Stemline Therapeutics Reports Third Quarter 2017 Financial Results

On November 9, 2017 Stemline Therapeutics, Inc. (Nasdaq: STML), a clinical-stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, reported financial results for the quarter ended September 30, 2017 (Press release, Stemline Therapeutics, NOV 9, 2017, View Source [SID1234521904]). The Company also reviewed recent clinical and regulatory events, and outlined key upcoming milestones:

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Pivotal Trial of SL-401 In Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

· On October 31, 2017, we announced that the pivotal trial of SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN) met its primary endpoint.

· We anticipate that a Biologics License Application (BLA) submission could begin in the fourth quarter of 2017 or first quarter of 2018.

· We plan to have detailed data on this trial presented at the upcoming 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, being held December 9-12 in Atlanta, GA.

Additional Clinical Trials — SL-401, SL-801, SL-701

· SL-401 is also being evaluated in ongoing Phase 1/2 trials in additional indications including certain myeloproliferative neoplasms (MPN) (chronic myelomonocytic leukemia [CMML] and myelofibrosis [MF]), acute myeloid leukemia (AML), and multiple myeloma, as a single agent or in combination with other agents. We expect to provide updates on some of these studies at ASH (Free ASH Whitepaper) and on into next year.

· SL-801 Phase 1 results in patients with advanced solid tumors were the subject of a presentation at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Congress in September. Dose escalation is ongoing, and the trial is currently enrolling patients in the eighth dosing cohort.

· SL-701 has completed dosing in a Phase 2 trial of patients with second-line glioblastoma. We plan to provide an update on the trial at the upcoming Society for Neuro-Oncology (SNO) meeting in November.

Third Quarter 2017 Financial Results Review

Stemline ended the third quarter of 2017 with $79.9 million in cash, cash equivalents and investments, as compared to $93.2 million as of June 30, 2017, which reflects cash expenditures of $13.3 million for the quarter. The company ended the third quarter of 2017 with 25.3 million shares outstanding.

For the third quarter of 2017, Stemline had a net loss of $16.1 million, or $0.68 per share, compared with a net loss of $9.9 million, or $0.56 per share, for the same period in 2016.

Research and development expense was $12.4 million for the quarter ended September 30, 2017, compared with $7.2 million for the quarter ended September 30, 2016, representing an increase of $5.2 million. The higher costs are primarily driven by an increase of $2.4 million in manufacturing expenses to support our upcoming potential BLA filing for SL-401. We also incurred $2.1 million in higher regulatory costs to support our potential BLA filing for SL-401.

General and administrative expense was $4.2 million for the quarter ended September 30, 2017, compared with $3.2 million for the quarter ended September 30, 2016, representing an increase of $1.0 million. The increase in expense was primarily attributable to $0.6 million in higher legal expenses. Additionally, the higher costs were due to $0.2 million of pre-launch expenses in support of a potential commercialization of SL-401 in BPDCN, if marketing approval from the FDA is received.

Iovance Biotherapeutics Announces New LN-144 Phase 2 Clinical Data from Metastatic Melanoma Trial to be Presented at SITC Meeting

On November 9, 2017 Iovance Biotherapeutics, Inc. (NASDAQ:IOVA), a biotechnology company developing novel cancer immunotherapies based on tumor-infiltrating lymphocyte (TIL) technology, reported early efficacy and safety data from Cohort 2 of the ongoing Phase 2 LN-144 metastatic melanoma trial (C-144-01) (Press release, Iovance Biotherapeutics, NOV 9, 2017, View Source;p=RssLanding&cat=news&id=2315820 [SID1234521876]). These data, being presented as a late-breaking poster (Poster #515) on Friday, November 10, 2017 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 32nd Annual Meeting in National Harbor, Maryland, show that administration of the company’s Generation 2 (Gen 2) manufacturing process in nine efficacy-evaluable metastatic melanoma patients resulted in a disease control rate (DCR) of 78%, which includes 3 confirmed partial responders (PRs) and a fourth PR awaiting confirmation. These patients had four median prior therapies. The Gen 2 manufacturing process reduces the process time to 22 days and the cell product is cryopreserved for ease of administration and handling.

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"The new study findings presented at SITC (Free SITC Whitepaper) indicate encouraging preliminary efficacy from patients in Cohort 2 with advanced metastatic melanoma disease, who were treated with our cryopreserved TIL product LN-144," said Maria Fardis, PhD, MBA, Iovance Biotherapeutics President and Chief Executive Officer. "We are excited to be able to offer patients a TIL product manufactured with a significantly shorter process, minimizing the time a patient has to wait to receive their TIL. The cryopreservation of the product further offers the clinical sites flexibility of scheduling the patient dosing. The reduction in duration of manufacturing will reduce our manufacturing costs as well. We intend to make the decision regarding which manufacturing process to utilize for our ongoing and future clinical trials by year-end and in advance of initiation of our regulatory interactions."

In addition to the late-breaking poster, a poster will be presented on the new Gen 2 manufacturing protocol, highlighting key improvements around the current high throughput commercial manufacturing method (Poster #203). A third poster will demonstrate utility of a new reagent used for TIL expansion and effector function (Poster #357), and a fourth poster will provide data regarding investigation of key quality attributes of TIL product (Poster #194). The company will also present two posters in the Clinical Trials in Progress session that include the design of LN-145 in head and neck and cervical cancer. The details of the poster presentations are as follows:

Cellular Therapy Approaches Late-Breaking Abstract

Title: Novel Cryopreserved Tumor Infiltrating Lymphocytes (LN-144) Administered to Patients with Metastatic Melanoma Demonstrates Efficacy and Tolerability in a Multicenter Phase 2 Clinical Trial
Authors: Sarnaik, et al.
Poster #: 515
Presentation date: Friday, November 10, 2017

Key Takeaways:

Preliminary efficacy data from nine patients in Cohort 2 shows a DCR of 78% including three partial responses and a fourth awaiting confirmation.
Preliminary data to date show that the safety profile of Gen 2 LN-144 therapy is similar to Gen 1 LN-144 therapy previously presented at ASCO (Free ASCO Whitepaper) 2017. The most common adverse events reported were pyrexia, anaemia and nausea.
Cellular Therapy Approaches Abstracts

Title: A Cryopreserved TIL Product, LN-144, Generated with an Abbreviated Method Suitable for High Throughput Commercial Manufacturing Exhibits Favorable Quality Attributes for Adoptive Cell Transfer
Authors: Wardell, et al.
Poster #: 203
Presentation date: Friday, November 10, 2017

Key Takeaways:

The Iovance Generation 2 manufacturing process produces a potentially potent TIL product with comparable quality attributes to the Generation 1 manufacturing process.
The abbreviated 22-day expansion platform allows for the rapid generation of TIL product for patients in urgent need of therapy. The cryopreserved product also offers flexibility of patient dosing.
The cryopreserved drug product introduces critical logistical efficiencies allowing flexibility in distribution, and overcomes traditional barriers to the wider application of TIL therapy.
Title: Studies of Key Quality Attributes for TIL Product, LN-144
Authors: Ritthipichai, et al.
Poster #: 194
Presentation date: Saturday, November 11, 2017

Key Takeaways:

Commercial manufacturing of TIL products will require a robust analytical platform to ensure consistent delivery of products meeting the critical quality attributes of cellular therapeutics.
Study shows TIL products manufactured by Iovance are composed of greater than 99% CD45+CD3+ T cells.
Residual melanoma cells in TIL products were below the limit of detection using a specialized assay developed for this assessment.
Immune Modulation, Cytokines, and Antibodies Abstract

Title: The T-cell Growth Factor Cocktail IL-2/IL-15/IL-21 Enhances Expansion and Effector Function of
Tumor-Infiltrating T cells in a Novel Process Developed by Iovance
Authors: Simpson-Abelson, et al.
Poster #: 357
Presentation date: Friday, November 10, 2017

Key Takeaways:

This study demonstrates the positive effects of adding IL-15 and IL-21 to the standard IL-2-containing cell culture in an ex vivo TIL generation protocol.
Clinical Trials (In Progress) Abstracts

Title: A Phase 2 Study to Evaluate the Safety and Efficacy Using Autologous Tumor Infiltrating Lymphocytes (LN-145) in Patients with Recurrent and/or Metastatic Squamous Cell Carcinoma of the Head and Neck
Authors: Leidner, et al.
Poster #: 221
Presentation date: Friday, November 10, 2017

Title: A Phase 2, Multicenter Study to Evaluate the Efficacy and Safety Using Autologous Tumor Infiltrating Lymphocytes (LN-145) in Patients with Recurrent, Metastatic, or Persistent Cervical Carcinoma
Authors: Jazaeri, et al.
Poster #: 220
Presentation date: Saturday, November 11, 2017

Additional information including the presentation schedule and full abstracts can be found on the conference website: View Source

Forward-Looking Statements
Certain matters discussed in this press release are "forward-looking statements". The Company may, in some cases, use terms such as "predicts," "believes," "potential," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. In particular, the Company’s statements regarding trends and potential future results are examples of such forward-looking statements. The forward-looking statements include risks and uncertainties, including, but not limited to, the efficacy, safety, tolerability and cost of the Gen 2 manufacturing process, the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for its current product candidates, including statements regarding the timing of initiation and completion of the trials; the timing of and its ability to obtain and maintain U.S. Food and Drug Administration or other regulatory authority approval of, or other action with respect to, its product candidates; the strength of Company’s product pipeline; the successful implementation of the Company’s research and development programs and collaborations; the success of the Company’s license or development agreements; the acceptance by the market of the Company’s product candidates, if approved; and other factors, including general economic conditions and regulatory developments, not within the Company’s control. The factors discussed herein could cause actual results and developments to be materially different from those expressed in or implied by such statements. A further list and description of the Company’s risks, uncertainties and other factors can be found in the Company’s most recent Annual Report on Form 10-K and the Company’s subsequent filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov or www.iovance.com. The forward-looking statements are made only as of the date of this press release and the Company undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstance.

10-Q – Quarterly report [Sections 13 or 15(d)]

BioTime has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, BioTime, 2017, NOV 9, 2017, View Source [SID1234521845]).

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10-Q – Quarterly report [Sections 13 or 15(d)]

Genomic Health has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Genomic Health, 2017, NOV 9, 2017, View Source [SID1234521911]).

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Fortress Biotech Reports Third Quarter 2017 Financial Results and Recent Corporate Highlights

On November 9, 2017 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products, reported financial results and recent corporate highlights for the third quarter ended September 30, 2017 (Press release, Fortress Biotech, NOV 9, 2017, View Source [SID1234521867]).

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Dr. Lindsay A. Rosenwald, Fortress’ Chairman, President and Chief Executive Officer, said, "Fortress enjoyed strong third quarter performance, beginning with the formation of a new gene therapy subsidiary company, Aevitas Therapeutics. Fortress’ other subsidiaries also continued to achieve important corporate and clinical milestones during the quarter. Avenue dosed the first patient in its pivotal Phase 3 study of IV tramadol for the management of moderate to moderately severe pain in patients following bunionectomy surgery. Additionally, Checkpoint dosed the first patient in its Phase 1 clinical study evaluating the safety and tolerability of its anti-PD-L1 antibody, CK-301, in selected recurrent or metastatic cancers. The FDA also granted Orphan Drug Designation to Checkpoint’s third-generation EGFR inhibitor, CK-101, for the treatment of EGFR mutation-positive NSCLC. Mustang Bio has continued to make meaningful progress throughout the quarter, with its common stock commencing trading on the NASDAQ Global Market, and the licensing of a CD20 CAR T cell therapy from the Fred Hutchinson Cancer Research Center, expanding its pipeline to six novel CAR T candidates."

Dr. Rosenwald continued, "We look forward to oral data presentations on both Mustang Bio’s and Caelum Biosciences’ clinical programs at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December, and plan to continue delivering on meaningful milestones for the remainder of the year."

Financial Results:

· As of September 30, 2017, Fortress’ consolidated cash, cash equivalents and short-term investments totaled $154.6 million, compared to $88.3 million at December 31, 2016, an increase of $66.3 million year-to-date. The September 30, 2017 consolidated cash, cash equivalents and short-term investments total excludes restricted cash of $16.9 million and cash deposits with clearing organizations of $1.0 million.
· Net revenue totaled $46.9 million for the third quarter of 2017 and $142.3 million for the first nine months of 2017, compared to $1.0 million for the third quarter of 2016 and $3.9 million for the first nine months of 2016. Net total revenue for the third quarter ending September 30, 2017 includes $2.5 million of Fortress revenue and $44.4 million of revenue from National Holdings Corporation ("National"), which Fortress acquired in September 2016, with no revenue attributable to National prior to the acquisition.

· Research and development expenses were $15.9 million for the third quarter of 2017, of which $14.2 million was related to Fortress Companies, and $34.7 million for the first nine months of 2017, of which $29.1 million was related to Fortress Companies. This compares to $7.3 million for the third quarter of 2016, of which $5.5 million was related to Fortress Companies, and $21.4 million for the first nine months of 2016, of which $15.8 million was related to Fortress Companies. Non-cash stock-based compensation expense included in research and development for the third quarter of 2017 was $1.6 million, compared to $0.9 million for the third quarter of 2016, and $4.8 million for the first nine months of 2017, compared to $3.4 million for the first nine months of 2016.
· Research and development expenses from license acquisitions totaled $0.3 million for the third quarter of 2017 and $3.4 million for the first nine months of 2017, compared to $1.0 million for the third quarter of 2016 and $3.1 million for the first nine months of 2016.
· General and administrative expenses were $15.1 million for the third quarter of 2017, of which $10.9 million was related to Fortress Companies, and $36.5 million for the first nine months of 2017, of which $23.8 million was related to Fortress Companies. This compares to $8.9 million for the third quarter of 2016, of which $3.8 million was related to Fortress Companies, and $25.4 million for the first nine months of 2016, of which $11.5 million was related to Fortress Companies. Non-cash stock-based compensation expenses included in general and administrative expenses were $2.6 million for the third quarter of 2017, compared to $2.0 million for the third quarter of 2016, and $6.9 million for the first nine months of 2017, compared to $5.4 million for the first nine months of 2016.
· National’s operating expenses totaled $47.7 million for the third quarter of 2017 and $139.2 million for the first nine months of 2017, with no expenses attributable to National prior to Fortress’ acquisition of the company in September 2016.
· Net loss attributable to common stockholders was $27.1 million, or $0.67 per share, for the third quarter of 2017, compared to a net loss attributable to common stockholders of $13.0 million, or $0.32 per share, for the third quarter of 2016. For the first nine months of 2017, net loss attributable to common stockholders was $56.5 million or $1.39 per share, compared to a net loss attributable to common stockholders of $37.7 million or $0.94 per share in the first nine months of 2016.