Jounce Therapeutics to Present at Deutsche Bank 43rd Annual Health Care Conference

On May 1, 2018 Jounce Therapeutics, Inc. (NASDAQ:JNCE), a clinical stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers for patient enrichment, reported that Richard Murray, Ph.D., chief executive officer and president of Jounce, will present at the Deutsche Bank 43rd Annual Health Care Conference on Tuesday, May 8, 2018 at 1:30 PM ET in Boston, MA (Press release, Jounce Therapeutics, MAY 1, 2018, View Source;p=RssLanding&cat=news&id=2345890 [SID1234525886]).

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A live webcast of the presentation will be available by visiting "Events and Presentations" in the Investors and Media section of Jounce’s website at www.jouncetx.com. A replay of the webcast will be archived for 30 days following the presentation.

Intellia Therapeutics Announces

First Quarter 2018 Financial Results

On May 1, 2018 Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology, announced financial results and operational progress for the first quarter of 2018 (Press release, Intellia Therapeutics, MAY 1, 2018, View Source [SID1234525885]).

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John Leonard, M.D., was appointed Intellia’s President and Chief Executive Officer in the first quarter of 2018, and one of his first initiatives was to broaden the Company’s strategy. "We are building the premier CRISPR-based genome editing company with leading in vivo and ex vivo capabilities," said Dr. Leonard. "We are very pleased with the scientific data generated from our in vivo non-human primate (NHP) studies, and the progress with our modular, scalable lipid nanoparticle (LNP) delivery system has allowed us to target a timeframe for our first Investigational New Drug (IND) submission. As we continue to execute on our full spectrum of in vivo and ex vivo genome editing platforms, we will share progress on our differentiated, wholly owned ex vivo approach, starting this month at the American Society of Gene and Cell Therapy Annual Meeting."

The Company announced today that it anticipates submitting an IND application for its lead indication, transthyretin amyloidosis (ATTR), by the end of 2019 and confirms plans to initiate IND-enabling studies in mid-2018. Over the past six months, ongoing NHP studies have demonstrated well-tolerated editing to therapeutically relevant levels of transthyretin (TTR) protein reduction (60 to 80 percent) after a single systemic administration via LNP delivery to NHP hepatocytes. Rates of editing were durable over the six-month period without re-dosing the animals. In support of the proposed IND submission, Intellia has narrowed the field of potential guides to its current development candidate for early human trials. The guide-optimization process used high-throughput screening to evaluate the entire TTR gene for those guides with high levels of activity and undetectable off-target cutting. The Company has completed studies to understand potential dosing regimens and is continuing studies on durability of the effect, both of which may expedite Phase I clinical trials. Intellia has also developed an enhanced LNP formulation through optimization campaigns that is currently being tested for multiple follow-on liver indications, and anticipates that this modular approach may minimize development timelines for each additional and subsequent liver-targeted product candidate.

Intellia has also demonstrated continued progression of its modular liver platform capability to knockout various targets of interest in the livers of mice, including SERPINA1 for alpha-1 antitrypsin deficiency (AATD) and HAO1 for primary hyperoxaluria type 1 (PH1), each of which has resulted in protein expression reductions believed to be therapeutically relevant. This initial knockout edit in AATD lays the groundwork for developing an approach that restores production of the missing protein in AATD, required for the amelioration of the disease.

The table below shows editing rates and corresponding protein reductions in the livers of mice for ATTR, AATD and PH1. ATTR and AATD both produce aberrant proteins hence treatment of these conditions requires reductions in the level of the disease-causing proteins. PH1 results from the low level activity of a particular protein for which treatment requires reducing the levels of substrate for that defective protein to metabolize, achieved by knocking out the gene that encodes HAO1. In each of these three cases, Intellia’s modular LNP delivery system achieved high levels of reduction of the targeted protein. These initial editing rates and corresponding protein reductions are evidence of Intellia’s ability to successfully target monogenic liver diseases by knocking out harmful genetic mutations.

Beyond the liver, the Company continues to advance its application of CRISPR/Cas9 technology to the central nervous system (CNS), including through its collaboration with Beverly Davidson, Ph.D., of the Children’s Hospital of Philadelphia, who will share updated LNP delivery data in a presentation at the American Society of Gene and Cell Therapy Annual Meeting later this month.

In ex vivo applications, Intellia seeks to develop allogeneic cellular therapies, which are cells derived from unmatched tissue donors, which are modified outside of the human body to allow them to be administered to an unrelated patient. This endeavor is supported through multiple efforts, including recently acquired access to intellectual property from researchers at the Karolinska Institutet and Intellia’s collaboration with Ospedale San Raffaele, announced in June of 2017.

In February of 2018, Cell Reports published Intellia’s first peer-reviewed paper entitled "A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing." This landmark paper documented Intellia’s delivery of Cas9 mRNA and single guide RNA using its proprietary LNPs to achieve a 97 percent reduction in mouse TTR protein levels in the liver, which was sustained for at least 12 months.

During the course of 2018, Intellia plans to share additional pre-clinical data on its TTR genome editing program, including the achievement of a near ten-fold reduction in the required dose, derived via improvements in potency, as well as other knockout targets and data on delivery via LNPs to the CNS of NHPs. Additionally, Intellia plans to share pre-clinical data on both immuno-oncology and autoimmune disease targets in 2018.

First Quarter 2018 Financial Results

Collaboration Revenue

Collaboration revenue was $7.5 million for the first quarter of 2018, compared to $6.2 million during the first quarter of 2017. The increase in collaboration revenue in 2018 was primarily driven by amounts recognized under Intellia’s collaboration agreement with Regeneron.

Since inception through March 31, 2018, the Company has received $112.1 million in funding from the collaborations with Novartis and Regeneron, excluding amounts received for equity investments, and had an accounts receivable balance of $7.5 million at March 31, 2018.

Operating Expenses

Research and development expenses increased by $9.1 million to $22.5 million during the first quarter of 2018, compared to $13.4 million during the first quarter of 2017. This increase was driven primarily by the advancement of Intellia’s research programs, research personnel growth to support these programs, as well as the expansion of the development organization, and includes laboratory supplies and research materials such as reagents.

General and administrative expenses increased by $1.7 million to $7.4 million during the first quarter of 2018, compared to $5.7 million during the first quarter of 2017. This increase was driven primarily by increased salary and related headcount-based expenses to support Intellia’s larger research and development organization, public company compliance, and administrative obligations.

The Company’s net loss was $21.4 million for the first quarter of 2018, compared to $12.6 million during the first quarter of 2017.

Balance Sheet

Cash and cash equivalents at March 31, 2018, were $327.8 million, compared to $340.7 million at December 31, 2017.

Financial Guidance

The Company’s primary uses of capital will continue to be for research and development programs, laboratory and related supplies, compensation costs for current and future employees, consulting, legal and other regulatory expenses, patent prosecution filing and maintenance costs for Intellia’s licensed intellectual property, and general overhead costs.

As of March 31, 2018, the Company had an accumulated deficit of $137.0 million. The Company expects losses to increase as it continues to incur significant research and development expenses related to the advancement of Intellia’s therapeutic programs and ongoing operations. Based on Intellia’s research and development plans and expectations related to the progress of the Company’s programs, the Company expects that the cash and cash equivalents as of March 31, 2018, as well as technology access and research funding from Novartis and Regeneron, will enable Intellia to fund operating expenses and capital expenditures through mid-2020, excluding any potential milestone payments or extension fees that could be earned and distributed under the collaboration agreements with Novartis and Regeneron or any strategic use of capital not currently in the base-case planning assumptions.

Insmed to Present at the Deutsche Bank 43rd Annual Health Care Conference

On May 1, 2018 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company focused on the unmet needs of patients with rare diseases, reported that Will Lewis, President and Chief Executive Officer of Insmed, will present at the Deutsche Bank 43rd Annual Health Care Conference in Boston, MA on Tuesday, May 8, 2018 at 4:10 p.m. EDT (Press release, Insmed, MAY 1, 2018, View Source [SID1234525884]).

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The presentation will be webcast live and can be accessed by visiting the investor relations section of the company’s website at www.insmed.com. The webcast will be archived for a period of 90 days following the conclusion of the live event.

Horizon Pharma plc to Participate in the UBS Global Healthcare Conference

On May 1, 2018 Horizon Pharma plc (NASDAQ:HZNP), reported that the company will participate in the following conference in May (Press release, Horizon Pharma, MAY 1, 2018, View Source [SID1234525883]):

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UBS Global Healthcare Conference

Date: May 22, 2018
Presentation Time: 9:30 a.m. ET
Location: New York
The presentation will be webcast live and may be accessed by visiting Horizon’s website at View Source A replay of the webcast will be available for the even

Incyte Reports 2018 First-Quarter Financial Results and Updates on Key Clinical Programs

On May 1, 2018 Incyte Corporation (Nasdaq:INCY) reports 2018 first-quarter financial results, highlighting strong growth in total product-related revenue and providing a status update on the Company’s development portfolio (Press release, Incyte, MAY 1, 2018, View Source [SID1234525882]).

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"Jakafi continues to grow with significant momentum as we bring the benefits of this first-in-class treatment to an increasing number of patients," stated Hervé Hoppenot, Incyte’s Chief Executive Officer. "We expect to be able to provide important updates from our development portfolio over the coming months—including the results of the first pivotal trial of Jakafi in graft-versus-host disease and initial data from our FGFR program in cholangiocarcinoma—as we continue to work on developing innovative therapies for patients in need."

Portfolio Update

Oncology – key highlights

Results from the REACH1 trial evaluating ruxolitinib in patients with steroid-refractory acute graft-versus-host disease (GVHD) are expected in the first half of 2018. Data emerging from this open-label, pivotal trial continue to support Incyte’s intention to submit an sNDA in the second half of 2018, seeking approval of ruxolitinib in this indication.

Initial data from the trial evaluating INCB54828 in patients with cholangiocarcinoma are expected in second half of 2018.

As previously announced, the external Data Monitoring Committee (eDMC) review of the pivotal Phase 3 ECHO-301 study evaluating epacadostat in combination pembrolizumab in patients with unresectable or metastatic melanoma determined that the study did not meet the primary endpoint of improving progression-free survival in the overall population compared to pembrolizumab monotherapy. The study’s second primary endpoint of overall survival also was not expected to reach statistical significance. Based on these results, and at the recommendation of the eDMC, the study has been stopped to enable patients and their physicians to consider alternative therapeutic options, and Incyte is also significantly downsizing the epacadostat development program.

In consultation with Incyte’s collaboration partners, and after the results of ECHO-301, the two pivotal trials of epacadostat in combination with pembrolizumab in lung cancer (ECHO-305 and ECHO-306) will be converted into randomized phase 2 trials. Enrollment will be discontinued in the four additional pivotal trials of epacadostat in combination with pembrolizumab, and in the two pivotal trials of epacadostat in combination with nivolumab; each of these studies will be amended to enable patients and their physicians to consider alternative therapeutic options. The pivotal trial in combination with durvalumab in Stage 3 lung cancer will not be initiated.

Incyte intends to continue to investigate epacadostat’s potential as a component of combination immunotherapy in proof-of-concept trials, which will include hypotheses distinct from combinations with PD-1 and PD-L1 antagonists.

Partnered – key highlights

In April 2018, the US. Food and Drug Administration (FDA) convened its Arthritis Advisory Committee to discuss the resubmission of the baricitinib NDA, which recommended approval of the 2mg dose of baricitinib as a once-daily oral medication for the treatment of moderately-to-severely active rheumatoid arthritis for adult patients who have had an inadequate response or intolerance to methotrexate. While the Advisory Committee unanimously supported the efficacy of the 4mg dose of baricitinib, it did not recommend approval of the 4mg dose of baricitinib for the proposed indication based on the adequacy of the safety and benefit-risk profiles. The FDA action date for baricitinib is in June 2018.

Corporate Update

In April 2018, Maria E. Pasquale joined the Incyte Executive Management team as Executive Vice President and General Counsel. Maria joined Incyte from Celgene Corporation, where for 17 years she held positions of increasing responsibility including Chief Counsel and Senior Vice President, Legal & Deputy General Counsel, where she led the legal department through Celgene’s global expansion. Most recently, Maria served as Celgene’s Executive Vice President and Global Chief Compliance Officer, responsible for GxP and healthcare compliance globally.

2018 First-Quarter Financial Results

The financial measures presented in this press release for the three months ended March 31, 2018 and 2017 have been prepared by the Company in accordance with U.S. Generally Accepted Accounting Principles ("GAAP"), unless otherwise identified as a Non-GAAP financial measure. Management believes that Non-GAAP information is useful for investors, when considered in conjunction with Incyte’s GAAP disclosures. Management uses such information internally and externally for establishing budgets, operating goals and financial planning purposes. These metrics are also used to manage the Company’s business and monitor performance. The Company adjusts, where appropriate, for both revenues and expenses in order to reflect the Company’s core operations. The Company believes these adjustments are useful to investors by providing an enhanced understanding of the financial performance of the Company’s core operations. The metrics have been adopted to align the Company with disclosures provided by industry peers. Reconciliations of GAAP net loss to Non-GAAP net income (loss) for the three months ended March 31, 2018 and 2017 have been included at the end of this press release.

Guidance related to research and development and selling, general and administrative expenses does not include estimates associated with any potential future strategic transactions.

Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used in conjunction with and to supplement Incyte’s operating results as reported under GAAP. Non-GAAP measures may be defined and calculated differently by other companies in our industry.

Revenues For the quarter ended March 31, 2018, GAAP net product revenues of Jakafi were $314 million as compared to $251 million for the same period in 2017, representing 25 percent growth. For the three months ended March 31, 2018, GAAP net product revenues of Iclusig were $21 million as compared to $14 million for the same period in 2017.

For the quarter ended March 31, 2018, GAAP product royalties from sales of Jakavi, which has been out-licensed to Novartis outside of the United States, was $41 million, as compared to $29 million for the same period in 2017. For the quarter ended March 31, 2018, GAAP product royalties from sales of Olumiant outside of the United States from Lilly were $6 million, as compared to less than $1 million for the same period in 2017.

For the quarter ended March 31, 2018, GAAP milestone revenues were $0 million, as compared to $90 million for the same period in 2017. GAAP milestone revenues in 2017 related to milestones earned from our collaborative partners.

For the quarter ended March 31, 2018, total GAAP revenues were $382 million as compared to $384 million for the same period in 2017. Total Non-GAAP revenues for the quarter ended March 31, 2018 were $382 million as compared to $294 million for the same period in 2017.

Cost of product revenues GAAP cost of product revenues for the quarter ended March 31, 2018 was $18 million, as compared to $15 million for the same period in 2017. Non-GAAP cost of product revenues for the quarter ended March 31, 2018 were $13 million, as compared to $9 million for the same period in 2017. Non-GAAP cost of product revenues exclude the amortization of licensed intellectual property for Iclusig relating to the acquisition of the European business of ARIAD Pharmaceuticals, Inc.

Research and development expenses GAAP research and development expenses for the quarter ended March 31, 2018 were $303 million as compared to $408 million for the same period in 2017. Decreased GAAP research and development expenses were driven primarily by upfront and milestone expenses of $209 million related to our collaborative agreements recorded in the quarter ended March 31, 2017 partially offset by an overall increase in development costs to advance our clinical pipeline. For the quarter ended March 31, 2018, GAAP research and development expenses included $12 million related to our collaboration agreement with Syros Pharmaceuticals, Inc. and $291 million of ongoing expenses.

Non-GAAP research and development expenses for the quarter ended March 31, 2018 were $266 million, as compared to $177 million for the same period in 2017. Non-GAAP research and development expenses exclude the cost of stock-based compensation of $24 million and $21 million for the quarters ended March 31, 2018 and 2017, respectively, and upfront consideration and milestones paid to our collaborative partners of $12 million and $209 million for the quarters ended March 31, 2018 and 2017, respectively.

Selling, general and administrative expenses GAAP selling, general and administrative expenses for the quarter ended March 31, 2018 was $121 million, as compared to $87 million for the same period in 2017. Increased GAAP selling, general and administrative expenses were driven by additional costs related to the commercialization of Jakafi.

Non-GAAP selling, general and administrative expenses for the quarter ended March 31, 2018 was $109 million, as compared to $78 million for the same period in 2017. Non-GAAP selling, general and administrative expenses exclude the cost of stock-based compensation.

Change in fair value of acquisition-related contingent consideration GAAP change in fair value of acquisition-related contingent consideration for the quarters ended March 31, 2018 and 2017 was $7 million.

Unrealized gain (loss) on long term investments GAAP unrealized gain on long term investments for the quarter ended March 31, 2018 was $23 million as compared to an unrealized loss of $6 million for the same period in 2017. The unrealized gain on long term investments for the quarter ended March 31, 2018 represents the fair market value adjustments of the Company’s investments in Agenus, Calithera, Merus, and Syros.

Expense related to senior note conversions GAAP expense related to senior note conversions for the quarter ended March 31, 2017 was $54 million related to the conversions of certain of our 2018 and 2020 convertible senior notes.

Net income (loss) GAAP net loss for the quarter ended March 31, 2018 was $41 million, or $0.19 per basic and diluted share, as compared to a net loss of $187 million, or $0.96 per basic and diluted share for the same period in 2017. Non-GAAP net loss for the quarter ended March 31, 2018 was $3 million, as compared to net income of $29 million for the same period in 2017. Non-GAAP net loss per share for the quarter ended March 31, 2018 was $0.01 per basic and diluted share, as compared to Non-GAAP net income per share of $0.15 per basic and $0.14 per diluted share for the same period in 2017.

Cash, cash equivalents and marketable securities position As of March 31, 2018 and December 31, 2017, cash, cash equivalents and marketable securities totaled $1.2 billion.

Future Non-GAAP financial measures may also exclude upfront and ongoing milestones relating to third-party collaboration partners, impairment of goodwill or other assets, changes in the fair value of equity investments in our collaboration partners, non-cash interest expense related to the amortization of the initial discount on our 2018 and 2020 Senior Notes and the impact on our tax provision of discrete changes in our valuation allowance position on deferred tax assets.