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Acorda Provides Financial and Pipeline Update for Third Quarter 2017

On October 30, 2017 Acorda Therapeutics, Inc. (Nasdaq:ACOR) provided a financial and pipeline update for the third quarter ended September 30, 2017 (Press release, Acorda Therapeutics, OCT 31, 2017, View Source [SID1234521332]).

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“We have had a constructive dialogue with the FDA since the issuance of its Refusal to File letter, and we plan to resubmit the INBRIJA NDA in the fourth quarter. We believe our resubmission reflects a strong package that incorporates feedback we received from FDA,” said Ron Cohen, M.D., Acorda’s President and CEO. “We are also on track to announce top-line data from our Phase 3 study of tozadenant in the first quarter of 2018.”

“INBRIJA and tozadenant are being developed as therapies for people with Parkinson’s, INBRIJA for on-demand use to treat symptoms of OFF periods and tozadenant as a daily oral treatment to increase overall ON time. If approved, they have the potential to position Acorda as a leader in the development of Parkinson’s therapy, creating substantial value for shareholders.”

Third Quarter 2017 Financial Results

AMPYRA (dalfampridine) Extended Release Tablets, 10 mg – For the quarter ended September 30, 2017, the Company reported AMPYRA net revenue of $132.6 million compared to $128.8 million for the same quarter in 2016.

FAMPYRA (prolonged-release fampridine tablets) – For the quarter ended September 30, 2017, the Company reported FAMPYRA royalties from sales outside of the U.S. of $3.1 million compared to $2.6 million for the same quarter in 2016.

Research and development (R&D) expenses for the quarter ended September 30, 2017 were $33.3 million, including $2.0 million of share-based compensation and $.03 million of restructuring expenses compared to $54.8 million, including $2.9 million of share-based compensation, for the same quarter in 2016.

Sales, general and administrative (SG&A) expenses for the quarter ended September 30, 2017 were $40.7 million, including $4.6 million of share-based compensation and $0.01 million of restructuring expenses compared to $54.4 million, including $7.1 million of share-based compensation for the same quarter in 2016.

The Company recorded a non-cash intangible asset impairment charge of $39.4 million in the quarter ended September 30, 2017 for Selincro. Selincro is currently marketed in Europe by the licensor for the reduction of alcohol consumption in alcohol dependent adults. The Company re-assessed its valuation assumptions, including expected future growth related to the expansion into new markets, and determined that the intangible asset was impaired.

Provision for income taxes for the quarter ended September 30, 2017 was $18.9 million, including $3.7 million of cash taxes, compared to a provision for income taxes of $3.0 million, including $1.0 million of cash taxes, for the same quarter in 2016.

The Company reported a GAAP net loss attributable to Acorda of $(25.2) million for the quarter ended September 30, 2017, or $(0.55) per diluted share. GAAP net loss in the same quarter of 2016 was $(12.7) million, or $(0.28) per diluted share.

Non-GAAP net income for the quarter ended September 30, 2017 was $20.1 million, or $0.43 per diluted share. Non-GAAP net loss in the same quarter of 2016 was $(1.9) million, or $(0.04) per diluted share. This quarterly non-GAAP net income measure, more fully described below under “Non-GAAP Financial Measures,” excludes share-based compensation charges, non-cash interest charges on our debt, restructuring expenses, changes in the fair value of acquired contingent consideration, intangible asset impairment charges and acquisition-related expenses. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At September 30, 2017, the Company had cash and cash equivalents of $192.5 million.

Guidance for 2017

The Company reiterates AMPYRA 2017 net revenue of $535-$545 million.
R&D expenses for the full year 2017 are expected to be $160-$170 million. This guidance is a non-GAAP projection that excludes share-based compensation and restructuring costs, as more fully described below under “Non-GAAP Financial Measures.”
The Company is reducing its SG&A expense guidance for the full year 2017 from $170-$180 million to $160-$170 million. This guidance is a non-GAAP projection that excludes share-based compensation and restructuring costs, as more fully described below under “Non-GAAP Financial Measures.”
The Company expects to be cash flow positive in 2017, with a projected year-end cash balance in excess of $200 million.
Third Quarter 2017 Highlights

INBRIJA (levodopa inhalation powder) in Parkinson’s disease
In August, the Company received a Refusal to File (RTF) letter regarding its NDA for INBRIJA. After constructive dialogue with the FDA, the Company expects to resubmit the NDA in Q4 2017.
As a result, the Company has revised the timing for its end-of-year submission of the Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) to Q1 2018.
INBRIJA is an investigational treatment for symptoms of OFF periods in people with Parkinson’s disease taking a carbidopa/levodopa regimen.
Tozadenant in Parkinson’s disease
The Company expects to report topline Phase 3 in Q1 2018.
Tozadenant is an investigational treatment for the reduction of OFF time in people with Parkinson’s disease.
AMPYRA (dalfampridine)
The Company filed its opening brief for its appeal to the U.S. Court of Appeals for the Federal Circuit of the District Court’s decision in the AMPYRA patent litigation. The defendants have filed their opposition and cross-appeal opening brief. Reply briefs from both parties are expected to be filed in November 2017, followed by oral argument to be scheduled by the appellate court.
Both BIO and PhRMA filed amicus briefs in support of the Company’s appeal, raising important issues in conjunction with biopharmaceutical innovation.
The Company expects to maintain exclusivity of AMPYRA at least through July 2018.
Webcast and Conference Call

The Company will host a conference call today at 8:30 a.m. ET. To participate, please dial (844) 579-6824 (domestic) or (763) 488-9145 (international) and reference the access code 95686626. A replay of the call will be available from 11:30 a.m. ET on October 31, 2017 until 2:59 p.m. ET on November 30, 2017. To access the replay, please dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference the access code 95686626. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.On October 30, 2017

Aviragen Therapeutics and Vaxart Enter into Merger Agreement

On October 30, 2017 Aviragen Therapeutics, Inc. (NASDAQ:AVIR), a company focused on the discovery and development of direct-acting antivirals to treat infections that have limited therapeutic options, and Vaxart, Inc., a privately-held, clinical-stage company focused on developing oral recombinant vaccines based on its proprietary delivery platform that allows for administration by tablet rather than by injection, reported that the companies have entered into a definitive merger agreement (Press release, Aviragen Therapeutics, OCT 30, 2017, View Source [SID1234639795]). The merger will result in a combined company, Vaxart, Inc., focused on developing orally-delivered therapeutics and prophylactics to address a variety of viral infections.

"We are thrilled with the prospect of combining forces with Aviragen, which will create a deep pipeline of antiviral products and allow Vaxart to accelerate development of the promising vaccine candidates that are based on our proprietary oral delivery platform," said Wouter Latour, M.D., Chief Executive Officer of Vaxart. "This transaction gives us the opportunity to build on the positive Phase 2 challenge study results we announced recently for our influenza oral tablet vaccine, as well as the excellent results we obtained in the safety and immunogenicity studies with our norovirus vaccine. Additionally, it will provide us access to Aviragen’s antiviral assets, including their BTA074 Phase 2 program for the treatment of condyloma caused by HPV, which is on track to complete enrollment this quarter and to report top-line safety and efficacy data in the second quarter of 2018."

"We believe our oral vaccine programs are significantly de-risked based on the positive clinical outcome of the BARDA-funded H1N1 influenza Phase 2 challenge study which serves as proof of concept for our technology platform as a whole," continued Latour, "and we look forward to taking our norovirus vaccine into a Phase 2 challenge study next. Norovirus is the leading cause of acute viral gastroenteritis in the United States, causing frequent outbreaks across the population, and we believe our oral tablet vaccine would be the optimal approach to address this unmet medical need."

The Vaxart technology platform has been engineered for the delivery of a wide range of oral vaccines, initially targeting norovirus, human papilloma virus (HPV), respiratory syncytial virus, and influenza, using a convenient and room temperature-stable tablet, which eliminates the need for injection. In clinical studies to date, Vaxart vaccines consistently generated broad systemic and local immune responses that could provide important advantages in preventing infection, as well as robust T cell responses that we believe are essential to obtain a therapeutic benefit in chronic viral infection and cancer.

"After a comprehensive review of strategic alternatives, we are delighted to announce this transaction with Vaxart, which will complement Aviragen’s focus on infectious diseases and position us to create both near and long-term value for our stockholders," said Joseph M. Patti, Ph.D., President and Chief Executive Officer of Aviragen Therapeutics. "Vaxart is well-funded to advance its norovirus and HPV antiviral vaccine programs, and together with BTA074, the combined companies are poised to provide meaningful value-creating data readouts."

Today, Vaxart will be announcing positive results from the company’s Phase 1b open-label, dose-ranging study assessing the safety and immunogenicity of VXA-G1.1-NN, Vaxart’s norovirus oral tablet vaccine, in 60 healthy adult volunteers. VXA-G1.1-NN met both the primary and secondary endpoints for safety and immunogenicity in the clinical trial. Based on the favorable clinical data, a Phase 2 norovirus challenge study is expected to begin in the second half of 2018. To date, Vaxart has dosed more than 300 adult volunteers with its vaccines for norovirus, respiratory syncytial virus and influenza.

About the Transaction

The exchange ratio in the merger agreement was determined by assigning $60 million in value to Aviragen for its financial and clinical assets and $90 million in value for Vaxart’s assets. On a pro forma basis, after giving effect to the number of shares of Aviragen common stock issued in the merger, Vaxart’s securityholders will own approximately 60% of the combined company and Aviragen securityholders will own approximately 40% of the combined company, subject to certain potential adjustments as described in the merger agreement. The transaction has been approved by the board of directors of both companies. The merger is expected to close in the first quarter of 2018, subject to the approval of the stockholders of each company as well as other customary conditions. Wouter Latour, M.D., will serve as Chief Executive Officer of the combined company.

Upon the closing of the transaction, the name of the combined company will become Vaxart, Inc. and shares of the combined are expected to continue trading on NASDAQ under the proposed ticker symbol "VXRT."

Stifel, Nicolaus & Company, Incorporated is acting as financial advisor to Aviragen, and Dechert LLP is serving as legal counsel to Aviragen. Cooley LLP is serving as legal counsel to Vaxart.

Aviragen will reduce its workforce by six to a total of 10 full-time employees, who will remain on board to complete the BTA074 Phase 2 clinical trial and assist with the transition of duties to the Vaxart management team.

Aviragen and Vaxart management will host a conference call this morning, Monday, October 30, 2017 at 8:30 a.m. EDT to discuss the planned merger. To participate in the conference call, please dial (877) 312-5422 (United States) or (253) 237-1122 (international) and refer to conference ID number 6295889. A replay of the conference call can be accessed under the Investors section of Aviragen’s website at www.aviragentherapeutics.com and on the Vaxart website at www.vaxart.com.

10-K/A [Amend] – Annual report [Section 13 and 15(d), not S-K Item 405]

10-Q – Quarterly report [Sections 13 or 15(d)]

Vertex Pharmaceuticals has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Vertex Pharmaceuticals, 2017, OCT 30, 2017, View Source [SID1234521308]).

Xynomic Pharma Acquires Global Rights of Boehringer Ingelheim’s BI 882370, a Novel and Potent RAF Inhibitor against Solid Tumors

On October 30, 2018 Xynomic Pharma, a clinical stage US oncology drug development company, reported that it has acquired exclusive global rights to develop, manufacture and commercialize BI 882370, a 2nd-generation RAF inhibitor, from Boehringer Ingelheim, a top-20 global pharmaceutical company (Press release, Xynomic Pharmaceuticals, OCT 30, 2017, View Source [SID1234527683]). Under the terms of the agreement Xynomic will pay upfront, milestone and royalty payments up to approximately $502 million.

BI 882370 is a potent and selective RAF inhibitor uniquely binding to the DFG-out conformation, whereas marketed BRAF inhibitors occupy the DFG-in conformation. BI 882370 inhibited proliferation of BRAFmut melanoma cell lines with 100x higher potency (EC50 1 – 10 nM) than vemurafenib (VEM), a marketed BRAF inhibitor.

In the colorectal cancer (CRC) animal models, BI 882370 was superior to VEM in both the Colo-205V600V/E model and HT-29V600V/E model. BI 882370 in combination with cetuximab induced tumor regressions in the less sensitive HT-29 model.

In melanoma’s G-361V600V/E model, BI 882370 was superior to VEM, marketed BRAF inhibitor dabrafenib (DAB), marketed MEK inhibitor trametinib (TRA) and DAB-TRA combination. In a second melanoma model A375V600E in which tumors developed resistance to VEM, the TRA-BI 882370 combination demonstrated superior efficacy over TRA-DAB combination.

There were no relevant findings in exploratory toxicology studies at exposures delivering efficacy superior to VEM, DAB and TRA.

"BI 882370, with an impressive efficacy and safety profile demonstrated in animal models, is well positioned to become a best-in-class 2nd-generation Pan-RAF inhibitor for the treatment of B-RAF mutant cancers including CRC and melanoma. We are honored to partner with BI, a global leader in oncology, and will move this asset into clinical testing expeditiously," said Y. Mark Xu, Chairman, CEO and President of Xynomic.

RAF inhibitors have attracted resurged and strong interest in oncology. Compared to 1st-generation, BI 882370 may provide an improved therapeutic window, enabling more pronounced and longer-lasting pathway suppression and thus resulting in improved efficacy.

Xynomic’s pipeline also includes Abexinostat, a potentially best-in-class HDAC inhibitor entering global pivotal Ph 3 trials against Non-Hodgkin’s lymphoma and renal cell carcinoma.