On August 1, 2018 Novavax, Inc. (Nasdaq:NVAX) reported it will report its second quarter 2018 financial and operating results following the close of U.S. financial markets on Wednesday, August 8, 2018 (Press release, Novavax, AUG 1, 2018, http://ir.novavax.com/news-releases/news-release-details/novavax-host-conference-call-discuss-second-quarter-financial-1 [SID1234528297]).

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Conference call details are as follows:

Date: August 8, 2018
Time: 4:30 p.m. U.S. Eastern Time (ET)
Dial-in number: (877) 212-6076 (Domestic) or (707) 287-9331 (International)
Passcode: 5886748
Webcast: www.novavax.com, "Investors"/ "Events"

Conference call and webcast replay:

Dates: Starting at 7:30 p.m. ET, August 8, 2018 until 7:30 p.m. ET August 15, 2018
Dial-in number: (855) 859-2056 (Domestic) or (404) 537-3406 (International)
Passcode: 5886748
Webcast: www.novavax.com, "Investors"/ "Events", until November 8, 2018

On August 1, 2018 Foamix Pharmaceuticals Ltd. (NASDAQ: FOMX), ("Foamix"), a clinical stage specialty pharmaceutical company focused on developing and commercializing proprietary topical foams to address unmet needs in dermatology, reported that it will report financial results for the three and six-month periods ended June 30, 2018, on Wednesday, August 8 after the markets close (Press release, Foamix, AUG 1, 2018, View Source [SID1234528363]).

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Due to the proximity of the Company receiving data from the confirmatory Phase 3 study of FMX101 in acne, the Company will not be hosting an earnings conference call. As previously disclosed, the Company expects to announce top line results from this Phase 3 study in acne during this current quarter and will address investor questions, including those regarding financial results, at that time

On August 1, 2018 MorphoSys AG reported its results for the second quarter of 2018(Press release, MorphoSys, AUG 1, 2018, View Source/medien-investoren/mediencenter/morphosys-ag-gibt-ergebnisse-des-zweiten-quartals-2018-bekannt" target="_blank" title="View Source/medien-investoren/mediencenter/morphosys-ag-gibt-ergebnisse-des-zweiten-quartals-2018-bekannt" rel="nofollow">View Source [SID1234528298]).

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The company has continued to expand its pipeline of proprietary and partner-developed programs and established a presence in the US to prepare for commercialization

Conference Call and Webcast on August 2, 2018 at 2:00 pm CEST

– ongoing discussions with the US Food and Drug Administration (FDA) on marketing approval of MOR208 as a potential treatment for aggressive lymphoma (DLBCL) within the existing status of the treatment breakthrough

– First clinical data from the ongoing Phase 2 trial with MOR208 plus idelalisib in CLL presented at the annual meeting of the European Hematology Association (EHA) (Free EHA Whitepaper)

– Jennifer Herron takes over leadership of the newly formed MorphoSys US Inc. and is responsible for building commercial structures for MOR208

– MorphoSys and Galapagos sign a worldwide licensing agreement with Novartis for MOR106. MorphoSys and Galapagos will jointly receive an upfront payment of € 95 million and significant potential future milestone payments and royalties in the double-digit percentage range

– Partner Janssen starts phase 2/3 program with Tremfya (R) in Crohn’s disease

– Partner Roche launches phase 3 trial with gantenerumab in patients with early Alzheimer’s disease

– US Nasdaq IPO and successful capital increase with gross proceeds of $ 239 million completed in April 2018

– Liquidity position increased to 450.5 million euros (as at 30 June 2018)

– Following the signing of a license agreement with Novartis for MOR106 and subject to US antitrust approval, MorphoSys is increasing its financial guidance for 2018 and expects sales of between EUR 67 and 72 million, EBIT of EUR -55 to -65 million and expenses for the development of proprietary programs and technology development from 87 to 97 million euros

MorphoSys AG (Prime Standard Segment, TecDAX, NASDAQ: MOR) reported its results for the second quarter of 2018.

"MorphoSys has made excellent progress in many areas during the quarter, and we have continued our constructive discussions with the FDA on a possible route to market approval for MOR208 for the treatment of aggressive lymphoma (DLBCL) and positive data from a current Phase 2 trial Study with MOR208 in chronic lymphocytic leukemia (CLL), "commented Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG. "With the formation of our new US subsidiary MorphoSys US Inc. and the appointment of Jennifer Herron as their director, we have laid the foundation for a strong sales presence to prepare for the potential future commercialization of MOR208, subject, of course, to approval for this development Drug by the FDA. "

"We are also very pleased with the progress made by our partners, notably the successful marketing of Tremfya (R) to treat psoriasis by Janssen, as well as the launch of additional pivotal development programs with Tremfya (R) in Crohn’s disease by Janssen and Gantenerumab in Alzheimer’s by Roche, "Dr. Moroney continues.

"An exciting quarter is behind us, and MorphoSys’s recent corporate developments are giving us an optimistic outlook for the future: In April, we completed our IPO on Nasdaq and signed an exclusive license agreement with Novartis for MOR106 shortly after the end of the quarter," said Jens Holstein , Chief Financial Officer of MorphoSys AG. "Our strong financial position allows us to provide the necessary resources for our most advanced MOR208 program, to continue developing our remaining pipeline programs and to expand our commercial activities in the US."

Financial Report for the second quarter of 2018 (IFRS, all figures rounded)

In the second quarter of 2018, MorphoSys continued to focus on drug discovery and development, both independently and for partners. Consolidated revenues in the second quarter of 2018 came to EUR 8.1 million (Q2 2017: EUR 11.7 million). The expected decrease compared to the second quarter of the previous year mainly results from the termination of the partnership with Novartis in 2017. As Janssen’s contractually agreed revenue share for the second quarter of 2018 has not yet been received due to the reporting periods of Janssen and MorphoSys, Based on Janssen / J & J’s publicly announced Tremfya (R) sales revenues, the second-quarter 2018 Tremfya (R) revenue was recognized in the second quarter of 2018.

Im Segment Proprietary Development konzentriert sich MorphoSys auf die Erforschung und Entwicklung eigener Produktkandidaten in den Bereichen Krebs und entzündliche Erkrankungen. In Q2 2018 verzeichnete dieses Segment Umsätze von 0,1 Mio. Euro (Q2 2017: 0,3 Mio. Euro). Im Segment Partnered Discovery setzt MorphoSys seine firmeneigene Technologie ein, um neue Antikörper für Pharmaunternehmen zu generieren. Das Unternehmen profitiert von den Entwicklungsfortschritten der Partner in Form von finanzierten Forschungsleistungen, Lizenzgebühren, erfolgsbasierten Meilensteinzahlungen und Tantiemen (Umsatzbeteiligungen). Im ersten Quartal 2018 beliefen sich die Umsätze in diesem Segment auf 8,1 Mio. Euro (Q2 2017: 11,5 Mio. Euro).

Die gesamten betrieblichen Aufwendungen erreichten im zweiten Quartal 2018 eine Höhe von 32,7 Mio. Euro (Q2 2017: 27,5 Mio. Euro). Die Aufwendungen für die Entwicklung eigener Produkte und Technologieentwicklung beliefen sich auf 23,7 Mio. Euro (Q2 2017: 18,6 Mio. Euro). Die Zunahme im Vergleich zum Vorjahr ist vor allem auf die Kosten für die Weiterentwicklung von MOR208 zurückzuführen.

Earnings before interest and taxes (EBIT) amounted to € -24.1 million in the second quarter of 2018 (Q2 2017: € -15.4 million). The Proprietary Development segment generated EBIT of
€ -24.6 million (Q2 2017: € -18.3 million). EBIT in the Partnered Discovery segment amounted to € 5.5 million (Q2 2017: € 6.8 million).
Consolidated net income amounted to EUR -23.5 million in the second quarter of 2018 (Q2 2017: EUR -16.1 million). Earnings per share reached -0.76 euros (Q2 2017: -0.56 euros).

At the end of the second quarter of 2018, MorphoSys had cash and cash equivalents of € 450.5 million, compared to € 312.2 million at December 31, 2017. This cash and cash equivalents are shown in the balance sheet in the following items: cash and cash equivalents ; financial assets at fair value, with changes recognized in profit or loss, and other current and non-current financial assets at amortized cost. The increase in cash and cash equivalents resulted primarily from the capital increase in the context of the successful Nasdaq IPO in April 2018 with gross proceeds of $ 239 million. Part of the cash and cash equivalents were used for operating expenses in the second quarter of 2018.

The total number of shares outstanding was 31,808,035 at the end of the second quarter of 2018 (31 December 2017: 29,420,758). The main reason for the increase in the number of shares was the capital increase in connection with the listing on the Nasdaq in April 2018.

Result for the first half of 2018

In the first six months of 2018, Group sales amounted to € 10.9 million (Q1-Q2 2017: € 23.6 million). Expenses for the development of own products and technology development amounted to 39.2 million Euro in the first six months of 2018 (Q1-Q2 2017: 37.3 million Euro). EBIT in the first six months of 2018 was thus EUR -43.2 million, compared with EUR -30.3 million in the first half of 2017.

Financial forecast and operational outlook for 2018

Following the recent signing of an agreement with Novartis for MOR106 and subject to the approval of the US antitrust authorities, MorphoSys is raising its financial guidance for 2018. Subject to approval by the US antitrust authorities, MorphoSys expects revenues of € 67 million to € 72 million and Earnings before interest and taxes (EBIT) of € -55 million to € -65 million. R & D expenses for proprietary programs and technology development are expected in a range of € 87 million to € 97 million. This forecast does not include additional revenues from potential future collaborations and / or licensing partnerships nor effects from possible in-licensing or development partnerships for new drug candidates.

In the Proprietary Development segment, MorphoSys expects the following events and activities for the current year:

MOR208

– L-MIND: Continued analysis of all 81 patients with relapsed / refractory diffuse large B-cell lymphoma (R / R DLBCL) enrolled in the study and presented updated clinical data at an appropriate medical conference.

– B-MIND: Continuation of the pivotal phase 3 trial with MOR208 plus bendamustine versus rituximab plus bendamustine in R / R DLBCL.

– COSMOS: continuation of Phase 2 trial with MOR208 in combination with idelalisib or Venetoclax in CLL / SLL and presentation of results from cohort B of the study (MOR208 plus Venetoclax) at an appropriate scientific conference.

– Commercial activities: Further build commercial structures for MOR208 in the US, under the umbrella of the newly formed MorphoSys US Inc., in anticipation of a potential launch, which is currently expected in 2020, subject to regulatory approval from the US Food and Drug Administration.

MOR202

– Multiple Myeloma (MM): MorphoSys has decided not to continue the development of MOR202 in MM beyond the completion of the ongoing Phase 1 / 2a trial; the final data is expected to be presented at one of the next medical conferences. MorphoSys will support its partner I-Mab as planned in the development of MOR202 for the Chinese market.

– Lung cancer (NSCLC): After Janssen discontinued a clinical trial with the CD38 antibody daratumumab in combination with a checkpoint inhibitor, MorphoSys has decided not to continue its activities in NSCLC for the time being.

– Other indications: MorphoSys continues to investigate the development of MOR202 in other indications.

MOR106: Continuing ongoing development with Galapagos as part of the new global licensing partnership with Novartis:

– Continuation of ongoing Phase 2 trial IGUANA in atopic dermatitis.

– Start of a Phase 1 study to investigate a subcutaneous formulation of MOR106.

– All future costs associated with the development of MOR106 will be borne by Novartis.

– Agreement between MorphoSys, Galapagos and Novartis is subject to approval by the US antitrust authorities.

MOR107: Continuation of pre-clinical studies of MOR107 with a focus on cancer indications to prepare a decision to conduct further clinical trials.

MOR103 / GSK3196165: Following the recent announcement by GSK that positive Phase 2b study results for GSK3196165 in rheumatoid arthritis will be presented at a future scientific conference and that the indication for osteoarthritis has been terminated, clinical data are expected to be published by GSK.

In the Partnered Discovery segment, MorphoSys 2018 expects the following events:

Tremfya (R) (Guselkumab): For several phase 3 studies in psoriasis, clinicaltrials.gov expects primary completion date in 2018, including a comparative study with Tremfya (R) and Cosentyx (R) (Secukinumab) in plaque psoriasis.

Other Affiliate Programs: Clinical data and possible regulatory milestones could be released throughout the year for many other affiliate programs.

MorphoSys will continue to expand its own development activities and review potential in-licensing, co-development and / or acquisition opportunities. In addition, the company plans to initiate further development programs of its own with the aim of maintaining and expanding the company’s position in its current therapeutic and technological fields of activity.

Percentage points ** Including MOR107, for which a Phase 1 trial with healthy volunteers was completed. MOR107 is currently undergoing preclinical research focusing on oncology. Due to ongoing studies in various indications, we continue to consider Tremfya (R) as a Phase 3 program

*** Includes MOR103 / GSK3196165, which is fully out-licensed to GSK.

MorphoSys will host a webcast public conference call on August 2, 2018, to present the financial results for the second quarter of 2018 and the outlook for 2018.

Dial-in dates for the analyst conference call (2:00 pm CEST) (Listening mode):
Germany +49 (0) 69 201 744 210
+49 (0) 89 204 049 750

Participant PIN 63419794 #

Please dial in ten minutes before the start of the conference. A live webcast and the presentation will also be available at View Source . About two hours after the end of the conference, you will be able to access an audio replay synchronized with the presentation and the transcript of the conference at View Source .

On august 1, 2018 United Therapeutics Corporation (NASDAQ: UTHR) reported its financial results for the quarter ended June 30, 2018 (Press release, United Therapeutics, AUG 1, 2018, View Source [SID1234528401]).

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"Our second quarter net revenues totaled $444 million and we are treating a larger number of patients, compared to the prior year, suffering from pulmonary arterial hypertension with our prostacyclin product franchise, which consists of Orenitram, Remodulin, and Tyvaso," said Martine Rothblatt, Ph.D., Chairman and Chief Executive Officer of United Therapeutics. "We also continued to invest in our innovative product pipeline, which includes seven Phase III clinical trials in cardiopulmonary diseases and oncology as well as programs in regenerative medicine and organ manufacturing to ultimately provide a cure for PAH and other end-stage organ diseases. We believe this product pipeline uniquely positions United Therapeutics to deliver long-term revenue growth to our stakeholders."

Update on SteadyMed Acquisition

Our previously-announced acquisition of SteadyMed Ltd. (SteadyMed) has satisfied two key closing conditions. On July 20, 2018, the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 expired, and on July 30, 2018, SteadyMed’s shareholders approved the transaction. Under Israeli law, closing may not occur until at least thirty days have passed since the SteadyMed shareholders approved the transaction. Assuming all remaining conditions to closing of this transaction are satisfied or waived, we expect the transaction to be completed in the third quarter of this year. SteadyMed’s lead drug product candidate is Trevyent, a development-stage drug-device combination product that combines SteadyMed’s PatchPump technology with treprostinil to treat pulmonary arterial hypertension.

Financial Results for the Three Months Ended June 30, 2018 compared to the Three Months Ended June 30, 2017

Key financial highlights include (dollars in millions, except per share data):

Three Months Ended
June 30,

Dollar

Percentage

2018

2017

Change

Change

Revenues

$

444.5

$

444.6

$

(0.1)

—

%

Net income (loss)

$

172.9

$

(56.0)

$

228.9

409

%

Non-GAAP earnings(1)

$

189.1

$

199.2

$

(10.1)

(5)

%

Net income (loss), per basic share

$

4.01

$

(1.25)

$

5.26

421

%

Net income (loss), per diluted share

$

3.98

$

(1.25)

$

5.23

418

%

Non-GAAP earnings, per diluted share(1)

$

4.36

$

4.37

$

(0.01)

—

%

(1)

See definition of non-GAAP earnings, a non-GAAP financial measure, and a reconciliation of net income to non-GAAP earnings below.

Revenues

The following table presents the components of total revenues (dollars in millions):

Three Months Ended
June 30,

Dollar

Percentage

2018

2017

Change

Change

Net product sales:

Remodulin

$

159.5

$

157.7

$

1.8

1

%

Tyvaso

105.9

104.2

1.7

2

%

Adcirca

109.8

120.6

(10.8)

(9)

%

Orenitram

49.5

46.0

3.5

8

%

Unituxin

19.8

16.1

3.7

23

%

Total revenues

$

444.5

$

444.6

$

(0.1)

—

%

Revenues for the three months ended June 30, 2018 decreased by $0.1 million as compared to the same period in 2017. Remodulin net product sales increased by $1.8 million due to a $16.7 million increase in U.S. net product sales partially offset by a $14.9 million decrease in international net product sales. U.S. net product sales increased due to an increase in quantities ordered from our U.S. distributors, which do not precisely reflect underlying patient demand, and a price increase implemented in April 2018, which was the first price increase for Remodulin since 2010. International net product sales decreased primarily due to a reduction in the price at which we sell Remodulin to an international distributor in connection with a transfer of additional regulatory and commercial responsibilities to that distributor in 2017. Tyvaso net product sales increased by $1.7 million primarily due to a price increase. Adcirca net product sales decreased by $10.8 million primarily due to a decrease in the number of bottles sold, partially offset by price increases that were determined by Lilly. Orenitram net product sales increased by $3.5 million primarily due to an increase in the number of patients being treated with Orenitram. Unituxin net product sales increased by $3.7 million primarily due to an increase in the number of vials sold and a price increase implemented in 2017.

Expenses

Cost of product sales. The following table summarizes cost of product sales by major category (dollars in millions):

Three Months Ended
June 30,

Dollar

Percentage

2018

2017

Change

Change

Category:

Cost of product sales

$

61.1

$

19.3

$

41.8

217

%

Share-based compensation expense (benefit)(1)

0.6

(0.4)

1.0

250

%

Total cost of product sales

$

61.7

$

18.9

$

42.8

226

%

(1)

Refer to Share-based compensation expense (benefit) below for discussion.

Cost of product sales, excluding share-based compensation. The increase in cost of product sales of $41.8 million for the three months ended June 30, 2018, as compared to the same period in 2017, was primarily due to a $40.7 million increase in the royalty expense for Adcirca. As a result of an amendment to our license agreement with Lilly, effective December 1, 2017 our royalty rate on net product sales of Adcirca increased from five percent to an effective rate of approximately 42.5 percent.

Research and development expense. The following table summarizes research and development expense by major category (dollars in millions):

Three Months Ended
June 30,

Dollar

Percentage

2018

2017

Change

Change

Category:

Research and development projects

$

79.1

$

61.6

$

17.5

28

%

Share-based compensation expense (benefit)(1)

3.2

(1.8)

5.0

278

%

Total research and development expense

$

82.3

$

59.8

$

22.5

38

%

(1)

Refer to Share-based compensation expense (benefit) below for discussion.

Research and development expense, excluding share-based compensation. The increase in research and development expense of $17.5 million for the three months ended June 30, 2018, as compared to the same period in 2017, was driven by the continued investment in our innovative product pipeline to treat cardiopulmonary diseases and cancer.

Selling, general and administrative expense. The following table summarizes selling, general and administrative expense by major category (dollars in millions):

Three Months Ended
June 30,

Dollar

Percentage

2018

2017

Change

Change

Category:

General and administrative

$

50.8

$

51.6

$

(0.8)

(2)

%

Sales and marketing

15.6

15.5

0.1

1

%

Share-based compensation expense(1)

16.7

0.3

16.4

NM

(2)

Total selling, general and administrative expense

$

83.1

$

67.4

$

15.7

23

%

(1)

Refer to Share-based compensation expense (benefit) below for discussion.

(2)

Calculation is not meaningful.

Share-based compensation expense (benefit). The following table summarizes share-based compensation (benefit) expense by major category (dollars in millions):

Three Months Ended
June 30,

Dollar

Percentage

2018

2017

Change

Change

Category:

Stock options

$

15.5

$

12.2

$

3.3

27

%

Restricted stock units

2.0

0.5

1.5

300

%

Share tracking awards plan (STAP)

2.7

(14.9)

17.6

118

%

Employee stock purchase plan

0.3

0.3

—

—

%

Total share-based compensation expense (benefit)

$

20.5

$

(1.9)

$

22.4

NM

(1)

(1)

Calculation is not meaningful.

The increase in share-based compensation expense of $22.4 million for the three months ended June 30, 2018, as compared to the same period in 2017, was primarily due to: (1) a $17.6 million increase in STAP expense related to an increase in our stock price during the three months ended June 30, 2018, as compared to a decrease in our stock price during the same period in 2017; and (2) a $3.3 million increase in stock option expense due to additional awards granted and outstanding in 2018.

Loss Contingency

In December 2017, we entered into a civil Settlement Agreement with the U.S. Government to resolve a DOJ investigation related to our support of 501(c)(3) organizations that provide financial assistance to patients. During the second quarter of 2017, we recorded a $210.0 million accrual relating to this matter, and ultimately paid this amount, plus interest, to the U.S. Government upon settlement.

Impairment of Investment in a Privately-Held Company

During the quarter ended June 30, 2017, one of our investments in a privately-held company experienced an event triggering an impairment analysis to evaluate the recoverability of our investment. We determined that the fair value of our investment as of June 30, 2017 was lower than its carrying value, resulting in an impairment charge of $46.5 million. As of June 30, 2017, the adjusted carrying value of our investment in this company was $53.5 million. The carrying value of this asset has not been further adjusted since June 30, 2017.

Income Tax Expense

The provision for income taxes was $45.0 million for the three months ended June 30, 2018, as compared to $100.2 million for the same period in 2017. The provision for income taxes is based on an estimated effective tax rate for the entire year. The estimated annual effective tax rate is subject to adjustment in subsequent quarterly periods if components used to estimate the annual effective tax rate are updated or revised. Our effective tax rate (ETR) as of June 30, 2018 and June 30, 2017 was approximately 21 percent and approximately 60 percent, respectively. Our ETR for the six months ended June 30, 2018 decreased as compared to the same period in 2017 due to the impacts of The Tax Cuts and Jobs Act as well as the $210.0 million accrual in connection with the civil settlement described above and the $46.5 million impairment charge described above that did not meet the criteria for tax deductibility at that time.

On August 1, 2018 Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, reported financial results and provided a business update for the second quarter ended June 30, 2018 (Press release, Blueprint Medicines, AUG 1, 2018, View Source [SID1234528283]).

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"In the second quarter, Blueprint Medicines continued to advance a broad portfolio, with progress across multiple programs," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "Importantly, we reported updated data from our Phase 1 EXPLORER trial in patients with advanced systemic mastocytosis that showed profound and durable clinical activity in nearly all patients. These data, combined with previously reported data from our ongoing Phase 1 NAVIGATOR trial in advanced gastrointestinal stromal tumors, reinforce our confidence in avapritinib as a potentially transformative therapy across multiple patient populations. By the end of this year, we expect to have four pivotal clinical trials of avapritinib underway, with the potential to rapidly advance toward approval in defined patient populations."

Clinical Programs:

Avapritinib: Gastrointestinal Stromal Tumors (GIST)

In June 2018, Blueprint Medicines announced the dosing of the first patient in its Phase 3 VOYAGER clinical trial, which will evaluate the safety and efficacy of avapritinib compared to regorafenib in patients with third- or fourth-line advanced GIST.
In June 2018, Blueprint Medicines presented data from a retrospective natural history study of patients with advanced PDGFRα D842V-driven GIST at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The data confirmed that patients with advanced PDGFRα D842V-driven GIST are unlikely to respond to currently available tyrosine kinase inhibitors (TKIs), illustrating the high unmet need for new therapies in this patient population with a short survival rate.
Blueprint Medicines continues to evaluate avapritinib in its Phase 1 NAVIGATOR clinical trial and anticipates presenting updated data across multiple patient populations, including PDGFRA-driven GIST, third-line or later GIST and second-line GIST, in the second half of 2018. Additionally, based on data from this trial, the Company plans to submit a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for avapritinib for the treatment of patients with PDGFRA-driven GIST and fourth-line KIT-driven GIST in the first half of 2019.
Avapritinib: Advanced Systemic Mastocytosis (SM)

In June 2018, Blueprint Medicines presented updated clinical data from its ongoing Phase 1 EXPLORER clinical trial of avapritinib in patients with advanced SM at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper). The data showed an overall response rate of 83 percent and durable ongoing responses up to 22 months. All evaluable patients showed marked decreases on one or more objective measures of mast cell burden, regardless of advanced SM subtype, previous treatment or starting dose level. The data also showed that avapritinib was generally well-tolerated. Most adverse events reported by investigators were Grade 1 or 2, and only three patients discontinued due to a treatment-related adverse event. Read the full data here.
Blueprint Medicines plans to initiate screening of patients for enrollment in PATHFINDER, a registration-enabling, open-label, single-arm Phase 2 clinical trial in patients with advanced SM, in the third quarter of 2018, and plans to initiate PIONEER, a registration-enabling, randomized, placebo-controlled Phase 2 clinical trial in patients with indolent and smoldering SM, by the end of 2018.
BLU-667: RET-Altered Solid Tumors

Blueprint Medicines continues to enroll patients in the expansion portion of its ongoing Phase 1 ARROW clinical trial of BLU-667 at a dose of 400 mg once daily. In the expansion, patients are being enrolled in four defined cohorts: RET-altered non-small cell lung cancer (NSCLC) patients previously treated with a TKI; RET-altered NSCLC patients who have not previously received any TKI treatment; patients with medullary thyroid cancer; and patients with other RET-altered solid tumors.
BLU-554: Hepatocellular Carcinoma (HCC)

In June 2018, Blueprint Medicines announced plans to initiate a proof-of-concept clinical trial with CStone Pharmaceuticals in China to evaluate BLU-554 in combination with CS1001, a clinical-stage anti-programmed death ligand-1 (PD-L1) immunotherapy being developed by CStone Pharmaceuticals, as a first-line treatment for patients with HCC. Additionally, the companies plan to expand Blueprint Medicines’ ongoing Phase 1 clinical trial of BLU-554 as a monotherapy to include new sites in Mainland China. The companies expect to submit an investigational new drug (IND) application for BLU-554 to the Chinese health authorities by the end of 2018, and plan to initiate the clinical trial evaluating BLU-554 in combination with CS1001 and the expansion of Blueprint Medicines’ ongoing clinical trial for BLU-554 as a monotherapy in Mainland China in 2019.
Corporate:

In June 2018, Blueprint Medicines announced an exclusive collaboration and license agreement with CStone Pharmaceuticals to develop and commercialize avapritinib, BLU-554 and BLU-667 in Mainland China, Hong Kong, Macau and Taiwan, either as a monotherapy or as part of a combination therapy. Under the terms of the agreement, Blueprint Medicines received an upfront cash payment of $40.0 million, will be eligible to receive up to approximately $346.0 million in potential milestone payments and tiered percentage royalties in the mid-teens to low twenties on annual net sales of each licensed product in the territory.
Blueprint Medicines recently received a $10.0 million milestone payment from Roche following the achievement of a research milestone.
Second Quarter Financial Results:

Cash Position: As of June 30, 2018, cash, cash equivalents and investments were $616.7 million, as compared to $673.4 million as of December 31, 2017. This decrease was primarily related to cash used in operating activities, partially offset by the $40.0 million upfront payment received in connection with Blueprint Medicines entering into the collaboration with CStone Pharmaceuticals and the $10.0 million milestone payment received from Roche.
Collaboration Revenues: Collaboration revenues were $41.4 million for the second quarter of 2018, as compared to $5.9 million for the second quarter of 2017. This increase was primarily due to revenue recognized under the collaboration agreement with CStone Pharmaceuticals.
R&D Expenses: Research and development expenses were $58.6 million for the second quarter of 2018, as compared to $33.3 million for the second quarter of 2017. This increase was primarily attributable to increased clinical and manufacturing expenses associated with advancing avapritinib, BLU-554 and BLU-667 further through clinical trials and increased personnel-related expenses. Research and development expenses included $4.3 million in stock-based compensation expenses for the second quarter of 2018.
G&A Expenses: General and administrative expenses were $12.3 million for the second quarter of 2018, as compared to $6.8 million for the second quarter of 2017. This increase was primarily attributable to increased personnel-related expenses and increased professional fees, including pre-commercial planning activities. General and administrative expenses included $3.5 million in stock-based compensation expenses for the second quarter of 2018.
Net Loss: Net loss was $27.0 million the second quarter of 2018, or a net loss per share of $0.62, as compared to a net loss of $33.4 million for the second quarter of 2017, or a net loss per share of $0.86.
Financial Guidance:

Based on its current plans, Blueprint Medicines expects that its existing cash, cash equivalents and investments, excluding any potential option fees and milestone payments under its existing collaborations with Roche and CStone Pharmaceuticals, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the second half of 2020.

Conference Call Information:

Blueprint Medicines will host a live conference call and webcast today at 8:30 a.m. ET. The conference call may be accessed by dialing (855) 728-4793 (domestic) or (503) 343-6666 (international) and referring to conference ID 5597837. A webcast of the conference call will be available in the Investors section of Blueprint Medicines’ website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.