On November 6, 2018 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL), reported financial results for the third quarter ended September 30, 2018, and also provided an update on the commercial launch of TAVALISSE for treatment of thrombocytopenia in adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment and its clinical development pipeline (Press release, Rigel, NOV 6, 2018, View Source [SID1234530861]).

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Recent Highlights

·Net product sales of $4.9 million for TAVALISSE during the third quarter

·On October 29, entered into an exclusive license and supply agreement with Kissei Pharmaceutical Co., Ltd. (Kissei) for development and marketing rights to fostamatinib in Asia; Rigel to receive upfront cash payment of $33 million, with the potential for up to an additional $147 million in milestone payments and product transfer price payments based on tiered net sales

· On October 4, the European Medicines Agency (EMA) validated the company’s Marketing Authorization Application (MAA) for fostamatinib1 in adult chronic ITP, initiating the review process

· Phase 3 trial design for fostamatinib1 investigational candidate in autoimmune hemolytic anemia (AIHA) to be submitted to U.S. Food and Drug Administration (FDA) in early November

"We continue to advance our corporate strategy with solid execution across all business areas. The success of our TAVALISSE commercial launch in the United States, our collaboration with Kissei in Asia, and our MAA validation highlight the expanding capabilities of our organization," said Raul Rodriguez, president and CEO of Rigel. "In parallel, our clinical development plans continue to increase the potential of our pipeline. For our investigational agents, we plan to initiate our Phase 3 study for fostamatinib in autoimmune hemolytic anemia in the first half of 2019 and we continue to explore potential drug development opportunities including for our IRAK1/4 inhibitor, R835."

Financial Update

For the third quarter of 2018, Rigel reported a net loss of $23.8 million, or $0.14 per share, compared to a net loss of $17.7 million, or $0.14 per share, in the same period of 2017.

For the third quarter of 2018, Rigel reported net product sales from TAVALISSE of $4.9 million. Rigel recognizes revenue using the sell-in methodology when products are delivered to its distributors. There were no product sales in the third quarter of 2017.

There were no contract revenues from collaborations in the third quarter of 2018. Contract revenues from collaborations of $900,000 in the third quarter of 2017 were related to a payment received from a license agreement with a third party.

Rigel reported total costs and expenses of $29.2 million in the third quarter of 2018, compared to $18.8 million for the same period in 2017. The increase in costs and expenses was primarily due to the increases in personnel costs as Rigel expanded its customer-facing team, third party costs to support Rigel’s ongoing commercial efforts for TAVALISSE in chronic ITP, as well as stock-based compensation expense related to certain performance-based stock options.

For the nine months ended September 30, 2018, Rigel reported net product sales from TAVALISSE of $6.7 million. There were no product sales for the nine months ended September 30, 2017. For the nine months ended September 30, 2018, Rigel reported a net loss of $73.7 million, or $0.47 per share, compared to a net loss of $52.1 million, or $0.43 per share, for the same period of 2017.

As of September 30, 2018, Rigel had cash, cash equivalents and short-term investments of $115.6 million, compared to $115.8 million as of December 31, 2017. With the $33.0 million upfront payment Rigel will receive under its collaboration agreement with Kissei, as discussed below, Rigel expects that its cash, cash equivalents and short-term investments will be sufficient to support its current and projected funding requirements, including the on-going commercial launch of TAVALISSE for chronic ITP in the U.S., into the first quarter of 2020.

Business Update

Since commercial launch in May 2018, demand for TAVALISSE in adult patients with previous treatment failure in cITP continues to grow, with broad usage seen in steroid refractory patients. TAVALISSE has been utilized by a broad base of prescribers and community physicians, and the payor response has been positive with an approval rate of 85-90%.

Outside of the U.S., the company continues to further its global commercialization strategy. Rigel has entered an exclusive license and supply agreement with Kissei for the development and commercialization of fostamatinib in all indications in Japan. The agreement also provides Kissei with rights to fostamatinib in China, Taiwan, and the Republic of Korea. In exchange, Rigel will receive an upfront cash payment of $33 million with the potential for up to an additional $147 million in development and commercial milestone payments. The company will also receive product transfer price payments in the mid to upper twenty percent range based on tiered net sales for exclusive supply of fostamatinib.

In the EU, which is the second largest market for adult chronic ITP, the EMA validated the MAA for fostamatinib1 in the indication. The review process was initiated in October and the company anticipates an opinion from the Committee on Human Medicinal Products (CHMP) of the EMA by the fourth quarter of 2019.

Rigel continues to progress with its expansion plans for fostamatinib in other indications1 and will submit its Phase 3 trial design for the treatment of warm AIHA (wAIHA) to the FDA in early November. The trial, designed in consultation with the FDA, is a placebo-controlled study of approximately 80 patients with primary or secondary wAIHA who have failed at least one prior treatment. The primary endpoint will be a durable hemoglobin response by week 24, defined as Hgb > 10 g/dL and > 2 g/dL

greater than baseline and durability of response, with the response not being attributed to rescue therapy. Enrollment is expected to begin in the first half of 2019.

About ITP
In patients with ITP, the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPOs) and splenectomy. However, not all patients are adequately treated with existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA
AIHA is a rare, serious blood disorder in which the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 40,000 adult patients in the U.S. and can be a severe, debilitating disease. To date, there are no disease-targeted therapies approved for AIHA, despite the unmet medical need that exists for these patients.

About R8351

The investigational candidate, R835, is an orally available, potent and selective inhibitor of IRAK1 and IRAK4 that has been shown preclinically to block inflammatory cytokine production in response to toll-like receptor (TLR) and the interleukin-1 (IL-1R) family receptor signaling. TLRs and IL-1Rs play a critical role in the innate immune response and dysregulation of these pathways can lead to a variety of inflammatory conditions. R835 is active in multiple rodent models of inflammatory disease including psoriasis, arthritis, lupus, multiple sclerosis and gout.

Conference Call and Webcast with Slides Today at 5:00PM Eastern Time
Rigel will hold a live conference call and webcast today at 5:00pm Eastern Time (2:00pm Pacific Time).

Participants can access the live conference call by dialing 855-892-1489 (domestic) or 720-634-2939 (international) and using the Conference ID number 1398326. The webcast, with slide presentation, can be accessed from Rigel’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About TAVALISSE
Indication
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information
Warnings and Precautions

· Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.

· Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to >3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.

· Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (>Grade 3), interrupt, reduce dose or discontinue TAVALISSE.

· Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.

· TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.

Drug Interactions

· Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.

· It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.

· Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.

· Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.

Adverse Reactions

· Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).

· Common adverse reactions (>5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.

Please see www.TAVALISSE.com for full Prescribing Information.
t side effects of prescription drugs to the FDA, visit www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).

TAVALISSE and RIGEL ONECARE are trademarks of Rigel Pharmaceuticals, Inc.

On November 6, 2018 Immune Design (Nasdaq: IMDZ), an immunotherapy company focused on next-generation therapies in oncology, reported financial results and a corporate update for the third quarter ended September 30, 2018 (Press release, Immune Design, NOV 6, 2018, View Source [SID1234530878]).

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"Given the promising data to date with G100, our intratumoral TLR4 cancer therapy, we are aggressively advancing an expanded clinical development plan for this proprietary agent," said Carlos Paya, M.D., Ph.D., President and Chief Executive Officer of Immune Design. "While preserving the option to develop our other approaches in the future, we believe that G100’s unique mechanism of action in B cell malignancies warrants our near-term focus. Its single agent and combination activity coupled with a preferable safety profile are differentiating features that we believe better position us for clinical success. We look forward to sharing data as they mature later this year and over the next 12-18 months."

Corporate Highlights

•
In October, the company announced prioritization of resources to support expanded clinical development of G100.
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Initial focus will be in combination with pembrolizumab in relapsed follicular lymphoma (FL) patients who have received three prior lines of systemic therapy.
▪
Pursuant to discussions with the FDA, these patients may represent an unmet medical need, which may allow for a single arm study and potential for accelerated approval path.
▪
In an open label setting scheduled to begin in the first quarter of 2019, the plan is to evaluate:
•
clinical activity based on Objective Response Rate (ORR) and Duration of Response; and
•
patients by "TLR4HIGH" expression, an emerging biomarker that may provide the opportunity to pre-select patients with a higher likelihood to respond to G100.
◦
In addition, the company:
▪
plans to evaluate G100 in earlier lines of lymphoma in combination with rituximab; and
▪
is evaluating the potential development of G100 in other indolent lymphomas, as well as aggressive lymphomas and solid tumors.

•
Upcoming Data Presentations
◦
As announced earlier today, G100 will be featured in three presentations at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting, November 9 and 10.
▪
"Higher dose single-agent intratumoral G100 (a TLR4 agonist) results in increased biomarker activity and improved clinical outcomes in patients with follicular lymphoma"
•
A new cohort of 18 follicular lymphoma patients who received 20ug of G100 with low-dose radiation showed increased biomarker activity and improved clinical outcomes in comparison to the 10ug dose (n=16), without the use of an anti-PD-1 antibody.

•
Patients receiving the 20ug dose showed a positive trend of more rapid and deeper abscopal responses than those receiving 10ug.
•
Patients receiving 20ug showed improved responses in the TLR4HIGH subpopulation:
◦
Patients receiving 20ug had a 60% ORR (6/10) as compared to 29% ORR (2/7).
◦
Approximately 60% of the patients in both groups tested positive for baseline
TLR4HIGH >50% TLR4 expression prior to G100 treatment.
▪
"Synergistic anti-tumor effects of TLR4 agonist G100 and anti-OX40 antibody"
▪
"The TLR4 agonist G100 enhances the efficacy of adoptive T-cell therapy"
◦
G100 will also be featured at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on December 2, 6-8 pm, in a presentation titled: "Long Term Follow-up of a Phase 2 Study Examining Intratumoral G100 Alone and in Combination with Pembrolizumab in Patients with Follicular Lymphoma."
▪
Follow up of the patient data presented at ASH (Free ASH Whitepaper) 2017 (n=26) from a randomized study comparing G100 with low-dose radiation +/- Keytruda (pembrolizumab).
▪
Responses are durable with a trend towards longer progression free survival (PFS) on the arm with pembrolizumab (11.1 months) vs. the arm without (7.4 months).

•
Together, Immune Design believes these new clinical and preclinical data:
◦
Support using the higher, 20ug dose of G100 in further development;
◦
Provide additional evidence of G100’s clinical activity; and
◦
Support the further development of G100 as a single agent and in combination with other therapies, initially in B cell malignancies.

Financial Results

Third Quarter

•
Immune Design ended the third quarter of 2018 with $107.5 million in cash and cash equivalents, short-term investments, and other receivables compared to $144.2 million as of December 31, 2017.
•
Net loss and net loss per share for the third quarter of 2018 were $14.0 million and $0.29, respectively, compared to $13.4 million and $0.52, respectively, for the third quarter of 2017.
•
Revenue did not materially differ over the comparative periods. Revenue for the third quarter of 2018 was $0.5 million and was primarily attributable to $0.2 million in collaboration revenue associated with the Sanofi G103 HSV2 vaccine collaboration and $0.2 million in product sales to collaboration partners and other third parties. Revenue for the third quarter of 2017 was $0.5 million and was primarily attributable to collaboration revenue associated with the Sanofi G103 collaboration.
•
Research and development expenses for the third quarter of 2018 were $11.2 million, compared to $10.2 million for the same period in 2017. The $1.0 million increase was primarily attributable to milestone payments of $1.7 million due to third parties as a result of the commencement of our SYNOVATE study, which was offset by a decrease in contract manufacturing services and personnel-related expenses.
•
General and administrative expenses did not materially differ over the comparative periods. For the three months ended September 30, 2018, general and administrative expenses were $3.8 million compared to $3.9 million for the same period in 2017.

Year-to-Date

•
Net cash used in operations for the nine months ended September 30, 2018 was $40.3 million.

•
Net loss and net loss per share for the nine months ended September 30, 2018 were $41.2 million and $0.85, respectively, compared to $39.9 million and $1.56, respectively, for the same period in 2017.
•
Revenue for the nine months ended September 30, 2018 was $1.7 million and was primarily due to $1.1 million in collaboration revenue associated with the Sanofi G103 collaboration and $0.6 million in product sales to our collaboration partners and other third parties. Revenue for the nine months ended September 30, 2017 was $6.7 million and was primarily attributable to $6.4 million in collaboration revenue associated with the Sanofi G103 collaboration and $0.3 million in product sales to collaboration partners other third parties.
•
Research and development expenses for the nine months ended September 30, 2018 were $32.5 million compared to $35.1 million for the same period in 2017. The $2.6 million decrease was primarily due to a decrease of $4.9 million in contract manufacturing costs and a slight decrease of $0.3 million in clinical trial costs. This decrease was offset by an increase of $0.9 million in personnel-related expenses and $1.7 million of milestone payments.
•
General and administrative expenses did not materially differ over the comparative periods. For the nine months ended September 30, 2018, general and administrative expenses were $11.8 million compared to $11.9 million for the same period in 2017.

Cash Guidance

Based on current expectations, Immune Design expects to have cash to fund operations into 2021.

Conference Call Information

Immune Design will host a conference call and live audio webcast this afternoon at 1:30 p.m. Pacific Time / 4:30 p.m. Eastern Time to discuss third quarter 2018 financial results and provide a corporate update.

The live call may be accessed by dialing 844-266-9538 for domestic callers and 216-562-0391 for international callers. A live webcast of the call will be available online from the investor relations section of the Immune Design website at View Source and will be archived there for 30 days. A telephone replay of the call will be available for five days by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference code 1359112.

On November 6, 2018 ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported that the following presentations by Company management at upcoming investor conferences will be webcast (Press release, ImmunoGen, NOV 6, 2018, View Source [SID1234530940]):

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Jefferies London Healthcare Conference
November 14, 2018 at 9:40 am ET/2:40 pm GMT
Evercore ISI HealthConX
November 27, 2018 at 8:45 am ET
30th Annual Piper Jaffray Healthcare Conference
November 28, 2018 at 11:30 am ET
A webcast of each presentation will be accessible live through the "Investors" section of the Company’s website, www.immunogen.com; a replay will be available in the same location for approximately two weeks.

On November 6, 2018 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage biopharmaceutical company focused on creating innovative cancer immunotherapies, reported financial results for the third quarter ended September 30, 2018 and provided updates on its clinical and preclinical programs (Press release, Arcus Biosciences, NOV 6, 2018, View Source [SID1234531019]).

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"This was another productive quarter for Arcus as we began dosing patients with our third product candidate, AB154, and made significant progress advancing our initial combination trials for AB928," said Terry Rosen, Ph.D., Chief Executive Officer at Arcus. "The starting dose for the dose-escalation portion of our AB928 combination trials is 75 mg once-daily, a dose that demonstrated significant inhibition of the adenosine 2a receptor pathway in our healthy volunteer study. We have incorporated extensive biomarker analysis into the design of our AB928 combination trials to determine if clinical responses observed in the trials can be attributed to the mechanism of action of AB928. We look forward to reporting initial clinical data for both AB928 and AB154, as well as data from our healthy volunteer study of AB680, our small-molecule CD73 inhibitor, in 2019."

Pipeline Updates and Poster Presentations

AB928 (dual A2aR/A2bR antagonist)

Initiated the first three AB928 combination trials in patients. AB928 is being evaluated in combination with other agents in the following Phase 1/1b dose-escalation trials, which are now enrolling patients:

AB928 in combination with Doxil in triple negative breast (TNBC) and ovarian cancers

AB928 in combination with mFOLFOX in colorectal and gastroesophageal cancers

AB928 in combination with AB122, the Company’s anti-PD-1 antibody, in advanced solid tumor types

The Company also expects the following AB928 dose-escalation trial to be open for enrollment shortly:

AB928 in combination with carboplatin/pemetrexed and pembrolizumab in non-small cell lung cancer (NSCLC)

In the above NSCLC trial, the Company also plans to explore AB928 combinations in the relapsed/refractory setting, including in patients previously treated with anti-PD-1 therapy.

Six posters to be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2018 Annual Meeting taking place November 7th through 11th:

"Development of biomarkers to assess adenosine generation & activity in support of clinical trials conducted with the adenosine receptor antagonist AB928" will highlight the development of biomarkers to enable the selection of patients and tumor types with the highest levels of CD73, the rate-limiting enzyme responsible for the production of adenosine.

"Selection of optimized drug candidates, dosing regimen, pharmacodynamic endpoints, tumor types, and biomarkers for translating inhibition of the adenosine pathway into effective anti-tumor activity" will highlight the Company’s strategy for identifying the optimal tumor types to target for each of AB928 and AB680.

Four "Trial in Progress" poster presentations will summarize the design of the Company’s four AB928 combination trials.

Presented final results from the Phase 1 double-blinded, randomized, placebo-controlled trial of AB928 in healthy volunteers in a poster presentation at ESMO (Free ESMO Whitepaper) in October. Data presented in this poster presentation support the selection of the starting dose of AB928 for clinical trials in patients.

Presented a poster on the ability of AB928 to relieve adenosine-mediated immune suppression at the Fourth AACR (Free AACR Whitepaper) International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) in September. The in vitro data presented demonstrate that AB928 prevents adenosine-mediated gene expression changes and suppression of immune cell function and suppresses tumor growth in syngeneic mouse models when administered as a monotherapy or in combination with anti-PD-1 or chemotherapy.

AB122 (anti-PD-1 antibody)

Two posters to be presented at the SITC (Free SITC Whitepaper) 2018 Annual Meeting in November:

"Preliminary results from an ongoing Phase 1 study of AB122 in patients with advanced solid tumors" will include pharmacokinetic, receptor occupancy, safety and clinical activity data from the Phase 1 dose-escalation trial for AB122.

"Development of a robust, simplified method to measure receptor occupancy in peripheral blood from patients treated with a novel anti-PD-1 agent, AB122" will demonstrate, together with the previous poster, that AB122 achieved significant inhibition of PD-1 in patients treated in the first two dosing cohorts of the Phase 1 dose-escalation trial.

Continued dosing patients in the Company’s Phase 1 dose-escalation trial for AB122. As of November 3, 2018, the Company had dosed 20 patients with AB122 evaluating different doses and dosing schedules. Based on data generated to date, the Company selected 240 mg as the dose for the Q2W (every 2 weeks) regimen for AB122.

AB154 (anti-TIGIT antibody)

Dosed the first cohort of patients in the dose-escalation portion of the ongoing Phase 1 trial for AB154 in Australia. This Phase 1 trial is evaluating AB154 in selected solid tumor types. The dose-escalation portion will be followed by the initiation of expansion cohorts in tumor types associated with high levels of TIGIT and/or CD155, the ligand for TIGIT, once the recommended doses for

AB154 as a monotherapy and in combination with AB122 have been identified. The Company plans to file an Investigational New Drug (IND) Application for AB154 in the U.S. by the end of the first quarter of 2019.

Presented a poster on the preclinical characterization of AB154 at the Fourth AACR (Free AACR Whitepaper) International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) in September. Data presented demonstrated that AB154 enhances the T cell activation effects of our anti-PD-1 antibody (AB122) in a mixed lymphocyte assay and that AB154 has sub-nanomolar potency on peripheral blood lymphocytes derived from both healthy donors and NSCLC patients.

Presented a poster at the SITC (Free SITC Whitepaper) 2018 Annual Meeting in November:

"Preclinical characterization of AB154, a fully humanized α-TIGIT antibody, for use in combination therapies" will highlight the Company’s development of a TIGIT occupancy assay, which is being implemented in the ongoing Phase 1 trial of AB154.

AB680 (small molecule CD73 inhibitor)

Received regulatory approval in Australia to initiate a healthy volunteer trial for AB680 (IV formulation). This trial is primarily designed to determine the safety, tolerability and pharmacokinetic profile of AB680 prior to initiating clinical testing of AB680 in cancer patients and is expected to begin dosing shortly. Preclinical data suggest that the half-life of AB680 should be sufficient for clinical dosing every two or three weeks.

Presented a poster on the preclinical pharmacokinetic and pharmacodynamic characterization of AB680 at the Fourth AACR (Free AACR Whitepaper) International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) in September. Data presented demonstrated that AB680 is a highly potent and selective small-molecule inhibitor of CD73 and that AB680 has a long projected human half-life.

IND-enabling studies for an oral formulation of AB680 are ongoing.

Corporate Updates

In October, Arcus announced that Kristin M. Hege, M.D., was appointed to its Board of Directors. Dr. Hege currently serves as Corporate Vice President, Translational Development, Hematology and Oncology and San Francisco site head at Celgene.

Upcoming Milestones

In the first half of 2019, the Company expects to:

Present initial data from the dose-escalation portion of the AB928 Phase 1/1b combination trials, which will include data on safety, biomarker analysis and clinical activity for the combinations, in the second quarter.

Initiate an expansion cohort to evaluate AB122 as a monotherapy to confirm that the activity of AB122 is similar to that of the approved anti-PD-1 antibodies.

Report safety and pharmacokinetic data from the Phase 1 trial of AB680 in healthy volunteers, and initiate the Phase 1 clinical program for AB680 in cancer patients.

In the middle of 2019, the Company expects to:

Initiate the first of the expansion cohorts for the AB928 combination trials.

In the second half of 2019, the Company expects to:

Present additional data from the dose-escalation portion of the AB928 Phase 1/1b combination trials.

Present initial data from the ongoing Phase 1 trial of AB154.

Third Quarter and Year-to-Date 2018 Financial Results

Cash Position: At September 30, 2018, cash and investments (which include cash equivalents and both short-term and long-term investments) were $265.6 million, compared to $175.7 million at December 31, 2017. The increase was primarily due to $124.7 million in net proceeds from the Company’s initial public offering in March.

Revenues: Collaboration and license revenues for the third quarter ended September 30, 2018 were $4.3 million, compared to $0.2 million for the same period in 2017. Collaboration and license revenues for the nine months ended September 30, 2018 were $6.8 million, compared to $0.2 million for the same period in 2017. The increase in revenues for both periods was attributable to revenues recognized from the Option and License Agreement the Company entered into with Taiho Pharmaceutical Co., Ltd in September 2017.

R&D Expenses: Research and development expenses for the third quarter ended September 30, 2018 were $12.9 million, compared to $21.4 million for the same period in 2017. The decrease was due to licensing costs of $15.0 million paid to WuXi Biologics in the third quarter ended September 30, 2017, partially offset by an increase in clinical and manufacturing costs related to the Company’s initiation of its AB928 combination and AB154 clinical trials, preclinical and manufacturing costs to prepare AB680 for clinical trials, an increase in R&D headcount to support the Company’s clinical operations and other programs, and an increase in lab supplies. Research and development expenses for the nine months ended September 30, 2018 were $38.2 million, compared to $35.1 million for the same period in 2017.

G&A Expenses: General and administrative expenses for the third quarter ended September 30, 2018 were $3.6 million, compared to $1.9 million for the same period in 2017. The increase was primarily due to higher legal and accounting fees and additional staff in key areas required to support a public company infrastructure, as well as increased facilities and office expenses related to our expanded facility in Hayward. General and administrative expenses for the nine months ended September 30, 2018 were $10.0 million, compared to $5.2 million for the same period of 2017.

Net Loss: Net loss for the third quarter ended September 30, 2018 was $10.8 million, compared to $23.1 million for the same period in 2017. The decrease in net loss was primarily attributable to the increase in revenue and changes in operating expenses noted above. Net loss for the nine months ended September 30, 2018 was $37.3 million, compared to $39.9 million for the same period in 2017.

Based on its current operating plan, the Company expects that its cash and investments as of September 30, 2018 will enable the Company to fund its anticipated operating expenses and capital expenditure requirements into 2021.

On November 6, 2018 CymaBay Therapeutics, Inc. (NASDAQ: CBAY) a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported financial results and a corporate update for the quarter and nine months ended September 30, 2018 (Press release, CymaBay Therapeutics, NOV 6, 2018, View Source [SID1234530759]).

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"We continue to make excellent progress advancing our lead candidate, seladelpar, in two indications — primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH)," said Sujal Shah, President and CEO of CymaBay. "Last week we announced the initiation of ENHANCE, a global Phase 3 registration study of seladelpar for patients with PBC. In addition, 52- and 26-week results from our Phase 2 PBC study will be featured in two late-breaking presentations at The Liver Meeting on November 12. We believe these data support the potential for seladelpar to offer patients with PBC improved efficacy and better tolerability than existing second line treatment while also significantly de-risking the ongoing Phase 3 study. Enrollment in our ongoing Phase 2b study of seladelpar for patients with NASH has been progressing well. We now expect to complete enrollment in the first quarter of 2019, one quarter ahead of our previous guidance. As the only highly selective and potent PPARδ agonist in development for liver disease, we think it may be particularly well suited to treat NASH because of its beneficial effects on lipid, glucose, and sterol metabolism, as well as its effects on inflammation and fibrogenesis. As we approach the end of 2018, our entire team remains committed to these core programs for which we look forward to providing further updates in 2019."

Third Quarter 2018 and Recent Business Highlights

Initiated ENHANCE, a global Phase 3 registration study evaluating 5 and 10 mg of seladelpar versus placebo for the treatment of PBC in patients that are inadequate responders to or are intolerant to ursodeoxycholic acid (UDCA)
The study is designed to support the submission of a global registration dossier with Health Authorities to obtain approval of seladelpar in PBC
Two late-breaking presentations featuring positive data from an ongoing Phase 2 study of seladelpar in PBC will be featured during The Liver Meeting hosted by the American Association for the Study of Liver Diseases (AASLD) in San Francisco, November 9-13, 2018
The 52-week composite responder rates in the ongoing Phase 2 study for the 5 mg / 5 mg to 10 mg titration and 10 mg seladelpar groups were 59% and 71%, respectively
Results suggest that seladelpar is not associated with drug-induced pruritus and may support the hypothesis that seladelpar decreases pruritus in PBC patients
This is the third consecutive year that data on seladelpar in PBC will be highlighted in a late-breaking presentation at The Liver Meeting
Patient recruitment in the placebo-controlled Phase 2b proof-of-concept study investigating seladelpar at three doses in biopsy-proven NASH is one quarter ahead of schedule and is now expected to be fully enrolled in the first quarter of 2019
The primary efficacy outcome is the change from baseline in liver fat content at 12 weeks as measured by magnetic resonance imaging using the proton density fat fraction method (MRI-PDFF)
The study also includes a second biopsy at 52 weeks to examine its activity on NASH and fibrosis
Appointed key individuals to expand and strengthen the development organization to execute the seladelpar Phase 3 program and deliver a high-quality registration package
Dr. Patricia Rohane, M.D., appointed Vice President, Clinical Development
Dr. Stephen Rossi, Pharm. D., appointed Vice President, Early Clinical Development
Kamal Sigel, M.S., appointed Vice President, Quality
Held $198.1 million in cash, cash equivalents and marketable securities at September 30, 2018. Existing cash is expected to fund the current operating plan into 2021.
Third Quarter 2018 Financial Results

Research and development expenses were $17.9 million in the third quarter of 2018 as compared to $4.2 million in the same period of 2017. The increase was primarily driven by increases in seladelpar-related clinical trial expenses from the expansion and extension of our PBC Phase 2 study, start-up activities related to our PBC Phase 3 study, the ongoing enrollment of our NASH Phase 2b study, and the execution of other NDA-enabling studies.
General and administrative expenses were $3.3 million in the third quarter of 2018 as compared to $2.2 million in the same period of 2017. The increase was driven primarily by employee compensation expense as we hired additional personnel to support our expanding operations.
Net loss was $18.6 million, or ($0.34) per diluted share in the third quarter of 2018, as compared to $8.2 million, or ($0.21) per diluted share in the same period of 2017. Net loss was higher primarily due to increased research and development expenses, partially offset by non-cash gains on the revaluation and extinguishment of our warrant liability.
Nine Months Ended September 30, 2018 Financial Results

No collaboration revenue was recognized in the nine months ended September 30, 2018. Collaboration revenue from Kowa Pharmaceuticals America, Inc. totaling $4.8 million was recognized in the same period of 2017.
Research and development expenses were $41.7 million in the nine months ended September 30, 2018 as compared to $12.3 million in the same period of 2017. The increase was primarily driven by increases in seladelpar-related clinical trial expenses from the expansion and extension of our PBC Phase 2 study, start-up activities related to our PBC Phase 3 study, the ongoing enrollment of our NASH Phase 2b study, and the execution of other NDA-enabling studies.
General and administrative expenses were $10.2 million in the nine months ended September 30, 2018, as compared to $9.5 million in the same period of 2017. The increase was driven primarily by higher compensation and consulting expenses, partially offset by decreases in severance and legal fees.
Net loss was $53.1 million, or ($0.93) per diluted share in the nine months ended September 30, 2018, as compared to $22.5 million, or ($0.71) per diluted share in the same period of 2017. Net loss was higher primarily due to increased research and development expenses and lower collaboration revenue.
Conference Call Details
CymaBay management will host a conference call today at 4:30 p.m. ET to discuss third quarter 2018 financial results and provide a business update. To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID# 13683385. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

About Seladelpar
Seladelpar is a potent, selective, orally active PPARδ agonist that is in development for the treatment of the liver diseases PBC and NASH. For PBC, seladelpar has received an orphan designation from the US Food and Drug Administration and the European Medicine Agency. Seladelpar also received the PRIority MEdicine (PRIME) status from the European Medicine Agency.