On May 4, 2021 Veracyte, Inc. (Nasdaq: VCYT) reported that new data relating to the Prosigna Breast Cancer Gene Signature Assay will be presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Breast Cancer Virtual Congress 2021 taking place May 5-8 (Press release, Veracyte, MAY 4, 2021, View Source [SID1234579116]).

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"New data accepted for presentation at this year’s ESMO (Free ESMO Whitepaper) Breast Cancer Congress provide further evidence for the importance of genomic testing in breast cancer, particularly to help physicians and patients make better informed treatment decisions based on the unique biology of individual patients’ tumors," said Bonnie Anderson, Veracyte’s chairman and chief executive officer. "We believe such data strengthen the backdrop for our Prosigna Breast Cancer Gene Signature Assay, and greatly appreciate the breast cancer experts who led and participated in these studies."

Following are details of the Prosigna-related posters accepted for presentation at the ESMO (Free ESMO Whitepaper) Breast Cancer Virtual Congress. These abstracts are available to meeting registrants on demand:

Title: Influence of PAM50 on therapeutic decisions in patients with early-stage Luminal Breast Cancer in a single centre

Poster: 62P

First Author: Alejandro Olivares-Hernandez, M.D., M.Sc., IBSAL – Instituto de Investigación Biomédica de Salamanca, Salamanca, Spain

Title: Consensus on the utility of breast cancer multigene signatures in routine clinical practice among European Breast Cancer clinicians – The PROCURE project

Poster: 24P

First Author:

Giuseppe Curigliano, M.D., Ph.D., IEO – Istituto Europeo di Oncologia, Milan, Italy

The first poster will present findings from a prospective study conducted at the University Hospital of Salamanca (Spain), evaluating the influence of the PAM50 gene signature, the foundation of the Prosigna assay, on therapeutic decision-making for 143 patients with early-stage breast cancer.

The second poster will feature initial findings and consensus from the first wave of the PROCURE (Prosigna Clinical Utility Review) project, a European study utilizing the Delphi methodology to generate consensus regarding the clinical utility of genomic tests, including the Prosigna Breast Cancer Gene Signature Assay, in breast cancer treatment. The study is led by an independent scientific committee of eight breast cancer experts with input from 141 breast cancer clinicians practicing in nearly a dozen European countries.

About Prosigna

The Prosigna Breast Cancer Gene Signature Assay is a prognostic genomic test, built from PAM50 molecular subtypes, which combines tumor gene expression with clinicopathologic factors to better inform treatment decisions. The assay is indicated in female breast cancer patients who have undergone either mastectomy or breast-conserving therapy in conjunction with locoregional treatment consistent with standard of care, either as a prognostic indicator for distant recurrence-free survival at 10 years in post-menopausal women with Hormone Receptor-Positive (HR+), lymph node-negative, Stage I or II breast cancer, or lymph node-positive (1–3 positive nodes, or 4 or more positive nodes), Stage II or IIIA breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors. Outside of the United States, the Prosigna Breast Cancer Gene Signature Assay also provides the intrinsic subtypes of the tumor tissue within three groups, low, intermediate and high, by the nCounter Analysis System through decentralized performance.

The Prosigna test is FDA 510(k) cleared in the United States for use on the nCounter Analysis System and is available for use when ordered by a physician. The Prosigna test has received CE Mark and is available for use by healthcare professionals in the European Union and other countries that recognize the CE Mark, as well as in Canada, Israel, Australia, New Zealand and Hong Kong. The assay is covered by Medicare and leading private payers in the United States, and is widely covered by government and private payers in the countries where it is available.

On May 4, 2021 VBL Therapeutics (Nasdaq: VBLT) reported that it will release its first quarter financial results for the period ended March 31, 2021 on Tuesday, May 11, 2021 before market open (Press release, VBL Therapeutics, MAY 4, 2021, View Source [SID1234579115]). Professor Dror Harats, M.D, Chief Executive Officer and Amos Ron, Chief Financial Officer, will host a conference call at 8:30am EDT the same day to discuss the results and provide a corporate update.

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On May 4, 2021 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported its financial results for the first quarter 2021 and reaffirmed its financial guidance for 2021 (Press release, Ultragenyx Pharmaceutical, MAY 4, 2021, View Source [SID1234579114]).

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"We continue to have strong Crysvita and Dojolvi launches and execute on our development priorities across our pipeline," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In 2021, we will have ongoing pivotal trials for three gene therapies, one pivotal monoclonal antibody study for osteogenesis imperfecta, our first mRNA program for GSDIII, and an antisense oligonucleotide for Angelman with our partner GeneTx. These six clinical programs along with our commercial and early-stage programs make ours one of the most diversified pipelines in rare disease."

First Quarter 2021 Financials

In the first quarter 2021, Crysvita revenue in Ultragenyx territories1 increased 46% versus the first quarter 2020. This increase is driven by demand from pediatric and adult patients with X-linked hypophosphatemia (XLH) and patients with tumor induced osteomalacia (TIO), which became Crysvita’s second approved indication in the United States in June 2020.

Dojolvi revenue in the first quarter 2021 continued to build on the launch momentum after the U.S. Food and Drug Administration (FDA) approval in June 2020. As of the end of the first quarter 2021 the company has received approximately 230 completed start forms from approximately 115 unique prescribers. This has led to approximately 180 patients on reimbursed therapy as of the end of March 2021.

First quarter 2021 revenue included $42.8 million related to the technology transfer as part of the Daiichi Sankyo strategic manufacturing partnership around the HeLa PCL and HEK293 technologies. This revenue is expected to taper significantly through the end of this year as these activities come to a close.

Total operating expenses of $206.0 million in the first quarter 2021 increased 31% or $49.0 million versus the first quarter 2020, primarily driven by a $50.0 million expense for the upfront payment on the closing of the license and collaboration agreement with Mereo Biopharma. Excluding this upfront payment, total operating expenses for the year are expected to increase modestly as the company prepares for and enrolls six clinical programs, including four registrational studies.

Net cash used in operations for the quarter ended March 31, 2021 was $159.3 million, compared to net cash used of $95.2 million for the same period in 2020. Cash, cash equivalents, and marketable debt securities were $1.0 billion as of March 31, 2021.

2021 Financial Guidance

Crysvita Guidance in Ultragenyx Territories
The company reaffirms the 2021 guidance range for Crysvita that was provided at the beginning of the year. This range is $180 million to $190 million and includes the North American profit share region and the other regions where product sales are recognized.

Program Updates and Upcoming Milestones

Dojolvi for the treatment of Long-chain Fatty Acid Oxidation Disorders, or LC-FAOD

Health Canada approved Dojolvi for the treatment of adult and pediatric patients with LC-FAOD in February 2021 and the therapy was made commercially available to patients in Canada beginning in April 2021.
Recently published data2 under France’s nominative Authorization for Temporary Use program , or Authorisations Temporaires d’Utilisation (ATU), of 18 pediatric and adult patients with LC-FAOD showed that Dojolvi (triheptanoin) led to reductions in LC-FAOD manifestations and was well-tolerated, with median follow-up duration of 22 months (range 9-228 months). When comparing the year prior to treatment to the first year receiving Dojolvi, annual emergency hospital care visits decreased from a mean of 1.12 to 0.17, or an 85% reduction, and the mean number of emergency home care events decreased from 16.82 to 2.83, an 83% reduction. Similarly, the cumulative annual number of days of emergency home care were reduced from 286 in the year prior to receiving Dojolvi to 51 in the first year receiving Dojolvi, an 82% reduction. Further improvements in the cumulative annual number of days of emergency home care were seen during the second year receiving Dojolvi.
The efficacy and safety profile reported under the ATU is consistent with the results from prior reported data from the company-sponsored studies.
GTX-102 for the treatment of Angelman Syndrome, partnered with GeneTx

A pre-application meeting with a national regulatory agency in Europe was held in which the company reviewed the available efficacy and safety data as well as its proposal for dosing and administration of GTX-102. Based on our discussion, the authority agreed in principle on the expansion of the trial to Europe using the proposed modified study design, dosing and administration strategy, pending review of the application. The application to enroll the clinical study in this region was recently submitted.
In Canada, an amendment to the open clinical trial agreement was submitted and included the additional data and updated protocol and is pending review.
Discussions with the FDA are continuing and a request for a meeting has been granted and will take place in the second quarter 2021. The meeting will review additional clinical data requested by the FDA regarding the previously treated patients that confirm that the serious adverse event in the previously treated patients has fully reversed as well as an amended dosing plan for the previously treated patients.
The Phase 1/2 study is expected to resume in 2021, with clinical data on some patients still available before the end of 2021.
DTX401 for the treatment of Glycogen Storage Disease Type Ia, or GSDIa

Ultragenyx completed the Scientific Advice process with the European Medicines Agency (EMA) and an End-of-Phase 2 meeting with the FDA.
The Phase 3 study design has been submitted and endpoints are being finalized with regulators.
The Phase 3 study is expected to initiate early in the second half of 2021.
DTX301 for the treatment of Ornithine Transcarbamylase, or OTC, Deficiency

Late in the first quarter 2021, Ultragenyx met with the FDA in an End-of-Phase 2 meeting where the Phase 3 design and endpoints were finalized. The Phase 3 study will include a 64-week primary efficacy analysis period and enroll approximately 50 patients 12 years of age and older, randomized 1:1 to DTX301 (1.7 x 10^13 GC/kg dose) or placebo. The co-primary endpoints are the change in 24-hour plasma ammonia levels and the percentage of patients who achieve a response as measured by discontinuation or reduction in baseline disease management.
The final Phase 3 study design incorporates Scientific Advice feedback from the EMA.
The Phase 3 study is expected to initiate in the second half of 2021.
UX701 for the treatment of Wilson Disease

During the first quarter 2021, the Investigational New Drug (IND) application cleared FDA review. A seamless, single-protocol Phase 1/2/3 study is expected to begin early in the second half of 2021.
UX053 for the treatment of Glycogen Storage Disease Type III, or GSDIII

The IND application for UX053 for the treatment of GSDIII, the company’s most advanced mRNA program, was cleared by the FDA in March 2021. Enrollment in a Phase 1/2 study is expected to begin in the second half of 2021.
1: Ultragenyx territories include the collaboration revenue from the North American profit share territory and other regions where revenue from product sales are recognized by Ultragenyx. This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.

2: View Source

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Tuesday, May 4, 2021, at 2 p.m. PT/ 5 p.m. ET to discuss the first quarter 2021 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 6883837. The replay of the call will be available for one year.

On May 4, 2021 Seres Therapeutics, Inc., (Nasdaq: MCRB), a leading microbiome company developing a novel class of multifunctional bacterial therapeutics designed to functionally interact with host cells and tissues to treat disease, reported first quarter 2021 financial results and provided business updates (Press release, Seres Therapeutics, MAY 4, 2021, View Source [SID1234579113]).

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"We continue to make steady progress across our broad microbiome pipeline and we look forward to an eventful remainder of the year with important clinical milestones," said Eric Shaff, President and Chief Executive Officer of Seres. "Our SER-287 ECO-RESET Phase 2b study in mild-to-moderate ulcerative colitis patients is fully enrolled and we anticipate topline clinical results in mid-year. Following successful SER-109 Phase 3 pivotal results in patients with recurrent C. difficile infection, we continue to enroll an open label study to expand our safety database. We are very pleased with the pace of study enrollment and we anticipate achieving target enrollment during the third quarter of 2021. Completion of the required SER-109 safety database will support a Biologics License Application (BLA) filing, and potentially pave the way for SER-109 to become the first-ever FDA-approved microbiome therapeutic."

Program and Corporate Updates

SER-109 Phase 3 ECOSPOR III study in recurrent C. difficile infection: SER-109, an investigational oral, live microbiome therapeutic, achieved a high rate of sustained clinical response in Seres’ Phase 3 clinical trial in patients with recurrent C. difficile infection (CDI).

In August 2020, Seres announced positive topline interim results from the SER-109 Phase 3 study, ECOSPOR III. The Phase 3 study (ClinicalTrials.gov identifier: NCT03183128) was a multicenter, randomized, placebo-controlled study that enrolled 182 patients with multiply recurrent CDI. Patients were randomized 1:1 to receive either SER-109 or placebo after standard of care antibiotic treatment. Study results demonstrated that ECOSPOR III achieved the study’s primary endpoint at eight weeks and demonstrated a sustained clinical response rate of approximately 88% at eight weeks post-treatment. SER-109 resulted in a 27% absolute reduction of recurrence of CDI compared to placebo at eight weeks post-treatment, which is a relative risk reduction of 68%. SER-109 was well tolerated, with no treatment-related serious adverse events (SAEs) observed in the active arm and an adverse event profile comparable to placebo. In addition, subsequent 24-week end-of-study data indicate that the clinical effect of SER-109 is durable.

Following the topline Phase 3 study results, the FDA reaffirmed its prior position regarding the efficacy requirements to support a SER-109 BLA submission, which were exceeded by the positive SER-109 ECOSPOR III study results, as well as its prior position that the safety database should be at least 300 subjects. Seres believes that ECOSPOR III will be a single pivotal efficacy study supporting product registration and expects to reach target enrollment for the safety database study in the third quarter of 2021.

In January 2021, Seres presented Phase 3 microbiome and metabolomic endpoint data at the Keystone Symposium, which provide mechanistic support for the SER-109 efficacy observed. The study data demonstrate that SER-109 bacterial species rapidly engraft into the gastrointestinal tract; engraftment was observed as early as one week post-treatment and was durable through eight weeks, confirming the biological activity of SER-109. SER-109 administration was also observed to rapidly shifted the gastrointestinal metabolic landscape, including a significant decrease in primary bile acids and an increase in secondary bile acids, providing a mechanistic basis for inhibition of C. difficile spore germination and vegetative growth.

Seres is conducting an ongoing SER-109 open-label study in patients with recurrent CDI (ClinicalTrials.gov identifier: NCT03183128), which also admits patients with a single recurrence of CDI, to expand the SER-109 safety database. The SER-109 open label study is being enrolled at more than 100 sites across the U.S. and Canada, representing a substantial increase over those that recruited subjects into the SER-109 Phase 3 ECOSPOR III study. Additional information is available at serescdiffstudy.com.

Seres continues to execute activities necessary to enable a SER-109 BLA submission. The Company is completing supportive market assessment work, including primary research with physicians and payers, and pricing and reimbursement analyses. Seres continues to hire, train, and deploy a medical science liaison team in order to increase market education efforts. The Company believes that a substantial commercial opportunity exists for SER-109. The recurrent CDI population includes approximately 170,000 patients in the U.S.

SER-287 Phase 2b ECO-RESET study in ulcerative colitis (UC): SER-287, an oral microbiome therapeutic candidate consisting of a bacterial consortium, is designed to restructure the gastrointestinal microbiome and reduce inflammation in individuals with UC. Seres has obtained FDA Fast Track designation for SER-287 in active mild-to-moderate ulcerative colitis. SER-287 targets restructuring the microbiome to reduce proinflammatory activity and modulate UC-relevant inflammatory pathways, potentially providing a much-needed non-immunosuppressive treatment option to patients suffering from ulcerative colitis. SER-287 has the potential to be used as both a monotherapy and in combination with other approved agents.

The SER-287 Phase 2b ECO-RESET study is a randomized, placebo-controlled, three-arm induction trial that has enrolled 203 patients with active mild-to-moderate ulcerative colitis who have had inadequate response or loss of response on prior therapy. In arm A, patients receive a short course of vancomycin preconditioning followed by ten weeks of the same daily regimen used in the arm of the Phase 1b trial that showed the highest clinical remission rate. In arm B, patients receive vancomycin preconditioning followed by two weeks of the SER-287 daily regimen as in the first arm, followed by eight weeks of a lower dose of SER-287. In arm C, patients receive placebo. The study’s primary endpoint is the proportion of patients achieving clinical remission at ten weeks. The study will assess endoscopic improvement as a secondary endpoint, as well as a range of microbiome and metabolomic measures.

Seres has completed enrollment in the SER-287 Phase 2b ECO-RESET induction study in patients with active mild-to-moderate ulcerative colitis and expects to report topline clinical study results in mid-2021, with additional microbiome biomarker data becoming available in H2 2021.

Publication of SER-287 Phase 1b study results: Data from a Phase 1b study demonstrated that SER-287 administration was associated with high rates of clinical remission, endoscopic improvement, modulation of the gastrointestinal microbiome, and a favorable tolerability profile. The paper, titled "A Phase 1b Safety Study of SER-287, a Spore-Based Microbiome Therapeutic, For Active Mild-To-Moderate Ulcerative Colitis," was published as the highlighted cover article in the January 2021 print edition of the leading journal Gastroenterology.

SER-301 Phase 1b study in adults with mild-to-moderate ulcerative colitis: Seres is enrolling its Phase 1b study for SER-301, an investigational oral, rationally-designed, cultivated microbiome therapeutic, and study enrollment is ongoing. SER-301 is being evaluated in a Phase 1b study in adults with mild-to-moderate ulcerative colitis. The study is being conducted in Australia and New Zealand and is designed to enroll approximately 65 subjects. The study objectives are to evaluate drug safety and pharmacokinetics and to evaluate clinical remission and other measures of efficacy as secondary endpoints.

The consortia of bacteria in SER-301 is designed to modify the microbiome and microbe-associated metabolites in the gastrointestinal tract and modulate pathways linked to gastrointestinal inflammation and epithelial barrier integrity in patients with ulcerative colitis. SER-301 was designed using Seres’ reverse translation discovery and development platforms. The design incorporated learnings from the SER-287 Phase 1b study related to the bacterial species and the microbiome functional signatures associated with clinical efficacy. Additionally, the design incorporated insights on the engraftment dynamics of different bacteria and also the association of specific bacteria with the modulation of inflammatory and immune pathways in human subjects that have been observed across our broader clinical portfolio and confirmed using our nonclinical human-cell based assays and in vivo models.

In December 2020, Seres received a $10 million milestone payment associated with the Phase 1b SER-301 clinical study initiation from Nestlé, the Company’s ex-North American collaborative partner for this program.

SER-155 Phase 1b clinical study activities: SER-155 is an investigational oral, rationally-designed, cultivated microbiome therapeutic designed to reduce the incidence of gastrointestinal infections, bacteremia, and graft versus host disease (GvHD) in immunocompromised patients, including patients receiving allogeneic hematopoietic stem cell transplantation. SER-155 is a consortium of bacterial species selected using microbiome biomarker data and from human clinical data and nonclinical human cell-based assays and in vivo disease models. The composition aims to decrease infection and translocation of antibiotic-resistant bacteria in the gastrointestinal tract and modulate host immune responses to decrease GvHD.

The SER-155 program is supported by a CARB-X grant. Seres continues to advance SER-155 toward a Phase 1b clinical study initiation in collaboration with Memorial Sloan Kettering Cancer Center.

SER-401 program update: Seres, in collaboration with study partners, The Parker Institute for Cancer Immunotherapy and The University of Texas MD Anderson Cancer Center, had announced the voluntary discontinuation of further enrollment in the Melanoma Checkpoint and Gut Microbiome Alteration With Microbiome Intervention (MCGRAW) study evaluating the safety and drug activity of SER-401 or fecal microbiota transplant (FMT) in combination with nivolumab in patients with metastatic melanoma. Seres has deprioritized further development of SER-401, and will prioritize rationally-designed, cultivated leads designed using the Company’s reverse translational platforms and learnings from the SER-401 trial. Future oncology programs aim to modulate host immunity/inflammation to improve response and tolerability of cancer drugs.

Upcoming investor event: Seres plans to hold a webcast and conference call on June 21, 2021 with a focus on the Company’s efforts to develop microbiome therapeutics for ulcerative colitis and the substantial opportunity for safe, effective alternative therapeutics for the over 700,000 individuals (U.S. estimate) suffering from UC. The event will include discussion of the ongoing SER-287 Phase 2b study, and SER-301 Phase 1b study. The Company anticipates topline SER-287 Phase 2b study clinical results in mid-2021, and additional microbiome biomarker data in H2 2021. Further details will be provided closer to the event.

Financial Results

Seres reported a net loss of $35.5 million for the first quarter of 2021, as compared with a net loss of $19.9 million for the same period in 2020. The first quarter net loss was driven primarily by clinical and development expenses, personnel expenses and ongoing development of the Company’s microbiome therapeutics platform.

Research and development expenses for the first quarter of 2021 were $29.3 million, compared with $21.7 million for the same period in 2020. The research and development expenses were primarily related to Seres’ late stage SER-109 and SER-287 clinical development programs.

General and administrative expenses for the first quarter of 2021 were $11.7 million, compared with $6.1 million for the same period in 2020. General and administrative expenses were primarily due to headcount, professional fees and facility costs.

Seres ended the first quarter of 2021 with approximately $272.5 million in cash, cash equivalents and short and long-term investments.

Conference Call Information

Seres’ management will host a conference call today, May 4, 2021, at 8:30 a.m. ET. To access the conference call, please dial 844-277-9450 (domestic) or 336-525-7139 (international) and reference the conference ID number 9967338. To join the live webcast, please visit the "Investors and News" section of the Seres website at www.serestherapeutics.com.

A webcast replay will be available on the Seres website beginning approximately two hours after the event and will be archived for at least 21 days.

On May 4, 2021 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and financial results for the first quarter ending March 31, 2021 (Press release, PTC Therapeutics, MAY 4, 2021, View Source [SID1234579112]).

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"Overall PTC has had a strong performance this quarter through all aspects of the company from discovery to commercial revenue," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "I would like to highlight the continued strong growth of the DMD franchise which has had one of our largest quarterly revenues to date. The other key milestone was the positive preliminary results in our PTC518 Huntington’s disease program demonstrating dose dependent lowering of the HTT mRNA. Analogous to the SMA program we are now well positioned with a clear path for success."

Key First Quarter and Other Corporate Updates:

The Duchenne muscular dystrophy (DMD) franchise had a total net product revenue of $90 million for Translarna (ataluren) and Emflaza (deflazacort) in the first quarter of 2021. This represents 32% growth over the first quarter of 2020 and one of PTC’s strongest quarterly commercial revenues to date.
Broader uptake due to new patients in existing geographies and geographic expansion drove Translarna growth.
Emflaza revenue growth was primarily due to increased new prescriptions, high compliance, and fewer discontinuations.
In March 2021, the European Medicines Agency (EMA) approved Evrysdi (risdiplam) in the European Union (EU). The first sale of Evrysdi in this region was recorded the following day, triggering a $20 million milestone payment to PTC. Evrysdi is a product of a collaboration between PTC, Roche and the SMA Foundation.
Preliminary results from the PTC518 Phase 1 healthy volunteer trial demonstrated dose-dependent reduction of Huntington mRNA beyond the 30-50% target.
PTC received Gallup’s Don Clifton Strengths-Based Culture Award, which reflects the Company’s ongoing deep commitment to its employees.
First Quarter Clinical Updates:

PTC has multiple clinical trials ongoing, three of which are registration-directed clinical studies:
The MIT-E Phase 2/3 trial with vatiquinone for mitochondrial epilepsy with data anticipated in the third quarter of 2022.
The MOVE-FA Phase 3 trial with vatiquinone for Friedreich ataxia with data anticipated in 2023.
The FITE19 Phase 2/3 clinical trial for PTC299 in patients with COVID-19 with an expected data readout in the second half of 2021.
The second Bio-e compound, PTC857 healthy volunteer study was recently completed, and data will be communicated in the second quarter.
The registration-directed Phase 3 PTC923 phenylketonuria (PKU) trial, APHENITY, is expected to initiate in mid-2021.
The Committee for Medicinal Products for Human Use (CHMP) has requested a clock stop in the aromatic L-amino acid decarboxylase (AADC) deficiency review process to allow for completion of its pre-approval inspections, which were delayed due to COVID-19. The CHMP opinion is now anticipated in the third quarter of 2021.
Due to COVID-related surgical delays, the AADC-deficiency biologics license application submission to the U.S. Food and Drug Administration is anticipated to be delayed by at least one quarter.
First Quarter 2021 Financial Highlights:

Total revenues were $117.9 million for the first quarter of 2021, compared to total revenues of $68.3 million for the first quarter of 2020, a 32% increase. Total revenue includes net product revenue of $91.3 million and collaboration and royalty revenue of $26.7 million in the first quarter of 2021.
Translarna net product revenues were $46.5 million for the first quarter of 2021, compared to $40.5 million for the first quarter of 2020. These results reflect an increase in net product sales in existing markets as well as continued geographic expansion into new territories.
Emflaza net product revenues were $43.5 million for the first quarter of 2021, compared to $27.5 million for the first quarter of 2020. These results reflect new patient prescriptions, high compliance, and fewer discontinuations.
Roche reported Evrysdi first quarter 2021 sales of approximately CHF 80 million. During the first quarter of 2021, the first commercial sale of Evrysdi in the EU triggered a $20 million milestone payment to PTC, which was reported as collaboration revenue.
U.S. GAAP (generally accepted accounting principles) R&D expenses were $134.5 million for the first quarter of 2021, compared to $90.1 million for the first quarter of 2020. The increase in R&D expenses reflects costs associated with increased investment in research programs, and advancement of the clinical pipeline.
Non-GAAP R&D expenses were $120.8 million for the first quarter of 2021, excluding $13.7 million in non-cash, stock-based compensation expense, compared to $81.9 million for the first quarter of 2020, excluding $8.2 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $61.1 million for the first quarter of 2021, compared to $58.2 million for the first quarter of 2020. The increase in SG&A expenses was associated with entering into a long-term lease for the Hopewell facility that commenced on July 1, 2020.
Non-GAAP SG&A expenses were $49.1 million for the first quarter of 2021, excluding $12.0 million in non-cash, stock-based compensation expense, compared to $51.2 million for the first quarter of 2020, excluding $7.0 million in non-cash, stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was $0.1 million for the first quarter of 2021, compared to $0.9 million for the first quarter of 2020. 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 $128.6 million for the first quarter of 2021, compared to net loss of $112.7 million for the first quarter of 2020.
Cash, cash equivalents and marketable securities was $988.4 million at March 31, 2021, compared to $1.1 billion at December 31, 2020.
Shares issued and outstanding as of March 31, 2021 were 70,405,905.
PTC Reaffirms Full Year 2021 Guidance as Follows:

PTC anticipates net product revenues for the DMD franchise for the full year 2021 to be between $355 and $375 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2021 to be between $825 and $855 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2021 to be between $725 and $755 million, excluding estimated non-cash, stock-based compensation expense of $100 million.

Today’s Conference Call and Webcast Reminder:

PTC will host a conference call to discuss the first quarter of 2021 corporate updates and financial results 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 4292410. 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.