On March 2, 2020 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported its fourth quarter financial results for 2019 (Press release, Intrexon, MAR 2, 2020, View Source [SID1234555062]).

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Recent Business Highlights:

The Company announced the appointment of Helen Sabzevari, PhD as President and CEO of Precigen and Randal J. Kirk as Executive Chairman effective as of January 1, 2020. The Company also changed its name to Precigen, Inc. from Intrexon Corporation and its Nasdaq stock symbol to PGEN from XON effective February 1, 2020;

The Company closed sales of a number of its assets for an aggregate of $65.2 million and sold $35 million of its common stock in January 2020, thereby alleviating the going concern qualification associated with its 2019 consolidated financial statements as well as further streamlining corporate focus;

The Company sold its ownership stake in AquaBounty Technologies, Inc. (Nasdaq: AQB) in October 2019 for proceeds of $21.6 million;

Precigen announced that the US Food and Drug Administration (FDA) granted orphan drug designation (ODD) to PRGN-3006, a first-in-class investigational therapy using Precigen’s non-viral UltraCAR-T therapeutic platform for patients with relapsed or refractory acute myeloid leukemia (AML) and higher risk myelodysplastic syndromes (MDS) (clinical trial identifier: NCT03927261);

Precigen ActoBio, Inc., a wholly-owned subsidiary of Precigen, collaboration partner Oragenics, Inc. (NYSE American: OGEN) completed enrollment in the Phase 2 trial of AG013, an easy to use oral rinsing system designed to prevent and treat oral mucositis; and

Triple-Gene LLC, a majority-owned subsidiary of Precigen, completed the Phase 1 trial enrollment and reported preliminary data of its investigational multigenic gene therapy INXN-4001 for the treatment of heart failure.

Fourth Quarter 2019 Financial Highlights:

Total revenues from continuing operations of $17.0 million;

Net loss of $169.2 million attributable to Precigen, or $(1.09) per basic share, of which $95.7 million was from discontinued operations and an additional $33.8 million was for non-cash charges related to continuing operations; and

Cash, cash equivalents, and short-term investments for continuing operations totaled $75.1 million at December 31, 2019.

Full Year 2019 Financial Highlights:

Total revenues from continuing operations of $90.7 million;

Net loss of $322.3 million attributable to Precigen, or $(2.09) per basic share, of which $116.2 million was from discontinued operations and an additional $70.4 million was for non-cash charges related to continuing operations.

"I am confident that we will make important advances this year in our mission to improve patient care through innovative gene and cell therapies," said Dr. Sabzevari. "We enter 2020 with cash resources that we believe are sufficient for us to deliver on several value-creating milestones during the year across our clinical pipeline. At the same time, we are laser-focused on aligning our portfolio, streamlining operations and maximizing organizational structures to improve operational efficiency going forward."

Fourth Quarter 2019 Financial Results Compared to Prior Year Period

Total revenues decreased $24.2 million from the quarter ended December 31, 2018. Collaboration and licensing revenues decreased $24.6 million, or 103%, from the quarter ended December 31, 2018 primarily due to the reacquisition of rights previously licensed to some of Precigen’s collaborators in the second half of 2018 and the result of which eliminated or substantially reduced revenues previously generated from those collaborations. Additionally, collaboration and licensing revenues from collaborations with other collaborators decreased due to lower demand for research and development services in the current year period.

Research and development expenses decreased $252.2 million, or 92%. The 2018 amounts include a $228.0 million expense related to in-process research and development reacquired from former collaborators. Selling, general and administrative (SG&A) expenses increased $6.3 million, or 28% which was primarily attributable to increased compensation expenses related to performance and retention incentives for SG&A employees, partially offset by (i) decreased share-based compensation expense which arose primarily from the departure of former employees during the first half of the current year; and (ii) fewer legal fees associated with the Company’s Trans Ova subsidiary. The Company also recorded a $29.6 million goodwill impairment charge in the fourth quarter of 2019 related to its Trans Ova subsidiary.

Full Year 2019 Financial Results Compared to Prior Year Period

Total revenues decreased $60.5 million from the year ended December 31, 2018. Collaboration and licensing revenues decreased $55.5 million, or 80%, from the year ended December 31, 2018 primarily due to the reacquisition of rights previously licensed to some of Precigen’s collaborators in the second half of 2018 and the result of which eliminated or substantially reduced revenues previously generated from those collaborations. Additionally, in 2018, the Company recognized additional revenues from the acceleration of previously deferred revenue upon mutual termination of certain collaborations. Product revenues decreased $4.7 million, or 17%, primarily due to lower customer demand in the beef and dairy industries resulting in fewer sales of pregnant cows and calf products. Gross margin on products also declined in the current period as a result of fewer products sold.

Research and development expenses decreased $264.4 million, or 72%. The 2018 amounts include $236.7 million of expenses related to in-process research and development reacquired from former collaborators. SG&A expenses decreased $24.9 million, or 20%. SG&A salaries, benefits, and other personnel costs decreased $14.9 million primarily due to decreased share-based compensation expense as a result of the reversal of previously recognized expense for unvested options granted to former employees as well as the conclusion of the vesting period for other previously granted stock options. Legal and professional fees decreased $6.1 million primarily due to fewer legal fees associated with the Company’s Trans Ova subsidiary. The Company also recorded a $29.6 million goodwill impairment charge in the fourth quarter of 2019 related to its Trans Ova subsidiary.

Conference Call and Webcast

Precigen will host a conference call today Monday March 2nd at 5:30 PM ET to discuss the results and provide a general business update. The conference call may be accessed by dialing 1-888-317-6003 (Domestic US), 1-866-284-3684 (Canada), and 1-412-317-6061 (International) and providing the number 4230814 to join the Precigen Conference Call. Participants may also access the live webcast through Precigen’s website in the Events section at View Source

Precigen: Advancing Medicine with Precision

Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. For more information about Precigen, visit www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

Trademarks

Precigen, UltraCAR-T, and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners.

On March 2, 2020 LabCorp (NYSE: LH) reported that members of the executive management team will participate in a fireside chat at the Barclays Global Healthcare Conference in Miami Beach on Tuesday, March 10 at 8:30 a.m. ET (Press release, LabCorp, MAR 2, 2020, View Source [SID1234555061]).

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A live audio webcast of the presentation will be available via the Company website at www.LabCorp.com and archived for replay.

On March 2, 2020 Ziopharm Oncology, Inc. ("Ziopharm" or the "Company") (Nasdaq: ZIOP), reported its financial results for the fourth quarter and year ended December 31, 2019 and provided a corporate update (Press release, Ziopharm, MAR 2, 2020, View Source [SID1234555060]).

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"In the 16 months since forging our corporate independence, we have advanced all our clinical programs, licensed critical intellectual property, recruited key personnel to the Company and Board, and fortified our financial position," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer. "In 2020, we will use this foundation to accelerate our commercial pathway, through enrollment at NCI to TCR-T trial, preparing TCR-T trials with MD Anderson Cancer Center, data readouts on Controlled IL-12 in recurrent GBM and enrollment to trial of CAR-T infused day after gene transfer."

Significant 2019 Achievements

NCI Phase 2 TCR Study. Under the T-cell receptor (TCR) T-cell therapy program, an investigational new drug (IND) application submitted by the National Cancer Institute (NCI) received clearance from the U.S. Food and Drug Administration (FDA) for a phase 2 clinical trial in multiple solid tumors to evaluate use of the Sleeping Beauty platform for TCR-T therapy.

This trial represents a first-in-human non-viral TCR-T trial at NCI

NCI has begun to screen patients for enrollment with patients going through the TCR identification and procurement process; with tumor resection, neoantigen identification, and TCRs made ready for infusion

Study protocol details are available on Clinicaltrials.gov: A Phase 2 Study Using the Administration of Autologous T-Cells Engineered Using the Sleeping Beauty Transposon/Transposase System to Express T-Cell Receptors Reactive Against Mutated Neoantigens in Patients With Metastatic Cancer (NCT0402436)

New MD Anderson Agreement. In October, the Company entered into a lease agreement to expand its R&D footprint and entered into a new Research and Development Agreement with MD Anderson Cancer Center which:

Enables Ziopharm-sponsored clinical studies at MD Anderson based on the Sleeping Beauty non-viral gene transfer platform to generate T cells targeting neoantigens in solid tumors; initially, two approaches will be evaluated infusing autologous T cells expressing

TCRs with specificities for multiple private neoantigens and administering autologous T cells expressing TCRs from a library targeting shared neoantigens in hotspots

Accelerates expansion of the library, licensed from NCI earlier in 2019, of TCRs against neoantigens in hotspots including mutated KRAS, TP53 and EGFR

Phase 2 Combination Study for Controlled IL-12. Under the Controlled IL-12 program, the Company initiated a phase 2 combination trial with Regeneron’s Libtayo to treat patients with recurrent glioblastoma (rGBM).

As prescribed in the study protocol, the Company expects to enroll up to 36 patients at approximately 10 sites. Enrollment is anticipated to be completed in 1H 2020.

This phase 2 trial builds on experience from a phase 1 combination study with another PD-1 inhibitor, OPDIVO, which completed enrollment last year, with additional data expected this year.

Controlled IL-12 Clinical Data. Data publications in 2019 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and Society for Neuro-Oncology (SNO) annual meetings showed Controlled IL-12, as monotherapy or in combination with a PD-1 inhibitor, resulted in immune-mediated anti-tumor effects in the setting of recurrent GBM. Encouraging results from the phase 1 monotherapy main trial were published in Science Translational Medicine. These publications and presentations are available on Ziopharm’s website.

IND Cleared for CAR-T Study. Under the CAR-T program, an IND was cleared by the FDA for a phase 1 clinical trial to assess CD19-specific CAR-T, produced using Rapid Personalized Manufacturing (RPM).

Up to 24 patients will be enrolled to evaluate infusion of donor-derived RPM CAR-T in patients with CD19+ leukemias and lymphomas who have relapsed after allogeneic bone marrow transplant. This study is being conducted at MD Anderson Cancer Center as an investigator-initiated trial.

The Company also expects partners at Eden BioCell to file an IND for an autologous RPM CD19 trial this year in Taiwan.

Expanded Team and Capabilities. The Company experienced significant growth in 2019, with strategic hires expanding capabilities and increasing staff nearly 50 percent to 73 employees at year end.

Notably, Ziopharm welcomed Sath Shukla, formerly of Vertex Pharmaceuticals, as Chief Financial Officer and Dr. Drew Deniger, who previously worked under Dr. Steven Rosenberg at the NCI, to lead the TCR program.

Chris Bowden, M.D., and Heidi Hagen were appointed as Directors, completing the repopulation of the Board as part of Ziopharm’s reemergence as an independent company.

Strengthened Balance Sheet. Ziopharm completed 2019 with approximately $79.7 million in cash, with another $20.3 million in capital pre-funded at MD Anderson available for the Company’s program.

Subsequent to the close of 2019, the Company raised approximately an additional $98 million through recent financing activities, Ziopharm has dramatically extended its funding horizon and can accelerate the buildout of its TCR program in Houston and the launch of Ziopharm-led TCR clinical trials for patients with solid tumors.

Anticipated Milestones for the First Half of 2020

Sleeping Beauty Cell Therapy Programs

Patient dosing in the NCI-led phase 2 TCR-T trial targeting solid tumors

Initiation of the CD19-specific CAR-T RPM phase 1 trial with membrane bound IL-15 at MD Anderson

Controlled IL-12 Gene Therapy Program

Complete enrollment and initial data readout for phase 2 combination trial with Libtayo

Interim data readout of phase 1 combination trial with OPDIVO

Interim data readout from phase 1 monotherapy trial in expanded cohort

Fourth Quarter 2019 Financial Results

Net loss of the fourth quarter of 2019, was $15.7 million, or $(0.09) per share, compared to net income of $194.5 million, or $1.29 per share, for the fourth quarter of 2018. Net income for the fourth quarter of 2018 reflects the forfeiture and return of all the Company’s Series 1 preferred stock held by a former corporate partner and the relinquishment of Ziopharm’s obligations under a separate agreement, accounting for approximately $207 million.

Research and development expenses were $10.2 million for the fourth quarter of 2019, compared to $8.2 million for the fourth quarter of 2018, primarily reflecting increased clinical trial activity.

General and administrative expenses were $5.8 million for the fourth quarter of 2019, compared to $4.6 million for the fourth quarter of 2018. The increase in general and administrative expenses for the fourth quarter of 2019 is primarily due to increased headcount, growth of intellectual property activity and expanded clinical activity.

The Company ended the fourth quarter 2019 with unrestricted cash resources of approximately $79.7 million.

In addition, a prepayment of approximately $20.3 million remains for work to be conducted by the Company at MD Anderson under the Company’s Research and Development Agreements.

Full Year 2019 Financial Results

Net loss applicable to the common shareholders for the year ended December 31, 2019 was $117.8 million, or $(0.70) per share, basic and diluted, compared to net income applicable to the common shareholders of $137.2 million, or $0.96 per share, basic and diluted, for the year ended December 31, 2018. Net income in 2018 reflects the forfeiture and return of all of the Company’s Series 1 preferred stock held by a former corporate partner and the relinquishment of Ziopharm’s obligations under a separate agreement, accounting for approximately $207 million.

Research and development expenses were $38.3 million for the year ended December 31, 2019, compared to $34.1 million for the year ended December 31, 2018. The increase in research and development expenses for the year ended December 31, 2019 is primarily due to expanded clinical trial activity.

General and administrative expenses were $19.5 million for the year ended December 31, 2019, compared to $19.9 million for the year ended December 31, 2018.

Conference Call and Webcast

Scheduled for today at 4:30 p.m. ET, the conference call can be accessed by dialing 1-844-309-0618 (U.S. and Canada) or 1-661-378-9465 (international). The passcode for the conference call is 6773016. To access the live webcast or the subsequent archived recording, visit the "Investors" section of the Ziopharm website at

www.ziopharm.com. The webcast will be recorded and available for replay on the Company’s website for two weeks.

On March 2, 2020 Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported financial results for the year ended December 31, 2019 (Press release, Zymeworks, MAR 2, 2020, View Source [SID1234555059]).

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"We’ve started the year off strong thanks to significant clinical progress in the second half of 2019 and a recent financing that has provided expanded resources to further advance global development of our clinical candidates, ZW25 and ZW49," said Ali Tehrani, Ph.D., Zymeworks’ President & CEO. "We plan to initiate registration-enabling studies for ZW25 in both biliary tract and gastric cancer, as well as explore additional indications, with a vision toward establishing ZW25 as the new foundational HER2 therapy. We also remain confident in the potential of ZW49 to be transformative in refractory and low HER2-expressing cancers and expect to begin expansion cohorts later this year."

2019 Business Highlights and Recent Developments

Strengthened Balance Sheet

In January 2020, Zymeworks completed an upsized US$320.8 million public financing to accelerate and expand global development of its lead clinical candidates, ZW25 and ZW49, and support further advancement of its novel preclinical programs.

Robust ZW25 Clinical Data and Initiation of Two Phase 2 Clinical Trials Pave the Way for Upcoming Registration-Enabling Studies

ZW25 has been well tolerated and has demonstrated promising anti-tumor activity both as a single agent and in combination with chemotherapy, supporting the planned initiation of two registration-enabling trials in refractory HER2-positive biliary tract cancer and first-line HER2-positive gastroesophageal adenocarcinomas. In addition, Zymeworks recently initiated a Phase 2 trial and collaboration with Pfizer to evaluate ZW25 in combination with palbociclib and fulvestrant in HER2-positive, hormone receptor-positive breast cancer.

ZW49 Advancing Phase 1 Dose-Escalation and Interim Clinical Update

Zymeworks’ second product candidate, ZW49, began a Phase 1 clinical trial to evaluate safety and anti-tumor activity, and to establish a recommended dose and schedule for expansion cohorts. A recent update highlighted that there had been no dose-limiting toxicities observed and the maximum tolerated dose had not been reached. The majority of treatment-related adverse events were grade 1 or 2, and were reversible and manageable on an outpatient basis. Preliminary results from these initial dose cohorts included anti-tumor activity.

Partner Programs Progress into the Clinic

In 2019, Eli Lilly entered the clinic with a novel bispecific; Merck, Celgene (now BMS), and Daiichi Sankyo advanced bispecific candidates toward clinical testing; GSK expanded its Azymetric partnership; and the first ZymeLink ADC collaboration was signed with Iconic Therapeutics. Zymeworks currently has nine active collaborations that offer up to US$7.9 billion in potential milestone payments as well as royalties on potential product sales.

Financial Results for the Year Ended December 31, 2019

Revenue in 2019 was $29.5 million as compared to $53.0 million in 2018. Revenue for both years was primarily comprised of non-recurring upfront fees, expansion payments and milestone payments from Zymeworks’ licensing and collaboration agreements. Revenue for 2019 included $8.0 million received from Eli Lilly for achievement of a development milestone upon their submission of an IND application, $7.5 million received upon BMS’s exercise of its commercial license option, $3.5 million received upon Daiichi Sankyo’s exercise of a commercial license option, and $7.0 million received in other development milestones and research support and other payments from our partners. Revenue in 2019 also included recognition of $3.5 million from deferred revenue relating to the upfront payment received in the prior year from BeiGene under the 2018 licensing and collaboration agreement for development of ZW25.

Revenue for 2018 included recognition of $23.5 million of the $60.0 million upfront fee from BeiGene associated with a new licensing agreement, an $18.0 million upfront fee related to a second licensing agreement with Daiichi Sankyo, a $5.0 million upfront fee related to a new licensing agreement with LEO Pharma, a $4.0 million research program expansion fee from BMS and $2.5 million in other milestones and research support payments from our partners.

For the year ended December 31, 2019, research and development expenses were $115.9 million as compared to $56.9 million in the prior year. The change was primarily due to an increase in activities related to the progression and expansion of ZW25 clinical studies and the associated manufacturing costs, as well as development activities for ZW49 in 2019, and an increase in other research and development activities, which include an increase in salaries and benefits expense as a result of an increase in headcount and non-cash stock-based compensation expense compared to the same period in 2018. Research and development expense included non-cash stock-based compensation expense of $14.3 million, including expense of $8.4 million related to the mark-to-market revaluation of certain historical liability classified equity awards.

For the year ended December 31, 2019, general and administrative expenses were $64.2 million as compared to $29.5 million in the prior year. The change was due to an increase in headcount to support our expanding research and development activities and non-cash stock-based compensation expense. General and administrative expense included non-cash stock-based compensation expense of $34.2 million including $27.5 million expense related to the mark-to-market revaluation of certain historical liability classified equity awards.

Net loss for the year ended December 31, 2019 was $145.4 million as compared to $36.6 million in 2018. This increase in net loss was primarily due to the variances in revenue, research and development expenses and general and administration expenses noted above.

Zymeworks expects research and development expenditures to increase over time due to the ongoing development of our product candidates and other clinical, preclinical, and regulatory activities. Additionally, Zymeworks anticipates continuing to receive revenue from its existing and future strategic partnerships, including technology access fees, milestones and research support payments. However, Zymeworks’ ability to receive these payments is dependent upon either Zymeworks or its collaborators successfully completing specified research and development activities.

As of December 31, 2019, Zymeworks had $298.9 million in cash and cash equivalents and short-term investments.

On March 2, 2020 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the fourth quarter and full year ending December 31, 2019 (Press release, PTC Therapeutics, MAR 2, 2020, View Source [SID1234555058]).

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"2019 was a year of outstanding execution for PTC. I’m proud of the progress made in every area of the business," said Stuart Peltz, Ph.D., CEO of PTC Therapeutics. "We’ve significantly expanded our rare disease portfolio, strengthened our global commercial engine and added vital gene therapy manufacturing capabilities, all while delivering on our revenue guidance. The innovation in our diverse, multiplatform pipeline continues to create value for stakeholders."

Key Fourth Quarter and Other Corporate Highlights:

Expanding commercial footprint including continued revenue growth

Total revenue for the full year 2019 was $307 million. The Duchenne muscular dystrophy (DMD) franchise, consisting of Translarna (ataluren) and Emflaza (deflazacort), continues to grow with 2019 revenue of $291 million.

PTC continues to leverage its strong Latin American infrastructure to support the launch of multiple products. In 2019, Translarna received approval from the Brazilian health regulatory authority (ANVISA) for ambulatory nmDMD patients 5 years and older. In addition, Tegsedi (inotersen) received ANVISA approval for the treatment of stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). In 2019, PTC established an early access program for patients with Familial Chylomicronemia Syndrome (FCS) in Latin America. PTC anticipates filing for approval of Waylivra (volanesorsen) for the treatment of FCS from ANVISA in 2H 2020.

Real-world data from the Translarna STRIDE registry demonstrated that boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) treated with Translarna and standard of care, preserved the ability to walk for 3.5 years longer than compared with those in a matched natural history cohort. Importantly, pulmonary function was also preserved in those treated with Translarna.

A recent publication on Emflaza adds to the growing body of evidence showing its relative benefit and supports the advantage of switching from prednisone to Emflaza. The data in the publication demonstrated that patients treated with Emflaza had better ambulatory function and were able to walk longer, had a lower risk of scoliosis, greater percentage lean body mass and lower weight than patients treated with prednisone.

Positive pivotal trials for risdiplam support planned global launch in broad range of SMA patient types

Following the U.S. Food and Drug Association’s (FDA) acceptance of the New Drug Application (NDA) for risdiplam, a Prescription Drug User Fee Act (PDUFA) date for risdiplam has been set for May 24, 2020.

Roche’s Marketing Authorization Application (MAA) filing with the European Medicines Agency (EMA) for risdiplam is expected mid-year 2020.

Data from part 2 of the FIREFISH trial will be presented during the American Academy of Neurology (AAN) 2020 Annual Meeting, from April 25 to May 1, 2020.

PTC gene therapy platform continues to make progress with regulatory filings and long-term lease of manufacturing facility

In January 2020, the EMA accepted the MAA for the potential approval of PTC-AADC for the treatment of Aromatic l-amino acid decarboxylase (AADC) deficiency. PTC expects the Committee for Medicinal Products for Human Use (CHMP) opinion by year end 2020.

PTC expects to submit a Biologics License Application (BLA) to the FDA in 2Q 2020.

PTC continues to anticipate that more than 300 global, addressable AADC patients will be identified by launch.

In 2019, PTC leased a state-of-the-art biologics manufacturing facility with supporting research and operations buildings in N.J. PTC expects manufacturing at this facility to begin in 2020.

Continued progress with PTC’s multi-platform, multi-product pipeline

The Phase 2 STAR trial in patients with nonsense mutation aniridia evaluating the effect of ataluren did not meet its primary endpoint of Maximum Reading Speed as measured by MNREAD. The results did show a trend in favor of ataluren and the results will be discussed with experts in the coming weeks, after which a decision will be made on a path forward for the program in this indication.

Topline data from the U.S. study to assess dystrophin levels in subjects with nmDMD after treatment with ataluren are expected in 2Q 2020.

In 2019, PTC declared a development candidate in its Huntington’s Disease program. The compound is currently in GLP safety toxicology studies and PTC expects to initiate a clinical trial by the end of the year. The Huntington’s Disease program was developed through PTC’s alternative splicing platform.

In 2020, PTC plans to initiate three clinical trials in two compounds that regulate inflammation and oxidative stress from its Bio-e (redox) platform. PTC acquired the Bio-e platform through an acquisition of assets from BioElectron Technology Corporation in 2019.

Potential registrational trial of PTC743 in patients with Mitochondrial Epilepsy (MEDS) expected to begin in 2Q 2020. MEDS is estimated to affect 5,000-6,000 patients in the U.S. and E.U. combined.

Potential registrational trial of PTC743 in patients with Friedreich Ataxia (FA) expected to begin in 3Q 2020. This is anticipated to be complementary to PTC-FA, a gene therapy candidate for the treatment of FA, which is expected to enter the clinic in 3Q 2020. FA is estimated to affect 25,000 patients worldwide.

PTC857 will enter into a Phase 1 healthy volunteer study in 3Q 2020. The first indication for PTC857 is planned to be GBA Parkinson’s disease based on strong pre-clinical rationale. This condition affects approximately 50,000-90,000 patients in the U.S.

Additional updates on PTC’s platforms and pipeline will be provided at PTC’s Analyst Day on June 16, 2020.

Fourth Quarter and Full-year 2019 Financial Highlights:

Total revenues were $96.5 million for the fourth quarter of 2019, compared to $86.3 million for the fourth quarter of 2018. Total revenues were $307 million for the full year 2019, compared to $264.7 million for the full year 2018.

Translarna net product revenues were $48.4 million for the fourth quarter of 2019, compared to $56 million for the fourth quarter of 2018. Translarna net product revenues were $190 million for the full year 2019, compared to $171 million for the full year 2018.

Emflaza net product revenues were $32.7 million for the fourth quarter of 2019, compared to $29.8 million for the fourth quarter of 2018. Emflaza net product revenues were $101 million for the full year 2019, compared to $92 million for the full year 2018.

GAAP R&D expenses were $81.8 million for the fourth quarter of 2019, compared to $53.6 million for the fourth quarter of 2018. GAAP R&D expenses were $257.4 million for the full year 2019, compared to $172 million for the full year 2018. The increase in R&D expenses reflects costs associated with advancing the gene therapy platform, increased investment in research programs, advancement of the clinical pipeline and the upfront $10M payment for the acquisition of the BioElectron assets.

Non-GAAP R&D expenses were $76.2 million for the fourth quarter of 2019, excluding $5.6 million in non-cash, stock-based compensation expense, compared to $49.7 million for the fourth quarter of 2018, excluding $4 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $236.6 million for the full year 2019, excluding $20.8 million in non-cash, stock-based compensation expense, compared to $155.9 million for the full year 2018, excluding $16.1 million in non-cash, stock-based compensation expense.

GAAP SG&A expenses were $63.5 million for the fourth quarter of 2019, compared to $48.7 million for the fourth quarter of 2018. GAAP SG&A expenses were $202.5 million for the full year 2019, compared to $153.6 million for the full year 2018. The increase in SG&A expenses was primarily due to continued investment to support our commercial activities.

Non-GAAP SG&A expenses were $57.7 million for the fourth quarter of 2019, excluding $5.8 million in non-cash, stock-based compensation expense, compared to $44.2 million for the fourth quarter of 2018, excluding $4.5 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $181.2 million for the full year 2019, excluding $21.3 million in non-cash, stock-based compensation expense, compared to $136.4 million for the full year 2018, excluding $17.2 million in non-cash, stock-based compensation expense.

Change in the fair value of deferred and contingent consideration was $12.4 million for the quarter ended December 31, 2019, compared to $19.3 million for the quarter ended December 31, 2018. Change in the fair value of deferred and contingent consideration was $48.4 million for the year ended December 31, 2019, compared to $19.3 million for the year ended December 31, 2018. The change is related to the fair valuation of the potential future consideration to be paid to former equity holders of Agilis, as a result of our merger with Agilis which closed in August 2018. Changes in the fair value were due to the re-calculation of discounted cash flows for the passage of time and changes to certain other estimated assumptions.

Net loss was $77.7 million for the fourth quarter of 2019, compared to net loss of $48.3 million for the fourth quarter of 2018. Net loss was $251.6 million for the full year 2019, compared to net loss of $128.1 million for the full year 2018.

Cash, cash equivalents, and marketable securities was $686.6 million at December 31, 2019, compared to $227.6 million at December 31, 2018.

Shares issued and outstanding as of December 31, 2019 were 61,935,870.

PTC Reaffirms Full Year 2020 Guidance as Follows

PTC anticipates full year DMD franchise net product revenues to be between $320 and $340 million.

PTC anticipates GAAP R&D and SG&A expense for the full year 2020 to be between $610 and $640 million.

PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2020 to be between $545 and $575 million, excluding estimated non-cash, stock-based compensation expense of approximately $65 million.

Today’s Conference Call and Webcast Reminder:
Today’s conference call will take place at 4:30 pm ET and can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 1732477. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. The accompanying slide presentation will be posted on the investor relations section of the PTC website. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.