On November 19, 2020 MannKind Corporation (NASDAQ:MNKD) reported that it has achieved the final development milestone under its licensing and collaboration agreement with United Therapeutics for the development and commercialization of a dry powder formulation of treprostinil (Press release, Mannkind, NOV 19, 2020, View Source [SID1234571430]).

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Treprostinil Technosphere ("TreT") is an investigational product currently being evaluated in clinical trials for the treatment of pulmonary arterial hypertension (PAH). All current clinical trials are now fully enrolled, including the BREEZE study in patients with PAH and a pivotal pharmacokinetics study in healthy subjects, both of which are being conducted by United Therapeutics, as well as a human factors study being conducted by MannKind. In addition, MannKind’s stability program for TreT has reached the milestone required for a regulatory filing.

"We are looking forward to working with United Therapeutics during the first part of 2021 to prepare an FDA submission for TreT," said Michael Castagna, Chief Executive Officer of MannKind.

MannKind has now received all of the milestone payments that were specified in its agreement with United Therapeutics. MannKind remains entitled to receive low double-digit royalties on net sales of TreT. MannKind will also manufacture supplies of TreT for United Therapeutics and will earn a manufacturing margin.

On November 19, 2020 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, reported the enrollment and dosing of the first patient in a Phase 1 clinical trial evaluating the ability of BP1002 to treat refractory/relapsed lymphoma and chronic lymphocytic leukemia (CLL) patients (Press release, Bio-Path Holdings, NOV 19, 2020, View Source [SID1234571429]).

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BP1002 targets the protein Bcl-2, which is responsible for driving cell survival in up to 60% of all cancers. High expression of Bcl-2 has been correlated with adverse prognosis for patients diagnosed with relapsed CLL or with relapsed, aggressive non-Hodgkin’s lymphoma. Preclinical studies have shown BP1002 to be a potent inhibitor against the Bcl-2 target, and the Company believes that its benign safety profile should enable BP1002 combination therapy with approved agents.

A total of six evaluable patients will be treated with BP1002 monotherapy in a standard 3+3 design, with a starting dose of 20 mg/m2. The treatment cycle consists of two doses per week over four weeks, resulting in eight doses administered over twenty-eight days. The primary objectives of the study include safety and tolerability of escalating doses of BP1002, recommended Phase 2 dose of BP1002, pharmacokinetics of BP1002 and BP1002 activity on Bcl-2 expression. Secondary endpoints include several efficacy measurements of tumor response.

"This study will mark a critical step in understanding the potential benefits that BP1002 may bring to patients suffering with advanced lymphoid malignancies," said Jorge Cortes, M.D., Director of the Georgia Cancer Center and Chairman of the Bio-Path Scientific Advisory Board. "Importantly, BP1002 activity is based on blocking the Bcl-2 messenger RNA and not the BH3 domain, as is the case with venetoclax. As a result, we believe BP1002 may provide a much-needed alternative for patients with malignancies that relapsed or are refractory to venetoclax."

"We are delighted to initiate this first-in-human clinical study of our second drug product candidate derived from the DNAbilize platform. Given the encouraging pre-clinical data and safety profile seen to date, we are very excited to begin this study, which is expected to demonstrate safety and to show initial efficacy signals in these indications with significant unmet medical need," said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings.

Ian W. Flynn, M.D. will serve as the national coordinating Principal Investigator for the Phase 1 trial. Dr. Flynn is the director of lymphoma research at the Sarah Cannon Research Institute. Other sites for the clinical trial include the Georgia Cancer Center at Augusta University and The University of Texas M.D. Anderson Cancer Center.

On November 19, 2020 Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported that the European Commission (EC) has granted Orphan Drug designation for zanidatamab, the company’s investigational HER2-targeted bispecific antibody, in patients with gastric cancer (Press release, Zymeworks, NOV 19, 2020, View Source [SID1234571428]).

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"We are encouraged by the European Commission’s and European Medicines Agency’s recognition of the benefit that zanidatamab can provide in the treatment of HER2-expressing gastric cancers as we continue to expand our clinical development globally," said Diana Hausman, M.D., Chief Medical Officer at Zymeworks.

The EC grants Orphan Drug designation to therapies that represent a significant benefit over existing treatments, are intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease, and where prevalence of the condition in the European Union (EU) is less than 5 in 10,000 persons. Orphan drug designation gives companies certain benefits, including 10 years of market exclusivity following regulatory approval, reduced regulatory fees, clinical protocol assistance, and access to research grants.

"While gastric cancer is a rare disease, European countries experience the highest incidence rates among all Western countries" said James Priour, Senior Vice President, Commercial, at Zymeworks. "The European Commission’s granting of Orphan Drug designation for zanidatamab represents an important step towards bringing this promising investigational medicine to patients in Europe and around the world."

Zymeworks previously received Orphan Drug designations for zanidatamab in gastric, biliary tract and ovarian cancers from the US FDA, as well as two Fast Track designations from the FDA for zanidatamab.

About Zanidatamab

Zanidatamab is a bispecific antibody, based on Zymeworks’ Azymetric platform, that can simultaneously bind two non-overlapping epitopes of HER2, known as biparatopic binding. This unique design results in multiple mechanisms of action including dual HER2 signal blockade, increased binding, and removal of HER2 protein from the cell surface, and potent effector function leading to encouraging antitumor activity in patients. Zymeworks is developing zanidatamab in multiple Phase 1, Phase 2, and registration-enabling clinical trials globally as a targeted treatment option for patients with solid tumors that express HER2. The US FDA has granted two Fast Track designations to zanidatamab, one as a single agent for refractory BTC and one in combination with standard of care chemotherapy, for first-line Gastroesophageal Adenocarcinoma (GEA). Zanidatamab has also received Orphan Drug designations for the treatment of biliary tract, gastric and ovarian cancers from the US FDA, in addition to Orphan Drug designation for the treatment of gastric cancer from the European Medicines Agency.

On November 19, 2020 Immunomic Therapeutics, Inc., ("ITI"), a privately-held clinical-stage biotechnology company pioneering the study of nucleic acid immunotherapy platforms, and CoImmune, Inc. ("CoImmune"), a privately held clinical-stage immuno-oncology company developing cell-based therapeutics to treat unmet medical needs in blood-borne and solid tumor indications, reported that the companies have entered into a license agreement for ITI to use CoImmune’s proprietary dendritic cell process for certain ITI cell therapy vaccine programs (Press release, Immunomic Therapeutics, NOV 19, 2020, View Source [SID1234571427]). The partnership highlights the oncology pipeline of ITI and CoImmune’s expertise and technology in the development and manufacture of cell-based therapeutics.

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Under the terms of the license agreement that supports this partnership, ITI will employ CoImmune’s process for manufacturing and cGMP work for the advancement of ITI-1020, ITI’s autologous dendritic cell (DC) investigational vaccine loading with mRNA encoding LAMP: pp65 for the treatment of newly diagnosed glioblastoma (GBM). CoImmune is eligible to receive milestone payments from ITI upon the successful achievement of certain agreed-upon programmatic goals, including initiation of clinical studies and regulatory submissions. In addition, CoImmune will be eligible to receive royalty payments based upon net sales of ITI-1020, if the product were to be approved in the future.

"We are excited to work with CoImmune, an emerging leader in the development and manufacture of cell-based immunotherapies," said Dr. Tim Coleman, Senior Vice President of Operations at Immunomic Therapeutics. "This agreement supports our immediate and long-term goals for developing and commercializing cancer immunotherapies designed from our investigational UNITE nucleic acid platform. We look forward to collaborating with CoImmune and harnessing their expertise in cell therapies to bring our innovative products to patients in need."

"Our industry is experiencing an increase in the emergence of cell-based therapeutics based on encouraging clinical results to date. Our team at CoImmune has a great deal of expertise in developing and manufacturing these products to efficiently stimulate the immune system against cancer and we are excited to partner with ITI," said Charles Nicolette, CEO of CoImmune. "By combining our team’s know-how with the unique approach to immune system stimulation by UNITE, we believe ITI is well positioned to move into advanced clinical development, initially targeting GBM."

About UNITE

ITI’s investigational UNITE platform, or UNiversal Intracellular Targeted Expression, works by fusing target antigens with the Lysosomal Associated Membrane Protein, an endogenous protein in humans, for immune processing. In this way, ITI’s vaccines (DNA or RNA) have the potential to utilize the body’s natural biochemistry to develop a broad immune response including antibody production, cytokine release and critical immunological memory. This approach could put UNITE technology at the crossroads of immunotherapies in a number of illnesses, including cancer, allergy and infectious diseases. UNITE is currently being employed in Phase II clinical trials as a cancer immunotherapy. ITI is also collaborating with academic centers and biotechnology companies to study the use of UNITE in cancer types of high mortality, including cases where there are limited treatment options like glioblastoma and acute myeloid leukemia. ITI believes that these early clinical studies may provide a proof of concept for UNITE therapy in cancer, and if successful, set the stage for future studies, including combinations in these tumor types and others. Preclinical data is currently being developed to explore whether LAMP nucleic acid constructs may amplify and activate the immune response in highly immunogenic tumor types and be used to create immune responses to tumor types that otherwise do not provoke an immune response.

On November 19, 2020 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported partner Juventas has completed the equivalent of $65 million financing and has initiated and enrolled the first patient in a Phase II registration study for CNCT19 (CD19 CAR-T) in China in patients with relapsed or refractory B-cell non-Hodgkin lymphoma (B-NHL) (Press release, CASI Pharmaceuticals, NOV 19, 2020, View Source [SID1234571426]).

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Dr. Wei-Wu He, CASI’s Chairman, and CEO, commented, "Initiating the Phase II B-NHL registration study and enrolling the first patient is an exciting milestone for the development of CNCT19. Our partner Juventas is also making good progress in the Phase I clinical trial for the treatment of relapsed or refractory acute lymphoblastic leukemia (B-ALL) and is expecting to start the Phase II study by the end of 2020. Its financing provides Juventas with resources to continue moving CNCT19 through registration and we remain excited about its potential as a first-line treatment for B-NHL. In addition, as a large (16%) shareholder of Juventas, we are pleased to see Juventas’ progress in their pipeline and expect its financing to help accelerate its pipeline development. Juventas is an example of CASI’s entrepreneurial partnership model that is built on two components, co-development and equity investment. We believe investments in our partners deepen our collaboration and provides additional potential return to our shareholders. With this approach, we will continue to build CASI’s pipeline, one asset at a time."

About CNCT19

CNCT19 targets CD19, a B-cell surface protein widely expressed during all phases of B-cell development and a validated target for B-cell driven hematological malignancies. CD19- targeted CAR constructs from several different institutions have demonstrated consistently high antitumor efficacy in children and adults with relapsed B-cell acute lymphoblastic leukemia (B-ALL), chronic lymphocytic leukemia (CLL), and B-cell non-Hodgkin lymphoma (B-NHL). CD19 antigen is the most frequently used target in the CAR-T cell therapy clinical trials for hematological malignancies such as leukemia and lymphoma. Juventas is responsible for the development of CNCT19. CASI and Juventas with co-commercialize together under the direction of the program’s joint steering committee.

About Juventas

Juventas Cell Therapy Ltd. is a China-based domestic company located in Tianjin City, China focused on cell therapy. The company’s lead product, CNCT19, devolved from the CD19 CAR-T, was originally created at the Institute of Hematology, Chinese Academy of Medical Sciences, one of the top hematology centers in China. CD19 CAR-T is used to treat patients with acute lymphoblastic leukemia and relapsed non-Hodgkin lymphoma.