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Supernus to Present at the 2018 Cowen Health Care Conference

on March 6 , 2018 – Supernus Pharmaceuticals, Inc. (NASDAQ:SUPN), a specialty pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system diseases, reported that the Company’s management will present an overview and update for the Company, and host investor meetings, at the Cowen 38th Annual Health Care Conference (Press release, Supernus, MAR 6, 2018, View Source [SID1234524467]).

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Date: Tuesday, March 13, 2018
Time: 10:00 a.m. ET
Place: The Boston Marriott Copley Place, Boston, Mass.

Investors interested in arranging a meeting with the Company’s management during this conference should contact the conference coordinator.

A live webcast of the presentation can be accessed by visiting ‘Events & Presentations’ in the Investor Relations Section on the Company’s website at www.supernus.com. An archived replay of these webcasts will be available for 60 days after the conference on the Company’s website.

Rigel Announces Fourth Quarter and Year End 2017 Financial Results and Provides Company Update

on March 6, 2018 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) reported financial results for the fourth quarter and year end 2017 (Press release, Rigel, MAR 6, 2018, View Source;p=RssLanding&cat=news&id=2336583 [SID1234524465]).

Recent Achievements

The U.S. Food and Drug Administration (FDA) is continuing its review of Rigel’s New Drug Application (NDA) for fostamatinib for the treatment of adult patients with chronic immune thrombocytopenia (ITP). The Prescription Drug User Fee Act (PDUFA) action date for the FDA to complete its review of the NDA is April 17, 2018.
The FDA awarded Orphan Drug Designation to fostamatinib for the treatment of warm antibody autoimmune hemolytic anemia (AIHA) on January 31, 2018.
Updated results from Stage 1 of Rigel’s fostamatinib Phase 2 AIHA trial showed an increased clinical response rate after an additional patient met the primary endpoint. This brings the Stage 1 response rate to 53% (9/17) of evaluable patients receiving fostamatinib.
"The milestones achieved by our team in 2017 have set Rigel up to realize our goal of building a commercial-stage company prepared to launch our first medicine," said Raul Rodriguez, president and CEO of Rigel. "We are excited about the potential of fostamatinib as a treatment option for patients with chronic ITP as well as the encouraging preliminary fostamatinib data in patients living with autoimmune hemolytic anemia, a rare disease for which there are no approved therapies."

For the fourth quarter of 2017, Rigel reported a net loss of $25.9 million, or $0.18 per basic and diluted share, compared to a net loss of $15.6 million, or $0.16 per basic and diluted share, in the same period of 2016.

There were no contract revenues from collaborations in the fourth quarter of 2017. Contract revenues from collaborations of $3.0 million in the fourth quarter of 2016 were related to the payment received pursuant to Rigel’s collaboration and license agreement with Bristol-Myers Squibb Company (BMS) for the discovery, development and commercialization of potential immuno-oncology therapeutics.

Rigel reported total costs and expenses of $26.2 million in the fourth quarter of 2017, compared to $18.8 million in the fourth quarter of 2016. The increase in costs and expenses was primarily due to the commercial launch preparation costs incurred for fostamatinib in ITP as well as costs for managing the NDA submission.

For the year ended December 31, 2017, Rigel reported contract revenues from collaborations of $4.5 million and a net loss of $78.0 million, or $0.62 per basic and diluted share, compared to contract revenues from collaborations of $20.4 million and a net loss of $69.2 million, or $0.73 per basic and diluted share, in 2016. Weighted average shares outstanding for the years ended December 31, 2017 and 2016 were 126.3 million and 94.4 million, respectively. Contract revenues from collaborations in 2017 are comprised of the $3.3 million payment Rigel received from BerGenBio AS pursuant to advancing a licensed AXL kinase inhibitor to a Phase 2 clinical study and a $1.2 million payment Rigel earned pursuant to a license agreement with a third party. Contract revenues from collaborations in 2016 were mainly comprised of the $13.4 million amortization of the upfront payment, $3.0 million contingent payment received and $290,000 in research service fees earned from BMS, as well as the $3.7 million contingent payment received from BerGenBio AS.

As of December 31, 2017, Rigel had cash, cash equivalents and short-term investments of $115.8 million, compared to $74.8 million as of December 31, 2016. Rigel expects that its cash, cash equivalents and short-term investments will be sufficient to support its current and projected funding requirements, including the launch of fostamatinib for chronic ITP in the U.S., through the next 12 months.

Corporate Update
Contingent on FDA approval of the NDA for fostamatinib for the treatment of chronic ITP, Rigel is preparing for a product launch in the second quarter of 2018.

Rigel continues to execute on its commercial readiness plan to support this potential launch, including establishing distribution channels with external partners, developing the systems needed to provide medication access, and hiring all key personnel. The last recruiting milestone will be the addition of the sales force pending product approval.

Portfolio Update
TAVALISSE (fostamatinib disodium) in Chronic ITP
Rigel is working with the FDA as it conducts its review of Rigel’s NDA for fostamatinib, an oral spleen tyrosine kinase (SYK) inhibitor, for the treatment of adult patients with chronic ITP.

Fostamatinib in Autoimmune Hemolytic Anemia (AIHA)
Rigel is evaluating the safety and efficacy of fostamatinib in patients with warm antibody AIHA. The Phase 2, open-label, multi-center, Simon two-stage study completed enrollment of Stage 1 in 2017. A clinical response in this trial was defined as achieving a hemoglobin level of greater than 10 g/dl and at least a 2 g/dl increase from baseline.

In February 2018, an additional patient in the Stage 1 extension study met the response criteria. As of February 2018, 53% (9 of 17) of evaluable patients achieved a response to fostamatinib treatment. Six patients achieved a response during the 12-week evaluation period, and an additional three patients met the response criteria in the extension study after 12 weeks of dosing. The safety profile was consistent with the existing fostamatinib safety database. Data from this study will be presented at the Thrombosis and Hemostasis Societies of North America meeting in San Diego, CA on March 8, 2018.

Stage 2 enrollment commenced in late 2017. Stage 2 follows the same protocol as Stage 1 and will include 20 patients. Rigel plans to meet with the FDA in the first half of 2018 to determine the regulatory development pathway of fostamatinib in AIHA.

On January 31, 2018, the FDA granted Orphan Drug designation to fostamatinib for the treatment of patients with AIHA.

Additional Product Development

Rigel completed enrollment of the second cohort in its blinded Phase 2 study of fostamatinib in IgA Nephropathy (IgAN). The study is evaluating the efficacy, safety, and tolerability of fostamatinib as measured by changes in proteinuria, renal function, and histology (comparing the pre- and post-study renal biopsies). The second cohort receives a higher dose of fostamatinib, 150mg BID, while the first cohort received 100mg BID. The primary efficacy endpoint is the mean change in proteinuria from baseline at 24 weeks. Rigel expects to have study results by April 2018.
During 2017, Rigel selected a molecule from its Interleukin-1 receptor-associated kinase (IRAK) program for preclinical development. The molecule was selected for development based on its ability to inhibit both the IRAK 1 and IRAK 4 signaling pathways in preclinical studies, potentially providing a profound clinical benefit in autoimmune and inflammatory diseases such as psoriasis, lupus, gout, psoriatic arthritis and multiple sclerosis. The Company expects to initiate clinical trials in mid-2018.
About ITP
In patients with ITP, the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPOs) and splenectomy. However, not all patients are adequately treated with existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA
Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder in which the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 40,000 adult patients in the US and can be a severe, debilitating anemia. To date, there are no disease-targeted therapies approved for AIHA, despite the tremendous medical need that exists for these patients.

Conference Call and Webcast Today at 5:00PM Eastern Time
Rigel will hold a live conference call and webcast today at 5:00pm Eastern Time (2:00pm Pacific Time).

Participants can access the live conference call by dialing (855) 892-1489 (domestic) or (720) 634-2939 (international) and using the Conference ID number 7289803. The conference call will also be webcast live and can be accessed from Rigel’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

Puma Biotechnology to Present at Cowen’s Annual Healthcare Conference

on March 6, 2018 -Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that Alan H. Auerbach, Chairman, Chief Executive Officer, President and Founder of Puma, will provide an overview of the Company at 11:20 a.m. EDT on Tuesday, March 13, at the Cowen and Company 38th Annual Health Care Conference (Press release, Puma Biotechnology, MAR 6, 2018, http://investor.pumabiotechnology.com/press-release/puma-biotechnology-present-cowens-annual-healthcare-conference [SID1234524464]). The conference will be held at the Boston Marriott Copley Place.

A live webcast of the presentation will be available on the Company’s website at www.pumabiotechnology.com . The presentation will be archived on the website and available for 30 days.

PTC Therapeutics Reports Fourth Quarter and Full Year 2017 Financial Results and Provides Corporate Update

On March 6, 2018 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the fourth quarter and full year ended December 31, 2017 (Press release, PTC Therapeutics, MAR 6, 2018, View Source [SID1234524463]).

"Our mission for the past 20 years has been to develop and bring treatments to patients with rare genetic disorders," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "We are thrilled with the progress we made in 2017. We now have two approved commercial products for patients living with Duchenne muscular dystrophy and have built a global commercial platform. We are also excited with the progress in our splicing platform including the rapidly advancing SMA program. Our commercial success and our developing pipeline positions PTC as a fast-growing, global rare disorder company."

Fourth Quarter and Full Year 2017 Financial Highlights:

Total revenues for the fourth quarter of 2017 were $78.0 million compared to $25.2 million in the fourth quarter of 2016. For the full year 2017, total revenues were $194.4 million compared to $82.7 million in 2016. The change in total revenue was a result of the expanded commercial launch of Translarna, the successful U.S. Emflaza launch and a $20 million milestone payment achieved from Roche for the initiation of the pivotal portion of the SMA clinical trial.
Translarna net product sales were $41.0 million for the fourth quarter of 2017, representing 63% growth over $25.1 million reported in the fourth quarter of 2016. For the full year 2017, Translarna generated $145.2 million in net product sales, representing 78% growth compared to $81.4 million in 2016.
Emflaza net product sales were $17.0 million for the fourth quarter of 2017 and $28.8 million for the full year 2017.
GAAP R&D expenses were $29.2 million for the fourth quarter of 2017 compared to $26.0 million for the same period in 2016. For the full year 2017, GAAP R&D expenses were $117.5 million compared to $117.6 million in 2016. The increase in GAAP R&D expense for the fourth quarter of 2017 as compared to the prior year period was due to increased clinical activities and regulatory spend.
Non-GAAP R&D expenses were $25.7 million for the fourth quarter of 2017, excluding $3.5 million in non-cash, stock-based compensation expense, compared to $21.9 million for the same period in 2016, excluding $4.1 million in non-cash, stock-based compensation expense. For the full year 2017, non-GAAP R&D expenses were $102.0 million, excluding $15.5 million in non-cash, stock-based compensation expense, compared to $100.0 million for 2016, excluding $16.8 million in non-cash, stock-based compensation expense and $0.8 million in one-time restructuring expense.
GAAP SG&A expenses were $35.5 million for the fourth quarter of 2017 compared to $24.2 million for the same period in 2016. For the full year 2017, GAAP SG&A expenses were $121.3 million compared to $97.1 million in 2016. The increase in SG&A expenses for the fourth quarter and year ended December 31, 2017, as compared to the prior year periods, was primarily due to the continued commercial support for the Emflaza launch.
Non-GAAP SG&A expenses were $32.5 million for the fourth quarter of 2017, excluding $3.0 million in non-cash, stock-based compensation expense, compared to $19.9 million for the same period in 2016, excluding $4.3 million in non-cash, stock-based compensation expense. For the full year 2017, non-GAAP SG&A expenses were $106.2 million, excluding $15.1 million in non-cash, stock-based compensation expense, compared to $77.3 million for 2016, excluding $18.2 million in non-cash, stock-based compensation expense and $1.6 million in one-time restructuring expense.
Net interest expense for the fourth quarter of 2017 was $3.4 million compared to net interest expense of $2.1 million in the same period in 2016. For the full year 2017, net interest expense was $12.1 million compared to net interest expense of $8.3 million in 2016. The increase in net interest expense for the fourth quarter and year ended December 31, 2017, as compared to the prior year periods, is primarily a result of increased interest expense related to the $40 million secured loan facility which we closed during the second quarter of 2017 partially offset by interest income from investments.
Net income for the fourth quarter of 2017 was $1.3 million compared to a net loss of $26.8 million for the same period in 2016. Net loss for the full year 2017 was $79.0 million, compared to $142.1 million for the same period in 2016.
Cash, cash equivalents, and marketable securities totaled approximately $191.2 million at December 31, 2017 compared to $231.7 million at December 31, 2016.
Shares issued and outstanding as of December 31, 2017, were 41.6 million.
2018 Guidance:

Full year 2018 net product revenues to be between $260 and $295 million. PTC anticipates Translarna net product revenue for the full year 2018 to be between $170 and $185 million. PTC projects a 5-year (December 31, 2022) compound annual growth rate of 15% for net product revenues representing continued strong growth year-over-year by increasing penetration in current countries and pursuing opportunities for label expansion. PTC anticipates Emflaza net product revenue for the full year 2018 to be between $90 and $110 million.
GAAP R&D and SG&A expense for the full year 2018 to be between $280 and $290 million.
Non-GAAP R&D and SG&A expense for the full year 2018 to be between $250 and $260 million, excluding estimated non-cash, stock-based compensation expense of approximately $30 million.
Key Fourth Quarter and Full Year 2017 Corporate Highlights:

Strong commercial execution has led to robust year over year revenue growth. In 2017, our Duchenne muscular dystrophy franchise generated $174 million dollars.
Translarna reported revenue of approximately $145 million dollars, a 78% increase over the prior year. The demand for Translarna continues to increase in established regions such as Western Europe, LATAM, and most recently in the Middle East and Central and Eastern Europe. PTC plans for continued growth for Translarna by increasing penetration in current countries and pursuing opportunities for label expansion. In January, we guided to a 5-year, 15% CAGR through year end of 2022.
Since launching in May, Emflaza reported revenue of approximately $29 million in 2017. The reception of Emflaza by both patients and healthcare providers has been very strong. We are focused on enabling all eligible patients to have access to this therapy. In line with PTC’s mission, PTC has established programs with the goal of ensuring that all eligible patients will have access to Emflaza regardless of financial or insurance status.
Data demonstrating that Emflaza is a differentiated product over prednisone were published in top-tier peer-reviewed journal and in recently published Duchenne treatment guidelines. These data indicate that Emflaza delays the loss of major milestones by 2 to 3 years compared to prednisone. Based on the understanding of both our own internally generated results and independently published data, we believe that Emflaza should be the standard of care for all Duchenne muscular dystrophy patients.
Regulatory update for Translarna in the US. The Office of New Drugs of the U.S. Food and Drug Administration has reiterated the FDA’s prior position and denied PTC’s appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren. In its response, the Office of New Drugs recommended a possible path forward for the ataluren NDA submission based on the accelerated approval pathway. This would involve a re-submission of an NDA containing the current data on effectiveness of ataluren with new data to be generated on dystrophin production. We intend to follow the FDA’s recommendation and will collect such dystrophin data using newer technologies via procedures and methods that will be mutually agreeable to us and the FDA. The response also stated that Study 041, which is currently enrolling, could serve as the confirmatory post-approval trial required in connection with the accelerated approval framework.
SMA program advancing with two registration-directed trials. The SUNFISH trial in the spinal muscular atrophy (SMA) program transitioned to the pivotal portion which triggered a $20M milestone payment from Roche in the fourth quarter of 2017. A dose has been selected in the FIREFISH trial and it is anticipated to transition to the pivotal stage in the coming weeks. A recent presentation at the International Scientific Congress on SMA in Krakow reviewed the ongoing, dose-finding Part 1 of FIREFISH in the Type 1 SMA infants highlighting survival data, as well as safety and interim clinical data. No patients had discontinued due to adverse events. Early interim clinical data reported no patient lost the ability to swallow and no patient has required tracheostomy or reached permanent ventilation. The program also includes an additional study, JEWELFISH, for patients who have previously received splicing therapies. The SMA program is a joint collaboration with Roche and the SMA Foundation.
PTC to provide details of its developing R&D pipeline at its Analyst Day, April 17th. PTC will outline its plans for the continued sustainable growth of its internally developed programs.

Protalix BioTherapeutics Reports 2017 Full Year Results and Provides Corporate Update

On march 6, 2018 Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx, reported its financial results for the full-year ended December 31, 2017 and provided a corporate update (Press release, Protalix, MAR 6, 2018, View Source;p=RssLanding&cat=news&id=2336416 [SID1234524462]).

"This has been an exciting year with a great partnership deal and good progress in all of our clinical programs," said Moshe Manor, Protalix’s President and Chief Executive Officer. "Looking ahead to 2018, we expect to generate and release more data regarding our clinical trials, and to continue the progress of our programs, both with potential partners and with our current partner, Chiesi Farmaceutici."

2017 and Recent Clinical and Corporate Highlights

Pegunigalsidase (PRX-102) for Fabry Disease

The Company has over 40 clinical trial sites actively recruiting patients for three trials, a significant portion of which are located in the United States. The trials are being run by leading opinion leaders in the Fabry disease field.

The Company expects to finalize enrollment in all three Fabry trials during 2018.

Pegunigalsidase alfa granted Fast Track designation from the U.S. Food and Drug Administration; the designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

Pegunigalsidase alfa granted Orphan Drug Designation by the European Commission which qualifies the Company for access to the centralized marketing authorization procedure, including applications for inspections and for protocol assistance. The designation was based on the Company having established medically plausible evidence that pegunigalsidase alfa will provide a significant benefit over existing treatments in the European Union for the treatment of Fabry disease.
Alidornase (PRX-110) for Cystic Fibrosis

The Cystic Fibrosis Foundation Grant Review is in the final stages of completion.
Oral antiTNF (OPRX-106) for Ulcerative Colitis

The Company expects to release top-line data this month, with full data planned to be presented in a medical conference later in the year.

Interim data was positive, with initial signs of efficacy across multiple clinically meaningful endpoints.
Alfataliglicerase for Gaucher Disease

The Company recognized alfataliglicerase sales of $7.1 million in Brazil for 2017. In addition, the Company shipped approximately $2.6 million worth of the drug during the current quarter.
Full-Year 2017 Financial Results

The Company recorded total revenues of $19.2 million during the year ended December 31, 2017, compared to $9.2 million for the same period of 2016. The increase resulted from an increase equal to $3.0 million of products sold to Brazil, and $7.0 million of drug substance sold to Pfizer Inc.

Research and development expenses for the year ended December 31, 2017, were $28.8 million, compared to $24.6 million for the same period in 2016. Selling, general and administrative expenses for the year ended December 31, 2017 were $11.5 million, compared to $9.4 million incurred during the same period in 2016.

Operating loss for the year ended December 31, 2017 was $36.4 million compared to $33.2 million for the year ended December 31, 2016.

For the year ended December 31, 2017, the Company reported a net loss of $47.2 million, excluding a one-time, non-cash net charge of $38.1 million in connection with the remeasurement of a derivative, or $0.36 per share, basic and diluted, compared to a net loss of $29.4 million, or $0.29 per share, basic and diluted, for the same period of 2016.

On December 31, 2017, the Company had $51.2 million of cash and cash equivalents, compared to $63.3 million at December 31, 2016, which is currently projected to fund operations into 2020. As of December 31, 2017, the Company had outstanding $5.9 million of its 4.5% convertible notes due September 2018 and $59.1 million of its 7.5% senior secured convertible notes due November 2021.
Conference Call and Webcast Information

The Company will host a conference call on Tuesday, March 6, 2018, at 8:30 am ET to review the clinical, corporate and financial highlights.

To participate in the conference call, please dial the following numbers prior to the start of the call: United States: +1-844-358-6760; International: +1-478-219-0004. Conference ID number 2636229.

The conference call will also be broadcast live and available for replay for two weeks on the Company’s website, www.protalix.com, in the Events Calendar of the Investors section. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software