NewLink Genetics to Participate in the Cantor Global Healthcare Conference

On SEptember 18, 2018 NewLink Genetics Corporation (NASDAQ:NLNK) reported that the Company will present at the 2018 Cantor Global Healthcare Conference on Wednesday, October 3, 2018, at 4:00PM ET in New York, New York (Press release, NewLink Genetics, SEP 18, 2018, View Source [SID1234530679]).

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A live webcast of the presentation will be available on the Company’s website at www.newlinkgenetics.com in the "Investors & Media" section under "Events and Presentations." An archived edition of the presentation will be available on NewLink Genetics’ website later that day.

G1 Therapeutics Announces Pricing of Offering of Common Stock

On September 18, 2018 G1 Therapeutics, Inc. (Nasdaq: GTHX), a clinical-stage oncology company, reported the pricing of an underwritten public offering of 3,000,000 shares of its common stock at a public offering price of $60.00 per share, for total gross proceeds of $180,000,000 (Press release, G1 Therapeutics, SEP 18, 2018, View Source [SID1234529789]). All of the shares in the offering will be sold by G1 Therapeutics. In addition, G1 Therapeutics has granted the underwriters a 30-day option to purchase up to an additional 450,000 shares of common stock at the public offering price, less the underwriting discount. The offering is expected to close on September 21, 2018, subject to customary closing conditions.

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J.P. Morgan Securities LLC and Cowen and Company, LLC are serving as joint book-running managers for the offering. Needham & Company, LLC and Wedbush Securities Inc. are acting as lead managers for the offering. BTIG, LLC and H.C. Wainwright & Co., LLC are acting as co-managers for the offering.

The shares are being offered pursuant to a "shelf" registration statement previously filed and declared effective by the Securities and Exchange Commission (the "SEC"). A preliminary prospectus supplement and accompanying prospectus relating to the offering have been filed with the SEC and are available on the website of the SEC at www.sec.gov. Copies of the final prospectus supplement and accompanying prospectus relating to the offering may be obtained, when available, from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (866) 803-9204; or from Cowen and Company, LLC, c/o Broadridge Financial Solutions, Attention: Prospectus Department, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (631) 592-5973.

This press release shall not constitute an offer to sell or a 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.

CBT Pharmaceuticals Initiates the APOLLO Oncology Clinical Trials Program

On September 18, 2018 CBT Pharmaceuticals (CBT), a U.S. and China-based innovative biopharmaceutical company committed to becoming a leader in the discovery and development of oncology combination therapies, reported the initiation of the APOLLO Oncology Clinical Trials Program (Press release, CBT Pharmaceuticals, SEP 18, 2018, View Source [SID1234529733]). The APOLLO series of trials will evaluate and investigate whether CBT’s proprietary assets can work in concert with other agents to improve single agent immunotherapy response rates, and, ultimately, to confer clinical benefit to patients with cancer. In Greek, the verb ‘apollymi’ means "to destroy," and in Greek mythology, Apollo is the god of healing.

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The APOLLO program has been initiated with the dosing of the first patient in the initial trial in the series: a Phase 1/2 open label, multi-center dose escalation and expansion study in which CBT’s proprietary c-Met inhibitor (bozitinib; CBT-101) will be administered concomitantly with anti PD-1 cancer immunotherapies. Utilizing biomarkers to identify patients likely to benefit from the combination, the trial will investigate whether the combination will improve single agent immunotherapy response rates as a result of the immunosuppressive tumor microenvironment due to tumor associated neutrophils (TANs).

"Initiating our APOLLO Oncology Clinical Trials Program and dosing the first patient is a major milestone for CBT as we advance our mission to improve the lives of cancer patients through combination treatment regimens," stated Sanjeev Redkar, PhD, Co-Founder and President. "The APOLLO program is designed to investigate our proprietary assets alongside each other and is focused on understanding the science and genetics to identify the appropriate patients likely to benefit from the regimen. We are grateful for the support of our investigators in running our series of trials."

The initial study is a two-arm clinical trial targeting locally advanced or metastatic disease: CBT-101 with CBT-501 (genolimzumab; anti-PD-1) in hepatocellular carcinoma (HCC), or CBT-101 and nivolumab in renal cell carcinoma (RCC). CBT-101 targets the epithelial to mesenchymal transition (EMT) pathway, and CBT-501 is CBT’s IgG4 humanized monoclonal antibody against the PD-1 membrane receptor on immune cells. Nivolumab (OPDIVO; Bristol-Myers Squibb Company) is approved for advanced kidney cancer.

Dr. Alex Powell, MBBS, FRACP, Affinity Oncology, Hollywood Private Hospital in Perth, Western Australia, added, "One of the newer paradigms for treating cancer patients is combining immunotherapy agents as this synergistic approach may provide improved outcomes. In this first trial in the series, we believe that giving a c-Met inhibitor concomitantly with an anti PD-1 therapy may produce a positive added response in patients with HCC and RCC where monotherapy treatment has proven effective. Affinity Oncology, and the entire Australia and New Zealand team, is thrilled to partner with CBT on this combination approach."

The primary objective of the Phase 1 portion of the trial will be to identify any dose limiting toxicities, evaluate overall safety, and assess the tolerability of CBT-101 and CBT-501 for HCC and CBT-101 and nivolumab for RCC. The Phase 2 primary objective is to assess preliminary efficacy by objective response rate (ORR) and duration of response (DOR) per irRECIST (Immune-related Response Evaluation Criteria In Solid Tumors). Secondary objectives include: determination of the recommended Phase 2 dose, determination of the pharmacokinetic (PK) parameters of CBT-101 and CBT-501 when administered in combination, assessment of clinical benefit rate, progression free survival, and overall survival at 6, 12, 18 and 24 months. Whole blood, serum, plasma and peripheral blood mononuclear cells will be collected to assess TANs. For additional information regarding the trial, please visit clinicaltrials.gov identifier: NCT03655613.

About Hepatocellular Carcinoma and Renal Cell Carcinoma

Hepatocellular Carcinoma (HCC)

Liver cancer is the sixth most common cancer in the world, and hepatocellular carcinoma (HCC) is the most common type of liver cancer. HCC occurs most often in people with chronic liver diseases, such as cirrhosis caused by hepatitis B or hepatitis C infection. While it is estimated that there will be approximately 42,000 new cases and 30,000 deaths from liver and intrahepatic bile duct cancer in the United States in 2018, about 83% of liver cancer cases occur in less developed countries. The highest incidence of liver cancer is in Asia and Africa.

Renal Cell Carcinoma (RCC)

Renal Cell Carcinoma (RCC) is the most common type of kidney cancer that begins in the lining of the renal tubules in the kidney. The renal tubules filter the blood and produce urine. Kidney cancer is the 12th most common cancer in the world with 338,000 new cases diagnosed in 2012. About 59% of kidney cancer cases occurred in more developed countries with the highest incidence in Northern America and Europe. It is estimated that there will be approximately 65,000 new cases and 15,000 deaths from kidney and renal pelvis cancer in the United States in 2018.

OBI Pharma Granted FDA Orphan Drug Designation for OBI-3424 for the Treatment of Acute Lymphoblastic Leukemia (ALL)

On September 18, 2018 OBI Pharma, Inc., a Taiwan biopharma company (TPEx: 4174), reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for OBI-3424 for the Treatment of Acute Lymphoblastic Leukemia (ALL) (Press release, OBI Pharma, SEP 18, 2018, View Source [SID1234529670]). OBI-3424 is a first in class DNA alkylating cancer therapeutic agent targeting aldo-keto reductase 1C3 (AKR1C3) overexpressing cancers.

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This is the second FDA orphan drug designation for OBI-3424. In July, 2018, OBI-3424 was granted orphan drug status for the Treatment of Hepatocellular Carcinoma (HCC). A Phase 1/2 study of OBI-3424 in patients with solid tumors, including HCC and castrate-resistant prostate cancer (CRPC), has commenced enrollment at the University of Texas M.D. Anderson Cancer Center.

Amy Huang, General Manager of OBI Pharma, noted, "This additional orphan drug designation for OBI-3424 by the FDA is a significant step in the development of this drug candidate in ALL, including T-ALL, an unmet medical need disease with limited treatment options. We are excited that the FDA has recognized the need to develop novel targeted therapeutic agents such as OBI-3424 in the fight against ALL".

About Acute Lymphoblastic Leukemia (ALL)

Acute Lymphoblastic Leukemia (ALL), also known as Acute Lymphocytic Leukemia, is a rare blood cancer affecting the maturation of B-cell and T-cell lymphoblasts from progenitor cells. The current prevalence of ALL in the US is around 86,462 cases in 2018. The disease affects primarily children, with 60% of cases occurring at age <20 years. The remission rate for pediatric ALL is approximately 90%, with overall survival around 60-70% in recent years. Current treatments for ALL have been less successful in both infant and adult patients, as well as patients with recurrent disease, leading to an unmet medical need for new treatments.

About Orphan Drug Designation (ODD)

The orphan drug designation provides OBI Pharma with potential benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees, and tax credits for qualified clinical trials. The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the U.S.

About OBI-3424

OBI-3424 is a first-in-class novel small-molecule prodrug that selectively targets cancers overexpressing the enzyme aldo-keto reductase 1C3 (AKR1C3), and selectively releases a potent DNA alkylating agent in the presence of the AKR1C3 enzyme. This selective mode of activation distinguishes OBI-3424 from traditional alkylating agents, such as cyclophosphamide and ifosfamide, which are non-selective.

AKR1C3 overexpression has been documented in a number of treatment-resistant and difficult-to-treat cancers including: hepatocellular carcinomas (HCC), castrate-resistant prostate cancer (CRPC), and acute lymphoblastic leukemia (ALL), including T-ALL. AKR1C3 is highly expressed in up to 15 solid and liquid tumors.

OBI Pharma holds worldwide rights for OBI-3424 with the exception of the following countries, whose rights are held by Ascenta Pharma: China, Hong Kong, Macao, Taiwan, Japan, South Korea, Singapore, Malaysia, Thailand, Turkey, and India.

Loxo Oncology Announces Accepted Abstracts at the 88th Annual Meeting of the American Thyroid Association

On September 18, 2018 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company developing highly selective medicines for patients with genomically defined cancers, reported that abstracts from its LOXO-292 and larotrectinib programs have been accepted for oral presentations at the 88th Annual Meeting of the American Thyroid Association to be held October 3-7, 2018, in Washington, DC (Press release, Loxo Oncology, SEP 18, 2018, View Source [SID1234529642]).

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The LOXO-292 oral presentation will provide an updated analysis of patients with RET mutant medullary thyroid cancer and RET fusion thyroid cancers enrolled in the dose escalation cohorts of the ongoing LIBRETTO-001 Phase 1/2 clinical trial. The larotrectinib oral presentation will provide an analysis of patients with TRK fusion thyroid cancer enrolled to the larotrectinib clinical program.

The schedule for the presentations is as follows:

LOXO-292 Oral Presentation Session Date & Time: October 6, 2018, 9:05 a.m.-9:20 a.m. ET
Title: Clinical Activity of LOXO-292, a Highly Selective RET Inhibitor, in Patients with RET-Altered Thyroid Cancers
Session Title: Clinical Short Call Oral
Presenter: Lori J. Wirth, M.D.

Larotrectinib Oral Presentation Session Date & Time: October 4, 2018, 1:50 p.m.-2:05 p.m. ET
Title: Activity of Larotrectinib in Patients with Advanced TRK Fusion Thyroid Cancer
Session Title: Thursday Clinical Oral Abstracts
Presenter: Marcia S. Brose, M.D., Ph.D.

About LOXO-292
LOXO-292 is an oral and selective investigational new drug in clinical development for the treatment of patients with cancers that harbor abnormalities in the rearranged during transfection (RET) kinase. RET fusions and mutations occur across multiple tumor types with varying frequency. LOXO-292 was designed to inhibit native RET signaling as well as anticipated acquired resistance mechanisms that could otherwise limit the activity of this therapeutic approach. LOXO-292 has been granted Breakthrough Therapy Designation by the U.S. FDA.

LOXO-292 is currently being studied in the global LIBRETTO-001 Phase 1/2 trial. For additional information about the LOXO-292 clinical trial, please refer to www.clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology Physician and Patient RET Clinical Trial Hotline at 1-855-RET-4-292 or email [email protected].

About RET-Altered Cancers
Genomic alterations in the RET kinase, which include fusions and activating point mutations, lead to overactive RET signaling and uncontrolled cell growth. RET fusions have been identified in approximately 2% of non-small cell lung cancer, 10-20% of papillary and other thyroid cancers, and a subset of other cancers. Activating RET point mutations account for approximately 60% of medullary thyroid cancer (MTC). Both RET fusion cancers and RET-mutant MTC are primarily dependent on this single activated kinase for their proliferation and survival. This dependency, often referred to as "oncogene addiction," renders such tumors highly susceptible to small molecule inhibitors targeting RET.

About Larotrectinib
Larotrectinib is an oral and selective investigational tropomyosin receptor kinase (TRK) inhibitor in clinical development for the treatment of patients with cancers that harbor a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body. In clinical trials, larotrectinib demonstrated anti-tumor activity in patients with tumors harboring NTRK gene fusions, regardless of patient age or tumor type. In an analysis of 55 RECIST-evaluable adult and pediatric patients with NTRK gene fusions, using a July 17, 2017 data cutoff, larotrectinib demonstrated a 75 percent centrally-assessed confirmed overall response rate (ORR) and an 80 percent investigator-assessed confirmed ORR, across many different types of solid tumors. The majority (93 percent) of all adverse events were grade 1 or 2.

Larotrectinib has been granted Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation and Orphan Drug Designation by the U.S. FDA.

In November 2017, Loxo Oncology and Bayer entered into an exclusive global collaboration for the development and commercialization of larotrectinib and LOXO-195, a next-generation TRK inhibitor. Bayer and Loxo Oncology are jointly developing the two products with Loxo Oncology leading the ongoing clinical studies as well as the filing in the U.S., and Bayer leading ex-U.S. regulatory activities and worldwide commercial activities. In the U.S., Loxo Oncology and Bayer will co-promote the products.

For additional information about the larotrectinib clinical trials, please refer to www.clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology Physician and Patient Clinical Trial Hotline at 1-855-NTRK-123 or visit www.loxooncologytrials.com/trk-trials.

About TRK Fusion Cancer
TRK fusion cancer occurs when a neurotrophic tyrosine receptor kinase (NTRK) gene fuses with another unrelated gene, producing an altered tropomyosin receptor kinase (TRK) protein. The altered protein, or TRK fusion protein, is constantly active, triggering a permanent signal cascade. These proteins become the primary driver of the spread and growth of tumors in patients with TRK fusion cancer. TRK fusion cancer is not limited to certain types of cells or tissues and can occur in any part of the body. NTRK gene fusions occur in various adult and pediatric solid tumors with varying prevalence, including appendiceal cancer, breast cancer, cholangiocarcinoma, colorectal cancer, GIST, infantile fibrosarcoma, lung cancer, mammary analogue secretory carcinoma of the salivary gland, melanoma, pancreatic cancer, thyroid cancer, and various sarcomas. Only sensitive and specific tests can reliably detect TRK fusion cancer. Next-generation sequencing (NGS) can provide a comprehensive view of genomic alterations across a large number of genes. Fluorescence in situ hybridization (FISH) can also be used to test for TRK fusion cancer, and immunohistochemistry (IHC) can be used to detect the presence of TRK protein.