1stOncology™: Cancer Intelligence Service – Page 15325 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Castle Biosciences Presents Prospective Data Supporting Use of DecisionDx-Melanoma Test to Inform Sentinel Lymph Node Biopsy Recommendations

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.

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 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.

ArQule Reports First Quarter 2018 Financial Results

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]).

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."

Allergan to Present at the Bank of America Merrill Lynch Healthcare Conference

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.

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.

Agenus Reports First Quarter 2018 Financial Results and Provides Corporate Update

On May 7, 2018 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology (I-O) company with a pipeline of immune checkpoint antibodies, cancer vaccines and adoptive cell therapies1, provided a corporate update and reported financial results for the first quarter ending March 31, 2018 (Press release, Agenus, MAY 7, 2018, View Source [SID1234526159]).

"Innovation and speed are the basis of our I-O business model with 5 INDs filed over the past 18 months, 6 INDs on track for this year and 2 additional INDs planned in the 1H of next year. We have treated over 100 patients with our CTLA-4 (AGEN1884) and/or PD-1 (AGEN2034) antibodies with clinical responses in some of the patients with advanced cancers," said Garo H. Armen, Ph.D., Chairman and CEO of Agenus. " We have also made progress with our commercial readiness with commercial grade antibodies already produced; our partnering discussions are maturing, and we are committed to bring these discussions to closure."

Milestones Achieved and Upcoming

Clinical update:
To date, we have:
Presented AGEN1884 (CTLA-4) & AGEN2034(PD-1) pharmacodynamic activity at AACR (Free AACR Whitepaper)2018;
Presented preclinical data on TIM-3 (INCAGN02390) and LAG-3 (INCAGN02385); Clinical trials planned for 2018;
Completed dose escalation of AGEN1884 & AGEN2034 combination;
Launched combination trials with AGEN1884 & AGEN2034, including trials in 2L cervical cancer.
In the coming year, we expect to:
Present efficacy data on AGEN1884 and AGEN2034
>100 patients treated; clinical activity observed;
Interim data review suggests patients with advanced cancers have clinical responses, including partial and complete responses in some patients;
We have shifted our development strategy for first approval from 1L NSCLC to 2L cervical cancer because of increasing hurdles and correspondingly longer timelines.
Advance our cervical cancer trial of AGEN1884 and AGEN2034 combination is currently enrolling patients.
File an IND on next generation CTLA-4 (AGEN1181) designed to improve T cell priming and Treg depletion;
File INDs for our bispecific antibodies designed to condition the tumor microenvironment through regulatory T cell depletion and other undisclosed mechanisms;
Advance efforts to launch a combination trial with CTLA-4, PD1 & our neoantigen vaccine, AutoSynVax + QS-21.
Manufacturing Update
Supplied GMP material for clinical programs and delivered commercial grade AGEN1884
We are also on track having already filled vials of commercial grade AGEN2034
QS-21 Stimulon update
SHINGRIX is the most effective shingles vaccines; GSK commercial sales projections have nearly tripled from expectations earlier in the year
AgenTus Cell Therapy Business
IND filing for lead candidate in 2019
First Quarter 2018 Financial Results

Cash and cash equivalents were $52.3 million and $60.2 million at March 31, 2018 and December 31, 2017 respectively.

For the first quarter ended March 31, 2018, we reported research and development expenses of $29.4 million, and $32.6 million for the same period in 2017. Our net loss of for the three months ended March 31, 2018 is $54.3 million or $0.53 per share compared to a net loss for same period in 2017 of $17.1 million, or $0.18 per share. The increased net loss reflects unfavorable items effecting the current quarter and favorable items effecting the same period last year; including, the loss on the extinguishment of our debt, increased change in the non-cash contingent considerations fair value adjustment as well as reduced revenue due to an accelerated milestone received during the first quarter of 2017 from Incyte.

Conference Call, Webcast and Prepared Statement Information

Agenus executives will host a conference call on Monday, May 7, 2018 at 11:00 a.m. Eastern Time. To access the live call, dial (844) 492-3727 (domestic) and (412) 317-5118 (international). Ask to be joined into the Agenus call. The call will also be webcast and will be accessible from the Company’s website at View Source or via the following link: View Source A replay will be available on the Company’s website approximately two hours after the call and will remain available for 90 days.

Aeterna Zentaris Reports First Quarter 2018 Financial and Operating Results

On May 7, 2018 Aeterna Zentaris Inc. (NASDAQ:AEZS) (TSX:AEZS) reported financial and operating results for the first quarter ended March 31, 2018 (Press release, AEterna Zentaris, MAY 7, 2018, View Source [SID1234526158]).

All Amounts are in U.S. Dollars

Recent Key Developments

Continue to exceed terms of licensing and assignment agreement with Strongbridge Ireland Limited in support of its Macrilen (macimorelin) commercial launch in the U.S. targeted for summer of 2018
Diligently prepare European Medicines Agency (EMA) D120 regulatory response to Marketing Authorization Application (MAA) of macimorelin for anticipated submission in July 2018
Actively seeking out-licensing partners for macimorelin in Europe
Expanding resources to actively assist in identifying potential marketed products in the U.S. market
Financial condition and capital structure significantly improved
— Upfront payment of $24 million received from Strongbridge in January 2018;
— As of March 31, 2018, $24.5 million of unrestricted cash and cash equivalents;
— Approximately 16.4 million Common Shares outstanding as of March 31, 2018
Financial Highlights

Revenues $24.7 million

Research and Development ("R&D") Costs $0.8 million

General and Administrative ("G&A") Expenses $2.8 million

Selling Expenses $1.6 million

Net Finance Income $1.9 million

Income Taxes $6.9 million

Net Income $14.4 million

Working Capital $19.6 million
Commenting on recent key developments, Michael V. Ward, President and Chief Executive Officer for Aeterna Zentaris, stated, "We ended first quarter 2018 in the strongest financial position in the past decade. Our licensing agreement of Macrilen (macimorelin) in the U.S. and Canada with Strongbridge demonstrates the success of our development initiatives and better positions us to monetize our rights by licensing in territories outside of the United States and Canada."

First Quarter Highlights

Revenues

Licensing revenue was $24.6 million for the three months ended March 31, 2018, as compared to $0.1 million for the same period in 2017. The increase is primarily due to the recognition in January 2018, of the $24.0 million upfront payment received from Strongbridge for the license of Macrilen(macimorelin) as earned revenue in accordance with International Financial Reporting Standards (IFRS) 15, Revenue from Contracts with Customers as it is a "right to use" license. In addition, due to events that occurred in 2018, we consider our performance obligations under the Zoptrex licensing agreements to be fulfilled, therefore we recognized deferred revenues of $0.5 million relating to non-refundable upfront payments it previously received for licensing and technology transfer arrangements that it entered into with respect to the development of Zoptrex in various territories.

Sales commission and other were $90,000 for the three months ended March 31, 2018, compared to $153,000 for the same period in 2017. During 2018, we received a $90,000 termination agreement payment from our customer. In 2017, those revenues mainly resulted from our sales team exceeding pre-established unit sales baseline thresholds under our co-promotion agreement to sell Saizen. We also generated sales commission in connection with our promotion of APIFINY.

Operating Expenses

R&D costs were $0.8 million for the three months ended March 31, 2018, as compared to $2.5 million for the same period in 2017. The decrease in R&D costs is mainly attributable to lower comparative third-party costs.

Additionally, the decrease in our R&D costs for the three months ended March 31, 2018, as compared to the same period in 2017, is attributable to lower employee compensation and benefits costs, as well as lower facilities rent and maintenance costs. A substantial portion of this decrease is due to the realization of cost savings in connection with our ongoing efforts to streamline our R&D activities.

Third-party costs attributable to Zoptrex and Macrilen (macimorelin) decreased considerably during the three months ended March 31, 2018 as compared to March 31, 2017, mainly since we completed the clinical portion of the ZoptEC trial and the Macrilen (macimorelin) trial in 2017 and 2016, respectively. Third-party costs attributable to Macrilen (macimorelin) incurred in 2017 are related to the detailed analysis of the results as well as the preparation of the New Drug Application filing, which was submitted on June 30, 2017.

Excluding the impact of foreign exchange rate fluctuations, we expect that we will incur overall R&D costs of between $1.0 million and $2.0 million for the year ended December 31, 2018.

G&A expenses were $2.8 million for the three months ended March 31, 2018, as compared to $1.9 million for the same period in 2017. The increase is primarily related to fees associated with the Strongbridge License Agreement.

Excluding the impact of foreign exchange rate fluctuations and the recording of transaction costs related to potential financing activities (not currently known or estimable), we expect that G&A expenses will range between $8.0 million and $10.0 million in 2018.

Selling expenses were $1.6 million for the three months ended March 31, 2018, as compared to $1.5 million for the same period in 2017. Most of the selling expenses for the three months ended March 31, 2018 were for the payment of fees for the execution of the Strongbridge License Agreement. For the three months ended March 31, 2017, the costs were for our sales force co-promotion activities as well as our sales management team. Based on currently available information, we expect selling expenses to range between $1.8 million and $2.1 million in 2018.

Net finance income was $1.9 million for the three months ended March 31, 2018, as compared to $1.5 million, for the same period in 2017. The increase in finance income is mainly attributable to the change in fair value of warrant liability. Such change in fair value results from the periodic "mark-to-market" revaluation, via the application of pricing models, of outstanding share purchase warrants. The closing price of our common shares, which, on the NASDAQ, fluctuated from $1.46 to $2.41 during the three months ended March 31, 2018, compared to $2.45 to $3.65 during the same period in 2017, also had a direct impact on the change in fair value of warrant liability.

Net income for the three months ended March 31, 2018 was $14.4 million (or $0.88 per share), as compared to a net loss of $4.1 million (or $0.31 per share) for the same period in 2017. The increase in net income for the three months ended March 31, 2018 is a result of the revenue recognition of the $24.0 million upfront payment received for the Strongbridge License Agreement.

Liquidity, Cash Flows and Capital Resources

At March 31, 2018, we had $24.5 million of cash and cash equivalents. We expect existing cash balances and operating cash flows will provide us with adequate funds to support our current operating plan for at least the next twelve months and for the foreseeable future.

Conference Call

The Company will host a conference call to discuss these results on Tuesday, May 8, 2018, at 8:30 a.m., Eastern Time. Participants may access the conference call by telephone using the following dial-in numbers:

Toll-Free: 877-407-8029, Confirmation #13679691
Toll: 201-689-8029, Confirmation #13679691
A replay of the conference call will also be available on the Company’s website for a period of 30 days. For reference, the Management’s Discussion and Analysis of Financial Condition and Results of Operations for the first quarter 2018, as well as the Company’s audited consolidated financial statements as at March 31, 2018, 2017, 2016 and 2015, can be found at www.zentaris.com in the "Investors" section.