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Bellicum Announces Interim BPX-601 Data and Corporate Restructuring

On October 29, 2020 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported interim data from its BPX-601 dose-escalation clinical trial in patients with relapsed/refractory metastatic pancreatic cancer (Press release, Bellicum Pharmaceuticals, OCT 29, 2020, View Source [SID1234569369]). Findings from the first four patients treated with BPX-601 followed by repeat rimiducid dosing showed evidence of rimiducid-mediated CAR-T cell activation. Clinically meaningful efficacy as measured by RECIST criteria was not observed.

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After an extensive review of its organization and programs, the company has implemented a restructuring plan, including a reduction in staff, to focus its efforts on its clinical GoCAR-T product candidates. For BPX-601, the first candidate incorporating iMC, the company expects to begin enrolling patients with metastatic castration-resistant prostate cancer (mCRPC) in the ongoing Phase 1/2 clinical trial before the end of the year and intends to review its plans in pancreatic cancer upon completion of the current safety cohort. For BPX-603, the company’s first dual-switch GoCAR-T candidate, Bellicum expects to initiate enrollment of patients with HER2+ solid tumors in a Phase 1/2 clinical trial also by the end of the year. In order to preserve operating capital for these clinical trials, the company plans to pause development of its BCMA GoCAR-NK program.

"The results we have observed in the BPX-601 study are encouraging in terms of safety and GoCAR-T cell activation, proliferation, and persistence. We are eager to investigate our technology further in new tumor types like mCRPC and against established target antigens like HER2," said Rick Fair, President and Chief Executive Officer of Bellicum. "We have concluded that Bellicum must reduce spending on preclinical programs and shift its resources to enable achievement of meaningful milestones in the clinic. We regret the impact this unavoidable decision will have on our departing employees and we sincerely thank them for their contributions and dedication."

Interim BPX-601 Data

As of July 9, 2020, four patients were treated with 5×106 BPX-601 cells/kg followed by 2-11 doses of rimiducid in cohort 5C of the Phase 1 dose escalation clinical trial. Interim results include the following observations:

Administration of BPX-601 and repeat doses of rimiducid was tolerated as follows:

No treatment-related adverse events ³Grade 2 were observed in these four patients; one treatment-related SAE (Grade 4 cytokine release syndrome) was reported in a patient treated after data cutoff

One genitourinary adverse event was reported (Grade 1 intermittent hematuria)

Four events of Grade 1 neurotoxicity (neurotoxicity, dysgraphia, and confusion x2) were reported in two patients

The safety profile observed was otherwise consistent with previous reports

Best Overall Response in these patients included 3 stable disease and 1 progressive disease

Evidence of repeat rimiducid-mediated CAR-T cell activation was observed as follows:

Rimiducid administration was associated with increased serum cytokine levels, including IL-5, TNF-α, and IFN-g

Rimiducid treatment was also associated with increased expression of activation markers (e.g. CD25) on peripheral CD4+ and CD8+ T cells, indicative of systemic immune modulation via BPX-601 iMC activation

In two evaluable subjects receiving >2 doses of rimiducid, repeat rimiducid dosing was not shown to increase the peak or AUC of circulating BPX-601 cells relative to single-dose rimiducid

Consistent with previous cohorts, rimiducid administration was associated with a transient decline followed by partial recovery in circulating BPX-601 cells

Corporate Restructuring

Under the restructuring program, the company will focus on the clinical development of BPX-601 and BPX-603, pause the BCMA GoCAR-NK program, and discontinue discovery research and new product development. Staff will be reduced by 79%, from 68 to 14 full-time employees by the end of 2020, and Bellicum expects to incur severance expenses of approximately $2.5 million. The company also intends to pay down all of its Oxford Finance debt obligations using cash on hand with payment of $27.4 million in principal plus applicable fees and accrued interest on or before October 30, 2020. These actions are expected to reduce the company’s expenses and extend its cash runway. The company now expects annual cash utilization of $25 to $30 million.

Idera Pharmaceuticals Reports Third Quarter Financial Results and Provides Corporate Update

On October 29, 2020 Idera Pharmaceuticals, Inc. ("Idera" or the "Company") (Nasdaq: IDRA) reported its financial and operational results for the third quarter ended September 30, 2020 (Press release, Idera Pharmaceuticals, OCT 29, 2020, View Source [SID1234569368]).

"Tilsotolimod is the most advanced TLR-9 agonist therapy in development, and we have made tremendous strides against our objectives for 2020," stated Vincent Milano, Idera’s Chief Executive Officer. "We continue to work diligently against our timelines for ILLUMINATE-301, which currently remain on track for data in the first quarter of 2021. In addition, our recent patent and allowed application for tilsotolimod method-of-use in colorectal and head and neck cancers as well as the continuation of ILLUMINATE-206 reinforce our ‘beyond melanoma’ strategy. We also have the financing to help our outstanding team continue to execute these key objectives."

Corporate Update

Since June 30, 2020, the following corporate updates were announced:

·The Company entered into a private placement of up to $20.0 million, with $5.1 million received in July 2020. The Company anticipates that its current cash, cash equivalents, and short-term investments will fund our operations through the second quarter of 2021. With this private placement, the Company has now entered into three financing vehicles since December 2019, which it believes could provide proceeds of up to $118.2 million to fund the potential NDA filing and commercial launch of tilsotolimod.
·The Company received a new U.S. Patent and allowed application for tilsotolimod, providing exclusivity through September 2037 when intratumoral tilsotolimod is used with certain immune checkpoint inhibitors in treating colorectal cancer (CRC) and head and neck squamous cell carcinoma (HNSCC).

ILLUMINATE (tilsotolimod) Clinical Development Updates

ILLUMINATE-301: Randomized phase 3 trial of tilsotolimod in combination with Yervoy* (ipilimumab) versus Yervoy alone in patients with anti-PD-1 refractory advanced melanoma:

•Primary endpoint family of overall response rate (ORR) by blinded independent central review using RECIST v1.1 and overall survival (OS);
•Trial initiated in March 2018;
•Enrollment completed in March 2020; and
•ORR and other preliminary data expected in the first quarter of 2021.

ILLUMINATE-206: Phase 2, open-label, multicohort, multicenter study to test the safety and effectiveness of tilsotolimod in combination with Yervoy and Opdivo* (nivolumab) for the treatment of solid tumors:

·Trial initiated in September 2019 with the microsatellite stable colorectal cancer (MSS-CRC) cohort;
·Initial safety run-in of 10 patients, which included Yervoy at 1 mg/kg every 8 weeks and Opdivo at 3 mg/kg every 2 weeks, showed that the regimen was generally well tolerated;
·Changes in the study design intended to improve potential outcomes in this patient population include increasing Yervoy dosing frequency to every 3 weeks and limiting the number of allowed prior lines of treatment to 2; and
·The Company has opened enrollment for the next 10 patients under the modified study design, with data anticipated in the second quarter of 2021.

ILLUMINATE-204: Phase 1/2 trial of tilsotolimod in combination with Yervoy or Keytruda± (pembrolizumab) in patients with anti-PD-1 refractory advanced melanoma:

·Final results from the recommended phase 2 dose (RP2D) of 8 mg of tilsotolimod in combination with Yervoy, which is the treatment regimen being evaluated in the Company’s registrational trial, ILLUMINATE-301, were shared in a Mini Oral presentation at the ESMO (Free ESMO Whitepaper) Virtual Congress in September 2020.

Third Quarter Financial Results

Research and development expenses for the three months ended September 30, 2020, totaled $4.8 million compared to $8.4 million for the same period in 2019. General and administrative expense for the three months ended September 30, 2020, totaled $2.7 million compared to $3.0 million for the same period in 2019. Additionally, during the three months ended September 30, 2020, we recorded $0.7 million and $12.4 million non-cash warrant revaluation loss and non-cash future tranche right revaluation loss, respectively, related to securities issued in connection with our December 2019 private placement transaction.

As a result of the factors above, net loss applicable to common stockholders for the three months ended September 30, 2020, was $20.6 million, or $0.59 per basic and diluted share, compared to net loss applicable to common stockholders of $11.1 million, or $0.39 per basic and diluted share, for the same period in 2019. Excluding the non-cash loss of approximately $13.1 million for the three months ended September 30, 2020, related to the securities issued in connection with the December 2019 private placement transaction, net loss applicable to common stockholders was $7.5 million, or $0.21 per basic and diluted share (calculated based upon the basic weighted-average number of common shares, due to the antidilutive effect of net loss).

As of September 30, 2020, our cash, cash equivalents, and short-term investments totaled $29.0 million. Based on our current operating plan, we anticipate that our current cash, cash equivalents, and short-term investments, will fund our operations through the second quarter of 2021.

Illumina Reports Financial Results for Third Quarter of Fiscal Year 2020

On October 29, 2020 Illumina, Inc. (NASDAQ: ILMN) reported its financial results for the third quarter of fiscal year 2020 (Press release, Illumina, OCT 29, 2020, View Source [SID1234569367]).

Third quarter 2020 results reflect strong sequential growth:

•Revenue of $794 million, an increase of 26% compared to the second quarter of 2020 and a 12% decrease compared to $907 million in the prior year period
•GAAP net income attributable to Illumina stockholders for the quarter of $179 million, or $1.21 per diluted share, compared to $234 million, or $1.58 per diluted share, for the prior year period
•Non-GAAP net income attributable to Illumina stockholders for the quarter of $150 million, or $1.02 per diluted share, compared to $286 million, or $1.93 per diluted share, for the prior year period. Non-GAAP net income excludes net gains from mark-to-market adjustments on our strategic investments, primarily from our marketable equity securities (see the "Reconciliation Between GAAP and Non-GAAP Net Income Attributable to Illumina Stockholders" table for a reconciliation of these GAAP and non-GAAP financial measures)
•Cash flow from operations of $153 million compared to $267 million in the prior year period
•Free cash flow (cash flow from operations less capital expenditures) of $105 million for the quarter compared to $218 million in the prior year period

"Our business accelerated in the third quarter with sequencing consumable revenue growing 29% from the second quarter," said Francis deSouza, Chief Executive Officer. "We’re also making progress incorporating genomics into the standard of care in non-invasive pre-natal testing, oncology therapy selection, and genetic disease diagnosis. Looking forward, we believe our planned acquisition of GRAIL will catalyze a new era of early cancer detection, transforming cancer survivability and opening up the largest clinical application of genomics we’ve seen."

Gross margin in the third quarter of 2020 was 66.2% compared to 71.5% in the prior year period. Excluding amortization of acquired intangible assets and the net impact from expenses and payroll credits related to COVID-19, non-GAAP gross margin was 67.4% for the third quarter of 2020 compared to 72.5% in the prior year period.

Research and development (R&D) expenses for the third quarter of 2020 were $172 million compared to $151 million in the prior year period. Excluding expenses related to COVID-19, non-GAAP R&D expenses as a percentage of revenue were 21.2% compared to 16.4% in the prior year period.

Selling, general and administrative (SG&A) expenses for the third quarter of 2020 were $192 million compared to $189 million in the prior year period. Excluding amortization of acquired intangible assets, gain on litigation, acquisition-related expenses, and expenses related to COVID-19, non-GAAP SG&A expenses as a percentage of revenue were 24.8% compared to 20.0% in the prior year period.

Depreciation and amortization expenses were $46 million and capital expenditures for free cash flow purposes were $48 million during the third quarter of 2020. At the close of the quarter, the company held $3.3 billion in cash, cash equivalents and short-term investments, compared to $3.4 billion as of December 29, 2019.
Updates since our last earnings release:

•Announced a definitive agreement to acquire GRAIL to accelerate commercialization and adoption of transformative multi-cancer screening genetic tests
•Released the NovaSeqTM 6000 v1.5 reagents to unlock deeper genomic discoveries by making a $600 genome more broadly available and catalyzing the next wave of adoption in smaller core labs
•Updated guidelines from the American College of Obstetricians and Gynecologists (ACOG) recommended Non-Invasive Prenatal Testing (NIPT) be made available to all pregnant women, regardless of maternal age or baseline risk
•Repurchased approximately $125 million of common stock in the third quarter with $295 million remaining for repurchase under our current plan
•Appointed Kathryne Reeves as Chief Marketing Officer

Financial outlook and guidance

As previously announced, Illumina has withdrawn its fiscal 2020 full year revenue and earnings per share guidance due to the uncertainties around the severity and duration of the COVID-19 pandemic.

Quarterly conference call information

The conference call will begin at 2:00 pm Pacific Time (5:00 pm Eastern Time) on Thursday, October 29, 2020. Interested parties may access the live teleconference through the Investor Info section of Illumina’s website under the "Company" tab at www.illumina.com. Alternatively, individuals can access the call by dialing 1 (866) 211-4597 or 1 (647) 689-6853 outside North America, both with conference ID 9488740.

A replay of the conference call will be posted on Illumina’s website after the event and will be available for at least 30 days following.

Statement regarding use of non-GAAP financial measures

The company reports non-GAAP results for diluted net income per share, net income, gross margins, operating expenses, operating margins, other income, and free cash flow in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The company’s financial measures under GAAP include substantial charges such as amortization of acquired intangible assets, non-cash interest expense associated with the company’s convertible debt instruments that may be settled in cash, and others that are listed in the itemized reconciliations between GAAP and non-GAAP financial measures included in this press release. Management has excluded the effects of these items in non-GAAP measures to assist investors in analyzing and assessing past and future operating performance. Additionally, non-GAAP net income attributable to Illumina stockholders and diluted earnings per share attributable to Illumina stockholders are key components of the financial metrics utilized by the company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

The company encourages investors to carefully consider its results under GAAP, as well as its supplemental non-GAAP information and the reconciliation between these presentations, to more fully understand its business. Reconciliations between GAAP and non-GAAP results are presented in the tables of this release.

OPKO Health Reports 2020 Third Quarter Business Highlights and Financial Results

On October 29, 2020 OPKO Health, Inc. (NASDAQ: OPK) reports business highlights and financial results for the three months ended September 30, 2020 (Press release, Opko Health, OCT 29, 2020, View Source;20.htm [SID1234569365]).

Third Quarter Business Highlights

•BioReference Laboratories’ COVID-19 PCR testing volume increased 61% over the second quarter of 2020. During the third quarter, BioReference Laboratories (BRL) processed approximately 3.5 million COVID-19 PCR tests and currently has the capacity to process more than 70,000 tests per day. In addition, the laboratory performed approximately 300,000 COVID-19 serology tests to measure SARS-CoV-2 specific antibody levels with the capacity to process more than 400,000 serology tests per day.

BRL announced additional COVID-19 testing agreements for New York City schools through a continued collaboration with New York City, the Department of Health and Hospital Corporation, the Test & Trace Corp. and the Department of Education. Through these agreements, BRL is testing students, teachers and staff in nearly 1,000 public schools across New York. BRL continues to provide COVID-19 testing services to numerous states, cities, professional sports associations and healthcare organizations, as well as to over 600 drive-thru and retail pharmacy testing sites nationwide.

•Positive somatrogon Phase 3 topline results reported from crossover pediatric study. A global Phase 3 study of somatrogon administered once-weekly to children 3 to <18 years of age with growth hormone deficiency met its primary objective of improved treatment burden compared to daily injection of Genotropin as measured by the mean overall Life Interference total score after 12 weeks of treatment.

•Somatrogon global regulatory submissions: Pfizer remains on schedule with respect to its regulatory submissions for marketing approval of somatrogon for children with growth hormone deficiency in the U.S. in the fourth quarter of this year and in Europe and Japan in the first half of 2021.

•RAYALDEE has received marketing authorizations in seven European countries. Vifor Fresenius Medical Care Renal Pharma, OPKO’s commercial partner for RAYALDEE in Europe, has received marketing authorizations for RAYALDEE for the treatment of secondary hyperparathyroidism in adults with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency in the United Kingdom, Germany, Sweden, Norway, Ireland, Denmark and the Netherlands. Market authorizations from Spain, Portugal, Italy and Switzerland are still pending. Market launch of RAYALDEE in authorized countries is expected to begin next year.
•RAYALDEE total prescriptions reported by IQVIA increased 13% compared with the third quarter of 2019. Total prescriptions for the three months ended September 30, 2020 increased to approximately 16,700, compared with approximately 14,800 for the third quarter of 2019. During the third quarter of 2020, demand for RAYALDEE was impacted by challenges in onboarding new patients because of the COVID-19 pandemic.

•RAYALDEE Phase 2 clinical trial initiated in patients with mild-to-moderate COVID-19. OPKO initiated a Phase 2 clinical trial with RAYALDEE as a treatment for mild to moderate COVID-19. The trial, "A Randomized, Double-Blind Placebo-Controlled Study to Evaluate the Safety and Efficacy of RAYALDEE (calcifediol) Extended-release Capsules to Treat Symptomatic Patients Infected with SARS-CoV-2 (REsCue)," is expected to enroll approximately 160 subjects, many with stage 3 or 4 CKD who are at increased risk for developing more severe illness. The REsCue trial will randomize COVID-19 outpatients in a 1:1 ratio to 4 weeks of treatment with RAYALDEE or placebo, and 2 weeks of follow-up. Primary efficacy endpoints are raising and maintaining serum total 25-hydroxyvitamin D within the range of 50-100 ng/mL and time to resolution of COVID-19 symptoms. Numerous independent studies report a correlation between vitamin D status and COVID-19 risk and severity.

•GeneDx enters into agreement with Pediatrix Medical Group to offer neonatal genomic services. In August 2020, BRL’s GeneDx subsidiary announced an agreement with Pediatrix Medical Group, the nation’s leading provider of maternal-fetal, and pediatric medical and surgical subspecialty physician services, to offer state-of-the-art, next-generation genomic sequencing to support clinical diagnosis in neonatal intensive care units staffed by neonatologists affiliated with Pediatrix. The sequencing is designed to enhance diagnostic capabilities in order to lessen the impact of disease and to facilitate the development of novel precision medicine solutions for pediatric care.

•BioReference Laboratories launches best-in-class next-generation sequencing assay. In September 2020, BRL, along with its GenPath specialty oncology division, announced the launch of OnkoSight Advanced, a next-generation sequencing (NGS) assay that enables revolutionary deoxyribonucleic acid (DNA) mutational profiling of tumor samples. OnkoSight Advanced NGS testing provides targeted gene content that is aligned with the latest National Comprehensive Cancer Network and World Health Organization guideline recommendations to provide critical insight into many of the most common cancer types. Each OnkoSight Advanced panel includes key biomarkers – Tumor Mutation Burden and Tumor-Only Microsatellite, critical when profiling advanced-stage malignancies to guide potential immunotherapy.

Third Quarter Financial Results

•Net income for the third quarter of 2020 was $23.7 million, or $0.04 per diluted share, compared with a net loss of $62.0 million, or $0.11 per share, for the comparable period of 2019. Consolidated revenues for the third quarter of 2020 were $428.1 million compared with $228.8 million for the comparable period of 2019.
•Diagnostics: Revenue from services in the third quarter of 2020 was $392.5 million compared with $181.1 million in the prior-year period, primarily due to increased COVID-19 testing volumes, partially offset by reduced clinical and genomic test volumes related to the pandemic and lower clinical and genomic test reimbursement. In addition, the Company received a $10.0 million grant from the CARES Act in the third quarter. Total costs and expenses were $346.4 million in the third quarter of 2020 compared with $197.5 million in the third quarter of 2019. This increase represents higher volumes from both COVID-19 testing and from the core testing business. Operating income was $46.2 million in the third quarter of 2020 compared with an operating loss of $16.4 million in the prior-year period, an improvement of $62.5 million.
•Pharmaceuticals: Revenue from products in the third quarter of 2020 was $28.7 million compared with $26.2 million in the third quarter of 2019, with the increase primarily attributable to higher sales at OPKO Chile and an increase in net sales of RAYALDEE to $8.1 million in the third quarter of 2020 compared with $7.4 million in the prior-year period. Revenue from transfer of intellectual property was $6.8 million in the third quarter of 2020 compared with $20.7 million in the third quarter of 2019 reflecting a decrease in the amortization of payments received from Pfizer with respect to somatrogon. Total costs and expenses were $49.9 million in the third quarter of 2020 compared with $61.1 million in the prior-year period, with the decline primarily attributable to lower research and development expenses due to the completion of the pediatric human growth hormone Phase 3 trial. The operating loss was $14.4 million in the third quarter of 2020 compared with $14.2 million in the third quarter of 2019.
•Cash and equivalents: Cash, cash equivalents and marketable securities were $36.3 million as of September 30, 2020, which reflects the repayment, in full, of its line of credit with JP Morgan utilizing cash generated from operations of $63.0 million during the three months ended September 30, 2020. In addition, the Company has availability under its line of credit with JP Morgan of $64.7 million and an unutilized $100 million credit facility that provides access to incremental capital on a non-dilutive basis.

CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update and discuss third quarter financial results in greater detail during a conference call and live audio webcast at 4:30 p.m. Eastern time today, October 29, 2020. Participants are requested to pre-register for the conference call using the link here, or dialing (888) 869-1189 or (706) 643-5902 and using conference ID 4542807. Upon registering, participants will receive dial-in numbers, an event passcode and a unique registrant ID to gain immediate access to the call and bypass the live operator. Participants may pre-register at any time, including up to and after the start of the call.

To access the live call via webcast, please click on the link OPKO 3Q20 Results Conference Call. Individual investors and investment community professionals who do not plan to ask a question during the call’s Q&A session are encouraged to listen to the call via the webcast.

For those unable to listen to the live conference call, a replay can be accessed for a period of time on OPKO’s website at OPKO 3Q20 Results Conference Call. A telephone replay will be available beginning approximately two hours after the completion of the conference call. To access the replay, please dial (855) 859-2056 or (404) 537-3406, and use conference ID 4542807.

PTC Therapeutics Reports Third Quarter 2020 Financial Results and Provides a Corporate Update

On October 29, 2020 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported financial results for the third quarter ending September 30, 2020 and provided a corporate update (Press release, PTC Therapeutics, OCT 29, 2020, View Source [SID1234569364]).

"I am very pleased by the rapid progress that we have made this quarter with the approval and strong U.S. launch of Evrysdi for spinal muscular atrophy and the continued growth across our global Duchenne muscular dystrophy franchise," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "We have also made significant advances across our clinical pipeline with a registration-directed trial in mitochondrial epilepsy initiated this quarter and an additional registration-directed trial in Friedreich ataxia to initiate before year end. The mission at PTC has always been to bring therapies to patients with high unmet medical need through investment in innovative technologies and we believe we are well positioned to bring long-term value for all of our stakeholders."

Key Third Quarter and Other Corporate Updates

●In August 2020, the U.S. Food and Drug Administration (FDA) approved Evrysdi (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older. In the early stages of launch, patients across a broad age range and SMA type have been treated. In October 2020, there were additional approvals in Brazil, Chile and Ukraine, further expanding the global reach of Evrysdi. Evrysdi is a product of the SMA collaboration between PTC, the SMA Foundation, and Roche.
●Also in August 2020, the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) filed by Roche for Evrysdi for the treatment of SMA. The EMA previously granted PRIME (PRIority MEdicines) designation to risdiplam for the treatment of SMA, providing a pathway for accelerated evaluation by the agency.
●The EMA’s acceptance of the MAA filed by Roche for Evrysdi for the treatment of SMA triggered a $15 million milestone payment to PTC and the first commercial sale of Evrysdi in the U.S. triggered a $20 million milestone payment to PTC. As a result, PTC recognized $35.0 million in collaboration revenue associated with Roche milestone events in the third quarter of 2020.
●In October 2020, Chugai Pharmaceutical, Co. Ltd, a member of the Roche group, announced that a new drug application for Evrysdi for the treatment of SMA was filed in Japan. Risdiplam received orphan drug designation from the Japan Ministry of Health, Labour and Welfare and the application is subject to a priority review. The filing in Japan triggered a $7.5 million milestone payment to PTC in the fourth quarter of 2020. Roche is executing an aggressive global regulatory strategy and now has over 20 additional applications filed for Evrysdi around the world.
●PTC has multiple aromatic L-amino acid decarboxylase (AADC) deficiency patient finding initiatives ongoing which continue to progress even amid COVID-19. PTC has expanded the number of
programs globally and in September 2020. PTC launched its newest program, PTC PINPOINT. The program offers testing at no charge to patients which can lead to an earlier diagnosis and treatment.

Third Quarter Clinical Updates

●Two-year data from Part 1 of the Evrysdi FIREFISH study were presented at the 25th International Annual Congress of the World Muscle Society (WMS). The data demonstrated that infants treated with Evrysdi continued to improve and achieve motor milestones.
●A registration-directed Phase 2/3 placebo-controlled trial to evaluate vatiquinone (PTC743) in children with mitochondrial epilepsy began enrollment in October 2020. This MIT-E trial is targeting the highly morbid and life-threatening symptom of refractory seizures in children with inherited mitochondrial disease. Vatiquinone, developed from PTC’s Bio-e platform, is an investigational oral small molecule that inhibits 15-Lipoxygenase, a key enzyme that regulates oxidative stress and inflammation response pathways that underpin neurological disease pathology including epilepsy. The FDA has granted vatiquinone orphan drug designation and pediatric rare disease designation for mitochondrial epilepsy.
●The Phase 3 vatiquinone Friedreich ataxia trial (MOVE-FA) is expected to initiate in the fourth quarter of 2020 and the Phase 3 PTC923 phenylketonuria (PKU) trial is expected to initiate in mid-2021.
●Muscle biopsies have been completed on all 20 enrolled subjects in PTC’s TranslarnaTM (ataluren) dystrophin study (Study 045). The samples from these biopsies are in the process of being analyzed and PTC expects to report top-line results in the first quarter of 2021.
●A Phase 1 study of PTC518 in healthy volunteers is expected to begin enrollment this quarter. PTC518 is another candidate from PTC’s validated splicing platform and is being developed for the treatment of Huntington disease.
●The Phase 1 single ascending dose study for PTC857 has been completed and dosing in the multiple ascending dose study is expected to be completed by the end of 2020. PTC857 was developed from the Bio-e platform with the potential to address multiple CNS disorders, with the first planned indication being glucocerebrosidase (GBA) Parkinson’s disease.
●A Phase 2/3 clinical trial for PTC299 for COVID-19 (FITE19) continues to enroll patients. PTC is expecting Stage 1 of this two-stage trial to be completed by the end of 2020 and continues to anticipate reporting top-line results from both stages in the first half of 2021. Stage 1 of this study is being conducted in the U.S., Brazil, Spain, and Australia.
●Due to COVID-19 related delays, PTC expects the EMA’s Committee for Medicinal Products for Human Use final opinion for the AADC deficiency application in the first half of 2021. PTC expects the biologics license application (BLA) for AADC deficiency to be submitted to the FDA in the first half of 2021.

Financial Highlights

●Total revenue was $118.4 million for the third quarter of 2020, compared to $71.4 million for the third quarter of 2019, a 66% year-over-year increase, Total revenue includes net product revenue of $82.7 million and collaboration and royalty revenue of $35.7 million in the third quarter of 2020.
●Total net product revenue across the commercial portfolio was $82.7 million for the third quarter of 2020, a 16% year-over-year increase.
●The Duchenne muscular dystrophy franchise continued to show strong performance in the third quarter with a 15% year-over-year increase. Emflaza continued its momentum from the second quarter of 2020 into the third quarter of 2020 driven by new patient prescriptions and ongoing
operational improvements in PTC’s commercial business. New Duchenne patients continue to be identified in Europe, LATAM and other key markets for Translarna.
●Translarna net product revenue was $43.4 million for the third quarter of 2020, compared to $48.3 million for the third quarter of 2019, a 10% year-over-year decrease. Revenue for the third quarter of 2020 was impacted by the timing of a group purchase order from Brazil, which is the primary driver for the year-over-year decrease compared to the third quarter of 2019.
●In October, PTC entered into a purchase agreement with the Brazil Ministry of Health for Translarna. The initial shipment was received by the Brazil Ministry of Health in October 2020 with the additional shipment expected in the first half of 2021.
●Emflaza net product revenue was $38.5 million for the third quarter of 2020, compared to $22.9 million for the third quarter of 2019. Growth in net product revenue was driven by new patient prescriptions and continued operational improvements and efficiencies in the commercial business.
●Roche reported initial Evrysdi August and September sales of approximately CHF 8 million. The acceptance of the MAA filed by Roche for Evrysdi for the treatment of SMA triggered a $15 million milestone payment to PTC and the first commercial sale of Evrysdi in the U.S. triggered a $20 million milestone payment to PTC. As a result, PTC recognized $35.0 million in collaboration revenue associated with Roche milestone events in the third quarter of 2020.
●Based on U.S. GAAP (Generally Accepted Accounting Principles), research and development (R&D) expenses were $93.0 million for the third quarter of 2020, compared to $63.1 million for the third quarter of 2019. The increase in R&D expenses reflects costs associated with advancing the gene therapy and Bio-e platforms, increased investment in research programs, and advancement of the clinical pipeline.
●Non-GAAP R&D expenses were $83.8 million for the third quarter of 2020, excluding $9.2 million in non-cash, stock-based compensation expense, compared to $58.1 million for the third quarter of 2019, excluding $5.0 million in non-cash, stock-based compensation expense.
●GAAP selling, general and administrative (SG&A) expenses were $57.8 million for the third quarter of 2020, compared to $49.3 million for the third quarter of 2019. The increase reflects continued investment to support PTC’s commercial activities including the expanding commercial portfolio.
●Non-GAAP SG&A expenses were $50.2 million for the third quarter of 2020, excluding $7.6 million in non-cash, stock-based compensation expense, compared to $43.8 million for the third quarter of 2019, excluding $5.5 million in non-cash, stock-based compensation expense.
●Change in the fair value of deferred and contingent consideration was $8.4 million for the third quarter of 2020, compared to $9.5 million for the third quarter of 2019. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.
●Net loss was $69.7 million for the third quarter of 2020, compared to net loss of $60.0 million for the third quarter of 2019.
●Cash, cash equivalents and marketable securities were $1,141.0 million as of September 30, 2020, compared to $686.6 million as of December 31, 2019. The cash, cash equivalents and marketable securities balance as of September 30, 2020 includes $650.0 million in consideration received related to the July 17, 2020 Royalty Purchase Agreement between PTC and RPI 2019 Intermediate Finance Trust ("RPI").
●Shares issued and outstanding as of September 30, 2020 were 67,809,560.

Non-GAAP Financial Measures

In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.

Conference Call and Webcast

PTC will host a conference call to discuss the third quarter of 2020 operational and financial today at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 7096445. 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. 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.