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Navidea Biopharmaceuticals Reports Third Quarter 2017 Financial Results

On November 7, 2017 Navidea Biopharmaceuticals, Inc. (NYSE MKT: NAVB) ("Navidea" or the "Company"), a company focused on the development of precision immunodiagnostic agents and immunotherapeutics, reported its financial results for the third quarter of 2017 (Press release, Navidea Biopharmaceuticals, NOV 7, 2017, View Source [SID1234521683]). Navidea reported total revenues for the quarter of $224,000. Net loss attributable to common stockholders was $1.5 million. Net revenues do not include the guaranteed payments from Cardinal Health 414, LLC ("Cardinal Health 414") because those are represented on the balance sheet in accounts receivable and were already included in the gain on sale of the line of business sold to Cardinal Health 414 for the nine months ended September 30, 2017.

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"We’ve made significant advancements in our pipeline, both on the diagnostic and therapeutics side so far this year. Efforts undertaken since the closing of the Cardinal Health 414 transaction to implement the new strategy have enabled the more rapid development of our proprietary technology. We have demonstrated significant market expansion potential with our imaging agent. We are actively pursuing an approval to utilize our activated macrophage technology as a biomarker. We have formally contacted the U.S. Food and Drug Administration ("FDA") and have scheduled our first meeting with them. In parallel, we are also pursuing an additional approval for our agent so we can administer it intravenously ("IV"). The FDA and many major pharmaceutical companies have indicated their significant interest in developing biomarkers that can assist in developing new therapeutics and in enabling objective monitoring of performance of existing therapies. With our best-in-class activated macrophage targeting system, we have been able to generate significant human imaging data and promising animal data with our therapeutic agents, reinforcing our optimism that this platform holds potential for the diagnosis and treatment of diseases in which macrophages play an important role," said Michael Goldberg, M.D., Navidea’s President and Chief Executive Officer.

Dr. Goldberg continued, "On the diagnostic side, we have generated data with both IV and subcutaneous formulations of Tc99m tilmanocept in rheumatoid arthritis ("RA"). We have completed all but a few of the control dosings in the Phase 1/2 dose escalation registrational study and expect to finalize the report on this study in the fourth quarter of this year. In nonalcoholic steatohepatitis ("NASH") and cardiovascular ("CV") disease, we will also be initiating dosing of an IV formulation shortly. For CV, we are working with the same team at Massachusetts General Hospital in Boston, who designed, managed and published the subcutaneous CV study that has attracted so much interest. In the IV study, we will also explore the ability to image central nervous system inflammation. With Kettering Medical Center in Ohio, we will shortly be dosing in NASH patients. On the therapeutic side, we have synthesized and tested delivery backbones that are one-tenth the size of the existing agents. As we explore formulation opportunities with our therapeutics, smaller agents provide better opportunities for creating therapeutics than can be delivered orally and topically. The newer agents retain the same very high binding we have achieved with our larger constructs. Finally, we have dosed in cancer models our MT1000 class of therapeutics, much more frequently (twice per day as opposed to twice per week) with the same total dose and as expected this resulted in much improved activity. "

Third Quarter 2017 Highlights and Subsequent Events

Executed a letter of intent for a sublicensing contract for worldwide research and development results with Cerveau Technologies, Inc. ("Cerveau") for using NAV4694, a beta-amyloid imaging agent being evaluated as an aid in the differential diagnosis of early-onset Alzheimer’s disease
Executed a letter of intent for an exclusive license with Cerveau for the development and commercialization of NAV4694 in Australia, Canada, China, and Singapore
Presented a late-breaking poster presented at the American College of Rheumatology Annual Meeting detailing the results of an IV-administered study in RA patients
Initiated series of regular investor-focused Q&A conference calls to improve Investor Relations strategy
IV-administration RA trial to complete enrollment in fourth quarter 2017
Launched NASH imaging study launch this quarter at Kettering Medical Center in Ohio
Initiate dosing in Phase 1/2 clinical imaging study in Kaposi’s Sarcoma
Financial Results

Our consolidated balance sheets and statements of operations have been reclassified, as required by current accounting standards, for all periods presented to reflect the line of business sold to Cardinal Health 414 as a discontinued operation. Accordingly, this discussion focuses on describing results of our operations as if we had not operated the discontinued operation during the periods being disclosed.

We recorded a $86.7 million net gain on the line of business sold to Cardinal Health 414 for the nine months ended September 30, 2017, including $16.5 million in guaranteed consideration, which was discounted to the present value of future cash flows. The proceeds were offset by $3.3 million in estimated fair value of warrants issued to Cardinal Health 414, $2.0 million in legal and other fees related to the sale, $800,000 in net balance sheet dispositions and write-offs, and $6.5 million in estimated taxes.
Total revenues for the third quarter of 2017 were $224,000, compared to $1.8 million in the third quarter of 2016. These revenues are grant-related and do not include the $1.7 million of payments received from Cardinal Health 414.
Research and development expenses for the third quarter of 2017 were $875,000, compared to $919,000 in the third quarter of 2016. The net decrease was primarily a result of decreases in net compensation costs coupled with decreased NAV4694 and NAV5001 development costs, offset by increases in Manocept development costs.
Selling, general and administrative expenses for the third quarter of 2017 were $1.7 million, compared to $1.8 million in the third quarter of 2016. The net decrease was primarily due to decreases in general support costs such as rent and depreciation, coupled with decreased net compensation costs, offset by net increased legal and professional services.
Navidea’s net loss attributable to common stockholders for the quarter ended September 30, 2017 was $1.5 million, or a $0.01 loss per share (basic), compared to a net loss of $59,000, or a $0.00 loss per share, for the same period in 2016.
Navidea ended the quarter with $6.6 million in cash and investments, not including the quarterly guaranteed earnout payment of $1.7 million from Cardinal Health 414 which was received after the quarter ended.
Conference Call Details

Investors and the public are invited to access the live audio webcast through the link below. Participants who would like to ask questions during the question and answer session must participate by telephone. Participants are encouraged to log-in and/or dial-in fifteen minutes before the conference call begins.

Event: Q3 2017 Earnings and Business Update Conference Call
Date: Wednesday, November 8, 2017
Time: 8:30 am (Eastern Time)
U.S. & Canada Dial-in: 1-866-548-4713 (toll free)
Conference ID: 6714834
Webcast
http://www.audio-webcast.com/cgi-bin/visitors.ssp?fn=visitor&id=5121
A live audio webcast of the conference call will also be available on the investor relations page of Navidea’s corporate website at www.navidea.com. In addition, the recorded conference call can be replayed and will be available for 90 days following the call on Navidea’s website.

Nektar Therapeutics Reports Financial Results for the Third Quarter of 2017

On November 7, 2017 Nektar Therapeutics (Nasdaq: NKTR) reported its financial results for the third quarter ended September 30, 2017 (Press release, Nektar Therapeutics, NOV 7, 2017, View Source [SID1234521684]).

Cash and investments in marketable securities at September 30, 2017 were $412.2 million as compared to $389.1 million at December 31, 2016. The cash balance includes the $150.0 million upfront payment from Nektar’s collaboration with Eli Lilly & Company for the development and commercialization of NKTR-358.

"Nektar’s immuno-oncology portfolio continues to expand as we add novel drug candidates to our growing pipeline," said Howard W. Robin, President and CEO of Nektar. "NKTR-214 is the first I-O agent to both increase tumor-infiltrating lymphocytes (TILs) and increase PD-1 expression on human immune cells, which uniquely complements checkpoint inhibition and other anti-cancer mechanisms. As the majority of cancer patients have tumors that do not express PD-L1 and these patients receive limited benefit from treatment with checkpoint inhibitors, the potential of NKTR-214 to help patients is significant. Finally, based on recent positive conversations with the agency regarding our regulatory plans for NKTR-181, we are now planning to submit an NDA for NKTR-181 by April 2018 with our data package of over 2,100 patients and healthy volunteers."

Revenue in the third quarter of 2017 was $152.9 million as compared to $36.3 million in the third quarter of 2016. Year-to-date revenue for 2017 was $212.2 million as compared to $128.0 million in the first nine months of 2016. Revenue in 2017 included recognition of $127.6 million of the $150.0 million upfront payment from Nektar’s collaboration with Eli Lilly & Company for the development and commercialization of NKTR-358.

Total operating costs and expenses in the third quarter of 2017 were $83.4 million as compared to $69.2 million in the third quarter of 2016. Year-to-date total operating costs and expenses in 2017 were $247.9 million as compared to $208.7 million for the same period in 2016. Total operating costs and expenses increased primarily as a result of increased research and development (R&D) expense.

Research and development expense in the third quarter of 2017 was $65.7 million as compared to $52.0 million in the third quarter of 2016. Year-to-date R&D expense for 2017 was $187.0 million as compared to $153.6 million for the same period in 2016. R&D expense was higher in the third quarter and first nine months of 2017 as compared to the same periods in 2016 primarily because of expenses for our pipeline programs, including Phase 3 clinical studies for NKTR-181, Phase 1/2 clinical studies of NKTR-214 and NKTR-358 and IND-enabling activities for NKTR-262 and NKTR-255.

General and administrative expense was $12.1 million in the third quarter of 2017 as compared to $10.3 million in the third quarter of 2016. G&A expense in the first nine months of 2017 was $40.0 million as compared to $31.5 million for the same period in 2016. G&A expense in the first nine months of 2017 includes a $3.3 million charge for a litigation settlement related to a cross-license agreement.

Net income in the third quarter of 2017 was $60.9 million or $0.39 basic income per share as compared to net loss of $43.2 million or $0.32 basic loss per share in the third quarter of 2016. Net loss in the first nine months of 2017 was $62.9 million or $0.41 basic loss per share as compared to $111.3 million or $0.82 basic loss per share in the first nine months of 2016.

The company also announced upcoming presentations at the following scientific congresses during the fourth quarter of 2017:

Society for Immunotherapy in Cancer (SITC) (Free SITC Whitepaper) 32nd Annual Meeting, National Harbor, MD:

Oral Presentation: "PIVOT-02: Preliminary safety, efficacy and biomarker results from the Phase 1/2 study of CD-122-biased agonist NKTR-214 plus nivolumab in patients with locally advanced/metastatic solid tumors"
Presenter: Dr. Adi Diab, Assistant Professor, Department of Melanoma Medical Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas
Session: Clinical Trials: Novel Combinations
Date: Saturday, November 11, 2017, 5:00 p.m. Eastern Time

Poster #P77: "The Novel IL-2 Cytokine Immune Agonist NKTR-214 Harnesses the Adaptive and Innate Immune System for the Treatment of Solid Cancers"
Presenter: Salah Eddine Bentebibel, University of Texas MD Anderson Cancer Center
Session: Biomarkers and Immune Monitoring
Date: Friday, November 10, 2017, 12:30-2:00 p.m. Eastern Time

Poster #P140: "NKTR-214 enhances anti-tumor T-cell immune responses induced by checkpoint blockade or vaccination"
Presenter: Meenu Sharma, University of Texas MD Anderson Cancer Center
Session: Cancer Vaccines
Date: Saturday, November 11, 2017, 12:30-2:00 p.m. Eastern Time

Poster #P274: "Combination of NKTR-214 and radiotherapy (RT) to reverse anergy and expand tumor-specific CD8 T-Cells"
Presenter: Joshua Walker, Oregon Health & Science University
Session: Combination Therapy
Date: Saturday, November 11, 2017, 12:30-2:00 p.m. Eastern Time

Poster #P275: "Harnessing the innate and adaptive immune system to eradicate treated and distant untreated solid tumors"
Presenter: Saul Kivimae, Nektar Therapeutics
Session: Combination Therapy
Date: Friday, November 10, 2017, 12:30-2:00 p.m. Eastern Time

Poster #P332: "Pre-clinical efficacy and tolerability of NKTR-255, a polymer-conjugated IL-15 for immuno-oncology"
Presenter: Peiwen Kuo, Nektar Therapeutics
Session: Combination Therapy
Date: Saturday, November 11, 2017, 12:30-2:00 p.m. Eastern Time

Poster #434: "Great Apes Adenoviral vaccine encoding neoantigens synergizes with immunomodulators to cure established tumors in mice"
Presenter: Anna Morena D’Alise, Nouscom srl
Session: Personalized Vaccines and Technologies/Personalized Medicines
Date: Saturday, November 11, 2017, 12:30-2:00 p.m. Eastern Time

American College of Neuropsychopharmacology 56th Annual Meeting, Palm Springs, CA:

Poster #T166: "Abuse potential of NKTR-181 in recreational opioid users: results from a randomized, double-blind crossover oral study"
Presenter: Snow Ge, Nektar Therapeutics
Session: Poster Session II
Date: Tuesday, December 5, 2017, 5:30-7:30 p.m. Pacific Time

Conference Call to Discuss Third Quarter 2017 Financial Results
Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time, Tuesday, November 7, 2017.

This press release and a live audio-only Webcast of the conference call can be accessed through a link that is posted on the home page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through Monday, December 11, 2017.

To access the conference call, follow these instructions:
Dial: (877) 881.2183 (U.S.); (970) 315.0453 (international)
Passcode: 4677348 (Nektar Therapeutics is the host)

In the event that any non-GAAP financial measure is discussed on the conference call that is not described in the press release, or explained on the conference call, related information will be made available on the Investor Relations page at the Nektar website as soon as practical after the conclusion of the conference call.

Novavax Reports Third Quarter 2017 Financial Results

On November 7, 2017 Novavax, Inc., (Nasdaq: NVAX) reported its financial results for the third quarter and nine months ended September 30, 2017 (Press release, Novavax, NOV 7, 2017, View Source [SID1234521686]).

Third Quarter Achievements:

· Novavax initiated a Phase 1/2 clinical trial of its nanoparticle influenza vaccine candidate with its proprietary Matrix-M adjuvant (NanoFlu) in older adults. The trial is a randomized, observer-blinded, active comparator-controlled trial in approximately 330 healthy older adults. The primary objective of the trial is to assess the safety and immunogenicity of two antigen concentrations (15 µg or 60 µg) of NanoFlu compared to a licensed influenza vaccine, Fluzone High-Dose (Fluzone HD).

· Novavax reported that NanoFlu demonstrated superior immunogenicity and protection compared to the market leader in a preclinical challenge study. In a head-to-head comparison study against Fluzone HD, NanoFlu demonstrated significantly stronger and broader immune responses against matched and unmatched influenza strains, including a series of "drifted" strains evolved over more than a decade of influenza seasons. NanoFlu was also protective in an established challenge model against both a matched and a ten-year old unmatched strain. Data from this study was published in the journal Vaccine.

· The Prepare clinical trial for infants via maternal immunization, supported by an $89 million grant from the Bill and Melinda Gates Foundation (BMGF), accelerated into the third global season of enrollment. Prepare’s global footprint is expected to grow from 16 sites in five countries in its first season of enrollment to over 90 sites in 12 countries in 2018.

Anticipated 2017 Event:

· Announce topline data from a Phase 1/2 clinical trial of the NanoFlu vaccine candidate in a head-to-head comparison with Fluzone HD before the end of 2017.

Summary

"We were very pleased to initiate and fully enroll the Phase 1/2 clinical trial of our NanoFlu vaccine candidate in older adults, following impressive results in preclinical challenge models. Given this timing, we are well positioned to announce data from the ongoing trial before the end of the year," said Stanley C. Erck, President and CEO. "We also continue to make significant progress in our RSV F Vaccine program, highlighted by the continued execution of the Phase 3 Prepare trial for infants via maternal immunization. We are also evaluating plans to conduct a clinical trial for older adults in 2018."

Financial Results for the Three and Nine Months Ended September 30, 2017

Novavax reported a net loss of $44.6 million, or $0.15 per share, for the third quarter of 2017, compared to a net loss of $66.3 million, or $0.24 per share, for the third quarter of 2016. For the nine months ended September 30, 2017, the net loss was $132.9 million, or $0.47 per share, compared to a net loss of $222.9 million, or $0.82 per share, for the same period in 2016.

Novavax revenue in the third quarter of 2017 was $8.4 million, compared to $3.2 million in the same period in 2016. This 158% increase was driven by higher revenue recorded under the BMGF grant of $89 million.

Research and development expenses decreased 21% to $41.9 million in the third quarter of 2017, compared to $53.0 million for the same period in 2016. The decrease was primarily due to reduced activities related to the development of the RSV F Vaccine for older adults, other general R&D project-related expenses and lower employee-related costs.

General and administrative expenses decreased 40% to $8.1 million in the third quarter of 2017, compared to $13.6 million for the same period in 2016. The decrease was primarily due to lower professional fees for pre-commercialization activities and lower employee-related costs.

Interest income (expense), net for the third quarter of 2017 and 2016 was ($3.0) million.

As of September 30, 2017, the company had $172.6 million in cash, cash equivalents and marketable securities, compared to $235.5 million as of December 31, 2016. Net cash used in operating activities for the first nine months of 2017 was $106.6 million, compared to $194.2 million for the same period in 2016. The decrease in cash usage was primarily due to decreased costs relating to our RSV F Vaccine and lower overall employee-related costs.

Conference Call

Novavax management will host its quarterly conference call today at 4:30 p.m. ET. The dial-in number for the conference call is (877) 212-6076 (Domestic) or (707) 287-9331 (International), passcode 2479419. A replay of the conference call will be available starting at 7:30 p.m. ET on November 7, 2017 until 7:30 pm ET on November 14, 2017. To access the replay by telephone, dial (855) 859-2056 (Domestic) or (404) 537-3406 (International) and use passcode 2479419.

A webcast of the conference call can also be accessed via a link on the home page of the Novavax website (novavax.com) or through the "Investor Info"/"Events" tab on the Novavax website. A replay of the webcast will be available through the "Investor Info"/"Events" tab on the Novavax website until February 7, 2018.

Regulus Reports Third Quarter 2017 Financial Results and Recent Events

On November 7, 2017 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, reported recent events and financial results for the three and nine months ended September 30, 2017 (Press release, Regulus, NOV 7, 2017, View Source [SID1234521688]).

Regulus Therapeutics Inc. Logo

"The third quarter was marked by the successful completion of our financing, as well as significant operational progress on our two clinical programs," said Jay Hagan, President and Chief Executive Officer of Regulus. "We are very pleased that both of our Alport studies are now active, the investigational new drug (IND) for the treatment of autosomal dominant polycystic kidney disease (ADPKD) has been filed, and a Phase I study remains on track to initiate by year-end."

Third Quarter Corporate Highlights and Recent Events

In July 2017, Regulus completed a public offering of its common stock, and received net proceeds of approximately $43.0 million after deducting underwriting discounts, commissions and other offering expenses.
In September, Regulus initiated HERA, the Phase II randomized, double-blinded, placebo-controlled study evaluating the safety and efficacy of RG-012 in Alport syndrome patients. In parallel, the Phase I renal biopsy study was also initiated.
Recently, Regulus filed an IND application with the FDA for RGLS4326, targeting microRNA-17 for the treatment of ADPKD.
Third Quarter Financial Results

Cash Position: As of September 30, 2017, Regulus had cash, cash equivalents and short-term investments of $71.4 million.

Research and Development (R&D) Expenses: R&D expenses were $12.7 million and $42.7 million for the three and nine months ended September 30, 2017, respectively, compared to $14.6 million and $49.3 million for the same periods in 2016. The decrease in R&D expenses for the three months ended September 30, 2017 compared to 2016 was driven by the planned reduction in personnel-related costs as a result of our May 2017 corporate restructuring. The decrease in R&D expenses for the nine months ended September 30, 2017 compared to 2016 was primarily driven by the wind-down of clinical activities related to the RG-101 program.

General and Administrative (G&A) Expenses: G&A expenses were $2.7 million and $13.8 million for the three and nine months ended September 30, 2017, respectively, compared to $4.8 million and $13.6 million for the same periods in 2016. The decrease in G&A expenses for the three months ended September 30, 2017 compared to 2016 was attributable to the planned reduction in personnel-related costs and non-cash stock-based compensation.

Revenue: Revenue was less than $0.1 million for each of the three and nine months ended September 30, 2017, compared to $0.2 million and $1.2 million for the same periods in 2016.

Net Loss: Net loss was $15.8 million and $57.5 million for the three and nine months ended September 30, 2017, respectively, compared to a net loss of $19.5 million and $61.8 million for the same periods in 2016. Basic and diluted net loss per share was $0.18 and $0.88 for the three and nine months ended September 30, 2017, respectively, compared to $0.37 and $1.17 for the same periods in 2016.

Conference Call Details

Regulus will host a conference call and webcast today at 5:00 p.m. Eastern Time to discuss third quarter 2017 financial results and corporate highlights. A live webcast of the call will be available online at www.regulusrx.com. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 9297128. To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), conference ID 9297128. The webcast and telephone replay will be archived on the Company’s website following the call.

Rigel Announces Third Quarter 2017 Financial Results and Provides Company Update

On November 7, 2017 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) reported financial results for the third quarter and nine months ended September 30, 2017 (Press release, Rigel, NOV 7, 2017, View Source;p=RssLanding&cat=news&id=2315079 [SID1234521689]).

Recent Achievements

On October 2, 2017, Rigel announced that the U.S. Food and Drug Administration (FDA) is not currently planning on holding an Oncology Drugs Advisory Committee (ODAC) meeting to discuss the New Drug Application (NDA) for fostamatinib, an oral spleen tyrosine kinase (SYK) inhibitor, in adult patients with chronic or persistent immune thrombocytopenia (ITP).
Rigel recently completed enrollment of Stage 1 of its Phase 2 study of fostamatinib for the treatment of warm antibody autoimmune hemolytic anemia (AIHA). On a top-line, preliminary basis, the Phase 2 study achieved the pre-specified primary efficacy endpoint for Stage 1. Given this achievement, Rigel is preparing to begin enrollment of Stage 2 of this study.
Rigel announced the closing of a public offering of common stock in October, with proceeds of $69,730,250, before deducting underwriting discounts and commissions and other estimated offering expenses payable by Rigel.
"Our NDA review is progressing smoothly," said Raul Rodriguez, president and chief executive officer of Rigel. "As we prepare for an FDA decision, we are simultaneously implementing our commercial plan for a potential launch of fostamatinib in ITP, as well as continuing our clinical studies of fostamatinib in other disorders."

For the third quarter of 2017, Rigel reported a net loss of $17.7 million, or $0.14 per basic and diluted share, compared to a net loss of $22.6 million, or $0.24 per basic and diluted share, in the same period of 2016.

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. Contract revenues from collaborations of $3.8 million in the third quarter of 2016 represent the remaining amortization of the $30.0 million upfront payment which was fully amortized in September 2016, pursuant to Rigel’s collaboration and license agreement with Bristol-Myers Squibb.

Rigel reported total costs and expenses of $18.8 million in the third quarter of 2017, compared to $26.5 million for the same period in 2016. The decrease in costs and expenses was primarily due to the decreases in personnel costs as a result of the reduction in workforce in September 2016 and the completion of the pivotal Phase 3 clinical trials in ITP in 2016, partially offset by the increase in costs related to the preparation for the potential commercial launch of fostamatinib in ITP.

For the nine months ended September 30, 2017, Rigel reported a net loss of $52.1 million, or $0.43 per basic and diluted share, compared to a net loss of $53.6 million, or $0.58 per basic and diluted share, for the same period of 2016.

As of September 30, 2017, Rigel had cash, cash equivalents and short-term investments of $68.1 million, compared to $74.8 million as of December 31, 2016. In October 2017, Rigel completed an underwritten public offering in which it received proceeds of approximately $65.3 million, net of underwriting discounts and commissions and estimated offering expenses. Rigel expects that its cash, cash equivalents and short-term investments will be sufficient to support its current and projected funding requirements, including the potential U.S. commercial launch, through at least the next 12 months. Rigel continues to evaluate ex-U.S. partnerships for fostamatinib and other partnering opportunities across its pipeline.

Corporate Update
Rigel continues to execute on its commercial readiness plan to support the potential commercial launch of fostamatinib in 2018. This includes the hiring of key personnel across several functions such as marketing, sales, market access, business operations and medical affairs.

Portfolio Update
TAVALISSE (fostamatinib disodium) in ITP
During the Mid-Cycle Communication teleconference, the FDA confirmed that it was not currently planning to hold an ODAC meeting to discuss the NDA for fostamatinib in patients with chronic or persistent ITP. The FDA indicated that the review of fostamatinib is proceeding according to the standard internal review timeline as described in the Guidance on Good Review Management Principles and Practices for Prescription Drug User Fee Act (PDUFA) Products. Under the PDUFA, the action date for the FDA to complete its review will be April 17, 2018.

Update on Fostamatinib in Autoimmune Hemolytic Anemia (AIHA)
The Phase 2, open-label, multi-center, Simon two-stage study of fostamatinib for the treatment of warm AIHA recently completed enrollment of Stage 1. The study is evaluating the safety and efficacy of fostamatinib, at 150mg BID (twice daily), in patients with warm AIHA who have previously received at least one treatment for this disease, but did not have a meaningful benefit and are still anemic.

Stage 1 of the 12-week study enrolled 17 patients. As of September 30, 2017, four patients responded during the 12-week evaluation period and an additional two patients met the response criteria in the extension study after 12 weeks of dosing, for a response rate of 35% (6/17) on fostamatinib (these data are preliminary and require further verification). A response was defined as achieving a hemoglobin level of greater than 10 g/dl and at least a 2 g/dl increase from baseline. The safety profile was consistent with the existing fostamatinib safety database.

Fostamatinib in IgA Nephropathy (IgAN)
Rigel has completed enrollment of the second cohort in its Phase 2 study of fostamatinib in IgAN. Similar to the first cohort, which reported results in January 2017, the study will evaluate the efficacy, safety, and tolerability of fostamatinib as measured by change in proteinuria, renal function, and histology (comparing the pre- and post-study renal biopsies). However, the second cohort evaluates a higher dose of fostamatinib, 150mg BID, while the first cohort evaluated 100mg BID. The primary efficacy endpoint is the mean change of proteinuria from baseline at 24 weeks.

Additional Product Development
During the second quarter of 2017, Rigel selected a molecule from its IRAK program for preclinical development. The molecule is differentiated in that it inhibits both the IRAK 1 and IRAK 4 signaling pathways, with potential to treat autoimmune and inflammatory diseases such as lupus, gout, psoriatic arthritis and multiple sclerosis. The company expects to initiate clinical trials in 2018.

About ITP
ITP affects approximately 65,000 adult primary patients in the US. 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, bleeding and fatigue. People suffering with chronic ITP may live with increased risk of severe bleeding events that can result in serious medical complication, 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 and as a result, there remains a significant medical need for additional treatment options for patients with chronic ITP.

About AIHA
AIHA is a rare, serious blood disorder where 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 US and can be a severe, debilitating anemia. To date, there are no FDA approved disease-targeted therapies for AIHA, despite the tremendous medical need that exists for these patients as disease relapse is common. Instead, physicians generally treat acute and chronic cases of the disorder with corticosteroids, IV immunoglobulin infusion, other immuno-suppressants, or splenectomy (surgical removal of the spleen).

About IgAN
IgAN (also known as Berger’s disease) is a chronic autoimmune disease associated with inflammation in the kidneys that diminishes their ability to filter blood. It is the most common primary glomerular disease affecting an estimated 82,500 to 165,000 cases in the US, with a higher prevalence in Asia. For as many as 25 percent of those living with IgAN, the disease results in end-stage renal failure requiring dialysis or kidney transplantation. Other than angiotensin blockade (primarily for blood-pressure control), there are no disease-targeted therapies for IgAN.

Conference Call and Webcast 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 4894258. The conference call will also be webcast live and 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.