On November 13, 2019 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for the third quarter of 2019 (Press release, Y-mAbs Therapeutics, NOV 13, 2019, View Source [SID1234551145]).

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"We are very pleased with our third quarter results, highlighted by prudent spending combined with notable progress in the preparation of our BLAs for naxitamab and omburtamab, as well commercial ramp-up for the potential launch of both compounds. In addition, we succesfully completed a follow-on offering in November, securing gross proceeds of $143.8 million, which we believe will – together with our existing cash – be sufficient to cover our operating costs through 2022," stated Thomas Gad, Founder, Chairman, President and Head of Business Development and Strategy.

Dr. Claus Moller, Chief Executive Officer, continued, "Over the third quarter, we have continued to work hard to make sure that our lead product candidates, naxitamab and omburtamab, advance towards rolling BLA submissions. We still expect the first portion of the rolling BLA for naxitamab to be submitted within the next few weeks, and we expect to file our BLA submission for omburtamab either via a rolling submission to begin in 2019 or early 2020 or via a single submission ahead of our expected completion date at the end of the first quarter of 2020.

Third Quarter 2019 and Recent Corporate Developments

Subsequent to the end of the third quarter, on November 1, 2019, Y-mAbs announced the pricing of a follow-on shelf public offering, resulting in gross proceeds to the Company of approximately $143.8 million.

·Also, subsequent to the end of the third quarter, on November 1, 2019, Y-mAbs announced that the requested omburtamab pre-BLA meeting with the FDA, had been converted to a general guidance meeting. Y-mAbs still expects to complete the omburtamab BLA submission by the end of the first quarter of 2020, and believes that the overall commercialization timeline will not be affected.

·Also, subsequent to the end of the third quarter, on October 28, 2019, Y-mAbs announced an update on omburtamab data, which was presented at the Interntional Society of Pediatric Oncology conference.

·After the close of the third quarter, on October 25, 2019, Y-mAbs announced an update on naxitamab data, which was presented at the Interntional Society of Pediatric Oncology conference.

On August 30, 2019, Y-mAbs announced the acceptance of abstracts concerning DSRCT for presentation at the Connective Tissue Oncology Society (CTOS) Annual Meeting for omburtamab.

· On July 8, 2019, Y-mAbs announced that it had completed a successful pre-BLA meeting with the FDA regarding a potential pathway for FDA approval of naxitamab for the treatment of relapsed/refractory high-risk neuroblastoma. During the meeting, the Company reached alignment with the FDA on an Accelerated Approval Pathway for naxitamab along with a rolling BLA submission.

· On July 1, 2019, Y-mAbs announced the status of patient recruitment for the Company’s two pivotal phase II trials, one for omburtamab for the treatment of central nervous system/leptomeningeal metastasis (CNS/LM) from neuroblastoma and the other for naxitamab for the treatment of relapsed/refractory high-risk neuroblastoma.

· Also, on July 1, 2019, Y-mAbs announced that the Company has entered into a development, manufacturing and supply agreement with SpectronRx in South Bend, Indiana, to secure access to clinical and commercial scale radiolabeling capacity for omburtamab. Under the terms of the agreement, SpectronRx has agreed to establish a manufacturing unit designated for Y-mAbs within its existing

facilities, at which Y-mAbs believes both clinical and commercial supply of radiolabeled omburtamab can be produced.

Third Quarter 2019 Financial Results

Y-mAbs reported a net loss of $23.9 million, or $0.70 per basic and diluted share, for the three months ended September 30, 2019, compared to a net loss of $11.4 million, or $0.42 per basic and diluted share, for the three months ended September 30, 2018.

For the nine months ended September 30, 2019, Y-mAbs reported a net loss of $57.9 million, or $1.69 per basic and diluted share, compared to a net loss of $29.2 million, or $1.08 per basic and diluted share, reported for the nine months ended September 30, 2018.

Operating Expenses

Research and Development

Research and development expenses were $19.7 million for the three months ended September 30, 2019, compared to $8.7 million for the three months ended September 30, 2018, an increase of $11.0 million. The increase in research and development expenses primarily reflects the following:

·$7.3 million increase in outsourced manufacturing for our two lead product candidates, naxitamab and omburtamab;

· $1.5 million increase in outsourced research and supplies to support expanding development activities; and

· $0.7 million increase in personnel costs.

Research and development expenses were $46.7 million for the nine months ended September 30, 2019, compared to $23.2 million for the nine months ended September 30, 2018, an increase of $23.5 million. The increase in research and development expenses primarily reflects the following:

· $14.5 million increase in outsourced manufacturing for our two lead product candidates, naxitamab and omburtamab;

· $5.0 million increase in outsourced research and supplies to support expanding development activities; and

·$1.9 million increase in personnel costs.

General and Administration

General and administrative expenses were $4.7 million for the three months ended September 30, 2019, compared to $2.7 million for the three months ended September 30, 2018, an increase of $2.0 million. Such increase in general and administrative expenses primarily reflects the following:

· $0.8 million increase in personnel costs; and

·$0.6 million increase in commercial infrastructure.

General and administrative expenses were $12.6 million for the nine months ended September 30, 2019, compared to $5.9 million for the nine months ended September 30, 2018, an increase of $6.7 million. Such increase in general and administrative expenses primarily reflects the following:

·$3.6 million increase in personnel costs; and

· $1.4 million increase in commercial infrastructure costs.

Cash and Cash Equivalents

The Company had approximately $98.2 million in cash and cash equivalents as of September 30, 2019, compared to $147.8 million as of December 31, 2018. The decrease of $49.6 million was primarily attributable to the increased costs of operation as the Company prepares for its submission of rolling BLAs for naxitamab and omburtamab and the build-up of the Company’s commercial infrastructure.

Webcast and Conference Call

The Company will host a conference call today at 4:30 pm eastern time. To participate in the call, please dial 877-407-0792 (domestic) or 201-689-8263 (international) and reference the access code 13696442. A webcast will be available at: View Source

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.

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 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INSTASYL) therapeutic platform, reported that it has commenced a proposed public offering of shares of its common stock (Press release, Phio Pharmaceuticals, NOV 13, 2019, View Source [SID1234551142]). All of the shares in the offering are to be sold by Phio. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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H.C. Wainwright & Co. is acting as exclusive placement agent for the offering.

Phio intends to use the net proceeds from this offering to fund the development of Phio immuno-oncology programs, for other research and development activities and for general working capital needs.

The shares of common stock described above are being offered by Phio pursuant to a shelf registration statement on Form S-3 (File No. 333-224031) filed with the Securities and Exchange Commission (the "SEC") on March 29, 2018 and declared effective by the SEC on April 6, 2018. The offering may be made only by means of a base prospectus and a related prospectus supplement. A preliminary prospectus supplement and accompanying prospectus related to the offering will be filed with the SEC. When available, electronic copies of the preliminary prospectus supplement and accompanying prospectus may be obtained by contacting H.C. Wainwright & Co., LLC, 430 Park Avenue, 3rd Floor, New York, New York 10022, via e-mail at [email protected] or via telephone at (212) 356-0530.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On November 13, 2019 IDEAYA Biosciences, Inc. (Nasdaq:IDYA), an oncology-focused precision medicine company committed to the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics,reported financial results for the third quarter ended September 30, 2019 (Press release, Ideaya Biosciences, NOV 13, 2019, View Source [SID1234551141]).

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"We have made significant progress on our Phase 1/2 tissue-agnostic basket trial evaluating IDE196 in patients with solid tumors harboring GNAQ or GNA11 (GNAQ/11) mutations, including metastatic uveal melanoma (MUM), cutaneous melanoma and colorectal cancer. This clinical trial progress, considered together with recent FDA feedback providing guidance on the regulatory pathway for IDE196 for treatment of MUM, are important enabling steps to advance this clinical-stage program," said Yujiro S. Hata, Chief Executive Officer and President at IDEAYA Biosciences.

IDEAYA continues to advance its MAT2A synthetic lethality program to treat patients having tumors with MTAP deletion. The Company remains committed to the research and development of a differentiated MAT2A inhibitor that could meaningfully enhance patient outcomes across a broad range of indications. IDEAYA also continues to progress its broad pipeline of synthetic lethality programs, including Pol theta for patients having tumors with BRCA or other homologous recombination deficiency (HRD) mutations, and Werner (WRN) for patients having tumors with high microsatellite instability (MSI).

Key highlights for IDEAYA’s research and development programs include:

Clinical Program IDE196

IDE196

Advanced IDEAYA’s Phase 1/2 tissue-type agnostic basket trial, which was initiated in June 2019 to evaluate IDE196 in solid tumors harboring activating GNAQ/11 mutations, and is entitled "A phase 1/2 study of IDE196 in patients with solid tumors harboring GNAQ/11 mutations or PRKC fusions" (ClinicalTrials.gov Identifier: NCT03947385)

Completed enrollment in the Phase 1 dose escalation portion of the Phase 1/2 clinical trial at sites in the U.S. and Australia

Anticipate selection of the dosing regimen and initiation of the Phase 2 portion of the clinical trial by year-end 2019

Expect to introduce a tablet formulation of IDE196 in Q1 2020 for use in the Phase 2 portion of the clinical trial

Enrolled our first cutaneous melanoma patient harboring a tumor with an activating GNAQ/GNA11 mutation in October 2019, a key milestone for evaluating IDE196 in solid tumors outside of metastatic uveal melanoma (MUM)

Expanded our relationship with Foundation Medicine, participating in their Smart Trials program for identification of non-MUM patients having tumors with activating GNAQ/11 mutations for potential enrollment in our Phase 1/2 basket trial

Anticipate release of interim data from the Phase 1/2 basket trial in Q2/Q3 2020

Obtained FDA feedback in End-of-Phase 1 meeting in Q4 2019, providing guidance on the regulatory pathway for IDE196 for treatment of MUM

Company’s proposed single-arm Phase 2 IDE196‑001 clinical trial may be adequate to support an NDA seeking Accelerated Approval for IDE196 monotherapy in metastatic uveal melanoma (MUM)

The single-arm potentially registration‑enabling Phase 2 clinical trial will target enrollment of 60 evaluable MUM patients

The primary endpoint is overall response rate (ORR) as determined by blinded independent central review (BICR), supported by

BICR‑determined duration of response (DOR) as a secondary endpoint

The 13-week GLP-compliant toxicology studies in 2 species is scheduled to initiate in November 2019, in support of FDA requirement that study results be submitted prior to enrollment of more than approximately 50 patients in the investigational arm of the clinical trial that will support a marketing application

Continued the ongoing Phase 1 clinical trial, entitled "A Phase I, multi-center, open-label, study of LXS196, an oral protein kinase C inhibitor, in patients with metastatic uveal melanoma" (ClinicalTrials.gov Identifier: NCT02601378), being conducted by Novartis

A confirmed Complete Response at the 200 mg BID dose level was observed at month 31 in one of four patients previously reported with confirmed Partial Response out of 30 total (28 evaluable) BID patients in this ongoing monotherapy arm of the Novartis clinical trial

Based on confirmation of the single arm trial design and anticipated rate of enrollment, we are anticipating submission of a New Drug Application (NDA) to the FDA in Q4 2021 to Q1 2022 for IDE196 in MUM.

Synthetic Lethality Preclinical Pipeline

MAT2A

We believe that data presented at the AACR (Free AACR Whitepaper)/NCI/EORTC conference in October 2019 demonstrates clinical activity of MAT2A as a biological target in patients having tumors with MTAP deletion.

Continuing our preclinical evaluation of potential drug candidates, including in the HCT116 engineered in-vivo model, and in multiple endogenous in-vivo models

Goal is to select a MAT2A inhibitor development candidate in the first half of 2020 with differentiated properties relative to Agios’ presented published compounds and Agios’ publicly-disclosed properties of AG270

Expect to file an IND for a differentiated MAT2A inhibitor development candidate in second half of 2020

"We continue to aggressively progress our programs, expand our capabilities and enhance our team. IDEAYA remains committed to its vision of improving lives through transformative precision medicines, and believes that the IDE196 tissue-type agnostic basket trial and our pipeline of synthetic lethality programs are key elements of this goal," said Yujiro S. Hata, Chief Executive Officer and President at IDEAYA Biosciences.

Corporate Updates

IDEAYA anticipates that existing cash, cash equivalents and short-term marketable securities of $109.4 million (as of September 30, 2019) will be sufficient to fund planned operations into the third quarter of 2021.

Financial Results

As of September 30, 2019, IDEAYA had cash, cash equivalents and short-term marketable securities totaling $109.4 million. This compared to cash, cash equivalents and short-term marketable securities of $90.0 million at December 31, 2018. The increase was primarily due to the receipt of $50.2 million in net proceeds from IDEAYA’s initial public offering, which was completed in May 2019, offset by cash used in operations.

Research and development expenses for the three months ended September 30, 2019 totaled $8.9 million compared to $12.5 million for the same period in 2018. The decrease was primarily due to license fees for our IDE196 license agreement with Novartis during the three months ended September 30, 2018, offset by an increase in costs in connection with IDEAYA’s Phase 1/2 clinical trial to evaluate IDE196 in solid tumors, and costs for personnel, consulting, and laboratory supplies in support of our research programs during the three months ended September 30, 2019.

General and administrative expenses for the three months ended September 30, 2019 totaled $2.7 million compared to $1.1 million for the same period in 2018. The increase was primarily due to an increase in costs for personnel, directors’ and officers’ liability insurance premiums, and professional fees in connection with becoming a publicly traded company as well as external legal patent expenses for our product candidates.

The net loss for the three months ended September 30, 2019 was $11.0 million compared to $13.0 million for the same period in 2018. Total stock compensation expense for the three months ended September 30, 2019 was $0.5 million compared to $0.3 million for the same period in 2018.