On October 29, 2020 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported that the company will deliver two poster presentations at The American Society of Dermatopathology (ASDP) 57th Virtual Annual Meeting, to be held on Nov. 5 – 11, 2020 (Press release, Castle Biosciences, OCT 29, 2020, View Source [SID1234569386]).

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Presentation details are as follows:

DecisionDx-Melanoma
Poster #: 592
Title: Identifying predictors of sentinel lymph node metastasis in cutaneous melanoma patients using molecular and clinicopathologic high-risk features
Session: Oral & Poster Abstract Defense 2
Presenter: Federico Monzon, M.D.
Date: Tuesday, November 10, 2020
Time: 11:30 a.m. – 12:30 p.m. Central time

DecisionDx DiffDx-Melanoma
Poster #: 296
Title: Development and validation of a diagnostic gene expression profile test for ambiguous or difficult to diagnose pigmented skin lesions
Session: Oral & Poster Abstract Defense 1
Presenter: Sarah Estrada, M.D., FCAP
Date: Monday, November 9, 2020
Time: 11:00 a.m. – 12:00 p.m. Central time

About DecisionDx-Melanoma

DecisionDx-Melanoma is a gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous melanoma metastasis or recurrence, as well as sentinel lymph node positivity, independent of traditional staging factors, and has been studied in more than 5,700 patient samples. Using tissue from the primary melanoma, the test measures the expression of 31 genes. The test has been validated in four archival risk of recurrence studies of 901 patients and six prospective risk of recurrence studies including more than 1,600 patients. Prediction of the likelihood of sentinel lymph node positivity has also been validated in two prospective multicenter studies that included more than 3,000 patients. Impact on patient management plans for one of every two patients tested has been demonstrated in four multicenter and single-center studies including more than 560 patients. The consistent performance and accuracy demonstrated in these studies provides confidence in disease management plans that incorporate DecisionDx-Melanoma test results. Through June 30, 2020, DecisionDx-Melanoma has been ordered more than 59,900 times for use in patients with cutaneous melanoma.

More information about the test and disease can be found at www.SkinMelanoma.com.

About Suspicious Pigmented Lesions

In the U.S., approximately 2 million skin biopsies of pigmented or melanocytic lesions are performed annually, which Castle estimates leads to the diagnosis of 130,000 invasive melanoma cases and more than 96,000 in situ melanoma cases. These biopsies are typically pigmented lesions for which the healthcare provider suspects melanoma. The majority of these biopsies receive a definitive diagnosis by the dermatopathologist using traditional microscopic analyses. However, approximately 300,000 of these biopsies are considered difficult-to-diagnose using this traditional method and require additional testing to clarify the likelihood that this lesion is benign or malignant. And though there are several options for further testing, Castle believes there is a need for improvements in objective tests to resolve this diagnostic dilemma.

On October 29, 2020 Arcus Biosciences, Inc. (NYSE:RCUS), an oncology-focused biopharmaceutical company working to create best-in-class cancer therapies, reported a collaboration with AstraZeneca (LSE/STO/Nasdaq: AZN) to evaluate domvanalimab (AB154), Arcus’s investigational anti-TIGIT antibody, in combination with Imfinzi (durvalumab) in a registrational Phase 3 clinical trial in patients with unresectable Stage III non-small cell lung cancer (NSCLC) (Press release, Arcus Biosciences, OCT 29, 2020, View Source [SID1234569385]). Imfinzi is the only immunotherapy approved for patients with unresectable Stage III NSCLC and was the first significant advancement in over twenty-five years for the treatment of patients with Stage III NSCLC whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy (CRT). The collaboration reflects Arcus’s commitment to ensuring the development of its portfolio of molecules in combinations and settings that maximize their value and bring their potential benefits to the broadest patient population possible.

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"This collaboration provides a unique opportunity for domvanalimab, our novel anti-TIGIT antibody, to be combined with the definitive standard of care in the curative-intent setting of unresectable Stage III NSCLC and to leverage AstraZeneca’s experience, deep knowledge and leadership within this indication," said Bill Grossman, M.D., Ph.D., Chief Medical Officer of Arcus. "With the aggressive development of our anti-TIGIT antibody in this additional setting, we are well positioned to be a leader in both the anti-TIGIT field and more broadly in the creation, development and commercialization of the next generation of innovative immuno-oncology combination therapies."

José Baselga, M.D., Ph.D., Executive Vice President, Oncology R&D of AstraZeneca, said: "This partnership creates an important opportunity to leverage the promise of Arcus’s anti-TIGIT antibody in Stage III NSCLC. This is a promising immunotherapy combination that has the potential to further enhance the efficacy and improvement of long-term survival that Imfinzi has already demonstrated in this setting, and to allow us to unlock the full potential of this medicine."

Results from the PACIFIC Phase 3 trial recently presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) annual meeting demonstrated that treatment with Imfinzi following CRT led to unprecedented survival in unresectable Stage III NSCLC, with an estimated 50% of patients surviving four years versus 36% for CRT alone, and 35% of patients not progressing after four years versus 20% for CRT alone. These data build on those reported in 2018 in The New England Journal of Medicine, demonstrating a significant benefit associated with Imfinzi treatment in the overall survival primary endpoint.

Arcus is currently evaluating domvanalimab, a new potential immuno-oncology backbone therapy in a three-arm randomized Phase 2 study, ARC-7, for first-line treatment of PD-L1-high metastatic NSCLC evaluating (1) zimberelimab monotherapy, versus (2) domvanalimab with zimberelimab versus (3) domvanalimab plus etrumadenant (AB928) with zimberelimab.

Under the terms of the agreement, each company will retain existing rights to their respective molecules and any future commercial economics. AstraZeneca will conduct the trial, and each company will supply its respective anti-cancer agent to support the trial. Pursuant to the terms of the agreement, the parties will share costs for the trial.

Consistent with the terms of the recently completed Arcus-Gilead partnership, Gilead maintains an option to co-develop and co-commercialize domvanalimab. If Gilead exercises its option to domvanalimab, the trial from this AstraZeneca collaboration is expected to form part of the Arcus and Gilead joint development program and Arcus’s portion of the trial costs would be shared with Gilead. The collaboration with AstraZeneca has the potential to expand domvanalimab’s clinical development program, accelerating the pathway to registration and adding value to the Arcus-Gilead alliance.

On October 29, 2020 Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) reported financial results for the third quarter of 2020 (Press release, Alexion, OCT 29, 2020, View Source [SID1234569384]). Total revenues in the third quarter were $1,588.7 million, a 26 percent increase compared to the same period in 2019. The negative impact of foreign currency on total revenues year-over-year was 2 percent, or $25.5 million, inclusive of hedging activities. On a GAAP basis, diluted EPS in the quarter was $2.62, compared to $2.08 in the prior year. Non-GAAP diluted EPS for the third quarter of 2020 was $3.24, a 16 percent increase versus the third quarter of 2019.

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"We have continued to build on our momentum from the first half of the year, delivering another strong quarter despite the ongoing challenges and uncertainty surrounding COVID-19. In the third quarter, we further progressed our LEAD-EXPAND-DIVERSIFY strategy with multiple regulatory approvals, the initiation of new Phase 3 trials and the integration of the Portola team," said Ludwig Hantson, Ph.D., Chief Executive Officer of Alexion. "Our foundation for the future is stronger than ever, and by maintaining our focus on serving patients and delivering for shareholders, I am confident that we will continue to build on our success to date and further advance our mission of delivering life-changing therapies to people with rare diseases and devastating conditions."

Third Quarter 2020 Financial Highlights

Net product sales were $1,588.3 million in the third quarter of 2020, compared to $1,263.1 million in the third quarter of 2019.
SOLIRIS net product sales were $1,042.3 million, compared to $990.5 million in the third quarter of 2019, representing a 5 percent increase.
ULTOMIRIS net product sales were $289.3 million, compared to $89.9 million in the third quarter of 2019, representing a 222 percent increase.
STRENSIQ net product sales were $189.4 million, compared to $154.3 million in the third quarter of 2019, representing a 23 percent increase.
KANUMA net product sales were $28.4 million in both of the third quarters of 2020 and 2019.
ANDEXXA/ONDEXXYA net product sales were $38.9 million in the third quarter of 2020.
GAAP cost of sales was $144.7 million, compared to $95.2 million in the third quarter of 2019. Non-GAAP cost of sales was $129.8 million, compared to $91.8 million in the third quarter of 2019.
GAAP R&D expense was $285.9 million, compared to $232.9 million in the third quarter of 2019. Non-GAAP R&D expense was $269.3 million, compared to $186.1 million in the third quarter of 2019.
GAAP SG&A expense was $334.2 million, compared to $299.3 million in the third quarter of 2019. Non-GAAP SG&A expense was $301.3 million, compared to $260.4 million in the third quarter of 2019.
GAAP income tax expense was $88.8 million, compared to $67.9 million in the third quarter of 2019. Non-GAAP income tax expense was $135.1 million, compared to $82.5 million in the third quarter of 2019.
GAAP diluted EPS was $2.62, compared to $2.08 in the third quarter of 2019. Non-GAAP diluted EPS was $3.24, compared to $2.79 in the third quarter of 2019.
COVID-19

We continue to take steps to proactively respond to the evolving COVID-19 pandemic and to plan for related uncertainties. We remain focused on continuing to serve patients, protecting the health and safety of our employees and the communities in which we live and work, and supporting patients in clinical trials. We are also focused on minimizing potential interactions that could contribute to the spread of the virus and put additional strain on healthcare systems through the use of innovative virtual means where possible.

Clinical Trials: We have implemented a pandemic response business continuity plan designed to protect patients and site staff safety while continuing our clinical trials with limited interruption to the extent we are able. The COVID-19 impact has varied by study and program, but there has been little timing impact on fully-enrolled trials. We have successfully re-initiated the majority of studies that had been temporarily paused. There has been, and may continue to be, an impact to the timing of trials that are enrolling patients and activating sites, or have not yet started to do so, based on local dynamics where these studies are being conducted.
Business Impact: We continue to take proactive measures designed to mitigate the risk of potential interruptions in supply and/or access to patients’ customary site-of-care locations. Treatment compliance rates across all our medicines have remained strong and continue to be slightly above expectations. We have also seen the predicted slowing of new patient initiations and delays in treatment starts, and we are continuing to closely monitor this environment as the pandemic continues.
Research and Development

PHASE 3/4

SOLIRIS – Neuromyelitis Optica Spectrum Disorder (NMOSD): A Phase 2/3 study of SOLIRIS in children and adolescents with NMOSD is underway.
SOLIRIS – Generalized Myasthenia Gravis (gMG): A Phase 3 study of SOLIRIS in children and adolescents with gMG is underway.
SOLIRIS – Guillain-Barre Syndrome (GBS): SOLIRIS in GBS has been granted SAKIGAKE designation by Japan’s Ministry of Health, Labour and Welfare (MHLW). Alexion plans to initiate a Phase 3 study of SOLIRIS in GBS in Japan in 2021, pending regulatory feedback.
ULTOMIRIS – Severe COVID-19: A Phase 3 randomized controlled trial of ULTOMIRIS in adults with COVID-19 who are hospitalized with severe pneumonia or acute respiratory distress syndrome is underway.
ULTOMIRIS – Paroxysmal Nocturnal Hemoglobinuria (PNH): A Phase 3 study of ULTOMIRIS in children and adolescents with PNH is underway.
ULTOMIRIS – Atypical Hemolytic Uremic Syndrome (aHUS): In September 2020, Japan’s MHLW approved ULTOMIRIS for adults and children with aHUS. A global Phase 3 study of ULTOMIRIS in children and adolescents with aHUS is underway.
ULTOMIRIS – 100 mg/mL: In October 2020, the U.S. FDA approved the ULTOMIRIS 100 mg/mL formulation for PNH and aHUS. In September 2020, Alexion announced a positive opinion for the ULTOMIRIS 100 mg/mL formulation from the European Medicines Agency Committee for Medicinal Products for Human Use. An application for approval is under review in Japan. This higher concentration formulation is designed to reduce infusion time by more than 60 percent to approximately 45 minutes.
ULTOMIRIS – Subcutaneous: The Phase 3 study of weekly subcutaneous (SC) ULTOMIRIS demonstrated PK-based non-inferiority versus intravenous ULTOMIRIS. Pending collection of 12-month safety and drug-device combination data, Alexion plans to file for approval in the U.S. and EU for the ULTOMIRIS SC formulation and device combination in PNH and aHUS in the third quarter of 2021.
ULTOMIRIS – gMG: As completion of full enrollment nears, screening of new patients has closed for the Phase 3 study of ULTOMIRIS in adults with gMG.
ULTOMIRIS – NMOSD: A Phase 3 study of ULTOMIRIS in NMOSD is underway.
ULTOMIRIS – Amyotrophic Lateral Sclerosis (ALS): A Phase 3 study of ULTOMIRIS in ALS is underway.
ULTOMIRIS – Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA): Alexion has initiated a Phase 3 study of ULTOMIRIS in adults with HSCT-TMA and plans to initiate a Phase 3 study in children with HSCT-TMA by the end of 2020.
ULTOMIRIS – Complement Mediated Thrombotic Microangiopathy (CM-TMA): Alexion plans to initiate a Phase 3 study of ULTOMIRIS in CM-TMA in the first half of 2021, pending regulatory feedback.
ALXN1840 – Wilson Disease: Enrollment is complete in a Phase 3 study of ALXN1840 in Wilson disease. Study results are expected in the first half of 2021.
CAEL-101 – Caelum Biosciences: In September 2020, Alexion and Caelum Biosciences announced the initiation of the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care therapy in AL amyloidosis. Enrollment is underway in two parallel Phase 3 studies – one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease.
ALXN2060 (AG10) – Eidos: Alexion holds an exclusive license to develop and commercialize ALXN2060 (AG10) in Japan. Eidos is currently evaluating AG10 in a Phase 3 study in the U.S. and Europe for ATTR cardiomyopathy (ATTR-CM) and plans to begin a Phase 3 study in ATTR polyneuropathy (ATTR-PN) in the second half of 2020. Alexion plans to initiate a Phase 3 bridging study of ALXN2060 for patients with ATTR-CM in Japan by the end of 2020.
ALXN2040 (Danicopan) – PNH with Extravascular Hemolysis (EVH): Alexion plans to initiate a Phase 3 study of ALXN2040 as an add-on therapy for PNH patients with EVH by the end of 2020.
ANDEXXA – Acute Intracranial Hemorrhage (ICH): The Phase 4 ANNEXA-I study – designed to provide clinical data supporting full approval – is underway to assess ANDEXXA compared to usual standard of care in patients presenting with acute intracranial hemorrhage while taking an oral Factor Xa inhibitor.
PHASE 1/2

ULTOMIRIS – Renal Diseases: Alexion plans to initiate a proof-of-concept trial of ULTOMIRIS in patients with IgA nephropathy and lupus nephritis in 2020.
ALXN1830: Due to COVID-19, Alexion discontinued the Phase 2 study of ALXN1830, administered intravenously, in warm autoimmune hemolytic anemia (WAIHA) and paused the Phase 1 study of a subcutaneous formulation of ALXN1830 in healthy volunteers. The paused Phase 1 study and new Phase 2 studies of subcutaneous ALXN1830 in gMG and WAIHA are planned to begin in 2021.
ALXN2040 – Geographic Atrophy (GA): Alexion plans to initiate a Phase 2 study of ALXN2040 in GA in the second half of 2021.
ALXN2050 – PNH: A Phase 2 study of ALXN2050 monotherapy in PNH is underway.
ALXN2050 – Renal Diseases: Alexion plans to initiate a proof-of-concept trial of ALXN2050 in patients with various renal diseases in 2021, pending regulatory feedback.
ALXN1720: Seven of nine cohorts are complete in a Phase 1 healthy volunteer study of ALXN1720, a novel anti-C5 albumin-binding bi-specific mini-body that is designed to bind and prevent activation of human C5. Remaining cohorts of the study, which had previously been paused due to COVID-19, have been restarted. Data are expected in the first half of 2021. Following successful completion of the Phase 1 study, Alexion plans to initiate Phase 2 studies of ALXN1720 in gMG and dermatomyositis (DM), pending regulatory feedback.
ANDEXXA – Urgent Surgery: ANDEXXA is currently being evaluated in a single-arm, open-label study in patients taking apixaban, rivaroxaban, edoxaban, or enoxaparin who require urgent surgery. The results of this study will inform the design of a randomized controlled clinical trial to expand the label in this population.
Cerdulatinib: Acquired as part of the Portola acquisition, cerdulatinib is a dual spleen tyrosine kinase and janus kinase (SYK/JAK) inhibitor being evaluated in a Phase 1/2a study in patients with relapsed/refractory chronic lymphocytic leukemia or B-cell or T-cell non-Hodgkin lymphoma.
Updated 2020 financial guidance assumes a GAAP effective tax rate of (5.0) to (4.5) percent and a non-GAAP effective tax rate of 15.5 to 16.0 percent. The 2020 GAAP and non-GAAP tax rates do not benefit from one-time events that benefited the tax rates in 2019.

Alexion’s financial guidance is based on current foreign exchange rates net of hedging activities and does not include the effect of acquisitions, license and other strategic agreements, intangible asset impairments, litigation charges, changes in fair value of contingent consideration, gains or losses related to strategic equity investments or restructuring and related activity outside of the previously announced activities that may occur after the issuance of this press release.

Conference Call/Webcast Information:

Alexion will host a conference call/audio webcast to discuss the third quarter 2020 results today at 8:00 a.m. Eastern Time. To participate in the call, dial 866-762-3111 (USA) or 210-874-7712 (International), conference ID 6582445 shortly before 8:00 a.m. Eastern Time. A replay of the call will be available for a limited period following the call. The audio webcast can be accessed on the Investor page of Alexion’s website at: View Source

On October 29, 2020 Novocure (NASDAQ: NVCR) reported financial results for the quarter ended September 30, 2020, highlighting revenue growth and financial strength as well as the advancement of the company’s clinical and product development programs (Press release, NovoCure, OCT 29, 2020, View Source [SID1234569383]). 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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(1) Adjusted EBITDA is a non-U.S. GAAP measurement of earnings before interest, taxes, depreciation, amortization and share-based compensation.

(2) 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.

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

"We delivered another record quarter of financial performance with $133 million in net revenues and $0.09 in earnings per share," said William Doyle, Novocure’s Executive Chairman. "Our financial strength positions us well to invest in our commercial, clinical and engineering priorities to sustain long-term growth and maximize shareholder value. With readouts from key clinical trials in multiple indications anticipated over the next few years and an increased focus on technology innovation, our confidence in the potential of Tumor Treating Fields to extend survival in some of the most aggressive forms of cancer continues to build."

"We further strengthened our foundation for growth in Q3 with efforts underway to ensure organizational readiness for the company’s next chapter," added Asaf Danziger, Novocure’s Chief Executive Officer. "We ended the quarter with 3,361 active patients on therapy, adding to the more than 17,000 patients treated to-date, globally. With plans to expand access to our approved indications into additional markets, we remain focused on positioning our company to serve many more patients in the future."

Third quarter 2020 financial update

For the quarter ended September 30, 2020, net revenues were $132.7 million, representing 44% growth compared to the third quarter 2019.

In the United States, net revenues totaled $92.6 million in the quarter ended September 30, 2020, representing 51% growth compared to the same period in 2019.
In Germany and other EMEA markets, net revenues totaled $28.2 million in the quarter ended September 30, 2020, representing 15% growth compared to the same period in 2019.
In Japan, net revenues totaled $7.5 million in the quarter ended September 30, 2020, representing 57% growth compared to the same period in 2019.
In Greater China, net revenues totaled $4.3 million in the quarter ended September 30, 2020, representing 205% growth compared to the same period in 2019.
For the three months ended September 30, 2020, the increase resulted primarily from an increase of 610 active patients in our currently active markets, and a durable improvement in the net revenues booked per active patient, as well as an increase in collaboration revenues from our partnership with Zai Lab.

We recorded $10 million in revenues from Medicare fee-for-service beneficiaries billed under the coverage policy effective on September 1, 2019 in the third quarter 2020. We have gained a good understanding of how to ensure timely processing of Medicare claims and have sufficient experience to recognize approximately two-thirds of the expected contribution from Medicare beneficiaries. In the third quarter 2020, we also recognized approximately $8 million in incremental net revenues compared to the first two quarters of 2020 resulting from the successful appeal of previously denied claims for Medicare fee-for-service beneficiaries billed prior to established coverage.

Cost of revenues for the three months ended September 30, 2020 was $28.4 million compared to $22.9 million for the same period in 2019, representing an increase of 24%. The increase in cost of revenues was primarily due to the cost of shipping transducer arrays to a higher volume of commercial patients and increasing shipments of equipment to Zai Lab, partially offset by benefits of ongoing efficiency initiatives and scale. Gross margin was 79% for the three months ended September 30, 2020 compared to 75% for the three months ended September 30, 2019.

Research, development and clinical trials expenses for the three months ended September 30, 2020 were $32.8 million compared to $18.8 million for the same period in 2019, representing an increase of 75%. This was primarily due to an increase in clinical trial and personnel expenses for our phase 3 pivotal and post-marketing trials, an increase in development and personnel expenses to support our product development programs, increased investments in preclinical research and the expansion of our medical affairs activities.

Sales and marketing expenses for the three months ended September 30, 2020 were $29.4 million compared to $23.8 million for the same period in 2019, representing an increase of 23%. This was primarily due to an increase in personnel and professional services costs to support our growing commercial business and reimbursement efforts and an increase in marketing expenses related to the launch of Optune Lua for MPM.

General and administrative expenses for the three months ended September 30, 2020 were $27.1 million compared to $22.7 million for the same period in 2019, representing an increase of 19%. This was primarily due to an increase in personnel costs, insurance premiums and professional services.

Net income for the three months ended September 30, 2020 was $9.3 million compared to net income of $1.9 million for the same period in 2019.

At September 30, 2020, we had $234.5 million in cash and cash equivalents and short-term investments, a decrease of $91.6 million compared to $326.1 million at December 31, 2019. The decrease in our cash, cash equivalents and short-term investments was primarily due to the prepayment of the 2018 credit facility in the amount of $150 million, partially offset by the cash flow from operations and the exercise of options and proceeds from the issuance of shares.

The 2018 credit facility prepayment included $150 million in principal repayment and $3 million in prepayment premium, plus accrued and unpaid interest and expenses payable through the payoff date. The un-amortized issuance costs in the amount of $0.5 million that were fully amortized upon the repayment and the prepayment premium were included in the Company’s third quarter 2020 finance expenses.

Third quarter 2020 operating statistics

There were 3,361 active patients at September 30, 2020, representing 22% growth compared to September 30, 2019, and 3% growth compared to June 30, 2020.

In the United States, there were 2,218 active patients at September 30, 2020, representing 19% growth compared to September 30, 2019.
In Germany and other EMEA markets, there were 902 active patients at September 30, 2020, representing 23% growth compared to September 30, 2019.
In Japan, there were 241 active patients at September 30, 2020, representing 51% growth compared to September 30, 2019.
Additionally, 1,371 prescriptions were received in the quarter ended September 30, 2020, representing 4% growth compared to the same period in 2019, and a 4% decrease compared to the quarter ended June 30, 2020. In the quarter ended September 30, 2020, 1,117 Optune prescriptions were written for patients with newly diagnosed glioblastoma.

In the United States, 955 prescriptions were received in the quarter ended September 30, 2020, representing a 4% increase compared to the same period in 2019.
In Germany and other EMEA markets, 330 prescriptions were received in the quarter ended September 30, 2020, representing 4% growth compared to the same period in 2019.
In Japan, 86 prescriptions were received in the quarter ended September 30, 2020, representing 2% growth compared to the same period in 2019.
Third quarter 2020 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 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 was $37.3 million for the three months ended September 30, 2020, an increase of $17.2 million, or 85%, from $20.1 million for the three months ended September 30, 2019. This improvement in fundamental financial performance was driven by net revenue growth coupled with an ongoing commitment to disciplined management of expenses.

Anticipated clinical milestones

Data from phase 2 pilot HEPANOVA trial in advanced liver cancer (Q1 2021)
Data from phase 2 pilot EF-31 trial in gastric cancer (2021)
Interim analysis of phase 3 pivotal LUNAR trial in non-small cell lung cancer (2021)
Interim analysis of phase 3 pivotal PANOVA-3 trial in locally advanced pancreatic cancer (2021)
Interim analysis of phase 3 pivotal INNOVATE-3 trial in recurrent ovarian cancer (2021)
Data from phase 3 pivotal METIS trial in brain metastases (2022)
Data from phase 2 pilot EF-33 trial with high-intensity arrays in recurrent glioblastoma (2022)
Final data from phase 3 pivotal LUNAR trial in non-small cell lung cancer (2023)
Final data from phase 3 pivotal PANOVA-3 trial in locally advanced pancreatic cancer (2023)
Final data from phase 3 pivotal INNOVATE-3 trial in recurrent ovarian cancer (2023)
Conference call details

Novocure will host a conference call and webcast to discuss second quarter 2020 financial results at 8 a.m. EDT today, Thursday, October 29, 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 5453859.

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 October 29, 2020 NOXXON Pharma N.V. (Euronext Growth Paris: ALNOX), a biotechnology company focused on improving cancer treatments by targeting the tumor microenvironment (TME), reported its interim 2020 results for the six months ended June 30, 2020 (Press release, NOXXON, OCT 29, 2020, View Source [SID1234569381]).

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"Despite the challenging operational environment posed by COVID-19, the NOXXON team, along with dedicated clinical researchers, was able to finalize the NOX-A12 plus immunotherapy trial in pre-treated microsatellite stable metastatic pancreatic and colorectal cancer patients. The final trial results including overall survival and the safety profile warrant further clinical development of NOX-A12 plus immunotherapy combinations. We also advanced our ongoing NOX-A12 plus radiotherapy trial in first-line brain cancer patients," said Aram Mangasarian, CEO of NOXXON. "In addition, the balance sheet of the company has been significantly strengthened and simplified, putting NOXXON in better negotiating position with potential industrial and financial partners."

Business Overview

NOXXON has been focused on clinical trials combining NOX-A12, its anti-CXCL12 tumor microenvironment targeting agent, in two distinct therapeutic combinations: 1) NOX-A12 plus immunotherapy (anti-PD1 checkpoint inhibitors) and 2) NOX-A12 plus radiotherapy. Each combination approach has a different underlying rationale and mechanism of action, and thus diversifies the risk of NOXXON’s clinical pipeline.

The combination approach of NOX-A12 plus standard of care radiotherapy is currently being tested in a dose escalation Phase 1/2 trial in newly diagnosed patients with aggressive brain cancer (glioblastoma) who would not benefit from standard of care chemotherapy and whose tumor cannot be fully resected by surgery. At multiple points in the trial, the independent Data Safety Monitoring Board (DSMB) reviewed safety and tolerability of the NOX-A12 combination and each time concluded that the trial should continue as planned. All patients in the low dose cohort completed six months of therapy in October 2020 and the data are very encouraging. Tumor volume reductions were observed in two of three patients during the six-month treatment, and in the third patient in the period after a second surgery following continued NOX-A12 treatment. Maximum tumor volume reductions were 6% and 60% for the first two patients. The third patient experienced 23% tumor volume reduction relative to the post-second surgery baseline. All patients in the mid dose cohort have been enrolled, with the first patient completing four months of combination therapy and other patients receiving their initial doses of NOX-A12 in October 2020. This means that six months of therapy for Cohort 2 will complete in April 2021.

NOXXON plans to advance the NOX-A12 plus radiotherapy combination in first-line brain cancer if the ongoing Phase 1/2 data warrant additional studies. NOXXON believes that such a pivotal trial following the current study could allow first filing of a market approval application for NOX-A12 in 2024 with first market approval targeted for 2025 if data are positive.

The Phase 1/2 trial studying the combination of NOX-A12 plus immunotherapy in metastatic pancreatic and colorectal cancer patients who had failed standard therapy reported final top-line data in September 2020. Both the NOX-A12 mechanistic data as well as the overall survival figures observed following treatment with the combination of NOX-A12 and anti-PD1 have been highly encouraging for the patient population treated in this study. The patients enrolled in the trial all had advanced disease with liver metastases and received on average their sixth-line of therapy in colorectal cancer and their fourth-line of therapy in pancreatic cancer. Despite the advanced disease and heavy pre-treatment, overall survival at one year was 20%, assessed using the Kaplan-Meier method. Notably, this group of longer-term survivors included two pancreatic cancer patients who had received their fourth-line of treatment.

NOXXON is planning to test the NOX-A12 plus immunotherapy combination in second-line pancreatic cancer with a dosing regimen of NOX-A12 optimized to induce anti-tumor immune responses. A two-step approach is planned for this indication with a first trial comparing two NOX-A12 chemotherapy combinations in second-line patients followed by a pivotal trial comparing the best combination to standard of care. With this approach, completion of the pivotal trial and filing of the first market approval application for this indication could be achieved in 2026 with approval targeted for 2027.

On the financing front, the company was able to raise €11.1 million net cash during the reporting period from a mix of private placements, convertible bonds and warrant exercises, thereby significantly strengthening its balance sheet.

NOXXON is closely monitoring the progress of COVID-19 and its potential impact on its operations. As requested by the European Medicines Agency (EMA), NOXXON has critically assessed the risks and benefits of therapy continuation and inclusion of new trial participants in its clinical trial of NOX-A12 combined with radiotherapy in first-line brain cancer patients. Following a thorough evaluation and discussion with the partners involved in the trial, it has been decided to continue both the treatment of enrolled patients and recruitment of additional patients. The safety of patients, hospital staff and employees, as well as the severity of the disease under study and the limited options currently available for treatment, were important factors in this decision. As there have been delays due to factors including COVID-19, NOXXON has added further centers to the trial to ensure adequate recruitment capacity to meet its targeted timelines. Overall, the impact on trial recruitment, the organization and the staff has been manageable.

The increased interest of investors in healthcare and the shift in the types of investors considering financing small-cap European biotech companies (particularly in France where over 150,000 new investors opened equity investment accounts according to the French regulator, the AMF), broadened the investor base of the capital market and had a positive impact on NOXXON’s ability to raise funds.

Business Highlights During First Half-Year of 2020

Significant strengthening of balance sheet – NOXXON raised €11.1 million net proceeds from multiple sources during the first half of 2020, including €7.3 million via private placements. The Dutch specialist fund Nyenburgh Investment Partners (NYIP) led the largest of the private placements announced on May 8, 2020. In addition, NOXXON has access to a remaining capacity of €16.2 million (nominal) from its convertible bonds financing with Atlas after this financing agreement was amended in October 2020.
Simplified capital structure – Increased price and liquidity during the reporting period allowed the conversion of the vast majority of outstanding warrants held by the investors Acuitas and Yorkville at the beginning of the period.
Timely advancement of NOX-A12 plus radiotherapy trial despite COVID-19– Phase 1/2 clinical trial of NOX-A12 plus radiotherapy in first-line brain cancer patients progressed well despite COVID-19. On April 2, 2020 NOXXON announced completion of patient recruitment for the first dose cohort in the Phase 1/2 brain cancer study of NOX-A12 plus radiotherapy. On April 24, 2020 the DSMB reviewed the available safety data from the low-dose group and validated recruitment of patients in the mid-dose group of NOX-A12. The recruitment of the first patient in the mid-dose group was announced on June 30, 2020.
More mature data from NOX-A12 plus immunotherapy trial –overall survival data from the Phase 1/2 NOX-A12 and immunotherapy combination trial in metastatic pancreatic and colorectal cancer patients supports the benefit to patients from NOX-A12 plus anti-PD-1 therapy. This data was presented by the principal investigator of the trial, Dr. Niels Halama, Head of Department of Translational Immunotherapy at the German Cancer Research Center (DKFZ), Heidelberg and Medical Oncologist at the German National Center for Tumor Diseases, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting on April 27, 2020.
New Supervisory Board Member – Oscar Izeboud joined the Supervisory Board of NOXXON on June 30, 2020. Oscar brings both a deep understanding of medicine and extensive experience in the financing and business side of biotechnology. While leading life science and healthcare investment banking at Kempen and NIBC, Oscar successfully closed more than 100 transactions, including seventeen IPOs and fifteen mergers or acquisitions. This experience combined with his operational biotech background makes him a valuable asset for NOXXON’s strategic development.
Business Highlights After June 30, 2020

July 2020 – NOXXON announced that the first brain cancer patient from the mid-dose cohort in the NOX-A12 plus radiotherapy study reached four weeks of treatment and that the DSMB confirmed safety and validated recruitment of additional patients.
September 2020 – Dr. Niels Halama presented final top-line clinical data from the Phase 1/2 NOX-A12 plus immunotherapy combination trial in colorectal and pancreatic cancer patients at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020.
October 2020 – NOXXON announced that two of the three planned dose cohorts were fully recruited in the NOX-A12 plus radiotherapy clinical trial.
First-half 2020 Financial Results (IFRS)

NOXXON Pharma did not generate any revenues in the first half of 2020 (H1 2020). The Group – NOXXON Pharma N.V. and NOXXON Pharma AG – does not expect to generate any revenues from its product candidates in development until the Group either signs a licensing agreement or obtains regulatory approval and commercializes its products or enters into collaborative agreements with third parties.

Other operating income decreased to €33 thousand in H1 2020 (vs. €274 thousand in H1 2019). Other services provided in 2020 generated lower other operating income than the sale of raw materials and a partial waiver of management and Supervisory Board members concerning their receivables from remuneration due from the Group in H1 2019.

NOXXON dedicated its resources to research and development (R&D) and general and administrative (G&A) expenses. R&D expenses decreased to €942 thousand in H1 2020 (vs. €1,062 thousand in H1 2019). The decrease in R&D expenses was mainly driven by lower costs for drug manufacturing, service fees and other costs related to clinical trials and preclinical testing, patent costs and consulting services, partly offset by higher personnel expenses.

G&A expenses decreased to €988 thousand in H1 2020 (vs. €1,238 thousand in H1 2019). The decrease in G&A expenses was mainly driven by lower legal, consulting and audit fees, lower public and investor relations and related expenses, as well as lower other expenses, partly offset by higher personnel expenses.

Foreign exchange losses increased to €7 thousand in H1 2020 (vs. €2 thousand in H1 2019) as a result of increased volume of purchases denominated in currencies other than Euro in H1 2020.

Finance cost increased from nil in H1 2019 to €4,173 thousand in H1 2020. Finance cost in H1 2020 was predominantly due to the Atlas convertible bonds financing with respect to the issuance and conversion of convertible notes into equity and the recognition of compound derivative financial instruments, the exercise of warrants of the Yorkville equity line financing, the cashless exercise of all remaining Acuitas warrants outstanding and fair value adjustments of warrants outstanding.

Finance cost in H1 2020 was non-cash finance cost, except for €105 thousand, thereof for transaction costs of €103 thousand borne by the company in conjunction with the issuance of convertible bonds.

Finance income (all non-cash) increased to €154 thousand in H1 2020 (vs. €75 thousand in H1 2019). The increase was due to the derecognition gain of compound derivative financial instruments in connection with the Atlas convertible bonds financing in H1 2020.

As a result of the above factors, the Group’s loss before income tax increased to €5,923 thousand in H1 2020 (vs. €1,953 thousand in H1 2019). The net cash used in operating activities amounted to €1,811 thousand in H1 2020 vs. €2,687 thousand in H1 2019.

Outlook

NOXXON is making progress in its ongoing Phase 1/2 trial of NOX-A12 plus radiotherapy in first-line, inoperable brain cancer (glioblastoma) patients who are shown by biomarker analysis of their tumor tissue to be resistant to the current standard of care chemotherapy. Currently, two of the three planned dose cohorts are fully recruited. If study results are positive, NOXXON plans to seek advice from authorities under its EU/US orphan drug designation to confirm that its planned approach is acceptable to complete development and achieve market approval in brain cancer. NOXXON’s partnering goal for this combination is the identification of industrial partners that will finance additional clinical trials in brain cancer and other indications where radiotherapy is core to the standard of care. NOXXON anticipates that at least partial top-line clinical data including post-treatment follow-up from the trial will be required to close a partnership in this area.

NOXXON published more mature data from the NOX-A12 clinical trial in metastatic microsatellite stable pancreatic and colorectal cancer patients in April 2020 and has published final top-line data in September 2020. NOXXON believes that further clinical trials are warranted based on this data, in particular in pancreatic cancer, where it plans to focus its near-term efforts. The goal of NOXXON is to find industrial partners that will not only provide anti-PD1 therapy but also financial support to conduct a trial.

To prepare for future trials leading to approval of NOX-A12, NOXXON has made additional investment commitments for the manufacturing of drug supply for clinical trials.

NOXXON’s long-term strategic plans now include the following trials by indication:

NOX-A12 plus radiotherapy in Brain Cancer

Completion of the ongoing Phase 1/2 dose escalation trial, potentially with an expansion of the dose chosen for the pivotal trial. Trial completion planned for 2021 (without any expansion).
Pivotal trial of NOX-A12 combined with radiotherapy in first-line MGMT promoter unmethylated glioblastoma patients vs. standard of care (assuming ongoing Phase 1/2 trial data supports further development) planned initiation in 2022, with first market authorization application targeted for 2024 and approval targeted for 2025.
NOX-A12 plus immunotherapy in Pancreatic Cancer

Two-arm Phase 2 "pick the winner" trial testing NOX-A12 plus anti-PD1 antibody with two different standard of care chemotherapy regimens to determine the choice of regimen for the pivotal trial. Trial initiation planned for 2021 and completion in 2023.
Pivotal trial of NOX-A12 combined with immunotherapy and standard of care in second-line pancreas cancer vs. standard of care, with market authorization application targeted for 2026 and approval targeted for 2027.
The second clinical stage asset, NOX-E36, is also being prepared for the next clinical trial. Manufacturing of clinical supply has been contracted and is projected to be available in mid-2021. Pre-clinical work comparing combination strategies for NOX-E36 in solid tumors to identify the most promising approaches are also advancing. NOXXON plans to initiate the first clinical trial of NOX-E36 combinations testing safety in 2021.

NOXXON continues to evaluate other indications and therapeutic combinations in which to test NOX‑A12 and NOX-E36 as well as the relative priority of such indications for the overall corporate strategy.

The Group will carefully monitor its available cash and calibrate additional financings through various sources in order to ensure its development plans and, to the extent deemed appropriate, maintenance of a sufficient cash runway. Considering cash and cash equivalents as well as financial assets as of June 30, 2020 of €10.7 million and available, secured financing of €11.5 million (nominal) as well as a subsequent amendment to this financing agreement increasing its capacity by an additional €4.7 million (nominal) drawable at the company’s discretion and subject to customary conditions being met, cash reach of NOXXON will be into Q1 2022, including the above planned manufacturing and clinical trial commitments.

The Half-Year Financial Report 2020 can be downloaded from the NOXXON website.