On May 2, 2019 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the first quarter ending March 31, 2019 (Press release, PTC Therapeutics, MAY 2, 2019, View Source [SID1234535538]).

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"We believe we are making great strides towards our vision of having many more treatments for patients of rare disorders," said Stuart Peltz, Ph.D., CEO of PTC Therapeutics. "The approval of Translarna expands our footprint in Brazil. We are now preparing for the launches for multiple products, including Tegsedi for hATTR and Waylivra for FCS in Latin America. These differentiated therapies for rare disorders have the potential to be part of growing our revenue base to $1.5 billion by 2023."

Key First Quarter and Other Corporate Highlights:

Expanding commercial platform

Translarna received approval from the Brazilian health regulatory authority (ANVISA) which will allow for expanded market access.

Waylivra received a positive CHMP opinion from the European medical authorities in the first quarter 2019 and will be referred to the European Commission for consideration. After ratification by the European Commission, PTC plans to initiate early access programs in Latin America in 2019.

Tegsedi application submitted to the Brazilian health regulatory authority (ANVISA) was granted priority review, with expected approval by year end 2019. PTC has initiated early access programs for Tegsedi to support patient diagnosis and monitoring in Latin America.

PTC has submitted a sNDA with the FDA for Emflaza for patients 2 to 5 years old and received an approval action date of July 4, 2019.

Advancing gene therapy portfolio

As previously disclosed, PTC plans to submit a BLA with the FDA in late 2019 followed by an MAA in Europe for the AADC deficiency gene therapy program with anticipated commercial launch in the United States in 2020.

Friedreich ataxia program is advancing with an expected IND submission in late 2019 and subsequent entry in the clinic.

PTC is in the process of finalizing a long-term lease on an existing biologics manufacturing facility for the gene therapy pipeline.

Risdiplam SMA data expected at American Academy of Neurology (AAN)

As previously disclosed, based on regulatory interactions an NDA and MAA is planned for the second half of this year with the intention to support a broad label to treat SMA Types 1, 2, & 3 patients.

The SMA program is a collaboration between PTC, Roche and SMA Foundation.

Net sales over $1B would be subject to mid-teens royalties to PTC from Roche, resulting in potential royalties to PTC in excess of $200M per year. Potential remaining regulatory and sales-based milestones are approximately $400M.

Growing pipeline and R&D capabilities

As previously disclosed, PTC’s splicing platform has generated another development candidate, PTC258, for Familial dysautonomia, a rare genetic neurological disorder causing life-threatening medical complications from birth. PTC258 is advancing through IND-enabling studies to enter the clinic in late 2019. This program is in collaboration with MGH and NYU

Translarna’s dystrophin study in currently enrolling for potential U.S. regulatory re-submission for accelerated approval in 2020.

PTC’s oncology portfolio continues to advance with studies in AML with PTC299 and DIPG & LMS for PTC596. PTC expects these studies to fully enroll by the end of 2019.

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.

First Quarter 2019 Financial Highlights:

Total revenues were $53.6 million for the first quarter of 2019, compared to $56.1 million for the first quarter of 2018.

Translarna net product revenues were $35.3 million for the first quarter of 2019, compared to $36.8 million for the first quarter of 2018. These results reflect lumpiness in ordering patterns from Latin America.

Emflaza net product revenues were $17.8 million for the first quarter of 2019, compared to $19.2 million for the first quarter of 2018. These results reflect first quarter dynamics including seasonality and a planned transition to a new specialty pharmacy distributor.

GAAP R&D expenses were $52.6 million for the first quarter of 2019, compared to $31.4 million for the first quarter of 2018. The increase in R&D expenses reflects costs associated with advancing the gene therapy platform and increased investment in research programs as well as advancement of the clinical pipeline.

Non-GAAP R&D expenses were $47.9 million for the first quarter of 2019, excluding $4.7 million in non-cash, stock-based compensation expense, compared to $27.6 million for the first quarter of 2018, excluding $3.7 million in non-cash, stock-based compensation expense.

GAAP SG&A expenses were $40.6 million for the first quarter of 2019, compared to $33.0 million for the first quarter of 2018. The increase in SG&A expenses were primarily due to continued investment to support our commercial activities.

Non-GAAP SG&A expenses were $36.0 million for the first quarter of 2019, excluding $4.6 million in non-cash, stock-based compensation expense, compared to $29.0 million for the first quarter of 2018, excluding $4.0 million in non-cash, stock-based compensation expense.

Change in the fair value of deferred and contingent consideration was $21.2 million for the first 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 $72.1 million for the first quarter of 2019, compared to net loss of $19.3 million for the first quarter of 2018.

Cash, cash equivalents, and marketable securities were $407.2 million at March 31, 2019, compared to $227.6 million at December 31, 2018.

Shares issued and outstanding as of March 30, 2019 were 58,418,790 .

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 measure 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 1559069. 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.

On May 2, 2019 Geron Corporation (Nasdaq: GERN) reported financial results for the first quarter ended March 31, 2019 (Press release, Geron, MAY 2, 2019, View Source [SID1234535556]). As of the quarter-end, the Company had approximately $170 million in cash and marketable securities, which is sufficient to support the commencement of late-stage development for imetelstat in lower risk myelodysplastic syndromes (MDS).

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"We continue to make good progress on our 2019 objectives," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "We are on track to assume sponsorship of the IND for imetelstat by the end of the second quarter, as expected, which will enable us to open enrollment of the planned Phase 3 portion of IMerge by mid-year. In addition, our expanding in-house leadership and drug development team will support the accomplishment of our 2019 objectives and beyond."

First Quarter 2019 Results

For the first quarter of 2019, the Company reported a net loss of $10.1 million, or $0.05 per share, compared to $7.2 million, or $0.04 per share, for the comparable 2018 period.

Revenues for the first quarter of 2019 were $57,000 compared to $318,000 for the comparable 2018 period. Revenues for 2019 and 2018 included royalty and license fee revenues under various non-imetelstat license agreements. The decrease in revenues for the three months ended March 31, 2019 compared to the same period in 2018 primarily reflects a reduction in the number of active license agreements as a result of patent expirations on the underlying technology.

Total operating expenses for the first quarter of 2019 were $11.4 million compared to $7.8 million for the comparable 2018 period.

Research and development expenses for the first quarter of 2019 were $5.9 million compared to $2.4 million for the comparable 2018 period. The increase in research and development expenses for first quarter of 2019 compared to the same period in 2018 primarily reflects higher imetelstat development costs under the former collaboration agreement with Janssen Biotech, Inc. (Janssen) due to Geron’s payment responsibility increasing from 50% to 100% following the termination of the collaboration agreement; new costs for the imetelstat program transition from Janssen to Geron, including for consultants and the Company’s contract research organization (CRO); and increased expenses related to additional development headcount.

General and administrative expenses for the first quarter of 2019 were $5.5 million compared to $5.3 million for the comparable 2018 period. The increase in general and administrative expenses in the first quarter of 2019 compared to same period in 2018 primarily reflects recruitment expenses for new members for the Company’s board of directors.

Interest and other income for the first quarter of 2019 was $1.2 million compared to $394,000 for the comparable 2018 period. The increase in interest and other income for 2019 compared to 2018 primarily reflects higher yields on the Company’s marketable securities portfolio.

Conference Call for Imetelstat Program Update

In lieu of a first quarter conference call, Geron will host a conference call to provide an imetelstat program update at 9:00 a.m. ET on Thursday, May 16, 2019.

Participants may access the conference call live via telephone by dialing domestically +1 (877) 303-9139 or internationally +1 (760) 536-5195. The conference ID is 1264477. A live, listen-only webcast will also be available on the Company’s website at www.geron.com/investors/events. If you are unable to listen to the live call, an archived webcast will be available on the Company’s website for 30 days.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat include a Phase 2/3 trial called IMerge in lower risk myelodysplastic syndromes (MDS) and a Phase 2 trial called IMbark in Intermediate-2 or High-risk myelofibrosis. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for the treatment of patients with transfusion-dependent anemia due to lower risk MDS who are non-del(5q) and refractory or resistant to an erythroid stimulating agent.

On May 2, 2019 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported that it is scheduled to participate in the following upcoming events (Press release, Arrowhead Pharmaceuticals, MAY 2, 2019, View Source [SID1234535572]):

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Bank of America Merrill Lynch 2019 Health Care Conference– Las Vegas, May 14-16, 2019
May 15, 8:40 a.m. PDT –Christopher Anzalone, Ph.D., Arrowhead’s president and chief executive officer, will deliver a corporate presentation

Vascular Discovery: From Genes to Medicine 2019 Scientific Sessions (ATVB|PVD) – Boston,
May 14-16, 2019 May 15, 11:00 a.m. EDT – So Wong, Ph.D., Arrowhead’s director of biology, will deliver a poster presentation titled, "Reduction in Angiopoietin-Like Protein 3 via RNA Interference Improves Dyslipidemias and Hepatic Steatosis"

2019 National Lipid Association Scientific Sessions – Aventura, FL,
May 16-19, 2019 May 18, 11:25 a.m. EDT – So Wong, Ph.D., Arrowhead’s director of biology, will deliver a poster presentation titled, "Personalized Medicine for Dyslipidemias by RNA Interference-Mediated Reductions in Apolipoprotein C3 or Angiopoietin-Like Protein 3"

20th Annual B. Riley FBR Institutional Investor Conference– Beverly Hills, May 22-23, 2019
May 23, 8:30 a.m. PDT –Christopher Anzalone, Ph.D., Arrowhead’s president and chief executive officer, will participate in a fireside chat presentation with Mayank Mamtani, B. Riley FBR research analyst

A copy of the presentation materials and webcast links, if the presentation is being webcast, may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

On May 2, 2019 Quanterix Corporation (NASDAQ:QTRX), a company digitizing biomarker analysis with the goal of advancing the science of precision health, reported that it will release its financial results for first quarter 2019 after the close of trading on Thursday, May 9, 2019 (Press release, Quanterix, MAY 2, 2019, View Source [SID1234535587]). Company management will host a conference call at 4:30 p.m., EDT to discuss Quanterix’ financial results and provide a business update. The call will be hosted by Kevin Hrusovsky, Chief Executive Officer, President and Chairman, Quanterix.

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Individuals interested in listening to the conference call may do so by dialing (833) 686-9351 for domestic callers, or (612) 979-9890 for international callers. Please reference the following conference ID:8955828. A live webcast will be accessible on the investor relations section of Quanterix’ website: View Source The webcast will be available on the Company’s website for one year following completion of the call.

On May 2, 2019 Apollo Endosurgery, Inc. ("Apollo") (Nasdaq: APEN), a global leader in less invasive medical devices for gastrointestinal and bariatric procedures, reported financial results for the first quarter ended March 31, 2019 (Press release, Lpath, MAY 2, 2019, View Source [SID1234535539]).

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First Quarter 2019 Highlights
•Endo-bariatric sales, representing our core business moving forward, increased 5% from $10.3 million to $10.8 million, with ESS sales increasing 13% and IGB sales declining 5%
•On a constant currency basis, endo-bariatric sales increased 9% with ESS sales increasing 17% and IGB sales equal to the prior year
•U.S. ESS sales increased 20% year-over-year in the quarter
Todd Newton, CEO of Apollo, commented, "We were pleased with our ESS product sales growth this quarter. We are also pleased with our Sx launch progress to date. The activity and resulting new account pipeline is encouraging to us that Sx will roll out very well as the year progresses. Similarly, we think our IGB product is positioned well for a return to growth."
First Quarter 2019 Results
U.S. Endo-bariatric sales increased 9% to $4.5 million, while outside the U.S. ("OUS") Endo-bariatric sales of $6.4 million increased 3% as reported and 10% in constant currency. Worldwide, total Endo-bariatric sales of $10.8 million increased 5% as reported and 9% constant currency. Of our Endo-bariatric product sales in the first quarter, 60% related to our Endoscopic Suturing System ("ESS" or "OverStitch") and 40% related to our Intragastric Balloon ("IGB" or "Orbera").
U.S. ESS product sales increased 20% to $3.0 million, while OUS ESS product sales of $3.5 million increased 7% as reported and 14% in constant currency. Worldwide ESS sales of $6.5 million grew 13% as reported and 17% constant currency. Growth was due to continuing new user adoption and greater product utilization in existing accounts.
U.S. IGB product sales decreased $0.2 million, or 9%, to $1.5 million in the first quarter of 2019. OUS IGB product sales were unchanged at $2.9 million, on an as reported basis, and increased 5% in constant currency as higher sales of Orbera365 were partially offset by weaker six-month balloon sales in other OUS markets. On a worldwide basis, IGB sales of $4.3 million declined 5% as reported and were flat in constant currency terms.
The divestiture of our Surgical products in December of 2018 affects the comparability of our total revenues in the first quarter of 2019 versus 2018. Total revenues in the first quarter of 2019 only include Surgical product sales in certain OUS markets where we continue to temporarily distribute these products. Total revenues in the first quarter of 2019 were $13.2 million, which included $1.7 million of Surgical product sales, compared to $15.7 million in the first quarter of 2018, which included $5.2 million of Surgical product sales, for a decrease of 16% or $2.5 million.
Gross margin for the first quarter 2019 was 55%, compared to 58% for the first quarter 2018 due to lower Surgical product sales following their divestiture and a greater proportion of our overall product sales coming from our ESS products, which realize a lower gross margin than our other products. Partially offsetting the gross margin effects of these changes in our product sales mix were the gross margin improvement projects we completed in late 2018.
Total operating expenses decreased $7.0 million to $9.8 million in the first quarter 2019, compared to the first quarter 2018. The decrease was primarily due to a one-time non-cash settlement gain of $5.6 million resulting from the resolution of a dispute regarding amounts charged for inventory purchases and transition services through 2016 following our December 2013 acquisition of the obesity intervention division of Allergan, Inc. Excluding this gain, total operating expenses decreased $1.4 million due largely to lower amortization expense resulting from the reduction in our intangible assets after the recent divestiture of our Surgical products.

Net loss for the first quarter 2019 was $2.8 million compared to $8.1 million for the first quarter 2018. The reduction in net loss was primarily due to the one-time settlement gain. Excluding this one-time gain, net loss would have been $8.4 million, an increase of $0.3 million due largely to lower Surgical product sales following our December 2018 divestiture, partially offset by the reduced amortization expense mentioned previously.

Cash, cash equivalents and restricted cash were $31.0 million as of March 31, 2019.
Conference Call
Apollo will host a conference call on May 2, 2019 at 3:30 p.m. Central Time / 4:30 p.m. Eastern Time to discuss Apollo’s operating results for the first quarter ended March 31, 2019.
To participate in the conference call dial (866) 393-4306 for domestic callers and (734) 385-2616 for international callers. The conference ID number is 7094276. A live webcast of the conference call will be made available on the "Events and Presentations" section of our Investor Relations website: www.ir.apolloendo.com.
A replay of the webcast will be made available on Apollo’s website, www.apolloendo.com following the event.
Non-GAAP Financial Measures
To supplement our financial results we are providing a non-GAAP financial measure, percentage revenue change in constant currency, which removes the impact of changes in foreign currency exchange rates that affect the comparability and trend of revenues. Percentage revenue change in constant currency is calculated by translating current foreign currency sales at last year’s exchange rate. This supplemental measure of our performance is not required by, and is not determined in accordance with GAAP.
We believe the non-GAAP financial measure included herein is helpful in understanding our current financial performance. We use this supplemental non-GAAP financial measure internally to understand, manage and evaluate our business, and make operating decisions. We believe that making non-GAAP financial information available to investors, in addition to GAAP financial information, may facilitate more consistent comparisons between the company’s performance over time with the performance of other companies in the medical device industry, which may use similar financial measures to supplement their GAAP financial information. However, our non-GAAP financial measure is not meant to be considered in isolation or as a substitute for the comparable GAAP metric.