On May 6, 2019 DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI) ("DelMar" or the "Company"), reported a biopharmaceutical company focused on the development of new cancer therapies, on May 3, 2019 presented data supporting Dianhydrogalactitol (VAL-083) as a potential therapy for pediatric brain tumors at the Society for Neuro-Oncology (SNO) Pediatric Neuro-Oncology Basic and Translational Research Conference held May 3-4, 2019 in San Francisco, CA (Press release, DelMar Pharmaceuticals, MAY 6, 2019, View Source [SID1234535749]). The Company, which has been working in collaboration with investigators from the University of California, San Francisco (UCSF), University of British Columbia, and the Vancouver Prostate Centre on evaluating VAL-083 as a treatment for diffuse intrinsic pontine glioma (DIPG), an extremely aggressive brain tumor, presented promising in vivo DIPG model data demonstrating VAL-083’s potential as a single agent, and in combination with Wee1 inhibitor AZD1775, in significantly prolonging survival.

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VAL-083 presentation highlights as a treatment for DIPG included:

As a single agent significantly increases median survival in DIPG in vivo compared to untreated controls
In combination with AZD-1775, a Wee1 inhibitor, further increases survival in DIPG in vivo
Is active against DIPG cell lines with varying genetic profiles including p53, H3.3/H3.1 and K27M mutations
Is synergistic with AZD1775 against DIPG and pediatric GBM cell lines
"We are encouraged by this data that demonstrates VAL-083’s ability as a single agent and in combination with Wee1 inhibitor AZD1775 to significantly increase survival in an in vivo DIPG model. An intact blood-brain barrier impeding drug penetration is a major obstacle to successful treatment of DIPG and VAL-083’s ability to concentrate in the cerebral spinal fluid (CSF) may offer an important advantage," commented Dr. Sabine Muller, Associate Professor of Clinical Neurology, UCSF.

DIPG is a difficult-to-treat, inoperable, rare pediatric brain tumor with very poor prognosis and a dismal survival outlook. Approximately 300 children in the U.S. are diagnosed with DIPG each year. While DIPGs are usually diagnosed when children are between the ages of 5 and 9, they can occur at any age in childhood. These tumors occur in boys and girls equally and do not generally appear in adults.

"While it’s certainly early in the evaluation process for the DIPG indication, I’m extremely pleased by these results demonstrating VAL-083’s potential in this difficult-to-treat pediatric brain cancer population. I am also encouraged to see the potential therapeutic benefit afforded by VAL-083 due to its ability to concentrate in the CSF. Recent data reported at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in April 2019 from our clinical study for newly-diagnosed, adult, unmethylated MGMT GBM patients demonstrated that drug levels in the CSF were generally higher in comparison to plasma levels at two hours after administration. This compares favorably to standard of care temozolomide where CSF concentration levels are approximately 80% lower than in plasma and may offer an important advantage for VAL-083," commented Saiid Zarrabian, DelMar’s Chief Executive Officer.

About VAL-083

VAL-083 (dianhydrogalactitol) is a "first-in-class," bifunctional DNA-targeting agent that introduces interstrand DNA cross-links at the N7-position of guanine leading to DNA double-strand breaks and cancer cell death. VAL-083 has demonstrated clinical activity against a range of cancers including GBM and ovarian cancer in historical clinical trials sponsored by the U.S. National Cancer Institute (NCI). DelMar has demonstrated that VAL-083’s anti-tumor activity is unaffected by common mechanisms of chemoresistance, including MGMT, in cancer cell models and animal studies. Further details regarding these studies can be found at:

View Source

VAL-083 has been granted orphan drug designations by the U.S. FDA Office of Orphan Products for the treatment of glioma, medulloblastoma and ovarian cancer, and in Europe for the treatment of malignant gliomas. VAL-083 has been granted fast-track status for the treatment of recurrent GBM by the US FDA.

On May 6, 2019 OnKure, Inc., the epigenetics-focused drug discovery and development company that is advancing the first Largazole-derived, histone deacetylase inhibitor into Phase 1 clinical trials, reported that its lead drug, OKI-179, has cleared the FDA IND regulatory hurdle to start Phase 1 trials (Press release, OnKure, MAY 6, 2019, View Source [SID1234535770]).

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OnKure’s platform is capable of transforming naturally occurring, biologically active lead compounds into commercial drugs that address a variety of diseases, with an emphasis on helping patients suffering from cancer. At the heart of OnKure’s solution to Class 1 histone deacetylase inhibition, OKI-179 is a drug with a potency and selectivity profile that is reminiscent of Romidepsin, arguably the most potent Class 1 histone deacetylase inhibitor currently approved by the FDA. However, while Romidepsin is administered by continuous infusion, OKI-179 is delivered orally and has demonstrated an impressive safety profile in preclinical studies.

"We are delighted that OKI-179 is poised to enter clinical development," said Tony Piscopio, co-founder, President and Chief Executive Officer of OnKure. "We are excited to see how this potential best-in-class molecule will perform in carefully selected patient populations."

Collaborating with OnKure on the clinical investigation of OKI-179 will be the University of Colorado, Anschutz Cancer Center, Aurora, CO and the University of Texas, Austin Dell Medical School.

"We are very excited to begin our first in human studies with OKI-179," said S. Gail Eckhardt, M.D., FASCO, OnKure’s Chief Medical Officer and Professor at UT Austin. She added, "Our preclinical data suggests that OKI-179 may provide patients with a safer and more convenient alternative to Romidepsin, both as a stand-alone agent and in rational combinations with other targeted therapies."

On May 6, 2019 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported financial results for the first quarter ended March 31, 2019, and provided a corporate update (Press release, Cellectar Biosciences, MAY 6, 2019, View Source [SID1234535788]).

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First Quarter and Recent Corporate Highlights

·Announced additional positive top-line results from a relapsed/refractory multiple myeloma cohort in the ongoing Phase 2 clinical study of CLR 131, the Company’s lead product candidate. In the cohort, CLR 131 achieved a 30% overall response rate in the first 10 evaluable patients. Patients received one 30-minute infusion of 25mCi/m2 and CLR 131 represented, on average, seventh line treatment. The Company previously announced an overall response rate of 33% as the fourth line average treatment in patients with R/R diffuse large B-cell lymphoma (DLBCL) also receiving the single, 25mCi/m2 dose of CLR 131.

·Announced median overall survival (mOS) of 22 months in Cohorts 1-4 of the Company’s ongoing Phase 1 clinical trial evaluating CLR 131 for the treatment of relapsed/refractory multiple myeloma. The mOS results were from 15 patients, all of whom were heavily pretreated and averaged five prior lines of systemic therapy.

·Received an exemption from the U.S. Food and Drug Administration (FDA) to the Import Alert placed on the Centre for Probe Development and Commercialization (CPDC) for the use of CLR 131 in connection with the Company’s pediatric Investigational New Drug Application (IND). This exemption has allowed Cellectar to initiate patient enrollment in its Phase 1 pediatric study for the treatment of select relapsed or refractory solid tumors including neuroblastoma, lymphomas and malignant brain tumors.

"The data we have seen thus far for CLR 131 are very compelling with demonstrated activity in at least 3 hematologic cancers. We continue to make significant progress in the clinic and are excited to now provide this promising treatment to pediatric cancer patients," said James Caruso, president and CEO of Cellectar. "We believe CLR 131 has the potential to be a meaningful part of the treatment regimine for patients battling life-threatening cancers and look forward to continuing to provide updates on our studies throughout 2019."

First Quarter Financial Highlights

Research and Development Expense: Research and development expense for the three months ended March 31, 2019 was $2.3 million, compared to $2.1 million in the three months ended March 31, 2018. The overall increase in research and development expense of $184,000, or 8%, was primarily a result of an increase in clinical project costs of approximately $285,000 related to the start-up of the pediatric study. Manufacturing and related costs increased as a result of an increase in patient recruitments for the on-going clinical trials. Pre-clinical studies decreased as some studies were concluding. The general research and development costs were relatively consistent.

General and Administrative Expense: General and administrative expense for the three months ended March 31, 2019 was approximately $1,321,000, compared to approximately $1,329,000 in the three months ended March 31, 2018 and remained relatively consistent.

Net Loss: Net loss for the three months ended March 31, 2019 was $(3.6) million, or a loss of $(0.76) per diluted share, compared to a net loss of $(3.5) million, or a loss of $(2.07) per diluted share, in the three months ended March 31, 2018.

Cash and Cash Equivalents: As of March 31, 2019, cash and cash equivalents were approximately $10.5 million compared to $13.3 million as of December 31, 2018. The Company believes this cash balance is adequate to fund our pipeline development and operations into the first quarter 2020.

About CLR 131

CLR 131 is a small-molecule, cancer-targeting radiotherapeutic PDC (proprietary phospholipid drug conjugate) designed to deliver cytotoxic radiation directly and selectively to cancer cells and cancer stem cells. CLR 131 is our lead therapeutic PDC product candidate and is currently being evaluated in both Phase 2 and Phase 1 clinical studies. In December 2014, the FDA granted orphan drug designation for CLR 131 for the treatment of multiple myeloma. In 2018, the FDA granted orphan drug and rare pediatric disease designations for CLR 131 for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. The FDA previously accepted our IND application for a Phase 1 open-label, dose-escalating study to evaluate the safety and tolerability of a single intravenous administration of CLR 131 in up to 30 children and adolescents with cancers including neuroblastoma, sarcomas, lymphomas (including Hodgkin’s lymphoma) and malignant brain tumors.

On May 6, 2019 DiaMedica Therapeutics Inc. (Nasdaq: DMAC) reported that its first quarter 2019 financial results will be released after market close on Monday, May 13th (Press release, DiaMedica, MAY 6, 2019, View Source [SID1234535751]). DiaMedica’s management will host a live conference call on Tuesday, May 14th at 7:00am Central Time to provide a business update and discuss the Company’s financial results.

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Conference Call details:

Date: Tuesday, May 14, 2019
Time: 7:00 AM CT
Web access: View Source
Dial In: (866) 962-3583 (domestic)
(630) 652-5857 (international)
Conference ID: 8177137
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 May 21, 2019, by dialing 1(855) 859-2056 (US Toll Free Dial In), (404) 537-3406 (international), replay passcode 8177137.

On May 6, 2019 Shattuck Labs, Inc. ("Shattuck"), a biopharmaceutical company, reported that patients are being treated in its Phase 1 dose escalation and expansion clinical trial of its molecule SL-279252 (PD1/OX40L), a bi-functional fusion protein (View Source) (Press release, Shattuck Labs, MAY 6, 2019, View Source [SID1234535771]). Sarah Cannon Research Institute in Nashville, Tennessee and MD Anderson Cancer Center in Houston, Texas are the first enrolling sites in this multi-center, global trial. This first-in-human study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamic, and anti-tumor activity of SL-279252 in patients with advanced solid tumors or lymphomas. Takeda Pharmaceutical Company Limited currently holds an exclusive option to enter into a license to develop and commercialize SL-279252. Further information about this trial can be found on clinicaltrials.gov.

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"We are excited to have initiated clinical studies for SL-279252. While some patients with cancer enjoy long-term benefit from antibody-based PD-1 blockade, a majority of patients unfortunately do not," explained Lini Pandite, M.D., Chief Medical Officer of Shattuck. "Pre-clinical studies demonstrate that SL-279252 binds simultaneously and with high affinity to PD-L1 and OX40, and stimulates anti-tumor T cell activity. Pre-clinical studies further demonstrate improved pharmacologic and anti-tumor activity compared to antibody-based PD-1 blockade, either alone or in combination with antibody-based OX40 stimulation. We look forward to learning more about SL-279252 in the clinic and expect to gain insight into whether it can improve upon antibody-based PD-1 blockade as a standard of care in multiple tumor types."

SL-279252 is a novel therapeutic derived from Shattuck’s proprietary Agonist Redirected Checkpoint (ARC) platform and its first molecule to begin clinical trials. The dual-sided nature of SL-279252 is designed to simultaneously block the PD-L1 inhibitory signal and stimulate OX40 signaling. Preclinical studies have demonstrated that SL-279252 potently stimulates anti-tumor T cell activity.

"Shattuck’s ARC platform technology combines checkpoint blockade with immune stimulation representing an approach that is highly differentiated from antibody-based platforms," said Phil Rowlands, Ph.D., Head, Oncology Therapeutic Area Unit, Takeda. "It is a great example of our commitment to collaborating with world-class partners to pursue novel immuno-oncology targets and next-generation platforms, that may one day deliver transformational medicines to patients."