On April 2, 2020 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, reported it has completed the latest (210 mg/m2) cohort in its European open label, single arm Phase 1/2 clinical trial of Annamycin for the treatment of relapsed or refractory acute myeloid leukemia ("AML"). A total of 19 patients have been treated in the US and Europe, and all results continue to show Annamycin to be safe, and, especially, all have shown Annamycin to be free of cardiotoxicity. Of those,10 have been treated at or above the FDA lifetime maximum anthracycline exposure.

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The Phase 1 portion of this clinical trial, which is described in more detail later in this press release, is designed to establish the safety of Annamycin and to determine the Recommended Phase 2 Dose to be used in the Phase 2 portion of the trial. While the Primary Endpoint of the Phase 1 portion is safety, a Secondary Endpoint is the assessment of efficacy generally defined as an improvement in bone marrow biopsy results sufficient to qualify patients for a potentially curative bone marrow transplant. The Company cautions not to place undue reliance on interim results.

The fourth cohort in Poland receiving a single dose of 210 mg/m2 in the Phase 1 dose escalation portion of the trial was completed with no adverse events and the trial will continue to the next cohort of 240 mg/m2. To date in the European trial, only one adverse event related to Annamycin has been reported; a patient experienced grade 2 mucositis (which resolved to grade 1 within 2 days). In the Company’s recently completed Phase 1 portion of a parallel US Phase 1/2 clinical trial, there were no unexpected serious adverse events (SAE) and no dose limiting toxicities (DLT) at any dose tested.

We refer to Annamycin as a "next generation anthracycline," because it is designed to provide enhanced therapeutic benefits when compared with traditional anthracyclines while reducing the potential for unwanted cardiotoxicity, or damage to the heart. This design intent has previously been validated with preclinical toxicology studies in animal models (as required by FDA) demonstrating Annamycin has little to no cardiotoxicity when compared with doxorubicin. Of the 19 patients treated thus far in both trials, none has shown any evidence of cardiotoxicity. This includes 10 patients in Poland who were treated at levels above the US maximum allowable cumulative anthracycline dose level (550 mg/m2), a limitation not imposed on our trial in Europe. If upheld in further studies, we believe this lack of toxicity would be an important differentiator between Annamycin and the currently approved anthracyclines, for which cardiotoxicity is a well-known treatment limitation.

Walter Klemp, Chairman and CEO of Moleculin commented, "Now that we are relying upon the European trial to establish an RP2D, and now that it is becoming apparent that the safety profile of Annamycin is even better than we expected, we will explore opportunities to increase the dosing increment between cohorts to speed up the establishment of the Recommended Phase 2 Dose or RP2D. We continue to target establishing the RP2D before the end of the year, but of course this will depend upon the continued rate of recruitment and the actual point at which we begin to see dose limiting toxicities."

The U.S. trial met its primary endpoint, demonstrating the safety of Annamycin in AML patients. Most importantly, all patients reflected the absence of cardiotoxicity (potential damage to the heart), as determined by echocardiograms, as well as cardiac health biomarkers, principally blood troponin levels. Based on testing to date, no patients in either the US or European trial have exhibited evidence of cardiotoxicity. Additionally, there were no unexpected serious adverse events (SAE) and no dose limiting toxicities (DLT) at any dose tested. Although a primary objective of the Phase 1 trial was to evaluate safety, the study also gathered data to support a preliminary assessment of the product’s efficacy. Among other things, the study recorded complete response (CR), partial response (PR), event-free survival (EFS), overall survival (OS; Kaplan-Meier), and time to and duration of remission/response. The Company reported efficacy in 33% of the US patients, even though the drug was dosed at what was expected to be sub-therapeutic levels. The evidence of efficacy consisted of 1 patient who achieved a "morphologically leukemia-free state," which the protocol defined as a CR with incomplete recovery of platelets or neutrophils, and another patient who had a substantial remission of leukemia cutis (a somewhat rare leukemia symptom), from diffuse to 3 lesions.
In all but one of the 7 relapsed patients treated in our Poland trial, a reduction in blasts in the bone marrow aspirate occurred. Of those 7, 2 qualified as PRs and 1 qualified as a bridge-to-transplant. In the last cohort, only 1 patient was relapsed, and that patient had a 33% reduction in bone marrow blasts. In all three patients in the 210 mg/m2 cohort (1 relapsed and 2 refractory), a reduction of circulating blasts to <2% was achieved during treatment.

On April 2, 2020 I-Mab (the "Company") (Nasdaq: IMAB), a clinical stage biopharmaceutical company committed to the discovery, development and commercialization of novel or highly differentiated biologics to treat diseases with significant unmet medical needs, particularly cancers and autoimmune disorders, reported that the first patient was dosed with TJC4 in a Phase 1/2a clinical trial evaluating its use in treating patients with relapsed or refractory acute myeloid leukemia (r/r AML) or myelodysplastic syndrome (MDS) in China (Press release, I-Mab Biopharma, APR 2, 2020, View Source [SID1234556100]). TJC4, also known as TJ011133, is an internally developed differentiated anti-CD47 monoclonal antibody that is designed to minimize inherent binding to normal red blood cells while preserving its strong anti-tumor activity.

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The clinical trial in China is a multi-center, open-label, single-arm study to evaluate the safety, tolerability, pharmacokinetics/pharmacodynamics and preliminary efficacy of TJC4 as a monotherapy in patients with r/r AML and MDS (CXSL1900039; NCT04202003;). I-Mab is also conducting a Phase 1 trial (NCT Number: NCT03934814) in the U.S. in patients with advanced solid tumors and lymphoma, with complete data from the dose escalation portion expected in the third quarter of 2020.

"We have been at full speed conducting clinical development of TJC4 in both U.S. and China," said Dr. Joan Shen, CEO of I-Mab. "Dosing the first patient in China represents another important milestone, both for our TJC4 program and for patients, as TJC4 stands out as a globally competitive, highly differentiated anti-CD47 asset for cancer treatment. Together with the Phase 1 dose escalation study of TJC4 in the U.S., we continue to leverage our clinical development capabilities and expertise to facilitate clinical validation of TJC4 in China and the U.S."

About CD47 and TJC4

CD47 is a glycoprotein over-expressed in a wide variety of cancers and delivers a "don’t eat me" signal to tumor-engulfing macrophages through its ligand SIRPα, and is one of the most promising immuno-oncology targets after PD-1 and PDL1.

TJC4 is a differentiated anti-CD47 monoclonal antibody and designed to minimize inherent binding to normal red blood cells by this class of monoclonal antibodies yet preserve its strong anti-tumor activities. Blockade of CD47 by TJC4 enables macrophages to engulf cancer cells. TJC4 recognizes a unique epitope on CD47 and exhibits minimal binding to red blood cells. The hematologic safety advantage of TJC4 has been demonstrated in a series of robust pre-clinical and toxicological studies including those in cynomolgus monkeys, while it maintains superb anti-tumor activities.

On April 2, 2020-Helix BioPharma Corp. (TSX: HBP) ("Helix" or the "Company"), an immuno-oncology company developing innovative drug candidates for the prevention and treatment of cancer, reported a corporate update (Press release, Helix BioPharma, APR 2, 2020, View Source [SID1234556099]).

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COVID-19
As countries across the globe face an unprecedented public health crisis due to COVID-19, Helix has taken various measures to protect the health and well-being of our staff while maintaining business continuity.

The office and laboratory of Helix in Canada have been following the recommendations of federal, provincial and regional authorities. This includes operating a safe and clean working environment, providing work at home facility and observing latest public health guidance such as ‘social distancing’. Except for a limited number of staff at the laboratory to maintain critical infrastructure operation, all Helix personnel are working from remote locations.

Clinical Development initiatives The Company’s U.S. Phase I L-DOS47 lung cancer study in combination with pemetrexed and carboplatin (LDOS001) has completed patient recruitment. Study reports are being compiled and an abstract has been submitted to a conference. As previously reported in the Company’s recently filed Q2 fiscal 2020 filings, the Company’s European Phase II L-DOS47 lung cancer study in Poland and Ukraine (LDOS003) no longer requires patient enrollment. The Company indicated that the first stage of the study related to dose escalation would be concluded and progression to the second stage of the study would only proceed if a third-party was willing to partner with the Company on the study and upon a confirmatory medical review.

The Company’s U.S. Phase 1b/II pancreatic study of L-DOS47 in combination with doxorubicin (LDOS006) continues to enroll patients and has dosed two patients. Given the COVID-19 crisis however, the Company expects patient enrollment will be impacted. Helix is working closely with the clinical trial site and the hospital to ensure best care is being provided to patients while ensuring Helix complies with their COVID-19 protocols.

Corporate initiatives COVID-19 has created significant uncertainty and has materially impacted equity markets globally at a time when the Company was in the planning process of up-listing to the U.S. and raising additional capital. Nevertheless, the Company continues to engage both U.S. and Canadian investment bankers and is preparing for a capital raise in combination with an up-listing on the NASDAQ. Though the Company previously expected to conduct road shows and face-to-face meetings with potential investors during the month of April 2020, COVID-19 has made it impossible to do so. Instead, the Company expects to conduct online meetings and conference calls.

The Company recently closed a $6,000,000 private placement which included the disposition of a 15.5% stake in the Company’s Polish subsidiary, Helix Immuno-oncology S.A. ("HIO"). As previously disclosed, the Company intends to fully divest its remaining 51.0% interest in HIO to raise additional capital to further fund the Company’s clinical development programs while retaining a licensing arrangement for future royalties and milestone payments.

As a result of the recent private placement the Company is in a sound financial position allowing management to operate as normally as possible, in light of COVID-19. Given that the duration and magnitude of the impact on the economy and the Company’s business from COVID-19 is unknown, the Company has implemented a review of 2 operations in order to reduce or defer spending where possible, while maintaining key clinical and business program priorities.

The Company thanks all its staff who responded quickly and professionally as well as all our stakeholders who continue to support our Company during these challenging times.

On April 2, 2020 Kite, a Gilead Company (Nasdaq: GILD), and Teneobio, Inc. reported the companies have entered into a license and collaboration agreement through which Kite will receive exclusive rights to certain antibodies directed to B-cell maturation antigen (BCMA) (Press release, Kite Pharma, APR 2, 2020, View Source [SID1234556098]). The fully human variable heavy chain of one such antibody is currently undergoing clinical evaluation in a chimeric antigen receptor (CAR) format for the treatment of patients with multiple myeloma in a Phase 1 clinical trial at the National Cancer Institute. Kite and Teneobio will also collaborate on the discovery of antibodies directed to four additional targets, using Teneobio’s proprietary Human Heavy-Chain Antibodies (UniAb) platform, to be used in CAR T cell therapies for multiple myeloma and other cancers.

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This press release features multimedia. View the full release here: View Source

CARs traditionally utilize an antigen-recognition domain made up of a single chain variable fragment (scFv). The binding domain derived from Teneobio’s BCMA antibody has key potential benefits, including minimal potential for immunogenicity due to its lack of murine elements, and its small size, which may allow for the creation of dual-targeting CAR T therapies.

"Kite is committed to pursuing novel CAR T therapies that have the potential to be meaningfully differentiated treatment options for people living with multiple myeloma," said Peter Emtage, PhD, Senior Vice President of Research at Kite. "Based on encouraging early clinical signals and unique attributes of the binding domain from Teneobio, this exciting collaboration will be central to our strategy of developing next-generation CAR T therapies with the potential to overcome the tumor microenvironment."

"We are pleased that Kite, a leader in cell therapy, has recognized the potential of Teneobio’s UniAb platform to enable differentiated CAR T cell therapies in treating multiple myeloma and other cancers," said Roland Buelow, PhD, CEO of Teneobio. "We look forward to working with Kite to advance our uniquely differentiated cancer-targeting UniAbs on behalf of patients in need of novel therapies."

Under the terms of the agreement, Teneobio will receive an upfront payment and will be eligible to receive additional payments based on achievement of certain clinical and regulatory milestones, as well as royalties on future potential sales.

The Anti-BCMA CAR T cell therapy using Teneobio’s UniAb platform is investigational and not yet approved by any regulatory authority. Its efficacy and safety have not been established.

On April 2, 2020 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported a global collaboration and option agreement with Janssen Biotech, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson (Press release, Fate Therapeutics, APR 2, 2020, View Source [SID1234556097]).

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Under the multi-year collaboration agreement, Janssen will contribute proprietary antigen binding domains for up to four tumor-associated antigen targets. The Company will apply its iPSC product platform to research and preclinically develop new iPSC-derived chimeric antigen receptor (CAR) NK and CAR T-cell product candidates. The Company will receive $50 million in cash and $50 million from the purchase by Johnson & Johnson Innovation – JJDC, Inc. of newly issued shares of the Company’s common stock at a price per share of $31.00. Janssen will also reimburse the Company for all activities conducted under the collaboration.

"We are delighted to enter this strategic collaboration, which brings together Janssen’s scientific and global commercialization leadership, deep domain expertise in oncology and proprietary technologies for targeting and binding certain tumors and our industry-leading iPSC product platform to develop novel off-the-shelf CAR NK and T-cell cancer immunotherapies," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "The collaboration strengthens our financial and operating position through a focused effort of developing cell-based cancer immunotherapies utilizing Janssen’s proprietary antigen binding domains, while enabling us to continue to exploit our deep pipeline of wholly-owned product candidates and further develop our off-the-shelf, iPSC-derived cell-based immunotherapies."

The Company will advance candidates under the collaboration to the filing of an Investigational New Drug (IND) application, after which Janssen will have the right to exercise its option for an exclusive license for the development and commercialization of collaboration candidates targeting the tumor-associated antigens. The Company will be primarily responsible for the manufacture of collaboration candidates, the cost of which will be paid for by Janssen. The Company is eligible to receive payments of up to $1.8 billion upon the achievement of development and regulatory milestones and up to $1.2 billion upon the achievement of commercial milestones, plus double-digit royalties on worldwide commercial sales of products targeting the antigens. In addition, the Company has the right to elect to co-commercialize each collaboration candidate in the U.S. and share equally in profits and losses in the U.S., subject to its payment of certain clinical development costs and adjustments in milestone and royalty payments.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 250 issued patents and 150 pending patent applications.