Illumina to Announce Fourth Quarter and Fiscal Year 2019 Financial Results on Wednesday, January 29, 2020

On January 13, 2020 Illumina, Inc. (NASDAQ:ILMN) reported that it will issue results for fourth quarter and fiscal year 2019 following the close of market on Wednesday, January 29, 2020 (Press release, Illumina, JAN 13, 2020, View Source [SID1234553115]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On the same day, at 2:00 pm Pacific Time (5:00 pm Eastern Time) Francis deSouza, President and Chief Executive Officer, and Sam Samad, Senior Vice President and Chief Financial Officer, will host a conference call with analysts, investors, and other interested parties to discuss financial and operating results.

Conference Call Details

The conference call will begin at 2:00 pm Pacific Time (5:00 pm Eastern Time) on Wednesday, January 29, 2020. Interested parties may access the live teleconference through the Investor Relations section of Illumina’s website under the "company" tab at www.illumina.com. Alternatively, individuals can access the call by dialing 1 (866) 211-4597, or 1 (647) 689-6853 outside North America, both with Conference ID 2966099.

A replay of the conference call will be posted on Illumina’s website after the event and will be available for at least 30 days following.

Tyvyt® (Sintilimab Injection) Combined with ALIMTA® (Pemetrexed) and Platinum Met Predefined Primary Endpoint in Phase 3 ORIENT-11 Study as First-Line Therapy in Nonsquamous NSCLC

On January 13, 2020 Eli Lilly and Company (NYSE: LLY) reported with Innovent Biologics, Inc. the results of a Phase 3 study in China; the ORIENT-11 trial of Tyvyt (sintilimab injection) in combination with ALIMTA (pemetrexed) and platinum in first-line advanced or recurrent nonsquamous non-small cell lung cancer (nsqNSCLC), without sensitive EGFR mutation or ALK rearrangement, met the predefined primary endpoint of progression-free survival (PFS) in an interim analysis (Press release, Eli Lilly, JAN 13, 2020, View Source [SID1234553135]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Based on the interim analysis conducted by the Independent Data Monitoring Committee (IDMC), sintilimab in combination with ALIMTA and platinum demonstrated a statistically significant improvement in PFS compared with placebo in combination with ALIMTA and platinum, which met the pre-defined efficacy criteria. The safety profile of sintilimab in this trial was consistent with previously reported studies, and no new safety signals were identified.

Relevant data will be presented at an upcoming medical conference. Based on the IDMC recommendations, Lilly and Innovent will initiate regulatory discussions for registration with the National Medical Products Administration (NMPA) in China in the near future.

Professor Li Zhang, Head of the Department of Internal Medicine, Sun Yat-sen University Cancer Center, stated: "In 2019, the National Cancer Center published Chinese data on lung cancer from 2015, showing an incidence of 20 percent and a mortality rate of about 27 percent, ranking it first among all cancer types. Patients who have nsqNSCLC without sensitive EGFR mutation or ALK rearrangement need more treatment options. Treatment with an anti-PD-1 monoclonal antibody in combination with chemotherapy may bring a greater survival benefit to this patient population. We are glad to see that these findings from this trial of sintilimab met the predefined primary endpoint in the interim analysis."

"We are excited about these results, which show Tyvyt plus ALIMTA and platinum significantly delayed disease progression in this patient population. This study is another example of the joint commitment from Lilly and Innovent to provide new treatment options to patients with lung cancer," said Dr. Wang Li, Senior Vice-President of Lilly China and Head of Lilly China Drug Development and Medical Affairs. "We would like to thank the patients, investigators and clinical trial sites that are participating in the study, and our colleagues from Innovent. We look forward to bringing this new treatment option to lung cancer patients in China."

Dr. Hui Zhou, Vice President and Head of Oncology Strategy and Medical Sciences, Innovent, said: "So far, Tyvyt is the only anti-PD-1 monoclonal antibody included in the New Catalogue of the National Reimbursement Drug List. It was officially approved by the NMPA on December 24, 2018 for the treatment of relapsed or refractory classic Hodgkin’s lymphoma after at least second-line system chemotherapy. Currently, we have several ongoing Phase 3 randomized clinical trials in lung cancer. With the encouraging result of ORIENT-11 we anticipate that sintilimab has the potential to benefit more patients with lung cancer and provide them more time with their families."

About ORIENT-11 Trial
ORIENT-11 is a randomized, double-blind, Phase 3 clinical trial to evaluate the efficacy and safety of Tyvyt (sintilimab injection) or placebo in combination with ALIMTA and platinum as first-line therapy for advanced or recurrent nsqNSCLC without sensitive EGFR mutation or ALK rearrangement (ClinicalTrials.gov, NCT03607539). The primary endpoint is progression-free survival (PFS) assessed by the Independent Radiographic Review Committee (IRRC) based on RECIST v1.1. The other secondary endpoints include overall survival (OS) and safety profile.

A total of 397 subjects have been enrolled in the ORIENT-11 trial and randomized in a 2:1 ratio to receive either sintilimab 200mg or placebo in combination with ALIMTA and platinum every three weeks for up to four cycles, followed by either sintilimab or placebo plus ALIMTA maintenance therapy. The subjects will receive treatment until radiographic disease progression, unacceptable toxicity or any other conditions that require treatment discontinuation. Conditional crossover is permitted.

About nsqNSCLC
Lung cancer is a malignancy with the highest morbidity and mortality rates in China. NSCLC accounts for about 80 percent to 85 percent of lung cancers. Approximately 70 percent of NSCLC are locally advanced or metastatic at initial diagnosis, rendering the patients with no chance of radical resection. Meanwhile, even after radical surgery patients still have a high chance of recurrence and eventually die from disease progression. About 70 percent of NSCLC in China are nonsquamous subtype and 50 percent of nsqNSCLC are without sensitive EGFR mutation or ALK rearrangement. These patients do not respond well to targeted therapy and there are limited treatment options available to them.

About Tyvyt (Sintilimab Injection)
Tyvyt (sintilimab injection), an innovative drug jointly developed in China by Innovent and Lilly, has been granted marketing approval by the NMPA for relapsed or refractory classic Hodgkin’s lymphoma after at least second-line system chemotherapy, and is included in the 2019 Guidelines of Chinese Society of Clinical Oncology for Lymphoid Malignancies. Tyvyt is the only PD-1 inhibitor with global quality that has been included in the new Catalogue of the National Reimbursement Drug List (NRDL) in November 2019.

Tyvyt is a type of immunoglobulin G4 monoclonal antibody, which binds to PD-1 molecules on the surface of T-cells, blocks the PD-1/ PD-Ligand 1 (PD-L1) pathway and reactivates T-cells to kill cancer cells. Innovent is currently conducting more than 20 clinical studies for sintilimab injection to evaluate its safety and efficacy in a wide variety of cancer indications, including eight registration or pivotal clinical trials.

Tyvyt (sintilimab injection) is not an approved product in the United States. ALIMTA (pemetrexed for injection) is not approved for use in combination with Tyvyt in the United States.

U.S. INDICATIONS FOR ALIMTA (pemetrexed for injection)
ALIMTA is indicated:

in combination with pembrolizumab and platinum chemotherapy for the initial treatment of patients with nonsquamous metastatic non-small cell lung cancer (mNSCLC) with no EGFR or ALK genomic tumor aberrations.
in combination with cisplatin for the initial treatment of patients with locally advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC).
as a single agent for the maintenance treatment of patients with locally advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) whose disease has not progressed after four cycles of platinum-based first-line chemotherapy.
as a single agent for the treatment of patients with recurrent metastatic nonsquamous non-small cell lung cancer (NSCLC) after prior chemotherapy. Limitation of Use: ALIMTA is not indicated for the treatment of patients with squamous cell non-small cell lung cancer.
in combination with cisplatin, for the initial treatment of patients with malignant pleural mesothelioma (MPM) whose disease is unresectable or who are otherwise not candidates for curative surgery.
U.S. IMPORTANT SAFETY INFORMATION FOR ALIMTA (pemetrexed for injection)

CONTRAINDICATION

ALIMTA is contraindicated in patients who have a history of severe hypersensitivity reaction to pemetrexed.
WARNINGS AND PRECAUTIONS

Myelosuppression and Increased Risk of Myelosuppression Without Vitamin Supplementation

ALIMTA can cause severe myelosuppression resulting in a requirement for transfusions and which may lead to neutropenic infection. The risk of myelosuppression is increased in patients who do not receive vitamin supplementation.
Prior to treatment with ALIMTA, patients must be instructed to initiate supplementation with oral folic acid. Intramuscular injections of vitamin B12 are also required prior to ALIMTA treatment. Folic acid and vitamin B12 supplementation should be continued during treatment and for 21 days after the last dose of ALIMTA as they may reduce the severity of treatment-related hematologic and gastrointestinal toxicities. Obtain a complete blood count at the beginning of each cycle. Do not administer ALIMTA until the ANC is at least 1500 cells/mm3 and platelet count is at least 100,000 cells/mm3. Permanently reduce ALIMTA in patients with an ANC of less than 500 cells/mm3 or platelet count of less than 50,000 cells/mm3 in previous cycles.
In Studies JMDB and JMCH, among patients who received vitamin supplementation, incidence of Grade 3-4 neutropenia was 15% and 23%, the incidence of Grade 3-4 anemia was 6% and 4%, and incidence of Grade 3-4 thrombocytopenia was 4% and 5%, respectively. In Study JMCH, 18% of patients in the ALIMTA arm required red blood cell transfusions compared to 7% of patients in the cisplatin arm. In Studies JMEN, PARAMOUNT, and JMEI, where all patients received vitamin supplementation, incidence of Grade 3-4 neutropenia ranged from 3% to 5%, and incidence of Grade 3-4 anemia ranged from 3% to 5%.
Renal Failure

ALIMTA can cause severe, and sometimes fatal, renal toxicity. Determine creatinine clearance before each dose and periodically monitor renal function during treatment with ALIMTA.
The incidences of renal failure in clinical studies in which patients received ALIMTA with cisplatin were 2.1% in Study JMDB and 2.2% in Study JMCH. The incidence of renal failure in clinical studies in which patients received ALIMTA as a single agent ranged from 0.4% to 0.6% (Studies JMEN, PARAMOUNT, and JMEI).
Withhold ALIMTA in patients with a creatinine clearance of less than 45 mL/min.
Bullous and Exfoliative Skin Toxicity

Serious and sometimes fatal, bullous, blistering, and exfoliative skin toxicity, including cases suggestive of Stevens-Johnson Syndrome/toxic epidermal necrolysis, can occur with ALIMTA. Permanently discontinue ALIMTA for severe and life-threatening bullous, blistering, or exfoliating skin toxicity.
Interstitial Pneumonitis

Serious interstitial pneumonitis, including fatal cases, can occur with ALIMTA treatment. Withhold ALIMTA for acute onset of new or progressive unexplained pulmonary symptoms such as dyspnea, cough, or fever pending diagnostic evaluation. If pneumonitis is confirmed, permanently discontinue ALIMTA.
Radiation Recall

Radiation recall can occur with ALIMTA in patients who have received radiation weeks to years previously. Monitor patients for inflammation or blistering in areas of previous radiation treatment. Permanently discontinue ALIMTA for signs of radiation recall.
Increased Risk of Toxicity With Ibuprofen in Patients With Renal Impairment

Exposure to ALIMTA is increased in patients with mild to moderate renal impairment who take concomitant ibuprofen, increasing the risks of adverse reactions of ALIMTA. In patients with creatinine clearances between 45 mL/min and 79 mL/min, avoid administration of ibuprofen for 2 days before, the day of, and 2 days following administration of ALIMTA. If concomitant ibuprofen use cannot be avoided, monitor patients more frequently for ALIMTA adverse reactions, including myelosuppression, renal, and gastrointestinal toxicity.
Embryo-Fetal Toxicity

Based on findings from animal studies and its mechanism of action, ALIMTA can cause fetal harm when administered to a pregnant woman. In animal reproduction studies, intravenous administration of pemetrexed to pregnant mice during the period of organogenesis was teratogenic, resulting in developmental delays and increased malformations at doses lower than the recommended human dose of 500 mg/m2. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with ALIMTA and for 6 months after the final dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with ALIMTA and for 3 months after the final dose.
DRUG INTERACTIONS

Ibuprofen increases exposure (AUC) of pemetrexed. In patients with creatinine clearance between 45 mL/min and 79 mL/min:
Avoid administration of ibuprofen for 2 days before, the day of, and 2 days following administration of ALIMTA.
Monitor patients more frequently for myelosuppression, renal, and gastrointestinal toxicity, if concomitant administration of ibuprofen cannot be avoided.
ADVERSE REACTIONS

Severe adverse reactions (Grade 3-4) occurring in ≥20% of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC) receiving ALIMTA in combination with pembrolizumab and platinum chemotherapy (carboplatin or cisplatin) versus ALIMTA with platinum chemotherapy + placebo for initial treatment (KEYNOTE-189), respectively, were fatigue (12% vs 6%); diarrhea (5% vs 3%); dyspnea (3.7% vs 5%); vomiting (3.7% vs 3%); nausea (3.5% vs 3.5%); rash (2% vs 2.5%); decreased appetite (1.5% vs 0.5%); constipation (1% vs 0.5%); and pyrexia (0.2% vs 0%).
Common adverse reactions (all grades) occurring in ≥20% of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC) receiving ALIMTA in combination with pembrolizumab and platinum chemotherapy (carboplatin or cisplatin) versus ALIMTA with platinum chemotherapy + placebo for initial treatment (KEYNOTE-189), respectively, were nausea (56% vs 52%); fatigue (56% vs 58%); constipation (35% vs 32%); diarrhea (31% vs 21%); decreased appetite (28% vs 30%); rash (25% vs 17%); vomiting (24% vs 23%); cough (21% vs 28%); dyspnea (21% vs 26%); and pyrexia (20% vs 15%).
USE IN SPECIFIC PATIENT POPULATIONS

Lactation: There is no information regarding the presence of pemetrexed or its metabolites in human milk, the effects on the breastfed infant, or the effects on milk production. Because of the potential for serious adverse reactions in breastfed infants from ALIMTA, advise women not to breastfeed during treatment with ALIMTA and for one week after the last dose.
Males of Reproductive Potential: ALIMTA may impair fertility in males of reproductive potential. It is not known whether these effects on fertility are reversible.
Pediatric Use: The safety and effectiveness of ALIMTA in pediatric patients have not been established. Adverse reactions observed in pediatric patients studied were similar to those observed in adults.
Patients with Renal Impairment: ALIMTA is primarily excreted by the kidneys. Decreased renal function results in reduced clearance and greater exposure (AUC) to ALIMTA compared with patients with normal renal function. No dose is recommended for patients with creatinine clearance less than 45 mL/min.
Geriatric: The incidences of Grade 3-4 anemia, fatigue, thrombocytopenia, hypertension, and neutropenia were higher in patients 65 years of age and older as compared to younger patients: in at least one of five randomized clinical trials.

Updated Corporate Slide Presentation

On January 13, 2020 Oncternal Therapeutics Presented the Corporate Presentation (Presentation, Oncternal Therapeutics, JAN 13, 2020, View Source [SID1234554377]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!


Deciphera Pharmaceuticals Provides Corporate Update and Highlights Key 2020 Milestones

On January 13, 2020 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported a corporate update and highlighted key 2020 milestones in conjunction with its presentation at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Deciphera Pharmaceuticals, JAN 13, 2020, View Source [SID1234553083]). The Company will webcast its presentation today at 11:00 a.m. PT (2:00 p.m. ET) at View Source

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"2019 was a year of many exciting accomplishments for Deciphera as we submitted our first New Drug Application (NDA) to the FDA for ripretinib in advanced gastrointestinal stromal tumors (GIST) based on positive results from the INVICTUS pivotal Phase 3 study and advanced our portfolio of wholly-owned product candidates," said Steve Hoerter, President and Chief Executive Officer of Deciphera. "We are preparing for a potential commercial launch in the U.S. and working to bring ripretinib to other parts of the world."

The Company announced today that two additional marketing applications have been submitted for ripretinib as part of the U.S. Food and Drug Administration’s (FDA) Project Orbis pilot program. The marketing applications of ripretinib for advanced GIST in Canada and Australia have both received priority review designations. The Project Orbis pilot program, an initiative of the FDA Oncology Center of Excellence, is designed to provide a framework for concurrent submission and review of oncology products among international partners. Additional information about Project Orbis can be found at: View Source

Mr. Hoerter continued, "In parallel with these efforts, we have laid the groundwork for further expansion across our pipeline of novel agents. We continued to activate sites and enroll patients in the INTRIGUE Phase 3 study in the 2nd line GIST patient population, advanced the DCC-3014 and rebastinib clinical development programs, and added a new internally-discovered candidate, DCC-3116, to our pipeline. We look forward to building on our momentum in 2020 as we work with the FDA towards a potential approval of ripretinib in advanced GIST and rapidly advance our additional programs."

In 2020, the Company seeks to achieve the following milestones:

Ripretinib
– Potential FDA approval and commercial launch of ripretinib in advanced GIST.
– Submit marketing authorization application to European Medicines Agency.
– Complete enrollment of INTRIGUE Phase 3 study in 2nd line GIST.
– Present Phase 1 study expansion data.

DCC- 3014
– Select Phase 2 dose for tenosynovial giant cell tumor (TGCT) patients and open expansion cohort.
– Provide update on Phase 1 data in TGCT patients.

Rebastinib
– Selected Phase 2 dose of 100 mg BID of rebastinib and activated Part 2 of Phase 1b/2 study in combination with carboplatin. (Completed January 2020)
– Present Phase 1b/2 data in combination with carboplatin.
– Present Phase 1b/2 data in combination with paclitaxel.

DCC-3116
– Submit an Investigational New Drug (IND) application to FDA.

Presentation at 38th Annual J.P. Morgan Healthcare Conference

Deciphera will webcast its corporate presentation from the 38th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 13, 2020 at 11:00 a.m. PT (2:00 p.m. ET). A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the Company’s website at deciphera.com. A replay of the webcast will be archived on the Deciphera website for at least two weeks following the presentation. In conjunction with the conference, the Company has also updated its corporate presentation which can be found here: View Source

Blueprint Medicines Announces 2020 Corporate Goals

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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