On November 13, 2019 Palatin Technologies, Inc. (NYSE American: PTN), a specialized biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin and natriuretic peptide receptor systems, whose product candidates are targeted, receptor-specific therapeutics for the treatment of diseases with significant unmet medical need and commercial potential, reported results for its first quarter ended September 30, 2019 (Press release, Palatin Technologies, NOV 13, 2019, View Source [SID1234551127]).

Quarter End and Recent Highlights

Hypoactive Sexual Desire Disorder / Vyleesi (bremelanotide injection)
Vyleesi is the first as needed treatment for premenopausal women with acquired, generalized HSDD.

AMAG Pharmaceuticals, Palatin’s North American licensee, launched Vyleesi nationally in September with its established women’s health sales force of approximately 125 sales representatives. In the four weeks since the national launch, more than 1,300 healthcare providers have prescribed Vyleesi, which has resulted in more than 3,000 prescriptions received by AMAG’s specialty pharmacy partners.

Anti-Inflammatory / Autoimmune Programs
Melanocortin agonist products are under development for the treatment of inflammatory and autoimmune diseases such as dry eye, uveitis, diabetic retinopathy and inflammatory bowel diseases (ulcerative colitis). We announced positive results of a micro-dose study of radiolabeled PL8177, a selective melanocortin receptor 1 ("MC1r") peptide agonist, using an oral, delayed-release, polymer formulation. The study met all primary and secondary endpoints. PL8177 has potential application in treatment of ulcerative colitis and other inflammatory bowel diseases. The FDA has granted orphan drug designation for PL8177 for the treatment of non-infectious intermediate, posterior, pan and chronic anterior uveitis. Non-infectious uveitis (NIU) is a group of inflammatory diseases that produces swelling and destroys eye tissue and can result in vision loss. A Phase 2 proof-of-concept clinical study with a systemic formulation in NIU patients is anticipated to commence in the first quarter of calendar year 2020. A Phase 2 proof-of-concept clinical study with an oral formulation in ulcerative colitis patients is anticipated to commence in the second quarter of calendar year 2020.

An IND application for PL9643, a melanocortin peptide agonist, and commencement of a Phase 2 clinical study in dry eye disease, are currently anticipated in the first quarter of calendar year 2020.

Natriuretic Peptide Receptor ("NPR") System Program
The Company has designed and is developing potential drug candidates that are selective agonist for one or more different natriuretic peptide receptors, including natriuretic peptide receptor-A ("NPR-A"), natriuretic peptide receptor B ("NPR-B"), and natriuretic peptide receptor C ("NPR-C"). Active collaborations with several institutions are ongoing for PL3994, an NPR-A agonist that has potential utility in the treatment of a number of cardiovascular diseases, including genetic and orphan diseases resulting from a deficiency of endogenous active NPR-A, and PL5028, a dual NPR-A and NPR-C agonist in development for cardiovascular diseases, including reducing cardiac hypertrophy and fibrosis. A Phase 2A clinical trial evaluating PL3994 in heart failure patients with preserved left ventricular ejection fraction will begin enrollment in the latter half of calendar year 2019. This trial is supported by a grant from the American Heart Association.

Genetic Obesity Program
The Company’s melanocortin receptor 4 ("MC4r") peptide PL8905 and orally, active small molecule PL9610 are currently under investigation for the treatment of rare genetic metabolic and obesity disorders. These programs are under internal evaluation for orphan designation and potential development.

Corporate
Cash, cash equivalents and accounts receivable balances at September 30, 2019 total $96.8 million.

Debt and related liabilities were fully paid off in July 2019.

In September 2019, the Company’s Board of Directors approved a plan to offer to purchase and terminate certain outstanding common stock purchase warrants through privately negotiated transactions. The purchase and termination program has no time limit and may be suspended for periods or discontinued at any time.

To date, the Company entered into several warrant termination agreements to repurchase and cancel previously issued warrants. The Company repurchased and cancelled in the aggregate 6.5 million warrants at an aggregate buyback price of $2.5 million.

First Quarter Fiscal 2020 Financial Results
Palatin reported a net loss of $(4.5) million, or $(0.02) per basic and diluted share, for the quarter ended September 30, 2019, compared to a net loss of $(5.7) million, or $(0.03) per basic and diluted share, for the same period in 2018.

The difference in financial results between the three months ended September 30, 2019 and 2018 was due to reductions in operating and interest expenses combined with increases in reported license and contract revenue and investment income.

Revenue
For the quarter ended September 30, 2019, Palatin recognized as revenue $97,379 in reimbursement of shared Vyleesi costs compared to $34,505 for the quarter ended September 30, 2018.

Operating Expenses
Total operating expenses for the quarter ended September 30, 2019 were $5.0 million compared to $5.7 million for the comparable quarter of 2018. The decrease in operating expenses was mainly attributable to decreases in salaries and other employee related expenses.

Other Income/Expense
Total other income, net was $361,603 for the quarter ended September 30, 2019 compared to total other expense, net of $(53,288) for the quarter ended September 30, 2018. The difference is primarily related to an increase in investment income and secondarily to a decrease in interest expense.

Cash Position
Palatin’s cash, cash equivalents, and accounts receivable total $96.8 million as of September 30, 2019, compared to cash, cash equivalents and accounts receivable of $103.8 million at June 30, 2019. Current liabilities were $1.9 million as of September 30, 2019, compared to $4.2 million as of June 30, 2019.

Palatin Drug Discovery and Development Programs
During the conference call and webcast, management will update and discuss next steps in Palatin’s portfolio of drug development programs. These include Palatin’s melanocortin receptor agonist peptides for treatment of inflammatory indications and natriuretic peptide receptor agonist compounds for treatment of cardiovascular and fibrotic diseases.

Conference Call / Webcast
Palatin will host a conference call and audio webcast on November 13, 2019 at 11:00 a.m. Eastern Time to discuss the quarter ended September 30, 2019 results of operations in greater detail and provide an update on corporate developments. Individuals interested in listening to the conference call live can dial 1-888-599-8686 (US/Canada) or 1-323-794-2575 (international), conference ID 1609183. The audio webcast and replay can be accessed by logging on to the "Investor/Webcasts" section of Palatin’s website at View Source A telephone and audio webcast replay will be available approximately one hour after the completion of the call. To access the telephone replay, dial 1-888-203-1112 (US/Canada) or 1-719-457-0820 (international), passcode 1609183. The webcast and telephone replay will be available through November 20, 2019.

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On November 13, 2019 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company"), a biopharmaceutical company developing innovative medicines based on cancer cell biology, reported financial results and business highlights for the third quarter 2019 (Press release, Cyclacel, NOV 13, 2019, View Source [SID1234551143]). The Company’s net loss applicable to common shareholders for the three months ended September 30, 2019 was $2.0 million. As of September 30, 2019 cash and cash equivalents totaled $13.0 million.

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"The investigators evaluating CYC065, our CDK2/9 inhibitor, as a single agent have reported that a heavily pretreated patient with MCL1 amplified endometrial cancer achieved a partial response (PR) with tumor shrinkage of 48%," said Spiro Rombotis, President and Chief Executive Officer of Cyclacel. "If this PR is confirmed with a follow up scan, it will provide further support that MCL1 dependent cancers may be sensitive to CYC065. We have previously reported that CYC065 durably suppresses MCL1 in cancer patients and believe that it is a leader amongst MCL1 suppressing medicines. In parallel, we have enrolled our first patient with an oral form of CYC065 and are continuing to recruit patients in Phase 1 studies evaluating CYC065 in combination with venetoclax in patients with relapsed or refractory AML, CLL or MDS. Altogether we are dosing patients in five clinical studies in pursuit of our strategy of overcoming cancer resistance through combinations of our candidates with approved drugs. With projected cash to the end of 2020 we look forward to delivering on multiple data outcomes from our ongoing studies."

Key Company Highlights

·Reported anticancer activity in part 2 of 065-01, the Phase 1 study of CYC065 as a single agent; a patient with MCL1 amplified endometrial cancer achieved partial response with 48% tumor shrinkage after 4 cycles of treatment at 213mg as reported by the investigators;
·Reached the second dose level in part 3 of 065-01 evaluating an oral form of CYC065 in patients with advanced cancers;
·Enrolled eight patients in the 065-03 Phase 1 study evaluating CYC065 in combination with venetoclax in patients with relapsed or refractory AML/MDS;
·Opened two new sites in the 065-02 study of CYC065 in combination with venetoclax in patients with relapsed/refractory CLL;
·Data from three ongoing studies have been selected for presentation at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The presentations will provide updates for CYC065 in combination with venetoclax in patients with relapsed or refractory AML/MDS or CLL and sapacitabine in combination with venetoclax in patients with relapsed or refractory AML/MDS; and
·Continued enrollment in part 2 of the 682-11 Phase 1/2 study evaluating an oral regimen of sapacitabine in combination with venetoclax in patients with relapsed or refractory AML/MDS.

Key Upcoming Business Objectives

·Presentations at the 61stAmerican Society of Hematology Annual Meeting;
·Report updated safety, pharmacokinetics and efficacy of CYC065 Phase 1 data with frequent dosing schedule in patients with advanced solid cancers;
·Report initial safety and PK data from the Phase 1 study of an oral formulation of CYC065;
·Report initial safety and proof of concept data from the CYC065-venetoclax Phase 1 study in relapsed/refractory AML and MDS;
·Report initial safety and proof of concept data from the CYC065-venetoclax Phase 1 study in relapsed/refractory CLL;
·Report initial data from the sapacitabine-venetoclax Phase 1/2 study in patients with relapsed or refractory AML or MDS;
·Report initial data from the CYC140 Phase 1 First-in-Human study in relapsed or refractory leukemias;
·Report data from the IST Phase 1b/2 trial of sapacitabine-olaparib combination in patients with BRCA mutant metastatic breast cancer when reported by the investigators; and
·Determine regulatory pathway and submissibility of sapacitabine in elderly AML patients.

Financial Highlights

As of September 30, 2019, cash and cash equivalents totaled $13.0 million compared to $17.5 million as of December 31, 2018. The decrease of $4.5 million was primarily due to net cash used in operating activities of $8.3 million, offset by net proceeds from a Common Stock Sales Agreement with H.C. Wainwright & Co., LLC of $4.1 million. In October 2019, we received $1.2 million in United Kingdom, research & development tax credits not included in the above amounts.

Research and development expenses were $1.1 million for the three months ended September 30, 2019 compared to $1.2 million for the same period in 2018.

General and administrative expenses were $1.3 million for each of the three months ended September 30, 2019 and 2018.

Other income, net for the three months ended September 30, 2019 was $0.2 million compared to $0.1 million for the same period of the previous year.

The accrued United Kingdom research and development tax credit was $0.3 million for each of the three months ended September 30, 2019 and 2018.

Net loss for the three months ended September 30, 2019 was $1.9 million compared to $2.1 million for the same period in 2018. With the projected cash-sparing benefits accruing from the alliance with The University of Texas MD Anderson Cancer Center, the Company believes that cash and marketable securities, which were approximately $13.0 million as of September 30, 2019, will be sufficient to finance operations through the end of 2020.

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 8636118

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.

On November 13, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, reported financial results for the quarter ended September 30, 2019 (Press release, Gamida Cell, NOV 13, 2019, View Source [SID1234551177]). The company also highlighted continued progress in advancing its clinical development candidates: omidubicel, an advanced cell therapy in Phase 3 clinical development as a potential life-saving treatment option for patients in need of bone marrow transplant, and GDA-201, an investigational, natural killer (NK) cell-based cancer immunotherapy in Phase 1 development in patients with non-Hodgkin lymphoma and multiple myeloma.

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Gamida Cell provides a company update and reports financial results for the third quarter of 2019. Webcast at 4:30pm ET here: View Source

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"During the quarter, we made important progress toward our goal of developing next-generation cell therapies with the potential to redefine standards of care for patients with blood cancers and rare, serious hematologic diseases," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We are nearing completion of patient enrollment in our multi-center, randomized Phase 3 study of omidubicel and are on track to report topline data from the study in the first half of 2020. Positive data from the study would enable us to file our first biologics license application next year."

"Our second cell therapy program, GDA-201, is also advancing, and next month we will report additional data from the ongoing Phase 1 study at the Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper)," Dr. Adams continued. "During the meeting, we will also show new gene expression data that reinforce our understanding of the mechanism of action underlying our NAM technology platform which enabled the generation of both of our development candidates."

Program Highlights

Announced presentation of new data at the 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper): Last week, Gamida Cell announced that additional data from the ongoing Phase 1 clinical study of GDA-201 will be presented during an oral session at the ASH (Free ASH Whitepaper) 2019 Annual Meeting, which is being held December 7 – 10 in Orlando, FL. The presentation, "Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer Cells (NAM-NK) in Patients with Refractory Non-Hodgkin Lymphoma (NHL) and Multiple Myeloma (MM)" (Abstract #777), will take place on Monday, December 9, at 3:15 p.m. ET.

Additionally, new research on the mechanism of action of Gamida Cell’s NAM-based cell expansion platform, which is designed to enhance the number and functionality of allogeneic donor cells, will also be shared during the meeting. The poster presentation, "Nicotinamide (NAM) Modulates Transcriptional Signature of Ex Vivo Cultured UCB CD34+ Cells (Omidubicel) and Preserves Their Stemness and Engraftment Potential" (Abstract #3718), will take place on Monday, December 9, from 6:00 – 8:00 p.m. ET.
Advanced the Phase 3 clinical study of omidubicel: Patient enrollment continued to progress in the Gamida Cell’s Phase 3 study of omidubicel in patients with high-risk hematologic malignancies. The international, randomized, multi-center study is designed to evaluate the safety and efficacy of omidubicel compared to standard umbilical cord blood for allogeneic bone marrow transplant in approximately 120 patients with no available matched donor. The company anticipates completing patient enrollment by the end of this year with topline data anticipated in first half of 2020.
Initiated health outcomes research for omidubicel: In September, Gamida Cell and the CIBMTR (Center for International Blood and Marrow Transplant Research) announced a research agreement to evaluate outcomes of patients with hematological malignancies who undergo allogeneic hematopoietic stem cell transplant (bone marrow transplant) from various donor sources. The recently launched observational study includes both retrospective and prospective data contemporaneous to the Phase 3 study of omidubicel. The goal of this real-world, observational study is to better understand the variables that influence the health outcomes of patients receiving a transplant from a source other than a fully matched family donor.
Continued to focus on activities required to successfully bring omidubicel to patients: Gamida Cell is continuing to advance key activities required to bring omidubicel to patients in a commercial setting, including building out manufacturing infrastructure, assembling an experienced commercial team with expertise in cell therapy and transplant, establishing hospital services and patient assistance programs, and exploring coverage and reimbursement models to enable access.
Progressed enrollment in the Phase 1/2 study of omidubicel in patients with severe aplastic anemia: Enrollment is ongoing in a Phase 1/2 clinical study of omidubicel in patients with severe aplastic anemia, a rare, life-threatening bone marrow failure disease. Earlier this year, encouraging data from the first cohort of patients were reported at the 2019 Transplantation & Cellular Therapy (TCT) Meeting that demonstrated that all three patients in the cohort successfully underwent a bone marrow transplant consisting of omidubicel plus a haploidentical stem cell graft. The rapid engraftment, sustained hematopoiesis and accelerated immune recovery observed enabled the initiation of a second cohort, where patients will be treated with omidubicel as a stand-alone graft.
Continued to prepare for the next clinical study of GDA-201: The company is continuing its work to enable a Phase 1/2 multi-dose, multi-center study of GDA-201 in patients with non-Hodgkin lymphoma, which is expected to begin in 2020. The decision to focus the next clinical study on non-Hodgkin lymphoma is based on the encouraging clinical data reported at the 2019 TCT Meeting which demonstrated the GDA-201 was generally well tolerated and clinically active, with multiple complete responses observed.
Third Quarter 2019 Financial Results

Research and development (R&D) expenses in the third quarter of 2019 were $7.4 million compared to $5.1 million for the same period in 2018. R&D expenses were higher in the third quarter of 2019 compared to the same period in 2018 due to the advancement of omidubicel and GDA-201.
General and administrative expenses were $4.6 million for the third quarter of 2019, compared to $2.4 million for the same period in 2018. The difference was attributable mainly to a $1.2 million increase in activities related to commercial readiness, as well as $1.0 million increase in professional services and other expenses, including an increase in expenses associated with being a publicly-traded company.
Finance income, net, was $1.7 million for the third quarter of 2019, compared to finance expenses, net, of $2.2 million for the same period in 2018. The net increase was primarily due to non-cash income resulting from the re-valuation of warrants, offset by non-cash expenses from the re-valuation of the Israeli Innovation Authority royalty-bearing grant liability.
Net loss for the third quarter of 2019 was $10.1 million, compared to a net loss of $9.8 million for the same period in 2018.
As of September 30, 2019, Gamida Cell had total cash, cash equivalents and available-for-sale assets of $68.1 million, compared to $60.7 million as of December 31, 2018.
2019 Financial Guidance

Gamida Cell continues to expect cash used for ongoing operating activities in 2019 to range from $35 million to $40 million, reflecting anticipated expenditures to advance the company’s clinical programs.

Gamida Cell expects that its current cash, cash equivalents and available-for-sale securities will support the company’s ongoing operating activities into the fourth quarter of 2020. This cash runway guidance is based on the company’s current operational plans and excludes any additional funding that may be received or business development activities that may be undertaken.

Conference Call Information

Gamida Cell will host a conference call today, November 13, 2019, at 4:30 p.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors" section of Gamida Cell’s website at www.gamida-cell.com. To participate in the live call, please dial 866-930-5560 (domestic) or 409-216-0605 (international) and refer to conference ID number 8653335. A replay of the webcast will be available approximately two hours after the event, for approximately 30 days.

About Omidubicel

Omidubicel (formerly known as NiCord), the company’s lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated rapid and durable time to engraftment and was generally well-tolerated.1 A Phase 3 study evaluating omidubicel in patients with leukemia and lymphoma is ongoing in the U.S., Europe and Asia.2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.3 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

About GDA-201

Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201 (formerly known as NAM-NK), an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.4

Omidubicel and GDA-201 are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On November 13, 2019 Constellation Pharmaceuticals, Inc. (Nasdaq: CNST), a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported that Jigar Raythatha, CEO, will present at three upcoming investor conferences (Press release, Constellation Pharmaceuticals, NOV 13, 2019, View Source [SID1234551128]):

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Jefferies London Healthcare Conference, 8:00 AM GMT/3:00 AM EST, November 20
Piper Jaffray Healthcare Conference in New York, 11:10 AM EST, December 3
Evercore ISI Healthcare Conference in Boston, 11:45 AM EST, December 4
A live audio webcast of these presentations and archives for replay will be available on the Investor Relations section of Constellation’s website at View Source The audio webcast replays will be available for 90 days following the live presentation.

On November 13, 2019 DiaMedica Therapeutics Inc. (Nasdaq: DMAC), a clinical-stage biopharmaceutical company focused on developing novel treatments for kidney diseases and neurological disorders, reported its financial results for the three and nine months ended September 30, 2019 (Press release, DiaMedica, NOV 13, 2019, View Source [SID1234551144]).

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Clinical Developments

DM199 for the Treatment of Chronic Kidney Disease

Phase Ib Clinical Study in Patients with CKD Completed

DiaMedica has completed its Phase Ib clinical trial of DM199, a recombinant form of the endogenous human tissue kallikrein protein (KLK1), in patients with moderate or severe Chronic Kidney Disease (CKD) caused by Type I or Type II diabetes mellitus. The study was performed to assess the pharmacokinetics (PK) of three dose levels of DM199 (3, 5 and 8 µg/kg), administered in a single subcutaneous dose, as well as the evaluation of safety, tolerability and secondary pharmacodynamic (PD) endpoints.

The Company previously announced positive interim results. PK profiles, at the 3µg/kg dose level, were similar between moderate and severe CKD patients, and consistent with healthy subjects (normal kidney function) tested previously. Therefore, the Company does not believe dosing adjustment is warranted, based on the presence or severity of CKD and a full renal study will likely not be required. Final study results indicated that DM199 was observed to be well tolerated with no dose-limiting tolerability. There were no deaths, no discontinuations due to a treatment-related adverse event (AE), and no treatment-related significant adverse events (SAEs). AEs were minor and consistent with standard treatment(s) in the CKD patient population.

Favorable overall PD results were also observed including short-term improvements in Nitric Oxide (NO), average increase of 35.2%, Prostaglandin E2 (PGE2), average increase of 41.2%, estimated glomerular flow rate (eGFR), average increase of 4.08 mL/min/1732, and the urinary albumin to creatinine ratio (UACR), average decrease of 18.7%. PD results appeared to be drug related in that greatest improvements occurred at approximately 24 hours after DM199 administration and subsequently declined.

DiaMedica intends to release full study results at an upcoming conference, including a breakout analysis of moderate and severe patients and results by dose level.

Phase II Clinical Study in CKD Caused by IgA Nephropathy and African Americans with Hypertension – Screening Commenced

The U.S. Food and Drug Administration (FDA) has accepted the Company’s Phase II clinical trial protocol for the treatment of CKD caused by rare or significant unmet diseases. This study is designed to investigate the safety, efficacy, PK and PD of DM199. Screening of patients has commenced and the enrollment and dosing is expected in the near term.

The Phase II trial named REDUX, latin for restore, is a multi-center, open-label investigation of approximately 60 participants with CKD, who are being enrolled in two cohorts (30 per cohort). The study is being conducted in the United States at up to 10 sites and will be focused on participants with CKD: Cohort I is focused on non-diabetic, hypertensive African Americans with Stage II or III CKD. African Americans are at greater risk for CKD than Caucasians, and those who have the APOL1 gene mutation are at an even higher risk. The study is designed to capture the APOL1 gene mutation as an exploratory biomarker in this cohort; Cohort II is focused on participants with IgA Nephropathy (IgAN). The study will evaluate two dose levels of DM199 within each cohort. Study participants will receive DM199 by subcutaneous injection twice weekly for 95 days. The primary study endpoints include safety, tolerability, blood pressure, proteinuria and kidney function, which will be evaluated by changes from baseline in eGFR and albuminuria, as measured by the UACR.

"We are pleased with the results from our CKD Phase Ib study and our ability to now commence Phase II studies," commented Dr. Harry Alcorn, DiaMedica’s Chief Medical Officer. "We look forward to our Phase II study building on our successful Phase Ib data and we have engaged a number of study sites that have the patient populations and experience in obtaining high quality data to better understand the potential of DM199 in benefitting individuals suffering with CKD, whose current treatment options are very limited."

DM199 for the Treatment of Acute Ischemic Stroke

DM199 Acute Ischemic Stroke Phase II "REMEDY" Trial Update – Enrollment Completed

DiaMedica has recently completed enrollment in the REMEDY trial, the Company’s Phase II study assessing the safety, tolerability and markers of therapeutic efficacy of DM199 in participants suffering from Acute Ischemic Stroke (AIS). Final enrollment was 92 participants. The markers of therapeutic efficacy will include multiple plasma-based biomarkers (e.g. C-reactive protein), the Modified Rankin Scale, National Institutes of Health Stroke Scale and the Barthel Index. These markers are assessed at multiple points throughout the study, including 90 days post-stroke.

Organizational Update

Dr. Sydney A. Gilman has joined DiaMedica as its Vice President of Regulatory Affairs. Dr. Gilman has over 30 years of pharmaceutical industry experience holding senior positions including at Elan Pharmaceuticals and Amylin Pharmaceuticals. Dr. Gilman also spent six years as a CMC reviewer with the FDA’s Center for Drug Evaluation and Research group. In his most recent role as founder and Senior Regulatory Consultant of Trident Rx Consulting, Dr. Gilman has been involved in the development and implementation of strategic plans for drug development and regulatory strategy for new drugs and biologics. In addition he has lead a variety of global regulatory submissions, including INDs, NDAs, BLAs, MAAs, global CTAs, 510Ks, PMAs, orphan and Fast Track submissions, and clinical study reports.

"We continue to make great progress with our clinical programs with the commencement of our Phase II CKD studies and the completion of enrollment in our REMEDY Phase II study, and I wish to thank our clinical team for their skill and dedication," stated Rick Pauls, DiaMedica’s President and CEO. "I would also like to welcome Dr. Gilman, who is a strong addition at this critical time. His proven track record in leading regulatory affairs is instrumental as we transition DM199 into Phase II and Phase III clinical trials and work to improve the lives of CKD and AIS patients."

Financial Results

Research and development (R&D) expenses increased to $1.6 million for the three months ended September 30, 2019, up from $1.2 million for the three months ended September 30, 2018, an increase of $0.4 million. R&D expenses increased to $6.1 million for the nine months ended September 30, 2019, compared to $3.1 million for the nine months ended September 30, 2018, an increase of $3.0 million. The increase for the nine months ended September 30, 2019 was due to costs of approximately $1.4 million incurred for a new production run of the DM199 drug substance, as well as costs incurred in conjunction with the Phase Ib and Phase II clinical studies in CKD patients and related non-clinical testing. Increased personnel and non-cash share-based compensation costs also contributed to the increase. The increase for the three months ended September 30, 2019 was due to costs incurred in conjunction with the Phase Ib and Phase II CKD studies and non-cash share-based compensation costs, partially offset by a decline in costs incurred in conjunction with the REMEDY Phase II stroke study.

General and administrative (G&A) expenses were $1.0 million for the three months ended September 30, 2019, compared to $777,000 for the three months ended September 30, 2018. G&A expenses increased to $2.7 million for the nine months ended September 30, 2019, up from $2.1 million for the nine months ended September 30, 2018. These increases were primarily due to costs associated with our status as a Nasdaq-listed U.S. public reporting company, which commenced in December 2018, including increased professional services, compliance and non-cash share-based compensation costs. Increased personnel costs also contributed to the increase on a year to date basis.

Total other income increased to $225,000 for the three months ended September 30, 2019, up from $157,000 for the prior year period. Total other income decreased to $683,000 for the nine months ended September 30, 2019, compared to $946,000 for the nine months ended September 30, 2018. The year-to-date decrease is primarily related to the initial recognition of R&D incentives from the Australian Government paid for qualifying research work performed by DiaMedica Australia Pty Ltd. during the nine months ended September 30, 2018. The increase in the quarterly comparison relates to increased study costs, compared to the prior year period, driving an increase in the eligible R&D incentive. The year-to-date decrease was partially offset by, and the current quarter increase was augmented by, increased interest income earned on marketable securities during the three and nine months ended September 30, 2019.

Balance Sheet and Cash Flow

The Company had cash and cash equivalents of $4.7 million, marketable securities of $5.0 million, current liabilities of $1.3 million and working capital of $9.6 million as of September 30, 2019, compared to $16.8 million in cash and cash equivalents, $1.3 million in current liabilities and $16.7 million in working capital as of December 31, 2018. The decreases in combined cash and cash equivalents and marketable securities and in working capital are due primarily to the Company’s operating loss incurred for the nine months ended September 30, 2019.

Net cash used in operating activities was $7.2 million for the nine months ended September 30, 2019, compared to $3.8 million for the nine months ended September 30, 2018. The net cash used in each of these periods primarily reflects the net loss for these periods, and was partially offset by non-cash charges for stock-based compensation and the net effects of changes in operating assets and liabilities.

Conference Call Information

DiaMedica management will host a conference call to discuss these results on Thursday, November 14, 2019, at 7:00 a.m. Central Time:

Date:

Thursday, November 14, 2019

Time:

7:00 AM CT / 8:00 AM ET

Web access:

View Source

Dial In:

(844) 557-8483 (domestic)

(825) 312-2381 (international)

Conference ID:

4594493

Interested parties may access the conference call by dialing in or listening to the simultaneous webcast. Listeners should log on to the website or dial in 15 minutes prior to the call. The webcast will remain available for play back on our website, under investor events and presentations, following the earnings call and for 12 months thereafter. A telephonic replay of the conference call will be available until November 21, 2019, by dialing (800) 585-8367 (US Toll Free), (416) 621-4642 (International), replay passcode 4594493.

About DM199
DM199 is a recombinant (synthetic) form of the human serine protease, KLK1. The KLK1 protein plays an important role in the regulation of diverse physiological processes including blood flow, inflammation, fibrosis, oxidative stress and neurogenesis via a molecular mechanism that increases production of nitric oxide and prostaglandin. KLK1 deficiency may play a role in multiple vascular and fibrotic diseases such as chronic kidney disease, retinopathy, stroke, vascular dementia, and resistant hypertension where current treatment options are limited or ineffective. DiaMedica is the first company to have developed a recombinant form of the KLK1 protein. The KLK1 protein, produced from porcine pancreas and human urine, has been used to treat patients in Japan, China and Korea for decades. DM199 is currently being studied in patients with chronic kidney disease and patients with acute ischemic stroke.