On January 3, 2022 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) for the Company’s lead drug candidate, REQORSA Immunogene Therapy, in combination with Merck & Co’s Keytruda in patients with histologically-confirmed unresectable stage III or IV non-small cell lung cancer (NSCLC) whose disease progressed after treatment with Keytruda (Press release, Genprex, JAN 3, 2022, View Source [SID1234597954]). In the first quarter of 2022, Genprex expects to initiate its Acclaim-2 clinical trial, which is an open-label, multi-center Phase 1/2 clinical trial evaluating REQORSA in combination with Keytruda, for this patient population. The Company previously received its first FTD for REQORSA in combination with AstraZeneca PLC’s Tagrisso in patients with histologically confirmed unresectable stage III or IV NSCLC, with EGFR mutations that progressed after treatment with Tagrisso.

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 thrilled to receive a second Fast Track Designation from the FDA for REQORSA in patients with late-stage NSCLC, this time in combination with the checkpoint inhibitor Keytruda," said Rodney Varner, President and Chief Executive Officer at Genprex. "This Fast Track Designation is an important step in our efforts to accelerate clinical development of REQORSA and another validation of the potential of REQORSA to treat the unmet medical need of patients with late-stage NSCLC. With a strong balance sheet of $42 million in cash as of the end of the third quarter of 2021 and expert clinical trial management led by Chief Medical Officer and industry veteran Mark Berger, MD who joined Genprex in September 2021, we are well positioned to advance our Acclaim-1 and Acclaim-2 clinical trials in a meaningful way in 2022."

Dr. Berger added, "Fast Track Designation provides a company opportunities to have more frequent engagement with the FDA to discuss the drug candidate’s development plan and also provides potential eligibility for priority review, which has a six-month review timeline. FDA may award Fast Track Designation if it determines that non-clinical or clinical data demonstrate the potential for a drug to address unmet medical need for a serious or life-threatening disease or condition. This provision is intended to facilitate development and expedite review of such drugs so that a product, if approved, can reach the market expeditiously. Advanced NSCLC represents a large patient population that is in desperate need of new therapies."

Previously presented preclinical data have shown synergy between REQORSA and Keytruda. Those data showed that REQORSA combined with Keytruda was more effective than Keytruda alone in increasing the survival of mice with a humanized immune system that had metastatic lung cancers. Those studies in mice with a humanized immune system also documented multiple effects of REQORSA on the immune system, such as an increase in Natural Killer cells and a decrease in PD-L1 expression on tumor cells, that is believed likely to contribute to the synergy seen with Keytruda.

Fast Track Designation recipients may also be eligible for accelerated approval or rolling review of the recipient’s Biologics License Application (BLA) if other qualifying criteria are met. In addition, Fast Track product candidates could be eligible for priority review if supported by clinical data at the time of BLA submission.

The initial intended disease indication for development of REQORSA is NSCLC. According to the World Health Organization, in 2020 lung cancer was the leading cause of cancer deaths worldwide, causing more deaths than colorectal, breast, liver or stomach cancers. There were more than 2 million new lung cancer cases and 1.8 million deaths from lung cancer worldwide. In the United States, according to the American Cancer Society, it is estimated that in 2021, there will be more than 235,000 new cases of lung cancer and more than 131,000 deaths from lung cancer. The American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) reports that NSCLC represents 84 percent of all lung cancers and the five-year survival rate for patients with NSCLC with distant spread is 7 percent.

For more information on the U.S. FDA’s Fast Track Designation, please visit the FDA’s Fast Track webpage.

On January 3, 2021 argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, reported that Tim Van Hauwermeiren, Chief Executive Officer, will present at the 40th Annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022 at 7:30 a.m. ET (Press release, argenx, JAN 3, 2022, View Source [SID1234597971]).

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!

A live webcast of the presentation may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website for approximately 30 days following the presentation.

On January 3, 2022 Everest Medicines Limited (HKEX 1952.HK, "Everest", or the "Company"), a biopharmaceutical company focused on developing and commercializing transformative pharmaceutical products in Asia, reported that Taiwan Food and Drug Administration (TFDA) has accepted the submission of a New Drug Application (NDA) for sacituzumab govitecan (SG) in adult patients with unresectable locally advanced or metastatic TNBC who have received two or more prior systemic therapies, at least one of them for metastatic disease (Press release, Everest Medicines, JAN 3, 2022, View Source [SID1234597989]). The treatment was granted Pediatric and Rare Severe Disease Priority Review Designation by the Taiwan FDA in July 2021.

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!

"This regulatory milestone is another important step in the ongoing global development of sacituzumab govitecan as a novel treatment option for patients with mTNBC. We will be working with the regulatory authorities in Taiwan to help bring the treatment to patients with metastatic TNBC who have limited alternative options." said Yang Shi, Chief Medical Officer for Oncology at Everest Medicines.

Under the trade name Trodelvy, the U.S. FDA previously granted accelerated approval to SG in April 2020 and then expanded its indication with full approval in April 2021 for adult patients with unresectable locally advanced or mTNBC who have received two or more prior systemic therapies, at least one of them for metastatic disease. In May 2021, Everest announced that the China National Medical Products Administration accepted its Biologics License Application with priority review for SG for adult patients with unresectable locally advanced or metastatic TNBC who have received two or more prior systemic therapies, at least one of them for metastatic disease.

In November 2021, Everest announced topline results for its Phase 2b EVER-132-001 study of SG, which met its primary endpoint with a 38.8% overall response rate (ORR). This study included 80 people in China, and the results were consistent with those from the global Phase 3 ASCENT study, thus showing similar efficacy in the Chinese population.

About Triple-Negative Breast Cancer (TNBC)

TNBC is the most aggressive type of breast cancer and accounts for approximately 15% of all breast cancers. The median age of breast cancer diagnoses tends to be younger in Asian than western countries, and the percentage of the TNBC molecular subtype has been increasing in the past 10 years. TNBC cells do not have estrogen and progesterone receptors and have limited human epidermal growth factor receptor 2 (HER2). Due to the nature of TNBC, effective treatment options are extremely limited compared with other breast cancer types. TNBC has a higher chance of recurrence and metastases than other breast cancer types. The average time to metastatic recurrence for TNBC is approximately 2.6 years compared with 5 years for other breast cancers, and the relative five-year survival rate is much lower. Among women with metastatic TNBC, the five-year survival rate is 12%, compared with 28% for those with other types of metastatic breast cancer.

About Sacituzumab Govitecan

Sacituzumab govitecan (SG) is a first-in-class antibody and topoisomerase inhibitor conjugate directed to the Trop-2 receptor, a protein overexpressed in multiple types of epithelial tumors, including metastatic TNBC and metastatic urothelial cancer (UC), where high expression is associated with poor survival and relapse. SG is approved for adults with metastatic TNBC in the United States, the European Union, Australia, Canada, Great Britain and Switzerland under the trade name Trodelvy and approval is based on data submitted from the Phase 3 ASCENT study. Review is also underway in Singapore and China through Everest Medicines. Trodelvy is also approved for use in metastatic UC in the United States and continues to be developed for potential use in other TNBC and metastatic UC populations. It is also being developed as an investigational treatment for hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) metastatic breast cancer and metastatic non-small cell lung cancer. Additional evaluation across multiple solid tumors is also underway.

Under a licensing agreement with Gilead Sciences, Inc., Everest Medicines has exclusive rights to develop, register, and commercialize sacituzumab govitecan for all cancer indications in Greater China, South Korea, and certain Southeast Asian countries. In October 2020, sacituzumab govitecan was included in the updated 2020 China Guidelines for the Standardized Diagnosis and Treatment of Advanced Breast Cancer, compiled by the Breast Cancer Expert Committee of the National Cancer Control Center, the Breast Cancer Professional Committee of the Chinese Anti-Cancer Association, and the Cancer Drug Clinical Research Professional Committee of the Chinese Anti-Cancer Association.

Followed by the Fast Track Designation and Orphan Drug Designation (ODD) to SG for the treatment of metastatic TNBC granted by the Ministry of Food and Drug Safety (MFDS) in South Korea, the NDA has been accepted in second-line mTNBC in December 2021. . In addition, Everest announced in January 2021 that it submitted a New Drug Application (NDA) to the Health Sciences Authority (HSA) of Singapore for SG for the treatment of patients with metastatic TNBC who have received at least two prior therapies for metastatic disease. That application is currently under review.

On January 3, 2022 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported their participation in upcoming investor conferences and invites investors to participate in one-on-one meetings (Press release, ERYtech Pharma, JAN 3, 2022, View Source [SID1234598106]):

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!

LifeSci Partners – 11thAnnual Corporate Access Event

Wednesday, January 5th – Friday, January 7thRegistration Link
To submit one-on-one meeting requests Click Here

H.C. Wainwright BioConnect Virtual Conference

Monday, January 10th – Thursday, January 13thRegistration Link

A webcast will be available for 90 days on the Webcasts section of the ERYTECH website on January 10th at 7am ET or by using the following link: View Source

If you are interested in arranging a one-on-one meeting, please contact your conference representative.

On January 3, 2021 Immix Biopharma, Inc. (Nasdaq: IMMX) ("ImmixBio" or the "Company"), a biopharmaceutical company pioneering Tissue-Specific Therapeutics (TSTx)TM targeting oncology and immuno-dysregulated diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for IMX-110 for the treatment of a life-threatening form of pediatric cancer in children, rhabdomyosarcoma. IMX-110, an investigational product, is currently being evaluated in a Phase 1b/2a clinical trial (Press release, Immix Biopharma, JAN 3, 2022, View Source [SID1234597956]).

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 FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children aged 18 years or younger and impact fewer than 200,000 people in the United States.

If a New Drug Application in the United States for IMX-110 is approved, ImmixBio may be eligible to receive a Priority Review Voucher (PRV) from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application, or may be sold or transferred.

"We are pleased by FDA’s acknowledgment of the urgent need for a safe and effective treatment for children with this devastating disease," stated ImmixBio’s Chief Executive Officer Ilya Rachman, M.D., PhD. "We are encouraged by our Phase 1b/2a clinical data in soft tissue sarcoma. IMX-110 is a tissue-specific therapeutic that simultaneously attacks all 3 components of the tumor micro-environment, severing the critical lifelines between the tumor and its metabolic and structural support. We believe our SMARxT platform generating Tissue-Specific Therapeutics represents a distinct alternative to the traditional ‘single target, single mutation’ development model."

Rhabdomyosarcoma ("RMS") is a high-grade, malignant neoplasm, the most common soft tissue sarcoma in pediatric and adolescent populations and which rarely occurs in adults. The prevalence of RMS in the United States is approximately 20,000 children of all ages. The five-year survival rate ranges from 20% to 30% for children in the high-risk group where cancer spreads widely in the body.

IMX-110 is the first clinical-stage product of ImmixBio’s SMARxT Tissue-Specific Platform, which produces Tissue-Specific Therapeutics that accumulate at intended therapeutic sites at 3 to 5 times the rate of conventional medicines. The FDA has already granted orphan drug designation (ODD) to IMX-110 for the treatment of soft tissue sarcoma.

ImmixBio recently shared clinical data across multiple soft tissue sarcoma subtypes in several heavily pretreated patients demonstrating median progression-free survival (PFS) of 4 months with zero drug-related severe adverse events and zero dose interruptions due to toxicity. The data can be viewed in the Immix Biopharma Corporate Presentation at View Source