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.

On May 15, 2017 Vical Incorporated (Nasdaq:VICL) reported financial results for the three months ended March 31, 2017 (Press release, Vical, MAY 15, 2017, View Source [SID1234519128]). Net loss for the first quarter of 2017 was $2.8 million, or $0.25 per share, compared with a net loss of $2.4 million, or $0.26 per share, for the first quarter of 2016. Revenues for the first quarter of 2017 were $3.2 million, compared with revenues of $4.6 million for the first quarter of 2016, reflecting revenues from Astellas Pharma Inc. for manufacturing services performed under our ASP0113 collaborative agreements.

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Vical had cash and investments of $39.2 million at March 31, 2017. The Company’s net cash burn for the first quarter of 2017 was $1.8 million, which was consistent with the Company’s full year guidance of between $8 million and $11 million.

Program updates include:

ASP0113 CMV Therapeutic Vaccine

The multinational Phase 3 registration trial in HCT recipients completed enrollment in September 2016 with a total of 515 subjects. Dosing in the trial was completed in April 2017 and the follow-up period is expected to be finished in September 2017. The primary endpoint of the trial is a composite of overall mortality and CMV end organ disease which will be assessed one year after transplantation. Astellas expects top-line data to be available in the first quarter of 2018. Vical and Astellas continue to make progress towards a potential BLA filing in 2018.
VCL-HB01 HSV-2 Therapeutic Vaccine

Recruitment into the Phase 2 trial of the VCL-HB01 HSV-2 therapeutic vaccine has been completed with a total of 261 subjects enrolled at 15 U.S. clinical sites. VCL-HB01 is formulated with Vaxfectin and encodes two full-length HSV-2 antigens gD and UL46, and is designed to reduce recurrences in patients with symptomatic genital HSV-2 infection. Healthy adult subjects, 18 to 50 years of age, have been randomized 2:1 to receive either vaccine or placebo to evaluate the efficacy and safety of the vaccine. The primary endpoint of the study is annualized lesion recurrence rate which is a clinically meaningful endpoint for both patients and treating physicians as it provides important information on the number of recurrences over time in this chronic disease setting. Vical expects to deliver top-line results during the second quarter of 2018.
VL-2397 Antifungal

The VL-2397 development program will be featured in four poster presentations and an oral presentation at the June ASM Microbe meeting in New Orleans.
Vical has completed its first-in-human Phase 1 trial of its novel antifungal, VL-2397. The randomized, double-blind, placebo-controlled trial was designed to evaluate safety, tolerability and pharmacokinetics of single and multiple ascending doses of intravenous VL-2397 in 96 healthy volunteers. Results point to a favorable safety and pharmacokinetic profile for VL-2397. The data will be highlighted in one of the four poster presentations at ASM Microbe.
Vical plans to conduct a Phase 2 efficacy study to evaluate VL-2397 for the treatment of invasive aspergillosis and is working with clinical experts and the FDA towards this objective. The FDA has granted Vical Qualified Infectious Disease Product (QIDP), Orphan Drug and Fast Track designations to VL-2397 for the treatment of invasive aspergillosis. Under the QIDP designation Vical has been able to interact intensively with the FDA on the design of the Phase 2 trial and in exploring an expedited development pathway for VL‑2397.
Invasive aspergillosis represents a major unmet medical need given the high mortality rate in immunocompromised patients, despite availability of current antifungal therapies.
Vical will conduct a conference call and webcast today, May 15, at noon Eastern Time, to discuss the Company’s financial results and program updates with invited participants. The call and webcast are open on a listen-only basis to any interested parties. To listen to the conference call, dial in approximately ten minutes before the scheduled call to (719)325-2361 (preferred), or (888)466-4462 (toll-free), and reference confirmation code 9047479. A replay of the call will be available for 48 hours beginning about two hours after the call. To listen to the replay, dial (719)457-0820 (preferred) or (888)203-1112 (toll-free) and enter replay passcode 9047479. The call will also be available live and archived through the events page at www.vical.com. For further information, contact Vical’s Investor Relations department by phone at (858)646-1127 or by e-mail at [email protected].

On May 15, 2017 Sophiris Bio Inc. (NASDAQ: SPHS) (the "Company" or "Sophiris"), a late-stage clinical biopharmaceutical company developing topsalysin (PRX302) as a first-in-class treatment for urological diseases, reported that successful data from its Phase 2a proof-of-concept study of topsalysin, which evaluated the drug as a focal treatment for localized prostate cancer, will be presented today as a poster at the 112th AUA meeting (Press release, Sophiris Bio, MAY 15, 2017, View Source [SID1234519127]).

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Abstract Title: "Intra-Prostatic Injection of PRX302 Focal Therapy in Treating Clinically Significant Low-Intermediate Risk Prostate Cancer: An Open Label, Proof-of-Concept Study"
Presenting Author: Edward J. Bass, University College London, London, UK
Poster Session: MP70: Prostate Cancer: Localized: Ablative Therapy 1
Time: May 15, 2017 from 7:00 a.m. – 9:00 a.m. ET

The poster will be available on the Company’s website at www.sophirisbio.com.

On May 15, 2017 Cleveland BioLabs, Inc. (NASDAQ:CBLI) reported financial results and development progress for the first quarter ended March 31, 2017 (Press release, Cleveland BioLabs, MAY 15, 2017, View Source [SID1234519125]).

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Cleveland BioLabs reported a net loss of $(1.7) million, excluding minority interests, for the first quarter of 2017, or $(0.15) per share, compared to a net loss, excluding minority interests, of $(0.7) million, or $(0.06) per share, for the same period in 2016. The increase in net loss was primarily due to an increase in the non-cash adjustment to our warrant liabilities and decreased revenues and expenses due to the completion of our development contracts with the Russian Federation Ministry of Industry and Trade ("MPT"), which was partially offset by reduced operating costs aligned with our streamlined focus primarily on pursuing a pre Emergency Use Authorization ("pre-EUA") with the U.S. Food and Drug Administration ("FDA") and a Marketing Authorization Application ("MAA") with the European Medicines Agency ("EMA") for entolimod as a medical radiation countermeasure.

As of March 31, 2017, the Company had $13.1 million in cash, cash equivalents and short-term investments, which, based on the Company’s current operational plan, is estimated to fund operations for at least one year beyond the filing date of our Form 10-Q.

Yakov Kogan, Ph.D., MBA, Chief Executive Officer, stated, "The pursuit of commercialization for entolimod as a medical radiation countermeasure remains our top priority. As previously announced, we are excited to have received agreement from the FDA to commence the in vivo biocomparability study in non-human primates. Following completion of this study and discussion of the study results with the FDA, we expect the agency to resume the review of our pre-EUA dossier."

"We are also excited to have received a positive opinion from EMA on our pediatric investigational plan and are diligently working on assembling our MAA for submission to EMA," added Dr. Kogan.


Other Operational Highlights

• Entolimod Oncology Indications. We have completed dosing of 40 patients in a clinical study of the safety and tolerability of entolimod as a neo-adjuvant therapy in treatment-naïve patients with primary colorectal cancer who are recommended for surgery. This study was conducted in Russia and partially funded by the development contract with the MPT. The goal is to accumulate additional clinical data regarding immune cell response to administrations of entolimod to guide future oncology development. The analysis of the data is ongoing.

• CBLB612 is a synthetic molecule that activates the Toll-like heterodimeric receptor 2/6 and stimulated white blood cell generation in preclinical studies. Recently we have completed dosing in a Phase 2, randomized, placebo-controlled clinical study of CBLB612 as myelosuppressive prophylaxis in patients with breast cancer receiving doxorubicin-cyclophosphamide chemotherapy and data analysis of this study is in progress.

• Mobilan is a recombinant non-replicating adenovirus that directs expression of TLR5 and its agonistic ligand, a secretory non-glycosylated version of entolimod we are also developing through our subsidiary, Panacela Labs, Inc. Two randomized, placebo-controlled, dose-ranging studies of Mobilan in men with prostate cancer are currently ongoing in the Russian Federation.


Further Financial Results

Revenue for the first quarter of 2017 decreased to $0.6 million compared to $0.8 million for the first quarter of 2016. The net decrease was primarily attributable to reduced revenue from our development contracts with MPT which completed in 2016. This decrease was partially offset by increased revenue from our Joint Warfighter Medical Research Program ("JWMRP") contract from the Department of Defense ("DoD") for the continued development of the entolimod as a medical radiation countermeasure.

Research and development costs for the first quarter of 2017 decreased to $1.4 million compared to $1.9 million for the first quarter of 2016. The reduction in research and development costs is due to completion of our development contract with MPT and was offset, in part, by continued preclinical development along with other drug manufacturing activities associated with our JWMRP contract.

General and administrative costs for the first quarter of 2017 decreased to $0.8 million compared to $1.2 million for the first quarter of 2016. This decrease was primarily attributable to reductions in personnel and other operating costs in connection with cost savings efforts to streamline operations.