AVIRAGEN THERAPEUTICS REPORTS FIRST QUARTER OF FISCAL YEAR 2018 FINANCIAL RESULTS

On November 2, 2017 Aviragen Therapeutics, Inc. (NASDAQ:AVIR) reported its financial results for the three months ended September 30, 2017 (Press release, Nabi Biopharmaceuticals, NOV 2, 2017, View Source [SID1234521518]).

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“Earlier this week we were pleased to announce the culmination of our strategic review process with the signing of a definitive merger agreement with Vaxart, which we believe complements Aviragen’s focus on infectious diseases. With recently reported positive safety and efficacy data in both influenza and norovirus, Vaxart is well-positioned to create both short and long-term value for our stockholders,” said Joseph M. Patti, Ph.D., President and Chief Executive Officer of Aviragen Therapeutics. “Post-merger, we believe that Vaxart will be well funded to advance its norovirus and HPV oral tablet vaccine programs, and together with BTA074, the combined companies are poised to provide several meaningful value creation clinical data readouts.”

Corporate Update:

Proposed Merger with Vaxart:

The exchange ratio in the merger agreement was determined by Vaxart assigning $60,000,000 in value to Aviragen for its financial and clinical assets, and $90,000,000 in value for its own assets. On a pro forma basis after giving effect to the number of shares of Aviragen common stock issued to Vaxart security holders in the merger, current Vaxart security holders will own approximately 60% of the combined company and current Aviragen security holders will own approximately 40% of the combined company. The transactions have been approved by the boards of directors of both companies. The merger is expected to close in the first quarter of calendar year 2018, subject to the approval of the stockholders of each company as well as other customary conditions.

Upon closing of the transaction, the name of the combined company will become Vaxart, Inc. and shares of the combined company are expected to continue trading on the NASDAQ Capital Market under the proposed ticker symbol VXRT. Wouter Latour, M.D., will serve as Chief Executive Officer of the combined company.




BTA074 (teslexivir):

The Phase 2 trial of BTA074, a topical antiviral treatment for condyloma caused by human papillomavirus (HPV), is ongoing and the Company anticipates that enrollment in the 210 patient trial will be completed in the fourth quarter of calendar year 2017. Top-line safety and efficacy data is expected in the second quarter of calendar year 2018.

Financial Results for the Three Month Period Ended September 30, 2017

The Company reported a net loss of $5.3 million for the three month period ended September 30, 2017, as compared to a net loss of $10.0 million in the same quarter of the prior fiscal year. Basic and diluted net loss per share was $0.14 for the three month period ended September 30, 2017, as compared to a basic and diluted net loss per share of $0.26 in the same period in 2016. The major components of net loss in both periods are detailed below.

Revenue was $0.1 million for the three month periods ended September 30, 2017 and 2016. The 2017 revenue relates to $0.1 million in non-cash royalty revenue related to certain royalty rights that were sold to HealthCare Royalty Partners III, L.P. (HCRP) in April 2016 and the cash will be passed through to HCRP. The 2016 revenue was comprised of $0.1 million in Relenza royalties.

Research and development expense decreased to $2.8 million for the three month period ended September 30, 2017 from $7.6 million in the same period in 2016. The $4.8 million decrease largely reflected reduced clinical trial activity and manufacturing costs as two of our three Phase 2 clinical trials finished in the third quarter of fiscal 2017.

General and administrative expense increased to $2.3 million for the three month period ended September 30, 2017 from $2.2 million for the same period in 2016 due mostly to higher legal fees.

The Company held $34.1 million in cash, cash equivalents, and short-term investments as of September 30, 2017.

NanoString Technologies Releases Operating Results for Third Quarter of 2017

On November 2, 2017 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported financial results for the third quarter of 2017 (Press release, NanoString Technologies, NOV 2, 2017, View Source [SID1234521519]). The results were consistent with the company’s announcement of preliminary revenue on October 11, 2017.

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Third Quarter Financial Highlights

Total revenue of $27.0 million, 13% year-over-year growth
Total product and service revenue of $16.9 million, 12% year-over-year decline
Consumables revenue of $10.7 million, including $1.7 million of Prosigna IVD kits, 7% year-over-year decline
Instrument revenue of $4.4 million, 36% year-over-year decline
Collaboration revenue of $10.1 million
“We have taken a number of actions to improve the growth of our life science business, including appointing new commercial leadership, expanding and specializing our sales channel, and launching a number of new consumable products, and we expect these initiatives will help to stabilize the business in the fourth quarter and return to growth over the course 2018,” stated Brad Gray, president and chief executive officer of NanoString. “Our management team is intensely focused on revitalizing the growth of our core business while advancing our pipeline of new instrument platforms, including Digital Spatial Profiling, which is scheduled to launch late next year.”

Recent Business Highlights

Increased installed base to approximately 570 nCounter Analysis Systems at September 30, 2017
Appointed J. Chad Brown as senior vice president, sales & marketing to lead commercial operations
Launched PanCancer IO 360TM gene expression panel for translational research, which assays key pathways from the tumor, microenvironment and immune system and includes more than 20 potentially predictive signatures of therapeutic response to immunotherapy
Entered into collaboration with the NSABP Foundation to study colorectal tumor samples using the PanCancer IO 360 panel to identify biomarkers of immune evasion that may inform the development of novel immunotherapies
Launched new Neuropathology gene expression panel for research of neurological disorders, such as Alzheimer’s Disease, Parkinson’s Disease, Amyotrophic Lateral Sclerosis, and Huntington’s Disease
Entered into $50 million collaboration agreement with Lam Research to advance development of the Hyb & SeqTM next generation sequencing platform
Presented proof-of-principle research demonstrating the capability of Hyb & Seq technology to perform liquid biopsy measurement by direct capture and sequencing of cell-free DNA
Third Quarter Financial Results

Revenue for the three months ended September 30, 2017 increased by 13% to $27.0 million, as compared to $23.9 million for the third quarter of 2016. Instrument revenue was $4.4 million, 36% lower than the prior year period, primarily due to a reduction in the number of systems sold. Consumables revenue, excluding Prosigna, was $9.0 million for the third quarter of 2017, 12% lower than in the comparable 2016 quarter due to a reduction in the consumable pull through from the installed base of systems. Prosigna IVD kit revenue was $1.7 million for the quarter, an increase of 47% over the third quarter of 2016. Collaboration revenue totaled $10.1 million, compared to $4.8 million for the third quarter of 2016. Gross margin on product and service revenue was 57% for the third quarter of 2017, compared to 58% for the third quarter of 2016.

Research and development expense increased by 30% to $11.4 million for the third quarter of 2017 versus $8.7 million for the third quarter of 2016, reflecting investments in new products and technologies under development for the life science research market, including Digital Spatial Profiling and Hyb & Seq technologies, as well as increased costs associated with biopharma collaborations. Selling, general and administrative expense increased by 18% to $18.4 million for the third quarter of 2017 compared to $15.6 million for the prior year period, reflecting added staffing, including expansion of the sales channel, and other costs supporting the company’s growth.

Net loss for the three months ended September 30, 2017 was $11.4 million, or a loss of $0.45 per share, compared with $10.1 million, or $0.51 per share, for the third quarter of 2016.

Outlook for 2017

The company has revised its financial outlook for 2017 as follows:

Total revenue in the range of $109 million to $112 million, which was previously $114 million to $118 million
Product and service revenue in the range of $68 million to $71 million, which was previously $81 million to $85 million
Gross margin on product and service revenue of approximately 56%, which was previously 57% to 58%
Operating expenses in the range of $119 million to $121 million, which was previously $123 million to $126 million
Operating loss in the range of $38 million to $41 million, which was previously $42 million to $46 million
Net loss per share in the range of $1.86 to $1.99, which was previously $2.03 to $2.20
Conference Call

Management will host a conference call today beginning at 1:30 pm PT / 4:30 pm ET to discuss these results and answer questions. Individuals interested in listening to the conference call may do so by dialing (888) 793-9492 for domestic callers, or (734) 385-2643 for international callers. Please reference Conference ID 88331168. To listen to a live webcast, please visit the investor relations section of the company’s website at: www.nanostring.com. A replay of the call will be available beginning November 2, 2017 at 7:30pm ET through 7:30pm ET on November 9, 2017. To access the replay, dial (855) 859-2056 or (404) 537-3406 and reference Conference ID: 88331168. The webcast will also be available on the company’s website for one year following the completion of the call.

OncoMed Announces Third Quarter 2017 Financial Results and Operational Highlights

On November 2, 2017 OncoMed Pharmaceuticals, Inc. (NASDAQ:OMED), a clinical-stage biopharmaceutical company focused on discovering and developing novel anti-cancer therapeutics, reported third quarter financial results (Press release, OncoMed, NOV 2, 2017, View Source [SID1234521520]). As of September 30, 2017, cash, cash equivalents and short-term investments totaled $113.6 million.

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“OncoMed remains committed to developing novel anti-cancer therapeutics to improve the lives of patients. With four clinical programs progressing, including the newly initiated Phase 1a study of GITRL-Fc (OMP-336B11), OncoMed is positioned to deliver value for both patients and our shareholders,” commented Sunil Patel, Executive Vice President and Chief Financial Officer. “We look forward to upcoming clinical updates from our navicixizumab, rosmantuzumab and anti-TIGIT programs in 2018.”

Pipeline Highlights

GITRL-Fc (OMP-336B11)

In September, OncoMed dosed the first patient in a Phase 1a single agent study of its wholly-owned GITRL-Fc in patients with advanced or metastatic solid tumors. GITRL-Fc is a fusion protein with an Fc-linked fully human trimer ligand and is designed to activate the co-stimulatory receptor GITR (glucocorticoid-induced tumor necrosis factor receptor) to enhance T-cell modulated immune responses. The Phase 1a study is designed to assess safety and tolerability of escalating doses.
Anti-TIGIT (OMP-313M32)

OncoMed now plans to initiate the Phase 1b portion of its anti-TIGIT trial to study anti-TIGIT in combination with anti-PD1 in the first half of 2018. The Phase 1b portion of the anti-TIGIT trial will be designed to assess safety and tolerability of escalating doses of the combination treatment.
OncoMed continues enrollment in the Phase 1a single-agent study of anti-TIGIT in patients with advanced or metastatic solid tumors. The Phase 1a study is designed to assess safety and tolerability of escalating doses, and interim data from this trial are expected to be reported by year-end 2018.
Navicixizumab (anti-DLL4/VEGF bispecific; OMP-305B83)

Enrollment continues in two Phase 1b multi-center, open-label, dose escalation and expansion studies of OncoMed’s anti-DLL4/VEGF bispecific antibody in combination with standard-of-care chemotherapies: one in patients with platinum-resistant ovarian cancer who have failed more than two prior therapies or prior bevacizumab and a second in patients with 2nd line metastatic colorectal cancer.
To date, OncoMed has enrolled over 80 patients across the Phase 1a and Phase 1b trials. Interim data are expected to be reported in 2018.
Rosmantuzumab (anti-RSPO3; OMP-131R10)

The Phase 1a/b multi-center, open-label, dose escalation and expansion study of OncoMed’s anti-RSPO3 antibody in patients with advanced solid tumors (Phase 1a) and in patients with previously treated metastatic colorectal or gastric cancer (Phase 1b; in combination with FOLFIRI) continues. As previously announced, enrollment in the study has recently been limited to patients that harbor an RSPO3 gene fusion.
Vantictumab (anti-Fzd; OMP-18R5) and Ipafricept (Fzd8-Fc; OMP-54F28)

OncoMed continues to evaluate potential partnering opportunities for Wnt/IO combinations.
Data were presented at the 2017 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) demonstrating that a 6-gene breast cancer signature significantly correlated with PFS and OS outcomes in OncoMed’s completed Phase 1b trial in 40 patients with baseline tumor assessments.
Third Quarter 2017 Financial Results

Cash, cash equivalents and short-term investments totaled $113.6 million as of September 30, 2017, compared to $184.6 million as of December 31, 2016.

Revenues were $5.1 million for the third quarter of 2017, a decrease of $0.8 million, compared to $5.9 million for the same period in 2016. The decrease in revenue was primarily due to lower revenue recognized from reimbursement of research and development costs for services performed in the third quarter of 2017.

Research and development (R&D) expenses were $12.2 million for the third quarter 2017, a decrease of $15.2 million, compared to $27.4 million for the same period in 2016. The decrease was primarily due to lower external research and development costs attributable to the decrease in Phase 2 clinical trial costs of demcizumab and tarextumab programs as a result of our discontinuation of the dosing of all patients of these programs, and a decrease in internal program costs due to reduced headcount as a result of the restructuring actions implemented in April 2017.

General and administrative (G&A) expenses were $3.9 million for the third quarter of 2017, a decrease of $0.6 million, compared to $4.5 million for the same period in 2016. The decrease was primarily attributable to a decrease in personnel costs, including stock-based compensation expenses, due to a decrease in headcount as a result of the restructuring actions implemented in April 2017.

Net loss for the third quarter of 2017 was $10.7 million ($0.28 per share), compared to $25.9 million ($0.77 per share) for the same period of 2016. The change in net loss from the prior year quarter was due to lower R&D and G&A expenses.

2017 Financial Guidance

OncoMed anticipates 2017 full-year cash utilization will be approximately $90 million. Based on the current plan, OncoMed anticipates that its current cash balance is sufficient to fund pipeline development and company operations through the third quarter of 2019, before considering potential opt-in milestones.

Corporate Updates

OncoMed Chairman and CEO, Paul Hastings, on Medical Leave: Mr. Hastings continues a personal leave of absence for medical reasons. As previously announced, Mr. Hastings will continue to be the Chief Executive Officer and Chairman of the Board of Directors during his leave of absence. In Mr. Hastings’ absence, OncoMed is led by the Office of the President consisting of Executive Vice President, Research and Development, John Lewicki, and Executive Vice President and Chief Financial Officer, Sunil Patel. In addition, OncoMed’s Board of Directors has appointed a special committee of the Board, consisting of Jack Lasersohn, lead director of the Board, Perry Karsen, Deepa Pakianathan and Rick Winningham, to work closely with Dr. Lewicki and Mr. Patel during Mr. Hastings’ medical leave.

Conference Call Today

OncoMed management will host a conference call today beginning at 4:30 p.m. ET / 1:30 p.m. PT to review third quarter 2017 financial results and corporate updates.

Analysts and investors can participate in the conference call by dialing 1 (855)-859-2056 (domestic) and 1(484) 756-4194 (international) using the conference ID# 4699289. The webcast of the conference call can be accessed live on the Investor Relations section of the OncoMed website, View Source

An audio replay of the conference call can be accessed by dialing 1 (855) 859-2056 (domestic) or 1 (404) 537-3406 (international) utilizing the conference ID number listed above. The web broadcast of the conference call will be available for replay through the OncoMed website.

PDL BioPharma Announces Third Quarter 2017 Financial Results

On November 2, 2017 PDL BioPharma, Inc. (PDL or the Company) (NASDAQ: PDLI) reported financial results for the third quarter ended September 30, 2017 including (Press release, PDL BioPharma, NOV 2, 2017, View Source [SID1234521521]):

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Total revenues of $62.7 million and $252.0 million for the three and nine months ended September 30, 2017, respectively.
GAAP diluted EPS of $0.14 and $0.56 for the three and nine months ended September 30, 2017, respectively.
GAAP net income attributable to PDL’s shareholders of $20.7 million and $88.4 million for the three and nine months ended September 30, 2017, respectively.
Non-GAAP net income attributable to PDL’s shareholders of $21.7 million and $73.7 million for the three and nine months ended September 30, 2017. A full reconciliation of all components of the GAAP to non-GAAP financial results can be found in Table 4 at the end of the release.
Revenue Highlights

Total revenues of $62.7 million for the three months ended September 30, 2017 included:
Royalties from PDL’s licensees to the Queen et al. patents of $1.4 million, which consisted of royalties earned on sales of Tysabri under a license agreement;
Net royalty payments from acquired royalty rights and a change in fair value of the royalty rights assets of $35.4 million, which consisted of the change in estimated fair value of our royalty right assets, primarily related to Depomed, Inc.;
Interest revenue from notes receivable financings to kaléo and CareView Communications of $6.1 million; and
Product revenues of $20.1 million, which consisted of $15.1 million from sales of Tekturna and Tekturna HCT in the United States, Rasilez and Rasilez HCT in the rest of the world (collectively, the Noden Products) and $5.0 million for sales and leasing of the LENSAR Laser System.
Total revenues increased by 17 percent for the three months ended September 30, 2017, when compared to the same period in 2016.
Royalties from PDL’s licensees to the Queen et al. patents were lower due to reduced sales of Tysabri that was manufactured prior to the patent expiry date;
The increase in royalty rights – change in fair value was primarily due to the current period increase in fair value of the Depomed, Inc. royalty asset by $22.0 million.
PDL received $26.3 million in net cash royalties from its royalty rights in the third quarter of 2017, compared to $15.3 million for the same period of 2016. The increase in cash royalties is mainly due to the launch of the authorized generic for Glumetza sold by Valeant Pharmaceuticals International, Inc. (Valeant) subsidiary, Oceanside Pharmaceuticals, Inc. PDL received royalties on the authorized generic equivalents under the same terms as the branded Glumetza.
The decrease in interest revenues was primarily due to the early repayment of the Paradigm Spine, LLC note receivable investment.
The increase in product revenues were derived from the sale and lease of the LENSAR Laser System, which PDL did not begin to recognize until May 11, 2017.
Total revenues increased by 42 percent for the nine months ended September 30, 2017, when compared to the same period in 2016.
The decrease in royalties from PDL’s licensees to the Queen et al. patents is due to the expiration of the patent license agreement with Genentech, Inc. and reduced royalties on Tysabri.
The increase in royalty rights – change in fair value was primarily due to the year-to-date increase in fair value of the Depomed, Inc. royalty asset by $144.3 million.
PDL received $74.4 million in net cash royalties from its royalty rights in the nine months ended September 30, 2017, compared to $47.2 million for the same period of 2016.
The decrease in interest revenues was primarily due to the early repayment of the Paradigm Spine, LLC note receivable investment and ceasing to recognize interest from the LENSAR note receivable.
Product revenue increased due to sales of the Noden Products, which PDL did not begin to recognize until the third quarter of 2016 and the sale and lease of the LENSAR Laser System, which PDL did not begin to recognize until May 11, 2017.
License and other revenue increased by $19.5 million primarily due to a $19.5 million payment from Merck as part of the previously announced settlement agreement to resolve the patent infringement lawsuits related to Keytruda.
Operating Expense Highlights

Operating expenses were $30.1 million for the three months ended September 30, 2017, compared to $21.0 million for the same period of 2016. The increase in operating expenses for the three months ended September 30, 2017, as compared to the same period in 2016, was primarily a result of the $5.6 million increase in costs of Noden and LENSAR product revenues, $5.0 million increase in Noden and LENSAR sales and marketing costs due to the increase in sales force headcount, and increase general and administrative expenses, partially offset by the $1.4 million decrease in amortization of the Novartis anniversary payment and contingent consideration.
Operating expenses were $88.1 million for the nine months ended September 30, 2017, compared to $40.7 million for the same period of 2016. The increase in operating expenses for the nine months ended September 30, 2017, as compared to the same period in 2016, was primarily a result of the $12.6 million increase in costs of Noden and LENSAR product revenues, the $12.4 million increase in amortization of intangible assets, the $11.2 million increase in Noden and LENSAR sales and marketing costs due to a increase in sales force headcount, the $8.7 million increase general and administrative expenses related to the Noden and LENSAR businesses being acquired by PDL in the prior year, and $4.7 million increase in research and development, partially offset by the $3.5 million decrease in acquisition related expenses related to the Noden acquisition in 2016.
Recent Developments

On October 27, 2017, PDL and Depomed, Inc. entered into a settlement agreement with Valeant Pharmaceuticals International, Inc. to resolve all matters addressed in the lawsuit filed by Depomed on September 7, 2017 relating to underpayment of royalties by Valeant. Under the terms of the Settlement Agreement, the litigation will be dismissed, with prejudice, and Valeant paid a one-time, lump-sum payment of $13.0 million, which will be transferred to PDL pursuant to the terms of the Depomed Royalty Agreement. The cash from the settlement agreement is expected to be received in Q4 2017 and has been reflected in the Depomed royalty rights asset discounted cashflow valuation as of September 30, 2017.
On October 26, 2017, PDL submitted a proposal to acquire Neos Therapeutics, Inc. for $10.25 per share in cash, which represented a premium of 40 percent to the closing price of Neos shares on October 25, 2017 and a premium of 41 percent to Neos’ share price prior to PDL’s initial proposal on June 23, 2017. The acquisition of Neos is consistent with PDL’s stated strategy for growth and is a logical next step in the execution of its strategic plan. In particular, the Company believes that this acquisition would create an attractive pediatric platform and foundation for future growth. Subsequently, Neos’ Board of Directors rejected PDL’s proposal and refuses to engage in a constructive dialogue with PDL management on behalf of Neos’ shareholders. PDL has a number of investment opportunities before it, of which Neos is only one. PDL’s proposal remains outstanding through November 8, 2017. PDL will evaluate all of its options in the interim.
On October 26, 2017, Biogen sent to PDL a notice of overpayment related to royalties on Tysabri on sales in the US, Spain, Italy and South Africa for $13.5 million through the period ending September 30, 2017. The notice states that the overpayment was the result of royalties being paid on product manufactured after the expiration of the Queen et al. patents. PDL received cash payments of $14.9 million during the third quarter of 2017. As a result of the receipt of this overpayment notice, royalty revenue from the Queen et al. patents was $1.4 million, in the third quarter of 2017, which was the net amount of $14.9 million cash received and the potential overpayment of $13.5 million. PDL recorded a refund liability for the potential overpayment amount of $13.5 million at September 30, 2017. Biogen indicated to us that royalty payment for Tysabri in the fourth quarter of 2017 will be $4.5 million leaving a net potential overpayment of $9.0 million. PDL is currently working with Biogen to resolve this issue.
In October 2017, PDL received a royalty payment from Valeant in the amount of $6.9 million for royalties earned on sales of Glumetza for the month of September. The royalty payment included royalties related to the authorized generic version of Glumetza.
On September 21, 2017, PDL entered into an agreement with a third-party purchaser, pursuant to which PDL sold its entire interest in the kaléo, Inc note. Pursuant to the agreement, the purchaser paid PDL an amount equal to 100% of the then outstanding principal plus a premium of 1% of the principal amount and accrued interest, for an aggregate cash purchase price of $141.7 million, subject to an 18-month escrow holdback of $1.4 million against certain potential contingencies.
Other Financial Highlights

PDL had cash, cash equivalents, short-term investments and other investments of $516.5 million at September 30, 2017, compared to $242.1 million at December 31, 2016.
Net cash provided by operating activities in the nine months ended September 30, 2017 was $58.1 million, compared with $86.1 million in the same period in 2016. The decrease was as a result of the fair value changes of PDL’s royalty rights.
PDL anticipates an estimated cash tax rate of 22% as the company begins to utilize available tax operating loss carry forwards and credits and expects an effective tax rate of approximately 41% in fiscal 2017, which is dependent on the mix and timing of income.
Conference Call and Webcast Details

PDL will hold a conference call to discuss financial results at 4:30 p.m. Eastern Time today, November 2, 2017.

To access the live conference call via phone, please dial (800) 668-4132 from the United States and Canada or (224) 357-2196 internationally. The conference ID is 8794857. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available beginning approximately one hour after the call through one week following the call, and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 8794857.

To access the live and subsequently archived webcast of the conference call, go to the Company’s website at View Source and go to “Events & Presentations.” Please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

PTC Therapeutics Reports Third Quarter 2017 Financial Results and Provides Corporate Update

On November 2, 2017 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the third quarter ending September 30, 2017 (Press release, PTC Therapeutics, NOV 2, 2017, View Source [SID1234521522]).

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“Our performance this quarter combined with our commercial, financial and R&D advancements should allow us to end 2017 in a strong position,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “Our commercial success is driven by our mission of improving the lives of patients with Duchenne.”

Third Quarter Financial Highlights:

Translarna net product sales were $32.0 million for the third quarter of 2017, representing 45% growth over $22.0 million reported in the third quarter of 2016.
EMFLAZA net product sales were $9.8 million for the third quarter of 2017.
Total revenues for the third quarter of 2017 were $41.9 million compared to $23.0 million in the same period of 2016. The change in total revenue was a result of the expanded commercial launch of Translarna and the successful U.S. EMFLAZA launch.
GAAP R&D expenses were $30.0 million for the third quarter of 2017 compared to $31.4 million for the same period in 2016. Non-GAAP R&D expenses were $26.4 million for the third quarter of 2017, excluding $3.6 million in non-cash, stock-based compensation expense, compared to $27.1 million for the same period in 2016, excluding $4.3 million in non-cash, stock-based compensation expense. The decrease in R&D expense for the third quarter of 2017 as compared to the prior year period was primarily due to the completion of our Phase 3 Translarna trials at the end of 2016 partially offset by start-up clinical activities and regulatory spend.
GAAP SG&A expenses were $31.4 million for the third quarter of 2017 compared to $23.7 million for the same period in 2016. Non-GAAP SG&A expenses were $27.9 million for the third quarter of 2017, excluding $3.5 million in non-cash, stock-based compensation expense, compared to $19.0 million for the same period in 2016, excluding $4.6 million in non-cash, stock-based compensation expense. The increase in SG&A expenses primarily related to the expansion of the U.S. commercial sales team in support of the launch of EMFLAZA.
Net interest expense for the third quarter of 2017 was $3.4 million compared to net interest expense of $2.1 million in the same period in 2016. The increase in net interest expense is primarily a result of increased interest expense related to the $40 million secured loan facility which we closed during the second quarter of 2017 partially offset by reduced interest income from investments.
Net loss for the third quarter of 2017 was $33.7 million compared to a net loss of $35.2 million for the same period in 2016.
Cash, cash equivalents, and marketable securities totaled approximately $169.3 million at September 30, 2017 compared to approximately $231.7 million at December 31, 2016.
Shares issued and outstanding as of September 30, 2017, were 41.5 million, which includes 0.1 million shares of unvested restricted stock awards.
2017 Guidance:

Translarna net sales guidance for 2017 is anticipated to be between $120 and $140 million. We now anticipate EMFLAZA net sales for 2017 to be between $20 and $25 million, an increase from our prior guidance of $15 to $20 million. This brings 2017 full year revenue guidance between $160 and $185 million, an increase from our prior guidance of $155 million to $180 million, including a $20 million milestone we achieved in mid-October, under our SMA program.
GAAP R&D and SG&A expense for the full year 2017 are now anticipated to be between $250 to $260 million. Excluding estimated non-cash, stock-based compensation expense of approximately $40 million, full year 2017 non-GAAP R&D and SG&A expense are now anticipated to be between $210 million and $220 million. These expenses will be primarily in support of the commercial availability of Translarna globally, the commercial launch of EMFLAZA in the U.S. and the continued research and clinical development of other product pipeline candidates.
We now expect to end 2017 with over $150 million of cash and cash equivalents, an increase from prior guidance of $120 million.
Key Third Quarter and other Corporate Highlights:

Filed Formal Dispute Resolution Request with U.S. FDA to appeal Complete Response Letter for ataluren. PTC received a Complete Response Letter from the Office of Drug Evaluation I of the U.S. Food and Drug Administration (FDA) for the New Drug Application (NDA) of the investigational medicine ataluren for the treatment of nonsense mutation dystrophinopathies. The letter stated that it is unable to approve the application in its current form. PTC has filed a formal dispute resolution request challenging this decision.
EMFLAZA revenue grew to $9.8M in third quarter. PTC is committed to enabling access to EMFLAZA for all patients in need, regardless of financial situation or insurance status. We demonstrated an increase in EMFLAZA prescriptions over the past quarter. There are currently over 1,500 patients on EMFLAZA and we estimate that there are approximately 9,000 Duchenne patients in the U.S. over the age of five who are eligible to be prescribed EMFLAZA.
Continued global expansion of Translarna results in revenue of $32.0M in third quarter. PTC continues to expand its strong global footprint, with sales generated in over 25 countries. This strong performance reflects continued uptake, sustainable pricing levels, and high ( > 90%) compliance to treatment.
SMA clinical program advanced into the pivotal portion of the study. In mid-October, the SUNFISH trial transitioned into the pivotal portion of the study which triggered a $20M milestone to PTC from Roche. Data from the SUNFISH trial was presented at the International Congress of the World Muscle Society. An interim analysis of the five cohorts treated with RG7916 for 28 days demonstrates an exposure-dependent increase in SMN protein.
Non-GAAP Financial Measures:
In this press release, the financial results and financial guidance of PTC are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial measures exclude stock-based compensation expense and one-time restructuring expenses relating to the reorganization of operations intended to improve efficiency and better align costs and employment structure with PTC’s strategic plans. These non-GAAP financial measures are provided as a complement to results reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the operating performance at PTC and the company’s future outlook. Quantitative reconciliations of GAAP financial measures are included in the tables below