On October 27, 2016 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported it will present two new studies at the American Society of Dermatopathology (ASDP) annual meeting being held Oct. 27-30, 2016 in Chicago, Ill (Press release, Myriad Genetics, OCT 27, 2016, View Source [SID1234516051]). The research being presented validates the accuracy of Myriad myPath Melanoma in differentiating benign skin nevi from malignant melanoma.

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"We are presenting landmark data from the largest outcomes-based study ever performed with a melanoma diagnostic," said Loren Clarke, M.D., medical director, Dermatology, Myriad Genetic Laboratories. "The myPath Melanoma test performed very well and identified patients with melanoma versus benign skin lesions with greater than 95 percent diagnostic accuracy, which is exceptional in molecular diagnostics for cancer, particularly given the extreme heterogeneity of melanoma."

"Pigmented or suspect skin lesions are difficult to diagnose in approximately 15 percent of cases," said Sancy Leachman, M.D., Ph.D., chair of the Department of Dermatology in the Oregon Health & Science University School of Medicine and director of the Melanoma Research Program at the Knight Cancer Institute. "A highly accurate biomarker like the myPath Melanoma test should help dermatologists augment their diagnosis of melanoma, improve patient care and lower healthcare costs."

Below are the featured presentations at ASDP (#ASDP2016).

Poster Presentation
Title: Diagnostic Distinction of Malignant Melanoma and Benign Nevi by a Gene Expression Signature and Correlation to Clinical Outcome​.
Presenter: Jennifer Ko.
Date: Friday, Oct. 28, 2016: 4:15 — 5:00 p.m. and Saturday, Oct. 29, 2016 10:00 — 10:45 a.m. CT.

In this study, research collaborators from the Cleveland Clinic, Stanford University and Nottingham University assessed the clinical accuracy (sensitivity and specificity) of the myPath Melanoma test against clinical outcomes in 182 patients with skin lesions (99 melanomas and 83 nevi) with more than 5 years of follow up. The results show that the myPath Melanoma test accurately differentiated benign lesions from melanoma with a sensitivity of 93.8 percent and a specificity of 96.2 percent when compared to known clinical outcomes. The diagnostic accuracy of the myPath Melanoma test was high even in a subset of difficult-to-diagnose cases and, in combination with two previous validation studies, the findings support its use as an adjunct method for the early and accurate diagnosis of melanoma.

Podium Presentation
Title: Gene Expression Signature as an Ancillary Method in the Diagnosis of Desmoplastic Melanoma​.
Presenter: Loren Clarke.
Date: Sunday, Oct. 30, 2016: 8:20 — 8:30 a.m. CT.

The objective of this study was to assess the accuracy of the myPath Melanoma test in the differentiation of desmoplastic melanoma (DM) from benign skin lesions. These lesions represent approximately one percent of melanomas, but are known to be very difficult to diagnose. The analysis included samples from 20 patients with DM and 27 from patients with benign moles (nevi). The results showed that the myPath Melanoma test was positive in 15 of the 20 known melanomas, negative in four and indeterminate in one. The myPath score was negative in 24 of the benign nevi and indeterminate in three. Based on these findings, the myPath Melanoma test demonstrated approximately 80 percent diagnostic accuracy in this very difficult-to-diagnose subtype.

For more information about the meeting, please visit the ASDP website at View Source Follow Myriad on Twitter via @MyriadGenetics to stay informed about news and updates from the Company.

About Melanoma
Melanoma is one of the fastest growing cancers in the United States and can strike people of all ages, races and skin types. With a one-in-50 lifetime risk of developing melanoma, nearly 76,000 Americans are expected to be diagnosed with Stage I-IV melanoma and another 68,000 will be diagnosed with melanoma in situ — totaling approximately 144,000 total diagnoses. Early and accurate diagnosis of melanoma is critical for long-term survival. For more information visit: www.mypathmelanoma.com/ and www.myriadpro.com/melanoma.

About Myriad myPath Melanoma
Myriad myPath Melanoma is a clinically validated test to be used as an adjunct to histopathology when the distinction between a benign nevus and a malignant melanoma cannot be made confidently by histopathology alone. The test measures the expression of 23 genes and accurately distinguishes melanoma from benign nevi.

On October 27, 2016 following the recent update on clinicaltrials.gov, AstraZeneca reported that the US FDA has placed a partial clinical hold on the enrolment of new patients with head and neck squamous cell carcinoma (HNSCC) in clinical trials of durvalumab as monotherapy and in combination with tremelimumab or other potential medicines (Press release, AstraZeneca, OCT 27, 2016, View Source [SID1234516048]). All trials are continuing with existing patients.

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The partial clinical hold on new patient enrolment relates only to head and neck cancer. Trials for durvalumab in different cancer types, as monotherapy or in combination with tremelimumab or other potential medicines, are progressing as planned, with pivotal data in lung cancer anticipated in the first half of 2017.

The FDA’s decision follows voluntary action by AstraZeneca to pause enrolment of new HNSCC patients while a detailed analysis is conducted of adverse events related to bleeding that were observed as part of routine safety monitoring of the Phase III KESTREL and EAGLE trials. Bleeding is a known complication in treatments of head and neck cancers primarily due to the nature of the underlying disease, the proximity of tumours to major blood vessels and use of prior cancer therapies, which may involve surgery and radiation.

AstraZeneca has submitted its analysis of the observed bleeding events to the FDA for review and is working closely with the Agency, providing the required information to resume new patient enrolment as soon as possible.

On October 27, 2016 TG Therapeutics, Inc. (NASDAQ:TGTX) reported the publication of preclinical data describing the synergy of the Company’s next generation, once daily, PI3K-delta inhibitor, TGR-1202, with the proteasome inhibitor carfilzomib and the unique effects of the combination to silence c-Myc in various preclinical lymphoma and myeloma models (Press release, TG Therapeutics, OCT 27, 2016, View Source [SID1234516047]). In addition, the manuscript also, for the first time, reports on TGR-1202’s unique complimentary mechanism of inhibiting the protein kinase casein kinase-1 (CK1) epsilon, which may contribute to the silencing of c-Myc and explain TGR-1202’s clinical activity in aggressive lymphoma, including Diffuse Large B-cell Lymphoma (DLBCL). The preclinical data are described further in the manuscript titled, "Silencing c-Myc Translation as a Therapeutic Strategy through Targeting PI3K Delta and CK1 Epsilon in Hematological Malignancies," which was published online yesterday in the First Edition section of Blood, the Journal of the American Society of Hematology (ASH) (Free ASH Whitepaper). The online version of the article can be accessed at www.bloodjournal.org.

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"We want to thank Dr. Deng, Dr. O’Connor, and the team from Columbia Presbyterian Medical Center and the Center for Lymphoid Malignancies for their exhaustive and comprehensive interrogation of TGR-1202 and the elucidation of this novel complimentary mechanism. For quite some time we have been presenting the differentiated safety profile observed with TGR-1202 and the differentiated chemical structure compared to other PI3K-delta inhibitors. This preclinical work demonstrates that TGR-1202 not only potently targets PI3K-delta but in addition uniquely targets a relatively novel kinase, CK1-epsilon, which perhaps offers another rationale for the differentiated activity and safety effects we have seen in patients. We look forward to exploring this exciting concept further in the recently launched clinical trial," stated Michael S. Weiss, the Company’s Executive Chairman and Interim Chief Executive Officer.

"The data in this paper clearly demonstrates that TGR-1202 and carfilzomib in combination is markedly synergistic and selectively silenced c-Myc compared to combinations with idelalisib and bortezomib. In addition, we were excited to identify and elucidate the previously unknown mechanism of TGR-1202 and its effect on CK1 epsilon which was not exhibited by either idelalisib or duvelisib based on a kinome profiling platform analyzed. We believe this research may help explain in part the preliminary activity demonstrated by TGR-1202 in DLBCL. Given TGR-1202’s distinct safety profile as a single agent and its uniquely demonstrated ability to be used in combination with other agents, we look forward to bringing this novel combination to the clinic in our recently announced Phase 1 study of TGR-1202 and carfilzomib in patients with lymphoma," stated Dr. Owen A. O’Connor, Professor of Medicine and Experimental Therapeutics, Director Lymphoid Malignancies at Columbia Presbyterian Medical Center.

Based on this extensive preclinical work, the Company recently announced the launch of a Phase 1/2 study to evaluate the safety and efficacy of TGR-1202 in combination with carfilzomib, in patients with relapsed or refractory lymphoma, particularly c-Myc driven lymphomas which are aggressive in nature. This study is currently open to enrollment at the Center for Lymphoid Malignancies, Columbia Presbyterian Medical Center, New York, NY. More information on this clinical study can be found at www.clinicaltrials.gov.

On October 27, 2016 Swedish Orphan Biovitrum AB (publ) (Sobi) reported its results for the third quarter 2016 (Press release, Swedish Orphan Biovitrum, OCT 27, 2016, View Source;Media/News/RSS/?RSS=View Source [SID1234516046]). Revenue for the quarter totalled SEK 1,171 M (786), an increase of 49 per cent compared to previous year. Based on strong performance across the portfolio and an earlier launch for Alprolix, the company has raised guidance for the full year.

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Business highlights Q3 2016

Elocta reimbursed in the UK, Italy, France and Spain
Alprolix reimbursed in the UK
Long term Elocta and Alprolix data presented at WHF 2016 World Congress
Orfadin capsule filing validated by Health Canada
Milan Zdravkovic appointed as SVP, Head of R&D
Financial highlights Q3 2016 (Q3 2015)

Total revenue of SEK 1,171 M (786), an increase of 49 per cent
Product revenue of SEK 1,009 M (645), an increase of 56 per cent (57 per cent at CER)
Gross margin of 67 per cent (62)
EBITA of SEK 282 M (97)
Ended the quarter with a cash position of SEK 824 M
Earnings per share 0.53 SEK (0.02)
"Results in the third quarter had a positive contribution from the ongoing launch of Elocta, from an earlier than anticipated launch for Alprolix and from Kineret. We continue to lay the foundation for a sustainable haemophilia business in Europe with both Elocta and Alprolix gaining several important reimbursement approvals in major markets", said Geoffrey McDonough, CEO and President at Sobi.

"A significant milestone after the quarter was the orphan designation approval by the European Commission for our development candidate SOBI003 – a chemically modified human recombinant sulfamidase for the treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome). MPS IIIA is a severe and debilitating disease with devastating consequences for patients, and there is presently no treatment available."

Financial Summary
Q3 Q3 Jan-Sep Jan-Sep Full year
Amounts in SEK M 2016 2015 Change 2016 2015 Change 2015
Total revenues1 1 171 786 49% 3 913 2 414 62% 3 228
Gross profit 782 486 61% 2 791 1 486 88% 2 007
Gross margin 67% 62% 71% 62% 62%
EBITA 282 97 >100% 1 334 343 >100% 433
EBIT (Operating profit/loss) 171 25 >100% 1 034 129 >100% 146
Profit/loss for the period 143 5 >100% 710 77 >100% 68
1Jan-Sep 2016 revenues include a one-time credit in Q1 of SEK 322 M relating to the first commercial sales of Elocta, and a one-time credit in Q2 of SEK 386 M relating to first commercial sales of Alprolix.
Outlook 2016 – guidance raised

For the full-year 2016, Sobi now expects revenues of SEK 5,125—5,200 M (4,800-5,000). Revenues include one-time credits for Elocta of SEK 322 M and for Alprolix of SEK 386 M which do not impact cash. Gross margin is now expected to be 70 per cent (68-70) and EBITA for the full-year in the range of SEK 1,475–1,525 M (1,200-1,300).

The original outlook was published 29 February 2016.

—

Sobi’s report for the third quarter 2016 can be found on View Source;Media/Financial-Reports/

On October 27, 2016 Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage developer of innovative RNA-targeted therapeutics, reported financial results for the three and nine months ended September 30, 2016 (Press release, Sarepta Therapeutics
, OCT 27, 2016, View Source;p=RssLanding&cat=news&id=2216471 [SID1234516045]).

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"We are thrilled that the first patient has been infused with EXONDYS 51. We are pleased with the early stages of the launch and that multiple insurance carriers are providing coverage for patients to have access to EXONDYS 51. We plan on providing a corporate update at the 35th Annual JP Morgan Healthcare Conference, after EXONDYS 51 has been available for a full quarter," said Edward Kaye, Sarepta’s chief executive officer.

"We believe our recent financing puts us in a strong financial position to execute on both our internal and external clinical development programs and global manufacturing development plans. We continue to evaluate the sale of the priority review voucher as a potential source of non-dilutive financing to help support these efforts and advance potential therapies for patients with DMD," said Sandy Mahatme, Sarepta’s chief financial officer.

Financial Results
For the third quarter of 2016, Sarepta reported a net loss of $56.7 million, or $1.18 per share, compared to a net loss of $51.9 million for the third quarter of 2015, or $1.25 per share. The incremental loss of $4.8 million was primarily the result of increased expenses related to the launch of EXONDYS 51.

Excluding $10.8 million of stock-based compensation expense and restructuring expenses, non-GAAP net loss for the third quarter of 2016 was $45.9 million, or $0.95 per share, compared to a non-GAAP net loss excluding $5.7 million of stock-based compensation expense of $46.3 million for the third quarter of 2015, or $1.11 per share.

No revenue was recognized for the three months ended September 30, 2016 and 2015.

Research and development expenses were $34.3 million for the third quarter of 2016, compared to $36.7 million for the third quarter of 2015, a decrease of $2.4 million. Non-GAAP research and development expenses (excluding $3.4 million of stock-based compensation and restructuring expenses) were $30.9 million for the third quarter of 2016, compared to $34.0 million (excluding $2.6 million of stock-based compensation expense) for the third quarter of 2015, a decrease of $3.1 million.

General and administrative expenses were $22.2 million for the third quarter of 2016, compared to $15.1 million for the third quarter of 2015, an increase of $7.1 million. Non-GAAP general and administrative expenses (excluding $7.4 million of stock-based compensation and restructuring expenses) were $14.8 million for the third quarter of 2016, compared to $12.0 million (excluding $3.1 million of stock-based compensation expense) for the third quarter of 2015, an increase of $2.8 million.

The Company had $406.6 million in cash, cash equivalents, short-term investments and restricted cash as of September 30, 2016 compared to $204.0 million as of December 31, 2015, an increase of $202.6 million. The increase was driven by the net proceeds received from the Company’s public offerings in June and September 2016, offset by the use of cash to fund the Company’s ongoing operations.

Use of Non-GAAP Measures
In addition to the GAAP financial measures set forth in this press release, the Company has included certain non-GAAP measurements: non-GAAP research and development expenses, non-GAAP general and administrative expenses, non-GAAP operating expense adjustments, non-GAAP net loss, and non-GAAP basic and diluted net loss per share, which present operating results on a basis adjusted for stock-based compensation and restructuring expenses.

Stock-based compensation expenses represent non-cash charges related to equity awards granted by Sarepta. Although these are recurring charges to operations, management believes the measurement of these amounts can vary substantially from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within management’s control. Therefore, management believes that excluding these charges from non-GAAP research and development expenses, non-GAAP general and administrative expenses, non-GAAP net loss and non-GAAP net loss per share facilitates comparisons of the Company’s operational performance in different periods.

Restructuring related expenses have been excluded from non-GAAP research and development expenses, non-GAAP general and administrative expenses, non-GAAP net loss and non-GAAP net loss per share as the Company believes that the adjustments for these items represent more closely the sustainability of the Company’s operating performance and understanding of its financial results.

The Company uses these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. The Company also believes these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating the Company’s performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of the Company’s financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP general and administrative expenses, non-GAAP operating expense adjustments, non-GAAP net loss, and non-GAAP basic and diluted net loss per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table "Reconciliation of GAAP to Non-GAAP Net Loss."

Recent Corporate Developments

Duchenne Muscular Dystrophy Program
–Sarepta Therapeutics Announces FDA Accelerated Approval of EXONDYS 51 (eteplirsen) injection, an Exon Skipping Therapy to Treat Duchenne Muscular Dystrophy (DMD) Patients Amenable to Skipping Exon 51
–Sarepta Therapeutics and Summit Enter Into Exclusive License and Collaboration Agreement for European Rights to Summit’s Utrophin Modulator Pipeline for the Treatment of Duchenne Muscular Dystrophy
–Catabasis Pharmaceuticals and Sarepta Therapeutics Announce a Joint Research Collaboration in Duchenne Muscular Dystrophy
–Sarepta Therapeutics Announces First Patient Dosed in Phase III Clinical Trial of SRP-4045 and SRP-4053 for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
–Sarepta Therapeutics Announces Favorable USPTO Decisions in Exon 51 and Exon 53 Composition of Matter Patent Interference Cases against BioMarin Pharmaceutical
Corporate Updates
–Sarepta Therapeutics Announces Pricing of $345 Million Public Offering of Common Stock

Conference Call
The Company will be hosting a conference call at 8:00 a.m. EDT, to discuss these financial results and other corporate updates. The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 7029987. Please specify to the operator that you would like to join the "Sarepta Third Quarter 2016 Earnings Call." The conference call will be webcast live under the investor relations section of Sarepta’s website at www.sarepta.com and will be archived there following the call for 90 days. Please connect to Sarepta’s website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

About EXONDYS 51
EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. Data from clinical studies of EXONDYS 51 in a small number of DMD patients have demonstrated a consistent safety and tolerability profile. The pivotal trials were not designed to evaluate long-term safety and a clinical benefit of EXONDYS 51 has not been established.

Important Safety Information
Adverse reactions observed in patients (N=8) treated with 30 or 50 mg/kg/wk of EXONDYS 51 with incidence ≥ 25% and higher than in the placebo group (N=4) (Study 1) were: balance disorder (38%), vomiting (38%) and contact dermatitis (25%). The most common adverse reactions were balance disorder and vomiting.

The following events were reported in ≥ 10% of patients treated with EXONDYS 51 for up to 208 weeks (N=88) and occurred more frequently than placebo in a controlled trial for 24 weeks (Study 1): vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.

There have been reports of transient erythema, facial flushing, and elevated temperature occurring on the day of EXONDYS 51 infusion.

For the full prescribing information please refer to U.S. Full Prescribing Information at www.EXONDYS51.com.