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.

On September 17, 2018 Cytori Therapeutics (NASDAQ: CYTX) ("Cytori" or the "Company") reported that it received FDA orphan drug designation for its ATI-1123 chemotherapy drug product candidate, an albumin-stabilized pegylated liposomal docetaxel, for the treatment of small cell lung cancer (Press release, Cytori Therapeutics, SEP 17, 2018, View Source [SID1234529464]).

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The FDA’s Orphan Drug Designation Program provides orphan status to drugs, such as ATI-1123, which are intended for the safe and effective treatment of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Sponsors of a drug receiving orphan designation qualify for various incentives including tax credits for clinical testing, 7 years of marketing exclusivity following FDA approval, and a waiver of prescription drug user fees.

Small cell lung cancer (SCLC), which accounts for approximately 15% of bronchogenic carcinomas with 33,375 new cases and 23,380 deaths estimated for 2017, has been identified by Cytori as a compelling target for ATI-1123. While SCLC is responsive to chemotherapy and radiation therapy, cure occurs only in ~20% of patients, generally restricted to those with limited stage disease. The remaining 80% of patients, including all patients with extensive disease, relapse within months of completing their initial therapy. Availability of 2nd line therapy options are limited, toxic, and provide little benefit in terms of extending survival. Topotecan is the only FDA-approved agent for 2nd line treatment of SCLC and is associated with an overall response rate of 24%, median response duration or time to progression of 14 weeks, and median overall survival of 25 weeks. Treatment usually involves a consecutive 5 day regimen of either IV or oral administration of drug, both which have black box warnings for severe myelosuppression as their use is associated with substantial morbidity including bone marrow suppression leading to neutropenia, thrombocytopenia and anemia requiring interventions of transfusion and growth factor support.

No major treatment advances for SCLC have occurred over the past 30 years. Hence, there remains a significant unmet need for novel agents with better safety profiles both for patients who cannot tolerate the adverse effects of 1st line chemo-radiotherapy and for relapsed/refractory patients who receive Topotecan. Cytori’s ATI-1123 has been designed to fill this need. ATI-1123’s combination of improved liposome stability, reduced toxicity, and superior delivery are expected to provide a therapeutic for SCLC that offers comparable or better efficacy to currently-available standards while having a less intensive administration routine and improved side effect profile.

Cytori is also exploring the development of ATI-1123 to address the shortcomings of docetaxel, a workhorse chemotherapy drug which generated $2.7B in worldwide sales at its peak. Compared to docetaxel, ATI-1123 may have potential to improve safety by removing the need for unwanted solvents, reduce morbidity by eliminating the requirement for standard pretreatment medications, provide better patient convenience and comfort via less time spent in the treatment center, decrease the cost of therapy, and enhance systemic docetaxel exposure.

A U.S. Phase 1 clinical study of ATI-1123 has been completed and published. Of the 29 patients in the study with cervical, gastric, melanoma, non-small cell lung, ovarian, pancreatic, prostate, thyroid, urachal and uterine cancers, 82% demonstrated a clinical benefit with ATI-1123. ATI-1123 exhibited an improved safety profile versus the Taxotere label with a 31% reduction in neutropenia and anemia. Further, ATI-1123 showed a 20% increase in maximum tolerated dose versus standard docetaxel and signs of efficacy with 1 partial responder.

On September 17, 2018 GamaMabs Pharma, a biotechnology company developing optimized therapeutic antibodies targeting the Anti-Müllerian Hormone Receptor II (AMHRII) for the treatment of cancer, reported that it has dosed the first patient in its Phase 2 clinical trial – of GM102 single agent and in combination with Trifluridine/Tipiracil (Lonsurf) – in patients with advanced or metastatic colorectal cancer (CRC) (Press release, GamaMabs Pharma, SEP 17, 2018, View Source [SID1234529581]).

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GM102 is a first-in-class glyco-engineered (low-fucose) monoclonal antibody targeting tumor antigen AMHRII. AMHRII is re-expressed in approximately 70% of colorectal cancer patients. GM102 exerts its anti-tumor activity through macrophage and NK cell engagement in the tumor microenvironment, resulting in enhanced tumor phagocytosis and ADCC (Antibody Dependent Cell Cytotoxicity).

This phase 2 study resulted from collaborative research with various European academic research centers and hospitals, which unveiled a wide AMHRII expression in human CRC tumors and a substantial expression of the target on tumor cells at the membrane level. These results were published at the AACR (Free AACR Whitepaper) Annual meeting in April 2018.

"The C201 study will investigate the anti-tumor activity of GM102 in this new indication and its potential synergism with Lonsurf for patients who have progressed on prior therapies. Most CRC tumors express moderate to large macrophage infiltration. GM102 therefore has a potential to enhance macrophage phagocytosis on tumor cells in addition to acting synergistically with Lonsurf," said Isabelle Tabah-Fisch, M.D., GamaMabs’ chief medical officer. "This is a key step in the development of our molecule, which has already shown excellent tolerability in patients with gynecological cancers and first signs of activity in the first patients in the phase 1a/1b C101 study, recently presented at the ASCO (Free ASCO Whitepaper) 2018 conference."

"We are still in serious need of active drugs for our CRC patients," said Professor B. Melichar, investigator at Olomouc University Hospital, Olomouc, Czech Republic. "AMHRII is yet another unexplored target in CRC. GM102 is the latest generation immunological agent to be tested in colorectal cancers, especially those which do not respond to checkpoint inhibitors. We look forward to evaluating this new agent and I am happy to have the first patient already enrolled in the study."

The European multicenter two-parallel non-randomized cohort C201 Phase 2 study will assess objective response rates, immunological changes in the tumor microenvironment, progression-free and overall survival rates for patients with advanced/metastatic colorectal cancers treated with GM102 single agent and in combination with Lonsurf. The study will enroll patients who have progressed after at least two lines of prior systemic therapies for metastatic or locally advanced disease and have received all prior available therapies (cohort 1) or are candidates to receive Lonsurf (cohort 2).

Lonsurf (trifluridine-tipiracil hydrochloride) is a chemotherapy drug, used to treat metastatic colorectal cancer. Servier commercializes Lonsurf in Europe and other countries outside of the United States, Canada, Mexico and Asia. Taiho Pharmaceutical has the right to develop and commercialize Lonsurf in the United States, Canada, Mexico, and Asia.

On September 17, 2018 Navidea Biopharmaceuticals, Inc. (NYSE American: NAVB) ("Navidea" or the "Company"), a company focused on the development of precision immunodiagnostic agents and immunotherapeutics, reported closing of a $3 million private placement (Press release, Navidea Biopharmaceuticals, SEP 17, 2018, View Source [SID1234530336]). The Company entered into a definitive securities purchase agreement with an existing investor, John K. Scott, Jr., pursuant to which the Company received aggregate gross proceeds of $3 million in exchange for the issuance of 18,320,610 shares of the Company’s common stock, par value $0.001 per share. The securities to be issued to Mr. Scott will represent approximately 10% of the Company’s outstanding common stock after such issuance. The securities are subject to a 180-day lock-up and there are no registration rights.

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The Company will use the proceeds from the private placement for general working capital purposes, including, but not limited to, research and development, and other operating expenses.

"Our family has been a long-term shareholder since 2003 and we now have confidence in the science, management and the direction of the company. This is why we have made this investment at this time. Our intent is to the give the company additional flexibility and stability," stated John K. Scott, Jr.

"We are very happy that an existing long-term shareholder continues to show faith in the potential of Navidea as well as the future direction of the Company led by a new, streamlined management team," commented Mr. Jed A. Latkin, Chief Executive Officer of Navidea. "The ability to quickly raise $3 million without having to pay any fees, give any warrants and at a price near market was an opportunity that the Company could not pass up. It also gives the Company significant runway to allow for the planned launch of the confirmatory rheumatoid arthritis study in the upcoming quarter."

This press release shall not constitute an offer to sell or the solicitation of an offer to buy securities, nor shall there be any sale of these securities in any state or other 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 17, 2018 Coherus BioSciences, Inc. (Nasdaq: CHRS), reported that senior management will be presenting at the 2018 Cantor Global Healthcare Conference being held in New York on Tuesday, October 2, 2018 at 8:00 am ET (Press release, Coherus Biosciences, SEP 17, 2018, View Source;p=RssLanding&cat=news&id=2367664 [SID1234529471]).

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The audio portion of the presentation will be available on the investors page of the Coherus BioSciences website at View Source