On May 05, 2016 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq:RDUS), a science-driven biopharmaceutical company that is committed to developing innovative therapeutics in the areas of osteoporosis, oncology and endocrine diseases, reported its financial results for the first quarter ended March 31, 2016, and provided recent corporate highlights (Press release, Radius, MAY 5, 2016, View Source [SID:1234511959]). As of March 31, 2016, Radius had $439.8 million in cash, cash equivalents and marketable securities.

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"We are extremely pleased to have submitted our first New Drug Application to the U.S. FDA for abaloparatide-SC for the treatment of postmenopausal osteoporosis and our MAA is currently under review in Europe. In anticipation of our first potential launch, we are developing our commercial plans and building our marketing organization in the U.S.," said Robert Ward, President and Chief Executive Officer of Radius. "To maximize the potential of this new investigational drug outside the U.S., we are continuing our productive partnering discussions and anticipate entering into a marketing collaboration prior to a potential first commercial launch."

Pipeline Updates

Abaloparatide-SC

In March 2016, Radius submitted a new drug application ("NDA") in the United States for the treatment of postmenopausal women with osteoporosis. In November 2015, Radius submitted a marketing authorisation application ("MAA") to the European Medicines Agency ("EMA"), which subsequently was validated and is currently undergoing regulatory review. We anticipate a CHMP scientific opinion in 2016.

Abaloparatide-TD

Radius also is developing abaloparatide-transdermal, which it refers to as abaloparatide-TD, based on 3M’s patented Microstructured Transdermal System technology for potential use as a short wear-time transdermal patch. Radius commenced a human replicative clinical evaluation of the optimized abaloparatide-TD patch in December 2015 with the goal of achieving comparability to abaloparatide-SC.

RAD1901

In December 2014, Radius commenced a Phase 1 multicenter, open-label, two-part, dose-escalation study of RAD1901 in postmenopausal women with advanced estrogen receptor positive and HER2-negative breast cancer in the United States. The Phase 1 study is designed to evaluate escalating doses of RAD1901 in Part A. The Part B expansion cohorts allow for an evaluation of additional safety, tolerability and preliminary efficacy. In addition, in December 2015, Radius commenced a Phase 1 FES-PET study in patients with metastatic breast cancer in the European Union, which includes the use of FES-PET imaging to assess estrogen receptor occupancy in tumor lesions following RAD1901 treatment. Upon completion of these studies, we plan to submit to present the data at an appropriate scientific meeting.

Radius Expects the Following Upcoming Milestones

Abaloparatide-SC
Receive opinion from the Committee for Medicinal Products for Human Use regarding the EMA’s review of the abaloparatide-SC MAA
FDA determination on acceptance of NDA for abaloparatide-SC for filing in the second quarter of 2016
Enter into a collaboration for the potential commercialization of abaloparatide-SC prior to a commercial launch
Abaloparatide-TD
Planned mid-year update on human replicative study of optimized abaloparatide-TD patch
RAD1901
Initiate expansion cohorts for Phase 1 dose escalation study in metastatic breast cancer patients
Report results at upcoming scientific meeting
Radius Expects To Make Presentations at the Following Upcoming Conferences

Bank of America Merrill Lynch 2016 Healthcare Conference, May 10-12, 2016, in Las Vegas, NV.
Abstract presentation at the ASCO (Free ASCO Whitepaper) Annual Meeting, June 3-7, 2016 in Chicago
"A Phase I study of RAD1901, an oral selective estrogen receptor degrader, in ER positive, HER2 negative, advanced breast cancer patients".
Recent Corporate Highlights

On April 1, 2016, at the 2016 Endocrine Society Annual Meeting, Dr. Felicia Cosman, an osteoporosis specialist and Professor of Medicine at Columbia University, presented "Abaloparatide-SC Significantly Reduces Vertebral and Nonvertebral Fractures and Increases BMD Regardless of Baseline Risk"

On April 15, 2016, three oral presentations were made from the abaloparatide-SC development program at the 2016 World Congress on Osteoporosis, Osteoarthritis and Musculoskeletal Diseases meeting in Malaga, Spain:

Dr. Lorie Fitzpatrick, Chief Medical Officer of Radius Health presented "Effects of Abaloparatide on Vertebral, Non-Vertebral, Major Osteoporotic and Clinical Fracture Incidence in Postmenopausal Women with Osteoporosis: Results of the Phase 3 Active Trial"

Dr. Serge Ferrari, Professor of Medicine, Geneva University presented "Eighteen Months of Treatment with Abaloparatide Followed by Six Months of Treatment with Alendronate in Postmenopausal Women with Osteoporosis- Results of the ACTIVExtend Trial"

Dr. Eugene McCloskey, Professor in Adult Bone Diseases, University of Sheffield presented "Effect of Investigational Treatment Abaloparatide for Prevention of Major Osteoporotic Fracture or Any Fracture is Not Altered by Baseline Fracture Probability"
Abaloparatide-SC as a treatment for postmenopausal women with osteoporosis is an investigational product and its safety and efficacy have not been established.

First Quarter 2016 Financial Results

For the three months ended March 31, 2016, Radius reported a net loss of $40.5 million, or $0.94 per share, as compared to a net loss of $17.1 million, or $0.47 per share for the three months ended March 31, 2015. The increase in net loss for the three months ended March 31, 2016 as compared to the three months ended March 31, 2015 was primarily due to an increase in research and development and general and administrative expenses, partially offset by a decrease in interest expense and an increase in interest income.

Research and development expenses for the three months ended March 31, 2016 were $27.5 million, compared to $11.6 million for the same period in 2015. The increase for the 2016 period as compared to the 2015 period was primarily attributable to an increase in professional contract services costs associated with the development of RAD1901 to support a Phase 1 study in metastatic breast cancer that commenced in late 2014 and a Phase 2b study in postmenopausal vasomotor symptoms that commenced in December 2015. This increase was also a result of an increase in compensation expense, including stock-based compensation, due to an increase in headcount from March 31, 2015 to March 31, 2016.

General and administrative expenses for the three months ended March 31, 2016 were $13.6 million, compared to $4.8 million for the same period in 2015. The increase for the 2016 period as compared to the 2015 period was primarily attributable to an increase in professional support costs and legal fees, including the costs associated with increasing headcount and preparing for the potential commercialization of abaloparatide-SC, subject to a favorable regulatory review.

As of March 31, 2016, Radius had $439.8 million in cash, cash equivalents and marketable securities. Based upon Radius’ cash, cash equivalents and marketable securities balance, Radius believes that, prior to the consideration of revenue from the potential future sales of any of its investigational products, it has sufficient capital to fund its development plans, U.S. commercial scale-up and other operational activities into 2018.

About Abaloparatide

Abaloparatide is an investigational therapy for the potential treatment of women with postmenopausal osteoporosis who are at an increased risk for a fracture. Abaloparatide is a novel synthetic peptide that engages the parathyroid hormone receptor (PTH1 receptor) and was selected for clinical development based on its favorable bone building activity.

Abaloparatide has completed Phase 3 development for potential use as a daily self-administered injection (abaloparatide-SC). In the fourth quarter of 2015, Radius’ Marketing Authorisation Application (MAA) for abaloparatide-SC for the treatment of patients with postmenopausal osteoporosis was validated and is currently undergoing regulatory review by the European Medicines Agency (EMA). Radius submitted a New Drug Application (NDA) for abaloparatide-SC to the US Food and Drug Administration (FDA) at the end of the first quarter of 2016.

Radius also is developing abaloparatide-transdermal (abaloparatide-TD) based on 3M’s patented Microstructured Transdermal System technology for potential use as a treatment for osteoporosis.

About RAD1901

RAD1901 is a selective estrogen receptor down-regulator/degrader (SERD), which at high doses is being evaluated for potential use as an oral non-steroidal treatment for hormone-driven, or hormone-resistant, breast cancer. RAD1901 is currently being investigated for potential use in postmenopausal women with advanced estrogen receptor positive (ER+), HER2-negative breast cancer, the most common form of the disease. Studies completed to date indicate that the compound has the potential for use as a single agent or in combination with other therapies for the treatment of breast cancer.

RAD1901 also is being evaluated in a Phase 2b study at low doses for potential relief of the frequency and severity of moderate to severe hot flashes in postmenopausal women with vasomotor symptoms. Additional information on the clinical trial program of RAD1901 is available on www.clinicaltrials.gov.

RAD140

RAD140 is a non-steroidal selective androgen receptor modulator (SARM) that is currently in preclinical development for potential use in multiple indications including cancer. RAD140 resulted from an internal drug discovery program focused on the androgen receptor pathway, which is highly expressed in many breast cancers. Due to its receptor and tissue selectivity, potent oral activity and long half-life, RAD140 could have clinical potential in the treatment of conditions where androgen modulation may play a role.

On May 5, 2016 DURECT Corporation (Nasdaq: DRRX) reported financial results for the first quarter of 2016 (Filing, Q1, DURECT, 2016, MAY 5, 2016, View Source [SID:1234512022]). Total revenues were $3.6 million and net loss was $7.9 million for the three months ended March 31, 2016 as compared to total revenues of $4.8 million and net loss of $4.9 million for the three months ended March 31, 2015.

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At March 31, 2016, we had cash and investments of $24.3 million, compared to cash and investments of $29.3 million at December 31, 2015. Subsequent to the end of the first quarter, we raised net proceeds of approximately $17.0 million from the sale of additional shares of common stock. Including these proceeds, our pro forma cash and investments at March 31, 2016 would have been approximately $41.3 million. At March 31, 2016, we had $19.7 million in short and long term debt.
"The highlight of the quarter was undoubtedly the resubmission of the REMOXY NDA, followed by its acceptance for review by the FDA and the establishment of a September 25, 2016 PDUFA date," stated James E. Brown, D.V.M., President and CEO of DURECT. "With respect to DUR-928, we have progressed into our first two patient studies with results anticipated during the course of this year. For POSIMIR, we continued the PERSIST Phase 3 trial which we are in the process of amending in response to an FDA recommendation."
Update of Selected Programs:

• Epigenomic Regulator Program. DUR-928, our Epigenomic Regulator Program’s lead product candidate, is an endogenous, small molecule, new chemical entity (NCE), which may have broad applicability in several metabolic diseases such as nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH), and in acute organ injuries such as acute kidney injury.
During the first quarter, we began our first patient trial utilizing DUR-928. This study is an open-label single-ascending-dose safety and pharmacokinetic Phase 1b trial of DUR-928 in NASH patients and matched control subjects. This study will be conducted in successive cohorts evaluating single-dose levels of oral DUR-928. After a PK/safety review at each dose, the study can proceed to the next higher dose. The study is being conducted in Australia, and we anticipate that we will start obtaining results from this trial in the second quarter of 2016. This study is designed to enable and inform a subsequent multi-dose study in NASH or other patients with other liver function impairment.
In addition, our protocol has been approved by the institutional review board for a second study in patients with DUR-928, also being conducted in Australia. This Phase 1b trial of DUR-928 is an open-label single-ascending-dose safety and pharmacokinetic study in patients with impaired kidney function and matched control subjects. This study will be

1
conducted in successive cohorts evaluating single-dose levels of DUR-928 administered by injection. After a PK/safety review at each dose, the study can proceed to the next higher dose. We anticipate that this study will be completed in 2016, and that this study will enable and inform subsequent trials for patients with either acute kidney injury or other kidney function impairment.

• REMOXY (oxycodone) Extended-Release Capsules CII. Based on our ORADUR technology, REMOXY is a unique long-acting formulation of oxycodone designed to discourage common methods of tampering associated with opioid misuse and abuse. Pain Therapeutics (our licensee) resubmitted the NDA on schedule in March 2016. In April 2016, Pain Therapeutics announced that the FDA had determined that the NDA was sufficiently complete to permit a substantive review and that September 25, 2016 is the target action date under the Prescription Drug User Fee Act (PDUFA). The extended release oxycodone market is greater than $2 billion in the U.S. alone, and we are eligible for a potential royalty on REMOXY between 6.0% to 11.5% of net sales depending on sales volumes.

• POSIMIR (SABER-Bupivacaine) Post-Operative Pain Relief Depot. In November 2015, we began enrolling patients for PERSIST, a new POSIMIR Phase 3 clinical trial, consisting of patients undergoing laparoscopic cholecystectomy (gallbladder removal) surgery. In a previous clinical trial of 50 patients undergoing laparoscopic cholecystectomy, POSIMIR was compared with the active control bupivacaine hydrochloride, against which POSIMIR demonstrated in a post hoc analysis an approximately 25% reduction in pain intensity on movement for the first 3 days after surgery (p=0.024), using the same statistical methodology specified for the current trial. We began recruiting patients for this trial with an intent to compare POSIMIR to placebo. Based on recommendations from the FDA received subsequent to the start of the trial, in April 2016 we decided to amend the PERSIST trial including by incorporating standard bupivacaine HCl as an active control. This change will add to the time and cost to complete the PERSIST trial, but we believe that a positive outcome from this trial design would result in a stronger NDA filing and potentially commercial advantages. This clinical trial is designed to generate data necessary to support an NDA resubmission.
POSIMIR is our investigational post-operative pain relief depot that utilizes our patented SABER technology and is intended to deliver bupivacaine to provide 3 days of pain relief after surgery. We are in discussions with potential partners regarding licensing development and commercialization rights to POSIMIR, for which we hold worldwide rights. We are also continuing to evaluate the requirements for commercializing POSIMIR on our own in the U.S., in the event that we determine that to be the preferred route of commercialization.

• Relday (Risperidone Program). Relday is a proprietary, long-acting, once-monthly subcutaneous injectable formulation of risperidone for the treatment of schizophrenia. In September 2015, Zogenix (our licensee) announced that they had completed a multi-dose Phase 1b trial with results consistent with the profile of risperidone and a previous Phase 1 single-dose clinical trial. Zogenix has stated that it is seeking a development and commercialization partner for Relday and that Relday is well-positioned to begin a Phase 3 program once a partner is secured.

•
ORADUR-ADHD Program. In 2013, we selected a formulation for the lead program in our ORADUR-ADHD (Attention Deficit Hyperactivity Disorder) program, ORADUR-Methylphenidate. This formulation was chosen based on its potential for rapid onset of
action, long duration with once-a-day dosing and target pharmacokinetic profile as demonstrated in a Phase 1 trial. In addition, this product candidate utilizes a small capsule size relative to the leading existing long acting products on the market and incorporates our ORADUR anti-tampering technology. Orient Pharma, our licensee in defined Asian and South Pacific countries, has initiated a Phase 3 study in Taiwan and anticipates completing it in 2016. We retain rights to all other markets in the world, notably including the U.S., Europe and Japan, and are engaged in licensing discussions with other companies.

• Business Development Activities. We have multiple programs that may potentially be licensed over the next 12-18 months. These include POSIMIR, DUR-928, ORADUR-ADHD (territories outside certain Asian and South Pacific markets), as well as various other programs which we have not described publicly in detail.
Earnings Conference Call
A live audio webcast of a conference call to discuss first quarter 2016 results will be broadcast live over the internet at 4:30 p.m. Eastern Time on May 5 and is available by accessing DURECT’s homepage at www.durect.com and clicking "Investor Relations." If you are unable to participate during the live webcast, the call will be archived on DURECT’s website under Audio Archive in the "Investor Relations" section.

On May 5, 2016 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the first quarter ending March 31, 2016 (Press release, PTC Therapeutics, MAY 5, 2016, View Source [SID:1234512058]).

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"We are working hard to make Translarna available to all Duchene muscular dystrophy patients globally who may benefit," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "To that end, we are in dialogue with the FDA about the Refuse to File letter we received earlier this year. In Europe, we are also continuing discussions with the CHMP surrounding our regulatory submissions for DMD and CF. We are seeing growth in Translarna global sales and continue to expand access for DMD patients outside of the U.S. We were pleased with the positive recommendation from NICE, and anticipate access for patients in England once the market access agreement with NHS England is finalized. We are also excited that the second SMA compound in our collaboration is advancing in the clinic."

Key First Quarter and other Corporate Highlights:

Actively pursuing regulatory approvals for Translarna in DMD globally. PTC has engaged in dialogue with the U.S. Food and Drug Administration (FDA) to discuss the matters described in the Agency’s Refuse to File letter regarding Translarna for nonsense mutation Duchenne muscular dystrophy (nmDMD) and to seek a potential path forward to bring Translarna to patients in the U.S. Given the sensitivity around these discussions as well as their iterative nature, PTC will provide an update once it has clarified the regulatory strategy for Translarna in the U.S.
In the first quarter of 2016, PTC submitted the ACT DMD Phase 3 results to the European Medicines Agency (EMA) in support of the marketing authorization for Translarna for the treatment of nmDMD in ambulatory patients aged five and over, and separately, submitted a request for renewal of its current conditional marketing authorization. PTC is in the process of responding to requests for supplementary information from the Committee for Medicinal Products for Human Use (CHMP) to assist in determining whether the results of ACT DMD support a positive benefit-risk assessment for Translarna. PTC anticipates that the CHMP will issue its recommendation in mid-2016.

In Canada, PTC plans to submit a New Drug Submission for Translarna to Health Canada incorporating the results of the company’s Phase 3 ACT DMD study in the second half of 2016.

PTC recently initiated a Phase 1 pharmacokinetics study to assess the effects of Translarna in Japanese healthy volunteers. This study is the first step in pursuing regulatory approval for Translarna for nmDMD in Japan, one of the world’s largest pharmaceutical markets.

Translarna revenue of $18.9M in first quarter, which represents 49% growth over prior quarter. PTC has established a strong global commercial footprint launching the first approved therapy in nmDMD. By the end of 2016, PTC anticipates expanding commercial access to Translarna in over 35 countries across Europe, the Middle East, Latin America and Asia Pacific.
Translarna received recommendation in draft guidance from NICE. In the UK, the National Institute for Health and Care Excellence (NICE) has recommended Translarna for ambulatory patients aged five years and older with nmDMD in connection with a Managed Access Agreement (MAA) with NHS England. The provision of patient access is subject to the completion of the MAA with NHS England and finalization of the NICE draft guidance. NICE recently extended the timeframe for finalization of its guidance.
German patients accessing Translarna through alternate distribution channel. In Germany, PTC has delisted Translarna from the pharmacy ordering system. Since delisting in April, initial orders for German patients have already been successfully fulfilled via a foreign importation pathway. This pathway allows patients with high unmet medical needs to receive reimbursed access to treatments not directly available in Germany.
ACT CF Phase 3 clinical trial on track for completion by year-end 2016 with top-line results expected early 2017. During the third quarter of 2015, PTC submitted a variation to its marketing authorization requesting EMA approval of Translarna for the treatment of nonsense mutation cystic fibrosis (nmCF) based on the company’s previous Phase 3 study. The company anticipates the CHMP will issue its opinion regarding this submission in mid-2016. PTC’s confirmatory Phase 3 ACT CF trial is currently ongoing, and there is substantial risk that the results from this trial will be required for approval.
SMA program progressing with proof of concept achieved in Phase 1 study of RG7916 and clinical study in SMA patients planned for second half of 2016. Clinical development of the spinal muscular atrophy (SMA) program, a collaboration with Roche and the SMA Foundation, continued with the completion of a Phase 1 study of RG7916 in healthy volunteers. Preliminary results indicate that RG7916 was well tolerated and treatment resulted in increases of full length SMN2 mRNA. A clinical study of RG7916 in SMA patients is expected to begin in the second half of 2016.
First Quarter Financial Highlights:

Translarna net product sales were $18.9 million for the first quarter of 2016, representing 49% growth over $12.7 million reported in the fourth quarter of 2015; and significant growth versus $5.1 million in the first quarter of 2015. Translarna net product sales were positively impacted by a significant order from Brazil, where purchasing is often fulfilled in six-month orders, partially offset by a lower sales price for existing stock in Germany as a result of a mandatory discount imposed by the German Federal Association of the Statutory Health Insurances (GKV-SV) prior to delisting. The effect of larger but less frequent orders from Brazil may result in fluctuations in quarterly sales reporting during the course of the year.
Total revenues for the first quarter of 2016 were $18.9 million compared to $7.5 million in the same period of 2015. The change in total revenue was a result of the expanded commercial launch of Translarna, partially offset by lower grant revenue.
Non-GAAP R&D expenses were $27.1 million for the first quarter of 2016, excluding $4.3 million in non-cash, stock-based compensation expense, compared to $23.3 million for the same period in 2015, excluding $4.6 million in non-cash, stock-based compensation expense. GAAP R&D expenses were $31.4 million for the first quarter of 2016 compared to $27.9 million for the same period in 2015. The increase in R&D expense for the first quarter of 2016 as compared to the prior year period was primarily due to additional costs associated with our ongoing clinical trials.
Non-GAAP SG&A expenses were $21.3 million for the first quarter of 2016, excluding $4.6 million in non-cash, stock-based compensation expense, compared to $12.5 million for the same period in 2015, excluding $5.1 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $25.9 million for the first quarter of 2016 compared to $17.6 million for the same period in 2015. The increase in SG&A expense for the first quarter 2016 as compared to the prior year period primarily resulted from additional costs associated with commercial activities in support of Translarna across Europe and other regions.
Net interest expense for the first quarter of 2016 was $2.0 million compared to net interest income of $0.5 million in the same period in 2015. The increase in interest expense is primarily a result of the $150 million convertible debt offering completed during the third quarter 2015. The debt was recorded on PTC’s balance sheet at a discount, which will be amortized over the life of the bond.
Net loss for the first quarter of 2016 was $41.2 million compared to a net loss of $37.9 million for the same period in 2015.
During the quarter, PTC announced a workforce reduction of approximately 18% of its employees and contractors, which will result in a one-time charge of approximately $2.6 million. PTC incurred $1.9 million of this charge in the first quarter.
Cash, cash equivalents, and marketable securities totaled approximately $299 million at March 31, 2016 compared to approximately $339 million at December 31, 2015.
Shares issued and outstanding as of March 31, 2016 were 34.3 million, which includes 0.4 million shares of unvested restricted stock.
2016 Guidance:

Total ex-U.S. Translarna nmDMD revenues for 2016 are anticipated to be between $65 and $85 million. This guidance assumes current exchange rates and the continued commercial expansion for Translarna in nmDMD outside of the U.S.
Operating expenses for the full year 2016 are anticipated to be between $185 million and $195 million, excluding expected non-cash stock-based compensation expense of approximately $40 million, for total operating expenses of approximately $225 million to $235 million. These expenses will be primarily in support of our ongoing clinical trials for Translarna in nmDMD and nmCF, commercial launch activities for Translarna outside of the US, and the continued research and clinical development of other product pipeline candidates.
PTC expects to end 2016 with cash and cash equivalents of approximately $200 million.
Non-GAAP Financial Measures

In this press release, PTC’s financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results exclude stock-based compensation expense. These results are provided as a complement to results reported in GAAP, because management uses these non-GAAP financial measures when assessing and identifying operational trends.

PTC Therapeutics, Inc.
Consolidated Statements of Operations
(In thousands, except per share data)


Three Months Ended

March 31,

2016

2015

Revenues:

Net product revenue
$18,878

$5,069

Collaboration and grant revenue
17

2,413

Total revenues
18,895

7,482

Operating expenses:

Research and development (1)
31,399

27,938

Selling, general and administrative (1)
25,938

17,615

Total operating expenses
57,337

45,553

Loss from operations
(38,442)

(38,071)

Interest (expense)/income, net
(1,956)

524

Other expense, net
(721)

(368)

Loss before income tax expense
(41,119)

(37,915)

Income tax expense
(114)

—

Net loss
($41,233)

($37,915)

Weighted-average shares outstanding (in shares):

Basic and diluted
33,919,169

33,067,752

Net loss per share – basic and diluted (in dollars per share)
($1.22)

($1.15)

(1) Non-cash share-based compensation expense

included in operating expenses are as follows:

Research and development
$4,328

$4,667

Selling, general and administrative
4,587

5,081

Total share-based compensation expense
$8,915

$9,748

PTC Therapeutics, Inc.
Summary Consolidated Balance Sheet
(In thousands, except share amounts)

March 31,

December 31,

2016

2015
Cash, cash equivalents and marketable securities
$298,712

$338,925
Total assets
$335,232

$365,281

Total debt
$93,366

$91,848
Total deferred revenue
396

139
Total liabilities
$139,231

$139,280

Total stockholders’ equity (33,919,684 and 33,916,559 common shares

issued and outstanding at March 31, 2016 and December 31, 2015, respectively)
196,001

226,001
Total liabilities and stockholders’ equity
$335,232

$365,281

Upcoming Events:

PTC will participate in the following upcoming conference:

Bank of America 2016 Healthcare Conference, May 12 at 9:20 a.m. in Las Vegas, NV
The presentation will be webcast live on the Events and Presentations page under the investor relations section of PTC’s website at www.ptcbio.com and will be archived for two weeks following the presentation. PTC’s current investor presentation is available at the same website location.

On May 05, 2016 Raptor Pharmaceutical Corp. (NASDAQ:RPTP) ("Raptor" or the "Company"), a biopharmaceutical company developing and commercializing transformative treatments for rare diseases, reported its financial results for the first quarter of 2016 and provided an update on recent corporate developments (Press release, Raptor Pharmaceutical, MAY 5, 2016, View Source [SID:1234512059]).

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Summary

Global net product revenue for PROCYSBI was $27.5 million for the first quarter ended March 31, 2016, a 34% increase compared to $20.5 million for the same period in 2015.

Net loss, including In-Process R&D (IPR&D) impairment and changes in the fair value of contingent consideration liability, on a GAAP basis was $41.6 million, or $0.49 per share, for the first quarter of 2016 compared to a GAAP net loss of $19.7 million, or $0.28 per share, for the same period in 2015.

Non-GAAP net loss, which excludes non-cash expenditures such as stock compensation, impairment of intangible assets and adjustment to the fair value of contingent consideration liability, was $14.1 million, or $0.17 per share compared to a net loss of $16.8 million, or $0.24 per share in the first quarter of 2015.

Cash and cash equivalents were $132.0 million as of March 31, 2016.

"Raptor achieved several significant milestones during the quarter, underscoring the progress we are making in building a premiere rare disease company," said Julie Anne Smith, President and CEO of Raptor. "PROCYSBI continues to deliver strong growth as a result of significant market penetration and continued high levels of patient compliance and we now look forward to revenues from the commercialization of QUINSAIRTM. We are focused on prioritizing key clinical programs to drive growth and being measured with our investments in selected development to extend our cash runway. As a result we are adjusting our non-GAAP cash operating expense guidance favorably for 2016. We look forward to continuing momentum as we work to bring QUINSAIR to patients in Europe and Canada, to make progress towards an NDA filing for MP-376 in cystic fibrosis (CF) and to initiate a Phase 2 MP-376 study in non-cystic fibrosis bronchiectasis (BE)."

First Quarter 2016 Business Highlights

Four new patents related to PROCYSBI were listed in the Orange Book during the quarter, bringing the total number of Orange Book-listed patents to five. In addition, PROCYSBI was granted additional U.S. orphan exclusivity in the two-to-six year old nephropathic cystinosis population until August 2022.

In March 2016, MP-376 received Qualified Infectious Disease Product (QIDP) designation for three distinct indications: the treatment of chronic pulmonary infections due to Pseudomonas aeruginosa, in patients with CF and in patients with BE, and in patients with nontuberculous mycobacteria infections (NTM). QIDP designation confers five years exclusivity under the Hatch-Waxman Act, which, in the case of CF, would be added to the seven years of orphan exclusivity for a total potential of 12 years of exclusivity, if approved for the indication.

In March 2016, Health Canada accepted the new drug submission for PROCYSBI in nephropathic cystinosis with Priority Review, which provides for a shortened review period of 180 days, compared to a standard review of 300 days. If approved, the shorter review time is expected to enable Raptor to bring PROCYSBI to market faster in Canada.

First Quarter 2016 Financial Results

Raptor provides non-GAAP financial measures, which it believes can enhance an overall understanding of its financial performance when considered together with GAAP figures. Refer to the section of this press release below entitled "Non-GAAP Financial Information and Other Disclosures" for further discussion on this subject.

Net Product Revenue

PROCYSBI global net revenue for the first quarter of 2016 was $27.5 million, compared to $20.5 million for the first quarter of 2015. The 34% revenue growth was driven by further market penetration in the U.S. and in Europe and named patient sales in other international territories.

Cost of Sales

Cost of sales was $4.3 million for the first quarter of 2016, compared to $3.7 million for the first quarter of 2015. The increase was due to higher quarterly sales year-over-year, which yielded higher direct costs and royalty expenses, and lower inventory reserves in 2016.

Research & Development (R&D)

R&D expenses for the first quarter of 2016 were $14.0 million compared to $16.6 million for the first quarter of 2015. The change was primarily due to a decrease in clinical trial expenses.

Selling, General and Administrative (SG&A)

SG&A expenses were $20.4 million for the first quarter of 2016 compared to $14.8 million for the first quarter of 2015. The increase in SG&A expenses was primarily a result of increased staffing and promotional support for commercial operations of PROCYSBI worldwide and administrative expenses.

In-Process R&D (IPR&D) and Contingent Consideration Liability Related to QUINSAIR Acquisition

The Company has revised its clinical development plans for MP-376. As a result, for the quarterly period ended March 31, 2016, based on a fair value assessment, Raptor recorded a non-cash impairment charge to IPR&D of $39.6 million and a partially offsetting contingent consideration credit adjustment of $14.7 million related to R&D stage projects from its October 2015 acquisition of QUINSAIR.

Interest Expense

Interest expense for the first quarter of 2016 was $5.0 million, compared to $4.5 million for the first quarter of 2015. The increase in interest expense was due to an increase in royalty fees based on net sales for the period.

Net Loss

GAAP net loss in the first quarter of 2016 was $41.6 million, or $0.49 per share, compared to a net loss of $19.7 million, or $0.28 per share, in the first quarter of 2015.

Non-GAAP net loss, which excludes the non-cash expenses of stock compensation expenses, impairment of intangible assets and adjustment to the fair value of the contingent consideration liability, for the first quarter of 2016 was $14.1 million, or $0.17 per share, compared to a net loss of $16.8 million, or $0.24 per share, in the first quarter of 2015.

Cash, Cash Equivalents

As of March 31, 2016, Raptor had $132.0 million in cash and cash equivalents compared to $157.4 million in cash and cash equivalents at the end of 2015.

2016 Full-Year Financial Guidance

Reiterating 2016 global net revenue guidance of $115 to $125 million.
Reiterating PROCYSBI revenue growth of 25% to 30% over 2015 revenues.
Lowering non-GAAP operating expense guidance, which excludes non-cash expenditures such as stock compensation, amortization and impairment of IPR&D and adjustment to the fair value of the contingent consideration liability, to $125 to $135 million from $135 to $145 million.
Product and Pipeline Updates

PROCYSBI for Nephropathic Cystinosis

PROCYSBI is currently commercially available to patients in the U.S., where it is the market leader in nephropathic cystinosis. PROCYSBI is also commercially available in Europe and is made available in additional markets through named patient sales.
In March 2016, Health Canada accepted for review the Company’s New Drug Submission (NDS) for PROCYSBI for the treatment of nephropathic cystinosis, with Priority Review status.
QUINSAIRTM

In April 2016, the Company launched QUINSAIR in Germany and Denmark for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adults with CF. Raptor anticipates launching the product in Canada in the second half of 2016.
MP-376 (Investigational Form of QUINSAIRTM)

In March 2016, MP-376 received QIDP designation for three distinct indications: the treatment of chronic pulmonary infections due to Pseudomonas aeruginosa, in patients with CF and in patients with BE, and in patients with NTM. Granting of the QIDP designation by the FDA highlights the potential of MP-376 to address a number of serious and life-threatening infections and provides Raptor with significant incentives for the development of MP-376, including Priority Review by the FDA, eligibility for Fast Track designation and a five-year extension of marketing exclusivity under the Hatch-Waxman Act. QIDP also paves the way for the FDA to apply greater regulatory flexibility in the case of high unmet medical need for serious life-threatening infections. Raptor holds orphan drug designation in the U.S. for MP-376 for the treatment of CF, which, when added to the five years of exclusivity associated with QIDP designation, would confer 12 years of regulatory exclusivity upon FDA approval.
Raptor expects to provide an update on regulatory discussions with the FDA concerning MP-376 in CF.
The Company intends to initiate a Phase 2 study for MP-376 in BE this year. The Company will provide additional clarity on the timing of key activities once the study design has been finalized.
RP103 for Huntington’s Disease (HD)

Based on prioritization of clinical programs and the best use of current capital, the Company will be exploring non-dilutive funding and partnering options.
RP103 for Mitochondrial Diseases

The second interim analysis, which occurred after 12 patients completed 24 weeks of treatment, indicated the trial should continue as planned. The treatment was generally safe and well tolerated and no unexpected safety signals were observed in this patient population.
Anticipated Upcoming Milestones

4Q 2016 — Potential PROCYSBI approval in Canada
2H 2016 — Potential PROCYSBI and QUINSAIR launches in Canada
2016 — Advancements toward an NDA submission for MP-376 in CF in the U.S.
2016 — Initiation of a Phase 2 study for MP-376 in BE
Conference Call and Webcast Information

Raptor will conduct a conference call and live audio webcast at 4:30 p.m. ET (1:30 p.m. PT) today. The live call may be accessed by dialing (877) 710-6201 for domestic callers or (616) 548-5611 for international callers and using the conference ID number 1390038. A live webcast of the conference call will be available online from the investor relations section of the Raptor website at www.raptorpharma.com. After the call, a webcast replay will be available on the Raptor website for 90 days while a telephone replay will be available for five days. This can be accessed by dialing (855) 859-2056 for domestic callers, or (404) 537-3406 for international callers, and using the conference ID number 1390038.

Non-GAAP Financial Information and Other Disclosures

Raptor uses non-GAAP financial measures, such as non-GAAP net loss, non-GAAP net loss per share and non-GAAP operating expense, to assess and analyze its operational results and trends, provide operating expense guidance, and to make financial and operational decisions. Raptor believes these non-GAAP financial measures are also useful to investors because they provide greater transparency regarding Raptor’s operating performance and exclude non-cash expenditures such as stock compensation, amortization and impairment of IPR&D, and change in the fair value of the contingent consideration liability related to acquisitions. These non-GAAP financial measures should not be considered an alternative to measurements required by GAAP, such as net loss, net loss per share and total operating expense, and should not be considered measures of Raptor’s liquidity. Non-GAAP financial measures should not be considered as a substitute for measures of financial performance calculated in accordance with GAAP and should only be used to supplement an understanding of Raptor’s operating results as reported under GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. Reconciliations between non-GAAP financial measures and GAAP financial measures are included in the table accompanying this press release after the unaudited consolidated financial statements. Raptor has not provided a GAAP reconciliation for its forward-looking non-GAAP financial measures, such as 2016 operating expense, because such measures are not readily determinable and not available without unreasonable efforts. Raptor cannot reliably predict without unreasonable efforts the timing or likelihood of outcomes that determine future impairments or changes in the fair value of contingent consideration liability nor the timing or amount of the factors that substantially contribute to the projection of stock compensation expense. Both are excluded from the forward-looking non-GAAP financial measures. The Company does not believe that such a reconciliation would be meaningful to stockholders.

As part of its disciplined approach to funding innovation, Amgen carefully evaluates each internal program to determine which ones will receive much-coveted development funding (Company Web Page, Amgen, MAY 5, 2016, View Source [SID:1234511964]). Occasionally, a program will come along that does not make Amgen’s resource allocation cut in their intended indication but shows great promise in other areas. This presents a dilemma. Does Amgen provide more funding to explore this new promising area? Or, does Amgen put this asset in the hands of a committed outside party? In one recent deal, Amgen found some middle ground. When a particular molecule showed promise outside its intended area, an external team committed to moving it forward was located, while Amgen holds an option to take it back at a later stage. This is how startup company Celimmune came to develop AMG 714.

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A New Direction for AMG 714
AMG 714 is an anti-IL-15 monoclonal antibody that, following a series of rheumatoid arthritis and psoriasis studies, Amgen deprioritized. But there was a scientist outside of Amgen who hadn’t forgotten about anti-IL-15. Dr. Francisco Leon, a leading expert in the development of therapeutics for celiac disease, had been following this antibody and a growing body of scientific literature suggesting IL-15’s central role in refractory celiac disease and non-responsive celiac disease. Dr. Leon believed AMG 714 was a ready tool to test the hypothesis that blocking IL-15 could benefit celiac patients.

Praise in Partnership
Intrigued, Amgen struck an agreement with Celimmune, where Dr. Leon serves as CEO and chief medical officer. Amgen has licensed AMG 714 to Celimmune, which has rights to develop, manufacture and commercialize AMG 714 on a worldwide basis excluding Japan. In connection with the license, Celimmune granted Amgen an exclusive option to acquire Celimmune, and thus to reacquire AMG 714, upon completion of certain clinical studies in patients with celiac disease. "We do not hesitate in going on record to speak to the ‘best-in-industry’ licensing and post-transactional alliance management support we have been so privileged to experience to date with Amgen," said Ashleigh Palmer, Executive Chairman, Celimmune weeks after the deal’s completion.

"Dr. Leon’s conviction was so strong and his credentials so compelling, we couldn’t help but take notice," said Jeremy Grunstein, an executive director from Amgen’s Business Development team. "Celimmune provided not only an avenue to test this important hypothesis, but also a mechanism for Amgen to reprioritize the program in its pipeline. It’s the kind of deal structure we’d like to replicate."

Celiac disease is a chronic hereditary systemic autoimmune and inflammatory disease triggered by gluten consumption, which can cause gastrointestinal dysfunction and debilitating symptoms including nutritional malabsorption. Celimmune plans to initiate Phase 2 studies of AMG 714 for the treatment of diet non-responsive celiac disease and type II refractory celiac disease (RCD-II), an in situ small bowel T-cell lymphoma.

Dr. Leon stated, "Celiac disease is the only common autoimmune disease without any approved medication. Published literature demonstrates that a gluten-free diet is not a solution for the majority of patients. As such, Celimmune is delighted to have an opportunity to license an experimental therapeutic that will test one of the main hypotheses in the pathophysiology of celiac disease, namely that IL-15 plays a central role in RCD-II and non-responsive celiac disease."

Ashleigh Palmer, Celimmune’s Executive Chairman, concurred, "AMG 714 could be the first drug to market for a celiac indication and there’s room for growth. In addition to celiac, AMG 714 could have longer-term expansion and life cycle management opportunities within adjacent gastrointestinal autoimmune diseases."

With this deal, Amgen is still making a bet on external innovation, but with science that originated from within Amgen.

"This deal is win-win-win. If Celimmune is successful, both parties will be rewarded and most importantly, patients will be one step closer to the first medicine available for celiac disease," said Grunstein.