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Unum Therapeutics to Present Preclinical Data on a New, Emerging Platform Technology for Solid Tumors at the SITC Annual Meeting

On November 6, 2018 Unum Therapeutics Inc. (NASDAQ: UMRX), reported that the Company will present preclinical data on a new proprietary technology platform called Bolt-On Chimeric Receptor, or BOXR, designed to improve the effectiveness of T cells in solid tumor cancers, at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting taking place November 7-11, 2018 in Washington, D.C (Press release, Unum Therapeutics, NOV 6, 2018, View Source [SID1234530852]).

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Unum has generated a large panel of novel bolt-on transgenes, evaluated using a unique screening strategy, and has identified lead candidates most likely to enhance T cell functions in the solid tumor microenvironment. The bolt-on transgenes enhanced immunometabolism or costimulation of both ACTR- and CAR-expressing T cells and significantly improved their function under stringent in vitro and in vivo conditions.

"We are excited by this new platform, which we believe has the potential to further bolster our ACTR technology pipeline, and highlights our innovative approaches to overcome immunosuppressive challenges in solid tumors," said Seth Ettenberg, Unum’s Chief Scientific Officer. "We look forward to continuing to explore the potential of this novel technology by selecting a lead development candidate to bring to patients in need in the clinical setting."

Details on the presentation are as follows:

Presentation Title: Select metabolic and costimulatory bolt-on transgenes enhance chimeric receptor-bearing T cell activity against solid tumors
Presenter:Luke Barron, PhD, Unum Therapeutics
Session Title: Cell-Based Therapies for Solid Tumors
Poster Number: 216
Date & Time: Saturday, November 10th, 12:20-1:50 and 7-8:30pm
Location:Walter E. Washington Convention Center, Hall E

Adaptimmune Reports Third Quarter 2018 Financial Results and Business Update

On November 6, 2018 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported financial results for the third quarter ended September 30, 2018, and provided a business update (Press release, Adaptimmune, NOV 6, 2018, View Source [SID1234530851]).

"We have now completed the three dose escalation cohorts of the studies with MAGE-A4 and MAGE-A10, our leading wholly owned programs. The Safety Review Committee has agreed that the higher pre-conditioning regimen and cell doses are tolerable and there were no dose limiting toxicities. These studies will now move into the expansion phase, which allows us to treat patients with up to ten billion cells, without a pre-determined stagger across a broad range of tumor types. We have also continued dosing patients in the AFP study with 100 million cells and anticipate escalating to Cohort 2 in early 2019. We expect to report our next clinical data by no later than our first quarter financial results in May 2019," said James Noble, Chief Executive Officer.

Clinical momentum in wholly owned programs

Ongoing MAGE-A10 and MAGE-A4 studies

· There are three ongoing studies with MAGE-A10 and MAGE-A4 SPEAR T-cells

· Two MAGE-A10 studies: one in non-small cell lung cancer (NSCLC) and a triple tumor study in bladder, melanoma, and head & neck cancers

· A MAGE-A4 basket study in NSCLC, bladder, melanoma, synovial sarcoma, myxoid/round cell liposarcoma (MRCLS), head & neck, ovarian, gastric, and esophageal cancers

· All three studies are first-in-human trials utilizing a modified 3+3 design with escalating target doses of 100 million (Cohort 1), 1 billion (Cohort 2), and 5 billion (Cohort 3) transduced SPEAR T-cells to evaluate safety, including dose limiting toxicities (DLTs)

· The preconditioning regimen in the first two cohorts was cyclophosphamide (600mg/m2/day) and fludarabine (30 mg/m2/day) on days -7, -6 and -5, and an extra day of fludarabine was added to the third cohorts and expansion phases, as clinical and translational data indicate that this extra day may be important for optimal T-cell expansion post-infusion

· Following the initial three cohorts, the Safety Review Committee (SRC) meets to decide whether to progress to the expansion phase, which has a target dose of 5 billion cells (range 1.2 to 10B) without pre-determined intervals between patient dosing

· The SRC recommended moving into the expansion phase for the MAGE-A10 triple tumor and MAGE-A4 basket studies

· As in the first two cohorts of these studies, there was no evidence of toxicity related to off-target binding or alloreactivity in the third cohorts at target doses of 5 billion cells

· Most adverse events were consistent with those experienced by cancer patients undergoing chemotherapy or other immunotherapies

ESMO data

· Initial safety data from the first two cohorts of the MAGE-A10 and MAGE-A4 studies were presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress (https://bit.ly/2PdB3CR)

· In brief, these data showed:

· Disease progressed for all eight patients treated in the first dose cohorts of the two MAGE-A10 studies (five patients with lung cancer, two with head & neck cancer, and one with melanoma)

· For the three patients treated in Cohort 2 of the MAGE-A10 study (all lung cancer patients), one patient died of pneumonia (unrelated to therapy) and two had stable disease (SD), albeit transient

· Of the six patients treated in Cohorts 1 and 2 of the MAGE-A4 basket study, best response was SD in four patients and progressive disease (PD) in two patients

·One patient in the MAGE-A4 basket study with SD had an overall 27% reduction of target lesions observed at Week 6, and was assessed as PD at the time of the second scan, which took place after the ESMO (Free ESMO Whitepaper) poster cut-off date

· No evidence of toxicity related to off-target binding or alloreactivity at target doses of 100 million or 1 billion cells

· Most adverse events consistent with those experienced by cancer patients undergoing chemotherapy or other immunotherapies

· Transduced cells detectable in peripheral blood at levels consistent with dose

Data from ongoing AFP study

· Dosing in Cohort 1 of AFP study is ongoing

· Anticipate dose escalation to Cohort 2 in early 2019.

NY-ESO data updates to be presented at SITC (Free SITC Whitepaper)

·The NY-ESO program transitioned to GSK in July 2018

·An abstract summarizing NY-ESO SPEAR T-cells in MRCLS was accepted for presentation at SITC (Free SITC Whitepaper), and is available online today

· Data in the abstract state that out of ten MRCLS patients, there were four with partial responses (PRs) and four with SD, as per investigator assessment

· These data will be updated in a poster at SITC (Free SITC Whitepaper)

· Overall, there was evidence of reduction in target lesions in seven patients out of eight evaluable patients

· The data submitted in the abstract included investigator assessments. These assessments showed a best response of four confirmed PR, one unconfirmed PR, and three patients with SD out of eight evaluable patients

· Two of the responses were confirmed before the minimum 28 days required by RECIST v1.1 (22 and 25 days), and the patients subsequently progressed

· Therefore, the response rate by RECIST, which will be presented in the poster, is two confirmed PRs and six patients with SD out of the eight evaluable patients

· Patients in the MRCLS study received the same preconditioning regimen as was used in Cohort 4 of the synovial sarcoma study, and these patients had less durable responses compared to Cohort 1 patients in the synovial sarcoma study, who received a more intense preconditioning regimen

· The most frequent AEs were consistent with those experienced by patients with cancer who are undergoing cytotoxic chemotherapy or other immunotherapies

· A second poster with NY-ESO data will also be presented at SITC (Free SITC Whitepaper) summarizing translational research conducted in the context of the NY-ESO synovial sarcoma study examining serum factors that lead to T-cell expansion with different preconditioning regimens (including the impact of fludarabine), tumor micro-environment analyses pre- and post-infusion, and SPEAR T-cell functionality post-infusion. This abstract is also available online.

Manufacturing

Adaptimmune on its way to becoming a fully integrated cell therapy company

· 2018 has been a successful year for manufacturing with the Navy Yard facility regularly producing target cell doses > 1 billion cells with more than 50% producing > 5 billion cells

· Producing cell doses across multiple solid tumor indications

· Cells have been manufactured for a number of patients who could enter the MAGE-A4 and/or MAGE-A10 expansion phases, once eligible

Other corporate news

Adaptimmune is focused on its next stage of development and in a strong position to deliver success with SPEAR T-cell therapies

· Announced the closing of a registered direct offering of Adaptimmune’s American Depositary Shares ("ADSs") (https://bit.ly/2MZFEIH) with net proceeds of approximately $100 million

· Adaptimmune intends to use the net proceeds from this offering to advance the Company’s wholly owned pipeline of SPEAR T-cell candidates through clinical trials as well as for other general corporate purposes

· Completed transition of NY-ESO IND to GSK and received approximately $26 million in milestone payments

· Funded through to late 2020 with cash and cash equivalents of $153.1 million and total liquidity(1) of $237.7 million

· Held annual Scientific Advisory Board meeting in October with Adaptimmune R&D leaders and external experts in immunology and oncology (bios available here: https://bit.ly/2PvHH4w); focused on optimal employment of NY-ESO learnings in ongoing and future studies as well as strategies for novel target identification

Financial Results for the three and nine month period ended September 30, 2018

· Cash / liquidity position: As of September 30, 2018, Adaptimmune had cash and cash equivalents of $153.1 million and Total Liquidity(1) of $237.7 million.

· Revenue: Revenue for the three and nine months ended September 30, 2018 was $40.8 million and $58.0 million, respectively, compared to $27.2 million and $33.6 million for the same periods of 2017. The revenue in the three and nine months ended September 30, 2018 includes $39.1 million of revenue for the license to NY-ESO, which commenced in September 2018.

· Research and development ("R&D") expenses: R&D expenses for the three and nine months ended September 30, 2018 were $23.5 million and $75.5 million, respectively, compared to $24.0 million and $62.2 million for the same periods of 2017. The R&D expenses in the nine months ended September 30, 2018 has increased compared to the same period in 2017 due to increased clinical trial and related manufacturing activities. R&D expenses in the three months ended September 30, 2018 compared to the same period in 2017 decreased due to the transfer of the NY-ESO program to GSK.

· General and administrative ("G&A") expenses: G&A expenses for the three and nine months ended September 30, 2018 were $10.3 million and $32.8 million, respectively, compared to $8.1 million and $22.3 million for the same periods of 2017. The increase was primarily due to increased personnel costs consistent with the Company’s planned infrastructure growth.

· Other (expense) income, net: Other expense for the three and nine months ended September 30, 2018 was $2.2 million and $10.5 million, respectively, compared to an income of $3.6 million and $7.2 million for the same periods of 2017. Other income primarily comprises unrealized foreign exchange gains, which fluctuate depending on exchange rate movements and the amount of foreign currency assets and liabilities.

· Net income (loss): Net income (loss) attributable to holders of the Company’s ordinary shares for the three and nine months ended September 30, 2018 was an income of $5.2 million and a loss of $59.3 million, respectively, ($0.01 and $(0.10) per ordinary share) compared to a loss of $0.9 million and $42.9 million, respectively, ($(0.00) and $(0.08) per ordinary share) in the same periods of 2017.

(1) Total liquidity is a non-GAAP financial measure, which is explained and reconciled to the most directly comparable financial measures prepared in accordance with GAAP below.

Financial guidance

The Company believes that its existing cash, cash equivalents and marketable securities will fund the Company’s current operations through to late 2020.

Conference call information

The Company will host a live teleconference and webcast at 8:00 a.m. EST (1:00 p.m. GMT) today. The live webcast of the conference call will be available via the events page of Adaptimmune’s corporate website at www.adaptimmune.com. An archive will be available after the call at the same address. To participate in the live conference call, please dial (833) 652-5917 (U.S.) or +1 (430) 775-1624 (International). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (2458438).

Supernus Announces Third Quarter 2018 Financial Results and Record Quarterly Revenue

On November 6, 2018 Supernus Pharmaceuticals, Inc. (NASDAQ: SUPN), a pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported record financial results for the third quarter of 2018 and related Company developments (Press release, Supernus, NOV 6, 2018, View Source [SID1234530847]).

"Supernus generated another strong quarter of growth, setting a new record for quarterly net product sales of $100 million," said Jack Khattar, President and CEO of Supernus Pharmaceuticals. "Despite the market introduction of new competitive preventive treatments for migraine, Trokendi XR continued to capture a greater portion of the topiramate market. For Oxtellar XR, we continue to prepare for the potential launch of the monotherapy indication for partial seizures."

Progress of Product Pipeline
Given the recently accelerated development timeline for SPN-812 that positions its potential regulatory approval and commercial launch ahead of SPN-810, the Company has directed its resources to prioritize filing of the New Drug Application (NDA) and potential commercial launch of SPN-812 in the United States.

As a result, the following are the updated plans and timelines for both product candidates:

SPN-812 – Novel non-stimulant for the treatment of ADHD

The Phase III program consists of four three-arm, placebo-controlled trials: P301 and P303 trials in patients 6-11 years old and P302 and P304 trials in patients 12-17 years old.
The Company expects to announce top-line data from P301 and P303 pediatric trials concurrently in early December 2018, and from P302, the first adolescent Phase III trial, by the end of December 2018. Top-line data from the second adolescent Phase III trial, P304, are expected by the end of the first quarter of 2019.
The Company expects to submit an NDA for SPN-812 in the second half of 2019, and to launch it, pending U.S. Food and Drug Administration (FDA) approval, in the second half of 2020.
SPN-810 – Treatment of Impulsive Aggression in patients with ADHD

As expected, the first Phase III trial (P301) has reached its original enrollment target. However, given the aforementioned prioritization of SPN-812 and that top-line data from the second Phase III trial (P302) is expected around mid-2019, the Company has decided to keep P301 enrollment active until data from both trials can be released concurrently instead of sequentially. This change does not impact the timing of submission of the NDA for SPN-810, given that the NDA submission is rate-limited by completion of the P302 trial and generation of data in the adolescent patient population.
The Company continues to observe enrollment in the open label extension (OLE) study at 90% or higher. On average, a patient in the OLE study remains on SPN-810 treatment for 9.5 months, which we believe is an encouraging sign of the tolerability and efficacy of SPN-810.
Patient dosing has been initiated in the Phase III trial in adolescent patients.
The Company expects to submit an NDA for SPN-810 in the second half of 2020, and to launch it, pending FDA approval, in the second half of 2021.
SPN-604 (formerly known as Oxtellar XR for Bipolar)

The Company continues to expect initiating pivotal Phase III studies for the treatment of bipolar disorder in the second half of 2019.
"We are pleased to announce the completion of enrollment in the first three Phase III trials for SPN-812," said Jack Khattar. "We look forward to reporting top-line data from these trials during December 2018. If successful, SPN-812 has the potential to be a novel non-stimulant for the treatment of ADHD that compares favorably to existing medications."

Operating Expenses
Research and development expenses in the third quarter of 2018 were $20.4 million, as compared to $13.0 million in the same quarter last year. The increase was due primarily to the initiation of the four Phase III clinical trials for SPN-812 in the second half of 2017 and, to a lesser extent, the OLE trials for SPN-812 and SPN-810.

Selling, general and administrative expenses in the third quarter of 2018 were $40.9 million, essentially unchanged compared to $40.8 million in the same quarter last year.

Operating Earnings and Earnings Per Share
Operating earnings in the third quarter of 2018 were $37.5 million, a 68.2% increase over $22.3 million in the same prior year period. The improvement in operating earnings was primarily due to increased net product sales, partially offset by increased research and development expenses.

GAAP net earnings in the third quarter of 2018 were $28.0 million, or $0.52 per diluted share, as compared to $16.0 million, or $0.29 per diluted share, in the same period last year. In addition to higher operating income, GAAP net earnings and diluted earnings per share for the third quarter of 2018 benefited from the reduction in the statutory U.S. federal income tax rate and, to a lesser extent, from stock option exercises.

Weighted-average diluted common shares outstanding were approximately 54.2 million in the third quarter of 2018, as compared to approximately 53.6 million in the third quarter of 2017.

As of September 30, 2018, the Company had $740.5 million in cash, cash equivalents, marketable securities and long term marketable securities, as compared to $273.7 million at December 31, 2017. This increase reflects net proceeds of $364.9 million from the issuance of convertible senior notes and warrants in March 2018, partially offset by purchases of convertible note hedges, as well as increased cash from operations in the nine months ended September 30, 2018.

Financial Guidance
For full year 2018, the Company is updating its prior guidance as set forth below:

Net product sales in the range of $388 million to $395 million, compared to the previously expected range of $385 million to $400 million.
Research and development expenses of approximately $95 million, including the one-time upfront expense of $15 million in the fourth quarter for the acquisition of Biscayne Neurotherapeutics, Inc.
Operating earnings in the range of $120 million to $125 million, compared to the previously expected range of $115 million to $125 million.
The Company expects an effective tax rate of approximately 23% to 25% for the fourth quarter of 2018.
Supernus ranked number one pharmaceutical company worldwide in Fortune’s "100 Fastest-Growing Companies" list for 2018 and number three in all industries
In August 2018, Fortune ranked qualifying companies based on revenue growth rate, EPS growth rate, and three-year annualized total return for the period ended June 29, 2018. In a review of Supernus and using their methodology, Fortune placed Supernus in the top spot in the pharmaceutical industry worldwide and the third spot across all industries.

To view the full list of Fortune’s 100 Fastest-Growing Companies go to: View Source

"I am so proud of our employees. They deserve all the recognition Supernus has received over the past few years, from making the Deloitte Technology Fast 500 list three years in a row to being ranked as the number one Fortune 100 fastest growing pharmaceutical company in the world," said Jack Khattar. "Their hard work and commitment to excellence and to our patients are second to none, and I am very fortunate to be working with such an incredible organization."

Conference Call Details
The Company will hold a conference call hosted by Jack Khattar, President and Chief Executive Officer, and Greg Patrick, Vice President and Chief Financial Officer, to discuss these results at 9:00 a.m. Eastern Time, on Wednesday, November 7, 2018. An accompanying webcast also will be provided.

Please refer to the information below for conference call dial-in information and webcast registration. Callers should dial in approximately 10 minutes prior to the start of the call.

Conference dial-in:
International dial-in:
Conference ID:
Conference Call Name:
(877) 288-1043
(970) 315-0267
2697616
Supernus Pharmaceuticals Third Quarter 2018 Earnings Conference Call

Following the live call, a replay will be available on the Company’s website, www.supernus.com, under "Investor Relations".

Queensland Government invests $1.4 million to support clinical trials and jobs growth

On November 6, 2018 QIMR Berghofer Medical Research Institute has reported its collaboration with US biopharmaceutical company Atara Biotherapeutics, entering into major agreements to manufacture and develop cellular immunotherapies for multiple sclerosis and some cancers (Press release, QIMR Berghofer Medical Research Institute, NOV 6, 2018, View Source [SID1234530846]).

QIMR Berghofer secured the deal thanks to a $1.4 million funding injection from the Queensland Government to expand and upgrade the Institute’s world-class, regulatory-approved cell therapy manufacturing facility, Q-Gen Cell Therapeutics.

The deal with Atara will allow world-first clinical trials to be established locally and in the United States and Europe, giving Queenslanders access to cutting-edge medical treatments.

Under the agreement, Q-Gen will manufacture the immunotherapies for the clinical trials, including those using patients’ own immune cells and others using cells from healthy donors.

QIMR Berghofer and Atara have also extended an existing agreement to collaborate on research and development of new T cell immunotherapies for cancers and autoimmune disorders associated with a number of viruses.

Queensland’s Minister for State Development, Manufacturing, Infrastructure and Planning, Cameron Dick, said the two agreements would support approximately 40 full-time jobs at QIMR Berghofer and Q-Gen.

"Through our government’s support, QIMR Berghofer can now expand its cell therapy facility Q-Gen Cell Therapeutics, including purchasing specialised laboratory equipment," he said.

"As a result of this facility upgrade, Atara has agreed to extend its agreement with QIMR Berghofer to collaborate on research and development of new immunotherapies for cancers and autoimmune disorders associated with a number of viruses.

"The two agreements will support 40 full-time jobs at QIMR Berghofer and its Q-Gen facility at Herston as well as grow QIMR Berghofer’s reputation as a world-leader in the immunotherapy field.

"Our government’s investment will strengthen our state’s push into the sophisticated advanced manufacturing of cell therapy products by enabling QIMR Berghofer to secure agreements and attract additional cell therapy manufacturing from not only Atara, but also other biomedical and pharmaceutical companies.

"Projects like this underpin the Queensland Biomedical 10-Year Roadmap and Action Plan, which was developed in close consultation with industry to diversify Queensland’s industry base, create high-value knowledge-based jobs of the future and drive export growth for the industry by 2027."

The Minister for Health Steven Miles said immunotherapy had emerged as the "fourth pillar" of cancer treatment, along with surgery, chemotherapy and radiotherapy.

"Immunotherapy works by training the immune system to recognise and destroy cancer and other harmful cells," he said.

"These agreements put Queensland’s biomedical capabilities on the world stage."

Atara has also exercised its option under an existing licence agreement with QIMR Berghofer to an exclusive, worldwide licence to develop and commercialise a specific T cell immunotherapy that "turbo charges" a patient’s immune cells to treat autoimmune conditions associated with the Epstein-Barr virus, such as multiple sclerosis.

All of the immunotherapies were developed by the head of QIMR Berghofer’s Tumour Immunology Laboratory, Professor Rajiv Khanna AO, and his team.

Professor Khanna said the agreements with Atara were a win for Queensland patients.

"We are delighted to partner with Atara and the Queensland Government to bring much-needed new T cell immunotherapies to patients," he said.

"We are very excited by the possibility that in future, we might be able to offer new treatment options to patients with certain virus-associated cancers and autoimmune conditions like multiple sclerosis."

QIMR Berghofer’s Director and CEO, Professor Frank Gannon, said the Queensland Government’s funding was a major boost for advanced manufacturing in the state.

"Q-Gen is the one of the largest dedicated cell therapy manufacturing facilities in Australia," he said.

"It is already a world-class facility that has secured regulatory approval to manufacture immunotherapies for clinical trials in Australia and the United States. It will now be upgraded to also manufacture for clinical trials in Europe and to meet the demand created by this deal with Atara.

"The Queensland Government’s support will allow us to expand our cell therapy manufacturing program, providing a major boost for Queensland’s biotech sector."

Atara’s Global Head of Research and Development, Dr Dietmar Berger, said he was excited about the collaboration with QIMR Berghofer and increasing biotechnology development in the region.

"Atara is delighted that the Queensland Government is supporting QIMR Berghofer, as well as the growth of the biotechnology community in Queensland," he said.

NewLink Genetics Announces Publication of Abstracts for Presentation at the Society for Immunotherapy for Cancer (SITC) Conference

On November 6, 2018 NewLink Genetics Corporation (NASDAQ:NLNK) reported that the following abstracts have been published and are now available on the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference 2018 website (Press release, NewLink Genetics, NOV 6, 2018, View Source [SID1234530845]).

Abstracts for poster presentation at the SITC (Free SITC Whitepaper) Annual Meeting, November 7-11, 2018

Abstract 11213: A phase 1a clinical trial of NLG802, a prodrug of indoximod with enhanced pharmacokinetic properties, Rixe, O., et al. (Poster #P331). Author will be present Friday, November 9th, 12:45-2:15 PM and 6:30-8:00 PM.
Abstract 10294: The immunogenomic impact of indoximod on the tumor microenvironment of melanoma patients, Yu, J., et al. (Poster #P142). Author will be present Saturday, November 10th, 12:20-1:50 PM and 7:00-8:30 PM.
Abstract 10304: Effects of indoximod plus gemcitabine/nab-paclitaxel on tumor microenvironment of patients with metastatic pancreatic cancer, Yu, J., et al. (Poster #P706). Author will be present Saturday, November 10th, 12:20-1:50 PM and 7:00-8:30 PM.
Posters will be accessible Friday, November 9th, and Saturday, November 10th, from 8 AM to 8 PM, in Exhibition Hall E of the Walter E. Washington Convention Center, Washington, D.C.