On May 1, 2019 ArQule, Inc. (Nasdaq: ARQL) reported its financial results for the first quarter, 2019 (Press release, ArQule, MAY 1, 2019, View Source [SID1234535491]).

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For the quarter ended March 31, 2019, the Company reported a net loss of $10,267,000, or $0.09 per share, compared with net loss of $6,532,000, or $0.07 per share, for the quarter ended March 31, 2018.

As of March 31, 2019, the Company had a total of approximately $92,223,000 in cash, cash equivalents, and marketable securities.

Key Highlights from Q1, 2019

ARQ 531, our potent and reversible dual inhibitor of both wild-type and C481S-mutant BTK. Reported in March that the first evaluable CLL patient with a C481S mutation enrolled in cohort 7 (65 mg QD) of our phase 1 trial achieved a partial response that has since been confirmed with a subsequent scan. This is in addition to a previously reported follicular lymphoma patient who had also achieved a partial response and continues on therapy. Cohort 7 has been cleared for safety and we have begun enrolling patients in cohort 8 (75 mg QD). No additional DLTs have been observed at any dose. We plan to present detailed, updated data from this ongoing trial at the European Hematological Association meeting in June
Miransertib, our potent and selective first-generation AKT inhibitor. Concluded interactions with the FDA and defined the registrational trial designs for both Proteus syndrome and PIK3CA-Related Overgrowth Spectrum (PROS). We have finalized the protocol and received the first conditional IRB approvals
ARQ 751, our highly potent and selective next-generation AKT inhibitor. Signal generation work in genetically-defined solid tumors continues, and we plan to present the final data set at a major conference by year end
Derazantinib, our FGFR inhibitor, partnered with Basilea and Sinovant, in a registrational trial for intrahepatic cholangiocarcinoma. Substantially completed the timely recruitment and transfer of clinical and other responsibilities to Sinovant and Basilea
Paolo Pucci, Chief Executive Officer of ArQule, commented, "We have made tremendous progress across our pipeline in Q1, and we are particularly pleased with the safety and dose dependent clinical activity profile that is emerging with ARQ 531."

"We are busy collecting data for our ARQ 531 presentation at EHA (Free EHA Whitepaper), and we are confident that we will demonstrate meaningful incremental clinical activity in addition to the two PRs already announced", commented Dr. Brian Schwartz, Chief Medical Officer of ArQule. "We are also pleased by the rapid review of the initial IRBs for our registrational trial with miransertib in Proteus syndrome and PROS."

Revenues and Expenses

Revenues for the first quarter, 2019, were $1,345,000 compared with revenues of $4,138,000 for the first quarter, 2018.

Research and development expenses in the first quarter, 2019 were $7,448,000 compared with $5,812,000 for the first quarter, 2018.

General and administrative expenses in the first quarter, 2019 were $4,300,000 compared with $2,351,000 for the first quarter, 2018.

2019 Financial Guidance

Our 2019 financial guidance has not changed. For 2019, ArQule expects revenue to range between $3 and $5 million. Net loss is expected to range between $40 and $43 million, and net loss per share to range between $(0.37) and $(0.39) for the year. ArQule expects to end 2019 with between $60 and $63 million in cash and marketable securities.

Conference Call and Webcast

ArQule will hold its first quarter financial results call today, May 1, 2019 at 9:00 a.m. ET. The live webcast can be accessed in the "Investors and Media" section of our website, www.arqule.com, under "Events and Presentations." You may also listen to the call by dialing (877) 868-1831 within the U.S. or (914) 495-8595 outside the U.S. A replay will be available two hours after the completion of the call and can be accessed in the "Investors and Media" section of our website, www.arqule.com, under "Events and Presentations."

On May 1, 2019 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a physician-led biopharmaceutical company focused on immuno-inflammatory and dermatological diseases, reported it will report financial results for the first quarter 2019, Wednesday, May 8th, after U.S. financial markets close (Press release, Aclaris Therapeutics, MAY 1, 2019, View Source [SID1234535490]).

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Management will conduct a conference call at 5:00 PM ET to discuss Aclaris’ financial results and provide a general business update. The conference call will be webcast live over the Internet and can be accessed by logging on to the "Investors" page of the Aclaris Therapeutics website, www.aclaristx.com, prior to the event. A replay of the webcast will be archived on the Aclaris Therapeutics website for 30 days following the call.

To participate on the live call, please dial (844) 776-7782 (domestic) or (661) 378-9535 (international), and reference conference ID 5737199prior to the start of the call.

On April 30, 2019 ALENTIS Therapeutics ("ALENTIS"), a Swiss-French biotech company developing novel therapeutics in advanced liver disease and cancer, reported the completion of a Series A financing of CHF 12.5million (Euro 11.1m; USD 12.5m) (Press release, Alentis Therapeutics, APR 30, 2019, View Source [SID1234556137]). The Swiss venture capital firms BioMedPartners and BB Pureos Bioventures co-led the round and were joined by Bpifrance, Schroder Adveq and the German High-Tech Gründerfonds (HTGF).

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Markus L.E. Ewert, PhD, MBA has joined the company as Chief Executive Officer. Markus was previously CBO at Ablynx, contributing to both its US IPO and highly successful sale to Sanofi. Before, he led global corporate development at GE Healthcare, had leadership positions at Novartis, and has a track record of building businesses based on innovations.

ALENTIS’ lead program is a humanized monoclonal antibody against a target that plays a key role in the pathology of liver fibrosis and hepatocellular carcinoma (HCC). Advanced liver disease and cancer are major clinical challenges world-wide and characterized by a poor outcome and limited therapeutic options.

ALENTIS’ research is powered by its proprietary drug discovery platform, which is based on a prognostic liver disease signature holding promise for the discovery and development of further drug candidates to treat advanced liver diseases and cancer.

ALENTIS is founded on ground-breaking research of the laboratory of Prof. Thomas Baumert, MD at the University of Strasbourg, the Inserm Institute for Viral and Liver Disease, the Laboratory of Excellence HepSYS and the Institut Hospitalo-Universitaire Strasbourg as well as other collaborators, including Prof. Yujin Hoshida, MD at the University of Texas Southwestern in Dallas. SATT Conectus, the organization for accelerating technology transfer in the Alsace territory (France) has been essential in providing, building and securing the IP portfolio that has been licensed to ALENTIS. BaseLaunch, a healthcare accelerator operated by BaselArea.swiss, has been instrumental as an early stage financial and operational supporter in the formation of the company. As a result of this tri-national collaboration, ALENTIS’ headquarters have been incorporated in Basel (Switzerland) with a subsidiary in Strasbourg (France) and a branch in Germany.

Thomas F. Baumert, MD, principal founder of ALENTIS and Professor of Medicine, Head Inserm Research Institute for Viral and Liver Diseases, University of Strasbourg and Strasbourg University Hospitals said: "Together with my colleagues we are excited to be translating our novel approaches and therapeutic compounds with a unique mechanism of action into clinical drug candidates, with the aim of improving the very poor outcomes of patients with advanced liver disease."

Andreas Wallnöfer, former Head of Clinical Research & Exploratory Development at F. Hoffmann-La Roche Ltd and General Partner at BioMedPartners added: "The pre-clinical data set of the ALENTIS lead molecule are most comprehensive and convincing: they support the therapeutic potential in advanced liver disease of different origins. Prof. Baumert’s research is truly translational and links pre-clinic and clinic through a prognostic gene expression signature, which is most valuable to optimally characterize the lead project and fuel further programs."

ALENTIS’ Board of Directors will comprise Neil Goldsmith, a co-founder and Chairman; Andreas Wallnöfer, General Partner at BioMedPartners; Martin Münchbach, Managing Partner at BB Pureos Bioventures; Benoit Barteau, Senior Investment Manager at BPI France, and Prof. Thomas F. Baumert, the principal founder.

On April 30, 2019 Helix BioPharma Corp. (TSX: HBP), ("Helix" or the "Company"), an immunooncology company developing innovative drug candidates for the prevention and treatment of cancer, has reported that the Trial Steering Committee ("TSC") reviewed safety data from the first dosing cohort of the Company’s LDOS003 study. No serious adverse events or dose limiting toxicities were observed (Press release, Helix BioPharma, APR 30, 2019, View Source [SID1234536460]). TSC recommended that Helix begin enrollment of patients into the second dosing cohort.

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LDOS003 is a Phase II, open-label, randomized study of immunoconjugate L-DOS47 in combination with vinorelbine and cisplatin as compared to vinorelbine and cisplatin alone in patients with lung adenocarcinoma. The study is divided into two parts. In part I, the maximum tolerated dose of L-DOS47, when given in combination with vinorelbine/cisplatin, will be determined. Cohorts of 3 patients will be recruited into three dosing cohorts (6, 9 and 12 µg/kg). All patients at a given dose level must complete the first treatment cycle (3-week period) before escalation in subsequent patients can proceed. In part II, after the maximum tolerated dose of L-DOS47 in combination with vinorelbine/cisplatin has been determined, a further 118 patients will be randomized (1:1) to receive L-DOS47 in combination with vinorelbine/cisplatin, or vinorelbine/cisplatin alone.

"We are very encouraged that the first dosing cohort of this L-DOS47 phase II study in combination with chemotherapy has been completed" said Heman Chao, Helix’s Chief Executive Officer. "We look forward to receiving more data as we advance the L-DOS47 development program."

About L-DOS47 clinical development
L-DOS47 is currently being clinically evaluated in three clinical studies, in the United States, Poland and Ukraine as a treatment for certain patients with non-small cell lung cancer ("NSCLC").

LDOS001 is a Phase I, open-label, dose escalation study being conducted in the United States at the University Hospitals Case Medical Center. The primary objective of the study is to determine the safety and tolerability of L-DOS47 in combination treatment with pemetrexed/carboplatin. The study will also evaluate the potential clinical benefit of L-DOS47 with this combination. Patient enrollment is active for this study.

LDOS002 is an open-label Phase I/II clinical study being conducted in Poland to evaluate the safety, tolerability and preliminary efficacy of ascending doses of L-DOS47, initially as a monotherapy, in patients with inoperable, locally advanced, recurrent or metastatic, non-squamous, stage IIIb/IV NSCLC. Patient enrollment has completed for this study.

LDOS003 is a phase II, open-Label, randomized study of L-DOS47 in combination with vinorelbine/cisplatin as compared to vinorelbine/cisplatin alone in patients with Lung adenocarcinoma. The 2 primary objectives of the study include safety, tolerability and efficacy of L-DOS47 in this combination treatment. Patient enrollment is about to commence for this study.

On April 30, 2019 Vividion Therapeutics, a biotechnology company with a revolutionary platform to discover small molecule therapeutics against biologically compelling but previously intractable targets, reported the completion of an oversubscribed $82 Million Series B financing (Press release, Vividion Therapeutics, APR 30, 2019, View Source [SID1234535882]). The financing was led by Nextech Invest, an oncology-focused investment firm, and included participation from additional new investors BVF Partners, Casdin Capital, Mubadala Ventures, Trinitas Capital, Mirae Asset Capital, Altitude Life Science Ventures, and Alexandria Venture Investments. Existing investors ARCH Venture Partners, Versant Ventures, Cardinal Partners and Celgene Corporation also participated in the round.

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In connection with the financing, Jakob Loven, Ph.D., Partner at Nextech Invest, will join the Vividion Board of Directors.

"Vividion’s unique approach has allowed us to discover and advance exquisitely selective small molecules against high value targets that have been extremely challenging to drug historically," said Dr. Diego Miralles, Chief Executive Officer of Vividion Therapeutics. "With over $165 million in cash and a remarkable team, we are well positioned with substantial runway to maximize the multiple opportunities provided by our unique platform, bringing therapeutics to many patients in need."

Vividion Therapeutics has pioneered an approach, based on the work of Professor Benjamin Cravatt at The Scripps Research Institute, that enablesscreening of small molecules against every protein in native biological systems. This unique platform identifies highly selective binders to previously intractable targets, agnostic to protein class and function. These can be developed into drugs utilizing a range of approaches, including direct and allosteric modulation of protein function, and targeted protein degradation. The company’s immediate focus is in oncology and immunology.

"We are witnessing a renaissance in the field of synthetic and medicinal chemistry and the numerous possibilities small molecule drug discovery brings to previously challenging target classes," said Loven. He added: "Vividion has successfully pioneered its platform to reveal druggable opportunities at an unprecedented scale and resolution to open a large range of therapeutic possibilities. We look forward to working with Vividion’s highly experienced team as they continue to build the platform and pipeline toward first-in-class medicines for underserved patients."