On August 25, 2021 Servier Pharmaceuticals, a growing leader in oncology committed to bringing the promise of tomorrow to the patients we serve, reported that the U.S. Food and Drug Administration (FDA) approved TIBSOVO (ivosidenib tablets) for the treatment of adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation as detected by an FDA-approved test (Press release, Servier, AUG 25, 2021, View Source [SID1234586893]). TIBSOVO is the first and only targeted therapy approved for patients with previously treated IDH1-mutated cholangiocarcinoma.

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 supplemental New Drug Application (sNDA) for TIBSOVO received Priority Review, which accelerated the review timeline and is typically given to drugs that may offer major advances in treatment or may provide a treatment where no adequate therapy exists.

"Servier has been focused on exploring the significant potential of inhibiting mutant IDH enzymes as a novel approach to treating cancers with high unmet needs, including cholangiocarcinoma," said David K. Lee, CEO, Servier Pharmaceuticals. "We are proud to bring to patients the first and only targeted therapy for previously treated IDH1-mutated cholangiocarcinoma. We are grateful to the patients, caregivers, investigators and study teams who made this achievement possible through their participation in the ClarIDHy clinical trial."

The FDA approval of this indication is supported by data from the ClarIDHy study, the first and only randomized Phase 3 trial for previously treated IDH1-mutated cholangiocarcinoma. Results from the ClarIDHy study demonstrated a statistically significant improvement in the primary endpoint of progression-free survival (PFS) by an independent review committee (hazard ratio [HR] 0.37; 95% CI [0.25, 0.54], p<0.001).1 The median PFS (95% CI) for TIBSOVO and placebo was 2.7 (1.6, 4.2) and 1.4 (1.4, 1.6) months, respectively. Thirty-two percent and 22% of patients randomized to TIBSOVO remained free of progression or death at 6 and 12 months, respectively, versus none on the placebo arm.

The study protocol specified that patients randomized to placebo could cross over to TIBSOVO at the time of disease progression, and a high proportion of patients in the placebo arm (70.5%) crossed over to TIBSOVO. The study also showed the key secondary endpoint of overall survival (OS) favoring patients randomized to TIBSOVO compared to those randomized to placebo; however, statistical significance was not reached.1 OS results are based on the final analysis of OS (based on 150 events which occurred 16 months after the final analysis of PFS. The median OS (95% CI) for TIBSOVO was 10.3 (7.8, 12.4) months; and placebo was 7.5 (4.8, 11.1) months without adjusting for crossover.

The safety profile observed in the study was consistent with previously published data.1 The most common adverse reactions (≥15%) in patients with cholangiocarcinoma were fatigue, nausea, abdominal pain, diarrhea, cough, decreased appetite, ascites, vomiting, anemia, and rash.

The recommended TIBSOVO dosage for previously treated IDH1-mutated cholangiocarcinoma is 500 mg orally once daily with or without food until disease progression or unacceptable toxicity.

"Patients living with IDH1-mutated cholangiocarcinoma, especially those whose disease progresses following chemotherapy, are in urgent need of new treatment options," said Rachna T. Shroff, MD, Associate Professor of Medicine, University of Arizona, and Chief of GI Medical Oncology at the University of Arizona Cancer Center. "In addition to an acceptable safety profile, TIBSOVO demonstrated an impressive, significant benefit in progression-free survival, underscoring its importance as a new option for patients battling this aggressive cancer."

Cholangiocarcinoma is a rare, aggressive cancer of the bile ducts within and outside of the liver. An estimated 8,000 people in the United States are diagnosed with cholangiocarcinoma each year. However, the actual number of these cases is likely to be higher, as cholangiocarcinoma can be hard to diagnose, and may be misclassified as other types of cancer.2

"Before today’s approval of TIBSOVO, there were no approved targeted therapies available to cholangiocarcinoma patients harboring the IDH1 mutation, and limited chemotherapy options available to patients with advanced disease," said Stacie Lindsey, Founder and CEO, Cholangiocarcinoma Foundation. "This approval brings new hope to the cholangiocarcinoma community and we are excited that this much-needed new therapeutic option is being made available to patients."

Servier Pharmaceuticals is introducing ServierONE Patient Support Services, a program that offers one-on-one support to help patients who are prescribed TIBSOVO or other Servier products navigate their cancer journey. Eligible patients will have access to financial assistance, emotional support and other resources. More information can be found at www.servierone.com.

TIBSOVO* is also approved in the U.S. as monotherapy for the treatment of adults with IDH1-mutated relapsed or refractory acute myeloid leukemia (AML) and for adults with newly diagnosed IDH1-mutated AML who are ≥75 years old or who have comorbidities that preclude the use of intensive induction chemotherapy.

About Cholangiocarcinoma

Cholangiocarcinoma is a rare, aggressive cancer of the bile ducts within and outside of the liver. IDH1 mutations occur in up to 20% of cholangiocarcinoma cases in the U.S. and are not associated with prognosis.3 Prior to the approval of TIBSOVO, there were no approved targeted therapies for IDH1-mutated cholangiocarcinoma and limited chemotherapy options are available in the advanced setting. Gemcitabine-based chemotherapy is often recommended for newly diagnosed advanced or metastatic disease.

On August 25, 2021 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported two oral presentations of clinical data will be presented at the upcoming European Society for Medical Oncology Congress (ESMO) (Free ESMO Whitepaper) 2021, reinforcing the clinical benefit of adagrasib, a KRASG12C inhibitor, and sitravatinib, a receptor tyrosine kinase inhibitor (Press release, Mirati, AUG 25, 2021, View Source [SID1234586892]). The congress will take place virtually from September 16 to 21, 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!

"Mirati continues to make important progress in demonstrating the clinical impact our investigational treatments may have on patient outcomes in lung and colorectal cancers," said Charles M. Baum, M.D., Ph.D., president and chief executive officer, Mirati Therapeutics, Inc. "The data we are presenting at ESMO (Free ESMO Whitepaper) will show how adagrasib alone and in combination with cetuximab offer encouraging clinical activity in patients with KRASG12C-mutated colorectal cancer. In addition, updated survival data from our Phase 2 study evaluating sitravatinib with nivolumab supports the potential of this combination for patients with advanced lung cancer whose tumors are resistant to checkpoint inhibitors."

Dr. Baum added, "We are investigating several combination approaches in our expanding pipeline with the goal of improving standards of care and the course of treatment for people with cancer. We are thankful to the investigators and patients, without whom our research would not be possible."

Learn more about Mirati’s development of therapies that target the genetic and immunological drivers of cancers at Mirati.com/science.

Mirati studies at ESMO (Free ESMO Whitepaper) Congress 2021 include:
*All times noted are Central European Summer Time (CEST)

Presentation Title: KRYSTAL-1: Adagrasib (MRTX849) as Monotherapy or Combined with Cetuximab in Patients With Colorectal Cancer Harboring a KRASG12C Mutation
Author: Jared Weiss
Abstract Number: LBA6
Session: Presidential Symposium II
Presentation Date/Time: Sunday, September 19, 2021 at 15:47-16:02 CEST | Channel 1

Presentation Title: MRTX-500: Phase 2 Trial of Sitravatinib (Sitra) + Nivolumab (Nivo) in Patients (Pts) With Nonsquamous (NSQ) Non–Small-Cell Lung Cancer (NSCLC) Progressing on or After Prior Checkpoint Inhibitor (CPI) Therapy
Author: Ticiana A. Leal
Abstract Number: 1191O
Session: NSCLC Proffered Paper Session II
Presentation Date/Time: Monday, September 20, 2021 at 14:10-14:20 CEST | Channel 4

About Adagrasib (MRTX849)

Adagrasib is an investigational, highly selective, and potent oral small-molecule inhibitor of KRASG12C that is optimized to sustain target inhibition, an attribute that could be important to treat KRASG12C mutated cancers, as the KRASG12C protein regenerates every 24-48 hours. Studies of adagrasib have shown that the drug has a long half-life, extensive tissue distribution and is well tolerated. Adagrasib has also shown single-agent responses in non-small cell lung cancer (NSCLC), colorectal cancer, pancreatic cancer, and other solid tumors with KRASG12C mutations. Adagrasib is a being evaluated in several clinical trials in combination with other anti-cancer therapies with strong scientific rationale in patients with advanced solid tumors. Registration-enabling studies are ongoing in NSCLC and colorectal cancer. For more information visit Mirati.com/science.

About Sitravatinib

Sitravatinib is an investigational spectrum-selective kinase inhibitor that potently inhibits receptor tyrosine kinases (RTKs), including TAM family receptors (TYRO3, Axl, Mer), split family receptors (VEGFR2, KIT) and RET. Sitravatinib is being evaluated in combination with nivolumab (OPDIVO), an anti-PD-1 checkpoint inhibitor, in patients whose cancers have progressed despite treatment with a checkpoint inhibitor. Sitravatinib’s potent inhibition of TAM and split family RTKs may overcome resistance to checkpoint inhibitor therapy through targeted reversal of an immunosuppressive tumor microenvironment, enhancing antigen-specific T cell response and expanding dendritic cell-dependent antigen presentation. Sitravatinib is being evaluated in multiple clinical trials to treat patients who are resistant to prior immune checkpoint inhibitor therapy and progressed on platinum doublet therapy, including the ongoing potentially registration-enabling Phase 3 trial of sitravatinib in combinations with a checkpoint inhibitor in non-small cell lung cancer (NSCLC). In addition, sitravatinib in combinations with checkpoint inhibitors are being evaluated in selected checkpoint inhibitor naïve patients.

On August 25, 2021 NovaRock Biotherapeutics Limited, a clinical-stage biopharmaceutical company delivering innovative specialty pharmaceuticals to address patients’ unmet medical needs, reported that they have entered into an exclusive license agreement and strategic partnership with Flame Biosciences (Press release, NovaRock Biotherapeutics, AUG 25, 2021, View Source [SID1234586891]).

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 very pleased to be partnering with Flame and their world-class clinical team," said Dr. Han Li, CEO of NovaRock. "The collaboration with Flame marks a solid step towards getting NovaRock’s innovative portfolio into the global market. The proceeds from this collaboration will be primarily used to fund the clinical advancement of NBL-012 and NBL-015 as well as the advancement of our preclinical programs."

NBL-015 is a fully human anti-Claudin 18.2 monoclonal antibody optimized through protein engineering to achieve enhanced ADCC, CDC, and ADCP effects. Its Investigational New Drug Application has also been approved by the U.S. FDA in May 2021.

Under the terms of the Agreement, NovaRock has granted Flame Biosciences the exclusive rights to NBL-015 outside of Greater China (including mainland China, Hong Kong, Macau, and Taiwan). Flame shall be responsible for the development, regulatory approval, and commercialization of NBL-015. NovaRock will, at Flame Biosciences’s expense, collaborate with Flame on the discovery and preclinical development of two new bispecific antibodies based on NovaRock’s NovaTE bi-specific antibody technology platform and subsequently grant Flame Biosciences the exclusive rights to further develop, manufacture and commercialize the Licensed Products. The lead product candidates from this collaboration are expected to enter clinical development in late 2023.

NovaRock will receive an upfront payment of US$7.5 million and is eligible to receive development milestone payments of up to US$172.5 million subject to achievement of the development milestone events. NovaRock is also eligible to receive sales milestone payments of up to US$460 million subject to the achievement of the sales milestone events and royalties based on a certain percentage of the net sales of the Licensed Products in the Territory.

About NBL-015

NBL-015 is a fully human anti-Claudin 18.2 monoclonal antibody optimized through protein engineering to achieve enhanced ADCC, CDC, and ADCP effects. Preclinical studies have demonstrated that NBL-015 has significant advantages over similar drugs in terms of low immunogenicity, good safety, high affinity, and high anti-tumor activity, providing a promising prospect of becoming the best-in-class target therapy to treat pancreatic and gastric cancer. NBL-015 has been granted the orphan-drug designation for the treatment of pancreatic cancer and gastric cancer, including cancer of gastroesophageal junction by the U.S. Food and Drug Administration (FDA). Its Investigational New Drug Application (IND) has also been approved by the U.S. FDA in May 2021.

About NovaTE bi-specific antibody technology platform

NovaTE is a novel tumor antigen and CD-137 bispecific antibody technology platform that is designed to selectively activate the antigen-experienced T cells in the tumor microenvironment. Its proprietary scaffold can maximize the tumor cell engaging and T cell activation while minimizing the systematic adverse events. Antibodies developed using NovaTE have demonstrated superior efficacy and safety in preclinical studies. Moreover, the unique bi-specific antibody structure offers exceptional stability and manufacturability.

On August 25, 2021 RenovoRx, Inc., a biopharmaceutical company and innovator in targeted cancer therapy, reported the pricing of its underwritten initial public offering of 1,850,000 units at a public offering price per unit of $9.00 (Press release, Renovorx, AUG 25, 2021, View Source [SID1234586890]). Each unit consists of one share of common stock and one warrant to purchase one share of common stock. The warrants have an exercise price of $10.80 per share and are exercisable for a period of five years after the issuance date. All of the units are being offered by RenovoRx. In addition, RenovoRx has granted the underwriters a 45-day option to purchase up to an additional 277,500 shares of its common stock and/or warrants to purchase up to an additional 277,500 shares of its common stock, at the initial public offering price, less the underwriting discounts and commissions.

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!

In connection with the offering, the Company’s common stock has been approved for listing on The Nasdaq Capital Market. The shares are expected to begin trading on The Nasdaq Capital Market on August 26, 2021, under the ticker symbol "RNXT." The offering is expected to close on August 30, 2021, subject to customary closing conditions.

RenovoRx Announces Pricing of Initial Public Offering

The gross proceeds to RenovoRx from the offering, before deducting the underwriting discounts and commissions and offering expenses, are expected to be $16.65 million.

Roth Capital Partners is acting as the sole book-running manager for the offering. Maxim Group LLC is acting as lead manager for the offering.

The securities described above are being offered by the Company pursuant to a registration statement on Form S-1 (Registration No. 333-258071) that was previously filed with the U.S. Securities and Exchange (the "SEC") and declared effective on August 25, 2021. This offering is being made only by means of a prospectus. A preliminary prospectus relating to and describing the terms of the offering has been filed with the SEC. Copies of the preliminary prospectus can be accessed through the SEC’s website at www.sec.gov. Copies of the final prospectus relating to the initial public offering can be obtained, when available, through the SEC’s website at www.sec.gov or from: Roth Capital Partners, LLC, 888 San Clemente Drive, Newport Beach, CA 92660, Attention: Equity Capital Markets at (800) 678-9147 or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On August 25, 2021 PharmaCyte Biotech, Inc. (NASDAQ: PMCB) (PharmaCyte or Company), 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 the Company will expand its product pipeline to again include its diabetes program and its malignant ascites program (Press release, PharmaCyte Biotech, AUG 25, 2021, View Source [SID1234586889]). Expanding the product pipeline was made possible after PharmaCyte closed on two public offerings, totaling approximately $90 million, including exercised warrants.

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 Chief Executive Officer of PharmaCyte, Kenneth L. Waggoner, said of the capital raises, "With the closing of two separate public offerings totaling approximately $90 million now complete, PharmaCyte can, for the first time in its history, operate comfortably in its development of treatments for hard-to-treat diseases without being constrained by financial resources.

"For a myriad of reasons, this is unquestionably good news for the Company, our shareholders and the patients we hope to treat. Most importantly, we now have the capital to complete the work necessary to satisfy the FDA’s requests to lift the clinical hold and receive an open Investigational New Drug application (IND) for our treatment in locally advanced, inoperable pancreatic cancer (LAPC). In addition, PharmaCyte is positioned to immediately move into a fully funded clinical trial in LAPC should the FDA lift the clinical hold. Also, because the Company is now well-capitalized, we can broaden our entire development pipeline, including our diabetes and malignant ascites programs.

"We were able to attract institutional investors to accomplish this substantial raise while maintaining our low-float, and we have no plans to raise additional capital any time soon."

Mr. Waggoner, discussing the expansion of the Company’s product pipeline, continued, "PharmaCyte remains laser focused on getting our leading product candidate, the treatment for LAPC, into a Phase 2b clinical trial. However, after deferring our diabetes and malignant ascites programs to dedicate every dollar to our treatment for LAPC, we are now ideally situated to reengage these two programs.

"We are excited to have the opportunity to continue what is very important work in the development of a treatment for Type 1 and insulin dependent Type 2 diabetes and for a treatment to delay the production and accumulation of malignant ascites fluid that results from abdominal tumors."

To learn more about PharmaCyte’s pancreatic cancer therapy and how it will work inside a patient’s body to treat locally advanced, inoperable pancreatic cancer, we encourage you to watch PharmaCyte’s documentary video at View Source