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PharmaCyte Biotech Successfully Completes 36 Month Master Cell Bank Stability Study

On December 28, 2021 PharmaCyte Biotech, Inc. (NASDAQ: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that it has successfully completed a 36-month stability study of the cells from its Master Cell Bank (MCB) (Press release, PharmaCyte Biotech, DEC 28, 2021, View Source [SID1234597810]). These cells will be encapsulated and then used to treat locally advanced, inoperable pancreatic cancer (LAPC). This stability study is one of the items that the U.S. Food and Drug Administration (FDA) requires PharmaCyte to complete for its clinical trial product, CypCaps, in an effort to lift the FDA’s clinical hold. This means that the cells used to produce the CypCaps have a shelf life of at least 36 months when stored in a vapor phase of liquid nitrogen. Vapor phase temperature for liquid nitrogen is between -140°C and -180°C.

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PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said of the completed 36-month stability study, "The ongoing study to determine the maximum shelf life of the MCB cells has reached another important milestone. Cells from our MCB successfully completed the required tests to prove that the cells are stable and remain active after being stored frozen for 36 months in a vapor phase of liquid nitrogen.

"Analysis of the cells from our MCB after 36 months showed that the cells passed all of the specified tests, including cell viability, identity testing, sterility, enzyme activity and cell potency as well as pH, label check and appearance of the cells.

"This study will continue in order to determine the maximum shelf life of the cells from our MCB. It is distinct from the other ongoing stability study on the shelf life of our CypCaps that continue to be stored at approximately -80°C. While the storage temperatures are different for the MCB cells and the CypCap cells, the tests for both stability studies are the same."

This ongoing stability study was initiated prior to the submission of PharmaCyte’s Investigational New Drug Application (IND) to the FDA. The information and data obtained from this stability study and other studies will form part of PharmaCyte’s Complete IND Submission of information that PharmaCyte will provide to the FDA to have the clinical hold lifted.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, we encourage you to watch the Company’s documentary video complete with medical animations at: View Source

CStone announced new drug approval of precision therapy AYVAKIT® (avapritinib) in Hong Kong, China for the treatment of PDGFRA D842V mutant gastrointestinal stromal tumors (GIST)

On December 28, 2021 CStone Pharmaceuticals ("CStone", HKEX: 2616), a leading biopharmaceutical company focused on research, development, and commercialization of innovative immuno-oncology therapies and precision medicines, reported that the new drug application (NDA) of first-in-class precision therapy AYVAKIT (avapritinib) has been approved in Hong Kong, China for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA D842V mutation (Press release, CStone Pharmaceauticals, DEC 28, 2021, View Source [SID1234597807]). The drug is the first precision therapy approved in Hong Kong, China for the treatment of patients with PDGFRA D842V mutant GIST.

Discovered by CStone’s partner Blueprint Medicines, AYVAKIT is a potent, selective and orally available inhibitor of KIT and PDGFRA mutant kinases. CStone has an exclusive collaboration and license agreement with Blueprint Medicines for the development and commercialization of AYVAKIT and certain other drug candidates in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for AYVAKIT in the rest of the world.

Dr. Frank Jiang, Chairman and CEO of CStone, said, "AYVAKIT is CStone’s first product approval in Hong Kong, China. Earlier this year, AYVAKIT was also approved in Mainland China and Taiwan. We are very glad to provide this innovative treatment to more GIST patients whose tumors harbor the PDGFRA D842V mutation. CStone is committed to bringing forward effective, innovative therapies to patients around the world. In the future, we will strive to accelerate the development of novel therapies to fulfill the unmet medical needs of more cancer patients."

The Hong Kong Department of Health (DOH) has approved AYVAKIT based on data from the NAVIGATOR study, an open-label, dose-escalation/dose-expansion phase I study designed to evaluate the safety and efficacy of AYVAKIT in patients with unresectable or metastatic GIST. In December 2020, the European Journal of Cancer (EJC) published updated data from the NAVIGATOR study enrolling PDGFRA D842V mutant GIST patients. In 38 patients with PDGFRA D842V mutant GIST who received a starting dose of 300 mg or 400 mg once daily, the overall response rate (ORR) was 95% (36/38 patients). In 28 of these patients who had a starting dose of 300 mg once daily, the ORR was 96% (27/28 patients). The disease control rate (DCR) of all dose groups was 100%. The median duration of response (DOR) of all dose groups was 27.6 months. The most common treatment-emergent adverse events were anemia, increased blood bilirubin, decreased white blood cell count, increased blood creatine phosphokinase, increased aspartate aminotransferase, face edema, eyelid edema, decreased neutrophil count and hair color changes. The data showed that AYVAKIT demonstrated robust, durable, and deep clinical activity, with a generally well-tolerated safety profile.

About Gastrointestinal Stromal Tumor (GIST)

GIST is a sarcoma, or tumor of bone or connective tissue, of the GI tract. Tumors arise from cells in the wall of the GI tract and occur most often in the stomach or small intestine. Most patients are diagnosed between the ages of 50 to 80, and diagnosis is typically triggered by GI bleeding, incidental findings during surgery or imaging, and in rare cases, tumor rupture or GI obstruction. About 5 to 6 percent of primary GIST cases are caused by a PDGFRA D842V mutation, the most common PDGFRA exon 18 mutation.

About AYVAKIT (avapritinib)

AYVAKIT (avapritinib) is a kinase inhibitor approved by the Department of Health (DOH), Hong Kong, China and Taiwan Food and Drug Administration (TFDA) under the brand name AYVAKIT for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA D842V mutation. The National Medical Products Administration (NMPA) of China has approved AYVAKIT for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA exon 18 mutation, including PDGFRA D842V mutations.

The U.S. Food and Drug Administration (FDA) has approved AYVAKIT for the treatment of two indications: adults with advanced systemic mastocytosis (Advanced SM), including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), and adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. This medicine is approved by the European Commission under the brand name AYVAKYT for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. AYVAKIT/AYVAKYT is not approved for the treatment of any other indication in the U.S., Europe or Greater China, or for any indication in any other jurisdiction by any other health authority.

Blueprint Medicines is developing AYVAKIT globally for the treatment of advanced and non-advanced SM. The FDA granted breakthrough therapy designation to AYVAKIT for the treatment of advanced SM, including the subtypes of ASM, SM-AHN, and MCL, and for the treatment of moderate to severe indolent SM.

These Were the Top 5 Venture Capital Deals in Houston in 2021

On December 27, 2021 Tvardi Therapeutics Inc. reported that closed its $74 million series B funding round led by new investors New York-based Slate Path Capital, Florida-based Palkon Capital, Denver-based ArrowMark Partners, and New York-based 683 Capital, with continued support and participation by existing investors, including Houston-based Sporos Bioventures (Press release, Tvardi Therapeutics, DEC 27, 2021, View Source [SID1234597809]).

"We are thrilled to move out of stealth mode and partner with this lineup of long-term institutional investors," says Imran Alibhai, CEO at Tvardi. "With this financing we are positioned to advance the clinical development of our small molecule inhibitors of STAT3 into mid-stage trials as well as grow our team."

VLP Therapeutics raises $21M Series A-1 for cancer treatment vaccine, infectious diseases vaccine R&D

On December 27, 2021 VLP Therapeutics, Inc. (VLPT) reported that it has signed an agreement for an investment of US$21 million in a Series A-1 round from six investors, consisting of two new investors: Nobelpharma Co., Ltd. and MUFG Bank, Ltd., and four existing investors: Sojitz Corporation, MIYAKO Capital Co., Ltd., Mr. Robert G. Hisaoka and SK Impact Fund, LLC (Press release, VLP Therapeutics, DEC 27, 2021, View Source [SID1234597776]).

With this funding, VLPT aims to further accelerate the research and development of a cancer treatment vaccine as well as prophylactic vaccines against malaria, dengue, etc. and move into clinical trials at the earliest date possible. This is an additional investment following US$16 million raised in a Series A round in March 2021.

With this funding VLPT aims to accelerate the R&D of a cancer treatment vaccine as well as malaria and dengue vaccines.

"I have long committed to the research and development of vaccines against cancer and infectious diseases so the people across the globe can lead normal lives," says Wataru Akahata, CEO and co-founder of VLPT. "We were fortunate enough to be able to raise funding in March to facilitate our cancer treatment vaccine R&D. This additional funding will now allow us to further accelerate our other R&D efforts in infectious diseases area as well. This means a lot as it helps us to push our scientific endeavors forward at much faster pace, enabling us to get one step closer in making a greater social impact."

I-Mab Announces IND Approval from China NMPA for Phase 2 Clinical Trial of Enoblituzumab in Combination with Pembrolizumab in Solid Tumors

On December 27, 2021 I-Mab (the "Company") (Nasdaq: IMAB), a clinical-stage biopharmaceutical company committed to the discovery, development, and commercialization of novel biologics, reported that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has approved I-Mab’s IND submission for the initiation of a phase 2 trial in China for enoblituzumab (also known as TJ271) in combination with pembrolizumab (Keytruda) in patients with solid tumors, including non-small cell lung cancer (NSCLC), urothelial carcinoma (UC), and other selected cancers (Press release, I-Mab Biopharma, DEC 27, 2021, View Source [SID1234597775]). I-Mab has acquired exclusive rights to develop and commercialize enoblituzumab in Greater China from MacroGenics (Nasdaq: MGNX).

Enoblituzumab is a highly differentiated humanized monoclonal antibody directed against the immune regulator B7-H3, which plays a key role in regulating immune response against cancers and is widely expressed in multiple cancers. The presence of B7-H3 in tumors is associated with the poor efficacy of neoadjuvant therapies. Enoblituzumab enhances the antibody-dependent killing of cancer cells and has demonstrated strong anti-tumor activity in preclinical studies. Additional preclinical data generated by I-Mab and preliminary clinical evidence from MacroGenics support increased efficacy for the combination of enoblituzumab and a PD-1 antibody against cancers.

The phase 2 clinical trial in China will evaluate the efficacy of the combination of enoblituzumab and pembrolizumab. The trial is designed as a "basket" clinical trial in patients with NSCLC, UC, and other selected cancer types based on previous studies conducted by MacroGenics. These previous studies have indicated that combination therapy resulted in anti-tumor activity in recurrent or metastatic NSCLC and squamous cell carcinoma of the head and neck (SCCHN).

"The initiation of the phase 2 clinical trial will accelerate the clinical development of enoblituzumab in China," said Dr. Andrew Zhu, President of I-Mab. "Enoblituzumab has become a key player against various advanced cancers and one of the Company’s core clinical assets. We are excited about the initiation of this clinical study and expect to bring this valuable compound to cancer patients with critical unmet medical needs."

Currently, MacroGenics is conducting a phase 2 study of enoblituzumab in combination with retifanlimab (PD-1 antibody) or tebotelimab (PD-1 & LAG-3 bispecific DART molecule) for first-line treatment of patients with recurrent or metastatic SCCHN.

About Enoblituzumab

Enoblituzumab is an investigational Fc-optimized monoclonal antibody that targets B7-H3, a member of the B7 family of immune regulator proteins. B7-H3 is widely expressed by many different tumor types and may play a key role in regulating the immune response to various types of cancer. Enoblituzumab has been or is currently being evaluated in clinical trials as a monotherapy or in combination with anti-PD-1-based therapies in patients with B7-H3-expressing cancers. I-Mab acquired the development and commercial rights from MacroGenics for Greater China.