On November 15, 2017 Neuralstem, Inc. (NASDAQ:CUR), a biopharmaceutical company focused on the development of nervous system therapies based on its neural stem cell technology, reported its financial results for the three and nine month periods ended September 30, 2017 (Press release, Neuralstem, NOV 15, 2017, View Source [SID1234522096]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continue to evaluate the full Phase 2 data set for NSI-189 to determine the optimal development path in major depressive disorder and for other conditions, including Angelman’s Syndrome following highly encouraging preclinical data in that setting. We expect to provide a detailed update on our corporate strategy, including development and regulatory plans, after our post-phase 2 meeting with the FDA in the first half of 2018," commented Rich Daly, Chairman and CEO.

"Our recent financing has further extended the company’s cash runway to sufficiently support continued research on NSI-189 and to support operations. We are encouraged by the emerging clinical profile of NSI-189 and look forward to presenting additional clinical data at the upcoming American College of Neuropsychopharmacology in December."

Recent Corporate & Clinical Highlights & Milestones

On November 6, 2017, we strengthened our clinical research team with the appointment of David Recker, MD, as Chief Medical Officer. Dr. Recker has more than 20 years of experience in drug development in multiple therapeutic areas including CNS and cell therapy and has been involved in numerous aspects of clinical strategy development, including product registration and marketing support, clinical trial development and execution, data interpretation, key opinion leader development and support.

On September 18, 2017, Cristina Csimma, Pharm.D, MHP joined the board of directors. Ms. Csimma brings extensive senior leadership experience in the biopharmaceutical industry, including expertise in drug development and regulatory and commercial processes.

On July 25, 2017, the Company announced top-line results from the exploratory Phase 2 clinical trial examining the efficacy of NSI-189 at 40 mg once daily (QD) and 40 mg twice daily (BID) compared to placebo for the treatment of major depressive disorder (MDD). The study, which utilized the two-staged sequential parallel comparison design (SPCD), did not meet its primary efficacy endpoint of a statistically significant reduction in depression symptoms on the Montgomery-Asberg Depression Rating Scale (MADRS). However, as reported in our topline results, the 40 mg QD dose was directionally positive on the MADRS and met statistical significance on several key secondary efficacy endpoints.

The company plans to present the results of the analysis of the secondary endpoints from the Phase 2 clinical trial of NSI-189 in MDD at a scientific meeting in the fourth quarter of this year.

Neuralstem plans to meet with the U.S. Food and Drug Administration in the first half of 2018 to discuss the clinical development path for NSI-189.
Neuralstem intends to submit data on NSI-566, its stem cell therapy product candidate, to FDA and to request Regenerative Medicine Advanced Technology, or RMAT, designation. The RMAT designation, intended to expedite the approval of safe and effective cell therapies, was created by the U.S. Congress as part of the recently-enacted 21st Century Cures Act. Neuralstem is evaluating NSI-566 in three indications: stroke, chronic spinal cord injury (cSCI), and Amyotrophic Lateral Sclerosis (ALS).
On September 5, 2017, the Company was awarded two additional patents by the United States Patent and Trademark Office (USPTO). These patents broadly protect methods for using neural stem cells to treat neurodegenerative disorders, a key component of the Company’s platform. The first new patent, U.S. Patent No. 9,744,194, covers methods of treating neurodegenerative disorders through transplantation of neural stem cells. The second new patent, U.S. Patent No. 9,750,769, covers neural stem cells engineered to express IGF-1, a neurotrophic molecule with broad therapeutic potential in the treatment of neurodegenerative disorders.
Financial Results for the Third Quarter Ended September 30, 2017

Cash Position and Liquidity: At September 30, 2017, cash and investments was $14.1 million as compared to $11.4 million at June 30, 2017. The $2.6 million increase is due to proceeds of $5.4 million, net, from a public offering of common stock and warrants. On August 1, 2017, the Company closed a public offering of 3,000,000 shares of common stock and 2,250,000 common stock purchase warrants at a public purchase price of $2.00 per share and accompanying warrant. Gross proceeds were $6.0 million and approximately $5.4 million, net.

Operating Loss: Operating loss for the quarter ended September 30, 2017 was $2.6 million compared to a loss of $4.9 million for the same period of 2016. The decrease in operating loss for the third quarter 2017 was primarily due to a decrease in clinical trial expenses related to the completion of the Phase 2 clinical trial of NSI-189 in MDD coupled with ongoing corporate restructuring and cost reduction efforts.

Operating loss for the nine months ended September 30, 2017 was $11.0 million compared to a loss of $15.0 million for the same period of 2016. The decrease in operating loss for the nine-month period was primarily due to ongoing corporate restructuring and cost reduction efforts partially offset by increases in clinical trial expenses as the Company completed the Phase 2 clinical trial of NSI-189.

Net Loss: Net loss for the quarter ended September 30, 2017 was $0.1 million, or $0.01 per share (basic) compared to a loss of $5.2 million, or $0.59 per share (basic), on a split adjusted basis for the same period of 2016. The decrease in net loss was primarily due to a decrease in operating expenses along with a $2.7 million non-cash, gain resulting from the fair value adjustment of outstanding liability classified stock purchase warrants.

Net loss for the nine months ended September 30, 2017 was $12.4 million, or $1.00 per share (basic), compared to a loss of $15.7 million, or $1.96 per share (basic), on a split adjusted basis for the same period of 2016. The decrease in net loss was primarily due to a decrease in operating expenses and interest expense due to the maturity of long-term debt in April 2017.

R&D Expenses: The $2.2 million, or 61% decrease, in research and development expenses for the quarter ended September 30, 2017, as compared to the comparable period of 2016, was primarily attributable to a $1.7 million decrease in clinical trial expenses due to the completion of NSI-189 Phase 2 clinical trial, a $0.3 million decrease in personnel, facility and other expenses related to ongoing corporate restructuring and cost reduction efforts and a $0.2 million decrease in non-cash stock based compensation expense.

The $2.3 million, or 25% decrease, in research and development expenses for the nine months ended September 30, 2017, as compared to the comparable period of 2016, was primarily attributable to a $2.2 million decrease in personnel, facility and other expenses related to ongoing corporate restructuring and cost reduction efforts and a $0.4 million decrease in non-cash stock based compensation expense partially offset by a $0.3 million increase in clinical trial expenses related to the completion of the Phase 2 clinical trial of NSI-189.

G&A Expenses: The $0.1 million, or 9% decrease, in general and administrative expenses for the quarter ended September 30, 2017, as compared to the comparable period of 2016, was primarily attributable to a decrease in cash based board of directors fees.

The $1.7 million, or 29% decrease, in general and administrative expenses for the nine months ended September 30, 2017 as compared to the comparable period of 2016 was primarily attributable to a $1.0 million decrease in non-cash stock based compensation expense coupled with a $0.8 million decrease in personnel related expense as a result of headcount reductions.

On November 15, 2017 ONCODESIGN (Paris:ALONC) (ALONC – FR0011766229), a biopharmaceutical group specialized in , precision medicine, reported that it has obtained positive results opening the way for the ALK1 kinase inhibitor discovery program to move on to the lead optimization phase (Press release, Oncodesign, NOV 15, 2017, View Source [SID1234522101]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

ALK1 is a kinase involved in angiogenesis. Tumoral angiogenesis is the mechanism by which new blood vessels form and infiltrate tumors to provide nutrients and oxygen and to dispose of the tumor’s cellular waste products. Inhibiting this mechanism is a promising line of research in the quest for new treatments for most types of cancer.

The ALK1 program has produced positive cellular results in a mechanistic model as part of the probe to lead phase. Oncodesign has thus decided to advance it to the lead optimization phase and commence an exhaustive series of in vivo biological tests, while conducting medicinal chemistry optimization of the inhibitor molecules identified. A medicinal chemistry team dedicated to the project will be set up to implement this decision.

"At the end of the Lead Optimization phase a drug candidate could be selected for preclinical trials and then clinical development", said Jan Hoflack, Oncodesign’s Chief Scientific and Operating Officer. "Today, our preclinical portfolio contains no fewer than 12 programs, and ALK1 is joining our most advanced programs. Our goal is to develop a best-in-class drug from this program with the potential to complement other anti-angiogenic approaches, currently a market worth over $10 billion. The addition of the drug discovery expertise of the François Hyafil research center in Paris-Saclay has enabled us to accelerate our most promising programs significantly. ALK1 is the first example of a project successfully moving on to a major new stage in its development by harnessing this new expertise."

After exploring molecules’ therapeutic potential in the probe qualification and probe orientation stages, molecules move on to the probe to lead phase. Molecules then undergo a further selection stage after medicinal chemistry optimization of their structure, and the programs are prioritized according to their activity in relevant cell models and their potential to become a drug.

The lead optimization phase aims to identify a drug candidate, a molecule meeting a large number of very exacting criteria to determine its suitability as a future drug. The selection of a drug candidate takes place at the end of the drug discovery phase, and the regulatory development phases then begin. Lead optimization can take up to 36 months, and the success rate is typically around 50%.

About kinases and Nanocyclix technology:

Kinases are a family of enzymes that play a key role in regulating most cell functions, such as proliferation, cell cycle progression, metabolism, survival/apoptosis, repair of damaged DNA, motility and response to the microenvironment.
Using its Nanocyclix technology module, Oncodesign identifies macrocyclic molecules capable of inhibiting both known and unexplored kinases in a powerful and targeted manner. A large variety of kinase inhibitors are thus explored continuously, and only the most promising inhibitor/targeted kinase combinations are selected for more in-depth investigations.
Oncodesign has built a project portfolio with tremendous potential to treat diseases with very substantial unmet medical needs. This portfolio contains both molecules already at an advanced stage of clinical development (a PET tracer for a specific type of lung cancer) and molecules at an earlier stage of development.

On November 15, 2017 Arvinas LLC, a private biotechnology company creating a new class of drugs based on protein degradation, reported it has expanded its ongoing license agreement with Genentech, a member of the Roche Group, for the development of new therapeutics using Arvinas’ novel PROTAC technology (Press release, Arvinas, NOV 15, 2017, View Source [SID1234558785]). The multi-year strategic license agreement, initiated in October 2015, will encompass additional disease targets and expand the collaboration.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Under the revised terms of the agreement, Arvinas is eligible to receive development and commercialization milestone payments in excess of $650 million based on achievement of certain predetermined milestones. In addition, Arvinas is eligible to receive tiered-royalties on sales of products resulting from the license agreement. Full financial terms have not been disclosed.

"Genentech’s decision to expand our original agreement to include additional disease targets shows the promise seen in our first two years together and further supports our targeted protein degradation platform as a novel drug modality to treat a broad array of diseases," said John Houston, Ph.D., President and Chief Executive Officer of Arvinas. "This expansion also supports our initial decision to work with Genentech in 2015 and we look forward to this growing collaboration."

The PROTAC Platform offers potential improvements over traditional small molecule inhibitors using the ubiquitin and proteasome system within a cell to degrade disease causing proteins. By removing target proteins directly rather than inhibiting them, PROTACs can provide multiple advantages over small molecule inhibitors, which can require high systemic exposure to achieve sufficient inhibition, often resulting in toxic side effects and eventual drug resistance.

On November 15, 2017 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that it will webcast a management presentation at the 29th Annual Piper Jaffray Healthcare Conference at 4:00 p.m. ET on Tuesday, November 28, 2017 (Press release, Regeneron, NOV 15, 2017, View Source [SID1234522097]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The session may be accessed through the Company’s web site, www.regeneron.com, on the ‘Events and Presentations’ page. An archived version of the presentation will be available for 30 days.

On November 15, 2017 Pharma reported the initiation of a Phase 1 clinical trial for its novel immune-modulator, PIN-2 (Press release, PIN Pharma, NOV 15, 2017, View Source [SID1234522102]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This first-in-human, proof-of-concept study is being conducted in oncology patients with solid tumors by PIN Pharma’s wholly owned Australian subsidiary, PIN Pharma Pty Ltd. The study is designed to show changes in the human immune system utilizing specific biomarkers that will both demonstrate and support PIN-2’s ability to link the innate and adaptive immune systems via antigen presenting cell activation (monocyte-derived dendritic cells) resulting in the generation of endogenous killer T cell immunity (CD8 cells).

"This study is supported by physiological and immunological data generated in our preclinical studies as well as in silico. We are confident that the study will confirm PIN-2’s mechanism of action and validate our preclinical models," said Colin Bier, President and CEO. "We anticipate having interim data by the upcoming JP Morgan conference this January, and are already seeing interest from pharma and venture funds to discuss the potential of this next generation immune-oncology therapy."

With the recent issues arising out of the current generation of immune-oncology compounds both in the clinic and on the market, PIN-2 has the potential to improve response rates that will allow better safety and tolerability either alone or in combination with existing treatments by enhancing innate immunity.