On May 6, 2019 ERYTECH Pharma (Euronext: ERYP—Nasdaq: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported a business update and reports its financial results for the quarter ended March 31, 2019 (Press release, ERYtech Pharma, MAY 6, 2019, View Source [SID1234536954]).

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"The first quarter of 2019 has been particularly intense in terms of execution of our late stage clinical trials and the extension of our manufacturing capacity, all while making progress on our earlier stage programs. Patient enrollment for TRYbeCA 1, our pivotal Phase 3 trial in second-line metastatic pancreatic cancer, continues ahead of plan in Europe, and we recently filed an IND in the United States in order to begin enrollment there," said Gil Beyen, CEO of ERYTECH. "Construction of the new manufacturing site in Princeton and the expansion of our Lyon manufacturing facility have been completed and validation is now ongoing."

Recent Business Highlights

TRYbeCA1, the pivotal Phase 3 trial evaluating ERYTECH’s lead product candidate eryaspase in second-line metastatic pancreatic cancer, is actively enrolling patients in several European countries. In view of opening the trial to patients in the United States, the company recently submitted an IND application to the US FDA. In this trial, ERYTECH is evaluating eryaspase in combination with standard chemotherapy (gemcitabine/nab-paclitaxel or an irinotecan-based regimen) compared to standard chemotherapy alone. The primary endpoint of the trial is overall survival. An interim efficacy analysis is planned when approximately two-thirds of events have occurred. The trial is expected to enroll approximately 500 patients at more than 120 sites in Europe and the United States.

TRYbeCA2, a Phase 2 proof-of-concept trial in patients with previously untreated metastatic triple-negative breast cancer (TNBC), is evaluating eryaspase in combination with gemcitabine and carboplatin chemotherapy, compared to chemotherapy alone in approximately 64 patients. The primary endpoint is objective response rate. First sites were initiated in December 2018, and the trial is now open for enrollment in Spain and France.

To ensure supply of the product for the clinical trials and potential initial commercial needs, the Company is establishing a GMP manufacturing facility in Princeton, New Jersey. It is also expanding its European manufacturing capacity in Lyon, France. Construction has now been completed at both sites and validation activities are ongoing, as planned.

ERYTECH is also advancing the preclinical programs to leverage its proprietary ERYCAPS encapsulation platform. The Company’s lead preclinical program and next product candidate is erymethionase, methionine-gamma-lyase encapsulated in red blood cells. Activities in support of initiating a Phase 1 clinical trial of erymethionase in solid tumor indications are ongoing. The company expects to begin this Phase 1 trial in Europe in the first quarter of 2020.

In preparation for the next stage of the company’s development, ERYTECH recently announced that its Board of Directors will propose the appointment of Jean-Paul Kress as a Director at the Annual General Meeting on June 21, 2019, with a view to appointing him as Chairman of the Board of Directors. Dr. Kress has over 25 years’ experience as a senior executive in international biotech and pharma groups. He was President and Chief Executive Officer of Syntimmune (Cambridge, MA, US) until the end of 2018, when the company was acquired by Alexion Pharmaceuticals.

Q1 2019 Financial Results

Net loss for the first quarter of 2019 was €11.7 million, stable from the same period in 2018. The €4.0 million increase in operating loss was offset in the period by the €4.1 million improvement in financial Income. The €4.0 million increase in operating loss was attributable to the €1.5 million increase in preclinical and clinical development expenses, mostly related to the Phase 3 clinical trial in pancreatic cancer, the €2.1 million increase in G&A expenses, primarily driven by professional fees in relation to the launch readiness of the Company’s additional production capacity, and the €0.4 million decrease in research tax-credit income. The €4.1 million improvement in financial Income was mainly related to the translation into Euro of the portion of the Company’s cash position denominated in U.S. Dollar, which had a positive foreign exchange impact and explained most of the €1.6 million financial Income in the first quarter of 2019, compared to a negative foreign exchange impact and a €2.5 million financial expense in the first quarter of 2018.

As of March 31, 2019, ERYTECH had cash and cash equivalents totaling €110.5 million (approximately $124 million), compared with €134.4 million on December 31, 2018. The €23.9 million decrease in cash position in the first quarter of 2019 was the result of a €25.2 million net cash utilization, comprised of a €15.9 million net cash utilization in operating activities, €8.9 million in capital expenditures and €0.4 million in loan and lease reimbursement, while the appreciation in the period of the U.S. Dollar against the Euro lead to a €1.3 million favorable currency exchange impact. Cash utilization in the first quarter of 2019 was, as anticipated, relatively high due to the non-recurring capital expenditure disbursements

for the production facility expansions in Lyon and in Princeton. As both projects are now close to completion, cash utilization is expected to be lower again in the coming quarters and the Company continues to expect its cash resources to be sufficient to fund operations until the end of 2020.
Key News Flow and Milestones Expected over Next 12 Months

First patient enrolled in TRYbeCA-2, Phase 2 proof-of-concept clinical trial in TNBC

Start of GMP production at Princeton facility and Lyon extension

Start of US patient enrollment in TRYbeCA 1, Phase 3 trial in second-line pancreatic cancer

Initiation of Phase 1 clinical trial with erymethionase

Q1 2019 Conference Call Details

ERYTECH management will hold a conference call and webcast on Tuesday, May 7th, 2019 at 02:30pm CEST / 08:30am EDT on business highlights and financial results for the first quarter of 2019. Gil Beyen, CEO, Eric Soyer, CFO/COO, and Iman El-Hariry, CMO, will deliver a brief presentation, followed by a Q&A session.

The call is accessible via the below teleconferencing numbers, followed by the Conference ID#: 2199912#:

USA/Canada: +1 (833) 818-6807

International Dial-In Number: +1 (409) 350-3501

France: +33 1 70 80 71 53

United-Kingdom: +44 2031070289

The webcast can be followed live online via the link: View Source

An archived replay of the call will be available for 7 days by dialing + 1 855 859 2056, Conference ID: 2199912#

An archive of the webcast will be available on ERYTECH’s website, under the "Investors" section at investors.erytech.com

2019 Financial Calendar:

General Assembly Meeting of Shareholders: Friday, June 21, 2019 at 9:30am CET in Paris

Quarterly financial updates:

Business Update and Financial Highlights for the 2nd quarter and first-half of 2019: September 17, 2019 (after U.S. market close), followed by a conference call and webcast on September 18, 2019 (2:30pm CET/8:30am ET)

Business Update and Financial Highlights for the 3rd quarter of 2019: November 7, 2019 (after U.S. market close), followed by a conference call and webcast on November 8, 2019 (2:30pm CET/8:30am ET)

ERYTECH will Present at the Following Upcoming Investor Conferences:

Gilbert Dupont Annual Healthcare Conference, May 23, Paris

Jefferies 2019 Global Healthcare Conference, June 4-7, New-York

European Midcap Event – Spring, June 18-19, Paris

JMP Securities Life Sciences Conference, June 19, New York

France Biotech, Health Tech Investor Day, June 25, Paris

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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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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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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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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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.