On March 28, 2018 Moleculin Biotech, Inc. (NASDAQ: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported its financial results for the year ended December 31, 2017 (Press release, Moleculin, MAR 28, 2018, View Source [SID1234525040]). Additionally, the Company announced potential upcoming milestones and recent corporate developments.

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Management Discussion
Walter Klemp, Chairman and CEO of Moleculin, said, "We continued to make significant progress in developing Moleculin’s distinctive cancer treatment technologies during 2017. We firmly believe that all three of our highly differentiated technologies have breakthrough potential in effectively treating various cancers. From those three core technologies, we now have six potential drug candidates, two of which we expect will commence clinical trials in 2018, with the possibility of a third before the end of the year.

"Our potentially disruptive technologies include Annamycin, a chemotherapy agent that is active against multidrug resistant tumor cells and has been designed to be non-cardio toxic (unlike currently approved drugs in this class); immuno-stimulating STAT3 inhibitors WP1066 and WP1732 that target glioblastoma and pancreatic cancer; and WP1122, an inhibitor of glycolysis that has been shown in preclinical testing to effectively block the energy supply required by cancer cells and effectively starves the cancer cells to death. Our diverse development portfolio gives Moleculin what I like to call multiple shots on goal."

The Company submitted an application in October 2017 for a Clinical Trial Authorization ("CTA") for Annamycin in Poland. Having met all the requirements, the Ethics Committee in Poland approved the Phase I/II trial of Annamycin for the treatment of relapsed or refractory acute myeloid leukemia ("AML") in December 2017. In March the Company received requests for and provided additional information to the Polish National Office. It expects a response from the Polish National Office in the first half of 2018 and at the earliest mid-April 2018. The start of clinical trials in Poland remains subject to confirmation and approval of the CTA by the Polish National Office. The Company can provide no assurance that it will receive such confirmation on a timely basis, if at all.

In addition, the Company continues to recruit and contract clinics both in the United States and Poland. In the U.S., the Company has one site — University Hospitals Cleveland Medical Center ("UHCMC") — recruiting patients and enrollment has begun. The Company can provide no assurance that it will continue enrollments or begin treatment on a timely basis, if at all.

Mr. Klemp continued, "Additionally, this past December we announced that a physician-sponsored Investigational New Drug ("IND") application for a Phase I trial of Moleculin’s WP1066 in patients with recurrent malignant glioma and brain metastasis from melanoma was allowed by the U.S. Food and Drug Administration ("FDA"). This will be our second drug in clinical trials. The trial will be conducted at the MD Anderson Cancer center to evaluate safety and efficacy. We believe WP1066 represents a new class of oncology drugs able to fight tumors on two fronts by directly inhibiting cell signaling supporting tumor activity, and independently stimulating a natural immune response. This constitutes a new approach to treating brain tumors and tumor metastasis to the brain.

"We also intend to request a clinical trial authorization in Poland for WP1220 for the topical treatment of Cutaneous T-Cell Lymphoma (CTCL), which we expect will become our third compound in clinical trials in 2018. WP1220 is one of our patented STAT3 inhibitors designed to be compatible with topical formulations and was selected based on its preclinical activity in CTCL cell lines and based on the need for better topical treatments for skin cancer.

"As we look ahead to 2019 and beyond, we are excited about a new molecule that we recently licensed from MD Anderson — WP1732 — that shares many of the same characteristics of WP1066, especially its ability to inhibit activated STAT3, which is widely considered a key transcription factor involved in the development and progression of tumors. WP1732 has demonstrated significantly different organ distribution in animal models, suggesting it could be especially well-suited to target systemic solid tumors including pancreatic cancer, one of the most deadly and difficult to treat.

"An important attribute of WP1732 is that it is more water-soluble than WP1066. So, while we have been focused on oral delivery of WP1066, WP1732 is ideally suited to intravenous ("IV") administration, which makes the delivery of the drug potentially more convenient and efficacious. We’ve already started the process of preparing the preclinical data necessary for an IND for WP1732 and we hope to have that preparation completed in 2018.

"I also want to acknowledge the outstanding Scientific Advisory Board that is part of the Moleculin brain trust. Waldemar Priebe, PhD., a Founder of Moleculin and the Company’s Founding Scientist, leads a team of world renown experts in various cancer fields that includes John Paul Waymack, MD; Elihu Estey, MD; and Jorge Cortes, MD. Together with our two Chief Medical Officers, Robert Shepard, MD (Annamycin), and Sandra Silberman, MD (New Products), their expertise and guidance have enabled us at Moleculin to successfully proceed in the development of our highly differentiated compounds. Our expectation is that 2018 will see significant progress in advancing our portfolio of unique cancer treatments," concluded Mr. Klemp.

Fourth Quarter Highlights and Recent Corporate Developments
Moleculin Announces Grant-Funded Collaboration to Expand Understanding of New Discovery – March 20, 2018, the Company announced it has entered into a collaboration with a team of scientists in Poland who have received a $300,000 research grant to expand the understanding of how Moleculin’s leading STAT3 inhibitor WP1066 and the Company’s newly discovered drug candidate, WP1732, create a blockade of transcription factor STAT3 leading to tumor cell death and immune-stimulating effects.

Moleculin Announces Pricing of $9 Million Registered Direct Offering – February 16, 2018, the Company announced that it has entered into a definitive agreement with institutional investors for a registered direct offering of securities with gross proceeds of approximately $9 million.

Moleculin Announces Breakthrough Discovery of a New Molecule for Cancer Treatment – February 15, 2018, the Company announced that, pursuant to its continued collaboration with MD Anderson it has developed and licensed what it believes, based on preclinical testing, is a major breakthrough in its effort to develop a new cancer treatment that selectively kills highly resistant tumors. Specifically, the Company has preclinical evidence to suggest it is capable of influencing a process known as ‘ubiquitination’ to block the activated form of STAT3, an important oncogenic transcription factor. The lead molecule resulting from this new discovery is called WP1732 and it not only appears to share the same key mechanistic properties with WP1066, it has markedly different organ distribution and its dramatically increased solubility makes it ideal for administration via standard IV injection. Importantly, preclinical testing has also shown that WP1732’s properties make it a promising candidate for treating pancreatic cancer.

Moleculin Announces Collaboration with Emory University to Develop Novel Treatment of Pediatric Brain Cancer – February 13, 2018, the Company announced it has entered into an agreement with Emory University to enable expanded cancer research on Moleculin’s WP1066 molecule for the possible treatment of medulloblastoma, a pediatric malignant primary brain tumor. Physician-scientists at Emory University and Children’s Healthcare of Atlanta have requested support to continue research aimed at the development of a novel treatment of medulloblastoma using WP1066 and Moleculin has agreed to supply them with a pure form of WP1066 for preclinical testing for the potential future treatment of patients with the disease. Emory studies so far have indicated that medulloblastoma may be particularly vulnerable to the ability of WP1066 to block the activated form of STAT3, a key signaling protein believed to contribute to the growth and survival of many tumors, including medulloblastoma.

Moleculin Announces Activity with Pancreatic Cancer Drug – February 7, 2018, the Company announced it has been able to show promising tumor suppression activity with its inhibitor of glycolysis, WP1122. The Company’s glycolysis inhibitors have shown a remarkable affinity for concentrating in the pancreas and has solid data showing the ability of WP1122 to inhibit pancreatic tumor growth in mice.

Leading Leukemia Experts Join Moleculin’s Science Advisory Board – January 17, 2018, the Company announced the expansion of its Science Advisory Board to include Drs. Jorge Cortes and Elihu Estey.

Jorge Cortes, M.D., is deputy chair and professor of medicine in the Department of Leukemia at MD Anderson Cancer Center where he directs the CML and AML Programs. Dr. Cortes received his medical degree in 1986 from the Universidad Nacional Autonoma de Mexico, and has been at MD Anderson since 1991. Dr. Cortes, whose clinical interest focuses on new drug development and the management of patients with myelodysplatic syndromes, acute and chronic leukemias, and myeloproliferative disorders, has authored over 900 peer-reviewed medical publications in top-tier journals including New England Journal of Medicine, Lancet Oncology, Lancet Hematology, Journal of Clinical Oncology, Leukemia, Blood and many others.

Elihu Estey, M.D., is a Professor of Medicine in the Division of Hematology at the University of Washington School of Medicine and a Full Member and Director of AML Clinical Research (non-transplant) Clinical Research Division, Fred Hutchinson Cancer Research Center. Dr. Estey has built a distinguished career in cancer research approaching 40 years of active clinical practice with AML patients, providing mentorships for many physicians that have risen to prominence in AML, lectured globally to professional audiences on cancer research and published more than 700 articles on hematologic malignancies, specifically on AML. Additionally, Dr. Estey serves on the European Leukemia Net (ELN) guidelines committee for AML and has served as an advisor for AML studies to the Oncology Drugs Advisory Committee ("ODAC") of the FDA.

Moleculin Expands Leukemia Development Portfolio with Immuno-Stimulating STAT3 Inhibitor – January 10, 2018, the Company announced it has expanded the Company’s development pipeline for the treatment of AML with an immuno-stimulating STAT3 inhibitor. Leading experts in the treatment of AML, Dr. Jorge Cortes and Dr. Sanjay Awasthi requested the Company to expand its clinical research to include WP1066, an immuno-stimulating agent and STAT3 inhibitor, to increase therapeutic options for AML patients. This would potentially be complementary and synergistic with Annamycin and existing first line treatments.

Moleculin Announces Polish Approval for Leukemia Clinical Trial – December 21, 2017, the Company announced that the Ethics Committee in Poland has approved the Company’s Phase I/II clinical trial of Annamycin for the treatment of relapsed or refractory AML.

Moleculin’s WP1066 Drug gets FDA Brain Tumor IND Clearance – December 5, 2017, the Company announced the physician-sponsored IND application for a Phase I trial of Moleculin’s drug WP1066 in patients with recurrent malignant glioma and brain metastasis from melanoma has been allowed by the FDA. WP1066 is the second of Moleculin’s drugs to enter clinical stage and represents a new class of oncology drugs able to fight tumors on two fronts by directly inhibiting cell signaling and independently stimulating a natural immune response. This IND was sponsored by Dr. Amy Heimberger, who will serve as the principal investigator for the Phase I trial at MD Anderson Cancer Center to evaluate safety and efficacy.

Moleculin Appoints Dr. Sandra Silberman as Chief Medical Officer – New Products – November 8, 2017, the Company announced the appointment of Dr. Sandra Silberman as Chief Medical Officer ("CMO") in charge of New Products.

Moleculin Announces MD Anderson has Filed an IND with the FDA on its Drug WP1066 for the Treatment of Brain Tumors – November 1, 2017, the Company announced that responses have been submitted to FDA requests for additional information relating to the physician-sponsored IND application to study WP1066 as a potential treatment for brain tumors.

Moleculin Requests Authorization from the Polish Government to Advance Annamycin – October 24, 2017, the Company announced that it has submitted its request for CTA in Poland which, if allowed, will enable a clinical trial to study Annamycin for the treatment of relapsed or refractory AML in Poland. This will be in addition to the previously announced allowance of Moleculin’s IND filing with the FDA.

Moleculin Announces 14 Qualified Clinical Sites Requesting Participation in Annamycin Trial – October 18, 2017, the Company announced that 14 qualified cancer clinics have requested to participate in its clinical trial to study Annamycin for the treatment of relapsed or refractory AML.

Moleculin Announces Strategic Collaboration to Develop Immuno-stimulating Drug – October 11, 2017, the Company announced that it has entered into an agreement to collaborate with the University of Bergen to expand research on WP1066 and early indications of a possible dual ability to increase immune system response to tumors while also suppressing tumor cell proliferation tumor cell and survival.

Moleculin Signs Agreement with First Hospital for Annamycin Trials – October 3, 2017, the Company announced it has entered into an agreement with the first of several hospitals desiring to become treatment sites for its clinical trial to study Annamycin for the treatment of relapsed or refractory AML.

Moleculin Announces FDA Approval of Annamycin IND – September 26, 2017, the Company announced that the FDA has allowed Moleculin’s IND for the study of Annamycin in relapsed or refractory AML to proceed. This allows Moleculin to begin clinical trials of Annamycin in the U.S.

Anticipated Milestone Potential Timeframe
Announcement that our IND for Annamycin has become effective and that we may begin clinical trials Accomplished
Initial IRB (Institutional Review Board) approvals and site initiations of various clinical sites participating in our Phase I/II clinical trial of Annamycin Accomplished and ongoing through Second Half of 2018
Establishment of a new RP2D for Annamycin Second Half of 2018
A clinician sponsored IND for WP1066 for treatment of adult brain tumors moving forward IND Accomplished; Trial expected to begin First Half of 2018
Announcement of initial Clinical Data for Annamycin trial 2018
Announcement of further benefits of our sponsored research agreement with MD Anderson Accomplished and Ongoing into 2019
Announce CTA for WP1220 for the treatment of cutaneous T-cell lymphoma (CTCL) 2018
Announce WP1122 and WP1732 move into preclinical work 2018
Announce the fourth drug approved for clinical trial 2019
Financial Results for the Year Ended December 31, 2017
Research and Development Expense. Research and development (R&D) expense was $4.5 million and $1.5 million for the years ended December 31, 2017 and 2016, respectively. The increase in R&D of approximately $3.0 million mainly represents an increase of approximately: $2.0 million associated with developing and testing drug product as we prepared for clinical trials; $0.4 million related to an increase in R&D headcount and associated payroll costs; $0.3 million for sponsored research and related expenses; and $0.3 million associated with license fees. The increase in R&D headcount mainly represents the associated costs of increasing the commitments of the Company’s part-time employees and the addition of a second Chief Medical Officer – New Products. These all are a reflection of the increased clinical and pre-clinical activity for its drug portfolio as compared to 2016.

General and Administrative Expense. General and administrative (G&A) expense was $4.1 million and $2.4 million for the years ended December 31, 2017 and 2016, respectively. The increase in G&A of approximately $1.7 million was mainly attributable to: (a) the increase in headcount and associated payroll costs, including additional stock-based compensation expense of $1.0 million; (b) approximately $0.4 million in legal, accounting, consulting, and other professional expenses; (c) $0.2 million in insurance expense; and (d) approximately $0.1 million in occupancy, office and other costs. These increases reflect the increase in support of the Company’s clinical activity described above as compared to 2016.

Net Loss. The net loss for the twelve months ended December 31, 2017 was $9.8 million, which included non-cash expenses of approximately $0.7 million, which was comprised almost completely of stock-based compensation.

Liquidity and Capital Resources
As of December 31, 2017, we had $7.7 million in cash. During 2017, via an equity offering in February of 2017 (the February 2017 Offering), the Company’s at-the-market issuance agreement (ATM), and the exercise of warrants associated with the February 2017 Offering, the Company issued 7.2 million shares of common stock and received $10.1 million in net proceeds. Subsequent to year-end the Company entered into a Purchase Agreement with certain Investors for the sale of 4,290,000 shares of its common stock at a purchase price of $2.10 per share. Concurrently with the sale of the common shares, pursuant to the Purchase Agreement, the Company also sold warrants to purchase 2,145,000 shares of common stock, which have an exercise price of $2.80 per share. This sale of common shares and warrants generated aggregate gross proceeds of approximately $9.0 million with net proceeds approximating $8.3 million (February 2018 Offering). The Company believes that its existing cash and cash equivalents as of December 31, 2017 along with the cash generated by the February 2018 Offering, will be sufficient to fund its planned operations into the first quarter of 2019. Such plans are subject to change depending on clinical enrollment and regulatory progress and the use and supply of drug product.

On March 28, 2018 Molecular Templates, Inc. (Nasdaq:MTEM) ("Molecular"), a clinical-stage oncology company focused on the discovery and development of the company’s proprietary engineered toxin bodies (ETBs), which are differentiated, targeted, biologic therapeutics for cancer, reported financial results for the fourth quarter of 2017. As of December 31, 2017, cash and cash equivalents totaled $58.9 million (Press release, Molecular Templates, MAR 28, 2018, View Source [SID1234525039]). Molecular’s current cash balance is expected to fund operations into late 2019.

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"We are excited by the continued progress of our pipeline as well as our partnership with Takeda," said Eric Poma, Ph.D., CEO and CSO of Molecular Templates. "We expect the remainder of 2018 to bring more data on our lead program MT-3724 in DLBCL, additional IND filings for other pipeline programs, and the potential for more business development transactions that would support additional programs with non-dilutive capital."

Company Highlights and Upcoming Milestones

Corporate

In 4Q17, Molecular expanded its senior management team with the additions of Adam Cutler as Chief Financial Officer; Barbara Ruskin J.D. Ph.D. as SVP, General Counsel and Chief Patent Officer; Nenad Sarapa M.D. M.S. as SVP of Clinical Development, and Conrad Jordaan as SVP of Finance and Corporate Controller.
On March 2, 2018, Molecular closed a $10 million debt facility with Perceptive Advisors. The proceeds were used to repay an existing debt facility with Silicon Valley Bank and will support Molecular’s build out of its GMP manufacturing facility, which should shorten the time from lead development to IND and better support Molecular’s own pipeline as well as partnerships.
MT-3724

MT-3724 (an ETB targeting CD20) is in an ongoing Phase Ib expansion study intended to better define the overall response rate to this candidate as a single-agent in heavily pre-treated diffuse large B-cell lymphoma (DLBCL) patients.
A brief update on the first three patients dosed in the MT-3724 Phase Ib expansion study was delivered at the World ADC Summit Europe today. Observations included the following:
One of the three patients achieved a partial response (PR) after a single dose of MT-3724. The PR was confirmed at the end of cycle 2 per protocol and the patient remains on study with continued dosing of MT-3724. The other two patients were assessed as having stable disease (SD) and progressive disease (PD).
A dose interruption and reduction was required in two of the first three patients in Phase Ib expansion. These patients had high body weights which resulted in high absolute doses of MT-3724 based on 75 mcg/kg dosing. The adverse events observed (grade 2 and 3) were reversible and dosing resumed at 50 mcg/kg, which has been generally well tolerated.
Based on these data, the deep and sustained clinical responses to MT-3724 observed at doses as low as 5 mcg/kg, as well as the near-complete peripheral B-cell depletion at doses up to 50 mcg/kg, the maximum tolerated dose (MTD) of MT-3724, has been defined as 50 mcg/kg with a maximum total drug per dose of 6,000 mcg, or 6 mg.
Enrollment in the Phase Ib expansion study continues, with further updates on results expected in 2Q18.
Nine DLBCL patients with low serum levels of rituximab have been treated at doses ranging from 5 mcg/kg to 75 mcg/kg in the Phase I dose-finding and Phase Ib expansion studies. In these nine patients, one complete response, two partial responses, three patients with stable disease (including one patient with a 48% reduction in tumor size), and three patients with progressive disease, were observed.
Based on the peripheral B-cell depletion observed at 50 mcg/kg and responses seen at doses as low as 5 mcg/kg, 50 mcg/kg appears to be an efficacious and well-tolerated dose.
Molecular also expects to initiate combination studies with MT-3724 in earlier lines of DLBCL therapy in 2Q18.
Takeda Collaboration

In December 2017, Takeda selected two targets for further research using Molecular’s ETBs. This has triggered $4 million in milestone payments to be paid by Takeda in 2018.
Takeda and Molecular are evaluating CD38 ETBs and could select a drug candidate for development by the end of 2Q18.
MT-4019

MT-4019, an ETB candidate that is designed to target CD38-expressing myeloma cancer cells, is progressing through IND enabling studies. If Takeda and Molecular do not select a joint candidate for development, Molecular anticipates filing an IND application for MT-4019 in mid-2018 to initiate a Phase I clinical trial in the United States in 2H18.
Research

Preclinical data for Molecular’s ETBs targeting PD-L1 (which incorporates Molecular’s Antigen Seeding Technology – a differentiated immune-oncology approach) and HER2 will be presented at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in April 2018
Molecular expects to file an IND application for an ETB targeting HER2 in 4Q18
Molecular expects to file an IND application for an ETB targeting PD-L1 (with antigen seeding) in 1Q19
Several other ETB candidates are in preclinical development targeting both solid and hematological cancers
Financial Results

The net loss attributable to common shareholders for the fourth quarter was $6.9 million, or $0.26 per basic and diluted share. This is compared to a net loss attributable to common shareholders for the same period in 2016, of $2.9 million, or $13.82 per basic and diluted share.

Revenues for the fourth quarter of 2017 were $0.8 million, compared to $0.4 million for the same period in 2016. Revenues in the fourth quarters of 2017 and 2016 were comprised of grant revenue from the Cancer Prevention & Research Institute of Texas ("CPRIT"). Total research and development (R&D) expenses for the fourth quarter of 2017 were $4.7 million, compared with $1.7 million for the same period in 2016. Total general and administrative (G&A) expenses for the fourth quarter of 2017 were $3.5 million, compared with $1.1 million for the same period in 2016.

Revenues for the year ended December 31, 2017 were $3.4 million, compared to $1.9 million for 2016. These revenues were primarily comprised of research and development revenues from our collaboration with Takeda of $1.9 million, and grant revenue from CPRIT of $1.0 million. Revenues for the same period in 2016 comprised of grant revenue from CPRIT. Total R&D expenses for the year ended December 31, 2017 were $9.5 million, compared to $8.0 million for 2016. Total G&A expenses for the year ended December 31, 2017 were $11.8 million, compared to $4.5 million for 2016.

The net loss attributable to common shareholders for the year ended December 31, 2017 was $24.1 million, or $2.11 per basic and diluted share, compared to a net loss attributable to common shareholders of $12.6 million or $59.04 per basic and diluted share, for 2016. As of December 31, 2017, cash and cash equivalents totaled $58.9 million. Molecular’s current cash balance is expected to fund operations into late 2019.

On March 28, 2018 Five Prime Therapeutics, Inc. (Nasdaq: FPRX), a biotechnology company discovering and developing innovative immuno-oncology protein therapeutics, reported that it initiated patient dosing in its Phase 1 clinical trial of FPA150, a first-in-class immuno-oncology antibody that targets B7-H4 (Press release, Five Prime Therapeutics, MAR 28, 2018, View Source [SID1234525038]).

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"We’re excited to advance into the clinic our targeted, first-in-class B7-H4 antibody, FPA150, which offers a differentiated approach to existing immunotherapies," said Helen Collins, M.D., Senior Vice President and Chief Medical Officer of Five Prime. "We’re studying FPA150 in patients whose tumors overexpress B7-H4 and in which there is high unmet need for immuno-oncology treatments, such as in breast, ovarian and endometrial cancer. We’re hopeful that a targeted therapy such as FPA150 may provide clinical benefit."

Five Prime designed the Phase 1 trial of monotherapy FPA150 with a dose-escalation phase in patients with solid tumors, followed by dose expansion in pre-specified cohorts in tumor types based on B7-H4 expression levels. The initial targeted tumors are advanced or metastatic breast, ovarian, endometrial and bladder cancers. Phase 1a dose escalation endpoints include identification of a maximum tolerated dose (MTD), safety, and pharmacokinetics (PK) of FPA150. Phase 1b dose expansion endpoints include objective response rate, as well as safety and PK.

About FPA150

FPA150 is a novel, fully human, afucosylated monoclonal antibody targeting B7-H4. B7-H4 expression is observed in multiple solid tumors, including breast, bladder and gynecologic cancers, and has been documented to correlate with poor prognosis. FPA150 is designed with a dual mechanism of action: blocking the T cell checkpoint activity of B7-H4 as well as delivering potent ADCC against tumor cells expressing B7-H4.

On March 28, 2018 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported financial results for the fourth quarter ended December 31, 2017 and provided an update on recent clinical and corporate developments (Press release, Deciphera Pharmaceuticals, MAR 28, 2018, View Source [SID1234525037]).

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"2017 was a highly productive year for Deciphera marked by significant progress with our lead program, DCC-2618. Data presented throughout the year continue to support our belief that DCC-2618 has the potential to serve as a much-needed therapy across multiple disease indications where treatment options for patients are limited," said Michael D. Taylor, Ph.D., President and Chief Executive Officer of Deciphera. "We have entered 2018 with strong momentum, and we were pleased to announce the initiation of our pivotal Phase 3 INVICTUS study of DCC-2618 in fourth-line plus GIST, which, if successful, could serve as the basis for a New Drug Application (NDA). Pending regulatory feedback in the US and Europe, we expect to initiate an additional pivotal Phase 3 study in second-line GIST patients later this year, and we plan to report data emerging from our Phase 1 expansion study cohorts over the course of the year."

Dr. Taylor added, "Following our successful IPO last year, we have a strong balance sheet and believe we are well-positioned for continued success across our full clinical-stage pipeline, including DCC-3014 and rebastinib, and look forward to sharing our progress throughout the coming months."

Recent Clinical and Corporate Developments

DCC-2618
Announced the initiation of the Phase 3 INVICTUS pivotal study in January 2018 evaluating the safety and efficacy of DCC-2618, in heavily pretreated patients with advanced gastrointestinal stromal tumors (GIST). The Company expects to report top-line data from the study in 2019.
Received Orphan Drug Designation from the European Medicines Agency in November 2017 for the treatment of GIST.
Provided an enrollment update from the ongoing Phase 1 clinical trial of DCC-2618 at the Annual Meeting of The Connective Tissue Oncology Society in November 2017. As of October 31, 2017, a total of 125 patients had been dosed with DCC-2618 of which 109 were GIST patients, including 54 GIST patients in three expansion cohorts of the Phase 1 trial, which are enrolling second-line, third-line, and fourth-to-fifth line GIST patients, respectively.
Reported data on eight patients with malignant gliomas in the ongoing Phase 1 clinical trial of DCC-2618 at the 22nd Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology in November 2017, including a 94% tumor reduction per RANO after 84 weeks (cycle 23, day 1) from a patient with glioblastoma multiforme (GBM) and amplification of three kinase genes, KIT, PDGFRα and VEGFR2.
Corporate Updates
Completed an initial public offering of 8,166,496 shares of its common stock in October 2017 at a public offering price of $17.00 per share, including shares pursuant to the partial exercise by the underwriters of their option to purchase additional shares. Deciphera Pharmaceuticals received net proceeds of approximately $124.6 million from the offering, after deducting underwriting discounts, commissions and offering expenses.
Fourth Quarter 2017 Financial Results

Cash Position: As of December 31, 2017, Deciphera Pharmaceuticals reported cash and cash equivalents of $196.8 million.

R&D Expenses: Research and development expenses for the fourth quarter of 2017 were $15.7 million compared to $6.5 million for the same period in 2016. The increase was primarily due to an increase in spending on the DCC-2618 program of $6.3 million as a result of clinical trial costs related to the Phase 1 trial as well as start-up activities related to the pivotal Phase 3 trial in fourth-line GIST, and increased manufacturing and preclinical study costs to support the current and planned clinical trials. In addition, facility and personnel related costs increased an aggregate of $2.9 million as the result of an increase in costs associated with early-stage drug discovery programs and headcount. Personnel costs for each of the fourth quarters of 2017 and 2016 included non-cash share-based compensation expense of $0.5 million and $0.2 million, respectively.

G&A Expenses: General and administrative expenses for the fourth quarter of 2017 were $4.7 million, compared to $2.0 million for the same period in 2016. The increase was primarily due to an increase in non-cash share-based compensation, which was $2.3 million and $0.4 million for each of the fourth quarters of 2017 and 2016, respectively. In addition, legal and professional fees increased as a result of ongoing business activities and operations as a public company.

Net Loss: For the fourth quarter of 2017, Deciphera reported a net loss of $19.9 million, or $0.62 per share, compared with a net loss of $8.6 million, or $0.74 per share, for the same period in 2016.

On March 28, 2018 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company") a biopharmaceutical company developing oral therapies that target the various phases of cell cycle control for the treatment of cancer and other serious disorders, reported its financial results and business highlights for the fourth quarter and full year ended December 31, 2017 (Press release, Cyclacel, MAR 28, 2018, View Source [SID1234525036]).The Company’s net loss applicable to common shareholders for the three months and year ended December 31, 2017 was $2.1 million and $14.9 million, respectively. As of December 31, 2017, cash and cash equivalents totaled $23.9 million.

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"We are greatly encouraged by our clinical progress in 2017, particularly in our transcriptional regulation program with CYC065, our lead CDK inhibitor candidate," said Spiro Rombotis, President and Chief Executive Officer of Cyclacel. "We have achieved clinical proof of mechanism with CYC065 by demonstrating durable reduction of Mcl-1 expression in Phase 1 patients for at least 24 hours after a single dose. We believe these unprecedented findings provide a strong rationale for evaluating CYC065 in combination with venetoclax in patients with chronic lymphocytic leukemia, or CLL, and neuroblastoma, a predominantly pediatric cancer with poor prognosis. In collaboration with an academic center and a pharmaceutical company we have developed a protocol for a Phase 1b/2 investigator-sponsored trial to evaluate combination treatment of an approved PARP inhibitor and sapacitabine in patients with BRCA mutant breast cancer. We have also completed analysis of the results from the SEAMLESS Phase 3 study of sapacitabine and plan to discuss the data with regulatory authorities. Finally, we improved our cash resources raising $14.9 million, net of expenses, which will fund currently planned programs through the first quarter of 2020. We look forward to reporting our progress during the remainder of 2018, including clinical data from our studies as they arise, such as the previously announced, oral presentation of Phase 1 data with CYC065 at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2018 Annual Meeting."

Fourth Quarter and Full-Year Highlights

Drug Development

Transcriptional Regulation Program: CYC065 CDK Inhibitor

In part 1 of an ongoing, first-in-human, single agent, ascending dose, Phase 1 study, prolonged reduction of Mcl-1 was observed in 11 out of 13 evaluable patients treated at the recommended Phase 2 dose, or RP2D, following a single dose of CYC065, which was generally well tolerated. Preliminary anticancer activity was observed in 5 patients, of which 4 were treated at the RP2D and 3 were reported by investigators to have molecular features of their cancers associated with CYC065’s mechanism of action, including amplification of Mcl-1, MYC or cyclin E. The trial is being conducted at the Dana Farber Cancer Institute in Boston. Part 2 of the Phase 1 translational study will evaluate additional dosing schedules in patients with advanced solid tumors, in particular those with amplification of Mcl-1, MYC or cyclin E, including subsets of high grade serous ovarian and uterine cancers. Several biomarkers relevant to CYC065’s mechanism of action will be assessed.

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The protocol for the Phase 1b study of a combination regimen of CYC065 and venetoclax in patients with relapsed or refractory CLL has been submitted to the US Food and Drug Administration or FDA. The study will evaluate safety, pharmacokinetics and pharmacodynamics of the combination, including biomarkers related to the mechanism of action of CYC065.

Discussions with principal investigators and/or cooperative groups progressed with the objective of evaluating CYC065 in both pediatric and adult patients with solid tumors. The Company is discussing with an investigator cooperative group a potential evaluation of CYC065 in patients with neuroblastoma, a mostly pediatric, life-threatening malignancy, frequently associated with MYCN amplification.

In another study, to be conducted as an investigator sponsored trial, CYC065 will be evaluated in adult and pediatric patients with leukemias, including acute myeloid leukemia, or AML, acute lymphocytic leukemia, or ALL, and in particular those with mixed lineage leukemia rearrangements, or MLL-r.

Preclinical data on the molecular rationale and therapeutic potential of CYC065 included an article published in the Journal of National Cancer Institute, reporting prominent antitumor activity against lung cancer cells through anaphase catastrophe, a novel, cancer specific, mechanism. CYC065 was found to be effective against lung cancer cell lines, including those with KRAS mutations. Additional preclinical data presented at the 2017 AACR (Free AACR Whitepaper) Annual Meeting, demonstrated therapeutic potential of CYC065 as a targeted anticancer agent. CYC065 substantially inhibited growth, triggered apoptosis, and induced anaphase catastrophe in murine and human lung cancer cells with known high metastatic potential. This was in marked contrast to effects in immortalized pulmonary epithelial murine and human cells. CYC065 markedly inhibited migration and invasion of lung cancer cells and affected distinctive pathways involved in DNA damage response, apoptosis, cell cycle regulation and cell migration.

DNA Damage Response (DDR) Program

In collaboration with an academic center and a pharmaceutical company we have developed a protocol for a Phase 1b/2 investigator-sponsored trial to evaluate safety and efficacy of a combination regimen of an approved PARP inhibitor and sapacitabine in patients with BRCA mutant breast cancer.

Enrollment has been completed in an extension of part 1 of the Phase 1 study evaluating a combination regimen of sapacitabine and seliciclib, Cyclacel’s first generation CDK inhibitor, in an enriched population of approximately 20 patients with BRCA positive advanced breast cancer. An ongoing part 3 of this study is testing a revised dosing schedule in additional patients, including BRCA positive, patients with breast, ovarian and pancreatic cancers.

SEAMLESS Phase 3 Study

Data from the SEAMLESS Phase 3 study of sapacitabine in elderly patients with AML were the subject of an oral presentation in December 2017 at the 59th ASH (Free ASH Whitepaper) Annual Meeting. The presentation included additional data from prespecified and exploratory analysis of subgroups that may benefit from treatment with the sapacitabine-decitabine alternating regimen. The Company believes that the subgroup results have defined a patient population for whom the sapacitabine regimen may represent an improvement over low intensity treatment by decitabine alone.

The Company has completed further statistical and exploratory analyses of the results from the SEAMLESS study and is preparing briefing documents for submission to regulatory authorities with the objective of determining a potential regulatory pathway for sapacitabine in AML.

PLK1 Inhibitor; CYC140

At the 2017 AACR (Free AACR Whitepaper) Annual Meeting, the Company presented preclinical data outlining the potential therapeutic utility of CYC140, a novel, polo-like kinase (PLK) 1 inhibitor, alone and in synergistic drug combinations, for the treatment of esophageal cancer and acute leukemia.

Investigator-Sponsored Trials (ISTs): Seliciclib in Rheumatoid Arthritis (RA)

The Independent Data Monitoring Committee, or IDMC, for the "TRAFIC" trial sponsored by the UK Medical Research Council, (ISRCTN 36667085) determined that part 1 of the study was successfully completed per protocol. The IDMC recommended continuation of the trial into part 2 to assess potential efficacy of seliciclib as an addition to existing anti-TNF therapy based on a composite outcome of response in patients with moderate to severe RA.

Corporate Developments

The Company raised net proceeds of approximately $13.7 million from an underwritten offering of common stock.

Cyclacel received notice from the European Patent Office of the grant of a European patent including claims to novel pharmaceutical formulations of sapacitabine.

2018 Key Upcoming Business Objectives

·Report updated CYC065 Phase 1 data in patients with advanced cancers

·Initiate CYC065 Phase 1b in relapsed/refractory CLL in combination with venetoclax

·Start enrollment in the Phase 1b/2 IST of a combination regimen of an approved PARP inhibitor and sapacitabine in patients with BRCA mutant breast cancer

·Start enrollment in the Phase 1b/2 IST of CYC065 in pediatric patients with neuroblastoma

·Update mature data from the part 1 extension of the sapacitabine and seliciclib combination in patients with BRCA positive advanced breast and complete part 3 enrollment of the sapacitabine and seliciclib combination in patients with BRCA positive, breast, ovarian and pancreatic cancers

·Submit CYC140, PLK1 inhibitor, IND application

·Conduct regulatory authority meetings regarding the SEAMLESS study of sapacitabine in AML

Financial Highlights

As of December 31, 2017, cash and cash equivalents totaled $23.9 million, compared to $16.5 million as of December 31, 2016. The increase of $7.4 million was primarily due to net proceeds of $13.7 million from a direct registered offering, $1.0 million from the sale of common stock through the ATM sales agreement with FBR Capital Markets & Co., $0.2 million warrant exercises and offset by $7.5 million of net cash used in operating activities.

There were no revenues for the three months and year ended December 31, 2017 compared to $0.3 million and $0.8 million for the same period of the previous year. The revenue is related to previously awarded, UK government grants being recognized over the period to progress IND-directed preclinical development of CYC140, a novel, PLK-1 inhibitor, which was completed in November 2016.

Research and development expenses were $0.7 million and $4.2 million for the three months and year ended December 31, 2017 as compared to $1.9 million and $9.5 million for the same periods in 2016. The decrease was primarily due to reduced study and clinical supply costs associated with completion of the SEAMLESS study and 2016 expenditure related the development of CYC140.

General and administrative expenses for the three months and year ended December 31, 2017 were $1.5 million and $5.3 million, compared to $1.5 million and $5.5 million for the same period of the previous year.

Other income (expense), net for the three months and year ended December 31, 2017 were $0.1 million and $1.0 million, compared to ($0.1) million and $0.4 million for the same period of the previous year. The increase is primarily related to income received under an Asset Purchase Agreement with Life Technologies Corporation, or LTC, (formerly Invitrogen Corporation and subsequently acquired by ThermoFisher Scientific), resulting from certain assets and intellectual property sold by the Company to LTC in December 2005.

United Kingdom research & tax credits were $0.2 million and $1.0 million for the three months and year ended December 31, 2017 as compared to $0.4 million and $2.0 million for the same periods in 2016.

Net loss for the three months and year ended December 31, 2017 were $1.9 million and $7.5 million compared to $2.8 million and $11.8 million for the same periods in 2016.

Conference call information:

US/Canada call: (877) 493-9121 / international call: (973) 582-2750

US/Canada archive: (800) 585-8367 / international archive: (404) 537-3406

Code for live and archived conference call is 4857905

For the live and archived webcast, please visit the Corporate Presentations page on the Cyclacel website at www.cyclacel.com. The webcast will be archived for 90 days and the audio replay for 7 days.