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]).

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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

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.

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"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 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that management will present a company overview at the 40th Annual J.P. Morgan Virtual Healthcare Conference on Monday, Jan. 10, at 7:30 a.m. EST (Press release, PTC Therapeutics, JAN 3, 2022, View Source [SID1234597953]).

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The presentation will be webcast live on the Events and Presentations page on the Investor section of PTC Therapeutics’ website at View Source and will be archived for 30 days following the presentation. It is recommended that users connect to PTC’s website several minutes prior to the start of the webcast to ensure a timely connection.

On January 3, 2022 Herantis Pharma Plc ("Herantis"), focusing on disease modifying therapies for debilitating neurodegenerative diseases, reported that Dr. Craig Cook, Chief Executive Officer, will hold 1×1 meetings and company presentations at the following investor conferences (Press release, Herantis Pharma, JAN 3, 2022, View Source,c3480839 [SID1234597949]):

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H.C. Wainwright BioConnect Conference, US – January 10th – 13th, 2022 (Virtual)

The presentation will be held on January 10th at 14:00 – 14:30 EET / 13:00 – 13:30 CET. 1×1 meetings will be scheduled during January 10-13th.

Biotech Showcase, US – January 10-12 and 17-19, 2022 (Virtual)

The presentation will be held on January 11th at 02:00 EET – 02:30 EET / 01:00 CET – 1:30 CET. 1×1 meetings will be scheduled during January 17-19th.

All presentations will be available via a digital library, which is accessible to event participants only. Please contact the conference organizers, or send an email to [email protected], if you wish to schedule a meeting with Herantis.

On January 3, 2022 Emergent BioSolutions Inc. (NYSE: EBS) reported that members of the company’s executive management team will participate in the following investor conferences in the first quarter of 2022 (Press release, Emergent BioSolutions, JAN 3, 2022, View Source [SID1234597948]):

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40th Annual J.P. Morgan Healthcare Conference
January 10 to 13, 2022
Company presentation scheduled on January 10 at 8:15 am PST
Click for Webcast
J.P. Morgan 2022 Global High Yield & Leveraged Finance Conference
February 28 to March 2, 2022
Company presentation scheduled on February 28 at 2:00 pm EST
Cowen & Co. 42nd Annual Health Care Conference
March 7 to 9, 2022
Presentation date and time will be updated on the Emergent website as the information becomes available
For conferences where a presentation is planned, the company’s webcast presentation may include a discussion of the company’s recent business developments as well as its financial results and guidance. The webcasts will be available both live, if possible, and by replay, and will be accessible from the Emergent website.