On May 8, 2018 CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, reported financial results for the quarter ended March 31, 2018, and provided an overview of recent accomplishments and plans for its research and development programs (Press release, CytRx, MAY 8, 2018, View Source [SID1234526247]).

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"We’ve had a strong start to 2018, highlighted by the presentation of statistically significant breakthrough data for our four new albumin binding ultra high potency LADR (Linker Activated Drug Release) drug candidates at AACR (Free AACR Whitepaper) 2018," said Eric Curtis, CytRx’s President and Chief Operating Officer. "The potential competitive advantages of the LADR assets over antibody-drug conjugates are significant, including broader therapeutic utility and patient populations, lower risk of immune responses, lower cost of goods, simpler manufacturing, and no requirement for antibody receptors. Looking ahead to the remainder of 2018, we are laser-focused on securing a strategic alliance for the innovative LADR technology, with the goal of closing a transaction during the fourth quarter of 2018."

First Quarter 2018 and Recent Highlights

Eric L. Curtis is Named President and Chief Operating Officer. In early May 2018, CytRx announced that its Board of Directors had affirmed the appointment of Mr. Curtis to be the President and Chief Operating Officer of CytRx. Mr. Curtis will be part of CytRx’s executive management team and will utilize his significant development and operational expertise to partner the Company’s LADR technology and assets, as well as maximize the success of CytRx’s scientific programs. He had previously served as an independent consultant for the Company’s internal development efforts and external partner relationships. Mr. Curtis, who holds an M.B.A., brings more than 25 years of life science leadership experience to CytRx, with a proven track record in oncology and orphan diseases.
NantCell Inc.’s Aldoxorubicin Abstract Selected for Presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2018 Annual Meeting. In April 2018, CytRx highlighted that an abstract submitted by aldoxorubicin licensee NantCell, Inc., a private subsidiary of NantWorks, LLC, entitled Lack of cardiac toxicity in patients treated with aldoxorubicin with doxorubicin equivalent doses beyond 1000mg/m2 was selected for a poster presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2018 Annual meeting, to be held June 1 – 5 in Chicago. Dr. Sant Chawla, Principal Investigator and Director of the Sarcoma Oncology Center in Santa Monica, California, will be the presenter. CytRx out-licensed global development, manufacturing, and commercialization rights for aldoxorubicin to NantCell, Inc. in July 2017.
Presented Statistically Significant Breakthrough LADR Candidate Data at the AACR (Free AACR Whitepaper) 2018 Annual Meeting. In April 2018, CytRx presented three posters highlighting breakthrough data relating to its LADR drug candidates at the AACR (Free AACR Whitepaper) 2018 Annual Meeting in Chicago. The posters describe the positive scientific findings that led to the Company’s decision to select auristatin E (AE) derivatives LADR-7 and LADR-8, and maytansine derivatives LADR-9 and LADR-10, as the LADR candidates eligible to advance toward IND-enabling studies. The compounds demonstrated excellent, long-term antitumor activity across a wide range of human solid tumor cancer types, including lung, breast, ovarian, head and neck, renal cell, and melanoma. PDF copies of the presented posters (abstracts #1657, #2661, and #3703) can be accessed here.
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Selected Four LADR Candidates Eligible for IND-Enabling Clinical Studies. In March 2018, CytRx announced that four LADR candidates are eligible for advancement into IND-enabling studies. These candidates, named LADR-7, LADR-8, LADR-9 and LADR-10 were chosen from two distinct classes of highly cytotoxic drugs, auristatins and maytansinoids.
Patterson Derivative Action is Dismissed. In March 2018, the Vice-Chancellor of the Delaware Court of Chancery ruled that CytRx’s motion to dismiss was granted, with prejudice. Patterson v. Kriegsman et al., C.A. No. 2017-0636-TMR.
Initiation of Pharma Partnering Activities for the LADR Ultra-High Potency Drug Candidates. In February and March 2018, CytRx made two announcements that highlighted efforts to strengthen its strategic alliance team. First, CytRx announced that it had entered into an exclusive agreement with Destum Partners, Inc., a leading strategic advisory firm serving companies in the life sciences industry, to assist in executing a strategic alliance for CytRx’s LADR drug candidates. CytRx also announced that Mr. Eric L. Curtis, MBA, joined its team to provide strategic counsel to the Chairman and CEO for the Company’s ongoing programs, including its LADR discovery platform and the ultra-high potency drug candidates. Mr. Curtis will foster introductions to strategic partners from his extensive network of big pharma and biotech companies. He brings a wealth of drug development and commercialization experience to CytRx, and he, along with Destum Partners, made introductions at AACR (Free AACR Whitepaper).
Partner NantCell Dosed First Patient in Clinical Trial Investigating Cell-Based Therapy in Combination with Multiple Anti-Cancer Agents, including Aldoxorubicin, in Patients with Metastatic Pancreatic Cancer. In January 2018, CytRx announced that aldoxorubicin licensee NantCell, Inc. (a private subsidiary of NantWorks, LLC) dosed the first patient in the Phase 1b portion of a Phase 1b/2 clinical trial for patients with metastatic pancreatic cancer. The trial will investigate high-affinity natural killer (haNK) cell therapy in combination with several anti-cancer agents, including aldoxorubicin, in patients with metastatic pancreatic cancer whose cancer has progressed on or following standard-of-care chemotherapy. This trial is a single-center, open-label, Phase 1b/2 clinical trial designed to evaluate the safety and efficacy of the various combination therapies with enrollment expected to be approximately 173 patients. The primary endpoint for the Phase 1b portion of the trial is safety and the primary endpoint for the Phase 2 portion of the trial is objective response rate (ORR) by RECIST.
Partner NantCell Dosed First Patient in Clinical Trial Evaluating Aldoxorubicin in Combination with Immuno-Oncology Agents and Cell-Based Therapies in Patients with Advanced Squamous Cell Carcinoma. In February 2018, CytRx announced that NantCell dosed the first patient in the Phase 1b portion of a Phase 1b/2 clinical trial for patients with advanced squamous cell carcinoma (SCC) of either the head and neck or non-small cell lung cancer, the second trial conducted by NantCell to investigate haNK cell therapy in combination with several anti-cancer agents, including aldoxorubicin, in certain high unmet need cancer indications. This single-center, open-label, Phase 1b/2 clinical trial is designed to evaluate the safety and efficacy of the various combination therapies, including combinations with aldoxorubicin, in subjects with SCC who have progressed on or after platinum-based chemotherapy and anti-PD1/PD-L1 therapy. Approximately 65 patients are expected to be enrolled. The primary endpoint for the Phase 1b portion of the trial is safety and the primary endpoint for the Phase 2 portion of the trial is objective response rate (ORR) by RECIST.
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First Quarter 2018 Financial Results

CytRx reported cash and cash equivalents of $35.1 million as of March 31, 2018.

Net loss for the quarter ended March 31, 2018, was $4.1 million, or $(0.15) per share, compared with a net loss of $11.0 million, or $(0.60) per share, for the quarter ended March 31, 2017, a reduction of $6.9 million. Net loss decreased by approximately 63% for the quarter ended March 31, 2018. During the first quarter of 2018, the Company recognized a non-cash gain of $0.45 million on the fair value adjustment of warrant derivative liabilities related to warrants issued in 2016, compared to a non-cash loss of $32,000 during the first quarter of 2017 related to now expired warrants.
Research and development (R&D) expenses were $1.5 million for the first quarter of 2018, which were primarily incurred for preclinical development of the new LADR albumin-binding, ultra-high potency drug candidates in the Company’s Freiburg, Germany lab. This is a reduction of approximately $5.3 million compared to R&D expenses of $6.8 million for the first quarter of 2017, which included clinical development expenses for aldoxorubicin.
General and administrative (G&A) expenses were $2.5 million for the first quarter of 2018, compared with $3.0 million for the first quarter of 2017, including non-cash stock-compensation expense of $0.4 million for the first quarter of 2018 as compared to 0.6 million for the first quarter of 2017. G&A expenses decreased by approximately 17 percent primarily due to a decrease in legal fees.
About the LADR Technology Platform
CytRx’s innovative LADR (Linker Activated Drug Release) technology employs a broad portfolio of novel linker molecules that selectively bind to circulating albumin and can be linked to a wide variety of anti-cancer payloads. The Company’s research efforts currently center on creating new molecules from the combination of ultra-high potency cytotoxic payloads with tunable linkers. The molecules that CytRx is currently evaluating concentrate at the tumor site providing targeted delivery of the cell killing payloads.

On May 8, 2018 Pieris Pharmaceuticals, Inc. presented Deutsche Bank Health Care Conference presentation (Presentation, Pieris Pharmaceuticals, MAY 8, 2018, View Source [SID1234526265]).

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On May 7, 2018 Allergan plc (NYSE: AGN), a leading global biopharmaceutical company, reported that Chief Commercial Officer William Meury will present at the Bank of America Merrill Lynch Healthcare Conference in Las Vegas, Nevada (Press release, Allergan, MAY 7, 2018, View Source(1) [SID1234526160]). The presentation will begin at 8:00 a.m. Pacific Time (11:00 a.m. Eastern Time) on Thursday, May 17, 2018.

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The presentation will be webcast live and can be accessed on Allergan’s Investor Relations website at www.allergan.com/investors. The webcast can also be accessed through the following URL: https://www.veracast.com/webca…

An archived version will be available within approximately one hour of the live presentation, and can be accessed at the same location for 90 days.

On May 7, 2018 ArQule, Inc. (Nasdaq: ARQL) reported its financial results for the first quarter of 2018 (Press release, ArQule, MAY 7, 2018, View Source [SID1234526161]).

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For the quarter ended March 31, 2018, the Company reported a net loss of $6,532,000, or $0.07 per share, compared with net loss of $7,576,000, or $0.11 per share, for the quarter ended March 31, 2017.

At March 31, 2018, the Company had a total of approximately $42,884,000 in cash and marketable securities.

Key Highlights

ARQ 531, our potent and reversible BTK inhibitor, demonstrated good oral bioavailability and pharmacokinetics in data presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) on April 15, 2018. The Phase 1a portion of the Phase 1a/b trial continues to recruit on schedule with no safety concerns, and we plan to present more advanced data at one or more major congresses later in 2018.
Miransertib, our lead proprietary AKT inhibitor, was featured in an oral presentation at AACR (Free AACR Whitepaper) in which it showed positive signs of activity in hormone- sensitive tumors with AKT1 or PI3K dysregulation. The Phase 1b trial of miransertib with anastrozole in patients with advanced endometrial cancer produced one complete response and three partial responses out of 8 patients and continues to recruit.
Miransertib has been granted Orphan Drug Designation by EMA for the rare disease, Proteus syndrome. We continue to make progress with our registrational strategy in Proteus syndrome; in addition, we are progressing our rare disease expansion strategy with the Phase 1/2 trial in PROS and Proteus syndrome which is recruiting on schedule.
The Company granted Basilea Pharmaceutica Ltd. ("Basilea") an exclusive license to develop and commercialize derazantinib, our pan-FGFR inhibitor, in all parts of the world except the People’s Republic of China, Hong Kong, Macau and Taiwan, where the Company has licensed rights to Sinovant Sciences, Ltd., a subsidiary of Roivant Sciences Ltd. Terms of the transaction include a $10 million upfront payment, an additional $326 million in regulatory and commercial milestones, and royalties on net sales ranging from single to double digits; Basilea will be responsible for all costs and expenses of development, manufacture and commercialization in its territory.
"We have continued to build on the momentum that we created during 2017 with presentations of important data at the recent AACR (Free AACR Whitepaper) meeting and the licensing of derazantinib to our partner, Basilea," said Paolo Pucci, Chief Executive Officer of ArQule. "This new partnership further supports our mid-term strategy by allowing us to develop derazantinib in ways that we could not have done on our own and by strengthening our balance sheet thus enabling us to focus more on our BTK and ATK programs in potential fast-to-market settings."

"After a very strong 2017 scientifically, our execution in 2018 continues to be at a high level, highlighted by continuing progress across all pipeline assets," said Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer of ArQule. "We are approaching therapeutic levels in the dosing of patients in the Phase 1a/b trial with our BTK inhibitor, ARQ 531, and look forward to presenting a comprehensive data set from that trial later this year. The AKT program is also progressing well in both rare diseases and oncology. We are launching an expansion of the Phase 1b trial with miransertib plus anastrozole in patients with advanced endometrial cancers and are executing our registrational strategy for Proteus and PROS."

Revenues and Expenses

Revenues for the quarter ended March 31, 2018, were $4,138,000 compared with revenues of zero for the quarter ended March 31, 2017. Research and development revenue in the quarter ended March 31, 2018 consisted of $3,000,000 from the February 2018 Roivant licensing agreement and $1,138,000 from our October 2017 non-exclusive license agreement for certain library compounds.

Research and development expenses in the first quarter of 2018 were $5,812,000, compared with $5,194,000 for the first quarter 2017.

Research and development expenses increased $0.6 million in the first quarter of 2018 compared to the first quarter of 2017 primarily due to higher outsourced pre-clinical, clinical and product development costs.

General and administrative expenses in the first quarter of 2018 were $2,351,000, compared with $2,074,000 for the first quarter of 2017. General and administrative expenses increased $0.3 million in the first quarter of 2018 compared to the first quarter of 2017 primarily due to higher labor related costs of $0.2 million and professional fees of $0.1 million.

2018 Updated Financial Guidance

As a result of the April 2018 exclusive license agreement with Basilea, our guidance for 2018 is being updated. For 2018, ArQule now expects revenue to range between $14 and $17 million. Net use of cash is expected to range between $27 and $29 million for the year. Net loss is expected to range between $16 and $21 million, and net loss per share to range between $(0.18) and $(0.24) for the year. ArQule expects to end 2018 with between $40 and $42 million in cash and marketable securities.

Conference Call and Webcast

ArQule will hold its first quarter 2018 financial results call today, May 7, 2018 at 9:00 a.m. ET. The live webcast can be accessed in the "Investors & Media" section of our website, www.arqule.com, under "Events & 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 "Investor and Media" section of our website, www.arqule.com, under "Events & Presentations."

On May 7, 2018 Castle Biosciences, Inc., the skin cancer diagnostics company providing molecular diagnostics to improve cancer treatment decisions, reported the presentation of data supporting clinical use of the DecisionDx-Melanoma test to inform sentinel lymph node biopsy (SLNB) recommendations (Press release, Castle Biosciences, MAY 7, 2018, View Source [SID1234526162]). The study was presented as a poster at the American College of Mohs Surgery (ACMS) 50th Annual Meeting in Chicago.

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The study found that the DecisionDx-Melanoma test result can be used with clinicopathologic factors to inform patient discussions and recommendations for SLNB in line with national melanoma clinical practice guidelines.

Study Background:

SLNB is recommended to assess prognosis of melanoma patients. Current guidelines recommend that clinicians discuss the SLNB procedure with patients who have a greater than 5% likelihood of SLN positivity; the guidelines do not generally recommend the procedure if a patient has a less than 5% likelihood of SLN positivity.
Previously, a patient’s risk for SLN positivity has been determined using traditional clinicopathologic features such as Breslow’s thickness and ulceration status. Importantly, it is recognized that older patients have a lower likelihood of SLN positivity but are at higher risk of recurrence and death from melanoma.
The DecisionDx-Melanoma test is a 31-gene expression profile (GEP) test that uses tumor biology to provide an individual risk of recurrence in cutaneous melanoma patients. The validity and performance of the test has been confirmed in three multicenter archival tissue studies involving 690 patients and three prospective studies involving 702 patients. The test has been shown to be an independent predictor of recurrence compared to clinicopathologic factors of Breslow’s thickness, ulceration status, mitotic rate and SLN status.
This study was designed to determine whether the test could be used along with clinicopathologic factors to improve identification of patients with a low likelihood of SLN positivity (<5%) as well as those with a higher likelihood.
Key Study Findings:

Using the DecisionDx-Melanoma GEP test in combination with clinicopathologic factors of age and T category, an algorithm predicting the likelihood of SLN positivity was validated in two independent, prospective, multicenter cohorts totaling 1,421 patients.
For patients with T1-T2 tumors older than 65 years of age who had a Class 1A test result (lowest risk of recurrence), SLN positivity was less than the 5% threshold below which guidelines do not generally recommend the procedure.
For patients with T1-T2 tumors who had a Class 2B test result (highest risk of recurrence), SLN positivity exceeded the 10% threshold for all age groups. Guidelines suggest that patients whose SLN positivity risk exceeds 10% be offered the SLNB procedure.
The impact of not performing SLN biopsy in Class 1A patients was evaluated based on a retrospective dataset of 690 patients with long-term follow-up. At 5 years, Class 1A patients with T1-T2 tumors had a melanoma-specific survival of 99.6%, overall survival of 98.2%, and distant metastasis-free survival of 95.3%.
"The incorporation of the DecisionDx-Melanoma test result, along with tumor thickness and age, helps to identify a group of patients who have a likelihood of a positive sentinel lymph node of less than 5%, suggesting they may be able to safely avoid the SLNB procedure," said Federico Monzon, M.D., FCAP, Chief Medical Officer at Castle Biosciences. "Based on these study results, the DecisionDx-Melanoma test can inform the discussion of SLNB options with melanoma patients and provides an important advance in the management of early stage melanoma patients."

Oral Presentation Features Initial Data from Cutaneous Squamous Cell Carcinoma GEP Test Development Program

Also at the meeting, Dr. Sarah Arron, M.D., Ph.D., Associate Professor in the Department of Dermatology at the University of California San Francisco, presented initial data from Castle Biosciences’ prognostic GEP test in development for cutaneous squamous cell carcinoma (cSCC).

The goal of the development program is to validate a prognostic test that can predict which cSCC patients are at higher risk of metastasis or recurrence and thus inform clinical management decisions. Data from a development cohort of samples show that preliminary predictive models can improve upon current staging methods. These results support the feasibility of the program to develop a clinically valuable test to predict which cSCC patients are at higher risk for recurrence.

"For patients with cutaneous squamous cell carcinoma, accurate prediction of their individual risk of recurrence or metastasis remains a challenge," commented Dr. Arron, who is an investigator for the study. "The development of a prognostic test that improves risk prediction could better inform important management decisions such as optimal surgical procedure, use of adjuvant radiation and selection of patients for SLNB or adjuvant immunotherapy, and has the potential to drive improvements in patient management."

Continued collaborative site recruitment and development activities are ongoing.

About DecisionDx-Melanoma

The DecisionDx-Melanoma test uses tumor biology to predict individual risk of melanoma recurrence and sentinel lymph node positivity independent of traditional factors. Using tissue from the primary melanoma, the test measures the expression of 31 genes. The test has been validated in three multi-center studies that have included 690 patients and have demonstrated consistent results. Performance has also been confirmed in four prospective studies including 702 patients. The consistent high performance and accuracy demonstrated in these studies, which combined have included over 1,300 patients, provides confidence in disease management plans that incorporate DecisionDx-Melanoma test results. Prediction of the likelihood of sentinel lymph node positivity has also been validated in two prospective multicenter studies that included over 1,400 patients. Clinical impact has been demonstrated in multi-center and single-center studies showing that test results impact clinical management decisions for one of every two patients tested. More information about the test and disease can be found at www.SkinMelanoma.com.