On November 2, 2016 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the third quarter ending September 30, 2016 (Press release, PTC Therapeutics, NOV 2, 2016, View Source [SID1234516313]).

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"For the past 18 years, we have focused significant effort towards developing Translarna for those affected by Duchenne," stated Stuart W. Peltz, Ph.D. Chief Executive Officer, PTC Therapeutics, Inc. "Over 400 Duchenne patients have participated in our clinical trials dating back to 2005 and the vast majority continue to remain on Translarna including approximately 130 boys in the United States. With this high compliance rate, we believe these actions speak to the benefit Translarna is providing to patients in the U.S. and around the world. In the E.U., regulatory discussions continue and we anticipate an opinion from the CHMP before the end of the year. With respect to the U.S., we will continue to escalate our appeal so that we may have the opportunity to have the Translarna NDA submission reviewed by the FDA. Our goal remains to deliver this novel therapy to nonsense mutation Duchenne patients globally."

Key Third Quarter and Other Corporate Highlights:

Translarna net sales of $22M in the third quarter represents 125% year-over-year growth. Commercial access to Translarna continued to expand during the quarter with a significant number of new patients coming from England. While pricing and reimbursement discussions with a number of European countries are ongoing, PTC has now successfully concluded pricing and reimbursement negotiations in both Italy and Romania. PTC anticipates growth from both additional penetration into existing markets as well as expanding access in new geographies across Central and Eastern Europe, the Middle East and Latin America.
EMA regulatory discussions regarding Translarna’s marketing authorization are ongoing. PTC continues to interact with the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) in connection with the company’s ongoing request for annual renewal of its marketing authorization for Translarna. Following the company’s CHMP meeting in October, the committee requested additional information regarding the risk-benefit profile of Translarna, its efficacy and the design of a potential trial that would provide comprehensive clinical data. While we have provided information in response to the CHMP’s requests and expect to continue to engage in further interactions, recent dialogue has introduced a higher degree of uncertainty as to the outcome. PTC anticipates that an opinion regarding its marketing authorization renewal request will be adopted by the CHMP before the end of 2016. The current marketing authorization remains valid while the EMA assessment is ongoing and until a decision is made by the European Commission.
Appeal process of FDA’s Refuse to File regarding Translarna for DMD continues. As PTC previously stated, the RTF appeal could be an iterative process requiring multiple rounds before a conclusion. Having had its appeal denied, the company intends to continue the appeal to higher levels of the FDA. PTC continues to believe that the FDA can only accomplish a proper assessment of the data and analyses based on the results of the company’s extensive clinical research and experience in more than 400 boys in the context of a full and fair review. This would include an advisory committee meeting that allows DMD clinical experts and representatives of the patient community to express their views on Translarna for the treatment of nmDMD. PTC intends to provide an update following a final conclusion of the appeal process or, alternatively, following a determination by the company to pursue an alternate regulatory strategy for advancing the potential approval of Translarna for the treatment of nmDMD in the U.S.
ACT CF Phase 3 clinical trial results expected late first quarter 2017. PTC’s confirmatory Phase 3 ACT CF clinical trial, a 48-week placebo-controlled trial designed to evaluate the efficacy of Translarna in patients six years of age or older with nonsense mutation cystic fibrosis, is ongoing. Following feedback from the FDA and the company’s CHMP rapporteurs during the third quarter, PTC modified the protocol for ACT CF. In-line with clinical trials for other approved CF therapies, the primary endpoint of lung function as measured by FEV1 was updated from relative change to absolute change in percent predicted FEV1. Pulmonary exacerbations will be an important secondary endpoint.
SMA program advancing with Phase 2 SUNFISH trial initiated in Type 2/3 patients. PTC’s joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation initiated a Phase 2 study in pediatric and adult Type 2/3 SMA patients. SUNFISH, is a two-part study investigating the safety, tolerability and efficacy of RG7916, an oral small molecule survival motor neuron 2 (SMN2) splicing modifier. The first part of the study will evaluate safety and tolerability through escalating doses of RG7916. After dose selection, the study will transition into the pivotal second part evaluating the efficacy of RG7916. Initiation of the pivotal second part of the study is expected to begin in 2017 and will trigger a $20 million milestone payment to PTC from Roche. A similarly designed two-part study to evaluate RG7916 in Type 1 SMA patients is expected to begin in the coming months.
PTC596 cancer stem cell program to advance in clinical development in 2017.
Phase 1 safety data from PTC’s clinical oncology program were recently presented at the European Society for Medical Oncology Congress. The ongoing Phase 1 dose-escalation study is evaluating the safety, tolerability and pharmacokinetics of PTC596 in patients with advanced solid tumors as a monotherapy. Preliminary results demonstrate that PTC596 is generally well tolerated at doses associated with exposures that achieved or exceeded efficacious target plasma concentrations in preclinical studies. Escalating doses are being evaluated for safety, pharmacodynamics, and to determine a target dose for subsequent studies. PTC596 is a novel, oral investigational drug that reduces the levels of BMI1, a protein that is required for cancer stem cell survival. Based on its proposed mechanism of action, PTC596 is expected to be most efficacious when used as part of combination therapy. Continued clinical development of PTC596 is being planned for 2017.
Third Quarter Financial Highlights:

Translarna net product sales were $22.0 million for the third quarter of 2016, representing a 125% increase versus $9.8 million in the third quarter of 2015. Net sales increased in conjunction with continued expansion of commercial access to Translarna for nonsense mutation DMD boys outside of the U.S.
Total revenues for the third quarter of 2016 were $23.0 million, an increase of $13.2 million or 135% compared to $9.8 million in the same period of 2015. The change in total revenue was primarily a result of the expanded commercial launch of Translarna.
GAAP R&D expenses were relatively flat at $31.4 million for the third quarter of 2016 compared to $30.6 million for the same period in 2015. Non-GAAP R&D expenses were $27.1 million for the third quarter of 2016, excluding $4.3 million in non-cash, stock-based compensation expense, compared to $26.8 million for the same period in 2015, excluding $3.8 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $23.7 million for the third quarter of 2016 compared to $21.4 million for the same period in 2015. Non-GAAP SG&A expenses were $19.0 million for the third quarter of 2016, excluding $4.6 million in non-cash, stock-based compensation expense, compared to $17.1 million for the same period in 2015, excluding $4.2 million in non-cash, stock-based compensation expense. The increase in SG&A expense for the third quarter 2016 as compared to the prior year period primarily resulted from additional costs associated with commercial activities in support of Translarna across Europe and other regions.
Net interest expense for the third quarter of 2016 was $2.1 million compared to net interest expense of $0.9 million in the same period in 2015. The increase in interest expense is primarily a result of the $150 million convertible debt offering completed during mid-third quarter 2015. The debt was recorded on PTC’s balance sheet at a discount, which will be amortized over the life of the bond.
Net loss for the third quarter of 2016 was $35.2 million, a decrease of $8.1 million compared to a net loss of $43.2 million for the same period in 2015.
Cash, cash equivalents, and marketable securities totaled approximately $248.3 million at September 30, 2016 compared to approximately $338.9 million at December 31, 2015.
Shares issued and outstanding as of September 30, 2016 were 34.3 million, which includes 0.2 million shares of unvested restricted stock.
2016 Guidance:

PTC expects to achieve total ex-US nmDMD Translarna net sales in the middle of our guidance of $65 to $85 million for 2016.
Operating expenses for the full year 2016 are now anticipated to be between $180 million and $190 million, excluding expected non-cash stock-based compensation expense of approximately $35 million, for total operating expenses of approximately $215 million to $225 million. These expenses are in support of our ongoing clinical trials for Translarna in nmDMD and nmCF, commercial launch activities for Translarna outside of the U.S., and the continued research and clinical development of other product pipeline candidates.
PTC expects to end 2016 with cash and cash equivalents of approximately $220 million.

On November 2, 2016 Aduro Biotech, Inc. (NASDAQ:ADRO) reported financial results for the third quarter 2016 (Press release, Aduro BioTech, NOV 2, 2016, View Source;p=RssLanding&cat=news&id=2218713 [SID1234516171]). Net loss for the three months ended September 30, 2016 was $35.1 million, or $0.54 per share, and for the nine months ended September 30, 2016 net loss was $61.6 million, or $0.96 per share, compared to a net income of $0.6 million, or $0.01 per share, and net loss of $42.3 million, or $1.09 per share respectively, for the same periods in 2015.

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Cash, cash equivalents and marketable securities totaled $387.1 million at September 30, 2016, compared to $431.0 million at December 31, 2015.

Third Quarter 2016 Financial Results

Revenue for the quarter and nine months ended September 30, 2016 was $3.8 million and $46.8 million, respectively, compared to $19.1 million and $38.6 million, for the same periods in 2015. The decrease in the third quarter of 2016 was primarily related to the full recognition of the Janssen upfront license fee in 2015. The increase for the nine months ended September 30, 2016 was primarily due to the receipt of a $35.0 million milestone payment from Novartis.

Research and development expenses for the quarter and nine months ended September 30, 2016 were $19.0 million and $66.9 million, respectively, compared to $11.8 million and $36.0 million for the same periods in 2015. The increase for the quarter was primarily due to licensing fees related to our STING technology platform and additional personnel-related costs which include stock-based compensation, partially offset by declines in contract manufacturing and clinical trial expenses for our pancreatic cancer program. The increase for the nine month period was primarily due to GVAX pancreas manufacturing expenses during the first half of 2016, and to a lesser extent due to additional personnel-related costs, contract research expenses, and licensing fees.

General and administrative expenses for the quarter and nine months ended September 30, 2016 were $8.6 million and $26.3 million, respectively, compared to $6.9 million and $19.0 million for the same periods in 2015. The increases in both periods were primarily due to additional personnel-related costs, including stock-based compensation, and the expansion of our office and laboratory facilities.

There was no loss from remeasurement of fair value of warrants during the quarter or nine months ended September 30, 2016 or for the third quarter of 2015. The $26.1 million loss from remeasurement of fair value of warrants for the nine months ended September 30, 2015 occurred in April 2015 when certain outstanding warrants were no longer subject to future remeasurement.

Provision for income taxes for the quarter and nine months ended September 30, 2016 was $11.7 million and $16.4 million, respectively. There was no provision for income taxes for the comparable periods in 2015. The income tax expense recorded for the quarter and nine months ended September 30, 2016 was primarily related to current and deferred federal income taxes.

On November 1, 2016 Array BioPharma Inc. (Nasdaq: ARRY), a biopharmaceutical company focused on the discovery, development and commercialization of targeted small molecule cancer therapies, reported results for its first quarter of fiscal 2017 and provided an update on the progress of its key clinical development programs (Press release, Array BioPharma, NOV 1, 2016, View Source;p=RssLanding&cat=news&id=2217854 [SID1234516133]).

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Array BioPharma. (PRNewsFoto/Array BioPharma Inc.) (PRNewsFoto/)
"We were pleased to report that COLUMBUS met its primary endpoint and demonstrated a robust PFS benefit associated with the combination of binimetinib plus encorafenib versus vemurafenib in patients with BRAF-mutant melanoma," said Ron Squarer, Chief Executive Officer of Array BioPharma. "We look forward to sharing results at SMR, partnering with global regulatory authorities on planned 2017 submissions for COLUMBUS and preparing for potential commercialization."

KEY COMPANY AND PIPELINE UPDATES
Binimetinib (MEK162) and encorafenib (LGX818)
Novartis continues to substantially fund all ongoing trials with binimetinib and encorafenib that were active or planned as of the close of the Novartis Agreements in 2015, including the NEMO and COLUMBUS Phase 3 trials. Reimbursement revenue from Novartis was approximately $129.0 million for the previous 12 months, of which $31.3 million was recorded over the past quarter.

COLUMBUS: Global Phase 3 trial of binimetinib plus encorafenib versus vemurafenib in BRAF-mutant melanoma patients
In September 2016, Array announced top-line results from Part 1 of the Phase 3 COLUMBUS study evaluating LGX818 (encorafenib), a BRAF inhibitor, and MEK162 (binimetinib), a MEK inhibitor, in patients with BRAF-mutant advanced, unresectable or metastatic melanoma. The study met its primary endpoint, with the combination of encorafenib and binimetinib ("combination") significantly improving progression free survival (PFS) compared with vemurafenib, a BRAF inhibitor, alone. In the analysis of the primary endpoint, the median PFS for patients treated with the combination was 14.9 months versus 7.3 months for patients treated with vemurafenib; Hazard ratio (HR) 0.54, (95% CI 0.41-0.71, p<0.001). The combination was generally well-tolerated and reported adverse events were overall consistent with previous clinical trial results of the combination in BRAF-mutant melanoma patients. Analysis of a secondary endpoint comparing the median PFS of patients treated with the combination to patients treated with encorafenib alone showed 14.9 months versus 9.6 months, respectively; HR 0.75, (95% CI 0.56-1.00, p=0.051), which did not reach statistical significance. A complete analysis of these results will be provided to global regulatory authorities as part of planned submissions in 2017. In addition, data from Part 2 of the COLUMBUS trial are anticipated in mid-2017 and will also be provided to global health authorities as part of planned regulatory submissions seeking approval of these product candidates.

Further results from Part 1 of the COLUMBUS trial will be presented at the Society for Melanoma Research Congress on November 9, 2016. Analysis of the secondary endpoint of overall survival (OS) was not planned as part of these initial results. Binimetinib and encorafenib are investigational medicines and are not currently approved in any country.

Melanoma is the fifth most common cancer among men and the seventh most common cancer among women in the United States, with more than 76,000 new cases and over 10,000 deaths from the disease expected in 2016. Novel therapies that target the RAS/RAF/MEK/ERK pathway have a strong scientific rationale for activity in this disease, as up to 50 percent of patients with metastatic melanoma have activating BRAF mutations, the most common gene mutation in this patient population. Current marketed MEK/BRAF combination agents have a run rate forecasted to approach $1 billion in annual worldwide sales.

NEMO: Global Phase 3 trial of binimetinib versus dacarbazine in NRAS-mutant melanoma patients
In September 2016, Array announced that the FDA has accepted its NDA for binimetinib in NRAS-mutant melanoma with a target action date under the Prescription Drug User Fee Act (PDUFA) of June 30, 2017. Also, the binimetinib Marketing Authorization Application (MAA) submitted by Pierre Fabre was validated and is currently under evaluation by the Committee for Medicinal Products for Human Use (CHMP). The FDA indicated that it plans to hold an advisory committee meeting (ODAC) in the first half of 2017 as part of the review process.

Activating NRAS mutations are present in up to 20 percent of patients with metastatic melanoma, and are a poor prognostic indicator for these patients. Treatment options for this population remain limited beyond immunotherapy, and these patients face poor clinical outcomes and high mortality.

BEACON CRC: Global Phase 3 trial of binimetinib, encorafenib and Erbitux (cetuximab) versus Erbitux in BRAF-mutant colorectal cancer (CRC) patients
Array is advancing BEACON CRC, a global Phase 3 trial of encorafenib and Erbitux (cetuximab), with or without binimetinib, versus standard of care in patients with BRAF-mutant CRC who have previously received first-or second-line systemic therapy. In September 2016, Array announced that it has reached agreement with the FDA regarding a Special Protocol Assessment (SPA) related to BEACON CRC. The SPA acknowledges that the design and planned analysis of BEACON CRC adequately address the objectives necessary to support a regulatory submission for the approval of the doublet regimen of encorafenib and Erbitux. The FDA also communicated that sharing evidence from the study that the triplet regimen (encorafenib, Erbitux and binimetinib) both met its primary endpoint (Overall Survival) as compared to the control arm, and demonstrated a clinically meaningful benefit as compared to the doublet regimen, would provide support for approval of the triplet regimen and that determination regarding approval of the triplet regimen will consider the totality of the data. Array expects to have data from the safety lead-in portion of the study in 2017.

BEACON CRC was initiated based on results from a Phase 2 study of the combination of encorafenib and cetuximab, with or without alpelisib, a selective PI3K alpha inhibitor, in patients with advanced BRAF-mutant CRC, which were presented at the 2016 ASCO (Free ASCO Whitepaper) meeting. Data from this study suggest that mOS for these patients may exceed one year, which is more than double several historical published benchmarks for this population.

Colorectal cancer is the third most common cancer among men and women in the United States, with more than 134,000 new cases and nearly 50,000 deaths from the disease projected in 2016. In the United States, BRAF mutations occur in 8 to 15 percent of patients with colorectal cancer and represent a poor prognosis for these patients.

ARRY-382
Phase 1/2 dose escalation study advancing with ARRY-382, a colony-stimulating factor-1 receptor (CSF-1R) inhibitor, in combination with pembrolizumab, a PD-1 antibody, for the treatment of patients with advanced solid tumors

Array is advancing a Phase 1/2 dose escalation immuno-oncology trial of ARRY-382 in combination with pembrolizumab (Keytruda), a Programmed Cell Death Receptor 1 (PD-1) antibody, in patients with advanced solid tumors. ARRY-382 is a wholly-owned, highly selective and potent, small molecule inhibitor of CSF-1R kinase activity.

The study will enroll up to 18 patients with selected advanced solid tumors to determine the maximum tolerated dose and/or recommended Phase 2 dose of the combination. In addition, the safety profile, pharmacodynamic effects, and preliminary assessment of activity of the combination will be assessed. With appropriate results, Array has the option to advance the combination into expansion cohorts of patients with metastatic melanoma or advanced non-small cell lung cancer (NSCLC). Array expects to have data from the dose-escalation portion of the study in 2017.

Results from a prior Phase 1 study designed to assess the safety, pharmacokinetics and pharmacodynamics of ARRY-382 for treating patients with cancer have been presented and a dose and schedule that demonstrates target engagement based on multiple pharmacodynamic biomarkers was identified for further study. ARRY-382 is an investigational new drug and is not currently approved for any use in any country.

ARRY-797 (ARRY-371797)
Phase 2 trial in patients with LMNA A/C-related dilated cardiomyopathy (LMNA-related DCM)
In August 2016, Array announced results from a Phase 2 study of ARRY-797, an oral, selective p38 mitogen-activated protein kinase inhibitor, in patients with LMNA-related DCM, a rare, degenerative cardiovascular disease caused by mutations in the LMNA gene and characterized by poor prognosis. The trial results were presented at the European Society of Cardiology Congress 2016. The results demonstrated an absolute mean change from baseline of 69 meters on the six-minute walk test (6MWT) at week 12, the study’s primary endpoint (baseline 6MWT ranged from 246 to 412 meters). This magnitude of improvement exceeded historical benchmarks for 6MWT that have served as the basis for recent approvals of other drugs in other rare diseases. ARRY-797 administration also resulted in sustained improvements in N-terminal pro-brain natriuretic peptide (NT-proBNP), functional capacity and cardiac function through 48 weeks in LMNA-related DCM patients. Patients who rolled over to a continuing treatment protocol maintained improvements in the 6MWT and NT-proBNP levels through 72 weeks of treatment. Other secondary endpoints measured including echocardiographic measures of left and right ventricular function and patient-reported outcomes using the Kansas City Cardiomyopathy Questionnaire (KCCQ), and both mirrored the improvements seen with the 6MWT. ARRY-797 was well tolerated with most patients experiencing mild to moderate adverse events, including stomatitis, acne and upper respiratory tract infection.

Taken together, the data to date suggest a path forward for this program, and Array has met with regulators to discuss the design of a study that could be the basis for marketing approval. Array is evaluating different options to advance the ARRY-797 program, including advancing it on its own, partnering the program for further development and commercialization or creating a separate company based on this asset.

LMNA-related DCM is estimated to occur in about 6,000 – 10,000 patients in the US. The number of patients currently identified with a molecular diagnosis is likely to be less than this estimate because of underutilization of genetic testing. Patients with LMNA-related DCM typically begin experiencing symptoms in their twenties or thirties and by age 45, nearly 70 percent of patients have had a heart transplant, have experienced a major cardiac event or have died. By comparison, only 25 percent of DCM patients who do not have LMNA mutations experience similar events by age 45. Currently, there are no disease-specific treatments approved for LMNA-related DCM. ARRY-797 is an investigational new drug and is not currently approved for any use in any country.

FINANCIAL HIGHLIGHTS
Raised $132.25 million in public offering
Array completed an underwritten public offering of 21,160,000 shares of its common stock at a price of $6.25 per share on October 3, 2016. The total gross proceeds from the offering are $132.25 million, before underwriting discounts and commissions and offering expenses.

First Quarter of Fiscal 2017 Compared to Fourth Quarter of Fiscal 2016 (Sequential Quarters Comparison)

Revenue for the first quarter of fiscal 2017 was $39.3 million, compared to $43.2 million for the prior sequential quarter, mainly driven by reduced Novartis reimbursed activity.
Cost of partnered programs for the first quarter of fiscal 2017 was $8.8 million, compared to $5.4 million for the prior quarter. The increase in cost of partnered programs was primarily due to the initiation of the BEACON CRC trial.
Research and development expense was $46.6 million, compared to $49.5 million in the prior quarter. The slight decrease in research and development expense is primarily related to the ongoing transition of binimetinib and encorafenib trials from Novartis to Array.
Net loss for the first quarter was $28.6 million, or ($0.20) per share, and was $25.0 million, or ($0.17) per share in the prior quarter.
First Quarter of Fiscal 2017 Compared to First Quarter of Fiscal 2016 (Prior Year Comparison)

Revenue for the first quarter of fiscal 2017 increased $23.1 million compared to the same quarter of fiscal 2016. The increase was primarily due to reimbursement revenue from Novartis.
Cost of partnered programs increased $2.6 million compared to the first quarter of fiscal 2016. The increase was primarily due to the BEACON CRC trial.
Research and development expense increased $25.6 million, compared to the first quarter of fiscal 2016. The increase was due to binimetinib and encorafenib expenses as we transitioned activity from the "Novartis Agreements."
Net loss for the first quarter of fiscal 2017 was $28.6 million, or ($0.20) per share, and was $21.0 million, or ($0.15) per share, for the same quarter in fiscal 2016.

On November 1, 2016 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, reported financial results for the three and nine months ended September 30, 2016 and provided a summary of corporate highlights (Press release, Regulus, NOV 1, 2016, View Source [SID1234516322]).

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"Our focus in the third quarter included the advancement of the RG-012 development program and addressing the clinical hold for RG-101 while expanding the pre-clinical pipeline," said Paul Grint, M.D., President and Chief Executive Officer of Regulus. "We continue to be excited with the progress of our research programs, and look forward to sharing an update at our first R&D day on December 6th."

Financial Results

Revenue: Revenue was $0.2 million and $1.2 million for the three and nine months ended September 30, 2016, respectively, compared to $1.9 million and $9.9 million for the same periods in 2015. Revenue for the three and nine months ended September 30, 2016 and 2015 consisted of amortization of up-front payments from Regulus’ strategic alliances and collaborations. Revenue for the three and nine months ended September 30, 2015 included $0.9 million and $4.1 million, respectively, for research services under Regulus’ strategic alliances and collaborations. Preclinical milestones earned under Regulus’ strategic alliances and collaborations were $0.3 million and $3.2 million for the three and nine months ended September 30, 2015, respectively.

Research and Development (R&D) Expenses: R&D expenses were $14.6 million and $49.3 million for the three and nine months ended September 30, 2016, respectively, compared to $11.0 million and $43.6 million for the same periods in 2015. The increases in R&D expenses were primarily driven by the advancement of our clinical programs and increased investment in our preclinical pipeline.

General and Administrative (G&A) Expenses: G&A expenses were $4.8 million and $13.6 million for the three and nine months ended September 30, 2016, respectively, compared to $4.2 million and $13.7 million for the same periods in 2015.

Net Loss: Net loss was $19.5 million, or $0.37 per share, and $61.8 million, or $1.17 per share, for the three and nine months ended September 30, 2016, respectively, compared to a net loss of $13.0 million, or $0.25 per share, and $48.5 million, or $0.95 per share, for the same periods in 2015.

Cash Position: Cash, cash equivalents, and short-term investments were $91.7 million at September 30, 2016, compared with $108.0 million at June 30, 2016 and $115.3 million at December 31, 2015.

Recent Events

In October, Dr. Timothy Wright joined Regulus as its Chief R&D Officer.
In September, Regulus initiated the HERA study, an international randomized, double-blind, placebo-controlled, multi-center Phase 2 clinical trial designed to evaluate the safety, pharmacodynamics, pharmacokinetics, dose selection, and preliminary efficacy of weekly RG-012 injections in approximately 30 patients with Alport syndrome. In order to address study design comments from European regulators, a multiple-ascending dose (MAD) study in healthy volunteers will be implemented (4-week repeat dosing) prior to expanding to Alport patients. Regulus anticipates the MAD study will be completed in the first half of 2017. Based on predicted enrollment rates, Regulus anticipates interim results from HERA in the first half of 2018.
In July, as anticipated, Regulus received written communication from the U.S. Food and Drug Administration (FDA) outlining information required to resolve the clinical hold for its Investigational New Drug (IND) for RG-101, which was announced on June 27, 2016. Based on the completion of additional mechanistic pre-clinical studies, Regulus expects a response to its submission from the FDA in the first quarter of 2017.
Upcoming Events

On November 13, 2016, Regulus will present three posters at American Association for the Study of Liver Disease (AASLD) in Boston.
On November 15, 2016 at 4:30 pm Eastern Time, Regulus will present a corporate overview at the Stifel 2016 Healthcare Conference in New York.
On November 18 and 19, 2016, Regulus will present two posters at American Society of Nephrology (ASN) Kidney Week in Chicago.
On December 6, 2016, Regulus will host its first R&D Day.
On December 13, 2016, Regulus will participate in the 4th Annual Boston Healthcare Conference.
On December 14, 2016, Regulus will participate in the BMO Capital Markets Prescriptions for Success Healthcare Conference.

On November 1, 2016 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), reported financial results for the third quarter ended September 30, 2016 and provided an overview of key milestones for the company’s lead drug candidates (Filing, Q3, Lexicon Pharmaceuticals, 2016, NOV 1, 2016, View Source [SID1234516134]).

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"We achieved a key milestone in the third quarter with the release of positive top-line results from our first pivotal Phase 3 clinical trial of sotagliflozin in patients with type 1 diabetes, followed by favorable results in October from a Phase 2 study that supported our Phase 3 dose selection and demonstrated results on several important measures beyond A1C," said Lonnel Coats, Lexicon’s president and chief executive officer. "We are enthusiastic about the clinical results demonstrated by sotagliflozin to date, and we are looking forward to announcing the results from two additional clinical trials, including our second pivotal Phase 3 study, by the end of the year."

Pipeline Progress

Telotristat ethyl is the first investigational drug in clinical studies to target tryptophan hydroxylase (TPH), the rate-limiting enzyme involved in serotonin production. Excess production of serotonin within metastatic neuroendocrine tumor cells can lead to carcinoid syndrome, a condition characterized by serious consequences including frequent and debilitating diarrhea, facial flushing, abdominal pain, and heart valve damage.

In July, the European Medicines Agency accepted a marketing authorization application filed for telotristat ethyl by Ipsen, Lexicon’s collaborator for the commercialization of telotristat ethyl in Europe and other countries outside the U.S. and Japan. The current Prescription Drug User Fee Act ("PDUFA") target action date for Lexicon’s new drug application with the U.S. Food and Drug Administration for telotristat ethyl is February 28, 2017.

Sotagliflozin, which is being developed as a potential treatment for type 1 and type 2 diabetes, is a dual inhibitor of sodium-glucose transporters 1 and 2 (SGLT1 and SGLT2), each of which modulates glucose levels, and is the first investigational medicine developed to target both of these two proteins.

In September, Lexicon announced positive top-line results of its first pivotal Phase 3 clinical trial of sotagliflozin in patients with type 1 diabetes, on a background of optimized insulin (inTandem1). Lexicon is conducting a second pivotal Phase 3 clinical trial (inTandem2) from which top-line results are expected in December 2016. Lexicon is also completing a Phase 2 clinical trial in collaboration with JDRF in young adults with type 1 diabetes, from which results are expected prior to the end of the year. A third type 1 diabetes Phase 3 clinical trial, inTandem3, is underway globally and is studying approximately 1,400 patients treated with sotagliflozin 400mg once daily or placebo on a background of any insulin therapy, but without insulin optimization prior to randomization.

Lexicon entered into a collaboration and license agreement with Sanofi in November 2015 under which Lexicon granted Sanofi an exclusive, worldwide, royalty-bearing right and license to develop, manufacture and commercialize sotagliflozin. Lexicon is responsible for all clinical development activities relating to type 1 diabetes and retains an exclusive option to co-promote and have a significant role, in collaboration with Sanofi, in the commercialization of sotagliflozin for the treatment of type 1 diabetes in the United States. Sanofi is responsible for all clinical development and commercialization of sotagliflozin for the treatment of type 2 diabetes worldwide and is solely responsible for the commercialization of sotagliflozin for the treatment of type 1 diabetes outside the United States. Sanofi is expected to commence Phase 3 clinical trials for sotagliflozin in patients with type 2 diabetes this year.

Financial Highlights

Revenues: Lexicon’s revenues for the three months ended September 30, 2016 increased to $27.7 million from $0.6 million for the corresponding period in 2015, primarily due to revenues recognized from the collaboration and license agreement with Sanofi. For the nine months ended September 30, 2016, revenues increased to $60.3 million from $2.7 million for the corresponding period in 2015.

Research and Development Expenses: Research and development expenses for the three months ended September 30, 2016 increased 127 percent to $52.5 million from $23.1 million for the corresponding period in 2015, primarily due to increases in external clinical and nonclinical research and development costs. For the nine months ended September 30, 2016, research and development expenses increased 113 percent to $137.8 million from $64.7 million for the corresponding period in 2015.

Change in Fair Value of Symphony Icon Purchase Liability: In connection with the acquisition of Symphony Icon, Lexicon made an initial estimate of the fair value of the liability for the associated base and contingent payments. Changes in this liability, based on the development of the programs and the time until such payments are expected to be made, are recorded in Lexicon’s consolidated statements of operations. The change in fair value of the Symphony Icon purchase liability was $(2.1) million and $3.4 million for the three months ended September 30, 2016 and 2015, respectively, and was $(0.7) million and $5.1 million for the nine months ended September 30, 2016 and 2015, respectively.

General and Administrative Expenses: General and administrative expenses for the three months ended September 30, 2016 increased 128 percent to $12.3 million from $5.4 million for the corresponding period in 2015, primarily due to increased costs in preparation for commercialization of telotristat ethyl. For the nine months ended September 30, 2016, general and administrative expenses increased 67 percent to $29.1 million from $17.4 million for the corresponding period in 2015.

Impairment Loss on Buildings: In 2014, Lexicon began to market its buildings and land in The Woodlands, Texas for sale. Lexicon recognized non-cash impairment losses on its buildings of $2.3 million for the three and nine months ended September 30, 2015 as a result of writing down the buildings to the estimated net selling price.

Consolidated Net Loss: Net loss for the three months ended September 30, 2016 was $36.0 million, or $0.35 per share, compared to a net loss of $35.3 million, or $0.34 per share, in the corresponding period in 2015. Net loss for the nine months ended September 30, 2016 was $109.0 million, or $1.05 per share, compared to a net loss of $91.4 million, or $0.88 per share, in the corresponding period in 2015. For the three and nine months ended September 30, 2016, net loss included non-cash, stock-based compensation expense of $1.9 million and $5.7 million, respectively. For the three and nine months ended September 30, 2015, net loss included non-cash, stock-based compensation expense of $1.7 million and $5.4 million, respectively.

Cash and Investments: As of September 30, 2016, Lexicon had $395.6 million in cash and investments, as compared to $429.4 million as of June 30, 2016 and $521.4 million as of December 31, 2015.