On March 8, 2018 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, reported its financial results for the fourth quarter and full year ended December 31, 2017 and provided an update on the company’s recent achievements and anticipated upcoming milestones (Press release, Audentes Therapeutics, MAR 8, 2018, View Source;p=RssLanding&cat=news&id=2337152 [SID1234524554]).

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"2017 was a transformational year marked by significant achievements across our portfolio of gene therapy product candidates," stated Matthew R. Patterson, President and Chief Executive Officer. "Among our many accomplishments, we are particularly pleased to have recently reported positive interim data from the first dose cohort of patients in ASPIRO, the Phase 1 / 2 clinical study of our product candidate AT132 to treat XLMTM. These promising initial data add to the growing body of evidence of the utility of AAV-based gene therapy to treat rare neuromuscular diseases, and we are optimistic that they signal a turning point in our ability to bring meaningful benefit to patients and families living with XLMTM."

Mr. Patterson continued, "As we look forward to the year ahead, we are excited by our many anticipated upcoming pipeline catalysts, including sharing additional data from our ASPIRO study in XLMTM, new data from our VALENS study in Crigler-Najjar, and advancing additional product candidates into the clinic for Pompe disease and CASQ2-CPVT. With our recently completed follow-on offering, we are well capitalized to continue our mission to develop transformative gene therapy products for patients living with serious, life-threatening rare diseases."

Recent Achievements & Upcoming Key Events

AT132 for X-Linked Myotubular Myopathy (XLMTM):
Announced positive interim data in the first three patients dosed in ASPIRO, the Phase 1 / 2 clinical study of AT132 to treat XLMTM. Plan to dose three additional patients in the first cohort of ASPIRO and to report additional interim data in the second quarter of 2018
Received Rare Pediatric Disease and Fast Track designations for AT132 from FDA
AT342 for Crigler-Najjar Syndrome:
Initiated dosing in the first cohort of patients in VALENS, the Phase 1 / 2 clinical study of AT342 to treat Crigler-Najjar Syndrome, in the first quarter of 2018 and plan to report preliminary data from VALENS in the second quarter of 2018
Received Rare Pediatric Disease and Fast Track designations for AT342 from FDA
AT982 for Pompe Disease:
Announced selection of the clinical development candidate, AT982, a novel AAV8 vector designed to express GAA in tissues relevant to Pompe disease, including skeletal muscle, the heart and the nervous system, and to reduce immunogenicity, thereby addressing the key limitations of existing ERT therapy for Pompe
Plan to file IND in mid-2018 and to initiate a Phase 1 / 2 clinical study in the fourth quarter of 2018
AT307 for CASQ2-CPVT:
Completed IND-enabling preclinical studies of AT307
Plan to file IND in the first quarter of 2018 and to initiate a Phase 1 / 2 clinical study in the fourth quarter of 2018
Initiated activities to identify CASQ2-CPVT patients and further characterize the disease burden and unmet medical need for patients living with CPVT
Manufacturing:
Completed manufacturing campaigns of all product candidates in our state-of-the-art, 2x500L bioreactor-scale cGMP facility, designed to support global commercial licensure
Continued optimization of our serum-free suspension mammalian cell culture manufacturing process, which we expect will support commercial production for all of our product candidates
Built a world-class scientific team to support manufacturing and development operations, including expertise in novel product design, process and assay development, and AAV immunology
Fourth Quarter and Full Year 2017 Financial Results

Cash Position: At December 31, 2017, Audentes had cash, cash equivalents, and short-term investments of $133.6 million. In January 2018, Audentes further strengthened its balance sheet with the completion of a follow-on financing, issuing 6,612,500 shares of common stock at an offering price of $35.00 per share, resulting in net proceeds of approximately $217.2 million after the deduction of underwriting discounts, commissions and estimated offering expenses. Current cash, cash equivalents and short-term investments are planned to fund operations into the second half of 2020.
Research and Development Expenses: Research and development expenses were $21.7 million for the fourth quarter of 2017 and $75.9 million for the year ended December 31, 2017, compared to $16.6 million and $48.8 million, respectively, for the same periods in 2016. The increase in research and development expenses was primarily attributable to an increase in research and development headcount and related facility costs, increased internal manufacturing costs, an increase in preclinical and clinical study costs for our development programs, and an increase in expenses related to expanded internal research activities.
General and Administrative Expenses: General and administrative expenses were $5.2 million for the fourth quarter of 2017 and $17.3 million for the year ended December 31, 2017, compared to $3.2 million and $11.3 million, respectively, for the same periods in 2016. The increase in general and administrative expenses was primarily attributable to increased general and administrative headcount and related facility costs, increased professional service and audit fees, and higher insurance costs.
Net Loss: Net loss was $24.4 million for the fourth quarter of 2017 and $90.2 million for the year ended December 31, 2017, compared to $19.7 million and $59.7 million, respectively, for the same periods in 2016.
Conference Call
At 4:30 p.m. Eastern Time today, Audentes management will host a conference call and a simultaneous webcast to discuss its fourth quarter 2017 financial results and provide a corporate update. To access a live webcast of the conference call, please visit the Events & Presentations page within the Investors + Media section of the Audentes website at www.audentestx.com. Alternatively, please call 1-833-659-8620 (U.S.) or 1-409-767-9247 (international) and dial the conference ID 5890848 to access the call.

A replay of the webcast will be available on the Audentes website for approximately 30 days.

Progenics Pharmaceuticals has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, Progenics Pharmaceuticals, 2018, MAR 8, 2018, View Source [SID1234524589]).

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On March 8, 2018 Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical-stage pharmaceutical company focused on discovering and developing novel small molecule drugs directed against tumor metabolism and tumor immunology targets for the treatment of cancer, reported its financial results for the fourth quarter and year ended December 31, 2017 (Press release, Calithera Biosciences, MAR 8, 2018, View Source [SID1234524555]). As of December 31, 2017, cash, cash equivalents and investments totaled $186.2 million.

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"2017 was a transformative year for Calithera as we advanced each of our internally discovered first-in-class, small molecule onco-metabolism clinical candidates into broad clinical programs and announced a partnership with Incyte." said Susan Molineaux, PhD, President and Chief Executive Officer of Calithera. "In 2018, we will be enrolling two randomized placebo-controlled trials of our oral glutaminase inhibitor for the treatment of patients with renal cell carcinoma and a Phase 2 trial for the treatment of patients with triple negative breast cancer. INCB001158, an inhibitor of arginase, will be in evaluated in three broad clinical trials for the treatment of patients with solid tumors in combination with a PD-1 inhibitor, chemotherapy, and epacadostat/PD-1 inhibitor, respectively."

Fourth Quarter 2017 and Recent Highlights

CB-839

• Presented Results of CB-839 in Combination with Cabozantinib in Renal Cell Carcinoma; Randomized Phase 2 Trial Planned. In February 2018, we presented preliminary results of the Phase Ib trial of CB-839 in combination with cabozantinib, an oral tyrosine kinase inhibitor at the 2018 Genitourinary Cancer Symposium. Preliminary results showed the combination demonstrated 40% overall response rate in advanced clear cell RCC patients, and 100% disease control with the safety profile of CB-839 plus cabozantinib generally consistent with that of cabozantinib monotherapy. On the basis of this efficacy and safety data, we plan to initiate a randomized double-blind placebo controlled trial in approximate 300 clear cell renal cell carcinoma patients who have previously received one or two prior lines of therapy. The Phase 2 trial, known as CANTATA, is planned to begin in the second quarter of 2018 and it is expected to take approximately two years to reach the primary endpoint analysis of progression free survival.

• Presented Results of CB-839 in Combination with Nivolumab. In November 2017, we announced initial data from the ongoing trial of CB-839 in combination with nivolumab, in patients with melanoma, renal cell carcinoma and non-small cell lung cancer. Responses were observed in three melanoma patients who were progressing on a checkpoint inhibitor at study entry, and disease stabilization was observed in patients with non-small cell lung cancer and renal cell carcinoma patients that had disease progression on a checkpoint inhibitor immediately prior to starting the CB-839/nivolumab combination. The collaboration with Bristol-Myers Squibb was expanded, and a joint development committee was established to guide the development and regulatory strategy.

• Presented Results of CB-839 in Combination with Paclitaxel for the Treatment of Triple Negative Breast Cancer. In December 2017, we presented updated clinical data from a Phase 2 expansion cohort of patients with triple negative breast cancer receiving CB-839 in combination with paclitaxel. Among all evaluable patients treated with CB-839 doses of at least 600 mg bid (n=37), there were 8 partial responses (22%) and disease control in 22 patients (59%). Exploratory biomarker analysis showed a trend for the strongest clinical benefit occurring in patients with desmoplastic stromal gene expression signatures. We plan to present an update on our TNBC development program in the fourth quarter of 2018.
INCB001158

• INCB001158 Initiated Combination Dosing. In October 2017, the first patient was treated in the Phase I cohort of INCB001158 dosed in combination with Keytruda (pembrolizumab), an anti-PD1 immune checkpoint inhibitor. INCB001158 is currently being evaluated in two Phase 1/2 clinical trials and a third trial is expected to begin in the first half of 2018.
Corporate

• Augmented Board of Directors and Management Team. In September 2017, Calithera appointed Blake Wise, President and Chief Operating Officer of Achaogen, to the company’s Board of Directors, and Sumita Ray as General Counsel.
Selected Fourth Quarter 2017 Financial Results

Cash, cash equivalents and investments totaled $186.2 million at December 31, 2017.

Collaboration revenue for the full year 2017 was $26.0 million, compared with zero in the prior year, and represents the portion of deferred revenue recognized from the Company’s collaboration and license agreement with Incyte. Collaboration revenue for the fourth quarter of 2017 was $7.3 million.

Research and development expenses for the full year 2017 were $43.1 million, compared with $27.7 million in the prior year. The increase of $15.4 million in 2017 was due to an increase in the CB-839 program to support our new and ongoing clinical trials, including our two Phase 2 trials, as well as investment in our early stage research programs, offset by a decrease in the INCB001158 program, primarily due to Incyte’s co-funding of development costs. Research and development expenses for the fourth quarter of 2017 were $15.5 million, compared to $6.6 million for the same period last year.

General and administrative expenses for the full year 2017 were $12.5 million, compared with $10.6 million in the prior year. The increase of $1.9 million in 2017 was primarily due an increase in professional services, including activities to support our collaboration and license agreements and Phase 2 clinical trials, and higher personnel-related costs. General and administrative expenses for the fourth quarter of 2017 were $3.3 million, compared to $3.0 million for the same period last year.

Net loss from operations for the three months and year ended December 31, 2017 was $11.0 million and $27.8 million, respectively.

Financial Guidance for 2018

Calithera expects its cash, cash equivalents and investments will be between $105 and $115 million at the end of 2018, and be sufficient to meet its current operating plan through 2020, exclusive of any new collaborations or partnerships, milestone payments, additional equity financings or other new sources.

Conference Call Information

Calithera will webcast a clinical update on CB-839 on Thursday, March 8th at 4:30 p.m. Eastern Time/ 1:30 p.m. Pacific Time. The call may be accessed by dialing (855) 783-2599 (domestic) or (631) 485-4877 (international), and referring to conference ID 3398144. To access the live audio webcast or the subsequent archived recording, visit the Investors section of the Calithera website at www.calithera.com. The webcast will be recorded and available for replay on Calithera’s website for 30 days.

On March 8, 2018 Affibody AB ("Affibody"), a clinical stage biopharmaceutical company developing a portfolio of innovative drug projects, reported the outcome of the rights issue that was decided at the EGM on November 23, 2017 (Press release, Affibody, MAR 8, 2018, View Source [SID1234575704]).

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The results from the rights issue was that all 3,691,905 share were subscribed by existing shareholders.

"We are very pleased to announce that the rights issue that was launched late last year has been completed. This funding is intended to enable us to effectively execute on our science driven experimental medicine model and reach several valuable milestones in the next twelve to eighteen months. Specifically, it will enable us to rapidly expand our clinical programs. We will also continue to systematically build our company based on the development of multiple new products from our innovative proprietary platform", said David Bejker, CEO of Affibody. "The support that our shareholders have extended us through this issue is a strong endorsement of our emerging drug pipeline, and we look forward to communicating the advancement of our expanding product portfolio throughout 2018."

Through the rights issue Affibody received SEK 199.4 million in total and the share capital is increased by a total of SEK 18,459,525 to SEK 86,144,480. The total number of shares in Affibody Medical AB is increased by 3,691,905 to 17,228,896.

DURECT has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, DURECT, 2018, MAR 8, 2018, View Source [SID1234524597]).

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