Tetraphase Pharmaceuticals Reports Fourth Quarter and Full-Year 2017 Financial Results, Highlights Achievements and Key 2018 Milestones

on march 6, 2018 Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH), a biopharmaceutical company focused on developing and commercializing novel antibiotics to treat life-threatening multidrug-resistant (MDR) infections,reported financial results for the fourth quarter and year ended December 31, 2017, provided an overview of recent achievements, and highlighted key milestones for 2018 (Press release, Tetraphase, MAR 6, 2018, View Source [SID1234524468]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"2017 was a year of tremendous progress for Tetraphase, as we successfully completed IGNITE4, our second positive phase 3 clinical trial evaluating IV eravacycline in complicated intra-abdominal infections (cIAI), and filed regulatory approval applications in the U.S. and in Europe for cIAI," said Guy Macdonald, President and Chief Executive Officer of Tetraphase. "Although we were very disappointed with the recently announced topline results from our IGNITE3 trial in complicated urinary tract infections (cUTI), in which eravacycline did not achieve statistical non-inferiority to ertapenem, we are excited to move forward with bringing eravacycline to market for the treatment of cIAI. We recently announced that the FDA has accepted our NDA submission for review and our PDUFA date is August 28, 2018."

Key Milestones for 2018

Potential approval of eravacycline in cIAI in US – 2H 2018

Potential approval of eravacycline in cIAI in Europe – 2H2018

Potential commercial launch of eravacycline in cIAI in the US – Q4 2018

Complete phase 1 MAD studies for TP-271 and TP-6076 – 2H 2018

Fourth Quarter and Recent Highlights

Entered into an exclusive licensing agreement with Everest Medicines Limited, a biopharmaceutical company based in China, to develop and commercialize eravacycline for the treatment of cIAI and other indications in mainland China, Taiwan, Hong Kong, Macau, South Korea, and Singapore.

Submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for IV eravacycline for the treatment of cIAI. Notified of a Prescription User Fee Act (PDUFA) goal date of August 28, 2018 for the FDA’s completion of its review. The NDA submission includes data from the IGNITE1 and IGNITE4 phase 3 clinical trials, in which twice-daily IV eravacycline was well tolerated and achieved high clinical cure rates in patients with cIAI. Both studies demonstrated statistical non-inferiority of eravacycline to two widely used comparators – ertapenem in IGNITE1 and meropenem in IGNITE4 – for the primary efficacy endpoint of clinical response at the test-of-cure (TOC) visit.

Announced top-line results from IGNITE3, the Company’s phase 3 clinical trial evaluating the efficacy and safety of once-daily intravenous (IV) eravacycline compared to ertapenem for the treatment of patients with cUTI, in which eravacycline did not achieve statistical non-inferiority to ertapenem. The Company is fully analyzing the data to understand the outcome and will provide an update when more information is available. Based on the results of the study, the Company does not plan to further evaluate eravacycline in cUTI and has ceased its development of an oral formulation of eravacycline for the treatment of cUTI.

Appointed Larry Tsai, M.D., Chief Medical Officer. Dr. Tsai joined Tetraphase in 2014 as senior medical director and became vice president, clinical development in 2015. He has nearly 20 years of experience in clinical practice and development. Dr. Tsai has provided significant expertise to the clinical team throughout the IGNITE phase 3 program for eravacycline, as well as in the early clinical development of the Company’s pipeline programs.

Appointed Larry Edwards, Chief Operating Officer. Mr. Edwards joined Tetraphase in July 2015 as vice president, marketing, served as vice president, commercial operations from January 2016 to December 2016 and as senior vice president, chief commercial officer from December 2016 to February 2017. He has over 20 years of experience in the infectious disease area. Mr. Edwards is responsible for all phases of the launch of eravacycline, assuming regulatory approval.
Fourth Quarter and Full-Year 2017 Financial Results
As of December 31, 2017, Tetraphase had cash and cash equivalents of $136.4 million and 51.4 million shares outstanding. The Company expects that its cash and cash equivalents, as well as expected revenue from its U.S. government awards, will be sufficient to fund operations through the first half of 2019.

For the fourth quarter of 2017, Tetraphase reported a net loss of $23.5 million, or $0.46 per share, compared to a net loss of $22.5 million, or $0.61 per share, for the same period in 2016. Revenues were $2.5 million compared to $1.1 million for the same period in 2016. Revenues for each period consisted of contract and grant revenue under the Company’s U.S. government awards for the development of Tetraphase compounds for the treatment of diseases caused by bacterial biothreat pathogens and for certain infections caused by life-threatening multidrug-resistant bacteria. The increase in revenue was mainly due to scope and timing of activities related to our Biomedical Advanced Research and Development Authority (BARDA) Contract conducted during the fourth quarter of 2017. Research and development (R&D) expenses for the fourth quarter of 2017 were $18.5 million compared to $19.3 million for the same period in 2016. The decrease in R&D expenses was primarily due to lower manufacturing costs in 2017 compared to the same period in 2016, offset, in part, by higher clinical costs related to our IGNITE3 clinical trial. General and administrative (G&A) expenses for the fourth quarter of 2017 were $7.9 million compared to $4.3 million for the same period in 2016. The increase in G&A was primarily due to pre-launch commercial investments, business development expenses and headcount related costs.

For the year ended December 31, 2017, Tetraphase reported a net loss of $114.8 million, or $2.63 per share, compared to a net loss of $77.5 million, or $2.11 per share, for the same period in 2016. Revenues were $9.7 million for the year ended December 31, 2017 compared to $5.1 million for the same period in 2016. As stated above, revenues for each period consisted of contract and grant revenue under the Company’s U.S. government awards for the development of Tetraphase compounds for the treatment of diseases caused by bacterial biothreat pathogens and for certain infections caused by life-threatening multidrug-resistant bacteria. This increase in 2017 was due to changes in the timing and scope of activities under the subcontract with respect to the BARDA and National Institute of Allergy and Infectious Diseases (NIAID) contracts and the addition of amounts received under the Combating Antibiotic Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) award. R&D expenses were $101.7 million for the year ended December 31, 2017 compared to $63.8 million for the same period in 2016. The increase was mainly due to costs associated with conducting our IGNITE3 and IGNITE4 phase 3 clinical trials and an increase in regulatory costs associated with eravacycline related to our marketing authorization application (MAA) and NDA filing activities. G&A expenses were $23.7 million for the year ended December 31, 2017 compared to $19.2 million for the same period in 2016. This increase was primarily due to pre-launch commercial investments, business development and legal expenses and headcount related costs.

About Eravacycline
Eravacycline is a novel, fully-synthetic fluorocycline antibiotic being developed for the treatment of cIAI and other serious infections, including those caused by MDR pathogens that have been highlighted as urgent public health threats by both the World Health Organization and the U.S. Centers for Disease Control & Prevention (CDC). In clinical trials, eravacycline has demonstrated potent activity against multidrug-resistant (MDR) pathogens, including carbapenem-resistant enterobacteriaceae (CRE), Acinetobacter baumannii, and colistin-resistant bacteria carrying the mcr-1 gene.

Eravacycline was investigated for the treatment of cIAI as part of the Company’s IGNITE (Investigating Gram-negative Infections Treated with Eravacycline) phase 3 programs. In IGNITE1, a pivotal phase 3 trial in patients with cIAI, twice-daily IV eravacycline met the primary endpoint by demonstrating statistical non-inferiority of clinical response compared to ertapenem, was well tolerated, and achieved high cure rates in patients with Gram-negative pathogens, including resistant isolates. The IGNITE1 data is serving as the basis of the Marketing Authorization Application (MAA) for IV eravacycline for the treatment of patients with cIAI now under review by the European Medicines Agency (EMA). In IGNITE4, a second phase 3 clinical trial in patients with cIAI, twice-daily IV eravacycline met the primary endpoint by demonstrating statistical non-inferiority of clinical response compared to meropenem, was well tolerated, and achieved high cure rates. The Company has used the results from IGNITE1 and IGNITE4 to support a New Drug Application (NDA) submission for IV eravacycline in cIAI. To date, eravacycline has been administered to over 2,700 patients.

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.