NY-ESO Data Presented at the Connective Tissue Oncology Society (CTOS) Annual Meeting Confirm Potential of Adaptimmune’s SPEAR T-Cell Therapy

On November 9, 2017 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, released updated data from the ongoing pilot study of NY‑ESO SPEAR T-cells in synovial sarcoma, as well as an overview of study design for the ongoing NY-ESO SPEAR T-cell trial in myxoid/ round cell liposarcoma (MRCLS) at the annual CTOS meeting at the Grand Wailea Resort in Hawaii (Press release, Adaptimmune, NOV 9, 2017, View Source;p=RssLanding&cat=news&id=2315812 [SID1234521851]).

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"The data from our ongoing pilot study in synovial sarcoma remain encouraging," said Rafael Amado, Adaptimmune’s Chief Medical Officer. "GSK exercised its option over our NY-ESO program and, as a result, these studies, including the pivotal registration trial in synovial sarcoma, will transition to GSK. The synovial sarcoma data, as well as results from other ongoing studies in the NY-ESO program, continue to inform development plans with our wholly owned pipeline of products. We believe the efficacy we have seen in synovial sarcoma is indicative of the potential of our SPEAR T-cell platform."

Data update from the ongoing pilot study of NY-ESO SPEAR T-cells in synovial sarcoma1
During an oral presentation, Dr. Sandra P. D’Angelo of the Memorial Sloan Kettering Cancer Center presented an update on all cohorts from Adaptimmune’s ongoing pilot study of NY-ESO SPEAR T-cells in synovial sarcoma. The data cut-off for this oral presentation was September 5, 2017 and results are summarized below.

NY-ESO SPEAR T-cells continue to be generally well-tolerated with initial efficacy observed in all cohorts including low expressors of NY‑ESO (Cohort 2)
Of the twelve patients treated in Cohort 1 (non-modified fludarabine (Flu) / cyclophosphamide (Cy) lymphodepletion regimen), five remain alive with a median predicted overall survival of 120 weeks (~28 months)
Confirmed responses have been observed across all cohorts as follows:
Cohort 1 (follow-up only; High Flu/Cy, High NY-ESO): 6 /12 (50%) patients (unchanged from ASCO (Free ASCO Whitepaper) 2017)2
Cohort 2 (ongoing; High Flu/Cy, Low NY-ESO): 3/10 (33%) patients (ASCO 2017: 2/5 [40%])
Cohort 3 (follow-up only): 1/5 (20%) patients (unchanged from ASCO (Free ASCO Whitepaper) 2017)
Cohort 4 (ongoing): 4/11 (36%) patients (ASCO 2017: 3/6 [50%]). Overall survival is not mature in this cohort; progression free survival is 23 weeks.
Peak and long-term expansion of NY-ESO SPEAR T-cells appears to correlate with clinical efficacy
All reported events of cytokine release syndrome resolved with supportive care, the majority of events were Grade 1 or 2, and there were no events of seizure, cerebral edema or encephalopathy
1 Oral presentation entitled: "Open label, non-randomized, multi-cohort pilot study of genetically engineered NY-ESO-1 SPEAR T-cells in HLA-A2+ patients with synovial sarcoma (NCT01343043)"

2 Data cut-off for ASCO (Free ASCO Whitepaper) 2017 was March 30, 2017

Overview of Study Design from the Trial in Progress Poster for NY-ESO SPEAR T-cells in MRCLS

Open-label, non-randomized pilot study evaluating the safety, tolerability, and antitumor activity of NY-ESO SPEAR T-cells in patients with MRCLS
Initially, 10 patients are planned to be enrolled, with potential to enroll an additional 5 patients
Patients who do not receive minimum cell dose or who do not receive T‑cell infusion may be replaced
Patients must be: ≥ 18 years old; HLA-A*02:01, *02:05, or *02:06 positive; have advanced (metastatic or inoperable) MRCLS expressing NY-ESO-1 at 2+/3+ intensity in ≥30% of tumor cells by IHC; measurable disease; prior systemic anthracycline therapy; have ECOG status 0 or 1; and adequate organ function.
Lymphodepletion regimen: Flu (30mg/m2/day) and Cy (600 mg/m2/day) for 3 days
Target dose of 1 – 8 × 109 transduced SPEAR T-cells
Efficacy assessed by overall response rate, time to response, duration of response, progression-free survival, and overall survival at weeks 4, 8, and 12, month 6, and then every 3 months until confirmation of disease progression
The study is open and enrolling
Ten patients have already been identified and enrolled

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

Ligand has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Ligand, 2017, NOV 9, 2017, View Source [SID1234521847]).

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

IntelGenx has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, IntelGenx, 2017, NOV 9, 2017, View Source [SID1234521914]).

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CytRx Reports Third Quarter 2017 Financial Results

On November 9, 2017 CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, reported financial results for the quarter ended September 30, 2017, and provided an overview of recent accomplishments and plans (Press release, CytRx, NOV 9, 2017, View Source [SID1234521860]).

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"The third quarter of 2017 was transformative for CytRx," said Steven A. Kriegsman, CytRx’s Chairman and CEO. "We executed a global strategic licensing transaction for aldoxorubicin, placing this clinically validated, post-Phase 3 asset into the hands of NantCell, Inc. to file the planned New Drug Application with the U.S. FDA. Further, NantCell is laying the groundwork to develop aldoxorubicin in combination with other anti-cancer agents, including immunotherapies and other innovative cell-based approaches, and recently communicated their plans to submit Investigational New Drug (IND) Applications to the U.S. FDA combining aldoxorubicin with certain immunotherapy protocols in patients with pancreatic and breast cancer."

Mr. Kriegsman continued, "Internally, our efforts and resources have been refocused on generating new candidates from our proprietary LADR (Linker Activated Drug Release) technology platform where our goal is to create a new class of rationally designed, breakthrough anti-cancer drugs with the potential to address a broad range of tumor types. Selection of a highly potent drug conjugate to bring into the clinic is at the forefront of our strategy heading into 2018, and we remain opportunistic in seeking out strategic alliances with big pharma."

Third Quarter 2017 and Recent Highlights

Highlighted Progress of LADR Candidates and Future Oncology Pipeline. In November 2017, CytRx provided an update on its R&D activities surrounding its proprietary LADR (Linker Activated Drug Release) Technology Platform. To date in 2017, the Company’s drug discovery laboratory, located in Freiburg, Germany, has tested over 75 rationally designed drug conjugates, and four lead candidates have been selected based on in vitro and animal preclinical studies, stability, and manufacturing feasibility. The Company’s goal is to nominate the next LADR candidate for clinical development in the first quarter of 2018 and to present these important preclinical data at an upcoming scientific meeting during the first half of 2018. Additional animal efficacy and toxicology testing of these lead candidates is underway, and CytRx’s goal is to file the first LADR-discovered, ultra-high potency conjugate IND for clinical trials in 2018.

Completed Reverse Stock Split. On November 1, 2017, CytRx completed a 1-for-6 reverse stock split of its issued and outstanding common stock. The split-adjusted shares of CytRx’s common stock will continue trading on the Nasdaq Capital Market under the Company’s existing symbol "CYTR," provided that the Company continues to comply with the Nasdaq listing requirements. The reverse stock split reduced the number of common shares outstanding to approximately 27.6 million as of the effective date. Authorized shares were also proportionally reduced to approximately 41.7 million, and the Company’s preferred stock was reduced to approximately 0.8 million shares. The reverse stock split was approved by the Company’s stockholders at a Special Meeting held on October 27, 2017.

Completed Strategic Realignment of Clinical Development Team. In November 2017, the Company announced a strategic realignment of its development team to focus its efforts on generating new drug candidates from its LADR Technology Platform. Hurley Consulting Associates, who have been providing strategic consulting to CytRx, will lead the company’s development and regulatory activities. Concurrently, the position of Senior Vice President of Drug Development has been eliminated, and Shanta Chawla, M.D., has retired.

Announced Upcoming NantCell INDs. In October 2017, CytRx announced that aldoxorubicin licensee NantCell, Inc. (a private subsidiary of NantWorks, LLC) plans to submit IND applications combining aldoxorubicin with its immunotherapy protocols for clinical trials in pancreatic and breast cancers. The planned clinical trials will enroll patients with cancers resistant to standard therapy and will employ adaptive designs allowing for expansion of treatment cohorts and modification of patient treatments based on tumor profiling and individual patient responses over time.

Completed Strategic Licensing Transaction with NantCell for Development of Aldoxorubicin. In July 2017, CytRx and NantCell executed a strategic licensing agreement for the global rights to aldoxorubicin. NantCell, led by Dr. Patrick Soon-Shiong, who developed and commercialized Abraxane, an albumin-mediated cytotoxic agent, received exclusive rights to develop and commercialize aldoxorubicin for all indications. Under the terms of the agreement, NantCell purchased $13 million of CytRx common stock at a per share price of $6.60 (split-adjusted), representing approximately a 92% premium to the market price at the time of the transaction. CytRx is eligible to receive up to an additional $343 million in regulatory and commercial milestones, plus increasing double-digit royalties on sales for aldoxorubicin’s lead indication of soft tissue sarcomas, and mid to high single-digit royalties for any additional indications. CytRx also issued NantCell a warrant to purchase on a split-adjusted basis up to 500,000 shares of common stock at $6.60 over the next 18 months. CytRx has been actively working with the NantCell team to transition the aldoxorubicin program, including both completed and ongoing studies.

Third Quarter 2017 Financial Results

CytRx reported cash, cash equivalents and short-term investments of $46.0 million as of September 30, 2017. During the third quarter, CytRx entered into a global strategic licensing agreement for aldoxorubicin with NantCell and received a strategic investment of $13.0 million. Concurrent with the closing of the aldoxorubicin license agreement, CytRx amended its existing long-term loan and made payments of $10 million during the quarter to the lender.

Net loss for the quarter ended September 30, 2017, was $5.1 million, or $(0.19) per share, compared with a net loss of $12.2 million, or $(0.80) per share, for the quarter ended September 30, 2016, a reduction of $7.1 million. During the third quarter of 2017, the Company recognized a non-cash gain of $3.8 million on the fair value adjustment of warrant derivative liability related to warrants issued in 2016, compared to a non-cash gain of $0.2 million during the third quarter of 2016 related to now expired warrants.

Research and development (R&D) expenses were $4.8 million for the third quarter of 2017, including approximately $2.6 million for aldoxorubicin, $1.0 million for pre-clinical development of new albumin-binding, ultra-high potency cancer drugs (Freiburg lab), and approximately $1.2 million for non-cash expenses and general operations of our clinical programs. This is a reduction of approximately 47 percent compared to R&D expenses of $8.9 million for the third quarter of 2016.

General and administrative (G&A) expenses were $3.4 million for the third quarter of 2017, compared with $2.8 million for the third quarter of 2016.

About CytRx Corporation

CytRx Corporation is a biopharmaceutical company specializing in research and clinical development of novel anti-cancer drug candidates that employ linker technologies to enhance the accumulation and release of drug at the tumor. Aldoxorubicin, CytRx’s most advanced drug conjugate, is an improved version of the widely used chemotherapeutic agent doxorubicin and has been out-licensed to NantCell, Inc. CytRx is also rapidly expanding its pipeline of ultra-high potency oncology candidates at its laboratory facilities in Freiburg, Germany, through its LADR (Linker Activated Drug Release) technology platform, a discovery engine designed to leverage CytRx’s expertise in albumin biology and linker technology for the development of a new class of potential breakthrough anti-cancer therapies.

Novelion Therapeutics Reports Third Quarter 2017 Financial Results and Announces Leadership Change

On November 9, 2017 Novelion Therapeutics Inc. (NASDAQ: NVLN), a biopharmaceutical company dedicated to developing and commercializing innovative new therapies for individuals living with rare diseases ("Novelion" or the "Company"), reported financial results for the third quarter ended September 30, 2017, announced that it has commenced a search for a new chief executive officer ("CEO") to lead the Company in the next stage of growth, and provided an overview of recent business highlights (Press release, QLT, NOV 9, 2017, View Source [SID1234521900]). Chief Executive Officer Mary Szela has resigned for personal reasons, effective immediately.

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To lead the company until a permanent CEO is appointed, the Board announced the creation of an interim Office of the Chief Executive Officer, comprised of Jeffrey Hackman, Novelion’s Chief Operating Officer, Jason Aryeh, Chairman of the Board of Directors and Mark Corrigan, Director. The Board has also formed a search committee, which has commenced efforts to identify candidates who will bring strong strategic and operational direction to the enterprise.

Jason Aryeh said, "Novelion’s strategy and mission will continue to focus on supporting access to our marketed therapies for patients globally and pursuing the development of our product portfolio in order to support long-term and sustainable growth. The board is confident in its ability to identify a successor who will effectively drive improved growth and shareholder value by reinvigorating the Company’s plans in support of our key objectives."

Mr. Aryeh continued, "We recognize the energy that Mary Szela brought to her role as CEO. Much has been accomplished in her tenure, including the merger to create Novelion, and we thank her for her dedication."

Third Quarter 2017 Highlights & Business Update


JUXTAPID: Novelion reported net revenues of JUXTAPID of $15.2 million in the third quarter of 2017, $10.3 million, or 68%, of which were from prescriptions written in the U.S.


MYALEPT: Novelion reported net revenues of MYALEPT of $13.5 million in the third quarter of 2017. $11.3 million, or 84%, of these revenues were from prescriptions written in the U.S.


Novelion reported total net revenues of $28.7 million in the third quarter of 2017.


The Company is continuing its efforts to stabilize JUXTAPID in the U.S., and continues to make progress with the launch in Japan.


Novelion ended the third quarter of 2017 with $70.5 million in unrestricted cash, compared with $83.3 million at the end of the second quarter of 2017.


Novelion’s marketing authorization application for metreleptin was accepted by the European Medicines Agency in January of 2017. In October 2017, the company submitted its responses to the Day 120 Questions, and based on current timelines, continues to anticipate EMA approval in the first half of 2018.


Novelion appointed Jeffrey Hackman as Executive Vice President and Chief Operating Officer, effective November 1, 2017. Mr. Hackman will lead the company’s commercial efforts, business development, manufacturing and supply chain initiatives. Mr. Hackman was most recently senior vice president, head of the US Internal Medicine and Oncology franchises for Shire. In this role, Mr. Hackman was a member of the US commercial leadership team and was responsible for sales, marketing, business insights and analytics for eight licensed products within Shire’s US rare disease portfolio.


Novelion appointed Murray Stewart, M.D. as Executive Vice President, Head of R&D, effective November 27, 2017. Dr. Stewart will lead the company’s clinical development activities, provide strategic regulatory guidance for the pipeline and commercial product initiatives, and maintain oversight of global medical affairs, publications and registry activities. Dr. Stewart will join Novelion from GlaxoSmithKline (GSK) where he is currently chief medical officer with global responsibility for patient well-being across the vaccines, pharmaceutical and consumer business units.


Novelion appointed Suzanne Bruhn, Ph.D. to its board of directors, effective October 1, 2017. Dr. Bruhn is president and chief executive officer of Proclara Biosciences, Inc. Prior to joining Proclara, Dr. Bruhn served as president and chief executive officer of Promedior, Inc. She also served as a member of the board of directors of Raptor Pharmaceuticals from 2011 until it was acquired by Horizon Pharma in 2016. Previously, Dr. Bruhn served as senior vice president, strategic planning and program management at Shire from 1998 until 2012.

2017 Financial Guidance

The Company reiterated its previously stated net revenues financial guidance for full year 2017 and expects:


Total net revenues between $135 million and $145 million;

JUXTAPID net revenues between $70 million and $75 million; and

MYALEPT net revenues between $65 million and $70 million.

Third Quarter 2017 Financial Results

On November 29, 2016, the Company completed its acquisition of Aegerion Pharmaceuticals, Inc. ("Aegerion"). The acquisition has been accounted for as a business combination in which Novelion was considered the acquirer of Aegerion. As such, under U.S. Generally Accepted Accounting Principles ("GAAP"), the financial statements of Novelion are treated as the historical financial

statements of the consolidated companies, with the results of Aegerion being included from November 29, 2016. This release also includes pro forma adjusted non-GAAP financial information showing pro forma results of operations of Novelion as if the acquisition had occurred on January 1, 2016. Reconciliation of the financial results on a GAAP versus non-GAAP basis are provided below the financial information that follows.

GAAP total net revenues for the third quarter of 2017 were $28.7 million compared to the prior year’s third quarter net revenues of $0. GAAP net revenues for JUXTAPID in the third quarter of 2017 were $15.2 million compared to $0 in the prior year. GAAP net revenues for MYALEPT in the third quarter of 2017 were $13.5 million compared to $0 for the same period in 2016.

GAAP total operating expenses for the third quarter of 2017 were $38.6 million compared to total operating expenses of $6.0 million for the same period in 2016. GAAP SG&A expenses were $21.4 million in the third quarter of 2017 compared to $3.2 million for the same period in 2016. GAAP R&D expenses were $17.1 million in the third quarter of 2017 compared to $2.9 million for the same period in 2016.

On a pro forma basis, during the third quarter of 2017, SG&A expenses were $20.2 million compared to $28.2 million for the same period in 2016. The decrease in pro forma SG&A expenses in the third quarter of 2017 compared with the same period in 2016 was primarily related to a reduction in headcount and legal and consulting fees.

On a pro forma basis, during the third quarter of 2017, R&D expenses were $16.9 million compared to $12.7 million for the same period in 2016. The decrease in R&D expenses in the third quarter of 2017 compared with the same period in 2016 was primarily related to a reduction in headcount.

GAAP net loss in the third quarter of 2017 was $49.7 million compared to GAAP net loss of $5.9 million during the same period in 2016.

On a pro forma basis, net loss in the third quarter of 2017 was $16.6 million, compared to $14.3 million for the same period in 2016.

As part of the Merger between QLT and Aegerion, the Company acquired inventory, a portion of which is classified as non-current based on its forecasted consumption exceeding one year. An excess and obsolescence analysis is run to determine the need to adjust inventory carrying values. In the third quarter, that analysis led to a write down of inventory of approximately $17.3 million.

First Nine Months of 2017 Financial Results

GAAP total net revenues for the first nine months of 2017 were $99.5 million compared to $0 for the same period of 2016. GAAP net revenues for JUXTAPID for the first nine months of 2017 were $51.9 million compared to $0 in same period in 2016. GAAP net revenues for MYALEPT for the first nine months of 2017 were $47.6 million compared to $0 for the same period in 2016.

GAAP total operating expenses for the first nine months of 2017 were $112.1 million compared to total operating expenses of $22.3 million for the same period in 2016. GAAP SG&A expenses were $72.4 million in the first nine months of 2017 compared to $13.6 million for the same period in 2016.

GAAP R&D expenses were $37.2 million in the first nine months of 2017 compared to $8.8 million for the same period in 2016.

On a pro forma basis, for the first nine months of 2017, SG&A expenses were $68.2 million compared to $111.5 million for the same period in 2016. For the first nine months of 2017, R&D expenses on a pro forma basis were $36.6 million compared to $37.9 million for the same period in 2016.

GAAP net loss for the first nine months of 2017 was $102.1 million compared to GAAP net loss of $33.0 million during the same period in 2016.

On a pro forma basis, net loss for the first nine months of 2017 was $26.7 million, compared to $111.7 million for the same period in 2016.

As of September 30, 2017, the Company’s consolidated unrestricted cash balance was $70.5 million, compared to $83.3 million at June 30, 2017 and $108.9 million at December 31, 2016. As of September 30, 2017, there were 18.6 million shares outstanding. At September 30, 2017, total debt was $325 million, reflecting the principal amount of convertible debt issued by Aegerion and consolidated as a result of the acquisition.