On August 7, 2018 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the second quarter ended June 30, 2018. In addition, the Company provided a clinical and business update (Press release, Syndax, AUG 7, 2018, View Source [SID1234528501]). As of June 30, 2018, Syndax had $98.4 million in cash, cash equivalents and short-term investments.

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"We made great progress across multiple programs during the first half of this year, including presentation of data from all three cohorts of ENCORE 601 and completion of target enrollment in ENCORE 602 and 603. We also initiated the first combination trial for the SNDX-6352 program, which will evaluate its safety in combination with durvalumab (IMFINZI)," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "We continue to expect the progression free survival results from E2112, our ongoing Phase 3 trial of entinostat plus exemestane in HR+, HER2- breast cancer, later this quarter, and the third prespecified interim analysis of overall survival in November. We also anticipate sharing next steps for the entinostat-KEYTRUDA (pembrolizumab) combination program in both non-small cell lung cancer and melanoma by the end of the year."

The Company also announced today that it plans to initiate a Phase 1 trial of its monoclonal antibody inhibitor of Colony-Stimulating Factor 1 Receptor (CSF-1R), SNDX-6352, in patients with chronic graft versus host disease (cGVHD). Enrollment in this trial is anticipated to begin by the end of the year, with initial data expected in the second half of 2019.

"We are excited to begin the evaluation of SNDX-6352 as a treatment for cGVHD, a novel clinical path for a CSF1-R inhibitor," said Michael L. Meyers, M.D., Ph.D., Chief Medical Officer of Syndax. "Preclinical findings support that CSF-1R inhibition may serve as an effective approach for treating this debilitating, often deadly side effect of allogenic hematopoietic stem cell transplantation. We look forward to learning more about the potential of SNDX-6352 in this indication."

Pipeline Updates

The Phase 3 registration trial of entinostat plus exemestane in advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer, E2112, is 98% enrolled as of the end of July. ECOG-ACRIN Cancer Research Group, the trial sponsor, had notified the Company that the Data Safety Monitoring Committee (DSMC) completed the final progression free survival (PFS) analysis in November 2017. The trial is proceeding as planned, and Syndax continues to anticipate that enrollment will be complete in the third quarter of 2018, at which time the result of the PFS analysis will be released to the Company. In addition, interim overall survival (OS) analyses are scheduled to occur every May and November. Two interim OS analyses have already occurred, with the next analysis expected this November.

The Company presented data from a subset of PD-(L)1 refractory non-small cell lung cancer (NSCLC) patients enrolled in the expanded Phase 2 ENCORE 601 cohort (n = 57) at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June. Updated data from all patients (n = 76) enrolled in this cohort will be presented at the World Conference on Lung Cancer Meeting in Toronto next month, including updated results from the Company’s biomarker analyses. The Company expects to communicate its development plans for entinostat in this indication in the fourth quarter.

The Company presented data from a subset of PD-1 refractory melanoma patients enrolled in the expanded Phase 2 ENCORE 601 cohort (n = 34) at the ASCO (Free ASCO Whitepaper) Annual Meeting in June. Updated results from the full cohort (n = 55) are expected by the end of this year, at which time the Company will make a decision on registration plans for entinostat in this indication.

Enrollment in the expanded stage 1 ENCORE 601 cohort of patients with microsatellite stable colorectal cancer (MSS-CRC, n = 37) is expected to complete in the third quarter. A decision on whether to continue to the second stage of this cohort is expected in the first half of 2019.

Target enrollment in both the Phase 2 portion of ENCORE 602, the Phase 1b/2 clinical trial evaluating the combination of entinostat plus Genentech’s PD-L1 inhibitor, atezolizumab (TECENTRIQ), in patients with triple negative breast cancer, and the Phase 2 portion of ENCORE 603, evaluating entinostat in combination with Pfizer/Merck KGaA’s PD-L1 inhibitor, avelumab (BAVENCIO), in patients with ovarian cancer, is complete. Topline results from each study are anticipated in the first half of 2019.

ENCORE 606, the Phase 1b/2 trial evaluating entinostat in combination with NKTR-214, Nektar’s CD122-biased agonist, is expected to begin enrolling patients in the first half of 2019.

Dosing of patients with solid tumors in the Phase 1/1b trial evaluating the safety of SNDX-6352 continues as planned. Testing of SNDX-6352 in combination with durvalumab (IMFINZI), AstraZeneca’s human monoclonal antibody directed against PD-L1, was recently initiated, and dosing of patients with SNDX-6352 as a monotherapy is ongoing. The Company anticipates identifying the recommended Phase 2 dose and schedule for SNDX-6352 monotherapy and in combination with durvalumab in the first half of 2019.

The Company expects to commence enrollment in a Phase 1 dose escalation trial of SNDX-6352 in patients with cGVHD by the end of the year. The objectives of this trial are to evaluate the safety and preliminary efficacy of SNDX-6352 in cGVHD and to identify a recommended Phase 2 dose and schedule. Initial results are anticipated in the second half of 2019.

Development of the Company’s portfolio of Menin-Mixed Lineage Leukemia (MLLr) inhibitors is ongoing. The Company continues to expect clinical trials to initiate in the first half of 2019.
Second Quarter 2018 Financial Results

As of June 30, 2018, Syndax had cash, cash equivalents and short-term investments of $98.4 million and 22,705,794 shares issued and outstanding.

On June 18, 2018, the Company signed an exchange agreement with Biotechnology Value Fund and certain affiliated funds ("BVF") under which BVF exchanged 2,000,000 shares of common stock for 2,000,000 Warrant Shares. BVF can exercise the Warrant Shares at an exercise price per share equal to $0.0001 per share. The warrant is issued for a period of 20 years.

In the third quarter of 2018, through August 6th, the Company sold 633,231 shares of its common stock with net proceeds of approximately $4.4 million pursuant to its at-the-market arrangement.

Second quarter 2018 research and development expenses increased to $14.9 million from $9.9 million for the comparable period in the prior year. The increases were primarily due to increased activities in manufacturing for SNDX-6352, increased development activities for the Menin-MLLr and ENCORE 602 programs partially offset by completion of pharmacology trials and lower program cost for E2112. Employee compensation increased due to increased headcount.

General and administrative expenses totaled $4.5 million during the second quarter of 2018, compared to $4.3 million for the comparable period in the prior year. The increase in general and administrative expenses was primarily due to increased pre-commercialization activities and increased patent related legal expenses.

For the three months ended June 30, 2018, Syndax reported a net loss attributable to common stockholders of $18.4 million or $0.74 per share compared to $13.6 million or $0.70 per share for the comparable prior year period.

Financial Guidance

Today the Company provided operating expense guidance for the third quarter and full year 2018. For the third quarter and full year 2018, research and development expenses are expected to be $14 to $16 million and $59 to $62 million, respectively, and total operating expenses are expected to be $18 to $20 million and $77 to $81 million, respectively. Total operating expenses for 2018 are expected to include approximately $6 million of non-cash stock compensation expense.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Tuesday, August 7, 2018.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 4980058
Domestic Dial-in Number: 1- 855-251-6663
International Dial-in Number: 281-542-4259
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.

On August 7, 2018 Protagonist Therapeutics, Inc. (Nasdaq:PTGX) reported its financial results for the second quarter ended June 30, 2018 (Press release, Protagonist, AUG 7, 2018, View Source;p=RssLanding&cat=news&id=2362550 [SID1234528527]).

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"Protagonist continues to advance well-differentiated therapeutic candidates discovered through its novel peptide technology platform," commented Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. "We very recently reported results from a comprehensive review of the data from the Phase 2 PROPEL study of PTG-100 for the treatment of moderate to severe active ulcerative colitis. We are pleased to conclude that this drug candidate showed signals of clinical efficacy, has no safety concerns, and warrants further clinical development for potential treatment of ulcerative colitis. We are planning to discuss next steps in the clinical development of PTG-100 with the U.S. Food and Drug Administration (FDA) and other global regulatory authorities in the second half of this year. We are glad to report on the continued progress of the clinical development of PTG-200 in collaboration with our partner Janssen for potential treatment of Crohn’s disease. In addition, we look forward to initiating an open label Phase 2 study in beta-thalassemia patients in the fourth quarter of 2018 with PTG-300, our product candidate for rare blood disorders."

Product Development Update:

PTG-100

In March 2018, Protagonist had announced discontinuation of the Phase 2 PROPEL study following a planned interim analysis conducted by an independent Data Monitoring Committee. The interim data had revealed an unusually high placebo rate of clinical remission (24 percent, approximately four times higher than historical norms for similar UC studies) that led to a futility decision and discontinuation of the trial. A re-read of the endoscopies by the subcontractor of the contract research organization (CRO) and a subsequent fully blinded re-read of the endoscopies by an independent third party, Robarts Clinical Trials, confirmed that a subset of the initial endoscopy reads provided by the CRO were in error. If the re-read of endoscopy results had been utilized for the interim futility analysis, the trial would have continued. Based on this entire analysis, the Company plans to discuss the next steps in advancing the clinical development of PTG-100 with the FDA and other global regulatory authorities in the second half of 2018.
PTG-200

In the fourth quarter of 2018, Protagonist expects a U.S. IND filing by its partner Janssen Biotech that will support the initiation of a global Phase 2 study of PTG-200, an oral, gut-restricted interleukin-23 receptor antagonist peptide in Crohn’s disease patients. This IND filing would trigger a milestone payment from Janssen of $25 million under the existing exclusive license and collaboration agreement between Janssen and Protagonist.
PTG-300

The results of a Phase 1 study in healthy volunteers and supportive pre-clinical data for PTG-300, an injectable hepcidin mimetic peptide, were the subject of oral presentations in June 2018 at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).
Protagonist completed discussions with U.S. and global regulatory agencies and filed a U.S. IND in the second quarter of 2018. The Company plans other global regulatory submissions to support the initiation of a global Phase 2 trial in patients with beta-thalassemia in the fourth quarter of 2018.
Preclinical Programs

The Company presented data related to preclinical product candidates, oral peptide agonists targeting the delta/mu opioid receptors, in a podium presentation at the Digestive Diseases Week conference in June 2018.
Corporate Update – Financing:

Protagonist recently announced the completion of an equity financing with investors including BVF Partners L.P. and their affiliates for gross proceeds of $22 million. Proceeds from the financing will be used to advance development of drug candidate PTG-100.
Financial Results

Protagonist reported a net loss of $8.7 million and $16.3 million, respectively, for the second quarter and first six months of 2018, as compared to a net loss of $15.0 million and $29.1 million, respectively, for the same periods of 2017. The decrease in net loss for both the second quarter and year-to-date periods was driven primarily by license and collaboration revenue recognized during the first and second quarters of 2018, partially offset by increased research and development (R&D) and general and administrative (G&A) expenses. The net loss for the second quarter and first six months of 2018 includes non-cash stock-based compensation of $1.6 million and $2.8 million, respectively, as compared to $1.0 million and $1.8 million, respectively, for the same periods of 2017.

License and collaboration revenue was $11.7 million and $22.5 million for the second quarter and first six months of 2018, respectively, and consisted of revenue from activities performed under the Janssen Collaboration Agreement. Protagonist did not recognize any license and collaboration revenue for the second quarter or first six months of 2017.

R&D expenses for the second quarter and first six months of 2018 were $17.7 million and $33.1 million, respectively, as compared to $12.0 million and $23.3 million, respectively, for the same periods of 2017. The increases in R&D expenses were primarily due to costs related to contract manufacturing and the preparation for and conduct of clinical trials for our product candidates. R&D expenses for the quarter also included an increase in salaries and employee-related expenses due to an increase in R&D personnel.

G&A expenses for the second quarter and first six months of 2018 were $3.2 million and $6.8 million, respectively, as compared to $3.1 million and $6.1 million, respectively, for the same periods of 2017. The increases in G&A expenses were primarily due to increases in salaries and employee-related expenses primarily due to an increase in headcount to support the growth of our operations, partially offset by a decrease in legal expenses.

Protagonist ended the second quarter with $125.2 million in cash, cash equivalents and investments. With the financing announced yesterday, the company expects to have sufficient financial resources to fund operations to mid-2020.

On August 7, 2018 Stony Brook University, iCell Gene Therapeutics, and the University of Louisville, have received Food and Drug Administration (FDA) clearance for an Investigational New Drug (IND) for the treatment of relapsed and refractory T-cell leukemia and lymphoma (Press release, Stony Brook University, AUG 7, 2018, View Source [SID1234528670]). The approach is the first to use chimeric antigen receptor engineered T-cells directed against the target protein CD4 (CD4CAR). Together, Stony Brook University, the University of Louisville, and iCell Gene Therapeutics expect the first in-human Phase I clinical trial to begin accruing patients before the end of 2018.

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"We are excited to partner with the University of Louisville and iCell Gene Therapeutics to offer this innovative first-in-human CAR T cell immunotherapy clinical trial for patients who are suffering from these extremely difficult to treat T cell lymphomas and leukemias," said Huda Salman, MD, Principal Investigator for the IND and an oncologist at Stony Brook University Cancer Center. "CD4CAR T cells may prove to be a promising and novel therapy in this setting."

"The development of this trial using CD4 as a target is the first of what we expect to be many CAR T-based clinical trials available to our patients over time," said Yusuf Hannun, MD, Director of the Stony Brook University Cancer Center. "The pending trial is an example of the type of bench-to-bedside research that is building up at Stony Brook due to the growing expertise and collaborative research environment we are creating and new opportunities that will emerge upon the opening of our Medical and Research Translation (MART) Building."

William Tse, MD, FACP, Chief of the Blood and Marrow Transplantation at the University of Louisville School of Medicine, is the Co-PI of the CD4CAR clinical trial at University of Louisville site.

About CD4CAR

CD4CAR is a novel development for the treatment of CD4+ T-cell malignancies. The CD4-redirected chimeric antigen receptor (CAR) T-cells are engineered to express an anti-CD4scFV antibody domain. CD4CAR has received Orphan Drug Designation by the FDA for Peripheral T cell Leukemia (PTCL) in 2016. Over the past few years, CAR T-cell therapy has proven its efficacy in clinical trials for various types of leukemia, lymphoma, and myeloma. CAR T-cell therapy is a type of adoptive immunotherapy. A CAR-engineered T-cell is genetically modified to express a protein on its surface with the capability to bind to a target protein on another cell. The CD4CAR is manufactured from the patient’s own cells to target CD4 expressed on tumor cells. Once these cells are infused back into a patient’s body through an IV, they multiply and attack tumor cells efficiently throughout the body.

About T-cell leukemias and lymphomas

Although there are clinical development programs ongoing with CAR T-cells for B-cell hematological malignancies, CD4 positive T-cell malignancies (T-cell lymphomas (TCLs) and T–cell acute lymphoblastic leukemia (T-ALLs)), have not been targeted by a CAR therapy in a human trial. TCLs account for 15–20 % of all non-Hodgkin’s lymphomas (NHLs), while T-ALLs affect about 25% of ALLs in adults. These malignant entities are significantly more difficult to treat in comparison to B-cell malignancies. Furthermore, T-cell malignancies almost exclusively have poorer outcomes with few exceptions, lower response rates, shorter times of disease control and survival. As a result, the standard of care for T-cell malignancies is not well-established and the only potential curative approach is allogeneic blood and marrow transplant (BMT) for which patients need to achieve complete disease control and to have suitable marrow donors. This leaves many patients with no curative options.

On August 7, 2018 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported the positive results from the lead-in period of the GENESIS trial, a double-blind, placebo-controlled Phase 3 trial comparing BL-8040 in combination with granulocyte colony-stimulating factor (G-CSF), to G-CSF alone, for the mobilization of hematopoietic stem cells (HSCs) used for autologous transplantation in multiple myeloma patients (Press release, BioLineRx, AUG 7, 2018, View Source;p=RssLanding&cat=news&id=2362414 [SID1234528751]).

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The open-label, single-arm, lead-in period of the study was designed to include up to 30 patients, with Data Monitoring Committee (DMC) review after completion of approximately 10, 20 and 30 patients, in order to assess safety and efficacy following treatment with BL-8040 plus G-CSF. Results of the first 11 patients show that BL-8040 in combination with standard G-CSF treatment is safe and tolerable. In addition, the data show that 9/11 patients (82%) reached the primary endpoint threshold of ≥ 6×106 CD34 cells/kg with only one dose of BL-8040 and in up to 2 apheresis sessions. Furthermore, 7/11 patients (64%) reached the threshold of ≥ 6×106 CD34 cells/kg in a single apheresis session only. These data demonstrate the potential of BL-8040 treatment to reduce the number of administrations and apheresis sessions, as well as hospitalization costs, related to the preparation of multiple myeloma patients for autologous HSC transplantation.

"Autologous HSC transplantation following high-dose chemotherapy has significantly improved outcomes for multiple myeloma patients," said Dr. John F. DiPersio, Chief, Division of Oncology at the Washington University School of Medicine, and lead investigator of the study. "Current practice involves mobilizing HSCs from the bone marrow to the peripheral blood, after which the cells are collected by apheresis. Results so far show that mobilizing HSCs with a single BL-8040 dose combined with G-CSF is not only safe and tolerable, but also demonstrates robust efficacy regarding the number of collected cells, and may reduce the number of required apheresis sessions to a single session in the majority of patients. This is a very encouraging result that, if corroborated in the placebo-controlled part of the trial, will be of great value to patients as well as to the medical community. I am looking forward to participating in the trial and to potentially improving the quality of treatment available to multiple myeloma patients."

"We are extremely encouraged by these results. Based on the robust data received from the first 11 patients, the DMC issued a positive recommendation to stop the lead-in part of the study and move immediately to the randomized placebo-controlled part of the study," stated Philip Serlin, Chief Executive Officer of BioLineRx. "This is the first Phase 3 trial for our lead BL-8040 program, and as such, it is an important milestone in BL-8040’s comprehensive development plan. We look forward to the top-line results from the randomized, double-blind, placebo-controlled part of the study, which are expected in 2020."

About the GENESIS Study

The GENESIS study is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of BL-8040 in combination with G-CSF, compared to placebo and G-CSF, for the mobilization of CD34 HSCs for autologous transplantation in multiple myeloma patients. The placebo-controlled part is designed to include 177 patients in more than 25 centers. Treatment will include 5 days of G-CSF, with a single dose of BL-8040 or placebo on day 4 with the option to expand treatment to up to 8 days of G-CSF and up to 2 days of BL-8040. Apheresis for collection of CD34 cells will be performed on day 5. An additional 3 apheresis sessions may be conducted if needed in order to reach the goal of ≥ 6×106 mobilized CD34 cells/kg.

The primary objective of the study is to demonstrate the superiority of a single dose of BL-8040 in combination with G-CSF, over placebo and G-CSF, in the mobilization of ≥ 6×106 CD34 cells/kg in up to 2 apheresis sessions, in preparation for autologous stem cell transplantation in multiple myeloma patients. Secondary objectives include time to engraftment of neutrophils and platelets, durability of the engraftment, as well as safety and other efficacy parameters.

About BL-8040

BL-8040 is a short synthetic peptide for stem cell mobilization and for treatment of hematological malignancies and solid tumors. It functions as a high-affinity best-in-class antagonist for CXCR4, a chemokine receptor that is directly involved in the retention of stems cells in the bone marrow, as well as tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity.

HSCs express CXCR4 and are retained in the protective bone marrow niche via binding to CXCL12 (also known as SDF-1). Blocking of the CXCR4-SDF1 interaction by BL-8040 leads to the mobilization of HSCs into the peripheral blood. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of HSCs.

Furthermore, BL-8040 induce mobilization of leukemic cells and immune-cells from the bone marrow, thereby sensitizing leukemic cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing cell death (apoptosis). BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

On August 7, 2018 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the second quarter ending June 30, 2018 (Press release, PTC Therapeutics, AUG 7, 2018, View Source [SID1234528767]).

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"We have made significant progress towards delivering on our strategic plan this year," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "This includes furthering our underlying business as well as executing on two external transactions. We look forward to bringing value to both patients and shareholders with the acquisition of Agilis, the LATAM commercialization rights for Tegsedi and Waylivra and realizing additional growth in the second half of the year."

Second Quarter Financial Highlights:

Total revenues for the second quarter of 2018 were $68.7 million, compared to $48.0 million in the same period in 2017. The change in total revenue was a result of Emflaza, which launched in May 2017, and the expanded commercialization of Translarna.

Translarna net product revenues were $47.8 million for the second quarter of 2018, representing 4% growth over $45.8 million reported in the second quarter of 2017.

Emflaza net product revenues were $20.3 million for the second quarter of 2018, from $2.1 million reported in the second quarter of 2017.

GAAP R&D expenses were $32.6 million for the second quarter of 2018, compared to $30.8 million for the same period in 2017. Non-GAAP R&D expenses were $28.7 million for the second quarter of 2018, excluding $3.9 million in non-cash, stock-based compensation expense, compared to $26.9 million for the same period in 2017, excluding $3.9 million in non-cash, stock-based compensation expense. The increase in GAAP and non-GAAP R&D expense was primarily due to increased investment in research programs and advancement of the clinical pipeline.

GAAP SG&A expenses were $33.5 million for the second quarter of 2018, compared to $28.9 million for the same period in 2017. Non-GAAP SG&A expenses were $29.4 million for the second quarter of 2018, excluding $4.1 million in non-cash, stock-based

compensation expense, compared to $24.9 million for the same period in 2017, excluding $4.0 million in non-cash, stock-based compensation expense. The increase in GAAP and non-GAAP SG&A expense was primarily due to continued investment in commercial activities to support the Duchenne muscular dystrophy franchise.

Net loss for the second quarter of 2018 was $9.5 million, compared to a net loss of $17.5 million for the same period in 2017.

In April 2018, PTC completed a public offering of 4,600,000 shares of common stock, resulting in net offering proceeds of $117.9 million.

Cash, cash equivalents, and marketable securities totaled approximately $296.1 million at June 30, 2018, compared to approximately $191.2 million at December 31, 2017.

Shares issued and outstanding as of June 30, 2018 were 46.7 million.

2018 Guidance

Full year 2018 net product revenues are anticipated to be between $260 and $295 million. PTC anticipates Translarna net product revenue for the full year 2018 to be between $170 and $185 million. PTC projects a 5-year (December 31, 2022) compound annual growth rate of 15% for net product revenues, representing continued strong growth year-over-year by increasing penetration in current countries and pursuing opportunities for label expansion. PTC anticipates Emflaza net product revenue for the full year 2018 to be between $90 and $110 million.

GAAP R&D and SG&A expense for the full year 2018 is anticipated to be between $280 and $290 million.

Non-GAAP R&D and SG&A expense for the full year 2018 is anticipated to be between $250 and $260 million, excluding estimated non-cash, stock-based compensation expense of approximately $30 million.

Key Second Quarter and Other Corporate Highlights:

Agreement to acquire Agilis Biotherapeutics gene therapy platform and four CNS clinical assets diversify and strengthen PTC’s existing pipeline. Following successful completion of the transaction, PTC plans a smooth transition of operations and to rapidly accelerate the programs. PTC plans to submit a Biologics Licensing Application with FDA in 2019 for the lead program in AADC deficiency, based on compelling long-term clinical data. An IND submission to the U.S. FDA is expected in 2019 for the second lead program in Friedreich ataxia. The transaction is expected to close in the third quarter of 2018.

In-licensed LATAM commercial rights to Tegsedi (inotersen) and Waylivra (volanesorsen) from Akcea Therapeutics. The collaboration leverages its global commercial infrastructure and adds to growing commercial pipeline. Tegsedi has received marketing authorization approval from the European Commission for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). PTC estimates that there are approximately 6,000 patients in LATAM and that it is well positioned to file for registration in key countries in LATAM for Tegsedi. Tegsedi has a PDUFA date of October 6, 2018. Waylivra is under

regulatory review in the U.S., Europe and Canada for the treatment of people with familial chylomicronemia syndrome (FCS). Waylivra recently received a positive vote from the FDA’s Division of Metabolism and Endocrinology Products Advisory Committee and has a PDUFA date of August 30, 2018. Waylivra is also in clinical development for Familial Partial Lipodystrophy, or FPL.

Compelling data from FIREFISH study and advancement of SMA clinical programs. Data presented at the CureSMA conference demonstrated that at Day 182, over 90% of the babies in the FIREFISH study achieved a greater than 4-point increase in CHOP-INTEND score compared to baseline. The CHOP-INTEND data were further supported by video presented by the investigator in which babies demonstrated improved motor function including head control, rolling from supine, and sitting. Three babies were depicted sitting independently. Updated SMA data including additional sitting babies from part 1 of the FIREFISH study will be presented at the upcoming World Muscle Congress. Part 2 of the pivotal FIREFISH study is ongoing. SUNFISH, a pivotal trial in Type 2/3 SMA patients is also ongoing.

European Commission ratifies CHMP’s positive opinion for the label expansion of Translarna for children as young as 2 years of age. The authorization allows PTC to market Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged two years and older in the 28 countries that are Member States of the European Union, as well as European Economic Area members Iceland, Liechtenstein and Norway.

Focus on intensifying the conversion of Emflaza bridge patients. There are currently hundreds of patients who have been prescribed Emflaza for whom commercial shipments were not yet made. PTC plans to intensify the efforts and programs to expedite the conversion of those prescriptions to commercial shipment in the second half of the year.

Publication of additional data demonstrating the clinically differentiated benefit of deflazacort. The results published in Muscle and Nerve demonstrated Duchenne muscular dystrophy patients treated with deflazacort had notably less decline from baseline in 6-minute walk distance at Week 48 than those treated with prednisone/prednisolone. The extrapolated time to loss of ambulation was 8.58 years for deflazacort and 4.74 years with prednisone/prednisolone. In addition to the time to delay of loss of ambulation, patients on deflazacort demonstrated a slowing of disease progression as measured by physical function endpoints.

Non-GAAP Financial Measures
In this press release, the financial results and financial guidance of PTC are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude stock-based compensation expense. This non-GAAP financial measure is provided as a complement to financial measures reported in GAAP because management uses this non-GAAP financial measure when assessing and identifying operational trends. In management’s opinion, this non-GAAP financial measure is 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. Quantitative reconciliations of non-GAAP financial measures to their closest equivalent GAAP financial measures are included in the tables below.

Today’s Conference Call and Webcast Reminder:
Today’s conference call will take place 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 8284468. A live, listen-only webcast of the conference call and corresponding slides 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 two weeks. PTC’s current Investor Presentation and R&D Day slides are available at the same website location.