On May 15, 2017 Zymeworks Inc. ("Zymeworks") (NYSE: ZYME; TSX: ZYME) a clinical-stage biopharmaceutical company dedicated to the discovery, development and commercialization of next-generation multifunctional biotherapeutics, initially focused on the treatment of cancer, reported financial results for the first quarter ended March 31, 2017 (Press release, Zymeworks, MAY 15, 2017, View Source [SID1234519134]).

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"During the first quarter, among other highlights, we received top-line data from the dose escalation stage of our on-going Phase 1 clinical trial of our lead product candidate, ZW25," said Ali Tehrani, Ph.D., Zymeworks’ President & CEO. "ZW25 has demonstrated preliminary anti-tumor activity across multiple cancer types in HER2 expressing patients who have progressed after several lines of treatment with HER2-targeted therapies."
First Quarter Highlights:
Preliminary data for ZW25 phase 1 clinical trial received
Orphan drug designation for ZW25 in gastric cancer received
Second research milestone with collaborator Eli Lilly announced
Two industry veterans added to Board of Directors
State-of-the-art lab facility opened

On May 3, 2017, subsequent to the first quarter, Zymeworks completed its initial public offering and sold 4,500,000 common shares at a price of $13.00 per share. In addition, Zymeworks has granted the underwriters an option, exercisable within 30 days of the date of its final prospectus relating to the IPO, to purchase up to an additional 675,000 common shares.

First Quarter Ended March 31, 2017 Financial Results
All amounts are in U.S. dollars. Zymeworks’ unaudited condensed consolidated financial statements are prepared in accordance with accounting principals generally accepted in the United States ("U.S. GAAP").

Revenues for the first quarter ended March 31, 2017 were $0.2 million compared to $0.3 million for the same period of 2016. The decrease in collaboration revenue of $0.1 million is due to a $0.3 million decrease in research support payments from Merck, which was partially offset by the increase in research support payments of $0.2 million from Daiichi.

Research and development expenditures for the first quarter ended March 31, 2017 were $9.1 million, compared to $7.9 million for the same period in 2016. The increase of $1.2 million, was primarily due to increased activities associated with our therapeutic platforms and early-stage research and discovery programs, recorded in other research activities.

General and administrative expenses in the first quarter ended March 31, 2017 were $6.3 million compared to $2.1 million for the same period in 2016. General and administrative expenses increased by $4.2 million, compared to the same period in 2016, primarily due to an increase in professional fees and compensation costs. The compensation cost increase was the result of higher share-based compensation expenses due to reclassification under U.S. GAAP of certain awards from equity to liability for accounting purposes as well as certain new hires. The increase in professional fees over the same period in 2016 was associated with consulting services as well as legal, intellectual property, assurance and taxation services.

Net loss for the three months ended March 31, 2017 was $15.9 million. Zymeworks expects that over the next several years, research and development expenditures will increase in connection with the ongoing development of product candidates and other clinical, preclinical and regulatory activities.

As of March 31, 2017, Zymeworks had $26.8 million in cash and cash equivalents and short-term investments, as well as $1.9 million in SR&ED and IRAP receivables. On May 3, 2017, subsequent to the first quarter, Zymeworks completed its initial public offering for a total of $58.5 million in aggregate gross proceeds.

On May 15, 2017 The biopharmaceutical company MOLOGEN AG (ISIN DE0006637200; Frankfurt Stock Exchange Prime Standard: MGN) reported that it has achieved the recruitment goal with the inclusion of currently 540 patients for the pivotal IMPALA study in metastatic colorectal cancer "mCRC" (Press release, Mologen, MAY 15, 2017, View Source [SID1234519133]). The aim of this study is to show that a switch maintenance therapy with the immunotherapeutic agent lefitolimod can lead to a prolongation of overall survival in patients with mCRC. With the finalization of recruitment, the company reached the next important milestone in the clinical study program of its lead product lefitolimod.
"We are pleased that the enrollment for IMPALA has been completed and that treatment with lefitolimod is so far well tolerated", said Dr. Matthias Baumann, Chief Medical Officer of MOLOGEN AG. "This is an important milestone for us as the pivotal IMPALA study is by far the largest study for us to date. We look forward to the study results in this high medical need indication, which will be available, once sufficient survival data have been collected. This is currently expected in 2019, which of course depends on the actual overall survival rates.

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Approximately 540 patients from more than 120 centers in eight European countries, including the five most important European pharmaceutical markets, participate in the study. The study will be evaluated once a certain number of deaths (events) have occurred, which is currently estimated to be reached around two years after completion of patient enrollment. MOLOGEN is continuously and closely monitoring the events and the corresponding timeline for the results.

Colorectal cancer
Colorectal cancer is one of the most common cancers worldwide with 1,361,000 cases in 2012. In the same year it accounted for approximately 694,000 deaths worldwide. In the US 134,000 cases have occurred in 2012, and 55,000 people have died from this disease.
About IMPALA
IMPALA (Immunomodulatory MGN1703 in Patients with Advanced Colorectal Carcinoma with tumor reduction during induction treatment) is a pivotal, randomized, international, multicenter, open-label phase III trial. The study aims to prove that a switch maintenance therapy with an active immunotherapy with lefitolimod leads to an increased overall survival of patients who have achieved a response during their first line treatment of metastatic colorectal cancer. The primary endpoint is overall survival and secondary study endpoints include progression-free survival, safety and tolerability, as well as Quality of Life (QoL).

The study is conducted in collaboration with three renowned national study groups: Arbeitsgemeinschaft Internistische Onkologie (AIO) in Germany, Grupo Españiol de Tratamiento de Tumores Digestivos (TTD) in Spain and Groupe Coopérateur Multidisciplinaire en Oncologie (GERCOR) in France. The steering committee consists of internationally recognized medical experts including Prof. David Cunningham, MD, Department of Medicine and Director of Clinical Research, Royal Marsden Hospital, London, UK, as coordinating investigator

On May 15, 2017 Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the discovery and development of medicines to control the expression of disease-driving genes, reported that the first patient has been dosed in the Phase 1 clinical trial of SY-1365, its first-in-class selective cyclin-dependent kinase 7 (CDK7) inhibitor, in patients with advanced solid tumors, including transcriptionally dependent cancers such as triple negative breast, small cell lung and ovarian cancers (Press release, Syros Pharmaceuticals, MAY 15, 2017, View Source [SID1234519132]).

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"SY-1365 represents a promising new approach for treating a number of aggressive cancers that have eluded treatment with other targeted approaches," said Anthony W. Tolcher, M.D., Director of Clinical Research at South Texas Accelerated Research Therapeutics (START), and a clinical investigator in the trial. "Certain cancers are particularly dependent on high expression of transcription factors for their growth and survival, and SY-1365 has shown substantial anti-tumor activity in preclinical models of these cancers. We’re pleased to have enrolled the first patient in this clinical trial and look forward to further investigating SY-1365 for patients with these difficult-to-treat solid tumors."

SY-1365 has shown significant anti-proliferative and pro-apoptotic activity in multiple preclinical models of difficult-to-treat solid tumors, including triple negative breast, small cell lung and ovarian cancers. SY-1365 has induced anti-tumor activity in both cell line-derived xenograft and patient-derived xenograft models of triple negative breast cancer, including complete regressions at a twice weekly dosing regimen consistent with the initial regimen being used in the Phase 1 clinical trial. In preclinical models, SY-1365 has also been shown to preferentially kill cancer cells over non-cancerous cells and can lower the expression of oncogenic transcription factors, including MYC.

"Patients with triple negative breast, small cell lung and ovarian cancers, as well as other transcriptionally dependent cancers, are in dire need of better treatment options," said David A. Roth, M.D., Chief Medical Officer of Syros. "Based on the strong preclinical data, we believe SY-1365 could provide a meaningful benefit for patients with these cancers. We have designed our Phase 1 trial to efficiently assess early proof of mechanism during the dose escalation phase and early anti-tumor activity by focusing the expansion phase of the trial on a set of transcriptionally dependent tumors that are most sensitive to CDK7 inhibition and for which early anti-tumor activity may be observed."

The Phase 1 trial is a multi-center, open-label trial that is expected to enroll approximately 70 patients with advanced solid tumors, including expansion cohorts focused on transcriptionally dependent cancers. The primary objective of the trial is to assess the safety and tolerability of escalating doses of SY-1365, with the goal of establishing a maximum tolerated dose and a recommended Phase 2 dose and regimen. The dose-escalation phase will be open to solid tumor patients for whom standard curative or palliative measures do not exist or are no longer effective. Following the dose-escalation phase, expansion cohorts are planned to further evaluate the safety and anti-tumor activity of SY-1365 in patients with triple negative breast, small cell lung and ovarian cancers, to confirm a recommended Phase 2 dose and regimen, and to enroll patients with tumors of any histology in a cohort focused on analyzing biopsied tumor tissue. Syros plans to expand future clinical development of SY-1365 into acute leukemias based on the data generated in this trial. Additional details about the trial can be found using the identifier NCT03134638 at www.clinicaltrials.gov.

On May 15, 2017 Fate Therapeutics, Inc. (NASDAQ:FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported business highlights and financial results for the quarter ended March 31, 2017 (Press release, Fate Therapeutics, MAY 15, 2017, View Source [SID1234519131]).

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"This has been an exciting quarter for us as we have successfully launched our multi-pronged clinical development strategy for FATE-NK100 across both hematologic and solid tumor malignancies. We also continue to be encouraged by investigator enthusiasm for ProTmune and look forward to sharing key clinical data in mid-2017 from the safety stage of our PROTECT study," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "Additionally, we are very pleased with the constructive feedback we received from regulatory authorities in both the United States and the United Kingdom regarding our specific manufacturing and development plans for our first-of-kind cell products derived from master pluripotent cell lines. These formal meetings confirmed our alignment with key regulatory agencies in bringing our off-the-shelf iPSC-derived cell products into human clinical trials."

Recent Highlights & Program Updates

Launched First-in-Human Clinical Trial of FATE-NK100. An investigator-initiated clinical trial of FATE-NK100, the Company’s first-in-class adaptive memory natural killer (NK) cell product candidate, was opened for patient enrollment at the Masonic Cancer Center, University of Minnesota (UMN) for the treatment of refractory or relapsed acute myelogenous leukemia. The VOYAGE study is utilizing accelerated dose-escalation to evaluate the safety and determine the maximum dose of a single intravenous infusion of FATE-NK100. The anti-tumor activity of FATE-NK100, including rates of complete response, clearance of minimal residual disease, disease-free survival and overall survival, will also be assessed.

Secured FDA Clearance of IND for FATE-NK100 in Advanced Solid Tumors. In May 2017, the U.S. Food and Drug Administration (FDA) cleared the Company’s investigational new drug (IND) application for the clinical investigation of FATE-NK100, including in combination with monoclonal antibody therapy, in subjects with advanced solid tumor malignancies. The Company plans to enroll subjects in the DIMENSION study concurrently across three FATE-NK100 treatment arms: as monotherapy for solid tumor malignancies, including small cell lung cancer and hepatocellular carcinoma; in combination with trastuzumab for advanced HER2+ cancers, including breast and gastric cancers; and in combination with cetuximab for advanced EGFR1+ cancers, including colorectal and head and neck cancers. Accelerated dose-escalation will be utilized to evaluate the safety and anti-tumor activity of FATE NK100 in an outpatient setting.

Conducted Formal Regulatory Engagements with FDA and MHRA for hnCD16-iNK Cell Product Candidate. Fate Therapeutics held a Pre-IND meeting with the FDA and a Scientific Advice meeting with the UK Medicines and Healthcare products Regulatory Agency (MHRA) in March and April 2017, respectively, to support the clinical translation of its induced pluripotent stem cell (iPSC)-derived NK cell products. These formal meetings reviewed the Company’s preclinical development, proposed manufacturing plans and clinical trial design for its first-of-kind NK cell product candidate, an off-the-shelf targeted cancer immunotherapy derived from an engineered iPSC line. Based on these interactions, the Company expects to file applications with both regulatory authorities within the next twelve months to conduct a first-in-human clinical trial for the treatment of cancer. In February 2017, Fate Therapeutics and UMN expanded their collaboration to initiate clinical translation of the Company’s iPSC-derived NK cell products, including its off-the-shelf targeted hnCD16-iNK cell product candidate derived from a master iPSC line engineered to express a proprietary high-affinity, non-cleavable CD16 receptor (hnCD16).

Granted Foundational iPSC Manufacturing Patent. In March 2017, the U.S. Patent and Trademark Office issued U.S. Patent No. 9,593,311 which protects cellular compositions comprising iPSCs and a WNT pathway activator, such as a GSK3 inhibitor. This latest issuance, which expires in 2029, continues to extend the Company’s dominant U.S. patent position covering OCT4-based cell reprogramming, including gene expression vectors and cell compositions necessary for generating iPSCs. The newly-patented compositions are critical for selecting and expanding iPSC clones and for maintaining clonal populations in a state of pluripotency, both of which are required to create master pluripotent cell lines for the manufacture of homogeneous cell products. The patent, which is owned by the Whitehead Institute for Biomedical Research and licensed exclusively to the Company for all therapeutic purposes, adds to the Company’s significant iPSC intellectual property portfolio of over 90 issued patents and 100 pending patent applications.
First Quarter 2017 Financial Results

Cash & Short-term Investment Position: Cash, cash equivalents and short-term investments as of March 31, 2017 were $82.3 million compared to $92.1 million as of December 31, 2016. The decrease was primarily driven by the Company’s use of cash to fund operating activities and to service principal and interest obligations under its loan agreement with Silicon Valley Bank.

Total Revenue: Revenue was $1.0 million for the first quarter of 2017 compared to $1.3 million for the comparable period in 2016. All revenue was derived from the Company’s research collaboration and license agreement with Juno Therapeutics.

Total Operating Expenses: Total operating expenses were $11.0 million for the first quarter of 2017 compared to $9.2 million for the comparable period in 2016. Operating expenses for the first quarter of 2017 included $0.9 million of stock compensation expense, compared to $0.8 million for the comparable period in 2016.

R&D Expenses: Research and development expenses were $8.0 million for the first quarter of 2017 compared to $6.6 million for the comparable period in 2016. The increase in R&D expenses was primarily related to an increase in third-party service provider fees to support the clinical development of ProTmune and FATE-NK100 and the preclinical advancement of the Company’s off-the-shelf iPSC-derived cellular immunotherapy programs.

G&A Expenses: General and administrative expenses were $3.0 million for the first quarter of 2017 compared to $2.6 million for the comparable period in 2016. The increase in G&A expenses was primarily related to an increase in intellectual property-related expenses.

Shares Outstanding: Common shares outstanding as of March 31, 2017 and December 31, 2016 were 41.4 million. Preferred shares outstanding as of March 31, 2017 and December 31, 2016 were 2.82 million, each of which is convertible into five shares of common stock. All preferred shares outstanding relate to the Company’s sale and issuance of 2.82 million shares of non-voting Class A convertible preferred stock to Redmile Group, LLC in November 2016.

On May 15, 2017 Moleculin Biotech, Inc. (NASDAQ: MBRX) ("Moleculin" or the "Company"), a preclinical pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center ("MD Anderson"), reported its financial and operating results for the first quarter ended March 31, 2017 and other recent developments (Press release, Advaxis, MAY 15, 2017, View Source [SID1234519112]).

First Quarter & Recent Highlights
Annamycin
Appointed Theradex Systems, Inc. as its contract research organization ("CRO") for its planned Phase I/II clinical trial for Annamycin for the treatment of relapsed or refractory acute myeloid leukemia ("AML").
Received Orphan Drug Designation by the U.S. Food and Drug Administration ("FDA") for the treatment of AML. The FDA grants orphan drug designation to drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including tax credits for qualified clinical trial costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.
Recently filed the IND application for Annamycin, with a Phase I/II approach with the intent of increasing the Maximum Tolerable Dose ("MTD"). In subsequent discussions, the FDA requested certain revisions to the protocol, additional information, and additional data related to Chemistry, Manufacturing and Controls ("CMC"). The Company has the additional information, has made the requested revisions to the protocol, and is working on developing the CMC data. In the interim, Moleculin has withdrawn the IND application in order to resubmit it when the requested data are available. The Company believes that the resubmission of the IND application will occur in time for the IND to go into effect prior to the end of July 2017 and allow for clinical trials. However, if the Company is unable to obtain the required CMC data on a timely basis, it will be delayed in resubmitting its IND application, which will delay the commencement of the clinical trials for Annamycin beyond July 2017.
Updated the Annamycin clinical strategy to add a Phase I arm to its next Phase II trial that leverages a potential increase in the MTD, which could increase the chance for positive outcomes. The Company believes that it will be able to publicly announce results from its Phase I/II clinical trial sometime in 2018.
WP1066
An MD Anderson physician is sponsoring a study of WP1066 for the treatment of brain tumors. While the Company is not participating in and has no influence on the conduct of this study, we understand that the sponsoring physician has submitted an IND to the FDA and the IND is on hold until documentation of Good Manufacturing Process or GMP production of WP1066 can be presented to the FDA, which Moleculin has agreed to provide. The Company expects that the sponsor’s IND will move forward in 2017 and may produce publishable clinical results in 2018.
Physician-scientists at another major US cancer center have requested and Moleculin has agreed to supply them with WP1066 for testing in a potential grant-funded clinical trial for children with Diffuse Intrinsic Pontine Gliomas (DIPG), a rare and very aggressive form of brain tumor. Studies conducted at this center have suggested that DIPG may be particularly sensitive to the inhibition of the activated form of a cell-signaling protein called STAT3, a primary target of WP1066, and their studies have demonstrated significant anti-tumor activity of WP1066 in DIPG in vitro and in vivo tumor models.
Corporate
Announced the closing of an underwritten public offering of securities for net proceeds of approximately $4.5 million. Roth Capital Partners and National Securities Corporation acted as joint book-running managers. Subsequently, approximately $0.8 million of additional funds have been received through the exercise of associated warrants issued in the offering bringing the total net raised in excess of $5 million.
Announced that Drs. Sandra Silberman and Paul Waymack have joined the Company’s Scientific Advisory Board ("SAB"). The Company’s current SAB also includes Dr. Waldemar Priebe (Chair) and Dr. Madeleine Duvic.
Planned Activities and Upcoming Potential Milestones
Anticipated Milestone Potential Timeframe
Announcement that our IND for Annamycin has become effective and that we may begin clinical trials End of July 2017
IRB (Institutional Review Board) approvals and site initiations of various clinical sites participating in our Phase I/II clinical trial of Annamycin Second Half of 2017
Establishment of a new MTD for Annamycin Second Half of 2017
A clinician sponsored IND for WP1066 for treatment of adult brain tumors moving forward Second Half of 2017
Announcement of Phase II data for Annamycin 2018
Announcement of further benefits of our sponsored research agreement with MD Anderson 2018
Walter Klemp, Chairman and CEO of Moleculin, stated: "We remain focused on developing the CMC data needed to submit our IND to move forward with the FDA by the end of July and to allow for clinical trials to begin. Additionally, we are pleased to have Theradex Systems as our CRO for our planned Phase I/II clinical trial for Annamycin. As we transition from a preclinical to a clinical stage company, we will continue to provide updates on our upcoming key milestones. We believe we have sufficient funds to pursue our planned operations into the first quarter of 2018."
Unaudited Financial Results for the Quarter Ended March 31, 2017
Research and development (R&D) expense was $0.68 million and $0.02 million for the three months ended March 31, 2017 and 2016, respectively. The increase of approximately $0.66 million is mainly due to the Company becoming fully operational post its June 1, 2016 Initial Public Offering ("IPO"). The difference mainly consists of increases of $0.15 million in sponsored research and research consultants, $0.13 million in employee related costs, $0.14 million in manufacturing and stability costs associated with the Company’s IND application, $0.1 million in regulatory counsel, $0.07 million in costs associated with the Company’s licenses, and $0.07 million of other costs. This increased activity represents the Company’s efforts in obtaining Orphan Drug designation for Annamycin and its associated IND application with the FDA.
General and administrative ("G&A") expense was $0.85 million and $0.31 million for the three months ended March 31, 2017 and 2016, respectively. The expense increase of approximately $0.54 million is mainly due to the Company becoming fully operational post its June 1, 2016 IPO. Specifically, these increases were attributable to $0.25 million associated with added headcount and associated payroll costs, $0.23 million in legal, auditing, and accounting costs, and $0.06 million in other G&A costs.
The Company recorded a gain of $1.06 million in the first quarter of 2017 for the change in fair value on revaluation of its warrant liability associated with the warrants issued in conjunction with its stock offering on February 14, 2017. The Company is required to revalue certain of its 2017 warrants at the end of each reporting period and reflect in the statement of operations a gain or loss from the change in fair value of the warrant in the period in which the change occurred. A gain results principally from a decline in the Company’s share price during the period and a loss results principally from an increase in the Company’s share price.
During the period, the Company settled a previously incurred expense utilizing shares of its common stock with an attributed value of $3.00 per share. The gain of $0.15 million reflects the difference in the Company’s share price in the open market as of the settlement date and $3.00 per share.
Interest expense includes expense accrued on convertible promissory notes issued in 2015 and 2016 bearing interest at the rate of 8% per annum.
The net loss for the three months ended March 31, 2017 was $0.33 million, which included the non-cash gains mentioned above aggregating to $1.21 million. Excluding this amount, the net loss for the period was $1.54 million, which is an increase of $1.21 million over the previous years’ $0.33 million net loss. Included in both net loss numbers for the three months presented was $0.11 million and $0.00 million for the 2017 and 2016, respectively, in stock based compensation.
As of March 31, 2017, the Company had $8.88 million of cash and cash equivalents compared to $5.00 million at December 31, 2016. In February 2017, Moleculin completed a public offering of its common stock and warrants, pursuant to which it received approximately $4.5 million in net proceeds, after deducting underwriting discounts and commissions and estimated offering expenses. Additionally, during the three months ended March 31, 2017, $0.80 million in cash was received due to warrants being exercised. Cash used in operations was $1.39 million for the first quarter of 2017. The Company believes that its existing cash and cash equivalents as of March 31, 2017 continues to be sufficient to fund planned operations into the first quarter of 2018.