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]).

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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.

On November 7, 2017 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers, reported that the company will be participating in the following upcoming investor conferences (Press release, Loxo Oncology, NOV 7, 2017, View Source [SID1234521631]):

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Stifel 2017 Healthcare Conference on November 14, 2017, in New York City. At 2:45 p.m. ET Joshua H. Bilenker, M.D., chief executive officer, will present a corporate overview.
Evercore ISI Biopharma Catalyst/Deep Dive Conference on November 30, 2017, in Boston. At 4:35 p.m. ET Joshua H. Bilenker, M.D., chief executive officer, and Jacob S. Van Naarden, chief business officer, will be participating in a fireside chat.
Live webcasts of the presentation and fireside chat will be available at View Source A replay of each webcast will be available on the company’s website for 30 days.

On November 7, 2017 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the US Food and Drug Administration (FDA) approved the supplemental New Drug Application (sNDA) for Alecensa (alectinib) for the treatment of people with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) as detected by an FDA-approved test (Press release, Hoffmann-La Roche, NOV 7, 2017, View Source [SID1234521630]). The approval is based on results from the phase III ALEX study, which showed Alecensa significantly reduced the risk of disease worsening or death (progression-free survival, PFS) by 47% (HR=0.53, 95% CI: 0.38, 0.73, p<0.0001) compared to crizotinib as assessed by independent review committee (IRC). Median PFS was 25.7 months (95% CI: 19.9, not estimable) for people who received Alecensa compared with 10.4 months (95% CI: 7.7, 14.6) for people who received crizotinib. The safety profile of Alecensa was consistent with that observed in previous studies. The study also showed that Alecensa significantly reduced the risk of the cancer spreading to or growing in the brain or central nervous system (CNS) compared to crizotinib by 84% (HR=0.16, 95% CI: 0.10, 0.28, p<0.0001). This was based on a time to CNS progression analysis in which there was a lower risk of progression in the CNS as the first site of disease progression for people who received Alecensa (12%) compared to people who received crizotinib (45%).

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"Our goal is to develop medicines that have the potential to significantly improve upon the standard of care," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "In our pivotal study, Alecensa significantly extended the time that people lived without their disease worsening compared to crizotinib and also showed a marked reduction in the risk of their cancer spreading to the brain."

Alecensa received Breakthrough Therapy Designation from the FDA in September 2016 for the treatment of adults with advanced ALK-positive NSCLC who have not received prior treatment with an ALK inhibitor. Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat serious or life-threatening diseases and to help ensure people have access to them through FDA approval as soon as possible. Results from the phase III ALEX study were simultaneously presented at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and published in The New England Journal of Medicine. Subsequently, Alecensa was recommended in the National Comprehensive Cancer Network (NCCN) guidelines as a treatment option for first-line ALK-positive metastatic NSCLC (Category 1, Preferred).

In addition to today’s approval, the FDA also converted Alecensa’s initial accelerated approval in December 2015 for the treatment of people with ALK-positive, metastatic NSCLC who have progressed on or are intolerant to crizotinib (second-line) to a full approval.

About the ALEX study1
ALEX (NCT02075840/B028984) is an open-label, randomised, active-controlled, multicentre, phase III study evaluating the efficacy and safety of Alecensa versus crizotinib in people with ALK-positive NSCLC who had not received prior systemic therapy for metastatic disease and whose tumours were characterised as ALK-positive by the VENTANA ALK (D5F3) CDx Assay, a immunohistochemistry (IHC) test developed by Roche Tissue Diagnostics. People were randomised (1:1) to receive either Alecensa or crizotinib. The major efficacy outcome measure of the ALEX study is PFS according to RECIST v1.1 as determined by investigator assessment. Additional efficacy outcome measures include: PFS as determined by IRC, time to CNS progression by IRC based on RECIST v1.1, objective response rate (ORR) and duration of response (DOR), and overall survival (OS). Additional exploratory outcome measures were CNS-ORR and CNS-DOR by IRC in people with measurable CNS metastases at baseline. The multicentre study was conducted in 303 people across 161 sites in 31 countries. OS data are currently considered immature with only about a quarter of events being reported.

Grade ≥ 3 adverse reactions were reported for 41% of patients treated with Alecensa. The most common Grade 3-4 adverse reactions (≥3%) were evidence of kidney dysfunction (increased creatinine; 4.1%), evidence of liver dysfunction (hyperbilirubinemia; 5%), low levels of sodium (hyponatremia; 6%), increased liver enzymes (aspartate transaminase; 6%, and alanine transaminase; 6%), and decreased red blood cells (anaemia; 7%). Serious adverse reactions reported in ≥2% of patients treated with Alecensa were lung infection (pneumonia; 4.6%) and renal impairment (3.9%).

About Alecensa
Alecensa (RG7853/AF-802/RO5424802/CH5424802) is a highly selective, CNS active, oral medicine created at Chugai Kamakura Research Laboratories and is being developed for people with NSCLC whose tumours are identified as ALK-positive. ALK-positive NSCLC is often found in younger people who have a light or non-smoking history.1 It is almost always found in people with a specific type of NSCLC called adenocarcinoma.3
Alecensa is currently approved in the United States, Europe, Kuwait, Israel, Hong Kong, Canada, South Korea, Switzerland, India, Australia, Singapore, Taiwan, Liechtenstein, Thailand, United Arab Emirates, Saudi Arabia, Argentina and Turkey for the treatment of people with advanced (metastatic) ALK-positive NSCLC whose disease has worsened after, or who could not tolerate treatment with, crizotinib and in Japan for people with ALK-positive NSCLC.

About Roche in lung cancer
Lung cancer is a major area of focus and investment for Roche, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have four approved medicines to treat certain kinds of lung cancer and more than ten medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.

Aclaris Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Aclaris Therapeutics, 2017, NOV 7, 2017, View Source [SID1234521657]).

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Acorda Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Acorda Therapeutics, 2017, NOV 7, 2017, View Source [SID1234521699]).

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