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Audentes Therapeutics Reports Fourth Quarter 2017 and Full Year Financial Results and Provides Corporate Update

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.

Stemline Therapeutics to Present at Upcoming Investor Conferences

On March 8, 2018 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, reported that Ivan Bergstein, M.D., Stemline’s CEO, will present at two upcoming investor conferences (Press release, Stemline Therapeutics, MAR 8, 2018, View Source [SID1234524545]):

The 30th Annual ROTH Conference on Monday, March 12, at 4:00 PM PT (7:00 PM ET), which is being held at The Ritz-Carlton, Orange County in Dana Point, California.

The Cowen and Company 38th Annual Health Care Conference 2018 on Wednesday, March 14, at 8:00 AM ET, which is being held at the Boston Marriott Copley Place in Boston, MA.
A live webcast of each presentation can be viewed on the company’s website at www.stemline.com.

Onconova Therapeutics, Inc. Reports Business Highlights and Full Year 2017 Financial Results

On March 8, 2018 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS), today provided a corporate update and reported financial results for the full year ended December 31, 2017 (Press release, Onconova, MAR 8, 2018, View Source [SID1234524544]).

"The recently completed year was a pivotal period for rigosertib development programs in MDS. We achieved important goals across our full pipeline, highlighted by the recently announced promising interim analysis and the advancement of the INSPIRE pivotal trial for rigosertib, our lead Phase 3 clinical candidate. With no FDA approved therapies available for patients with higher-risk MDS who are refractory to hypomethylating agents, Onconova has taken a leadership position in this indication. Looking ahead, we now expect topline analysis to be concurrent with enrollment completion, which can be achieved in the first half of 2019," said Dr. Ramesh Kumar, President and Chief Executive Officer.

"We also announced three important collaborations in recent months. Regional licensing of our pre-IND stage next generation CDK 4/6 inhibitor for Greater China which we believe advnaces this program on the IND track and towards clinical data in 2019. Our collaboration with the National Cancer Institute for clinical development of rigosertib in children suffering from incurable inherited diseases (RASopathies) could provide Onconova with the opportunity to establish a rare disease development program. Finally, our licensing agreement for rigosertib in Latin America further expands the global commercial footprint of rigosertib, and is in addition to our existing partnership in Japan and Korea. Execution of these transactions we believe indicates the ability to leverage our late stage and pipeline assets to finance multiple programs. Based on the progress achieved in our oral rigosertib-azacitidine combination Phase 2 program in front-line MDS indications, we expect to secure additional collaborations and regional partnerships to help support a pivotal Phase 3 trial for oral rigosertib."

INSPIRE Trial of IV Rigosertib in 2nd Line Higher-risk (HR) MDS

Interim Analysis (IA)

On January 17, 2018, Onconova announced that it is moving forward with its Phase 3 INSPIRE pivotal trial following the interim analysis and the Data Monitoring Committee’s (DMC) recommendation, together with unanimous approval by the Executive Committee overseeing this trial. The DMC recommended continuation of the trial with a one-time expansion in enrollment, using a pre-planned sample size re-estimation, consistent with the Statistical Analysis Plan.
The expanded INSPIRE study will increase enrollment by adding 135 patients to the original target to reach a total enrollment of 360 patients.
At the topline analysis of the INSPIRE trial, the primary endpoint of overall survival will be analyzed in both the ITT population and the Very High Risk (VHR) subgroup.
In the INSPIRE trial enrollment so far, the predefined subgroup of VHR patients constitutes greater than 70% of patients enrolled to date.
The Company remains blinded to the interim analysis results.
Trial Progress

The INSPIRE study is open in more than 170 sites in 22 countries across four continents.
More than half of the expanded study is now enrolled.
The Company is planning to add sites in Europe and new territories, including in Latin America, in concert with our new partner Pint Pharma ("Pint").
The INSPIRE trial was designed with stringent selection criteria so as to identify a more homogenous MDS patient population. Accordingly, extensive eligibility verification and trial site education are integral to the Company’s plan.
Oral Rigosertib in Combination with Azacitidine for 1st-line HR-MDS

Pivotal Phase 3 Trial Protocol

Phase 2 Expansion Trial is expected be fully enrolled this month with the addition of more than 40 patients.
Onconova plans to present initial data from this study at a scientific conference in 2018, highlighting the results of dose selection and optimization of the combination regimen.
On March 2, 2018, Onconova presented data relating to the mechanism of action of rigosertib in combination with azacitidine at the AACR (Free AACR Whitepaper) Special Conference. The results suggested potential novel clinical strategies to improve outcomes for patients with higher-risk MDS and reversal of resistance to treatment with epigenetic therapies.
Progress in Business Development around Rigosertib and Pipeline Products

Onconova and Pint Pharmaceutical Announce Licensing Agreement for Rigosertib in Latin America

On March 5, 2018, Onconova and Pint announced that they had entered into a Latin American licensing agreement for rigosertib. Pint is a private, European-based pharmaceutical company focused on the development, registration and commercialization of specialty-based treatments for the Latin American market.
Under the terms of the agreement, Pint will make an investment in Onconova totaling up to $2.5 million by purchasing shares at a premium to market. In addition, Pint will make potential additional regulatory, development and sales-based milestone payments to Onconova of up to $42.75 million and pay double digit tiered royalties on net sales in Latin America.
Rigosertib Collaboration for Pediatric RASopathies

On January 4, 2018, Onconova announced that it had entered into a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), part of the National Institutes of Health. Under the terms of the CRADA, the NCI will conduct research, including preclinical laboratory studies and a clinical trial, on rigosertib in pediatric cancer associated RASopathies. The RASopathies are a group of rare diseases which share a well-defined molecular basis in expression or defects involving Ras Effector Pathways.
License and Collaborative Development Agreement with HanX Biopharmaceuticals for ON 123300

On December 19, 2017, Onconova announced the signing of a license and collaboration agreement with HanX Biopharmaceuticals, Inc., a company focused on development of novel oncology products, for the further development, registration and commercialization of ON 123300 in China. ON 123300 is a first-in-class dual inhibitor of CDK4/6 + ARK5, which is currently in advanced pre-clinical development. This compound has the potential to overcome the limitations of current generation CDK 4/6 inhibitors.
Under the terms of the agreement, Onconova will receive an upfront payment, and is eligible to receive potential regulatory and commercial milestone payments, as well as royalties on Chinese sales. HanX will provide all funding required for Chinese IND enabling studies performed for Chinese Food and Drug Administration IND approval. The Companies also intend for these studies to comply with US Food and Drug Administration (FDA) standards. Accordingly, such studies may be used by Onconova for an IND filing with the FDA. Onconova will maintain global rights outside of China.
Pre-clinical Stage CDK4/6 + ARK5 Inhibitor Program

Following signing of the collaboration agreement with HanX, Onconova initiated a pre-IND process with the U.S. Food and Drug Administration (FDA).

Presentations of Data

Rigosertib in MDS at the ASH (Free ASH Whitepaper) 2017 Meeting

Onconova delivered two poster presentations highlighting drug activity and the mechanism of action of rigosertib in MDS during the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Atlanta in December, 2017.
Among the highlights of the presentation were: Oral rigosertib as a single agent demonstrated activity in a Phase 2 trial for lower-risk MDS; 32% of 62 evaluable patients, and 44% of patients receiving optimal dosing, achieved transfusion independence; and new data on the molecular basis of the combination therapy with rigosertib and azacitidine in epigenetic studies in patient derived stem cells.
Full Year 2017 Financial Results:

Cash and cash equivalents as of December 31, 2017, totaled $4.0 million, compared to $21.4 million as of December 31, 2016. Subsequently, on February 12, 2018, Onconova announced the closing of a $10 million underwritten public offering of 9,947,500 shares of common stock or common stock equivalents and warrants to purchase an aggregate of 994,750 shares of Onconova’s Series A convertible preferred stock, including the exercise in full of the underwriter’s option to purchase additional securities, at the public offering price of $1.01 per share and accompanying Preferred Stock Warrant. Onconova also issued to the underwriter a preferred stock warrant to purchase 49,737.5 shares of Series A convertible preferred stock. Based on the Company’s cash burn for 2017 and its current projections, Onconova expects that cash and cash equivalents will be sufficient to fund ongoing trials and operations into the third quarter of 2018.
Net loss was $24.1 million for the year ended December 31, 2017, compared to $19.7 million for the year ended December 31, 2016, primarily due to the lack of collaboration cost sharing revenue in the 2017 period and a smaller change in fair value of warrant liability in the 2017 period.
Research and development expenses were $19.1 million for the year ended December 31, 2017, and $20.1 million for the comparable period in 2016.
General and administrative expenses were $7.4 million for the year ended December 31, 2017, and $9.2 million for comparable period in 2016.
The Company will host a conference call on March 8th at 9:00 a.m. Eastern Time to provide a corporate update and discuss fourth quarter and full-year financial results. Interested parties may access the call by dialing toll-free (855) 428-5741 from the US, or (210) 229-8823 internationally and using conference ID: 2947108.

The call will also be webcast live. Please click here to access the webcast.

A replay will be available at this link until June 29, 2018

Molecular Templates to Present at the Cowen and Company 38th Annual Health Care Conference

On March 8, 2018 Molecular Templates, Inc., (Nasdaq:MTEM) a clinical stage biopharmaceutical company focused on the discovery and development of Engineered Toxin Bodies (ETBs), a new class of targeted biologic therapies that possess unique mechanisms of action in oncology, reported that its management will provide a corporate overview at the Cowen and Company 38th Annual Health Care Conference, taking place March 12-14 at the Boston Marriott Copley Place in Boston, MA (Press release, Molecular Templates, MAR 8, 2018, http://ir.mtem.com/news-releases/news-release-details/molecular-templates-present-cowen-and-company-38th-annual-health [SID1234524543]).

Presentation Details
Date: Tuesday, March 13
Time: 11:20am Eastern Time
Location: The Boston Marriott Copley Place
Webcast: http://wsw.com/webcast/cowen46/mtem/

TG Therapeutics, Inc. Announces Fourth Quarter and Year-End 2017 Financial Results and Business Update

On March 8, 2018 TG Therapeutics, Inc. (NASDAQ: TGTX) reported its financial results for the fourth quarter and year ended December 31, 2017 and provided recent company developments along with a business outlook for 2018 (Press release, TG Therapeutics, MAR 8, 2018, View Source [SID1234524542]).

Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "2017 was a truly pivotal year for our company with the presentation of positive results from our first Phase 3 trial, the GENUINE trial, the completion of enrollment in our second Phase 3 trial, the UNITY-CLL trial, and the launch of our Phase 3 ULTIMATE trials in Multiple Sclerosis. These major milestones along with strong enrollment in our UNITY-NHL registration directed program sets the stage for an even more impactful 2018 and 2019." Mr. Weiss continued, "We are extremely pleased to enter 2018 with a healthy balance sheet and look forward to multiple value creating milestones this year, including presenting topline ORR data from our Phase 3 UNITY-CLL trial and, ideally, filing our first BLA and/or NDA later in the year."

2017 Highlights

● Orphan Drug Designation: Orphan drug designation was granted for the combination of ublituximab (TG-1101) and umbralisib (TGR-1202) for the treatment of Chronic Lymphocytic Leukemia (CLL) and Diffuse Large B-Cell Lymphoma (DLBCL).
● Ublituximab Publication: Data from the Phase 1/2 trial of ublituximab was published in the British Journal of Haematology.
● GENUINE Phase 3 Data: Positive data from the Phase 3 GENUINE trial of ublituximab in combination with ibrutinib in patients with high risk CLL was presented at the ASCO (Free ASCO Whitepaper) annual meeting and Lugano Lymphoma Conference.
● ULTIMATE Phase 3 Trials in MS: Received a Special Protocol Assessment (SPA) for the Phase 3 ULTIMATE I and II studies in relapsing forms of multiple sclerosis, commenced enrollment into the global studies and presented clinical and MRI data from the supportive Phase 2 trial of ublituximab in RMS at various conferences throughout the year.
● UNITY-CLL Phase 3 Enrollment: Full enrollment in the UNITY-CLL Phase 3 Trial was completed in October 2017
● Umbralisib Grant: Umbralisib was selected for a grant by the National Multiple Sclerosis Society to support the development of umbralisib as an oral B-Cell targeted treatment option in progressive Multiple Sclerosis (PMS).
● Anti-PD-L1 Entered the Clinic: The Company advanced its anti-PD-L1 monoclonal antibody into clinical development, with the first patient being dosed in a Phase I clinical trial.

Key Objectives for 2018

● UNITY-CLL Phase 3 Trial: We plan to present top-line overall response rate results from our UNITY-CLL Phase 3 trial in both front line and relapsed or refractory CLL and also prepare a BLA/NDA filing for accelerated approval.
● GENUINE Phase 3 BLA Filing: A Biologics Licensing Application (BLA) filing will be prepared for ublituximab plus ibrutinib for potential accelerated approval based on the results from GENUINE.
● UNITY-NHL: Aggressively enroll into all cohorts of the UNITY-NHL clinical trial with the goal of completing enrollment into all cohorts.
● ULTIMATE Phase 3 Trials: Aggressively recruit to our Phase 3 MS trials.
● Data Presentations: Present new and updated data from ongoing trials at various scientific meetings throughout the year.

Financial Results for the Fourth Quarter and Full Year 2017

● Cash Position: Cash, cash equivalents, investment securities, and interest receivable were $84.8 million as of December 31, 2017. Pro-forma cash, cash equivalents, investment securities, and interest receivable as of December 31, 2017 (excluding our first quarter 2018 operations) are approximately $114.6 million, after giving effect to $29.8 million of net proceeds from the utilization of the Company’s at-the-market ("ATM") sales facility during the first quarter of 2018.

● R&D Expenses: Research and development (R&D) expenses were $26.0 million and $102.5 million for the three and twelve months ended December 31, 2017, respectively, compared to $22.3 million and $69.2 million for the three and twelve months ended December 31, 2016, respectively. Included in research and development expenses for the three and twelve months ended December 31, 2017, in addition to our other clinical expenses, are $6.1 million and $26.6 million, respectively, of manufacturing and CMC expenses for Phase 3 clinical trials and potential commercialization. The increase in R&D expenses for both the three and twelve months ended December 31, 2017, is primarily due to the ongoing clinical development programs and related manufacturing costs for TG-1101 and TGR-1202.

● G&A Expenses: General and administrative (G&A) expenses were $5.1 million and $16.3 million for the three and twelve months ended December 31, 2017, respectively, as compared to $1.8 million and $9.9 million for the three and twelve months ended December 31, 2016, respectively. The increase in G&A expenses for the three and twelve months ended December 31, 2017 relates primarily to non-cash compensation expenses related to equity incentive expense recognized during 2017.

● Net Loss: Net loss was $30.9 million and $118.5 million for the three and twelve months ended December 31, 2017, respectively, compared to a net loss of $23.7 million and $78.3 million for the three and twelve months ended December 31, 2016, respectively.

● Financial Guidance: The Company believes its cash, cash equivalents, investment securities, and interest receivable on hand as of December 31, 2017, inclusive of the proceeds raised subsequent to the year-end will be sufficient to fund the Company’s planned operations through mid-2019.

Conference Call Information

The Company will host an investor conference call today, Thursday, March 8, 2018 at 8:30am ET, to discuss the Company’s 2017 financial results and provide a business outlook for 2018.

In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Year-End 2017 Earnings Call. A live audio webcast of this conference call will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com. An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call