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.

On November 13, 2019 Brickell Biotech, Inc. ("Brickell") (Nasdaq: BBI), a clinical-stage pharmaceutical company focused on developing innovative and differentiated prescription therapeutics for the treatment of debilitating skin diseases, reported financial results for the third quarter ended September 30, 2019 and provided a corporate update (Press release, Vical, NOV 13, 2019, View Source [SID1234551140]).

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"Becoming a publicly listed company earlier this year was a transformative milestone for Brickell and we remain confident about the potential of our lead asset, sofpironium bromide, for primary axillary hyperhidrosis," commented Robert Brown, Chief Executive Officer of Brickell. "Ten million people in the United States suffer from this disease which can have a negative impact on a patient’s social life, well-being, emotional and mental health."

Business and Recent Developments

In September 2019, Brickell announced completion of its merger with Vical Incorporated ("Vical"), following approval by Vical’s shareholders, and commenced trading on The Nasdaq Capital Market under the ticker symbol "BBI". Vical contributed approximately $35 million to the combined company in addition to an R&D financing arrangement entered into with NovaQuest Capital Management that provides up to $25 million in funding.

The long-term safety study for sofpironium bromide is fully enrolled with 300 subjects and is on track to be completed in the first quarter of 2020. Earlier this year, Brickell’s development partner, Kaken Pharmaceutical Co. Ltd. ("Kaken"), achieved positive pivotal Phase 3 results in its clinical study conducted in Japan. To date there have been 19 clinical studies conducted by Brickell and Kaken of sofpironium bromide gel that encompass over 1,200 subjects.

On October 23, 2019, Bodor Laboratories, Inc. and Nicholas S. Bodor (collectively, "Bodor") filed a complaint against Brickell disputing certain aspects of the license agreement between the parties with respect to sofpironium bromide ("Complaint"). As a result, NovaQuest notified the Company that additional development funding for sofpironium bromide was suspended temporarily. Subsequently, Brickell filed a motion to dismiss the Complaint, initiated arbitration proceedings against Bodor and asserted claims against Bodor for tortious interference and material breach of the license agreement by Bodor.

The sofpironium bromide pivotal Phase 3 studies in axillary hyperhidrosis are ready to commence in the United States, pending developments in the ongoing dispute resolution process with Bodor.

Financial Results

Cash, cash equivalents, and short-term investments were $25.7 million as of September 30, 2019 compared to $8.1 million as of September 30, 2018.

Revenue was $1.2 million for the third quarter of 2019 compared to $3.0 million for the third quarter of 2018. The decrease is due primarily to the completion of certain research and development activities during the three months ended September 30, 2019 for which funding is provided under a license and collaboration agreement with Kaken.

Research and development expenses were $3.3 million for the third quarter of 2019 compared to $4.1 million for the third quarter of 2018. The decrease in research and development expenses is primarily due to a decrease in clinical studies costs associated with sofpironium bromide following the completion of certain clinical trials.

General and administrative expenses were $3.9 million for the third quarter of 2019 compared to $1.2 million for the third quarter of 2018. The increase in general and administrative expenses is primarily due to an increase in professional fees for legal, accounting, and auditing services, including merger-related costs.

The Company’s net loss was $4.8 million for the third quarter of 2019, and $13.0 million for the nine months ended September 30, 2019, compared to $2.5 million for the third quarter of 2018, and $5.6 million for the nine months ended September 30, 2018.

About Sofpironium Bromide

Sofpironium bromide, is a proprietary new molecular entity that belongs to a class of medications called anticholinergics. Anticholinergics block the action of acetylcholine, a chemical that transmits signals within the nervous system that are responsible for a range of bodily functions, including activation of the sweat glands. Sofpironium bromide was retrometabolically designed. Retrometabolic drugs are designed to exert their action topically and are potentially rapidly metabolized once absorbed into the blood. This proposed mechanism of action may allow for highly effective doses to be used while limiting systemic side effects.

About Hyperhidrosis

Hyperhidrosis is a life-altering medical condition where a person sweats more than the body requires to regulate its temperature. More than 15 million people, or 4.8% of the population of the United States, are believed to suffer from hyperhidrosis. Axillary (underarm) hyperhidrosis is the targeted first indication for sofpironium bromide and is the most common occurrence of hyperhidrosis, affecting an estimated 65% of patients in the United States or 10 million individuals. Doolittle et al. Arch Dermatol Res (2016).

On November 13, 2019 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for cancer, reported financial results for the third quarter ended September 30, 2019 (Press release, Personalis, NOV 13, 2019, View Source [SID1234551139]).

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Third Quarter 2019 Highlights

Record revenues of $17.2 million in the third quarter of 2019, versus $11.7 million in the third quarter of 2018, an increase of 47%

Received a new $38.1 million task order from the VA’s Million Veteran Program, increasing our unfulfilled orders with the VA MVP to approximately $82.5 million

Announced important customer and partner relationships, including agreements with Invectys, the University of New Mexico and the New Mexico Cancer Care Alliance

"I’m very pleased that we again achieved record quarterly revenues and grew 47% over the third quarter of last year. In September, we received a new order from the VA’s Million Veteran Program, which increased our cumulative orders received to date from the VA MVP to approximately $145 million," said John West, Chief Executive Officer. "In addition, many of our biopharmaceutical and prospective customers are actively evaluating our new platform ImmunoID NeXT and feedback has been resoundingly positive. We continue to anticipate ramping revenue volume of the NeXT platform increasingly throughout 2020."

Third Quarter 2019 Financial Results

Revenues were $17.2 million in the three months ended September 30, 2019, up 47% from $11.7 million in the same period of the prior year. Third quarter revenue growth was driven by an increase in volume for testing and analytical services provided to the U.S. Department of Veterans Affairs Million Veteran Program (VA MVP). In the third quarter, the VA MVP accounted for $12.9 million, or 75%, of revenues and the remaining $4.3 million, or 25%, was from biopharma and all other customers.

Gross margin was 32.8% for the three months ended September 30, 2019, compared with 38.5% in the same period of the prior year.

Operating expenses were $11.4 million for the three months ended September 30, 2019, compared with $6.2 million in the same period of the prior year.

Net loss was $6.9 million for the three months ended September 30, 2019 and net loss per share was $0.22 based on a weighted-average basic and diluted share count of 31.1 million, compared with a net loss of $3.6 million and a net loss per share of $1.19 on a weighted-average basic and diluted share count of 3.1 million last year.

Cash, cash equivalents, and short-term investments were $127.3 million as of September 30, 2019.

2019 and 2020 Outlook

Personalis expects full year 2019 revenues to be in the range of $64.5 million to $65.0 million, and full year 2020 revenues to be in the range of $77.5 million to $83.5 million.

Webcast and Conference Call Information

Personalis will host a conference call to discuss the third quarter financial results after market close on Wednesday, November 13, 2019 at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time. The conference call can be accessed live over the phone (866) 220-8061 for U.S. callers or (470) 495-9168 for international callers, using conference ID: 8749318. The live webinar can be accessed at View Source

On November 13, 2019 Alpine Immune Sciences, Inc. (NASDAQ:ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer, autoimmune/inflammatory, and other diseases, reported a corporate update and financial results for the third quarter ended September 30, 2019 (Press release, Alpine Immune Sciences, NOV 13, 2019, View Source [SID1234551138]).

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"The third quarter of 2019 included several significant milestones for Alpine, highlighted by completion of enrollment in our Phase 1 study in healthy volunteers for our lead program, ALPN-101," said Mitchell H. Gold, MD, Executive Chairman and Chief Executive Officer of Alpine. "We believe ALPN-101 has the potential to deliver significantly differentiated efficacy outcomes across a range of inflammatory and autoimmune diseases. We look forward to highlighting our upcoming Phase 1/2 BALANCE study of ALPN-101 during an oral presentation at the ASH (Free ASH Whitepaper) annual meeting next month."

"In addition, we are actively preparing for the initiation of the Phase 1 NEON-1 study of our lead immuno-oncology program, ALPN-202," Dr. Gold said. "At Alpine, we share a deep commitment and drive to deliver the next generation of immunotherapies to patients suffering from debilitating and deadly diseases and I am proud of the work our entire team has done in the transformation of Alpine to an established, clinical-stage company."

Recent Pipeline and Company Highlights

ALPN-101 Clinical Advancement

Completed enrollment of Phase 1 Study of ALPN-101: On October 30, 2019, Alpine announced completion of enrollment of its Phase 1 study of ALPN-101, a first-in-class dual ICOS/CD28 antagonist. Results from this trial will help inform further development of ALPN-101 in serious autoimmune and inflammatory diseases.
Overall, ALPN-101 was generally well-tolerated, without evidence of cytokine storm, cytokine release, or clinical immunogenicity while being dosed as single or multiple doses, intravenously or subcutaneously.
Preliminary analyses indicate well-behaved pharmacokinetics and pharmacodynamics, including on-target inhibition of immune functionality, such as antibody responses to keyhole limpet hemocyanin (KLH) and ex vivo staphylococcal enterotoxin B (SEB)-induced cytokine responses.
These initial findings will be included as part of an oral presentation at ASH (Free ASH Whitepaper) next month, and further details after completion of final analyses are expected to be reported in presentations in the first half of 2020.
New ALPN-101 Data Presented at American College of Rheumatology (ACR) Annual Meeting: Earlier this week, Alpine presented new preclinical data on ALPN-101 in two posters during sessions at ACR. The new data highlighted ALPN-101’s novel dual mechanism of action which modulates unique inflammatory pathways, distinct from the other biologic therapies singly targeting the ICOS or CD28 pathways, and results in potent efficacy in multiple disease models. The full posters from ACR can be viewed here.
Upcoming Oral Presentation at American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting: Alpine will present on its upcoming Phase 1/2 BALANCE study of ALPN-101 in steroid-resistant or steroid-refractory acute Graft-versus-Host Disease (GvHD). Dr. Jan Hillson, Senior Vice President, Clinical Development for Alpine, will present on December 8, 2019 at 11:00 a.m. ET. The full abstract can be viewed here.
ALPN-202 Preparing for Phase I Study

New ALPN-202 Data Presented at Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting: On November 8, 2018, Alpine presented two posters with new preclinical data on its lead oncology program ALPN-202, a conditional CD28 costimulator and dual checkpoint inhibitor. The new preclinical data further highlight ALPN-202’s novel mechanism of action and its superior anti-tumor activity, as well as its potential to be uniquely effective as a monotherapy and in combination with immune checkpoint inhibitors or chemotherapy. The full posters from SITC (Free SITC Whitepaper) can be viewed here.
Data presented at SITC (Free SITC Whitepaper) supports the advancement of ALPN-202 towards its first Phase 1 clinical trial, which Alpine expects to initiate, upon authorization, in the first quarter of 2020.
Financial Results for Third Quarter and Nine Months Ended September 30, 2019

Alpine recorded a net loss of $11.5 million and $12.1 million for the third quarters ended September 30, 2019 and 2018, respectively, and $35.7 million and $25.4 million for the nine months ended September 30, 2019 and 2018, respectively.

Research and development expenses for the third quarter ended September 30, 2019 were $9.5 million compared to $10.5 million for the third quarter ended September 30, 2018. For the nine months ended September 30, 2019 and 2018, research and development expenses were $30.0 million and $20.0 million, respectively. The company expects a continued increase to research and development activities to support the clinical advancement of its ALPN-101 and ALPN-202 programs.

General and administrative expenses for the third quarter ended September 30, 2019 were $2.5 million compared to $1.9 million for the third quarter ended September 30, 2018. For the nine months ended September 30, 2019 and 2018, general and administration expenses were $7.4 million and $5.8 million, respectively. The increase was primarily attributable to professional and legal services and an increase in facility costs to support the growth and expansion of our business.

Cash Position

As of September 30, 2019, Alpine had cash, cash equivalents, restricted cash, and short-term investments totaling $47.0 million. For the nine months ended September 30, 2019, net cash used in operating activities was $29.7 million compared to $19.0 million for the nine months ended September 30, 2018.

Management will provide updates to cash guidance on the conference call scheduled to discuss third quarter 2019 financial results.

Conference Call and Webcast Details

Alpine will host a conference call to discuss key data, clinical highlights and provide an update on third quarter 2019 results on Wednesday, November 13, 2019 at 4:30 p.m. ET. To access the live call by phone, dial (877) 407-0789 (domestic) or (201) 689-8562 (international). To access a live webcast of the call, please visit the Investor Relations section of the Alpine Immune Sciences website at www.alpineimmunesciences.com. The recorded webcast will be available for replay for approximately 30 days following the call.

About ALPN-101

ALPN-101 is a novel Fc fusion protein of a human inducible T cell costimulator ligand (ICOSL) variant immunoglobulin domain (vIgD), and a first-in-class therapeutic designed to inhibit simultaneously the CD28 and ICOS inflammation pathways. CD28 and ICOS are closely related costimulatory molecules with partially overlapping roles in T cell activation likely connected to multiple autoimmune and inflammatory diseases. In preclinical models of graft versus host disease, inflammatory arthritis, connective tissue disease and multiple sclerosis, ALPN-101 demonstrates efficacy superior to blockade of the CD28 or ICOS pathways alone.

About ALPN-202

ALPN-202 is a first-in-class, conditional CD28 costimulator and dual checkpoint inhibitor, which has the potential to improve upon the efficacy of combined checkpoint inhibition without significant toxicities. Preclinical studies of ALPN-202 have successfully demonstrated superior efficacy in tumor models compared to checkpoint inhibition alone. We anticipate initiation, upon authorization, of the first-in-human clinical study of ALPN-202 to begin in the first quarter of 2020.

On November 13, 2019 Heron Therapeutics, Inc. (Nasdaq: HRTX), a commercial-stage biotechnology company focused on improving the lives of patients by developing best-in-class treatments to address some of the most important unmet patient needs, reported that Barry Quart, Pharm.D., President and Chief Executive Officer of Heron Therapeutics, will present at the Stifel 2019 Healthcare Conference on Tuesday, November 19, 2019 at 8:35 a.m. EST at the Lotte New York Palace hotel (Press release, Heron Therapeutics, NOV 13, 2019, View Source [SID1234551137]).

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A live webcast of the presentation will be available on the Company’s website at www.herontx.com in the Investor Resources section. A replay of the presentation will be archived on the site for 60 days.