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LG to Unveil Capsule-Based Craft Beer System at CES 2019

Tuesday December 11 th 2018

Automated Process and Exclusive Technologies
Deliver Total DIY Solution for Beer Enthusiasts

TORONTO, Dec. 10, 2018 /CNW/ – LG Electronics (LG) will debut a capsule-based craft beer making machine, LG HomeBrew, at CES 2019. A winner of a 2019 CES Innovation Award, the state-of-the-art device makes the brewing process incredibly easy thanks to its single-use capsules, optimized fermentation algorithm and convenient self-cleaning feature, the ideal product for anyone seeking the satisfaction of creating their own cold, tasty beer.

LG’s unique capsule-based system brings revolutionary simplicity to the art of brewing with one-touch activation. With a set of single-use capsules – which contain malt, yeast, hop oil and flavoring – and the press of a button, users can relax as LG HomeBrew automates the whole procedure from fermentation, carbonation and aging to serving and cleaning. A free companion app (for Android and iOS devices) lets users check HomeBrew’s status at any time, anywhere.

HomeBrew not only offers an incredibly simple way to make craft beer, it also enhances the quality of beer it makes. An optimized fermentation algorithm intelligently controls the fermenting process with precise temperature and pressure control for guaranteed brewing success. The clever capsule system also removes all the cleaning-related frustrations associated with traditional home-brewing methods. It automatically sanitizes using nothing more than hot water, ensuring everything is hygienically clean for the next batch.

Designed with discerning beer lovers in mind, HomeBrew allows for the in-home production of various popular styles insuring excellent results with every batch. Five distinctive, richly-flavored beers are sure to impress: hoppy American IPA, golden American Pale Ale, full-bodied English Stout, zesty Belgian-style Witbier and dry Czech Pilsner. It can produce up to five litres of premium quality beer in approximately two weeks depending on the beer type.

“LG HomeBrew is the culmination of years of home appliance and water purification technologies that we have developed over the decades,” said Song Dae-hyun, president of LG Electronics Home Appliance Air Solution Company. “Homebrewing has grown at an explosive pace but there are still many beer lovers who haven’t taken the jump because of the barrier to entry and these are the consumers we think will be attracted to LG HomeBrew.”

Visitors to CES 2019 from January 8-11 will be able to experience LG’s revolutionary HomeBrew at booth #11100 in the Las Vegas Convention Center.

About LG Electronics Home Appliance Air Solutions

LG Electronics is a global leader in home appliances, air conditioning and air quality systems. The company is creating total solutions for the home with its industry leading core technologies. LG is committed to making life better for consumers around the world by providing thoughtfully designed products, including refrigerators, washing machines, dishwashers, cooking appliances, vacuum cleaners, built-in appliances, air conditioners, air purifiers and dehumidifiers. Collectively, these products deliver enhanced convenience, superb performance, great savings and compelling health benefits. For more information, please visit  

About LG Electronics Canada Inc.

The LG brand was established in 1995. The company is a global leader in electronics, information and communications products, with more than 117 operations around the world, and annual worldwide revenues of more than US $49 billion. LG Electronics Canada Inc. is comprised of five business units – Mobile Communications, Home Appliance, Home Entertainment, Business Solutions and Commercial Air Conditioning. The company has offices in Toronto and Vancouver. LG Electronics Canada Inc. is focused on delivering award-winning products known for blending style and technology. These innovative products include cell phones, flat screen TVs, laptop computers and home appliances. For more information please visit

SOURCE LG Electronics, Inc.

A top YouTuber did a ‘blind’ test to find the very best smartphone camera, and the iPhone lost in the first round (AAPL, GOOG, GOOGL)

Tuesday December 4 th 2018

Marques Brownlee, iPhone vs BlackberryMKBHD/YouTube

  • On paper, and in technical comparisons, the iPhone XS ranks among the world’s best smartphones when it comes to photography.
  • But in a blind test conducted by popular YouTube tech vlogger Marques Brownlee, the iPhone XS flunked out in the first round.
  • Crazier still, it lost against a BlackBerry smartphone. Google’s Pixels, renowned for their camera quality, fared just as poorly.

One of the main reasons that people buy the iPhone is for its ability to take high-quality, detailed photos. And it certainly does that!

But based on the results of a new video from YouTube vlogger Marques “MKBHD” Brownlee, it seems like another factor may be far more important: Brightness.

In a massive blind photo test that Brownlee conducted over social media, he pit 16 different smartphones against each other. Both the iPhone X and the iPhone XS flunked out in the first round to less capable smartphones — Xiaomi’s Pocophone F1 and TCL’s BlackBerry Key2, respectively.

That’s right: Apple’s flagship iPhone from this year and last failed out in the first round, against phones that are barely considered competition normally.

Google’s flagship Pixel line did just as poorly, albeit against more technically competitive devices.

Marques Brownlee (Huwaei Mate 20 Pro vs Pocophone)MKBHD/YouTube

His test was simple: Put two photos of the same subject next to each other and have his millions of social media followers vote on which looked better to them. 

It’s hardly a scientific poll, but that’s not the point — what you see is what matters.

Most people are looking at photos on smartphone screens, through social media apps that compress images. They’re using apps on their smartphone to edit images before sharing. They’re trying to see faces clearly. Does the image “pop?” Is it bright? 

That kind of interaction with photos leads to a different type of preference.

“The most important thing to people, when viewing these photos straight out of camera, was just exposure — brightness, basically,” Brownlee says in the video. “Nine times out of ten, the brighter, more saturated, more punchy-contrasty photo, won. Every single round — it’s pretty consistent.” 

Marques Brownlee (MKBHD)Marques Brownlee/YouTube

It says a lot about what actually matters in smartphone cameras, and what may matter to you.

Are you taking a lot of extremely detailed photos with your smartphone? If the answer is no, then maybe you can wait a little longer next time before upgrading your smartphone — or maybe it’s finally time to start considering those mid-range, less expensive smartphones.

If nothing else, the video is a fascinating look into modern smartphone camera options — see it for yourself right here:

Sunesis Pharmaceuticals Announces Presentation of Preliminary Data from Phase 1b/2 Trial of Vecabrutinib in Patients with CLL and Other B-Cell Malignancies at ASH Annual Meeting

Sunday December 2 nd 2018

SOUTH SAN FRANCISCO, Calif., Dec. 02, 2018 (GLOBE NEWSWIRE) — Sunesis Pharmaceuticals, Inc. (Nasdaq:SNSS) today announced the presentation of results from the Company’s Phase 1b/2 clinical trial of its non-covalent BTK inhibitor vecabrutinib in adults with relapsed/refractory chronic lymphocytic leukemia (CLL) and other B-cell malignancies. The results will be presented today, December 2, from 6:00-8:00 p.m. PT in a poster session titled “CLL: Therapy, excluding Transplantation: Poster IIâ€� at the 60th American Society of Hematology (ASH) Annual Meeting in San Diego, California. The poster, titled “Preliminary Safety, Pharmacokinetic, and Pharmacodynamic Results from a Phase 1b/2 Dose-Escalation and Cohort-Expansion Study of the Noncovalent, Reversible Bruton’s Tyrosine Kinase Inhibitor, Vecabrutinib, in B-Lymphoid Malignancy Patients with Prior BTKi Therapy,â€� Abstract No. 3141, is available at

“To date, vecabrutinib has demonstrated both an encouraging safety profile and evidence of pharmacodynamic activity in CLL and other B cell cancer patients both with and without the BTK C481 mutation,� said Dayton Misfeldt, Sunesis interim Chief Executive Officer. “We believe vecabrutinib has significant potential to be an important new treatment for ibrutinib-resistant B-cell malignancy patients, and its additional activity as an ITK inhibitor suggests further directions for clinical investigation. We look forward to continuing the dose escalation, as we believe that the target dose level is likely to be between 100 mg and 300 mg BID. We are excited to be working with such thoughtful and diligent investigators at eight premier sites across the U.S., and we thank our investigators for their continued support.�

Data reported today were available from 11 of 13 treated patients. These included 7 with relapsed/refractory CLL, two with mantle cell lymphoma (MCL), and two with Waldenstrom macroglobulinemia (WM). Patients had received an average of 5 lines of prior therapy, and all had progressed on prior covalent BTK inhibitor treatment. Four of the 7 CLL patients had BTK C481 mutations. Currently, 4 patients are on study: one in Cycle 2, one in Cycle 3, and two new subjects who are in Cycle 1 and are anticipated to complete the 50 mg cohort.

The poster builds vecabrutinib’s profile in three key areas:

  • Safety: data on treatment-emergent adverse events (TEAEs) were available for 10 patients. The most common TEAEs of any grade were anemia (70%) and neutropenia and night sweats (50% each). Grade 3 drug-related AEs were anemia, neutropenia, leukocytosis, and ALT increase (10% each). In the second cohort, one patient experienced a dose-limiting toxicity of an inadequate number of Cycle 1 doses administered due to a drug-related grade 3 ALT elevation, resulting in expansion of the cohort to 6 patients.
  • Pharmacokinetics: the pharmacokinetic profile of the 50mg dose is approximately dose proportional to the 25 mg dose. The next dose levels are expected to produce plasma concentrations associated with consistently high inhibition of BTK.
  • Pharmacodynamics: vecabrutinib inhibition of BTK phosphorylation was rapid and sustained in the 5 patients who had adequate baseline signal for analysis. Decreases in serum concentrations of key cytokines associated with B-cell malignancies, CCL2, CCL3, and CCL4, were also observed in 7 patients, consistent with inhibition of BTK signaling.

Webcast Information

The data will be further discussed as part of an analyst and investor event being held in San Diego today, December 2, at 8:00 p.m. PT, with the slide webcast commencing at 8:30 p.m. PT. The event is intended for institutional investors and sell-side analysts only. Please contact for more information. The live webcast of the event, with slides, will be available to all on the Investors section of the Sunesis website at and will be archived for 90 days.

About Vecabrutinib

Vecabrutinib (SNS-062) is a selective, oral, reversible, non-covalent inhibitor of Bruton’s tyrosine kinase (BTK). BTK is a validated target for the treatment of B-cell malignancies driven by B-cell receptor signaling. Vecabrutinib retains its activity in the presence of a BTK C481S mutation, the most common mutation seen in ibrutinib-resistant CLL patients. In preclinical studies, vecabrutinib demonstrated potent activity in both wild-type and C481S-mutant BTK. Vecabrutinib has also been shown to inhibit a select number of other kinases including IL2-inducible T-cell kinase (ITK), which may improve T cell function. In a Phase 1a randomized, double-blind, placebo-controlled single ascending dose study in healthy volunteers, vecabrutinib demonstrated improved pharmacokinetics over ibrutinib, and sustained inhibition of BTK. Vecabrutinib is now being investigated in a Phase 1b/2 study in patients with relapsed CLL and other B-cell malignancies.

About Sunesis Pharmaceuticals

Sunesis is a biopharmaceutical company developing new therapeutics for the treatment of hematologic and solid cancers. Sunesis has built an experienced drug development organization committed to improving the lives of people with cancer. The Company is focused on advancing its novel kinase inhibitor pipeline, with an emphasis on its oral non-covalent BTK inhibitor vecabrutinib. Vecabrutinib is currently being evaluated in a Phase 1b/2 study in adults with chronic lymphocytic leukemia and other B-cell malignancies that have progressed after prior therapies. The Company’s proprietary PDK1 inhibitor SNS-510 is in preclinical development. PDK1 is a master kinase that activates other kinases important to cell growth and survival including members of the AKT, PKC, RSK, and SGK families. Sunesis plans to submit an IND for SNS-510 in 2019. Sunesis is exploring strategic alternatives for vosaroxin, a late-stage investigational product for relapsed or refractory AML. Sunesis also has an interest in the pan-RAF inhibitor TAK-580 which is licensed to Takeda. TAK-580 is in a clinical trial for pediatric low-grade glioma.

For additional information on Sunesis, please visit

SUNESIS and the logos are trademarks of Sunesis Pharmaceuticals, Inc.

This press release contains forward-looking statements, including statements related to the continued development of vecabrutinib (SNS-062), including the timing and preliminary results of Phase 1b/2 trial of vecabrutinib and the therapeutic potential of vecabrutinib, further development and potential of its kinase inhibitor pipeline, and planned development of SNS-510 and TAK-580. Words such as “believe,â€� “expect,â€� “future,â€� “look forward,â€� “potential,â€� “will,â€� and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Sunesis’ current expectations. Forward-looking statements involve risks and uncertainties. Sunesis’ actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, the risk related to the timing or conduct of Sunesis’ clinical trials, including the vecabrutinib Phase 1b/2 trial, the risk that Sunesis’ clinical or preclinical studies for vecabrutinib, SNS-510 or other product candidate may not demonstrate safety or efficacy or lead to regulatory approval, the risk that data to date and trends may not be predictive of future data or results, risks related to the timing or conduct of Sunesis’ clinical trials, that Sunesis’ development activities for vecabrutinib or SNS-510 could be otherwise halted or significantly delayed for various reasons, that Sunesis may not be able to receive regulatory approval of vecabrutinib, or SNS-510 in the U.S. or Europe, and risks related to Sunesis’ ability to raise the capital that it believes to be accessible and is required to fully finance the development and commercialization of vecabrutinib, SNS-510 and other product candidates. These and other risk factors are discussed under “Risk Factors” and elsewhere in Sunesis’ Quarterly Report on Form 10-Q for the quarter ended September 30, 2018 and Sunesis’ other filings with the Securities and Exchange Commission.  Sunesis expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein reflect any change in Sunesis’ expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based.


Kura Oncology Announces Proof of Concept in Angioimmunoblastic T-Cell Lymphoma, Validation of CXCL12 as a Therapeutic Target of Tipifarnib in Peripheral T-Cell Lymphoma

– Two CRs, four PRs (46% ORR) observed in advanced AITL patients in Phase 2 trial of tipifarnib in PTCL –

– AITL and other PTCL patients with high CXCL12 expression experienced 50% ORR and 90% clinical benefit with tipifarnib after median of three prior therapies –

– High expression of CXCL12 observed in approximately 40% of PTCL and was associated with poor prognosis for standard-of-care therapy –

– Management to host webcast today at 11:30 p.m. ET / 8:30 p.m. PT –

SAN DIEGO, Dec. 02, 2018 (GLOBE NEWSWIRE) —  Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported preliminary data in angioimmunoblastic T-cell lymphoma (AITL) and CXCL12+ peripheral T-cell lymphoma (PTCL), the two expansion cohorts in its Phase 2 clinical trial of its lead drug candidate tipifarnib in patients with relapsed or refractory PTCL.

The data, presented today at the American Society of Hematology (ASH) Annual Meeting in San Diego, showed encouraging activity with tipifarnib in late-stage PTCL patients, including a significant association between CXCL12 expression and clinical benefit, and proof of concept in AITL, an aggressive form of T-cell lymphoma often characterized by high levels of CXCL12 expression. A copy of the poster is available on the Company’s website at

“The mechanism of action of farnesyl transferase inhibitors has remained elusive for several decades,� said Antonio Gualberto, M.D., Ph.D., Head of Development and Chief Medical Officer of Kura Oncology. “Our initial data in HRAS mutant head and neck cancer provided strong evidence of activity in tumors driven by this oncogene. However, many other tumors such as T- and B-cell lymphomas, myeloid leukemias, pancreatic or breast cancers, in which anecdotal evidence of tipifarnib activity has been reported, do not usually carry HRAS mutations. We believe the preliminary results reported at ASH validate our observation that the CXCL12 pathway is a therapeutic target of tipifarnib and provide a potential path to expand the development of tipifarnib well beyond HRAS mutant solid tumors by using CXCL12-related biomarkers to enrich for patients most likely to benefit from treatment. We will continue our efforts to identify these patient subsets and to bring this important drug candidate to patients in need.�

As of November 21, 2018, a total of 39 patients were enrolled in the ongoing Phase 2 trial, including 19 patients with AITL (16 patients in the AITL extension cohort and 3 patients in the previous portion of the study). Six of the 16 AITL patients were not evaluable as of the data cutoff date, including two who were pending initial efficacy assessments. Of the 13 evaluable AITL patients, two achieved a complete response (CR) and four achieved a partial response (PR), for an objective response rate (ORR) of 46% (six of 13). According to the study protocol, the AITL cohort is considered positive when four or more responses are observed.

The study also identified a particularly responsive subset within AITL and non-AITL patients. Specifically, patients with a high ratio of expression of CXCL12 to its receptor CXCR4 experienced a 50% ORR (five of 10) and a 90% clinical benefit rate (nine of 10 with either complete response, partial response or stable disease) with tipifarnib. Patients in this Phase 2 trial had a median of three prior lines of therapy (range 1-7). The high CXCL12/CXCR4 expression ratio had 90% sensitivity and 93% specificity to identify PTCL patients likely to benefit from tipifarnib.

In addition to the Phase 2 clinical data, the results from two ancillary, non-clinical studies were also reported at ASH. In the first, the expression of CXCL12 and CXCR4 was investigated using tumor bank samples of PTCL patients treated with standard-of-care agents. Worse prognosis was observed in PTCL patients with high CXCL12/CXCR4 expression ratio, indicating that CXCL12 is a negative prognostic factor for standard PTCL therapy. In the second study, the effect of the incubation of stroma cells with tipifarnib on CXCL12 secretion was investigated in a mouse model of bone marrow culture. Tipifarnib reduced secretion of the CXCL12 chemokine from the stromal cells, providing a potential mechanism of action for the observed clinical activity.

“To our knowledge, these results position tipifarnib as the first CXCL12 inhibitor with reported proof-of-concept data, marking a significant advancement in our development strategy,� said Troy Wilson, Ph.D., President and CEO of Kura Oncology. “Our data suggest that as many as 40% of PTCL patients express high CXCL12. In addition, the discovery of CXCL12-related biomarkers offers the potential to increase the opportunity for tipifarnib into other hematological and solid tumor indications. We intend to explore those indications while continuing to gather additional data from our ongoing Phase 2 study and expect to provide an update at a medical meeting next year.�

The Phase 2 study was designed to investigate the anti-tumor activity of tipifarnib in patients with relapsed or refractory PTCL. After preliminary data suggested that CXCL12 expression was associated with tipifarnib’s clinical activity, two expansion cohorts were added to enroll patients with tumors expected to overexpress CXCL12: AITL tumors, and those non-AITL tumors that lack a single nucleotide variation in the 3’-untranslated region (3′-UTR) of the CXCL12 gene (CXCL12+ PTCL). CXCL12 is a stroma-derived chemokine that promotes the progression of lymphoma and other hematological and solid tumors carrying the CXCR4 receptor, and our previous results had suggested an association between CXCL12 expression and the most common form of the 3’-UTR CXCL12 variant. In the expansion cohorts, both the presence or absence of this variant and the expression levels of CXCL12 and CXCR4 were assessed.

In the expansion cohorts in the Phase 2 trial tipifarnib was dosed at 300 mg twice daily on days 1-21 of a 28-day cycle. The reported data indicated that tipifarnib was generally well-tolerated, with adverse events consistent with its known safety profile. The most common treatment-related adverse events (grade ≥ 3) were hematology-related, including neutropenia, thrombocytopenia, leukopenia, febrile neutropenia and anemia.

Last week, the U.S. Patent and Trademark Office (USPTO) issued a new patent for tipifarnib as a method of treating patients with AITL. In May 2018, the USPTO issued a patent for tipifarnib as a method of treating patients with certain CXCL12-expressing cancers, including PTCL. Both patents expand protection for tipifarnib, providing exclusivity in the U.S. to 2037.

Webcast Information

Kura’s management will host a webcast at 11:30 p.m. ET / 8:30 p.m. PT today, December 2, 2018, following the conclusion of the poster presentation at the ASH 2018 Annual Meeting. The live audio webcast and slides of the presentation will be available from the Investors and Media section of the company website at, and will be archived there for 30 days.

About Tipifarnib

Kura Oncology’s lead drug candidate, tipifarnib, is a potent and highly selective inhibitor of farnesylation, a key cell signaling process implicated in cancer initiation and development. Tipifarnib was previously studied in more than 5,000 cancer patients and showed compelling and durable anti-cancer activity in certain patient subsets, however no molecular mechanism of action had previously been determined that could explain its activity across a range of diverse clinical indications, including squamous tumors that carry mutant HRAS, as well as in lymphoid, myeloid and solid tumors that do not carry HRAS mutations. Leveraging advances in next-generation sequencing as well as emerging information about cancer genetics and tumor biology, Kura is seeking to identify those patients most likely to benefit from tipifarnib. In November 2018, Kura initiated its first global, registration-directed trial of tipifarnib in patients with recurrent or metastatic HRAS mutant HNSCC.

About Kura Oncology

Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer. The Company’s pipeline consists of small molecule drug candidates that target cancer signaling pathways where there is a strong scientific and clinical rationale to improve outcomes by identifying those patients most likely to benefit from treatment. Kura’s lead drug candidate is tipifarnib, a farnesyl transferase inhibitor, for which the Company has initiated a registration-directed trial of tipifarnib in recurrent or metastatic patients with HRAS mutant HNSCC. In addition, tipifarnib is being evaluated in multiple other Phase 2 clinical trials in solid tumor and hematologic indications. Kura’s pipeline also includes KO-947, an ERK inhibitor, currently in a Phase 1 dose-escalation trial, and KO-539, a menin-MLL inhibitor, currently in IND-enabling studies. For additional information about Kura Oncology, please visit the Company’s website at

Forward-Looking Statements

This news release contains certain forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, the efficacy, safety and therapeutic potential of tipifarnib and the Company’s other product candidates, the conduct, results and timing of Kura Oncology’s clinical trials, including the Phase 2 clinical trial of tipifarnib in patients with PTCL, plans regarding future clinical trials and development and commercial activities, the regulatory approval path for tipifarnib and expectations regarding intellectual property and biomarkers related to Kura Oncology’s product candidates. Factors that may cause actual results to differ materially include the risk that compounds that appeared promising in early research or clinical trials do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials, the risk that Kura Oncology may not obtain approval to market its product candidates, uncertainties associated with performing clinical trials, regulatory filings and applications, risks associated with reliance on third parties to successfully conduct clinical trials, the risks associated with reliance on outside financing to meet capital requirements, and other risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. You are urged to consider statements that include the words “may,� “will,� “would,� “could,� “should,� “believes,� “estimates,� “projects,� “promise,� “potential,� “expects,� “plans,� “anticipated,� “intends,� “continues,� “designed,� “goal,� or the negative of those words or other comparable words to be uncertain and forward-looking. For a further list and description of the risks and uncertainties the Company faces, please refer to the Company’s periodic and other filings with the Securities and Exchange Commission, which are available at Such forward-looking statements are current only as of the date they are made, and Kura Oncology assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.


Pete De Spain
Vice President, Investor Relations
Corporate Communications
(858) 500-8803

Robert H. Uhl
Managing Director
Westwicke Partners, LLC
(858) 356-5932

Jason Spark
Managing Director
Canale Communications
(619) 849-6005

Blueprint Medicines Announces Updated Results from Ongoing EXPLORER Clinical Trial of Avapritinib Demonstrating Broad Clinical Activity and Significant Symptom Reductions in Patients with Systemic…

CAMBRIDGE, Mass., Dec. 2, 2018 /PRNewswire/ — Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, today announced updated results for the Phase 1 EXPLORER clinical trial of avapritinib, a potent and highly selective inhibitor of D816V mutant KIT, the common disease driver in nearly all patients with systemic mastocytosis (SM). The updated EXPLORER trial data in patients with advanced SM showed durable clinical responses that deepened over time, regardless of disease subtype, prior therapy or starting dose. Avapritinib was generally well-tolerated, and most adverse events (AEs) reported by investigators were Grade 1 or 2. These results will be presented today in an oral presentation at the 60th American Society of Hematology Annual Meeting and Exposition in San Diego, California.


As of the data cutoff date of September 30, 2018, the updated results from the ongoing EXPLORER trial showed an overall response rate (ORR) of 83 percent. Twenty-four percent of patients had a complete response with a full or partial recovery of peripheral blood counts (CR/CRh). Responses deepened over time, with a median time to initial response of two months and a median time to CR/CRh of nine months. The median duration of response (DoR) was not reached, and the 12-month DoR rate was 76 percent.

In addition, statistically significant improvements in patient-reported disease symptoms were observed. A 41 percent mean reduction (p=0.043) in patient-reported disease symptoms was demonstrated on the Advanced SM Symptom Assessment Form (AdvSM-SAF), the first patient-reported outcomes tool designed specifically to assess advanced SM.

“Systemic mastocytosis is a complex rare disorder that causes debilitating symptoms across all forms of the disease and reduced survival in advanced patients,” said Jason Gotlib, M.D., professor of Medicine, Hematology, at the Stanford University Medical Center and an investigator on the EXPLORER trial. “The updated EXPLORER study results show that selectively targeting D816V mutant KIT with avapritinib led to profound and durable clinical activity in patients with advanced systemic mastocytosis, including significant improvements in patient-reported disease symptoms and quality of life. Combined with encouraging preliminary data from an initial cohort of indolent systemic mastocytosis patients from the EXPLORER study, these results highlight the potential of avapritinib to improve objective and subjective measures of disease burden across the spectrum of mastocytosis subtypes.”

“The data further validate Blueprint Medicines’ precision therapy approach, where we target genetic drivers of disease with potent and highly selective inhibitors,” said Andy Boral, M.D., Ph.D., Chief Medical Officer of Blueprint Medicines. “With robust clinical data from the EXPLORER trial, favorable FDA feedback on potential registration pathways and Breakthrough Therapy Designation for advanced systemic mastocytosis, we believe avapritinib has a strong foundation for expedited development across all forms of the disease. In particular, the new data showing a significant decrease in symptom burden in patients enrolled in the EXPLORER trial increase our confidence in avapritinib’s potential in indolent systemic mastocytosis.”

Data Highlights from the Ongoing Phase 1 EXPLORER Clinical Trial

As of the data cutoff date of September 30, 2018, 67 patients were treated with avapritinib in the dose escalation and expansion portions of the Phase 1 EXPLORER clinical trial, including 23 patients with aggressive SM (ASM), 30 patients with advanced SM with an associated hematological neoplasm (SM-AHN), seven patients with mast cell leukemia (MCL) and seven patients with indolent or smoldering SM. Forty patients (60 percent) had a prior treatment, including 14 patients (23 percent) who had previously received Rydapt® (midostaurin).

Safety Data

As of the data cutoff date, avapritinib was generally well-tolerated. Most AEs were reported by investigators as Grade 1 or 2. Across all enrolled patients, 52 patients (78 percent) remained on treatment as of the data cutoff date. Three patients (4 percent) discontinued treatment with avapritinib due to treatment-related AEs.

Across all grades, the most common non-hematological treatment-emergent AEs (regardless of relationship to avapritinib) reported by investigators (15 percent) were periorbital edema, fatigue, nausea, diarrhea, peripheral edema, vomiting, cognitive effects, hair color changes, arthralgia, dizziness and abdominal pain. The most common hematological treatment-emergent AEs reported by investigators (10 percent) were anemia, thrombocytopenia and neutropenia. Grade 3 and 4 treatment-related AEs occurred in 44 patients (66 percent), and these events were most commonly hematological AEs, typically in patients with low blood counts (cytopenias) at study entry.

Clinical Activity Data

IWG-MRT-ECNM Assessments and Objective Measures of Mast Cell Burden                               

As of the data cutoff date, 29 patients were evaluable for response by the modified IWG-MRT-ECNM criteria, a rigorous method for assessing clinical response in advanced SM patients with regulatory precedent in the U.S. and Europe. Responses were centrally reviewed by a committee of SM experts.

Avapritinib demonstrated durable clinical responses across all doses studied and in each subtype of advanced SM – ASM, SM-AHN and MCL. The duration of treatment was up to 31 months as of the data cutoff date, with a median follow-up time of 14 months in evaluable patients.

Across all evaluable patients at all doses, the ORR was 83 percent. Seven patients had a CR/CRh (24 percent, two pending confirmation), 14 patients had a partial response (48 percent, three pending confirmation) and three patients had clinical improvement (10 percent, one pending confirmation). Three of the pending responses were previously confirmed responses (two partial responses, one clinical improvement) that are transitioning to a deeper response. No patients had documented disease progression per modified IWG-MRT-ECNM criteria.

In addition, strong clinical activity was demonstrated in evaluable patients treated with a starting dose of less than or equal to 200 mg once daily (QD), the dose under evaluation in the ongoing registration-enabling Phase 2 PATHFINDER clinical trial in patients with advanced SM. These 10 patients had an ORR of 90 percent and a CR/CRh rate of 50 percent (one complete response pending confirmation).

All patients evaluable on objective measures of mast cell burden showed reductions from baseline. These results were shown regardless of disease subtype, prior therapy (including midostaurin or the investigational agent DCC-2618), or co-mutation status. These measures consisted of declines in bone marrow mast cells, serum tryptase, spleen volume and KIT D816V mutant allele burden.

At baseline, 22 SM patients received steroids for mastocytosis symptoms. As of the data cutoff date, 18 patients (80 percent) decreased their steroid dose, including nine patients (41 percent) who were able to entirely discontinue their steroids.

Patient-Reported Outcomes

As of the data cutoff date, 32 patients in the dose expansion portion of the Phase 1 EXPLORER trial were evaluated using the AdvSM-SAF, the first patient-reported outcomes tool developed specifically for advanced SM patients. It was designed to evaluate symptoms across the gastrointestinal domain (abdominal pain, diarrhea, nausea and vomiting) and skin domain (spots, itching and flushing), as well as fatigue. Patients reported their symptoms daily using an electronic diary.

In advanced SM patients, benefits were shown across each individual symptom studied. There was a 41 percent improvement from baseline in the AdvSM-SAF Total Symptom Score (p=0.043). The most symptomatic patients (n=16, top 50th percentile) had the largest mean improvement in the AdvSM-SAF Total Symptom Score (46 percent, p=0.038).

In addition, an improvement in patient-reported quality of life was observed. As of the data cutoff date, 30 patients were evaluated using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-30), a validated and commonly used patient-reported outcomes tool in oncology clinical trials. Results showed a statistically significant improvement in quality of life score, approaching levels observed in healthy aged-matched controls, with a pronounced improvement observed in the most symptomatic patients.

Proof-of-Concept in Indolent and Smoldering SM

All evaluable patients with indolent and smoldering SM showed profound reductions in bone marrow mast cells, serum tryptase, spleen volume and KIT D816V mutant allele burden. The data also showed avapritinib led to improvements in patient-reported symptoms. These encouraging data support Blueprint Medicines’ planned Phase 2 PIONEER clinical trial in patients with indolent and smoldering SM.

About the Clinical Development Program for Avapritinib in SM

Blueprint Medicines is pursuing a broad clinical development program for avapritinib across advanced, indolent and smoldering forms of SM. Avapritinib is currently being evaluated in two ongoing clinical trials for advanced SM: the Phase 1 EXPLORER clinical trial and the registration-enabling Phase 2 PATHFINDER clinical trial.

The Phase 1 EXPLORER clinical trial of avapritinib was designed to identify the recommended Phase 2 dose for further study and demonstrate proof-of-concept in advanced SM. The dose escalation portion is complete, and the expansion portion of the trial is enrolling patients with ASM, SM-AHN and MCL at multiple sites in the United States and United Kingdom. Trial objectives include assessing safety and tolerability, response per modified IWG-MRT-ECNM criteria and patient-reported outcomes.

The Phase 2 PATHFINDER clinical trial is an open-label, single-arm, registration-enabling clinical trial in patients with advanced SM. Patient dosing is now ongoing in the clinical trial, which is designed to enroll up to 60 advanced SM patients at sites in the United States, Canada and Europe. The primary efficacy endpoints are ORR and DoR based on modified IWG-MRT-ECNM criteria.

Blueprint Medicines expects to initiate the Phase 2 PIONEER clinical trial, a randomized, placebo-controlled, registration-enabling trial in patients with indolent and smoldering SM, by the end of 2018. The trial’s primary endpoint will be symptom reductions for avapritinib versus placebo based on the Indolent and Smoldering SM Assessment Form Total Symptom Score. All patients who complete the dose-finding (part 1) and placebo-controlled efficacy (part 2) portions of this trial will have an opportunity to receive avapritinib in an open-label extension (part 3).

SM patients and clinicians interested in ongoing or planned clinical trials can contact the Blueprint Medicines study director at or 1-617-714-6707. Additional details are available at, or

About SM

SM results from the abnormal proliferation and survival of mast cells, which mediate allergic responses. There are several forms of the disease, including indolent SM, smoldering SM and three advanced subtypes – ASM, SM-AHN and MCL. The KIT D816V mutation drives approximately 90 to 95 percent of all SM cases, causing debilitating and difficult-to-manage symptoms such as pruritus, flushing, headaches, bone pain, nausea, vomiting, diarrhea, anaphylaxis, abdominal pain and fatigue. While these effects occur across SM patients, symptom burden and poor quality of life are the predominant disease manifestations of indolent and smoldering SM. Advanced SM patients experience organ damage and a median overall survival of about 3.5 years in ASM, two years in SM-AHN and less than six months in MCL.

Currently, there are no approved therapies that selectively inhibit KIT D816V in advanced SM, and no approved therapies for indolent and smoldering SM. New treatments are needed that are more effective and better tolerated than existing advanced SM therapy, as well as for indolent and smoldering SM patients whose symptoms are often not well controlled with symptom-directed therapies.

About Avapritinib

Avapritinib is a potent and selective oral inhibitor of KIT and PDGFRA mutant kinases. It is a type 1 inhibitor designed to target the active kinase conformation; all oncogenic kinases signal via this conformation. Avapritinib has demonstrated broad inhibition of KIT and PDGFRA mutations associated with gastrointestinal stromal tumors (GIST), and the most potent activity against activation loop mutations, which currently approved therapies for GIST do not inhibit. In contrast with existing multi-kinase inhibitors, avapritinib has shown marked selectivity for KIT and PDGFRA over other kinases. In addition, avapritinib is uniquely designed to selectively bind and inhibit D816V mutant KIT, the primary driver of disease in approximately 90 to 95 percent of all SM patients. Preclinical studies have shown avapritinib potently inhibited KIT D816V at sub-nanomolar potencies with minimal off-target activity.

Blueprint Medicines is initially developing avapritinib, an investigational medicine, for the treatment of advanced GIST, advanced SM, and indolent and smoldering SM. The U.S. Food and Drug Administration has granted avapritinib two Breakthrough Therapy Designations, one for the treatment of PDGFRα D842V-driven GIST and one for advanced SM.

Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of avapritinib and certain other drug candidates in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for avapritinib in the rest of the world.

About Blueprint Medicines

Blueprint Medicines is developing a new generation of targeted and potent kinase medicines to improve the lives of patients with genomically defined diseases. Its approach is rooted in a deep understanding of the genetic blueprint of cancer and other diseases driven by the abnormal activation of kinases. Blueprint Medicines is advancing multiple programs in clinical development for subsets of patients with gastrointestinal stromal tumors, hepatocellular carcinoma, systemic mastocytosis, non-small cell lung cancer, medullary thyroid cancer and other advanced solid tumors, as well as multiple programs in research and preclinical development. For more information, please visit

Cautionary Notes Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding plans and timelines for the clinical development of avapritinib, including plans and timelines for the ongoing Phase 1 EXPLORER clinical trial, ongoing Phase 2 PATHFINDER clinical trial and planned Phase 2 PIONEER clinical trial; expectations regarding the potential for the Phase 1 EXPLORER clinical trial, Phase 2 PATHFINDER clinical trial or Phase 2 PIONEER clinical trial to be registration-enabling for avapritinib in SM; Blueprint Medicines’ ability to implement its clinical development plans for avapritinib in SM; expectations regarding the potential benefits of avapritinib in treating patients with SM, including advanced, indolent and smoldering SM; expectations regarding the development of avapritinib as a treatment for patients with SM, including advanced, indolent and smoldering SM; expectations regarding the potential for expedited development of avapritinib; and Blueprint Medicines’ strategy, business plans and focus. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks and uncertainties related to the delay of any current or planned clinical trials or the development of Blueprint Medicines’ drug candidates, including avapritinib, BLU-554, BLU-667 and BLU-782; Blueprint Medicines’ advancement of multiple early-stage efforts; Blueprint Medicines’ ability to successfully demonstrate the safety and efficacy of its drug candidates; the preclinical and clinical results for Blueprint Medicines’ drug candidates, which may not support further development of such drug candidates; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; Blueprint Medicines’ ability to develop and commercialize companion diagnostic tests for its current and future drug candidates, including companion diagnostic tests for avapritinib for PDGFRα D842V-driven GIST, BLU-554 for FGFR4-driven hepatocellular carcinoma and BLU-667 for RET-driven non-small cell lung cancer; the success of Blueprint Medicines’ current and future collaborations, including its cancer immunotherapy collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. and its collaboration with CStone Pharmaceuticals. These and other risks and uncertainties are described in greater detail in the section entitled “Risk Factors” in Blueprint Medicines’ Quarterly Report on Form 10-Q for the quarter ended September 30, 2018, as filed with the Securities and Exchange Commission (SEC) on October 30, 2018, and any other filings that Blueprint Medicines has made or may make with the SEC in the future. Any forward-looking statements contained in this press release represent Blueprint Medicines’ views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements.


Rydapt® is a registered trademark of Novartis AG. All other trademarks and trade names in this press release are the property of Blueprint Medicines Corporation or used with permission.

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