On February 27, 2020 BostonGene Corporation, a biomedical software company, reported the appointment of Nathan Fowler, M.D., to the newly created position of Chief Medical Officer (CMO) (Press release, BostonGene, FEB 27, 2020, View Source [SID1234554929]). Dr. Fowler joins BostonGene as it moves into the advanced stages of commercializing its personalized medicine solutions.

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Dr. Fowler brings more than 20 years of patient care experience to BostonGene—most recently as a Professor of Medicine in the Department of Lymphoma/Myeloma at the University of Texas, MD Anderson Cancer Center. In addition to his role as CMO at BostonGene, Dr. Fowler will retain his affiliation with MD Anderson, continuing his involvement in the treatment of lymphoma patients while furthering his clinical research.

"We’re thrilled to welcome Dr. Fowler to BostonGene as a senior member of our management team," said Andrew Feinberg, President and CEO at BostonGene. "His extensive experience researching and developing new therapies, combined with his commitment to discovering innovative solutions to treat patients, is invaluable to BostonGene as we continue to develop our solutions designed to treat the person, not the diagnosis."

"I am honored to join BostonGene as its Chief Medical Officer," said Nathan Fowler, M.D. "I have devoted my entire medical career to finding innovative solutions for cancer treatment, and I firmly believe BostonGene’s solution is fundamentally going to change how we treat cancer patients. I look forward to working with cancer centers from around the globe to drive change and improve the outcomes of all patients battling cancer."

Dr. Fowler received his medical degree from the University of Texas, having completed his internship and residency there as well. He went on to complete a fellowship in hematology/oncology at Georgetown University Hospital in Washington D.C. At MD Anderson Cancer Center, Dr. Fowler focused on developing groundbreaking new therapies for cancer patients and led the clinical research program for nearly ten years. He is considered one of the world’s foremost experts in drug development for blood cancers, and his direct work led to the FDA approval of several novel agents in lymphoma. He also led development of one of the first combination immunotherapy regimens for untreated lymphomas, and is the primary investigator for the ongoing international phase III study with the combination.

Dr. Fowler has published over 150 articles in peer-reviewed journals as well as numerous textbooks on innovative approaches to cancer therapy. Prior to his work in medicine, Dr. Fowler served in the U.S. Army as a nuclear, biological and chemical defense specialist. In addition, he serves as the President and Founder of Halo House Foundation, a non-profit organization providing housing to blood cancer patients receiving treatment.

On February 27, 2020 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, reported it will host a conference call and webcast to report its financial results for the fourth quarter and full year ended December 31, 2019 on March 12, 2020 at 8:30 a.m. ET (Press release, X4 Pharmaceuticals, FEB 27, 2020, View Source [SID1234554928]).

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The conference call can be accessed by dialing (866) 721-7655 (domestic) or (409) 216-0009 (international), followed by the conference ID: 3816258. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. The webcast replay will be available on the website approximately two hours after the completion of the call.

On February 27, 2020 -NantHealth, Inc. (NASDAQ: NH), a next-generation, evidence-based, personalized healthcare company, reported two expanded programs with Eviti Connect, NantHealth’s healthcare payer solution and provider decision support software (Press release, NantHealth, FEB 27, 2020, View Source [SID1234554927]). A leading U.S. Medicaid health plan expanded its coverage and one of the largest non-profit, rural health plans in the country offering coverage for 165,000 members will continue to drive high-value oncological care with Eviti Connect.

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Eviti Connect streamlined the pre-authorization process for cancer treatments and reduced denials and appeals for this leading U.S. Medicaid health plan, while providing oncologists in their network with access to a comprehensive library of evidence-based treatment options for optimal decision making. For one of the largest non-profit rural health plans in the country, Eviti Connect improved consistency in the use of evidence-based medicine, simplified processes, and streamlined treatment approvals that enable cancer patients to begin treatment quickly and efficiently.

"Implementation of the Affordable Care Act has resulted in a much more diverse Medicaid population," said Dr. William Flood, Chief Medical Officer for Eviti at NantHealth. "Medicaid plans now must address more members and a higher prevalence of cancer. Eviti’s comprehensive evidence-based medical library and experienced oncology medical staff enables Medicaid programs to evolve and meet these new challenges quickly."

The Medicaid health plan experienced compelling results in their two-state pilot program. During the two-year pilot, which covered approximately 250,000 members, the insurer noted a significant improvement in the use of evidence-based medicine for member oncology care as a result of providers submitting treatment requests via Eviti Connect. The successful pilot led the insurer to expand the program from two to 10 U.S. states.

For Eviti Connect to continue to help assure quality, high-value care for members with cancer, the non-profit, rural health plan signed a three-year renewal agreement. Under terms of the agreement, the health plan’s provider network will continue to benefit from Eviti Connect decision support solution, which includes evidence-based treatment regimens, condition-specific clinical trial options, and the potential for more efficient preauthorization and expedited reimbursement.

"This renewal of Eviti Connect by a large health plan demonstrates our continued leadership in delivering solutions that align the priorities of physicians, patients, and payers at the moment of clinical prescribing to promote the best care for patients," said Mark Mozley, Senior Vice President, Global Sales & Marketing, NantHealth. "We are pleased to continue this relationship momentum and support our clients’ goals to exceed industry standards for the authorization of oncology care."

NantHealth’s Eviti Connect is a web-based healthcare payer solution that electronically connects health plans and oncology practices for the authorization of treatment plans in near real-time. Eviti Connect reduces the administrative time involved in obtaining authorizations at the drug level, assures appropriate reimbursement at the regimen level, and validates cancer treatment options overall. It also provides the ability to have peer-to-peer conversations between prescribing oncologists and NantHealth oncologists.

On February 27, 2020 Novocure (NASDAQ: NVCR) reported financial results for the quarter and year ended December 31, 2019, highlighting continued commercial execution, strengthening financial performance and clinical and product development programs intended to unlock future value (Press release, NovoCure, FEB 27, 2020, View Source [SID1234554925]). Novocure is a global oncology company working to extend survival in some of the most aggressive forms of cancer by developing and commercializing its innovative therapy, Tumor Treating Fields.

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Fourth quarter and full year 2019 highlights include:

(1) An "active patient" is a patient who is receiving treatment under a commercial prescription order as of the measurement date, including patients who may be on a temporary break from treatment and who plan to resume treatment in less than 60 days.

(2) A "prescription received" is a commercial order for Optune or NovoTTF-100L that is received from a physician certified to treat patients for a patient not previously on Optune or NovoTTF-100L. Orders to renew or extend treatment are not included in this total.

"We start 2020 with substantial momentum from an exceptional 2019," said Asaf Danziger, Novocure’s Chief Executive Officer, "In 2019, we generated $351 million in net revenues and added over $80 million in cash on hand to our balance sheet. The financial strength derived from our existing indications allows us to fund significant investments in clinical and product innovation to advance our proprietary cancer therapy platform. Our focus on strengthening our foundation and executing our strategic plan to deliver value to our patients, employees, and shareholders is unwavering."

"In 20 years of preclinical research across a wide variety of tumor cell lines and cancer models, Tumor Treating Fields has demonstrated a consistent anti-mitotic effect. We are enrolling patients in six clinical programs, including four randomized, phase 3 pivotal trials, and are very optimistic about the potential application of Tumor Treating Fields beyond our currently approved indications," continued William Doyle, Novocure’s Executive Chairman. "Also, new research is pointing the way to further improving the efficacy of the Tumor Treating Fields platform, and we are advancing multiple product development programs to this end. We are excited about the potential to significantly improve patient outcomes as we work to extend survival in some of the most aggressive forms of cancer."

Fourth quarter 2019 operating statistics and financial update

For the quarter ended December 31, 2019, net revenues were $99.2 million, representing 42% growth compared to the fourth quarter 2018.

In the United States, net revenues totaled $65.9 million in the quarter ended December 31, 2019, representing 49% growth compared to the same period in 2018.
In Germany and other EMEA markets, net revenues totaled $25.8 million in the quarter ended December 31, 2019, representing 14% growth compared to the same period in 2018.
In Japan, net revenues totaled $5.6 million in the quarter ended December 31, 2019, representing 95% growth compared to the same period in 2018.
In Greater China, net revenues totaled $1.9 million in the quarter ended December 31, 2019, representing 138% growth compared to the same period in 2018.
There were 2,909 active patients at December 31, 2019, representing 22% growth compared to December 31, 2018, and six percent growth compared to September 30, 2019.

In the United States, there were 1,952 active patients at December 31, 2019, representing 19% growth compared to December 31, 2018.
In Germany and other EMEA markets, there were 765 active patients at December 31, 2019, representing 17% growth compared to December 31, 2018.
In Japan, there were 192 active patients at December 31, 2019, representing 109% growth compared to December 31, 2018.
Additionally, 1,380 prescriptions were received in the quarter ended December 31, 2019, representing five percent growth compared to the same period in 2018, and five percent growth compared to the quarter ended September 30, 2019. In the quarter ended December 31, 2019, 1,079 Optune prescriptions were written for patients with newly diagnosed glioblastoma.

In the United States, 1,002 prescriptions were received in the quarter ended December 31, 2019, representing six percent growth compared to the same period in 2018.
In Germany and other EMEA markets, 285 prescriptions were received in the quarter ended December 31, 2019, representing an 11% decrease compared to the same period in 2018.
In Japan, 93 prescriptions were received in the quarter ended December 31, 2019, representing 79% growth compared to the same period in 2018.
For the three months ended December 31, 2019, cost of revenues was $24.8 million compared to $23.0 million for the same period in 2018, representing an increase of 8%. The increase was primarily due to the cost of shipping transducer arrays to a higher volume of commercial patients. Gross margin was 75% for the three months ended December 31, 2019 and 67% for the three months ended December 31, 2018.

Research, development and clinical trials expenses for the three months ended December 31, 2019, were $23.7 million compared to $15.0 million for the same period in 2018, representing an increase of 58%. This was primarily due to an increase in clinical development expenses driven by our ongoing phase 3 pivotal trials and an increase in costs associated with product development and medical affairs.

Sales and marketing expenses for the three months ended December 31, 2019, were $26.8 million compared to $21.2 million for the same period in 2018, representing an increase of 26%. This was primarily due to increased marketing expenses related to the launch of NovoTTF-100L and an increase in personnel costs to support our growing commercial business.

General and administrative expenses for the three months ended December 31, 2019 were $23.8 million compared to $19.1 million for the same period in 2018, representing an increase of 25%. This was primarily due to an increase in personnel costs and an increase in professional services.

Net income for the three months ended December 31, 2019, was $4.3 million compared to net loss of $15.6 million for the same period in 2018.

At December 31, 2019, we had $177.3 million in cash and cash equivalents and $148.8 million in short-term investments, for a total balance of $326.1 million in cash, cash equivalents and short-term investments. This represents an increase of $13.5 million in cash and investments since September 30, 2019.

Fourth quarter 2019 non-U.S. GAAP measures

We also measure our performance based upon a non-U.S. GAAP measurement of earnings before interest, taxes, depreciation, amortization and shared-based compensation ("Adjusted EBITDA"). We believe Adjusted EBITDA is useful to investors in evaluating our operating performance because it helps investors evaluate and compare the results of our operations from period to period by removing the impact of earnings attributable to our capital structure, tax rate and material non-cash items, specifically share-based compensation.

Adjusted EBITDA increased by $13.1 million, or 314%, to $17.3 million for the three months ended December 31, 2019 from $4.2 million for the three months ended December 31, 2018. This improvement in fundamental financial performance was driven by top-line growth and disciplined execution.

Anticipated clinical milestones

Interim analysis of phase 3 pivotal LUNAR trial in non-small cell lung cancer (H2 2020)
Data from phase 2 pilot HEPANOVA trial in advanced liver cancer (2021)
Data from phase 3 pivotal METIS trial in brain metastases (2021)
Interim analysis of phase 3 pivotal PANOVA-3 trial in locally advanced pancreatic cancer (2021)
Final data from phase 3 pivotal LUNAR trial in non-small cell lung cancer (2022)
Interim analysis of phase 3 pivotal INNOVATE-3 trial in recurrent ovarian cancer (2022)
Final data from phase 3 pivotal PANOVA-3 trial in locally advanced pancreatic cancer (2022)
Final data from phase 3 pivotal INNOVATE-3 trial in recurrent ovarian cancer (2024)
Conference call details

Novocure will host a conference call and webcast to discuss fourth quarter and full year 2019 financial results at 8 a.m. EST today, Thursday, February 27, 2020. Analysts and investors can participate in the conference call by dialing 855-442-6895 for domestic callers and 509-960-9037 for international callers, using the conference ID 1769503.

The webcast, earnings slides presented during the webcast and the corporate presentation can be accessed live from the Investor Relations page of Novocure’s website, www.novocure.com/investor-relations, and will be available for at least 14 days following the call. Novocure has used, and intends to continue to use, its investor relations website, as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD.

On February 27, 2020 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, reported financial results for the fourth quarter and year ended December 31, 2019, and provided a business update (Press release, Adaptimmune, FEB 27, 2020, View Source [SID1234554922]).

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"The last 12 months have been transformative. We reported responses in five different solid tumors, confirming that our SPEAR T-cell platform can treat a wide range of cancers. We also validated the potential of our allogeneic platform by demonstrating that we can generate functional T-cells from stem cells, and by signing our first major strategic deal in five years with Astellas," said Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer. "With our passionate and skilled teams, and our world class capabilities, we are developing our cell therapy pipeline for a range of tumor indications, aiming to launch our first product in 2022 for people with sarcoma."

Responses in five solid tumor indications demonstrate SPEAR T-cell potential to treat cancer

Based on compelling response data in synovial sarcoma from the Phase 1 trial announced in May of last year, and updated at ESMO (Free ESMO Whitepaper) and CTOS, the Phase 2 SPEARHEAD-1 trial was initiated with ADP-A2M4 in synovial sarcoma and myxoid/ round cell liposarcoma (MRCLS). The product was granted Orphan Drug Designation, for the treatment of soft tissue sarcomas, and Regenerative Medicine Advanced Therapy designation, for synovial sarcoma, by the US FDA. The Company aims to launch ADP-A2M4 for sarcoma in 2022.

In January of this year, partial responses in liver, melanoma, gastro-esophageal junction, and head and neck cancers were reported. These early data in multiple indications demonstrate the potential of Adaptimmune’s SPEAR T-cell platform across multiple targets and a range of solid tumors. Further updated data will be presented at upcoming medical / scientific meetings.

Partnerships to develop next-generation and off-the-shelf cell therapies

In January of this year, a co-development and co-commercialization agreement with Astellas, through its wholly owned subsidiary Universal Cells, Inc., was announced for stem-cell derived allogeneic CAR-T and TCR T-cell therapies. The Company has received an upfront payment of $50 million under the agreement and is entitled to receive research funding of up to $7.5 million per year.

This agreement covers the co-development and co-commercialization of up to three T-cell therapies and leverages Astellas’ Universal Donor Cell Platform and Adaptimmune’s stem-cell derived allogeneic T-cell platform. This new collaboration may encompass both CAR-T and TCR T-cell approaches, including Adaptimmune’s novel HLA-independent TCR ("HiT") platform.

In 2019, Adaptimmune announced agreements with Alpine Immune Sciences and Noile-Immune to develop further next-generation products.

Leadership, manufacturing and financial updates strengthen fully integrated cell therapy company position

Adrian Rawcliffe assumed the role of Chief Executive Officer effective September 1, 2019 and John Lunger became Chief Patient Supply Officer effective August 1, 2019. In January 2020, a series of changes to the R&D leadership were announced, including the appointment of Elliot Norry as Chief Medical Officer. These leadership changes strengthen the scientific and clinical organization from early to late stage and accelerate the application of translational science learnings to therapeutic candidates and trials, as Adaptimmune becomes a late-stage cell therapy company aiming to launch a commercial product in 2022.

Adaptimmune’s in-house cell manufacturing facility located at the Navy Yard in Philadelphia, PA, is achieving a 25-day processing time for production of SPEAR T-cells. 95% of patient batches manufactured in 2019 met manufacturing criteria set for those batches. The Navy Yard facility was approved as a manufacturing source for a number of the Company’s clinical trials in Europe. The Company also produced its first GMP batch of lentiviral vector using an in-house, proprietary suspension process at its dedicated manufacturing space within the Cell and Gene Therapy Catapult Manufacturing Centre at Stevenage, UK.

Finally, on January 24, 2020, the Company closed an underwritten public offering of 21,000,000 American Depository Shares (ADSs) which, together with the full exercise by the underwriters on February 7, 2020 of their option to purchase an additional 3,150,000 ADSs, generated net proceeds of approximately $89.8 million. Following the agreement with Astellas and the public offering of ADSs described above, the Company is funded into 2H 2021.

Planned 2020 milestones

First Half of 2020

·Full summary of the ADP-A2M4 Phase 1 trial at a medical conference
·Safety update on Cohorts 1 and 2 of the ADP-A2AFP Phase 1 trial at a medical conference
·Update on Cohort 3 of the ADP-A2AFP Phase 1 trial at a medical conference
·initiation of PD1 / PDL1 inhibitor combination trial with ADP-A2M4 in head & neck cancer
·Data update from allogeneic program at a scientific meeting

Second Half of 2020

·12-month durability data from patients with synovial sarcoma from the ADP-A2M4 Phase 1 trial at a medical conference
·Updates on dose escalation cohorts from the SURPASS trial at medical conferences
·Update on the ADP-A2M4 Phase 1 radiation sub-study at a medical conference
·Update on the ADP-A2AFP expansion cohort at a medical conference

Financial Results for the fourth quarter and year ended December 31, 2019

·Cash / liquidity position: As of December 31, 2019, Adaptimmune had cash and cash equivalents of $50.4 million and Total Liquidity1 of $89.5 million.
· Revenue: Revenue for the fourth quarter and year ended December 31, 2019 were $0.7 million and $1.1 million compared to $1.5 million and $59.5 million for the same periods of 2018. The revenue recognized for the year ended December 31, 2019 is due to development work on the third target program under the GSK Collaboration and License Agreement. Revenue for the year ended December 31, 2018 included $39.1 million of license revenue and $20.4 million of development revenue due to the performance under the NY-ESO transition program and the PRAME development plan, which were completed in 2018.
·Research and development ("R&D") expenses: R&D expenses for the fourth quarter and year ended December 31, 2019 were $20.4 million and $97.5 million, compared to $22.8 million and $98.3 million for the same periods of 2018. The decrease in R&D expenses for the year ended December 31, 2019 was driven by a reduction in subcontracted expenses and clinical trial costs following the transfer of NY-ESO to GSK in 2018 and a decrease in share-based compensation expenditure, offset by recognition of accrued purchase commitments for clinical materials and payments for in-process research and development (following collaborations with Alpine Immune Sciences, Inc. and Noile-Immune Biotech, Inc.).
·General and administrative ("G&A") expenses: G&A expenses for the fourth quarter and year ended December 31, 2019 were $10.7 million and $43.4 million, compared to $10.8 million and $43.6 million for the same periods of 2018.
·Net loss: Net loss attributable to holders of the Company’s ordinary shares for the fourth quarter and year ended December 31, 2019 was $29.4 million and $137.2 million ($(0.22) per ordinary share) compared to $36.2 million and $95.5 million ($(0.16) per ordinary share) in the same periods of 2018.

Financial Guidance

The Company believes that its existing cash and cash equivalents and marketable securities, Total Liquidity, together with the net proceeds received from the underwritten public offering in January 2020, the additional net proceeds generated from the exercise in full of the underwriters’ option in February 2020 and the upfront payment received under its agreement with Astellas in January 2020, will fund the Company’s current operating plan into the second half of 2021.

Conference Call Information

The Company will host a live teleconference and webcast to provide additional details at 8:00 a.m. EST (1:00 p.m. GMT) today, February 27, 2020. 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, if preferred, please dial (833) 652-5917 (U.S. or Canada) 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 (6083408).