On January 13, 2020 Novocure (NASDAQ: NVCR) reported operating statistics and preliminary, unaudited net revenues and cash balances for the fourth quarter and full year 2019 (Press release, NovoCure, JAN 13, 2020, View Source [SID1234553102]). Novocure plans to discuss these results with investors at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco. Novocure also plans to highlight key clinical and product development programs.

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Fourth quarter and full year 2019 highlights include:

The preliminary, unaudited results described in this press release are estimates only and are subject to revision until the company reports its full, audited financial results for the fourth quarter and full year 2019 on Feb. 27, 2020.

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.

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 continued to make substantial progress in 2019 towards becoming a global oncology leader with $351 million in net revenues, representing 42% annual revenue growth, and four advancing phase 3 pivotal trials creating the potential for substantial market expansion over the next five years," said Asaf Danziger, Novocure’s CEO. "Our ongoing focus on product innovation generated 33 new patent applications in 2019 and we ended the year with $326 million cash on hand, an increase of $80 million versus 2018."

"At Novocure, we are working to extend survival in some of the most aggressive forms of cancer by developing and commercializing our innovative therapy, Tumor Treating Fields," continued William Doyle, Novocure’s Executive Chairman. "Our established commercial business fuels a strong balance sheet and cash position which provides broad flexibility to support a growing body of clinical evidence and deliver meaningful product innovation. We look forward to providing a full company update and discussing our 2019 financial results on our February conference call."

Fourth quarter 2019 operating statistics and preliminary financial highlights

On a preliminary, unaudited basis, for the quarter ended December 31, 2019, net revenues were $99.2 million, representing 42% growth compared to the fourth quarter 2018, and full year 2019 net revenues were $351.3 million, representing 42% growth compared to December 31, 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.
At December 31, 2019, on a preliminary, unaudited basis, Novocure 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 $80.2 million in cash and investments since September 30, 2019.

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)
Fourth quarter and full year 2019 financial results conference call

Novocure will host a conference call and webcast to discuss fourth quarter and full year 2019 financial results at 8 a.m. EST on 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 January 13, 2020 Oncology Venture A/S ("OV" or the "Company") reported that an update of its ongoing clinical development of the PARP inhibitor, 2X-121, will be presented at an upcoming meeting in Boston on January 28-30th (Press release, Oncology Venture, JAN 13, 2020, View Source [SID1234553101]).

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Oncology Venture, a company developing novel cancer treatments along with its best in class companion diagnostic technology, the Drug Response Predictor (DRP), today announces that an update of the company’s ongoing clinical development of its proprietary PARP inhibitor 2X-121 "Titled: A novel drug specific multigene mRNA biomarker for the PARP/Tankyrase inhibitor 2X-121" has been accepted for poster presentation at the upcoming PARP & DDR Inhibitor Summit taking place in Boston on January 28th-30th, gathering VPs, Heads and Directors of Translational Oncology, Target Discovery, and Early Drug Development companies. The poster includes data from the use of a 2X-121 specific DRP biomarker that the company has developed to enable the selection of patients most likely to respond to the drug.

Title: "A novel drug specific multigene mRNA biomarker for the PARP/Tankyrase inhibitor 2X-121"

Session: Poster Session

Date and Time: January 29th at 3:15 pm Eastern Time

Presenter: Dr. Marie Foegh

"We are delighted to be presenting some of our development data in support of our PARP inhibitor 2X-121 at the upcoming PARP & DDR summit meeting as we expect it will enhance the awareness for our unique compound and the DRP platform." said Steve Carchedi, President and Chief Executive Officer of Oncology Venture. "Our novel multi-targeted approach to hitting both PARP and Tankyrase, we believe will provide an improved clinical impact compared to other PARP inhibitors currently approved and/or under development. We look forward to sharing the data set following its presentation later this month".

The company has chosen to publish this announcement now, even though the company is of the opinion that the contents of this announcement does not constitute information which will impact the company’s share price.

However, in the light of the recent events the company finds that expedited access to this information is relevant in order to provide the highest level of transparency for the members of the general public interested in the news flow from the company.

On January 13, 2020 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported corporate goals for 2020, which provide a path to achieve the company’s "2020 Blueprint" strategy for launching its global commercial business (Press release, Blueprint Medicines, JAN 13, 2020, View Source [SID1234553100]).

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"2020 is off to a great start for Blueprint Medicines with the recent FDA approval of our first medicine and a U.S. launch now underway. As we complete our evolution into a fully-integrated biopharmaceutical company this year, we will also aim to bring a second product to market, expand across multiple indications and extend our global commercial footprint with our first anticipated regulatory approval in Europe," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "In addition, our future growth will be fueled by an expanded strategic focus on systemic mastocytosis and related mast cell disorders, which represent a large population of underserved patients with significant medical needs. This focus is anchored by avapritinib, which is now FDA-approved for the treatment of PDGFRA exon 18 mutant GIST. Avapritinib was specifically designed to treat the underlying cause of systemic mastocytosis and has demonstrated remarkable and consistent clinical activity across the spectrum of the disease."

RECENT PORTFOLIO MILESTONES AND 2020 GOALS

Avapritinib: gastrointestinal stromal tumors (GIST)

In January 2020, announced the U.S. Food and Drug Administration (FDA) granted a full approval to AYVAKIT for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Read the press release here and visit www.AYVAKIT.com for full Prescribing Information.
2020 goals:

Report top-line data from the Phase 3 VOYAGER trial of avapritinib in third-line GIST in the second quarter of 2020
Gain regulatory approval and launch avapritinib in fourth-line GIST in the U.S. in the second quarter of 2020
Gain regulatory approval and launch avapritinib in PDGFRA D842V GIST in Europe in the third quarter of 2020
Present the registration dataset from the VOYAGER trial of avapritinib in third-line GIST in 2020
Submit a supplemental new drug application (NDA) to the FDA for third-line GIST in the second half of 2020
Avapritinib and BLU-263: systemic mastocytosis (SM) and other mast cell disorders

Today announced an update on the planned submission of a supplemental NDA to the FDA for avapritinib for advanced SM. Based on ongoing discussions with the FDA, the company plans to focus its supplemental NDA on data from patients in the Phase 1 EXPLORER and Phase 2 PATHFINDER trials who started at the proposed indicated dose of 200 mg once daily (QD), supported by pooled data from all doses. To allow for a sufficient number of patients and follow-up, Blueprint Medicines now plans to submit the supplemental NDA to the FDA in the second half of 2020.
In December 2019, reported initial data from Part 1 of the PIONEER trial of avapritinib in patients with indolent SM. The data showed rapid and robust reductions in serum tryptase, a measure of mast cell burden, in patients treated with 25, 50 or 100 mg QD of avapritinib. All dose levels of avapritinib tested were well-tolerated, and no patients discontinued treatment due to an adverse event (AE). Read the press release here.
Today announced updated data from Part 1 of the PIONEER trial have been accepted for presentation as a late-breaking oral abstract at the American Academy of Allergy, Asthma & Immunology 2020 Annual Meeting in Philadelphia on March 14, 2020.
2020 goals:

Present updated data from Part 1 of the PIONEER trial of avapritinib in indolent SM in the first quarter of 2020
Initiate a Phase 1 trial of BLU-263 in healthy volunteers in the first half of 2020
Submit a supplemental NDA to FDA for avapritinib for advanced SM in the second half of 2020
Present updated data from the EXPLORER and PATHFINDER trials of avapritinib in advanced SM in the second half of 2020
Complete enrollment of Part 2 of the PIONEER trial of avapritinib in indolent SM by the end of 2020
Pralsetinib: RET-altered cancers

In January 2020, reported top-line data from the Phase 1/2 ARROW trial of pralsetinib in RET fusion-positive non-small cell lung cancer (NSCLC) as of a data cutoff date of November 18, 2019 in patients treated with the proposed indicated dose of 400 mg QD. In patients with previously treated RET fusion-positive NSCLC, the overall response rate (ORR) was 61 percent (95% CI: 50-72%) per central review (two responses pending confirmation), and the median duration of response was not reached. In patients with treatment-naïve RET fusion-positive NSCLC, the ORR was 73 percent (95% CI: 52-88%) per central review (all responses confirmed), with 12 percent of patients achieving a complete response. Pralsetinib was well-tolerated, and most reported AEs were Grade 1 or 2 with only four percent of patients discontinuing treatment with pralsetinib due to treatment-related AEs. In addition, the company announced it had initiated a rolling NDA submission to the FDA for pralsetinib for the treatment of RET fusion-positive NSCLC. Read the press release here.
Today announced the activation of the first trial site for the company’s Phase 3 AcceleRET Lung trial of pralsetinib in patients with first-line RET fusion-positive NSCLC. More information about the trial is available at www.clinicaltrials.gov (NCT04222972).
2020 goals:

Complete the submission of a rolling NDA to the FDA for pralsetinib for RET fusion-positive NSCLC in the first quarter of 2020
Submit an NDA to the FDA for pralsetinib for previously treated RET-mutant medullary thyroid cancer (MTC) in the second quarter of 2020
Submit an MAA to EMA for pralsetinib for RET fusion-positive NSCLC in the second quarter of 2020
Present registration datasets from the Phase 1/2 ARROW trial of pralsetinib in RET fusion-positive NSCLC and RET-mutant MTC in 2020
Initiate a Phase 3 trial of pralsetinib in first-line RET-mutant MTC in the second half of 2020
Gain regulatory approval and launch pralsetinib in RET fusion-positive NSCLC in the U.S. by the end of 2020
Research portfolio

In November 2019, at Blueprint Medicines’ first R&D Day, announced four new research programs enabled by the company’s integrated precision medicine platform. Read the press release here.
Today announced the nomination of a potential first-in-class development candidate for the treatment of resistant EGFR-positive triple mutant NSCLC.
2020 goals:

Nominate up to two additional development candidates by the end of 2020

On January 13, 2020 Geron Corporation (Nasdaq: GERN), a late-stage clinical development biopharmaceutical company, reported 2019 accomplishments and key development priorities for 2020 (Press release, Geron, JAN 13, 2020, View Source [SID1234553099]). The Company is currently enrolling patients in the Phase 3 IMerge clinical trial of imetelstat in lower risk myelodysplastic syndromes (MDS), and plans to complete enrollment by the end of 2020 with top-line results expected by mid-year 2022.

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"From regaining control of the imetelstat program and building an impressive in-house development team to initiating the Company’s first Phase 3 clinical trial, 2019 was a pivotal year for Geron," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "We plan to build on the progress achieved in 2019 as we set our 2020 priorities and finalize our development plans."

2019 – A Year of Accomplishments

Advancing the Imetelstat Development Program

In May 2019, Geron assumed the imetelstat investigational new drug (IND) sponsorship from Janssen Biotech, Inc. (Janssen), and executed on its 2019 imetelstat development plans, including advancing into late-stage clinical development with the initiation of the Phase 3 IMerge clinical trial in lower risk MDS.

Geron completed the transition of the imetelstat program, including the transfer of the remaining non-clinical, manufacturing and ex-U.S. clinical and regulatory responsibilities from Janssen at the end of September 2019.

Throughout the year, Geron continued to build a solid foundation of extensive hematology-oncology and drug development expertise in key functional areas by attracting senior leadership with extensive experience, including many new development team members and executives with prior experience with imetelstat. This highly capable in-house team will support current and future imetelstat development plans, including plans to explore additional indications, as well as provide the ability to evaluate other hematology-oncology assets to expand the Company’s pipeline in the future.

Commencing the Phase 3 in Lower Risk MDS

In June 2019, Geron presented updated efficacy and safety data for the Phase 2 IMerge clinical trial. The presentation highlighted meaningful and durable transfusion independence, activity across different MDS subtypes and potential disease-modifying activity in lower risk MDS with imetelstat treatment.

Geron achieved a significant milestone in August 2019 with the opening of the Phase 3 IMerge clinical trial for patient screening and enrollment, and the first patient was dosed in October. Many key aspects from the Phase 2 portion of IMerge remained the same for the Phase 3 portion, including the primary and secondary endpoints, the dose and schedule of imetelstat administration, and the target patient population. This trial is an important step in developing imetelstat as a potential alternative for lower risk MDS patients who have limited treatment options.

Regulatory Interactions Related to Myelofibrosis (MF)

The United States Food and Drug Administration (FDA) granted Fast Track designation to imetelstat for the treatment of adult patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment, or relapsed/refractory MF, in September 2019. The FDA’s Fast Track Program is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious conditions and supported by data that demonstrate the potential to address an unmet medical need.

Geron announced that it conducted an End of Phase 2 meeting with the FDA to discuss the results of the IMbark Phase 2 clinical trial of imetelstat in patients with relapsed/refractory MF in December 2019.

Development Priorities for 2020

Geron is setting development priorities that are expected to shape the Company’s 2020 milestones, which will be discussed at a conference call to be held in March 2020 in connection with the announcement of the Company’s year-end 2019 financial results.

Building Momentum in Phase 3 IMerge Clinical Trial in Lower Risk MDS

The Company expects to complete enrollment for the Phase 3 IMerge clinical trial in 2020, which is a key step toward achieving top-line results from the trial by mid-year 2022. As of the beginning of 2020, approximately 40% of the planned clinical sites are open for enrollment.

Determining Potential Late-Stage Development in MF

Based on feedback from the End of Phase 2 meeting, Geron plans to submit several Phase 3 trial design proposals in MF, and to have further discussions with the FDA regarding a potential regulatory approval path. Subsequent to these additional discussions, and after considering the timing and resources required, as well as other clinical development opportunities for imetelstat, Geron plans to make a decision regarding potential late-stage development of imetelstat in MF.

Broadening Imetelstat Program with Additional Hematologic Myeloid Malignancy Indication

Geron expects to expand development of imetelstat within hematologic myeloid malignancies by initiating a proof-of-concept study in an additional indication in 2020.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat consist of IMerge, a Phase 2/3 trial in lower risk myelodysplastic syndromes (MDS), and IMbark, a Phase 2 trial in Intermediate-2 or High-risk myelofibrosis (MF). Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.

On January 13, 2020 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC) (TSX: ONC.TO), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported a recap of a Key Opinion Leader call sponsored by ROTH Capital Partners which was held on Thursday, January 9, 2020 (Press release, Oncolytics Biotech, JAN 13, 2020, View Source [SID1234553098]). The KOL call focused on recent data presented at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition and highlighted that carfilzomib promotes reovirus infection, that pelareorep upregulates PD-L1, and that delivery of additional data from ongoing pelareorep studies in multiple myeloma is planned for presentation at ASCO (Free ASCO Whitepaper) in June 2020.

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The KOL call was hosted and conducted by the equity research department at ROTH Capital and featured two of Oncolytics’ multiple myeloma clinical investigators:

Dr. Craig Hofmeister M.D., Winship Cancer Institute/Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta, GA.
Dr. Flavia Pichiorri Ph.D., Judy and Bernard Briskin Center for Multiple Myeloma Research, City of Hope, Duarte, CA and Department of Hematologic Malignancies Translational Science, Beckman Research Institute, City of Hope, Duarte, CA.
"I think that carfilzomib promotes pelareorep infection by suppressing the innate antiviral response and our data suggest that it does not get in the way of T-cell activation," said Dr. Hofmeister. "Pelareorep infection, not proteasome inhibition, can upregulate PD-L1 expression on myeloma cells and the adaptive immune system can then assist in clearing infected tumor cells. The combination in fact enhances the body’s immune attack on infected myeloma cells."

Dr. Hofmeister confirmed that adding PD-1 inhibition is appropriate in these patients, as viral infection with replication has been associated with an increase in PD-L1 tumor expression preclinically. "We are seeing some signs of cytokine release with associated clinical responses and we look forward to describing the clinical results in more detail at ASCO (Free ASCO Whitepaper) 2020," Dr. Hofmeister added.

The call also discussed the competitive landscape for refractory multiple myeloma and the paucity of available therapies to treat these patients.

"Reovirus is the only strategy I see in the market that is completely different and may be able to activate the immune system of these patients," said Dr. Pichiorri. "It would be a salvage therapy for now, but so many patients need a salvage therapy after being refractory to other therapies."

Oncolytics would like to thank Doctors Pichiorri and Hofmeister for their time and insightful commentary, as well as ROTH Capital for hosting the call.

About Pelareorep

Pelareorep is a non-pathogenic, proprietary isolate of the unmodified reovirus: a first-in-class intravenously delivered immuno-oncolytic virus for the treatment of solid tumors and hematological malignancies. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype through innate and adaptive immune responses to treat a variety of cancers and has been demonstrated to be able to escape neutralizing antibodies found in patients.