On February 18, 2020 ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company applying a precision medicine approach to developing genetically-targeted therapies for cardiovascular diseases, reported financial results for the year ended December 31, 2019 and provided a corporate update (Press release, Arca biopharma, FEB 18, 2020, View Source [SID1234554421]).

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Dr. Michael Bristow, ARCA’s President and Chief Executive Officer, commented, "During 2019, we continued to advance development of Gencaro as a potential genetically-targeted treatment for heart failure patients with atrial fibrillation. In published or submitted for presentation/publication material, we have identified important new effectiveness evidence from the GENETIC-AF trial that have enhanced our understanding of the clinical profile of Gencaro. These data have allowed us to broaden the design of the single, pivotal Phase 3 clinical trial that was developed after consultation with the U.S. Food and Drug Administration via a Special Protocol Assessment agreement. There are currently no FDA approved drug therapies indicated for the treatment of atrial fibrillation in heart failure patients with left ventricular ejection fraction values greater than 40%. Based on our clinical data to date, we believe Gencaro has the potential to help address this substantial unmet medical need. We look forward to further evaluating the pharmacogenetic benefits of Gencaro for patients with our targeted genetic profile in PRECISION-AF, the Phase 3 clinical trial, which, subject to obtaining additional financing, we plan to initiate in the fourth quarter of 2020."

Pipeline Update – Second Half Review

GencaroTM (bucindolol hydrochloride) – a pharmacologically unique beta-blocker and mild vasodilator being developed as a potential genetically-targeted treatment for atrial fibrillation (AF) in patients with heart failure (HF).

The U.S. Food and Drug Administration (FDA) issued a Special Protocol Assessment (SPA) agreement for a single Phase 3 clinical trial (PRECISION-AF) to examine Gencaro as a genetically-targeted therapy for the prevention of AF recurrence in certain heart failure patients.
PRECISION-AF is designed as a double-blind, active-controlled, multicenter, international, adaptive study comparing Gencaro with TOPROL-XL (metoprolol succinate) for the prevention of AF recurrence or all-cause mortality in approximately 400 heart failure patients who have left ventricular injection fraction (LVEF) values ≥ 40% and ≤ 55% and the genotype which ARCA believes responds best to Gencaro (ADRB1 Arg389Arg).
Clinical data from the GENETIC-AF Phase 2B clinical trial was published in the Journal of the American College of Cardiology: Heart Failure in May 2019 and was presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting in September 2019. These data indicate that the response to Gencaro may be greater in HF patients with less severe left ventricular dysfunction, a patient population with no FDA approved drug therapeutic options for AF prevention or heart failure.
Subject to securing additional financing, ARCA anticipates initiating PRECISION-AF in the fourth quarter of 2020.
AB171 – a thiol-substituted isosorbide mononitrate being developed as a potential genetically-targeted treatment for HF and peripheral arterial disease (PAD).

Subject to securing additional financing, the Company anticipates conducting non-clinical studies to support a potential IND submission and initiation of clinical development in 2021.
2019 Summary Financial Results

Cash and cash equivalents were $8.4 million as of December 31, 2019, compared to $6.6 million as of December 31, 2018. ARCA believes that its current cash and cash equivalents will be sufficient to fund its operations, at its current cost structure, after giving effect to potential cost reductions, through the end of the third quarter of 2020.

Research and development (R&D) expenses for the year ended December 31, 2019 were $1.8 million compared to $4.2 million for 2018. The $2.4 million decrease was primarily due to decreased clinical expenses following the completion of the GENETIC-AF clinical trial. If the PRECISION-AF clinical trial is initiated in the second half of 2020, R&D expense in 2020 is expected to be higher than 2019.

General and administrative (G&A) expenses were $4.0 million for 2019 compared to $3.9 million for 2018. The Company expects G&A expenses in 2020 to be consistent with those in 2019 as it maintains administrative activities to support its ongoing operations.

Total operating expenses for 2019 were $5.8 million compared to $8.1 million for 2018. The decrease in total operating expenses was primarily attributable to the decrease in R&D expense due to the completion of the GENETIC-AF clinical trial.

Net loss was $5.5 million, or $4.15 per basic and diluted share, for 2019 compared to $7.9 million, or $10.31 per basic and diluted share, for 2018.

The Company will need to raise additional capital, and/or complete a partnership or other possible strategic transaction, to fund future operations and develop Gencaro or any other product candidates.

On February 18, 2020 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that the company is scheduled to present at the Cowen 40th Annual Healthcare Conference in Boston on Tuesday, March 3, 2020 at 11:20 a.m. ET (Press release, Agios Pharmaceuticals, FEB 18, 2020, https://investor.agios.com/news-releases/news-release-details/agios-present-cowen-40th-annual-healthcare-conference-tuesday [SID1234554420]).

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A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation

On February 18, w2020 Agilent Technologies Inc. (NYSE: A) reported revenue of $1.36 billion for the first quarter ended Jan. 31, 2020, up 5.7% year over year (and up 2.4% on a core(1) basis) (Press release, Agilent, FEB 18, 2020, https://www.agilent.com/about/newsroom/presrel/2020/18feb-gp2004.html [SID1234554419]).

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On a GAAP basis, first-quarter net income was $197 million or 63 cents per share. This compares with $504 million or $1.57 per share in the first quarter of fiscal year 2019. GAAP earnings per share were down 60% year over year. Non-GAAP(2) net income was $252 million or 81 cents per share during the quarter compared with $244 million or 76 cents per share during the first quarter a year ago. Non-GAAP earnings per share were up 7% year over year.

"The Agilent team delivered a strong start to 2020. Our revenues were above expectations with growth across all regions and markets, and earnings per share at the top end of our guidance," said Mike McMullen, Agilent president and CEO. "Our first-quarter results provide clear evidence our growth strategy is working."

Financial Highlights

Life Sciences and Applied Markets Group

First-quarter revenue of $638 million from Agilent’s Life Sciences and Applied Markets Group (LSAG) was up 5% year over year (and down 2% on a core(1) basis). LSAG’s operating margin was 24.8%.

Agilent CrossLab Group

First-quarter revenue of $470 million from the Agilent CrossLab Group (ACG) grew 6% year over year (up 7% on a core(1) basis). ACG’s operating margin was 25.4%.

Diagnostics and Genomics Group

First-quarter revenue of $249 million from Agilent’s Diagnostics and Genomics Group (DGG) grew 6% year over year (up 7% on a core(1) basis). DGG’s operating margin was 13.5%.

Second-Quarter and Full-Year Outlook

Agilent expects second-quarter 2020 revenue in the range of $1.28 billion to $1.32 billion. Second-quarter 2020 non-GAAP(3) earnings are expected to be in the range of 72 cents to 76 cents per share after factoring in the potential impact of the coronavirus disease 2019 (COVID-19).

The company is maintaining its previous outlook and expects revenue of $5.50 billion to $5.55 billion for fiscal year 2020. Fiscal year 2020 non-GAAP(3) earnings guidance also remains unchanged in the range of $3.38 to $3.43 per share.

The outlook is based on Jan. 31, 2020, currency exchange rates.

Conference Call

Agilent’s management will present additional detail regarding the company’s first-quarter 2020 financial results on a conference call with investors today at 1:30 p.m. PST. This event will be webcast live in listen-only mode. Listeners may log on at www.investor.agilent.com and select "Q1 2020 Agilent Technologies Inc. Earnings Conference Call" in the "News & Events — Calendar of Events" section. The webcast will remain available on the company’s website for 90 days.

Additional financial information can be found at www.investor.agilent.com by selecting "Financial Results" in the "Financial Information" section.

On February 18, 2020 Pharma Two B Ltd., a privately held company developing innovative therapeutics based on previously approved drugs for Parkinson disease, reported the appointment of David S. Tierney M.D. as Chief Executive Officer, effective immediately as well as the establishment of a fully owned U.S. subsidiary to serve as the marketing base of the Company’s activities (Press release, Pharma Two B, FEB 18, 2020, View Source [SID1234554416]).

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Pharma Two B’s main product, P2B001, is targeting patients with early stage Parkinson’s Disease. According to the Michael J. Fox Foundation for Parkinson’s Research (MJFF), Parkinson’s disease is estimated to affect nearly 1 million people in the United States and more than 6 million people worldwide. The Company estimates that approximately 15%-20% of these patients could be defined as early stage patients.

"We are excited to have David, a highly accomplished pharmaceutical executive with proven business development, medical and financial acumen, join Pharma Two B as CEO," said Ehud Marom, Chairman of Pharma Two B’s Board of Directors. "David has a successful track record in drug approvals, developing and executing plans of commercial organizations as well as raising equity capital raising from both private and VC investors, and the public markets, including the completion of an IPO."

Dr. Tierney stated, "I am delighted to lead Pharma Two B at this pivotal time and believe there are substantial opportunities to create significant value by registering P2B001 and building the commercial infrastructure to launch the product in the U.S. in 2021. This plan will include securing the needed financial resources for a successful launch through quality financings with high quality investors that will prepare the Company for an IPO and or strategic combination."

P2B001 is a low dose, sustained release combination of pramipexole and resagiline. The drug is orally dosed once daily with no titration. P2B001 is near the end of the Phase 3 pivotal study and the Company is preparing for an NDA submission next year via the 505(b)(2) regulatory pathway. Dr. Tierney added, "I look forward to working with our team and board members, completing the ongoing Phase 3 study and building the infrastructure to support a successful launch, including marketing and collaboration with potential partners."

About David S. Tierney, M.D.

Prior to David’s appointment as CEO of Pharma Two B, he was President and CEO of BioPharmX Corp (NYSE American: BPMX) and of Icon Bioscience, Inc., where he led a team that in 2018 received U.S. FDA approval of its New Drug Application (NDA) for DEXYCU (dexamethasone intraocular suspension), a dropless, long-acting therapeutic for treating inflammation associated with cataract surgery. Icon was merged with EyePoint Pharmaceutical, Inc (NASDAQ: EYPT) in March 2018.

Dr. Tierney served as President and COO of Oceana Therapeutics, Inc., a specialty therapeutic company he co-founded in 2008. Oceana quickly established a global commercial network and by 2011 was acquired by Salix Pharmaceuticals. In 2000, David was appointed President & CEO of Hydro Med Sciences (HMS), a research firm with a promising drug delivery platform. Under his leadership, HMS emerged as Valera Pharmaceuticals, a fully integrated, commercial, specialty pharma company that successfully completed an initial public offering in 2005. Valera has since been merged into Endo Pharmaceuticals (NASDAQ: ENDP). In total, Dr. Tierney has over 30 years of experience in executive and management positions in the pharmaceutical industry. Dr. Tierney received his medical degree from Royal College of Surgeons in Ireland.

David Tierney is the recipient of two Ernst & Young Entrepreneur of the Year awards, first in 2005 and again in 2011, respectively, reflecting his accomplishments at Valera and Oceana. He was also featured in the inaugural 2010 Irish Life Science 50, an honor presented by the President of Ireland in conjunction the Irish Voice and Irish America Magazine. Additionally, in 2005, 2006, and 2011, PharmaVOICE 100 named David one of the most inspiring people in health sciences.

On February 18, 2020 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, reported the final data from its CTCL clinical trial of WP1220 for the treatment of cutaneous T-cell lymphoma (CTCL), which was published and presented by Dr. M. Sokolowska-Wojdylo in conjunction with the 4th Annual World Congress of Cutaneous Lymphomas in Barcelona, Spain on February 13, 2020 (Press release, Moleculin, FEB 18, 2020, View Source [SID1234554415]). The final results supported the safety of topical WP1220 and demonstrated a median improvement in the Composite Assessment of Index Lesion Severity (CAILS) score of 56% in treated (index) lesions for patients completing the study.

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Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)

"WP1220 is part of our p-STAT3 inhibitor drug portfolio. Late last year, we announced the preliminary results of this proof of concept Phase 1 trial. For years, p-STAT3 (the activated form of STAT3) has been considered an ‘undruggable’ target because of the difficulty of reaching and affecting this cell-signaling protein," commented Walter Klemp, Moleculin’s Chairman and CEO. "We are pleased to conclude this trial and demonstrate a therapeutic effect from a p-STAT3 inhibitor which could be considered a significant breakthrough in cancer research."

Introduction & Objectives: Mycosis Fungoides or MF, the most common variant of CTCL, is a disease with symptomatic, disfiguring skin lesions. STAT3, an oncogenic transcription factor, has been identified as a critical regulator of MF, whereby the activation of STAT3 through phosphorylation (p-STAT3) has been linked to tumor proliferation and suppression of immune responses. WP1220, a synthetic compound, potently inhibits the activity of p-STAT3 and the growth of CTCL cell lines. This Phase 1b study was designed to demonstrate the safety and efficacy of WP1220 after topical treatment of MF.

Results: Of 5 subjects enrolled, 9 lesions were assessed according to the CAILS scoring system. The only adverse event (AE) was mild contact dermatitis in one subject felt not to be related to the drug. 4 of the 5 subjects had significant improvement in CAILS scores on index lesions, with a median reduction of 56% (range 25%-94%). Improvement was noted within 7 days of treatment initiation and maintained 1 month after discontinuation. Independent dermatologic review based on photographic documentation was conducted and corroborated these findings.

Conclusions: WP1220, an inhibitor of p-STAT3, shows safety and significant efficacy in MF after topical treatment. We believe this is the first demonstration in humans that inhibition of p-STAT3 with topical therapy has efficacy in CTCL. A larger Phase 2 study is now being planned.