On January 11, 2021 Novocure (NASDAQ: NVCR) reported operating statistics and preliminary, unaudited net revenues and cash balances for the fourth quarter and full year 2020 (Press release, NovoCure, JAN 11, 2021, View Source [SID1234573839]). Novocure plans to discuss these results with investors at the 39th Annual J.P. Morgan Virtual Healthcare Conference. Novocure also plans to highlight key clinical and product development programs.

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(1) 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 2020 on or about Feb. 25, 2021.

(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.

"Our track record of execution extended throughout 2020 with significant achievements furthering our three overarching priorities," said William Doyle, Novocure’s Executive Chairman. "We sustained our commercial momentum with $494 million in net revenues, representing 41% annual revenue growth; launched three new clinical trials, expanding our development pipeline to include eight ongoing global studies involving nearly 3,000 patients across seven solid tumor cancers; and, increased our investments in product development."

"We continue to build on twenty years of innovation and execution to pioneer an emerging modality in cancer care," said Asaf Danziger, Novocure’s CEO. "Through our clinical and product development efforts, we believe we are just beginning to unlock the potential of the Tumor Treating Fields platform to extend survival in some of the most aggressive forms of cancer. We look forward to providing a full company update and discussing our 2020 financial results on our February conference call."

Fourth quarter 2020 operating statistics and preliminary financial highlights

On a preliminary, unaudited basis, for the quarter ended December 31, 2020, net revenues were $144.0 million, representing 45% growth compared to the fourth quarter 2019, and full year 2020 net revenues were $494.4 million, representing 41% growth compared to full year 2019.

In the United States, net revenues totaled $97.7 million in the quarter ended December 31, 2020, representing 48% growth compared to the same period in 2019.
In Germany and other EMEA markets, net revenues totaled $33.8 million in the quarter ended December 31, 2020, representing 31% growth compared to the same period in 2019.
In Japan, net revenues totaled $7.9 million in the quarter ended December 31, 2020, representing 42% growth compared to the same period in 2019
In Greater China, net revenues totaled $4.5 million in the quarter ended December 31, 2020, representing 132% growth compared to the same period in 2019.
We recorded $9 million and $36 million in revenues from Medicare fee-for-service beneficiaries billed under the coverage policy effective on September 1, 2019 for the three and twelve month periods ended December 31, 2020, respectively. 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 fourth quarter of 2020, we also recognized approximately $11 million in incremental net revenues compared to the first half of 2020 resulting from the successful appeal of previously denied claims for Medicare fee-for-service beneficiaries billed prior to established coverage.

There were 3,411 active patients at December 31, 2020, representing 17% growth compared to December 31, 2019, and one percent growth compared to September 30, 2020.

In the United States, there were 2,193 active patients at December 31, 2020, representing 12% growth compared to December 31, 2019.
In Germany and other EMEA markets, there were 953 active patients at December 31, 2020, representing 25% growth compared to December 31, 2019.
In Japan, there were 265 active patients at December 31, 2020, representing 38% growth compared to December 31, 2019.
Additionally, 1,411 prescriptions were received in the quarter ended December 31, 2020, representing two percent growth compared to the same period in 2019, and three percent growth compared to the quarter ended September 30, 2020. In the quarter ended December 31, 2020, 1,160 Optune prescriptions were written for patients with newly diagnosed glioblastoma.

In the United States, 962 prescriptions were received in the quarter ended December 31, 2020, representing a four percent decrease compared to the same period in 2019.
In Germany and other EMEA markets, 349 prescriptions were received in the quarter ended December 31, 2020, representing 22% growth compared to the same period in 2019.
In Japan, 100 prescriptions were received in the quarter ended December 31, 2020, representing eight percent growth compared to the same period in 2019.
At December 31, 2020, on a preliminary, unaudited basis, Novocure had $235 million in cash and cash equivalents and $608 million in short-term investments, for a total balance of $843 million in cash, cash equivalents and short-term investments. This represents an increase of $608 million in cash and investments since September 30, 2020.

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)
Fourth quarter and full year 2020 financial results conference call

Novocure will host a conference call and webcast to discuss fourth quarter and full year 2020 financial results at 8 a.m. EST on Thursday, February 25, 2021. 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 3965899.

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 11, 2021 Ultimovacs ASA ("Ultimovacs", ticker ULTI), reported its participation in the Phase II DOVACC collaboration study with the Nordic Society of Gynaecological Oncology – Clinical Trial Unit (NSGO-CTU), the European Network of Gynaecological Oncological Trial Groups (ENGOT) and AstraZeneca, to conduct a randomized Phase II clinical trial to evaluate Ultimovacs’ proprietary universal cancer vaccine, UV1, in combination with AstraZeneca’s durvalumab and olaparib in patients with relapsed ovarian cancer (Press release, Ultimovacs, JAN 11, 2021, View Source [SID1234573838]).

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Carlos de Sousa, Chief Executive Officer at Ultimovacs stated: "Now that the formal collaboration with NSGO-CTU/ENGOT and AstraZeneca is in place and preparations for the study are well underway, I am happy to provide further details on the DOVACC Phase II clinical trial, which was first announced in May of 2020. A key benefit of UV1 is its safety profile, which enables a triple combination study with a PD-L1 inhibitor and now for the first time also with a PARP inhibitor, in an indication in desperate need of new treatment options. Ultimovacs has four Phase II trials evaluating UV1, including more than 500 patients in total, supporting its universal potential across many cancer indications and as part of various treatment combinations."

"The NSGO-CTU is a highly regarded oncology society that has strong connections with similar organizations across Europe through the ENGOT collaboration," commented Jens Bjørheim, Chief Medical Officer at Ultimovacs. "The collaboration with the NSGO-CTU and ENGOT is exciting for us as they are very experienced with conducting clinical trials in the field of gynaecological oncology. Their expertise and network will support the recruitment of patients at multiple participating study sites across Europe."

"Our purpose at the NSGO-CTU and ENGOT is to improve treatment options in gynaecological cancer indications by coordinating clinical trial efforts across countries," added Dr. Mansoor Raza Mirza, Medical Director of the NSGO-CTU and Chair of the ENGOT. "The evaluation of UV1, durvalumab and olaparib in this Phase II triple combination study is an important next step in changing the standard of care for patients with ovarian cancer. We look forward to working with Ultimovacs to reach our mutual goal."

DOVACC is a multi-center, multinational, randomized Phase II clinical trial sponsored by the NSGO, the leading gynaecological oncology research society in the Nordic and Baltic regions. The trial is designed to evaluate Ultimovacs’ proprietary UV1 cancer vaccine in combination with AstraZeneca’s durvalumab, a PD-L1 checkpoint inhibitor and its PARP inhibitor, olaparib, the maintenance therapy for BRCA-mutated advanced ovarian cancer. The trial will be conducted at more than 30 hospitals in around 10 European countries. The Company expects to treat the first patient in the first half of 2021. Topline data on the primary endpoint is expected in 2023.

The second-line maintenance study will enroll patients with high-grade BRCA negative ovarian cancer after partial or complete response following the second round of chemotherapy. The study includes three arms treating a total of 184 patients. The first arm will enroll 46 patients receiving the PARP inhibitor olaparib. The 46 patients enrolled in the second arm will receive olaparib and the checkpoint inhibitor durvalumab. The third arm will include 92 patients that will receive Ultimovacs’ UV1 vaccine in combination with both AstraZeneca drugs. The primary endpoint is progression-free survival (PFS) in the treatment arm with solely the PARP inhibitor olaparib, versus PFS in the triple combination treatment arm. Under the terms of the collaboration, Ultimovacs will provide its UV1 vaccine and AstraZeneca will provide the PD-L1 and PARP inhibitors for the study.

Innovation Norway has granted Ultimovacs NOK 10 million, approximately EUR 1 million, to support the execution of the Phase II DOVACC study. In addition, the successful private placement in May of this year raised NOK 160 million, approximately EUR 16 million, allowing Ultimovacs to continue to implement the Company’s clinical development plan and fund its operations through the expected readout of the primary endpoint in the DOVACC study in 2023.

Ultimovacs management will host a webcast on Monday, January 11, 2021, at 11:00 a.m. CET to discuss the DOVACC clinical trial in more detail. The webcast will also feature a short video statement from lead investigator Dr. Mirza. The live webcast can be accessed through the investor section of Ultimovacs’ corporate website at www.ultimovacs.com. The webcast replay will be available for at least 72 hours following the call.

About UV1

UV1 is a peptide-based vaccine inducing a specific T cell response against the universal cancer antigen telomerase. UV1 is being developed as an "off-the-shelf" therapeutic cancer vaccine which may serve as a platform for use in combination with other immunotherapy which requires an ongoing T cell response for their mode of action. To date, UV1 has been tested in four phase I clinical trials in a total of 82 patients and maintained a positive safety and tolerability profile as well as encouraging signals of efficacy.

About UV1 Clinical Programs

As a universal cancer vaccine, UV1’s unique mechanism of action has the potential to be applicable across most cancer types. The clinical development of the UV1 vaccine includes four randomized, multinational, Phase II combination trials: INITIUM, NIPU, DOVACC and FOCUS, recruiting over 500 patients in total. The INITIUM trial is an Ultimovacs-sponsored clinical trial recruiting 154 patients with metastatic malignant melanoma to evaluate UV1 in combination with ipilimumab and nivolumab as first-line treatment. The NIPU study is testing UV1 in combination with checkpoint inhibitors ipilimumab and nivolumab as second-line treatment in 118 patients with advanced malignant pleural mesothelioma, a rare lung cancer. The study is sponsored by Oslo University Hospital and Bristol-Myers Squibb is providing the checkpoint inhibitors for this study. The DOVACC study is sponsored by the Nordic Society of Gynaecological Oncology. In total, 184 patients with high-grade ovarian cancer will be enrolled to evaluate UV1 in combination with durvalumab and olaparib, both provided by AstraZeneca. FOCUS is an investigator-sponsored, randomized clinical trial enrolling 75 patients with metastatic head and neck cancer receiving pembrolizumab as standard of care, and will evaluate the impact of adding UV1 to this regimen. Ultimovacs anticipates announcing data on the primary endpoints for the NIPU and INITIUM studies in 2022 and for the DOVACC and FOCUS studies in 2023. (Press release, Ultimovacs, JAN 11, 2021, View Source [SID1234573838])

On January 11, 2021 Propanc Biopharma, Inc. (OTC: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that pancreatic proenzymes versus T-Cell therapy when targeting solid tumors such as pancreatic, ovarian and colorectal cancers (Press release, Propanc, JAN 11, 2021, View Source [SID1234573837]). The analysis is prepared by the Company’s Chief Executive Officer, Mr. James Nathanielsz, in collaboration with joint lead researcher, Professor Macarena Perán, from the University of Jaén, Granada, Spain.

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This press release features multimedia. View the full release here: View Source

Cell differentiation therapy using pancreatic proenzymes has shown to degrade the fibrotic tissue on the surface of solid tumors and therefore might impair tumor engrafting, tumor niche formation and even cancer stem cell subpopulation activation. (Photo: Business Wire)
Cell differentiation therapy using pancreatic proenzymes has shown to degrade the fibrotic tissue on the surface of solid tumors and therefore might impair tumor engrafting, tumor niche formation and even cancer stem cell subpopulation activation. (Photo: Business Wire)

"We are making significant inroads in the way we treat cancer today, but there is a genuine need to continually challenge ourselves to improve the standard of care for many cancer types," said James Nathanielsz. "At Propanc, we share a vision to develop and commercialize a novel approach using pancreatic proenzymes for the long-term treatment and prevention of metastatic cancer from solid tumors. Our goal is to reduce the threat of cancer by extending life meaningfully, but not at the expense of great toxicity. This humanitarian cause affects us all."

Everybody knows what cancer is. Cells in the body begin to divide rapidly and uncontrollably in the body, with an ability to migrate from one location and spread to distant sites. However, when a cell becomes undifferentiated, forgetting how to do its job and investing all its energy in proliferating, it becomes cancerous. Unlike normal cells, cancer cells multiply, but do not differentiate. Most common therapies take advantage of the uncontrolled proliferation and kill these cells by targeting the cell division machinery. These therapies are effective, but affect healthy cells as well, particularly those with a high cell turn over, inducing undesirable effects. More recently, scientific advancements have meant that T-cell therapies are considered a tremendous improvement compared to older treatments. T-cell therapy involves using specific T-cells from the patient’s own immune system. Doctors take a type of white blood cell from the patient’s body and genetically change the cells in a lab so they can better find the cancer. Then millions of these target-seeking cells are put back into the patient.

The use of cancer-specific T-cells is a clever strategy to use the natural weapons from the body against cancer cells. This is a genuine targeted therapy, which kills cancer by recognizing antigen targets expressed on the cancer cell surface. This novel strategy is promising, although it still has some challenges. Of most importance is the health of patient’s T-cells, which may decline due to age, or degeneration induced by the cancer itself, which is not ideal. There are also limitations with regards to efficacy and safety, and they are highly expensive. Resistance can develop over time, as specific antigens mutate, causing tumor escape and disease relapse. Furthermore, a patient can have serious side effects, including very high fevers and dangerously low blood pressure days after treatment. Other serious side effects include neurotoxicity, or changes in the brain that cause swelling, confusion, seizures, or severe headaches. Another problem is that T-cells can kill off some of the good B-cells that help fight germs, so the patient may be at higher risk for infection. Finally, when factoring in all the costs associated with T-cell therapies, hospitals may charge as much as $1.5 million or more to avoid losing money.

So, whilst enhancing a patient’s immune response to attack cancer has genuine merit, other ways to stop cancer are needed to further reduce the threat of cancer from a killer disease to a chronic (long term) illness. Another approach to stop cancer is not by targeting cell death, but inducing cell differentiation. This is known as cell differentiation therapy. The key consideration is how to convince the malignant cells to stop proliferating and return to their role as a specific cell type.

So, what are the advantages of cell differentiation therapy over other strategies, like T-Cell therapy? Firstly, cell differentiation therapy does not target cell death, so healthy cells are not compromised. Cell differentiation therapy induces cancer cells to differentiate and become non-proliferative (non-replicating), so they die naturally. Cell differentiation therapy acts not only against cancer cells, but interestingly can turn cancer stem cells (undifferentiated cells) towards completely differentiated, i.e., normal cells. Significantly, once the cancer stem cells are completely differentiated, they are no longer hidden from the immune system. This means that the body’s immune response can more effectively target the cancer, and therefore, in theory, will be complementary to immunological approaches like T-Cell therapy, by improving response rates and reducing toxicity.

More than 100 years ago, a comparative embryologist Professor John Beard first proposed that pancreatic enzymes represent the body’s primary defense against cancer and would prove useful as a cancer treatment. Since then, scientists have endorsed Beard’s hypothesis with encouraging data from patient treatment. After extensive laboratory research over the last decade and limited human testing by compassionate use, there is evidence that pancreatic proenzymes reduces cancer cell growth via promotion of cell differentiation, enhances cell adhesion (cell to cell contact) and suppresses metastasis (cancer spread), has no serious side effects and improves patient survival. The unique approach targets and eradicate cancer stem cells, which can migrate to other organs triggering explosive tumor growth, causing the patient to relapse after standard treatments that do not target non-dividing cells. Eighty percent of cancers are from solid tumors and metastasis is the main cause of patient death, therefore the potential of cell differentiation therapy using pancreatic proenzymes is significant. Given they are derived from natural sources, pancreatic proenzymes are also not cost prohibitive.

There is little doubt that both the T-Cell based and cell differentiation therapy approaches have emerged to address the limited efficacy of chemotherapy and radiation therapy for patients with advanced solid tumors. Although both therapies bear a slight resemblance because they enhance the immune response, they are not comparable by their mode of action. It is also understood the tumor micro-environment promotes the appearance of new cancer stem cells, derived from non-stem cancerous cells by secreting several biomarkers, such as IL6 (interleukin 6), HGF (hepatocyte growth factor), or TGFβ-163 (tumor growth factor beta-163). Consequently, it is critical to impact the tumor micro-environment in order to effectively eradicate the tumor. Cell differentiation therapy using pancreatic proenzymes has shown to degrade the fibrotic tissue on the surface of solid tumors and therefore might impair tumor engrafting, tumor niche formation and even cancer stem cell subpopulation activation.

Whilst T-Cell therapy has helped to advance the treatment of cancer, there are new and exciting approaches which are complementary and may provide a long-term solution to the treatment and prevention of metastatic cancer from most common solid tumors. Cell differentiation therapy using pancreatic proenzymes is based on the original work by John Beard, a professor of embryology at Edinburgh University over 100 years ago, using fresh pancreatic extracts. Through advancements in science and technology, there is an opportunity to introduce an improved version of this hypothesis, as a long-term therapeutic approach to treat metastatic cancer from solid tumors, which today, remains the main cause of patient death for sufferers.

Bibliography

"Antitumor efficacy of chymotrypsinogen and trypsinogen," P. Hernández, E. López-Ruiz, M. A. García, J. A. Marchal, J. Kenyon, M. Perán.
"In vitro treatment of carcinoma cell lines with pancreatic (pro)enzymes suppresses the EMT programme and promotes cell differentiation", M. Perán, J.A. Marchal, M.A. García, J. Kenyon & D. Tosh.
"A formulation of pancreatic proenzymes provides potent anti-tumour efficacy: a pilot study focused on pancreatic and ovarian cancer", M. Perán, E. López-Ruiz, M. A. García, S. Nadaraia-Hoke, R. Brandt, J. A. Marchal & J. Kenyon.
"Pancreatic proenzymes treatment suppresses BXPC-3 pancreatic Cancer Stem Cell subpopulation and impairs tumour engrafting," P. Hernández-Camarero, E. López-Ruiz, C. Griñán-Lisón, M.A. García, C. Chocarro-Wrona, J.A. Marchal, J. Kenyon & M. Perán.
"Trypsinogen and Chymotrypsinogen: Potent Anti-Tumour Agents," A. González-Titos, P. Hernández-Camarero, S. Barungi, J.A. Marchal, J. Kenyon & M. Perán. *
*Draft manuscript under review

On January 11, 2021 Aptevo Therapeutics Inc. ("Aptevo" or the "Company") (NASDAQ:APVO), a clinical-stage biotechnology company focused on developing novel immuno-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported an update on its ongoing APVO436 phase 1 clinical trial (Press release, Aptevo Therapeutics, JAN 11, 2021, View Source [SID1234573836]).

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The Company noted that patient dosing in cohorts 1 through 8 has been completed and enrollment in cohort 9 has commenced. A total of 36 patients have been enrolled and treated with APVO436 to date. No evidence of dose-limiting toxicities (DLTs) was observed in cohorts 5-8.

"2020 was a very busy and exciting year at Aptevo, with progress on many fronts. Notably, two patients in cohort 6 of our APVO436 Phase 1a clinical trial achieved complete remission. This development greatly encouraged our research partners and our inhouse teams, who have poured their passion and energy into this trial," said Mr. Marvin White, President and CEO of Aptevo Therapeutics.

"In 2021, we look forward to advancing our APVO436 clinical trial and improving patient outcomes, while recognizing that each patient is unique. Our first patient in cohort 6 who had complete remission continues therapy, while the second patient in cohort 6 who also had complete remission progressed and discontinued therapy. Importantly, we are now in a critical phase of the study, within the therapeutic range,"

"We believe that the work we are doing is meaningful and important, and we will continue to focus on developing candidates that target cancers, ultimately increasing a patients’ chance of survival. We have multiple candidates moving towards clinical development, and our ADAPTIR and ADAPTIR-FLEX technology platforms are uniquely positioned to develop and advance our studies," concluded Mr. White.

About APVO436

APVO436 is an optimized bispecific antibody candidate designed to simultaneously target CD123 and CD3 and redirect T-cell cytotoxicity to the tumor. It is currently being evaluated in a Phase 1/1b open-label, dose-escalation study evaluating the safety and pharmacokinetic profile. APVO436 was built on Aptevo’s proprietary ADAPTIR protein therapeutic platform. Focused on generating novel, targeted bispecific antibody-based immunotherapies for cancer the ADAPTIR platform offers key advantages over other bispecific formats, derived in part from the flexible and modular nature of the ADAPTIR structure. These advantages include: (i) achieving potent biological activity and extended half-life while retaining desirable manufacturing characteristics and (ii) unique properties for redirecting T-cell cytotoxicity (RTCC) compared to other bispecific platforms, including a favorable cytokine release profile.

On January 11, 2021 Blueprint Medicines Corporation (NASDAQ: BPMC) reported an update on key portfolio milestones and outlined a strategic roadmap to become the world’s leading precision therapy company (Press release, Blueprint Medicines, JAN 11, 2021, View Source [SID1234573835]).

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"For the first time, we enter a new year as a fully integrated, global biopharmaceutical company, with four regulatory approvals in the United States and Europe in 2020, a pipeline of eight wholly owned or partnered precision therapies, and the strongest financial position since our inception," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "With this solid foundation, we are now scaling our ambition and aim to make real the promise of precision medicine to improve and extend life for as many people with cancer and hematologic disorders as possible. We will do this by bringing our medicines to more patients globally, rapidly advancing a wave of new therapeutic candidates to clinical proof-of-concept, and further expanding our platform-enabled research pipeline."

In addition, Blueprint Medicines reported the achievement of several portfolio milestones:

AYVAKIT received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of moderate to severe indolent systemic mastocytosis (SM), which encompasses the majority of patients with SM, highlighting the medical need in this population as well as the clinical potential of AYVAKIT to demonstrate substantial improvement over the current standard of care.
Positive top-line results from a Phase 1 trial in healthy volunteers showed BLU-263 was well-tolerated across a range of single- and multiple-ascending doses predicted to potently inhibit D816V mutant KIT, the underlying SM disease driver. These data support development of BLU-263 as a potential treatment for patients with SM and other mast cell disorders.
The company nominated a selective, brain-penetrant development candidate for treatment-resistant double-mutant EGFR-driven non-small cell lung cancer (NSCLC), with the potential to be first-in-class, showing potent activity against the activating L858R or exon 19 deletion mutations and the acquired C797S mutation, the most common on-target resistance mutation to osimertinib.
The company nominated a development candidate targeting MAP4K1, a kinase believed to play a role in T-cell regulation, with the potential to be best-in-class. The program was developed under the company’s cancer immunotherapy collaboration with Roche. In addition, Blueprint Medicines and Roche have amended their agreement to focus on MAP4K1 and one additional undisclosed target, collectively identified as the most promising targets of the collaboration to date.
Entering 2021, the company’s key strategies and goals include:

1. Accelerate global adoption of AYVAKIT and GAVRETO (pralsetinib)

AYVAKIT, a selective KIT and PDGFRA inhibitor, is approved in the U.S. and Europe for the treatment of patients with unresectable or metastatic gastrointestinal stromal tumor driven by certain PDGFRA mutations.

Obtain FDA approval and launch AYVAKIT in advanced SM in the U.S. in the second half of 2021.
Submit a Type II variation marketing authorization application (MAA) to the European Medicines Agency (EMA) for AYVAKYT (avapritinib) for advanced SM in the first quarter of 2021.
Present registrational data from the PATHFINDER trial of AYVAKIT in advanced SM in the first half of 2021.
Complete enrollment of the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced SM in mid-2021.
GAVRETO, a selective RET inhibitor, is approved in the U.S. for the treatment of patients with certain advanced or metastatic RET fusion-positive NSCLC, RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer. Under a global collaboration, Blueprint Medicines and Roche are developing and commercializing GAVRETO for the treatment of RET-altered cancers.

Obtain regulatory approval from the European Commission and launch GAVRETO in RET fusion-positive NSCLC in Europe in the first half of 2021.
Submit a Type II variation MAA to the EMA for GAVRETO for RET-altered thyroid cancers in the second half of 2021.
Initiate a GAVRETO cohort in Roche’s TAPISTRY tumor-agnostic platform trial in the second half of 2021.
Submit marketing applications for GAVRETO for RET-altered NSCLC and thyroid cancers across multiple additional global geographies in 2021.
2. Advance a new wave of innovative therapeutic candidates into clinical development, with plans to achieve rapid proof-of-concept and regulatory approval.

BLU-263, a next-generation selective KIT inhibitor

Initiate the Phase 2 HARBOR trial of BLU-263 in patients with non-advanced SM in mid-2021.
Development candidates for treatment-resistant EGFR-driven NSCLC

Initiate a Phase 1 trial of BLU-945, a triple-mutant EGFR inhibitor, in patients with treatment-resistant EGFR-driven NSCLC in the first half of 2021.
Initiate a Phase 1 trial of the company’s double-mutant EGFR inhibitor in patients with treatment-resistant EGFR-driven NSCLC by the end of 2021.
Present foundational preclinical data for the company’s double-mutant EGFR inhibitor in the first half of 2021.
Present preclinical data supporting combination of the company’s wholly owned double- and triple-mutant EGFR inhibitors in treatment-naïve EGFR-driven NSCLC in the second half of 2021.
Development candidate targeting MAP4K1, under the cancer immunotherapy collaboration with Roche

Present foundational preclinical data in the first half of 2021.
3. Further expand the company’s precision medicine pipeline with a focus on delivering transformational benefit to patients with cancer and hematologic disorders.

Expand pipeline with one or more development candidates in 2021.
Pursue external opportunities to complement the company’s precision medicine pipeline.
Financial Guidance

Based on its current operating plans, Blueprint Medicines continues to anticipate its existing cash, cash equivalents and investments, together with anticipated future product revenues, will provide sufficient capital to enable the company to achieve a self-sustainable financial profile.