 months (95% CI 4.5, not estimable) and 7.5 months (95% CI 4.4, 12.2), respectively. In patients with HER2 YVMA insertions, the most frequent mutation, ORR was 90.0%, DoR was 9.7 months, and PFS was 9.9 months.

The safety profile of BAY 2927088 was found to be manageable, and consistent with previous reports. Treatment-related adverse events (TRAEs) were reported in 95.5% of patients, with grade 3 TRAEs reported in 40.9%. Diarrhea was the most common TRAE (86.4%, 25.0% grade 3), followed by rash (43.2% grade 1 or 2) and paronychia (25.0% grade 1 or 2). Three patients (6.8%) discontinued due to TRAEs.

About BAY 2927088
BAY 2927088 is an investigational agent and has not been approved by any health authority for use in any country, for any indication. It is currently being evaluated as a potential new targeted treatment option for patients with NSCLC harboring HER2 activating mutations. BAY 2927088 is an oral, reversible tyrosine kinase inhibitor (TKI) that potently inhibits mutant human epidermal growth factor receptors 2 (HER2), including HER2 exon 20 insertions and HER2 point mutations, as well as epidermal growth factor receptors (EGFR), with high selectivity for mutant vs wild-type EGFR. Investigational agent BAY 2927088 is derived from Bayer’s strategic research alliance with the Broad Institute of MIT and Harvard in Cambridge, MA, USA.

About Breakthrough Therapy Designation
This Breakthrough Therapy designation for BAY 2927088 is supported by preliminary clinical evidence from the Phase I/II, open-label, multicenter first-in-human study (NCT05099172) evaluating the safety, pharmacokinetics and preliminary efficacy of BAY 2927088 in adult patients with advanced NSCLC harboring HER2 or EGFR. The Breakthrough Therapy designation is a process designed to expedite the development and review of novel medicines that are intended for the prevention or treatment of serious, life-threatening diseases or conditions that severely impact the quality of life for which there is no existing treatment, or where sufficient evidence indicates advantages of the novel drug over available treatment options.

About Non-Small Cell Lung Cancer (NSCLC)
Lung cancer is the leading cause of cancer-related deaths worldwide. NSCLC is the most common type of lung cancer, accounting for more than 85% of cases. Activating HER2 mutations are found in 2% to 4% of advanced NSCLC. 80% of people diagnosed with NSCLC have already progressed to advanced stages, which makes it more difficult to treat. Patients with HER2-mutant NSCLC currently face limited treatment options, highlighting an urgent need for more effective therapies.

On September 9, 2024 Evaxion Biotech A/S (NASDAQ: EVAX) ("Evaxion"), a clinical-stage TechBio company specializing in developing AI-Immunology powered vaccines, reported new exciting clinical phase 2 data for its lead compound EVX-01 (Press release, Evaxion Biotech, SEP 9, 2024, View Source [SID1234646429]). The data show that 11 out of 16 patients had objective clinical responses, equaling a 69% Overall Response Rate (ORR). 15 out of the 16 patients had reduction of their tumors (target lesions).

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This topline data is part of a one-year interim analysis of the ongoing phase 2 trial assessing EVX-01 in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in patients with advanced melanoma (skin cancer). The complete one-year clinical data will be presented at a poster session at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024, taking place in Barcelona, Spain, from September 13-17, 2024.

"We are very excited about these data, which strongly support both the clinical profile of EVX-01 as a promising personalized cancer treatment and the unique predictive capabilities of our AI-Immunology platform. To present phase 2 efficacy data for an AI-designed vaccine is a major milestone for Evaxion. Huge unmet medical needs remain in the field of melanoma, and we believe that EVX-01 could potentially be an improved treatment option for patients.
We look forward to presenting the complete one-year dataset at ESMO (Free ESMO Whitepaper), discussing the data with potential partners and advancing the phase 2 trial towards its completion next year," says Christian Kanstrup, CEO of Evaxion.

Evaxion’s innovative approach to develop personalized cancer vaccines builds on its AI-Immunology platform. The vaccines are designed to target the unique genetic makeup of an individual’s tumor and are tailored to the patients’ immune system, potentially enhancing the efficacy of treatment and improving patient outcomes.

Webinar on September 18
Evaxion will be hosting an online webinar featuring key opinion leader and the trial’s principal investigator, Professor Georgina V. Long, on September 18, 2024, at 19:00 CEST/13.00 EST. The webinar can be attended through registration via this link.

In the webinar, Professor Long will present the data from the one-year interim analysis and discuss challenges in the medical treatment of advanced melanoma. In the end, a Q&A session will be held, and participants are encouraged to present questions.

ESMO presentation details:

Abstract Title: Phase 2 study of AI-designed personalized neoantigen cancer vaccine, EVX-01, in combination with pembrolizumab in advanced melanoma
Abstract#: 1084P
Poster#: 2904
Track: Melanoma and other skin tumours
Location: Hall 6
Date/Time: September 14 at 12:00 – 13:00 CEST
Presenter: Dr. Paola Queirolo, Director, Medical Oncology of Melanoma, Sarcoma and Rare Tumors, European Institue of Oncology, Milan, Italy

About EVX-01
EVX-01 is a personalized peptide-based cancer vaccine intended for first-line treatment of multiple advanced solid cancers. It is Evaxion’s lead clinical asset.

EVX-01 is a personalized therapy designed with our AI-Immunology platform and is tailored to target the unique tumor profile and immune characteristics of each patient. It engages the patient’s immune system to fight off cancer by mounting a targeted response against tumors.

In the completed Phase 1/2a clinical trial (NCT03715985), assessing EVX-01 in combination with a PD-1 inhibitor, eight of twelve metastatic melanoma patients (67%) had objective clinical responses, with two complete and six partial responses.

In addition, vaccine-induced T cells were detected in all patients and a significant correlation between clinical response and the AI-Immunology predictions was observed, underlining the predictive power of the platform.

About EVX-01 phase 2 clinical trial
The Phase 2 clinical study (NCT05309421) is a self-sponsored open-label, single-arm, multi-center trial carried out in collaboration with leading principal investigators and research centers from Italy and Australia. The trial aims to evaluate the efficacy and safety of EVX-01 vaccination in combination with MSD’s anti-PD1 therapy KEYTRUDA (pembrolizumab) in treatment-naive patients with metastatic or unresectable malignant stage III or IV melanoma. KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA. Merck Sharp & Dohme LLC supplies KEYTRUDA (pembrolizumab) for the trial.

About melanoma
Melanoma accounts for approximately 1 in 5 of the 1.5 million new skin cancer cases estimated globally in 2020 with approximately 325,000 cases and 57,000 deaths. The global burden from melanoma is estimated to increase to 510,000 new cases and 96,000 deaths by 2040 (Arnold et al., JAMA Dermatology 2022). The global market for melanoma treatments is estimated to grow to $7.4 billion by 2029.

On September 9, 2024 Theratechnologies Inc. ("Theratechnologies" or the "Company") (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, reported that the Company’s President and CEO, Paul Lévesque will be presenting at two investor conferences in September (Press release, Theratechnologies, SEP 9, 2024, View Source [SID1234646444]). Members of the Theratechnologies management team will also be available for one-on-one meetings throughout the conferences.

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H.C. Wainwright 26th Annual Global Investment Conference
Date: September 9-11, 2024
Location: Lotte New York Palace Hotel
Presentation Details: Virtual presentation accessible online to conference attendees on the morning of Monday, September 9, 2024

2024 Cantor Global Healthcare Conference
Date: September 17-19, 2024
Location: InterContinental Barclay Hotel, New York
Presentation Details: Fireside chat on Wednesday, September 18 at 8:00-8:30 a.m. ET in Track 4, Empire Ballroom 1