On September 11, 2019 Athenex, Inc. (Nasdaq: ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported that its partner, Guangzhou Xiangxue Pharmaceutical Co., Ltd., initiated a Phase I study in China of KX2-361 (formerly known as KX-02) oral treating advanced malignant solid tumors (Press release, Athenex, SEP 11, 2019, View Source [SID1234573887]).

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KX2-361, which is being developed for the treatment of glioblastoma multiforme (GBM), is the second compound derived from Athenex’s Src kinase inhibition platform. It is a closely related structural analog of Athenex’s tirbanibulin (formerly known as KX2-391 or KX-01) with a distinct ability to cross the blood brain barrier, making it a potential therapeutic candidate for treating brain cancers, including GBM, as well as brain metastases. Studies of KX2-361 oral in preclinical mouse GBM tumor models resulted in complete tumor eradication as well as extended survival compared to the current standard of care, temozolomide.

"KX2-361 is a potentially valuable treatment option for GBM patients, and we are initiating this Phase I study on the strength of encouraging results in preclinical studies," stated Mr. YongHui Wang, Chairman of Xiangxue Pharmaceutical. "We are excited to advance this promising candidate in the clinic to potentially improve the lives of patients with GBM."

"We are impressed by the R&D capabilities of our partner and delighted by their decision to advance another product candidate from our Src kinase inhibition platform," stated Dr. Johnson Lau, Chairman and Chief Executive Officer of Athenex. "KX2-361 may potentially expand the range of cancers we can address with our broad oncology pipeline, especially for rare diseases with significant unmet medical need like GBM."

The Phase I clinical study in China is a single-center, open-label dose escalation trial that will enroll 36-72 patients with advanced malignant solid tumors who have no standard treatment or standard treatment failed.

In 2012, Athenex out-licensed KX2-361 to Xiangxue Pharmaceutical for development and marketing in Greater China and Singapore. In May 2017, the China National Medical Products Administration (NMPA) (formerly China FDA) allowed Xiangxue’s IND to commence clinical trials of KX2-361 in China. Athenex also received U.S. IND allowance for KX2-361 in 2014 and the product candidate was granted Orphan Drug Designation by the U.S. FDA.

On September 11, 2019 Genprex, Inc. (NASDAQ: GNPX), a clinical-stage gene therapy company, reported that independent researchers reported in a recent study that TUSC2, a tumor suppressor gene and the active agent in Genprex’s Oncoprex immunogene therapy, prevented tumor growth in triple-negative breast cancer (TNBC), which is currently considered an incurable cancer with limited therapeutic options (Press release, Genprex, SEP 11, 2019, View Source [SID1234539442]). Genprex has no affiliation with these researchers.

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The study, published in Nature, first found MicroRNA-138 as a diagnostic biomarker for TNBC, which currently lacks targeted therapies due to its inability to express the estrogen and progesterone hormone receptors and the human epidermal growth factor receptor 2 (HER2), thus the name for triple-negative breast cancer. Depletion of miR-138 was found to lead to apoptotic cell death in vitro and prevented tumorigenesis in vivo. TUSC2 was found to be a direct target of miR-138, and TUSC2 mimics the effects of miR-138 knockdown, preventing tumor growth. The researchers deduced that TUSC2 is a downstream tumor suppressor that is directly repressed by miR-138.

The study reports that triple-negative breast cancer is an extremely aggressive subtype of breast cancer which is associated with poor prognosis and high mortality rates. The lack of targeted treatment for triple-negative breast cancer makes it an increasingly feared diagnosis.

Genprex is conducting clinical and pre-clinical research to evaluate the effectiveness of TUSC2 when combined with targeted therapies and immunotherapies for non-small cell lung cancer. Existing pre-clinical data also suggest that TUSC2 may be effective against glioblastoma, head and neck cancer, kidney cancer, and soft tissue sarcomas. Now, this new independent study raises the possibility that TUSC2 expression, through miR-138 targeting, may also be used to treat the most aggressive subset of breast cancer.

"The results of the study evaluating TUSC2 for the treatment of triple-negative breast cancer are encouraging," said Rodney Varner, Genprex’s Chairman and Chief Executive Officer. "We believe that the data reported in this Nature article by independent researchers supports our belief that TUSC2 may be effective to treat a variety of cancers, including some of the most deadly types of cancer."

On September 11, 2019 Fate Therapeutics, Inc. (the "Company" or "Fate Therapeutics") (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that it has commenced an underwritten public offering of its common stock (Press release, Fate Therapeutics, SEP 11, 2019, View Source [SID1234539459]). Fate Therapeutics intends to use the net proceeds from the offering to fund clinical trials and nonclinical studies, the manufacture of its clinical product candidates, the expansion of its cGMP compliant manufacturing operations, the conduct of preclinical research and development, and for general corporate purposes. All shares of common stock to be sold in the offering will be offered by Fate Therapeutics. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or the actual size or terms of the offering.

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Jefferies and Citigroup are acting as joint book-running managers for the offering.

The securities described above are being offered by Fate Therapeutics pursuant to an automatic shelf registration statement on Form S-3 (File No. 333-228513) that was previously filed by Fate Therapeutics with the Securities and Exchange Commission (the "SEC") and automatically became effective upon filing on November 21, 2018.

A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website at View Source A copy of the preliminary prospectus supplement and accompanying prospectus can be obtained by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by e-mail at [email protected] or by telephone at (877) 821-7388; or Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (800) 831-9146.

This press release does not constitute an offer to sell or the solicitation of an offer to buy these securities, 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 September 11, 2019 FORMA Therapeutics, Inc. reported that abstracts for the company’s next generation IDH1m inhibitor, olutasidenib (FT-2102), have been accepted for two presentations at the 2019 Society for NeuroOncology Annual Meeting, taking place November 20-24, 2019 in Phoenix, Arizona (Press release, Forma Therapeutics, SEP 11, 2019, View Source [SID1234539443]). Olutasidenib was designed to have a differentiated efficacy, durability and safety profile, as well as blood brain barrier penetrance, and is intended to treat all cancers with IDH1 mutations, including acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), gliomas and other solid tumors.

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"I am proud of the progress FORMA has made in the first eight months of 2019 and am truly excited for this next phase of growth for our company centered on our wholly-owned therapeutic pipeline focused on rare hematologic diseases and cancers," said Frank Lee, CEO of FORMA Therapeutics. "Gliomas are the most common, aggressive and difficult-to-treat primary brain tumors, with current treatment options limited to surgery, chemotherapy and radiation. Furthermore, gliomas typically affect relatively young patients. We look forward to presenting data regarding the brain penetrant activity of olutasidenib as well as initial results from our ongoing Phase 1b/2 study in patients with relapsed/refractory IDH1 mutant gliomas this November at SNO."

Olutasidenib Status in Gliomas/Solid Tumors:

An ongoing Phase 1b/2 study of olutasidenib is enrolling patients with gliomas or other advanced solid tumors with an IDH1 mutation.
Data from olutasidenib clinical trials have been submitted for presentation at medical meetings in the fourth quarter of 2019. Abstracts accepted for presentation at SNO include:
Oral Presentation: FT-2102 – A Potent and Selective Brain Penetrant Inhibitor of Mutant Isocitrate Dehydrogenase
Date and Time: Wednesday, November 20, 2019, 9:45-9:55 AM, MST
Oral Abstract Session: BBB Physiology at the SNO-SCIDOT Joint Conference on Therapeutic Delivery to the CNS
Presenter: Maria Ribadeneira, PhD, FORMA Therapeutics
E-Talk Presentation: Phase 1 study of FT-2102, an inhibitor of mutant IDH1, in patients with relapsed/refractory IDH1 mutant gliomas: preliminary safety and clinical activity
Date and Time: Saturday, November 23, 2019, 5:20 PM – 5:24 PM, MST
E-Talks Session: Group 1: Adult Therapeutics/Immunology Rare Tumors
Presenter: Macarena de la Fuente, MD, Sylvester Cancer Center, University of Miami
About Olutasidenib (FT-2102)

FORMA’s most advanced clinical asset, olutasidenib was designed as a potent and selective next generation inhibitor of mutated isocitrate dehydrogenase 1 (IDH1m) intended to treat patients with relapsed/refractory acute myeloid leukemia (RR/AML) or myelodysplastic syndrome (MDS), and patients with glioma and other solid tumors with an IDH1 mutation. IDH1 is a natural enzyme that is part of the normal metabolism of all cells, but when mutated its activity can promote blood malignancies and solid tumors. IDH1 mutations are present in up to 16% of patients with AML and over 80% of patients with low-grade gliomas. In AML, hypermethylation driven by IDH mutations inhibits normal differentiation of progenitor cells leading to accumulation of immature blasts. Quality of life declines with each successive line of treatment for AML and well-tolerated treatments in relapsed disease remain an unmet need. In gliomas, IDH1 mutations occur early in the tumor pathogenesis and persist throughout progression from a neural stem or progenitor cell. Gliomas are the most common, aggressive and difficult-to-treat primary brain tumors and high-grade gliomas are associated with poor long-term prognosis, with a median survival ranging from 5 to 7 years. Treatment options for relapsed glioma are limited. The rationale for targeting IDH1m is to reverse the oncogenic hypermethylated state to reduce tumor burden through induction of differentiation of immature blasts in AML and by slowing or stopping cancer cell growth in glioma.

In addition to the ongoing Phase 1b/2 study in patients with gliomas and other advanced solid tumors with an IDH1 mutation, a multi-cohort study with olutasidenib as a single agent and in combination with azacitidine is currently enrolling patients with AML and MDS, including a pivotal arm with olutasidenib as a single agent in R/R AML. Patients with treatment-naïve AML and R/R MDS with IDH1m are also being evaluated. Top-line data in patients with R/R AML are expected in the first half of 2020. If there is a positive outcome in R/R AML patients, a new drug application (NDA) is expected to be filed in the second half of 2020.

On September 11, 2019 Fate Therapeutics, Inc. (the "Company" or "Fate Therapeutics") (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported the pricing of an underwritten public offering of 8,600,000 shares of its common stock at a public offering price of $17.50 per share, before underwriting discounts, for an aggregate offering of approximately $150.5 million (Press release, Fate Therapeutics, SEP 11, 2019, View Source [SID1234539460]). Fate Therapeutics has granted the underwriters a 30-day option to purchase up to an additional 1,290,000 shares of its common stock. The proceeds to Fate Therapeutics from this offering are expected to be approximately $141.2 million after deducting underwriting discounts and commissions and other estimated offering expenses but excluding any exercise of the underwriters’ option. Fate Therapeutics intends to use the net proceeds from the offering to fund clinical trials and nonclinical studies, the manufacture of its clinical product candidates, the expansion of its cGMP compliant manufacturing operations, the conduct of preclinical research and development, and for general corporate purposes. All shares of common stock to be sold in the offering are being offered by Fate Therapeutics. The offering is expected to close on or about September 16, 2019, subject to customary closing conditions.

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Jefferies, Citigroup, SVB Leerink and Wells Fargo Securities are acting as joint book-running managers for the offering. Wedbush PacGrow and Cantor Fitzgerald & Co. are acting as co-managers for the offering.

The securities described above are being offered by Fate Therapeutics pursuant to an automatic shelf registration statement on Form S-3 (File No. 333-228513) that was previously filed by Fate Therapeutics with the Securities and Exchange Commission (the "SEC") and automatically became effective upon filing on November 21, 2018. The securities may be offered only by means of a prospectus.

A preliminary prospectus supplement related to the offering was filed with the SEC on September 11, 2019 and is available on the SEC’s website at View Source and a final prospectus supplement related to the offering will be filed with the SEC and will be available on the SEC’s website at View Source. Copies of the final prospectus supplement and the accompanying prospectus for the securities being offered may also be obtained, when available, by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by e-mail at [email protected] or by telephone at (877) 821-7388; Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (800) 831-9146; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525 ext. 6132 or by email at [email protected]; or Wells Fargo Securities, LLC, Attention: Equity Syndicate Department, 375 Park Avenue, New York, New York 10152, by email at [email protected] or by telephone at (800) 326-5897.

This press release does not constitute an offer to sell or the 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.