On March 18, 2019 Celyad (Euronext Brussels and Paris, and Nasdaq: CYAD), a clinical-stage biopharmaceutical company focused on the development of CAR-T cell-based therapies, reported at R&D Day in New York the continued progress of the its pipeline of proprietary CAR-T therapies for the treatment of hematological malignancies and solid tumors, based on its short hairpin RNA (shRNA) platform (Press release, Celyad, MAR 18, 2019, View Source [SID1234534464]).

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 are very pleased with the recent progress of our preclinical CAR-T pipeline," noted Dr. Christian Homsy, CEO of Celyad. "Together with CYAD-101, our next generation allogenic shRNA platform should allow us to leapfrog the competition in the allogeneic CAR-T landscape. In addition, CYAD-02 has shown encouraging preclinical data of increased CAR-T cell expansion, persistence and anti-tumor efficacy. We continue to tap into our deep expertise and knowledge in cell therapy manufacturing and our all-in-one vector approach provides tremendous flexibility, versatility and efficiency in the design of novel, CAR-T therapies."

Corporate Updates

Lead asset CYAD-01 continues to advance in clinical trials for the treatment of patients with relapsed/refractory (r/r) acute myeloid leukemia (AML). The Company reported that additional dosing and schedule optimization are under evaluation in the THINK Phase 1 trial. In addition, the Company reported that interim data from the DEPLETHINK Phase 1 trial evaluating CYAD-01 following preconditioning chemotherapy shows the CAR-T cell therapy is well-tolerated at the initial dose levels following preconditioning chemotherapy. Future clinical updates from the Phase 1 THINK and DEPLETHINK trials are anticipated by mid-2019.
Company also highlighted its operational excellence for 2018 including a 94% manufacturing success rate and a twofold increase year-over-year in the number of patients treated.
shRNA Platform

In October 2018, Celyad announced it had entered into an exclusive agreement with Horizon Discovery Group for the use of its shRNA technology to generate a novel, next-generation, non-gene-edited allogeneic platform for CAR-T therapies. Horizon Discovery’s SMARTvector technology to express shRNA optimized by Celyad provides an alternate method to knockout the TCR complex in allogeneic CAR-T therapies compared to gene editing techniques as well as the specificity to target a broad range of proteins.
Utilization of the shRNA platform allowed the Company to develop the next generation of autologous, NKG2D-based CAR-T candidate, CYAD-02, and the novel, non-gene edited allogeneic CYAD-200 series of CAR-T candidates.
In addition, the shRNA platform complements the Company’s strong intellectual property of six U.S. patents related to allogeneic T-cell technology and producing TCR deficient cells expressing a CAR construct.
Autologous CAR-T candidate: CYAD-02

CYAD-02 incorporates shRNA technology to target NKG2D ligands MICA/MICB. In preclinical AML models, CYAD-02 shows an encouraging increase in in vitro proliferation and in vivo persistence and anti-tumor activity. The Company plans to generate additional preclinical proof-of-concept data for the program throughout 2019 and plans to submit an Investigational New Drug (IND) application for CYAD-02 in first half 2020.
Non-gene edited allogeneic CAR-T candidates: CYAD-200 series

Celyad utilizes two technologies based on non-gene edited approaches to modulate the T-cell receptor (TCR) complex to generate allogeneic CAR-T therapies. The first approach utilizes our TCR-inhibitor molecule (TIM) and is tailored to our NKG2D-based CAR-T clinical candidate CYAD-101. The second approach leverages shRNA technology exclusively licensed from Horizon Discovery to target the CD3ζ component to knockdown the expression of the TCR/CD3 complex on the surface of the T-cell.
In vivo data demonstrate that shRNA targeting of CD3ζ effectively protects against Graft-versus-Host Disease (GvHD) to a level equivalent to CRISPR-Cas9 based knock-out. Furthermore, results from preclinical tests show significant increase in persistence of allogeneic T cells using shRNA targeting when compared to gene editing technologies, such as CRISPR-Cas9.
Celyad announced plans to develop three disruptive first-in-class non-gene-edited allogenic CAR-T candidates leveraging the shRNA SMARTvector platform, including:
CYAD-211: B-cell maturation antigen (BCMA) targeting CAR-T therapy for the treatment of multiple myeloma, which is expected to enter the clinic by mid-2020.
CYAD-221: CD19 targeting CAR-T therapy for the treatment of B-cell malignancies, which is expected to enter the clinic by late 2020.
CYAD-231: Dual specific CAR-T targeting NKG2D and an undisclosed membrane protein, which is expected to enter the clinic by early 2021.
In addition, the company continues to investigate additional undisclosed targets using the shRNA platform to pair with CARs to develop, differentiated next-generation cell therapies for the treatment of both hematological malignancies and solid tumors.

On March 18, 2019 vTv Therapeutics Inc. (Nasdaq:VTVT) (the "Company") reported that it has entered into a definitive agreement with certain institutional investors for the purchase and sale in a registered direct offering (the "Registered Direct Offering") of 3,636,364 shares of the Company’s Class A common stock (the "Common Stock") at a price of $1.65 per share for aggregate gross proceeds of approximately $6.0 million (Press release, vTv Therapeutics, MAR 18, 2019, View Source;p=RssLanding&cat=news&id=2391591 [SID1234534415]). The offering is expected to close on or about March 20, 2019, subject to the satisfaction of customary closing conditions.

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!

H.C. Wainwright & Co. is acting as the exclusive placement agent.

The Company also entered into a letter agreement (the "Letter Agreement") with MacAndrews & Forbes Group LLC ("M&F Group"), the Company’s largest shareholder, pursuant to which M&F Group has committed to purchase, at the Company’s option and exercisable on demand during a one-year period after the date of the Letter Agreement, up to 5,454,545 unregistered shares of Common Stock at a fixed price of $1.65 per share for aggregate proceeds of approximately $9.0 million assuming the Company elects to sell the entire amount. M&F Group may also exercise a right to purchase those shares on the same terms three times during the same one-year period.

"This financing allows us to initiate two important trials to study therapeutics for underserved populations, one for Type 1 diabetics who rely primarily on insulin and a second for diabetics with Alzheimer’s," said Steve Holcombe, the chief executive officer of vTv Therapeutics. "We expect readouts from these trials in the first quarter of next year and fourth quarter of next year, respectively."

The Company intends to use the net proceeds from the Registered Direct Offering and the Letter Agreement to fund start-up activities for current and future clinical trials, in addition to ongoing business operations. The clinical trials include:

A phase 2 and phase 3 clinical trial under a single protocol designed to investigate the safety and efficacy of azeliragon in patients with mild Alzheimer’s disease and type 2 diabetes as evidenced by elevated HbA1c.
The part 2 confirmatory phase of the ongoing Simplici-T1 Study, a 12-week study to evaluate TTP399 as an add-on to insulin therapy for type 1 diabetics. This trial is being conducted in partnership with JDRF.
The Common Stock in the Registered Direct Offering is being offered and sold by the Company pursuant to a shelf registration statement on Form S-3 (Registration No. 333-223269) that was previously filed with the Securities and Exchange Commission ("SEC") and declared effective on March 19, 2018. The Registered Direct Offering of shares of Common Stock will be made only by means of a prospectus supplement that forms a part of the registration statement. A prospectus supplement and the accompanying prospectus relating to the Registered Direct Offering will be filed with the SEC. Copies of the prospectus supplement and the accompanying prospectus relating to the Registered Direct Offering may be obtained, when available, from H.C. Wainwright & Co., LLC, 430 Park Avenue 3rd Floor, New York, NY 10022, or by calling (646) 975-6996 or by emailing [email protected] or at the SEC’s website at View Source

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

On March 18, 2019 IntelGenx Technologies Corp. (TSX VENTURE:IGX)(OTCQX:IGXT) reported that it will release its fourth quarter and full year 2018 financial results after market close on March 22, 2019 (Press release, IntelGenx, MAR 18, 2019, View Source;Conference-Call-to-Follow-on-March-25-2019/default.aspx [SID1234534437]).

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!

An associated conference call will be hosted by Dr. Horst G. Zerbe, President and Chief Executive Officer, and Mr. Andre Godin, Executive Vice-President and Chief Financial Officer, on March 25, 2019 to discuss the results and provide a business update. Details of the conference call and webcast are below:

Date: Monday, March 25, 2019

Time: 8:00 a.m. EDT

Conference dial-in: (833) 231-8269

International dial-in: (647) 689-4114

Conference ID: 1297963

Webcast Registration: Click here

Following the live call, a replay will be available on the Company’s website, www.intelgenx.com, under "Investor Relations".

On March 18, 2019 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat age-related diseases, reported its financial results for the fourth quarter ended December 31, 2018 (Press release, CohBar, MAR 18, 2019, View Source [SID1234534465]).

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 focus on getting CB4211 back into the clinic as soon as possible, and have engaged in direct discussions this month with the FDA about our revised clinical plan," said Philippe Calais, CohBar’s interim chief executive officer. "At the same time, our increased investment in research is enabling significant progress in evaluating our new peptides in an expanded range of disease models, and uncovering mechanisms that open new potential opportunities for additional therapeutic targets. We also continue to make progress optimizing novel peptides as potential therapeutics for type 2 diabetes and cancer. These developments on multiple fronts reinforce our belief in the therapeutic potential of our portfolio of mitochondrial peptides to treat multiple age-related diseases."

Fourth Quarter 2018 and Recent Clinical, Research and Business Highlights:

CB4211 Clinical Study Update. The company retained Dr. James Leyden, Professor Emeritus of Dermatology at University of Pennsylvania, a well-respected expert and advisor to U.S. and European regulatory agencies, who reviewed the clinical data related to the mild but persistent indurations observed in the company’s temporarily-suspended Phase 1a/1b clinical trial. Dr. Leyden reported his conclusions that there were no significant safety issues and participated in the company’s call with the FDA in early March. The company is submitting additional information to the FDA, with the goal of resuming clinical activities as soon as possible.

CohBar Pipeline Update. During the fourth quarter and more recently, the company continued to advance its optimization and evaluation of novel analogs of its previously discovered peptides, and the identification of potential mechanisms and disease targets. In the area of type 2 diabetes research, the company recently discovered the interaction between a family of novel peptide analogs with effects on glucose tolerance in animals and a key cell surface receptor. This receptor plays an important role in a number of age-related diseases. CohBar submitted an abstract on this discovery for presentation at a major scientific meeting scheduled for later this year.

Appointed Dr. Philippe Calais as Interim CEO. Dr. Calais became interim CEO in December, bringing to the company more than 30 years of large and small-cap biopharmaceutical experience in product development and commercialization, partnerships, collaborations and financings. He most recently served as Chief Executive Officer of Isarna Therapeutics B.V., after having led several clinical stage biopharmaceutical companies in Canada and in Europe, and Univalor LTD, a large technology transfer organization in Canada. Earlier in his career, he served in multiple roles at F. Hoffmann-La Roche.

Expanded the Company’s Board of Directors. Dr. Phyllis Gardner joined the CohBar Board in February 2019, adding broad and deep expertise in academia, medicine, pharmacology, drug delivery, biotechnology and corporate investing and governance to the CohBar board. Dr. Gardner’s background includes roles as a senior scientist at ALZA Corporation, a partner at Essex Woodland Health Ventures and over 30 years as a distinguished professor of medicine at Stanford University School of Medicine.

Investment Community Outreach. During the fourth quarter, CohBar presented an overview of the company and its clinical development program at The BIO Investor Forum. The company also met with investors, bankers and analysts during the JP Morgan Healthcare conference in January 2019, and presented at the BIO CEO and Investor Conference in February. At BIO CEO, Dr. Calais was featured on a panel entitled "Attacking Biological Mechanisms of Aging to Extend Healthspan."

Media Coverage. An article entitled "CohBar Develops Targeted Mitochondria Based Therapeutics for Metabolic Disorders that Cause Age-Related Diseases," and an interview with Dr. Calais, were published in the February 15, 2019 issue of Beikoku Seiyaku Gyokai Shuho. Beikoku Seiyaku Gyokai Shuho is a weekly report covering the U.S. pharmaceutical market. Its subscribers include some of Japan’s largest pharmaceutical and other life sciences companies.
During the fourth quarter and more recently, CohBar’s founders, Dr. Pinchas Cohen and Dr. Nir Barzilai, continued to be recognized as international leaders in the study of aging, age-related diseases and mitochondrial science.

Dr. Cohen was featured as a keynote speaker at "The Barshop Symposium on Exercise Regulation of Biological Aging" in San Antonio, Texas and at "The Gerontological Society of America" in Boston, Massachusetts. Dr. Cohen also co-authored three studies related to mitochondrial peptides published during the quarter and recently: "Characterizing the Protective Effects of SHLP-2, a Mitochondrial Derived Peptide, in Macular Degeneration," in Nature Scientific Reports (October 2018), "Humanin is a Novel Regulator of Hedgehog Signaling and prevents Glucocorticord-induced Bone Growth Impairment," in FASEB Journal (January 2019), and "MOTS-c, an Equal Opportunity Insulin Sensitizer," Journal of Molecular Medicine (February 2019).

Dr. Barzilai was awarded the prestigious international Fondation IPSEN Longevity Prize during the 22nd Geriatric Society of America meeting held on November 17, 2018 in Boston, Massachusetts. Dr. Barzilai also delivered keynote lectures at the "Happy Aging Day," in Brussels, Belgium, "The Cristofalo Annual Lectureship," at The University of Pennsylvania, "Endokrinologisches Symposium," in Berlin, Germany, "The Longevity Therapeutics Summit," in San Francisco, California, "The Science and Business of Aging," Boston, Massachusetts, and "The Taft Lectureship at the Nutrition Center," also in Boston, Massachusetts. Dr. Barzilai also authored four published articles: "Effects of FOXO3 Polymorphisms on Survival to Extreme Longevity in Four Centenarian Studies," in The Journals of Gerontology, "Sarcosine is Uniquely Modulated by Aging and Dietary Restriction in Rodents and Humans," in Cell Reports, "PopCluster: an Algorithm to Identify Genetic Variants with Ethnicity-dependent Effects," in Bioinformatics, and "Models and Studies of Aging: Executive Summary of a Report from the U13 Conference Series," in the Journal of American Geriatrics Society.
Fourth Quarter 2018 Financial Highlights

Cash and Investments. CohBar had cash and investments of $22,182,768 on December 31, 2018, compared to $8,452,459 on December 31, 2017.

R&D Expenses. Research and development expenses were $2,085,662 in the three months ended December 31, 2018, compared to $1,791,212 in the prior year quarter. The increase was primarily due to the transition of our lead MBT Candidate from preclinical to clinical stage, resulting in an increase in clinical activity costs incurred in the current year quarter partially offset by a decrease in preclinical activity costs incurred in the prior year quarter.

G&A Expenses. General and administrative expenses were $2,009,604 for the three months ended December 31, 2018, compared to $1,059,565 in the prior year quarter. The increase in general and administrative expenses was primarily due to severance and non-cash stock-based compensation costs related to the termination of our former CEO, recruiting costs, and increased director fees.

Net Loss. For the three months ended December 31, 2018, net loss was $4,190,125, or $0.10 per basic and diluted share, compared to a net loss of $2,833,396, or $0.07 per basic and diluted share, for the three months ended December 31, 2017.
Fourth Quarter Investor Call and Slide Presentation:

Date: March 18, 2019
Time: 5:00 p.m. ET (2:00 p.m. PT)

Conference Audio

Dial-in U.S. and Canada: (800) 289-0438
Dial-in International: (323) 794-2423
Conference ID No.: 8144256
Slide Presentation

Go to www.webex.com, click on the ‘Join’ button and enter Meeting Number 923 629 679 and Password CWBR, or
Go to www.cohbar.com and click on Q4 Shareholder Presentation at top of homepage.
We kindly request that you please call into the conference audio and log into WebEx approximately 10 minutes prior to the start time so that we can begin promptly.

An audio replay of the call will be available beginning at 8:00 p.m. Eastern Time on March 18, 2019, through 11:59 p.m. Eastern Time on April 8, 2019. To access the recording please dial (844) 512-2921 in the U.S. and Canada, or (412) 317-6671 internationally, and reference Conference ID# 8144256. The audio replay along with the slide presentation will also be available on the homepage at www.cohbar.com from March 18, 2019 through April 8, 2019.

About CB4211

CohBar’s lead program is based on CB4211, a first-in-class mitochondria based therapeutic (MBT) that has demonstrated significant therapeutic potential in preclinical models of nonalcoholic steatohepatitis (NASH) and obesity. CB4211 is a novel and improved analog of MOTS-c, a naturally occurring mitochondrial-derived peptide (MDP) which was discovered in 2012 by CohBar founder Dr. Pinchas Cohen and his academic collaborators and has been shown to play a significant role in the regulation of metabolism. In July 2018, CB4211 entered a Phase 1a/1b clinical trial which includes a potential activity readout relevant to NASH and obesity. In November 2018, the company announced the temporary suspension of the trial to address mild injection site reactions that were unexpectedly persistent. NASH has been estimated to affect as many as 12% of adults in the U.S., and there is currently no approved treatment for the disease.

On March 18, 2019 Puma Biotechnology, Inc. (Nasdaq: PBYI), a biopharmaceutical company, reported that updated results from the cervical cancer cohort of SUMMIT, an ongoing Phase II basket trial examining the efficacy of neratinib in HER2-mutated cancers, were reported at the Society of Gynecologic Oncology (SGO) 2019 Annual Meeting in Honolulu, Hawaii (Press release, Puma Biotechnology, MAR 18, 2019, View Source [SID1234534416]). "Neratinib in patients with HER2-mutant, metastatic cervical cancer: findings from the phase II SUMMIT ‘basket’ trial," was presented during the Scientific Plenary Session by Anishka D’Souza, M.D., Assistant Professor of Clinical Medicine, Keck School of Medicine of University of Southern California (USC). SGO selected this abstract as the recipient of the 2019 SGO Presidential Award. Slides from the presentation are available on the Puma Biotechnology website.

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 Phase II SUMMIT ‘basket’ trial is an open-label, multicenter, multinational study to evaluate the safety and efficacy of neratinib administered daily to patients who have solid tumors with activating, somatic HER2 mutations. The cervical cancer cohort was comprised of 11 patients with advanced and/or metastatic disease treated with neratinib monotherapy. Patients received a median of 2 (range 1–4) prior regimens in the recurrent or metastatic setting before entering this trial. Six patients (54.5%) had been previously treated with bevacizumab prior to entering the study; 7 patients (63.6%) had received prior surgery; and 9 patients (81.8%) received prior radiation therapy. The objective response rate was 27.3% (95% CI: 6.0%–61.0%). The clinical benefit rate was 54.5% (95% CI: 23.4%–83.3%) and included 3 patients with confirmed partial responses and 3 patients with stable disease that lasted greater than 16 weeks. The median progression free survival was 7.0 months (95% CI: 0.7–20.1 months).

The safety profile observed in neratinib-treated cervical cancer patients in SUMMIT was consistent with that reported for HER2-amplified metastatic breast cancer. The most frequently observed adverse event was diarrhea, any grade (n=9, 81.8%) including 1 (9%) grade 3 diarrhea event. The duration of grade 3 diarrhea was 1 day. None of the diarrhea events resulted in dose reduction, dose discontinuation or hospitalization.

"Somatic HER2 mutations represent a distinct class of oncogenic driver mutations that appear to be clinically actionable for metastatic cervical cancers. Treatment with neratinib led to durable responses and disease control in metastatic patients with HER2-mutant cervical cancer," said Dr. D’Souza, who practices oncology at the USC Norris Comprehensive Cancer Center.

Alan H. Auerbach, CEO and President of Puma Biotechnology, added, "We are very pleased with the activity seen with neratinib in this cohort of patients with HER2-mutated cervical cancer. We look forward to the further development of neratinib in this patient population."