On May 20, 2024 Cantex Pharmaceuticals, Inc., a clinical-stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions for which new treatments are urgently needed, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Cantex’ azeliragon, a well-tolerated once-a-day pill, for the treatment of pancreatic cancer (Press release, Cantex, MAY 20, 2024, View Source [SID1234643471]). This new azeliragon orphan drug designation adds to azeliragon’s previous orphan drug designation for the treatment of glioblastoma, received in early 2023.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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!

Pancreatic cancer, unless controlled at its earliest stages, often spreads to local tissues and to distant organs. Although surgery can be curative if pancreatic cancer is diagnosed at its very earliest stages, once the cancer has spread to distant organs, the current treatment has some, although limited, efficacy. New treatments are clearly needed.

Cantex has an ongoing clinical trial studying the safety and efficacy of azeliragon in patients refractory to first-line treatment of metastatic pancreatic cancer. This clinical trial is enrolling metastatic pancreatic cancer patients in several world-class cancer treatment centers in the U.S.

"Receiving FDA orphan drug status for azeliragon for the treatment of pancreatic cancer underscores the significant unmet need for novel treatment options for patients with pancreatic cancer, particularly for those patients with locally advanced, unresectable, or metastatic disease," commented Stephen G. Marcus, M.D., Chief Executive Officer of Cantex. "This designation strengthens our continued commitment to developing new azeliragon treatment options for patients with pancreatic cancer, as well as for other cancers and their complications, including glioblastoma, brain metastasis and breast cancer."

FDA Orphan Drug Designation provides Cantex with seven years of azeliragon marketing exclusivity from the time of product launch for the orphan indication, and several other important benefits, including assistance in the drug development process, tax credits for clinical costs, and exemptions from certain FDA fees.

About Azeliragon

Azeliragon is an orally administered capsule, taken once daily, that inhibits interactions of the receptor for advanced glycation end products (known as RAGE) with certain ligands, including HMGB1 and S100 proteins in the tumor microenvironment. Azeliragon was discovered by and originally under development for Alzheimer’s disease by vTv Therapeutics Inc. (NASDAQ: VTVT) from which Cantex licensed worldwide rights to azeliragon. Clinical safety data from these trials, involving more than 2000 individuals dosed for periods up to 18 months, indicate that azeliragon is very well tolerated.

Cantex has ongoing Phase 2 clinical trials in pancreatic cancer, glioblastoma, brain metastasis, breast cancer, and a Phase 3 trial in hospitalized patients with pneumonia. These trials are based on azeliragon’s robust preclinical data as well as its extensive clinical safety information from randomized placebo-controlled clinical trials.

On May 20, 2024 YGION Biomedical GmbH, a company dedicated to developing individualized neoantigen-based cancer vaccines, reported the completion of a Series A financing round of €15 million from an Austrian private trust (Press release, YGION Biomedical, MAY 20, 2024, View Source [SID1234643470]). The proceeds will be used to further develop YGION’s unique YGNITETM technology platform and advance the lead program YG-01 into preclinical and clinical development.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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!

Founded in 2022, YGION’s high-profile team of experienced biotech veterans and entrepreneurs are focused on developing individualized "plug-and-play" cancer vaccines through a proprietary suite of technologies to identify, produce and target the delivery of relevant peptide neoepitopes resulting in safe, exceptionally potent, and precise targeted activation of the patient’s immune system.

"We are on the cusp of a breakthrough in individualized cancer treatments" said Dr. Wolfgang Fischl, Chief Executive Officer of YGION. "This financing is truly exciting for YGION as we see it as a strong validation of our unique approach, and we are grateful for the support of our investors enabling us to take a significant step towards clinical validation of our technology. With the continued demonstration of the efficacy of our approach, we are open to additional investors and collaborators interested in our ground-breaking approach."

"We are confident that with our technology, we can realize the promise of simple, safe and effective cancer vaccines" added Dr. Geert Mudde, Chief Technology Officer of YGION. "Our YGNITETM platform gives us the unique ability to identify, select and synthesise relevant neoantigens, and combined with our CARGONAUTTM immune-modulating carrier induces a powerful cell-mediated tumor specific response that targets and eliminates malignant cells with unmatched safety."

YGION’s lead program YG-01 is currently in preclinical development.

On May 20, 2024 Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) reported the successful completion of CUPID[1], a first-in-human Phase I dose escalation study of TLX592 in patients with advanced prostate cancer (Press release, Telix Pharmaceuticals, MAY 20, 2024, View Source [SID1234643469]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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!

TLX592 (225Ac-PSMA-RADmAb) is Telix’s investigational "next generation" targeted alpha therapy (TAT) for the treatment of prostate cancer and is the first clinical program to utilise Telix’s proprietary RADmAb engineered antibody technology. The RADmAb approach accelerates blood clearance and reduces bone marrow residence time compared with standard monoclonal antibodies (mAbs), while retaining target selectivity, internalisation and retention. The RADmAb platform is currently under pre-clinical and clinical evaluation for multiple cancer targets.

The CUPID (64Cu PSMA Imaging and (Bio) Distribution) study is a 3+3 mass dose escalation study with four patient cohorts intended to evaluate the safety, tolerability, pharmacokinetics, biodistribution and radiation dosimetry of TLX592. The study utilises copper-64 (64Cu) which is detectable by Positron Emission Tomography (PET) as a surrogate for actinium-225 (225Ac), enabling a successful proof-of-targeting study as well as predictive dosimetry calculations for future studies with 225Ac. Preliminary results in 11 evaluable patients enrolled in the study demonstrated accelerated blood kinetics compared to the standard antibody TLX591, while demonstrating similar (favourable) on-target and off-target biodistribution and hepatic clearance. There were no serious adverse events observed in the study.

Based on these encouraging results, Telix expects to advance TLX592 into a therapeutic Phase I/II study with 225Ac in the second half of 2024, subject to regulatory approval. TLX592 further deepens Telix’s PSMA[2]-targeting prostate cancer therapy portfolio and supplements its lead investigational radio antibody-drug conjugate (rADC) TLX591 (177Lu rosopatamab tetraxetan), currently being investigated in the ProstACT GLOBAL Phase III study. The Company intends to publish and present non-clinical and clinical data supporting these results at several upcoming symposia.

Dr David N. Cade, Group Chief Medical Officer at Telix stated, "The CUPID study demonstrated clearly how theranostic approaches can be used to streamline novel radiopharmaceutical drug development. In this case, PET imaging was used to dose-find a targeting agent for future use with an alpha emitter, while establishing basic safety and utility parameters that will greatly inform ongoing development of this product candidate.

"There is a significant unmet need for novel targeting platforms that may be used with alpha emitting isotopes and avoid renal toxicity and other off-target effects, such as the exocrine gland uptake typical of PSMA small molecule agents. We are excited to progress TLX592 into therapeutic studies where our aim is to develop this agent for both early metastatic prostate cancer and late-stage patients who are no longer responding to lutetium therapy. We would like to thank all participants for their commitment to the CUPID study."

On May 20, 2024 Genentech, a member of the Roche Group reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for inavolisib, an investigational oral therapy, in combination with palbociclib (Ibrance) and fulvestrant, for the treatment of adult patients with PIK3CA-mutated, hormone receptor-positive, human epidermal growth factor receptor 2-negative, locally advanced or metastatic breast cancer, following recurrence on or within 12 months of completing adjuvant endocrine treatment (Press release, Genentech, MAY 20, 2024, View Source [SID1234643467]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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!

"We are pleased that the FDA granted Breakthrough Therapy Designation for inavolisib in recognition of the substantial clinical benefit observed with this regimen," said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. "This promising inavolisib-based regimen could transform the PI3K inhibitor class, potentially becoming the standard of care for this patient population in the first-line setting."

Breakthrough Therapy Designation is designed to accelerate the development and regulatory review of medicines intended to treat serious or life-threatening conditions where preliminary clinical evidence has indicated they may demonstrate substantial improvement over existing therapies.

The FDA’s decision is based on positive Phase III INAVO120 results, which showed the inavolisib-based regimen reduced the risk of disease worsening or death (progression-free survival) by 57% compared to palbociclib and fulvestrant alone (15.0 months vs. 7.3 months; hazard ratio [HR]=0.43, 95% CI: 0.32-0.59, p<0.0001). Overall survival (OS) data were immature at this time, but a clear positive trend has been observed (stratified HR=0.64, 95% CI: 0.43-0.97, p=0.0338 (boundary of 0.0098). Follow-up for OS will continue to the next analysis. These data reinforce the potential for this inavolisib-based regimen to benefit patients with PIK3CA-mutated locally advanced or metastatic breast cancer.

PIK3CA is one of the most commonly mutated genes in advanced or metastatic breast cancer. Despite the prevalence of PIK3CA mutations, many patients are not tested until later in their treatment journey. Early testing for PIK3CA prior to initiating first-line treatment helps clinicians make a personalized treatment decision.

Data from INAVO120 are also being submitted to other global health authorities, including the European Medicines Agency.

Inavolisib is currently being investigated in three company-sponsored Phase III clinical studies (INAVO120, INAVO121, INAVO122) in PIK3CA-mutated locally advanced or metastatic breast cancer in various combinations. We continue to evaluate potential clinical development program expansion opportunities to address patient unmet needs in various tumor types across oncology.

About inavolisib

Inavolisib is an investigational, oral targeted treatment with best-in-class potential that could provide well-tolerated, durable disease control and potentially improved outcomes for people with PIK3CA-mutated, HR-positive, HER2–negative, locally advanced or metastatic breast cancer, who often have a poor prognosis and are in urgent need of new treatment options. Inavolisib has been designed to help minimize the overall burden and toxicity of treatment and is differentiated from other PI3K inhibitors due to its high potency and specificity for the PI3K alpha isoform versus other isoforms, and unique mechanism of action that facilitates the degradation of mutated PI3K alpha.

About the INAVO120 study

The INAVO120 study [NCT04191499] is a Phase III, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of inavolisib in combination with palbociclib and fulvestrant versus placebo plus palbociclib and fulvestrant in people with PIK3CA-mutated, hormone receptor (HR)-positive, HER2-negative, locally advanced or metastatic breast cancer whose disease progressed during treatment or within 12 months of completing adjuvant endocrine therapy and who have not received prior systemic therapy for metastatic disease.

The study included 325 patients, who were randomly assigned to either the investigational or control treatment arm. The primary endpoint is progression-free survival, as assessed by investigators, defined as the time from randomization in the clinical trial to the time when the disease progresses, or a patient dies from any cause. Secondary endpoints include overall survival, objective response rate, and clinical benefit rate.

Beyond INAVO120, inavolisib is currently being investigated in two additional company-sponsored Phase III clinical studies in PIK3CA-mutated locally advanced or metastatic breast cancer in various combinations:

in combination with fulvestrant versus alpelisib plus fulvestrant in HR-positive/HER2-negative breast cancer post cyclin-dependent kinase 4/6 inhibitor and endocrine combination therapy (INAVO121; NCT05646862), and
in combination with dual HER2 blockade vs dual HER2 blockade and optional physician’s choice of endocrine therapy as a maintenance treatment in HER2-positive disease (INAVO122; NCT05894239).

About Hormone Receptor-Positive Breast Cancer

HR-positive breast cancer is the most prevalent type of all breast cancers, accounting for approximately 70% of cases. A defining feature of HR-positive breast cancer is that its tumor cells have receptors that attach to one or both hormones – estrogen or progesterone – which can contribute to tumor growth. People diagnosed with HR-positive metastatic breast cancer often face the risk of disease progression and treatment side effects, creating a need for additional treatment options. The PI3K signaling pathway is commonly dysregulated in HR-positive breast cancer, often due to activating PIK3CA mutations, which have been identified as a potential mechanism of intrinsic resistance to standard of care endocrine therapy in combination with cyclin-dependent kinase 4/6 inhibitors.

About Genentech in Breast Cancer

Genentech has been advancing breast cancer research for more than 30 years with the goal of helping as many people with the disease as possible. Our medicines, along with companion diagnostic tests, have substantially improved outcomes for HER2-positive breast cancer. As our understanding of breast cancer biology rapidly improves, we are working to identify new biomarkers and approaches to treatment for other subtypes of the disease, including estrogen receptor-positive breast cancer, which is a form of hormone receptor-positive breast cancer, the most prevalent type of all breast cancers.

On May 20, 2024 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, reported it will be hosting a webcasted R&D breakfast panel featuring prominent scientific and medical thought leaders to discuss topline overall survival data from its phase 2 clinical trial of CAN-2409, its multimodal biological immunotherapy candidate, in Non-Small Cell Lung Cancer (NSCLC) (Press release, Candel Therapeutics, MAY 20, 2024, View Source [SID1234643466]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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 event will be held on Monday, June 3, 2024, at 7:00 AM Central Time, during the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago.

Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel, will be hosting the event and moderating the guest panel, which includes:

Charu Aggarwal, MD, MPH, FASCO
Leslie M. Heisler Associate Professor for Lung Cancer Excellence, Perelman School of Medicine, University of Pennsylvania
Roy S. Herbst, MD, PhD
Chief of Medical Oncology
Yale School of Medicine
Candel Research Advisory Board
Daniel H. Sterman, MD, FCCP, ATSF, DAABIP
Professor and Director, Pulmonary, Critical Care and Sleep Medicine
NYU Langone Health
A live webcast will be available by selecting Events and Presentations, under the News & Events tab, in the Investors section on Candeltx.com. A replay of the webcast will be archived for up to 90 days following the session date.

EDITOR’S NOTE: Media representatives interested in attending the event should please contact Kyle Evans at [email protected].