On August 6, 2019 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the Phase 1 study of its recombinant crisantaspase molecule, JZP-458, met its efficacy and safety objectives (Press release, Jazz Pharmaceuticals, AUG 6, 2019, View Source [SID1234538231]). The company plans to initiate a single-arm, pivotal Phase 2/3 study evaluating JZP-458 as a potential treatment option for patients with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) who are hypersensitive to E. coli-derived asparaginase products.

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"Jazz is committed to the ALL patient community, and we are pleased to advance this development program with the goal of bringing a new treatment option to ALL and LBL patients who are hypersensitive to E. coli-derived asparaginase products as soon as possible," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development of Jazz Pharmaceuticals. "Following a meeting with the U.S. Food and Drug Administration, we are finalizing the Phase 2/3 study protocol in collaboration with the Children’s Oncology Group and plan to initiate the study later this year."

A recombinant crisantaspase Phase 1 study in healthy volunteers in the U.S. met safety and efficacy objectives with efficacy based on measurement of serum asparaginase activity levels. Results of this Phase 1 study will be submitted for presentation at an upcoming medical meeting.

About JZP-458
JZP-458 is a recombinant crisantaspase that uses a novel Pseudomonas fluorescens expression platform. It is being developed for use as a component of a multi-agent chemotherapeutic regimen in the treatment of pediatric and adult patients with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) who are hypersensitive to E. coli-derived asparaginase products. A Phase 1 study of healthy volunteers was recently completed, and a single-arm, pivotal Phase 2/3 study is planned for initiation later in 2019.

About Acute Lymphoblastic Leukemia (ALL)
Acute Lymphoblastic Leukemia (ALL) is a cancer of the blood and bone marrow that can progress quickly if not treated.1 Leukemia is the most common cancer in children, and about three out of four of these cases are ALL.2 Adults can also develop ALL, and about four of every 10 cases of ALL diagnosed are in adults.3 The American Cancer Society estimates that almost 6,000 new cases of ALL will be diagnosed in the U.S. in 2019.4 Asparaginase is a core component of multi-agent chemotherapeutic regimens in ALL.5 However, asparaginase treatments derived from E. coli are associated with the development of hypersensitivity reactions.6

About Jazz Pharmaceuticals plc
Jazz Pharmaceuticals plc (Nasdaq: JAZZ), a global biopharmaceutical company, is dedicated to developing life-changing medicines for people with limited or no options. As a leader in sleep medicine and with a growing hematology/oncology portfolio, Jazz has a diverse portfolio of products and product candidates in development, and is focused on transforming biopharmaceutical discoveries into novel medicines. Jazz Pharmaceuticals markets Sunosi (solriamfetol), Xyrem (sodium oxybate) oral solution, Defitelio (defibrotide sodium), Erwinaze (asparaginase Erwinia chrysanthemi) and Vyxeos (daunorubicin and cytarabine) liposome for injection in the U.S. and markets Defitelio (defibrotide), Erwinase and Vyxeos 44 mg/100 mg powder for concentrate for solution for infusion in countries outside the U.S. For country-specific product information, please visit www.jazzpharmaceuticals.com/medicines. For more information, please visit www.jazzpharmaceuticals

On August 6, 2019 XOMA Corporation (Nasdaq: XOMA), reported its second quarter 2019 royalty asset portfolio highlights and financial results (Press release, Xoma, AUG 6, 2019, View Source [SID1234538182]).

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"The second quarter began with our latest acquisition of rights to future milestones and royalties associated with five development-stage anti-thrombotic hematology assets from Aronora, Inc. Of the five assets, three are being developed under a collaboration with Bayer, a global leader in hematology therapeutics," stated Jim Neal, Chief Executive Officer at XOMA. "We believe we are well-positioned to continue executing on our royalty-aggregator strategy to create near- and long-term value for shareholders."

Second Quarter 2019 Updates About Partnered Assets in Development
"The quarter also was marked with three notable developments within XOMA’s portfolio of partnered assets. Novartis made two important iscalimab data presentations and began dosing patients in a gevokizumab oncology study. Sesen Bio announced its agreement with the U.S. Food and Drug Administration to move forward on a Biologics License Application for Vicinium," Mr. Neal added. "As well, we received notification that clinical programs are being launched for several additional assets to which XOMA holds a royalty interest."

Novartis and iscalimab
During a late-breaker session at the American Transplant Congress held in June, Novartis presented data on iscalimab (CFZ533) that showed 60% of iscalimab-treated transplant patients have normal kidney histology at least one year after transplant, compared with 0% with tacrolimus (current standard of care)1. Less than half of donated kidneys last 10 years, so durability is a significant unmet need for patients who are living with or waiting for a transplant2. More than 100,000 patients are on the U.S. kidney transplant waiting list with a chronic shortfall of donors3. Iscalimab could prove to be a valuable new option for these transplant patients.

Data from a separate iscalimab study were presented at the European Congress of Rheumatology 2019. The presentation, titled Subcutaneous Dosing of the Novel Anti-CD40 Antibody Iscalimab Achieves Target Drug Exposure and Clinical Efficacy in Primary Sjögren’s Syndrome; Results of a Phase IIa Randomized Open Label Two Arm Parallel Group Trial, concluded the study results further support the safety and efficacy of iscalimab in primary Sjögren’s syndrome and the suitability of subcutaneous dosing for future development. From a clinical point of view, the ability to treat patients with a subcutaneous formulation would help reduce the ever-increasing demands on infusion clinics.

Novartis and gevokizumab
Gevokizumab (VPM087), an anti-IL1β monoclonal antibody that XOMA discovered and initially developed, is now actively progressing in a Novartis oncology development program. Recently the first patient was dosed with gevokizumab in the dose-finding portion of a study in combination with standard of care anti-cancer therapies in patients with metastatic colorectal cancer, metastatic gastroesophageal cancer, and metastatic renal cell carcinoma.

Sesen Bio, Inc.
Sesen Bio had a successful pre-Biologics License Application (BLA) meeting with the U.S. Food and Drug Administration (FDA) regarding Vicinium for the treatment of patients with high-risk, Bacillus Calmette-Guérin unresponsive, non-muscle invasive bladder cancer. Sesen has stated it intends to begin filing its BLA in the fourth quarter of 2019.

Other partnered assets in development
Multiple clinical studies were initiated with assets for which XOMA holds a royalty interest. Takeda expanded its TAK-079 development program with a Phase 1 study to evaluate subcutaneous TAK-079 added to standard of care regimens in participants with newly diagnosed multiple myeloma. Molecular Templates announced the FDA accepted its Investigational New Drug application for TAK-169, an engineered toxin body targeting CD38. Molecular Templates and its partner, Takeda, expect to initiate an open-label Phase 1 dose escalation and expansion study in relapsed/refractory multiple myeloma patients. Rezolute, Inc., disclosed it has resumed its Phase 1 study for AB101 and dosed the first patient in the study’s second cohort. Aronora initiated a new study to evaluate the safety and efficacy of ProCase (AB002) in patients with end stage renal disease on chronic hemodialysis.

Additionally, AVEO Oncology announced positive results from the investigator-initiated Phase Ib Ficlatuzumab-Cytarabine Trial In Patients With Relapsed And Refractory Acute Myeloid Leukemia study. Data showed six of 12 patients who received ficlatuzumab and cytarabine at the maximally tolerated dose achieved a complete response. Study results were presented in a poster session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019.

Business Highlights
Acquired the rights to potential royalty payments and a portion of the potential milestone payments associated with five anti-thrombotic hematology assets from Aronora, Inc. Three of the assets are covered by a collaboration with Bayer, a global leader in hematology therapeutics. Two of the collaboration assets are in early to mid-stages of development and the third is a Phase 2 candidate that is subject to an option. The Company also agreed to acquire the rights to potential royalty payments and a portion of the potential upfront and milestone payments associated with two unpartnered hematology programs from Aronora. XOMA made a $6 million payment and will make an additional payment of up to $3 million to Aronora upon fulfillment of certain other conditions. In return, XOMA will receive, on average, low single-digit royalties on future sales of these five products and 10% of the milestones associated with each of the assets. In addition, the Company could pay Aronora sales-based milestones on each asset if XOMA’s royalty receipts related to each program exceed certain thresholds.

"From our legacy history with our phage display platform, we understand the value of a technology platform. It serves as an engine that can generate multiple product candidates, all of which have the potential to produce milestone and royalty revenues. During the second quarter, we added a second technology platform relationship to our royalty interest portfolio. Sonnet BioTherapeutics, Inc., has a platform and pipeline that were developed using XOMA’s phage display technology," concluded Mr. Neal. Sonnet has developed a Fully Human Albumin Binding platform focused on enhancing half-life and tumor targeting to enhance immune response to improve cancer survival. Its approach is unique in that their team is developing bi- and tri-functional therapies to stimulate and/or block immune-modulating targets. Sonnet believes its technology can activate and sustain an immune response selectively against the targeted tumor. XOMA will receive milestones and a low single-digit royalty on future commercial sales of any therapeutic product that utilizes licensed XOMA intellectual property.

Financial Results
XOMA recorded total revenues of $1.0 million for the second quarter of 2019, compared with $2.3 million recorded for the second quarter of 2018. The decrease for the three months ended June 30, 2019, as compared to the same period in 2018, was due primarily to $1.8 million recognized under the Company’s license agreement with Rezolute in the second quarter of 2018.

Research and development expenses were $0.7 million for the second quarter of 2019, compared to $0.4 million for the second quarter of 2018. The increase of $0.3 million for the three months ended June 30, 2019, compared to the same period of 2018, was primarily due to a $0.5 million pass-through license fee incurred based on the achievement of a development milestone by one of our partners, partially offset by a $0.1 million decrease in salary and related expenses.

General and administrative expenses were $4.9 million for the second quarter of 2019, compared to $4.4 million for the second quarter of 2018. The increase of $0.5 million for the three months ended June 30, 2019, as compared to the same period of 2018, was primarily due to increases of $0.3 million in stock-based compensation expenses and $0.2 million in legal and accounting expenses.

In the second quarter of 2019, XOMA recorded $0.4 million in total interest expense, as compared to $0.2 million in the corresponding period of 2018. The increase in interest expense compared with 2018 is primarily due to the outstanding Silicon Valley Bank (SVB) loan balance. In the second quarter, XOMA borrowed an additional $3.0 million under the SVB loan agreement, and as of June 30, 2019, a total of $10.5 million was outstanding.

Total other income, net was $1.1 million for the second quarter of 2019, compared to other expense of $1.2 million for the second quarter of 2018. During the three months ended June 30, 2019, the fair value of the long-term equity securities held by XOMA increased by $31,000. During the three months ended June 30, 2019, XOMA was party to four sublease agreements, compared with two sublease agreements for the same period in 2018, resulting in $0.8 million in sublease income during the second quarter of 2019 and $0.4 million in the corresponding period of 2018. In the second quarter of 2018, XOMA recognized $1.0 million of other income under the agreement with Ology Bioservices related to the disposition of XOMA’s biodefense business in March 2016; no further payments are due.

Net loss for the second quarter of 2019 was $4.1 million, compared to $1.9 million for the second quarter of 2018.

On June 30, 2019, XOMA had cash and cash equivalents of $42.3 million. The Company ended December 31, 2018, with cash and cash equivalents of $45.8 million. The Company’s current cash and cash equivalents are expected to be sufficient to fund its operations for multiple years.

Tom Burns, Chief Financial Officer of XOMA, commented, "XOMA congratulates Rezolute for its recent announcement that its investors have exercised their full combined $20 million option to purchase shares of Rezolute common stock. In conjunction with this, we recently received $2.9 million from Rezolute."

On August 6, 2019 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the second quarter ended June 30, 2019 and provided a corporate update (Press release, BioCryst Pharmaceuticals, AUG 6, 2019, View Source [SID1234538200]).

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"We have just returned from the 2019 HAEA National Patient Summit with more than 1,000 attendees from the U.S. and around the world. Patients’ excitement about BCX7353 was resounding as they told us an oral option with our safety and efficacy profile could change their lives and the lives of their family members with HAE. Our customers want our product, and we cannot wait to deliver it to them next year," said Jon Stonehouse, president and chief executive officer of BioCryst.

"We are on-track to submit an NDA to the FDA in the fourth quarter, followed by regulatory submissions in Europe and Japan in the first quarter of 2020. We are also preparing for the commercial launch of BCX7353 in the U.S. later in 2020, and we look forward to seeing informative clinical data with our oral Factor-D inhibitor, BCX9930, next quarter," Stonehouse added.

Upcoming Key Milestones

HAE Program – BCX7353

Submit a new drug application (NDA) for oral once-daily BCX7353 for the prevention of HAE attacks with the FDA (Q4 2019)

Submit a marketing authorization application for oral once-daily BCX7353 for the prevention of HAE attacks with the European Medicines Agency (EMA) and Japanese Pharmaceuticals and Medical Devices Agency (PMDA) (Q1 2020)

The company now plans to begin ZENITH-2, a Phase 3 clinical trial of oral BCX7353 (750 mg) for the acute treatment of HAE in 2020, pending the completion of its interactions with regulators on the Phase 3 program and additional CMC formulation work on the acute oral formulation. The company had previously planned to begin ZENITH-2 this summer.
Complement Oral Factor D Inhibitor Program – BCX9930

Report results from ongoing Phase 1 trial of BCX9930 (Q4 2019). The Phase 1 data will inform plans for a proof of concept study in PNH patients in 2020.
ALK-2 Inhibitor Program – BCX9250

Begin a Phase 1 clinical trial of BCX9250, an oral ALK-2 kinase inhibitor for treatment of FOP, in healthy subjects (2H 2019)
Recent Corporate Developments

On July 1, 2019, the company announced it had appointed Megan Sniecinski as chief business officer.
On June 27, 2019, the company announced it had begun enrollment of a Phase 1 trial of BCX9930, an oral Factor D inhibitor discovered and developed by BioCryst, for the treatment of complement-mediated diseases. The trial will evaluate the safety and tolerability and characterize the pharmacokinetic and pharmacodynamic profiles of BX9930 in single and multiple ascending doses of BCX9930 in healthy subjects.
On May 21, 2019, the company announced the successful outcome of APeX-2, a Phase 3 randomized, double-blind, placebo-controlled trial of once-daily, oral BCX7353 for the prevention of hereditary angioedema (HAE) attacks.
On May 10, 2019, the company announced the successful outcome of a randomized, placebo-controlled Phase 1 clinical trial to evaluate intravenous (IV) galidesivir in healthy volunteers.
Second Quarter 2019 Financial Results

For the three months ended June 30, 2019, total revenues were $1.4 million, compared to $12.5 million in the second quarter of 2018. The decrease was primarily due to $7.0 million of deferred revenue and a $5.0 million milestone recognized in the second quarter of 2018, both associated with the EMA’s approval of peramivir (ALPIVABTM).

Research and development (R&D) expenses for the second quarter of 2019 increased to $27.7 million from $21.0 million in the second quarter of 2018, primarily due to increased spending as our HAE programs have progressed and our complement-mediated diseases program entered clinical testing. In addition, the company began recognizing stock option expense for two tranches of performance-based options totaling approximately $2.0 million of expense in the second quarter of 2019. While this expense is allocated to both R&D and G&A, it had a more meaningful impact on R&D expenses for the quarter.

General and administrative (G&A) expenses for the second quarter of 2019 decreased to $8.7 million, compared to $9.5 million in the second quarter of 2018. The decrease was primarily due to a $4.9 million reserve recorded in the second quarter of 2018 for concern regarding the collectability of the EMA approval milestone for peramivir, as well as merger-related costs. These decreases were partially offset by an overall increase in G&A expenses as we prepare for the commercial launch of BCX7353 and an increase in legal costs associated with our ongoing Seqirus UK Limited (Seqirus) dispute.

Interest expense was $3.0 million in the second quarter of 2019, compared to $2.2 million in the second quarter of 2018. The increase was primarily associated with enhancements to the company’s secured credit facility in July 2018 and February 2019.

Net loss for the second quarter of 2019 was $37.6 million, or $0.34 per share, compared to a net loss of $18.5 million, or $0.19 per share, for the second quarter of 2018.

Cash, cash equivalents and investments totaled $97.5 million at June 30, 2019, and reflect a decrease from $128.4 million at December 31, 2018. Operating cash use for the second quarter of 2019 was $26.3 million. Net operating cash use for the first six months of 2019 was $53.4 million as compared to $41.3 million for the first six months of 2018.

Financial Outlook for 2019

BioCryst continues to expect net operating cash use to be in the range of $105 to $130 million, and its operating expenses to be in the range of $120 to $145 million. The company’s operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the company’s stock, as well as by the vesting of the company’s outstanding performance-based stock options. Although not in our operating expense guidance above, approximately $3.5 million of stock option expense will be recognized in the remaining two quarters of 2019 associated with the two tranches of performance-based options mentioned above.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 8954869. A live webcast of the call will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 8954869.

On August 6, 2019 Ribon Therapeutics, a clinical stage biotechnology company, developing first-in-class therapeutics targeting novel enzyme families activated under cellular stress conditions, reported the appointments of Sudha Parasuraman, M.D., and Edward "Tad" Stewart to the newly created positions of Chief Medical Officer (CMO) and Chief Business Officer (CBO), respectively (Press release, Ribon Therapeutics, AUG 6, 2019, View Source [SID1234538216]). Dr. Parasuraman is a board-certified hematologist-oncologist with more than 20 years in the healthcare industry and broad experience across the spectrum of oncology drug development. Mr. Stewart brings more than 20 years of executive experience in the biotechnology area with a focus on business development, strategy and commercialization.

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"Sudha has had a distinguished career as a successful drug developer in the biopharmaceutical industry with expertise in leading clinical strategy, design and management of novel oncology programs, experience that aligns perfectly with our goal to bring first-in-class therapies to patients in need," said Victoria Richon, Ph.D., President and Chief Executive Officer, Ribon Therapeutics. "Tad brings to Ribon significant business development and strategy experience, perfectly suited to help us maximize the opportunities we see with our platform. On behalf of our entire team, I want to welcome both Sudha and Tad and we look forward to their contributions."

"Ribon has pioneered a completely novel area of human biology and rapidly used translational methods to bring those discoveries forward. Their lead program RBN-2397 is a testament to this approach and is now set to enter the clinic in the third quarter of this year," said Dr. Parasuraman. "This has established Ribon as the leader in developing potential first-in-class therapeutics that target stress response pathways. When activated, these enzymes are pivotal in disease progression and survival, making them very suitable for therapeutic intervention. I’m excited to join the Ribon team on their journey in bringing this exciting science to patients."

"Ribon’s platform enables thorough interrogation of stress response pathways, an expanding set of targets and rapid candidate generation," said Mr. Stewart. "Ribon has identified multiple monoPARP and NADase targets in oncology, neuro-degeneration and inflammatory diseases, and has tremendous capabilities to rapidly develop potent and selective inhibitors to target them. The speed with which the company has been able to execute on bringing their lead program toward the clinic is impressive and I look forward to helping expand the potential value of that platform for the company and for patients."

Dr. Parasuraman brings more than 20 years of industry and academic practice experience in hematology, oncology, and pediatrics to her work at Ribon. Most recently she served as CMO for X4 Pharmaceuticals, where she led their immuno-oncology and rare disease programs. Prior to X4, she was VP, Global Medical Affairs at uniQure Inc, where she built and led a team developing gene therapies for rare diseases. Prior to uniQure, she held senior positions at Novartis, where she led the early development program for the CDK4/6 inhibitor, ribociclib, and helped pave the way for accelerated registration studies. She was responsible for leading innovative clinical trials in the Signature Program and the commercial strategies for early-stage programs. Dr. Parasuraman also served as Medical Director at Millennium Pharmaceuticals (now Takeda Oncology) where she was the medical lead for various programs including the clinical development and lifecycle management of the first-in-class cancer therapy, VELCADE. Before joining industry, Dr. Parasuraman was a faculty member at Harvard Medical School and staff physician at Children’s Hospital of Boston, Dana Farber Cancer Institute, and Brigham and Women’s Hospital. Dr. Parasuraman completed her fellowship in pediatric hematology-oncology at St. Jude Children’s Research Hospital in Memphis, TN, and pediatric residencies at Children’s Hospital of Michigan and Los Angeles County/University of Southern California Medical Center. She received her medical degree from the University of Madras, India.

Mr. Stewart has more than 20 years of strategic biopharmaceutical business development experience, including over fifteen years at Merrimack Pharmaceuticals. During his time at Merrimack, Tad served in various roles as part of the executive leadership team, including leading the company’s business development efforts and heading the company’s Commercial Business Unit. Tad’s experience at Merrimack spanned its growth from a pre-clinical organization to a clinical development company and, ultimately, to a commercial enterprise, including the successful launch of a marketed oncology product (ONIVYDE) and the execution of several strategic transactions that supported the long-term growth of the company. Most recently he served as President and CEO of Commense, Inc., a company focused on developing pediatric immunomodulators based on manipulating the human microbiome. Prior to Commense, Tad served as CBO for Crescendo Biologics, a novel antibody platform company. Prior to Merrimack, Tad worked as a consultant with clients across the biotech, pharmaceutical and medical device industries. Tad holds an M.B.A. from the Johnson School at Cornell University and a B.S. in Biology from Bates College.

On August 6, 2019 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the second quarter ending June 30, 2019 (Press release, PTC Therapeutics, AUG 6, 2019, View Source [SID1234538232]).

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"Over the second quarter we’ve made important progress towards our strategic plan of building a robust diversified orphan drug franchise," said Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics. "We believe that one of PTC’s key differentiators is our ability to work across multiple scientific platforms to bring orphan products to patients. Our 5-year vision elucidates PTC’s mission of bringing differentiated therapies to patients living with rare disorders that have limited or no treatment options. By fulfilling this mission, we will be creating value for all of our stakeholders."

Key Second Quarter and Other Corporate Highlights:

Commercial portfolio

Translarna received approval from the Brazilian health regulatory authority (ANVISA) and subsequently, PTC has entered into its first annual contract with the Brazilian Ministry of Health.
Emflaza received FDA approval for a label expansion to include DMD patients aged 2-5 years. This demographic is estimated to comprise approximately 25% of the prevalent DMD population in the U.S.
First commercial patient on Tegsedi in Latin America received treatment. The regulatory application submitted to the Brazilian health regulatory authority (ANVISA) was granted priority review, with expected approval by year end 2019.
We are working with Akcea to review the recent clinical results for Waylivra in Familial Partial Lipodystrophy (FPL) and we will determine the potential commercial strategy in LATAM.
The CHMP adopted a negative opinion to expand the label for Translarna to include non-ambulatory DMD patients. PTC has requested a re-examination with a tentative date for an opinion in October.
Advancing gene therapy portfolio & infrastructure

PTC has signed a long-term lease agreement securing a state-of-the-art biologics facility to support the Company’s expansion into multiple gene therapy programs. The facility is currently operating under cGMP standards by its current tenant, Bristol-Myers Squibb, and will be fully transitioned to PTC by mid-2020. PTC intends to consolidate its discovery and research operations in the same campus.
The expected BLA for our gene therapy candidate to treat patients with AADC deficiency is on track for submission to the FDA in Q4 2019, with anticipated commercial launch in the U.S. in 2020.
PTC has expanded its gene therapy portfolio by entering into a strategic licensing agreement with Odylia Therapeutics for multiple preclinical programs to treat rare inherited retinal disorders. The lead program is for Leber Congenital Amaurosis (LCA6), a rare early onset retinal dystrophy.
Completed strategic equity investment in MRI Interventions provides PTC with devices that directly delivers gene therapies into the CNS.
Risdiplam remains on track for NDA submission with the FDA in 2H19

Data from pivotal FIREFISH and SUNFISH studies were presented at AAN and demonstrate continued clinical benefit with risdiplam in Type 1, 2, and 3 SMA.
Risdiplam continues to be well tolerated at all doses across studies and there have been no drug related safety findings leading to withdrawal.
Planned NDA filing with the FDA is on track for the second half of this year with the intention to support a broad label to treat SMA Types 1, 2, & 3 patients. Filing of the MAA in the EU is expected to occur in the first half of 2020.
PTC re-iterates full year 2019 guidance:

PTC anticipates full year DMD franchise net product revenues to be between $285 and $305 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2019 to be between $395 and $405 million.
PTC anticipates non-GAAP R&D and SG&A expense for the full year 2019 to be between $360 and $370 million, excluding estimated non-cash, stock-based compensation expense of approximately $35 million.
Second quarter 2019 financial highlights:

Total revenues were $85.5 million for the second quarter of 2019, compared to $68.7 million for the second quarter of 2018.
Translarna net product revenues were $57.8 million for the second quarter of 2019, compared to $47.8 million for the second quarter of 2018. These results reflect the expanded commercialization of Translarna.
Emflaza net product revenues were $27.6 million for the second quarter of 2019, compared to $20.3 million for the second quarter of 2018. These results reflect the continued transition to a new specialty pharmacy distributor.
GAAP R&D expenses were $60.0 million for the second quarter of 2019, compared to $32.6 million for the second quarter of 2018. The increase in R&D expenses reflects costs associated with advancing the gene therapy platform, increased investment in research programs and advancement of the clinical pipeline.
Non-GAAP R&D expenses were $54.5 million for the second quarter of 2019, excluding $5.5 million in non-cash, stock-based compensation expense, compared to $28.7 million for the second quarter of 2018, excluding $3.9 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $49.2 million for the second quarter of 2019, compared to $33.5 million for the second quarter of 2018. The increase in SG&A expenses reflects continued investment in commercial activities including our expanding commercial portfolio.
Non-GAAP SG&A expenses were $43.8 million for the second quarter of 2019, excluding $5.4 million in non-cash, stock-based compensation expense, compared to $29.4 million for the second quarter of 2018, excluding $4.1 million in non-cash, stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was $5.3 million for the second 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 $41.8 million for the second quarter of 2019, compared to net loss of $9.5 million for the second quarter of 2018.
Cash, cash equivalents, and marketable securities were $363.5 million at June 30, 2019, compared to $227.6 million at December 31, 2018.
Shares issued and outstanding as of June 30, 2019 were 58,707,185.
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 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.

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 6594813. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. The accompanying slide presentation will be posted on the investor relations section of the PTC website. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for two weeks.