On November 2, 2017 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the third quarter ending September 30, 2017 (Press release, PTC Therapeutics, NOV 2, 2017, View Source [SID1234521522]).

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“Our performance this quarter combined with our commercial, financial and R&D advancements should allow us to end 2017 in a strong position,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “Our commercial success is driven by our mission of improving the lives of patients with Duchenne.”

Third Quarter Financial Highlights:

Translarna net product sales were $32.0 million for the third quarter of 2017, representing 45% growth over $22.0 million reported in the third quarter of 2016.
EMFLAZA net product sales were $9.8 million for the third quarter of 2017.
Total revenues for the third quarter of 2017 were $41.9 million compared to $23.0 million in the same period of 2016. The change in total revenue was a result of the expanded commercial launch of Translarna and the successful U.S. EMFLAZA launch.
GAAP R&D expenses were $30.0 million for the third quarter of 2017 compared to $31.4 million for the same period in 2016. Non-GAAP R&D expenses were $26.4 million for the third quarter of 2017, excluding $3.6 million in non-cash, stock-based compensation expense, compared to $27.1 million for the same period in 2016, excluding $4.3 million in non-cash, stock-based compensation expense. The decrease in R&D expense for the third quarter of 2017 as compared to the prior year period was primarily due to the completion of our Phase 3 Translarna trials at the end of 2016 partially offset by start-up clinical activities and regulatory spend.
GAAP SG&A expenses were $31.4 million for the third quarter of 2017 compared to $23.7 million for the same period in 2016. Non-GAAP SG&A expenses were $27.9 million for the third quarter of 2017, excluding $3.5 million in non-cash, stock-based compensation expense, compared to $19.0 million for the same period in 2016, excluding $4.6 million in non-cash, stock-based compensation expense. The increase in SG&A expenses primarily related to the expansion of the U.S. commercial sales team in support of the launch of EMFLAZA.
Net interest expense for the third quarter of 2017 was $3.4 million compared to net interest expense of $2.1 million in the same period in 2016. The increase in net interest expense is primarily a result of increased interest expense related to the $40 million secured loan facility which we closed during the second quarter of 2017 partially offset by reduced interest income from investments.
Net loss for the third quarter of 2017 was $33.7 million compared to a net loss of $35.2 million for the same period in 2016.
Cash, cash equivalents, and marketable securities totaled approximately $169.3 million at September 30, 2017 compared to approximately $231.7 million at December 31, 2016.
Shares issued and outstanding as of September 30, 2017, were 41.5 million, which includes 0.1 million shares of unvested restricted stock awards.
2017 Guidance:

Translarna net sales guidance for 2017 is anticipated to be between $120 and $140 million. We now anticipate EMFLAZA net sales for 2017 to be between $20 and $25 million, an increase from our prior guidance of $15 to $20 million. This brings 2017 full year revenue guidance between $160 and $185 million, an increase from our prior guidance of $155 million to $180 million, including a $20 million milestone we achieved in mid-October, under our SMA program.
GAAP R&D and SG&A expense for the full year 2017 are now anticipated to be between $250 to $260 million. Excluding estimated non-cash, stock-based compensation expense of approximately $40 million, full year 2017 non-GAAP R&D and SG&A expense are now anticipated to be between $210 million and $220 million. These expenses will be primarily in support of the commercial availability of Translarna globally, the commercial launch of EMFLAZA in the U.S. and the continued research and clinical development of other product pipeline candidates.
We now expect to end 2017 with over $150 million of cash and cash equivalents, an increase from prior guidance of $120 million.
Key Third Quarter and other Corporate Highlights:

Filed Formal Dispute Resolution Request with U.S. FDA to appeal Complete Response Letter for ataluren. PTC received a Complete Response Letter from the Office of Drug Evaluation I of the U.S. Food and Drug Administration (FDA) for the New Drug Application (NDA) of the investigational medicine ataluren for the treatment of nonsense mutation dystrophinopathies. The letter stated that it is unable to approve the application in its current form. PTC has filed a formal dispute resolution request challenging this decision.
EMFLAZA revenue grew to $9.8M in third quarter. PTC is committed to enabling access to EMFLAZA for all patients in need, regardless of financial situation or insurance status. We demonstrated an increase in EMFLAZA prescriptions over the past quarter. There are currently over 1,500 patients on EMFLAZA and we estimate that there are approximately 9,000 Duchenne patients in the U.S. over the age of five who are eligible to be prescribed EMFLAZA.
Continued global expansion of Translarna results in revenue of $32.0M in third quarter. PTC continues to expand its strong global footprint, with sales generated in over 25 countries. This strong performance reflects continued uptake, sustainable pricing levels, and high ( > 90%) compliance to treatment.
SMA clinical program advanced into the pivotal portion of the study. In mid-October, the SUNFISH trial transitioned into the pivotal portion of the study which triggered a $20M milestone to PTC from Roche. Data from the SUNFISH trial was presented at the International Congress of the World Muscle Society. An interim analysis of the five cohorts treated with RG7916 for 28 days demonstrates an exposure-dependent increase in SMN protein.
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, non-GAAP financial measures exclude stock-based compensation expense and one-time restructuring expenses relating to the reorganization of operations intended to improve efficiency and better align costs and employment structure with PTC’s strategic plans. These non-GAAP financial measures are provided as a complement to results 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 operating performance at PTC and the company’s future outlook. Quantitative reconciliations of GAAP financial measures are included in the tables below

On November 2, 2017 Radius Health, Inc. (“Radius” or the “Company”) (Nasdaq:RDUS), reported its financial results for the third quarter ended September 30, 2017, and provided a business update (Press release, Radius, NOV 2, 2017, View Source [SID1234521524]).

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“It is very encouraging for Radius to see TYMLOS gaining traction in the market,” said Jesper Høiland, President and CEO of Radius. “In addition to our stated goal of gaining market leadership for TYMLOS, we are excited by the positive response to the product by physicians, payors and patients. With the recent capital raise, we are also well funded to start investing in expanding the TYMLOS label and advancing the development of our other therapeutic candidates in the pipeline.”

“TYMLOS is proving itself to be an important treatment for a high unmet medical need, as demonstrated by the continued increase in lives covered through managed care contracts,” said David Snow, Chief Commercial Officer of Radius. “Postmenopausal women with osteoporosis at high risk for fractures deserve to have therapies that safely and effectively reduce that risk, with lower out of pocket costs. Our sales team is achieving strong reach and call frequency and we are continuing to expand payer acceptance while we see anabolic class volume stabilizing.”

TYMLOS (abaloparatide injection)

Third quarter reported sales of TYMLOS in the U.S. (the first full quarter since its launch) were approximately $3.5 million. Radius received FDA approval for TYMLOS on April 28, 2017 for the treatment of postmenopausal women with osteoporosis at high risk of fracture, and began shipments to wholesalers at the end of May 2017.

In September 2017, Radius presented results from the completed ACTIVExtend study in an abstract titled “Sustained Fracture Risk Reduction with Sequential Abaloparatide/Alendronate: Results of ACTIVExtend” at the ASBMR 2017 Annual Meeting in Denver, Colorado. In ACTIVExtend, patients who had completed 18 months of TYMLOS or placebo in the ACTIVE Phase 3 trial were transitioned to receive 24 additional months of open-label alendronate. Patients who received a sequential therapy of TYMLOS followed by alendronate demonstrated statistically significant fracture risk reductions through 3.5 years. At the 43-month timepoint, the previous TYMLOS-treated patients had a significant 84 percent relative risk reduction (p<0.0001) in the incidence of new vertebral fractures compared with women who received placebo followed by alendronate. Additionally, TYMLOS followed by alendronate demonstrated a 39 percent relative risk reduction in nonvertebral fractures (p=0.038), compared with women who received placebo followed by alendronate.

The Company expects to submit a labeling supplement to the FDA in connection with the ACTIVExtend results by the end of 2017.
Pipeline Updates

Abaloparatide — Subcutaneous (SC)

European MAA
Radius’ European Marketing Authorisation Application (MAA) for abaloparatide-SC for the treatment of postmenopausal women with osteoporosis is under review by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). On July 21, 2017, the CHMP issued a second Day-180 List of Outstanding Issues. Radius is working with the CHMP to address these issues, and discussed preliminary responses with the Rapporteurs in a formal Clarification Meeting. We expect an opinion from the CHMP regarding the MAA prior to the end of 2017.
Male Osteoporosis Trial
We recently gained agreement with the FDA on the design of a clinical trial in men with osteoporosis, which, if successful, will form the basis of an sNDA seeking to expand the use of TYMLOS to treat men with osteoporosis at high risk for fracture. The study will be a randomized, double-blind, placebo-controlled trial that will enroll approximately 225 men with osteoporosis.
The primary endpoint is change in spine bone mineral density (“BMD”) at 12 months compared with placebo. TYMLOS has demonstrated in previous clinical trials that it increases BMD in postmenopausal women. The study will include specialized high-resolution imaging of bone structure in a subset of the study participants. We expect to initiate the trial in the first quarter of 2018.
Abaloparatide-Transdermal Patch (TD)

We have scheduled a meeting with the FDA in January 2018 to align on a regulatory pathway for a pivotal study (e.g. bioequivalence or BMD) for abaloparatide-TD and we are also discussing manufacturing arrangements with 3M Company related to potential commercial supplies of abaloparatide-TD. We are on track to complete manufacturing scale-up, production, and other required activities needed to initiate a pivotal study. The pharmacokinetic profile of an optimized abaloparatide-TD patch was successfully modified so as to improve comparability to abaloparatide-SC. We believe that the transdermal patch program, if approved, will offer patients who suffer from osteoporosis a convenient alternative.
Elacestrant (RAD1901)

In a meeting to discuss the elacestrant breast cancer development program, the FDA indicated that, depending on the study results, which must demonstrate an improvement over then available therapies, the planned single-arm Phase 2 trial could be considered a pivotal study for accelerated approval as long as a confirmatory study is ongoing by the time of the NDA submission. We will provide further study details when the Phase 2 study is initiated, which we expect will be in early 2018. In October, elacestrant received FDA Fast Track designation supporting a rapid speed to market strategy.
Elacestrant is also being evaluated at low doses as an estrogen receptor ligand for the potential relief of the frequency and severity of moderate to severe hot flashes in postmenopausal women with vasomotor symptoms. We are currently reviewing our elacestrant vasomotor development program and plan to provide an update by the end of 2017.
RAD140

In September 2017, the Company announced that the first patient had been enrolled in the Company’s Phase 1 study of RAD140, a nonsteroidal selective androgen receptor modulator (SARM) undergoing clinical evaluation for the treatment of hormone receptor positive breast cancer. The clinical trial is designed to evaluate the safety and maximum tolerated dose of RAD140 in approximately 40 patients.
Radius Anticipates the Following Milestones

Abaloparatide
Receive Committee for Medicinal Products for Human Use (CHMP) opinion regarding the EMA’s review of the abaloparatide-SC MAA before the end of 2017
Submit a labeling supplement in connection with the ACTIVExtend data to the FDA by 2017 year end
Provide updates on the potential regulatory pathway for an abaloparatide-transdermal patch (TD) pivotal study following a scheduled meeting with the FDA in January 2018 and discussions with 3M Company for potential commercial supplies of abaloparatide-TD
Initiate a male osteoporosis study in the first quarter of 2018
Enter into a partnership for the potential commercialization of abaloparatide-SC outside the US and Japan prior to commercial launch in the European Union
Elacestrant
Initiate Phase 2 single-arm monotherapy clinical trial in metastatic breast cancer patients in early 2018
Complete review of the elacestrant vasomotor development program and provide an update by the end of 2017
RAD140
Continue enrollment in the Phase 1 study
Corporate Update

The Company also announced today that Ansbert Gadicke, M.D., has resigned from the Board of Directors of the Company effective November 8, 2017, after having served on the Board of the Company and its predecessor since 2003. Following Dr. Gadicke’s resignation, the Company expects to reduce the size of the Board from 10 to 9 members.

Radius Expects to Make Presentations at the Following Upcoming Conferences

On December 5-9, 2017, Gary Hattersley, PhD, Chief Scientific Officer, will present at the San Antonio Breast Cancer Symposium and Radius will host one-on-one meetings
On January 8-11, 2018, Jesper Høiland, Radius President and CEO, will present and host one-on-one meetings at the 36th JP Morgan Annual Healthcare Conference in San Francisco
On March 12-14, 2018, Jesper Høiland, Radius President and CEO, will present and host one-on-one meetings at the 38th Cowen Annual Healthcare Conference
Third Quarter 2017 Financial Results

Three Months Ended September 30, 2017
For the three months ended September 30, 2017, Radius reported a net loss of $57.8 million, or $1.31 per share, compared to a net loss of $46.2 million, or $1.07 per share, for the three months ended September 30, 2016.

For the three months ended September 30, 2017, Radius reported TYMLOS net product revenues of about $3.5 million, which reflects the first full quarter of recorded sales. Radius had no revenue in the three months ended September 30, 2016 as the FDA approved TYMLOS on April 28, 2017.

Research and development expense for the three months ended September 30, 2017, was $21.0 million compared to $27.5 million for the three months ended September 30, 2016, a decrease of $6.5 million, or 24%. This decrease was primarily driven by a $3.4 million decrease in vasomotor project related spending, a $2.0 million decrease in abaloparatide-SC project costs, and a $1.1 million decrease in RAD1901 oncology project costs.

Selling, general, and administrative expense for the three months ended September 30, 2017, was $47.7 million compared to $19.2 million for the three months ended September 30, 2016, an increase of $28.5 million, or 148%. This increase was primarily the result of an increase of approximately $10.1 million in professional fees and support costs, including the costs associated with increasing headcount and preparing for the commercialization of TYMLOS in the United States. This increase was also driven by a $15.2 million increase in compensation expense, including stock-based compensation, due to the increase in headcount.

Nine Months Ended September 30, 2017
For the nine months ended September 30, 2017, Radius reported a net loss of $183.2 million, or $4.21 per share, compared to a net loss of $130.1 million, or $3.02 per share, for the nine months ended September 30, 2016.

For the nine months ended September 30, 2017 Radius reported TYMLOS net product revenues of about $4.4 million, which reflects the first full quarter of reported sales. Radius had no revenue in the nine months ended September 30, 2016 as the FDA approved TYMLOS on April 28, 2017.

Research and development expense for the nine months ended September 30, 2017, was $60.2 million compared to $81.8 million for the nine months ended September 30, 2016, a decrease of $21.6 million, or 26%. This decrease was primarily driven by a $14.9 million decrease in RAD1901 project costs, a $14.1 million decrease in abaloparatide-SC project costs, and a $2.2 million decrease in development costs associated with abaloparatide-TD. This decrease was partially offset by a $9.7 million increase in compensation expense, including stock-based compensation, due to an increase in headcount.

Selling, general, and administrative expense for the nine months ended September 30, 2017, was $135.9 million compared to $50.1 million for the nine months ended September 30, 2016, an increase of $85.8 million, or 171%. This increase was primarily the result of an increase of approximately $27.9 million in professional fees and support costs during the nine months ended September 30, 2017, including the costs associated with increasing headcount and preparing for the commercialization of TYMLOS in the United States. This increase was also driven by a $49.4 million increase in compensation expense, including stock-based compensation, due to an increase in headcount.

As of September 30, 2017, Radius had $468.1 million in cash, cash equivalents and marketable securities. Based upon our cash, cash equivalents and marketable securities balance as of September 30, 2017, we believe that, prior to the consideration of proceeds from partnering and/or collaboration activities, we have sufficient capital to fund our development plans, U.S. commercial and other operational activities for not less than twelve months from the date of this press release.

On November2, 2017 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for cancer, respiratory and other diseases, reported that will host a Q3 2017 Investor Call on Thursday, November 9, 2017 at 10:00 AM (EST) to discuss financial results and provide a corporate update (Press release, Pieris Pharmaceuticals, NOV 2, 2017, View Source [SID1234521487]).

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To access the call, participants may dial 877-407-8920 (US & Canada) or 412-902-1010 (International) at least 10 minutes prior to the start of the call.

An archived replay of the call will be available for 30 days by dialing (Toll Free US & Canada): 877-660-6853, (International): 201-612-7415, Conference ID #: 13661472.

On November2, 2017 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for cancer, respiratory and other diseases, reported that will host a Q3 2017 Investor Call on Thursday, November 9, 2017 at 10:00 AM (EST) to discuss financial results and provide a corporate update (Press release, Pieris Pharmaceuticals, NOV 2, 2017, View Source [SID1234521487]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

To access the call, participants may dial 877-407-8920 (US & Canada) or 412-902-1010 (International) at least 10 minutes prior to the start of the call.

An archived replay of the call will be available for 30 days by dialing (Toll Free US & Canada): 877-660-6853, (International): 201-612-7415, Conference ID #: 13661472.

On November2, 2017 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for cancer, respiratory and other diseases, reported that will host a Q3 2017 Investor Call on Thursday, November 9, 2017 at 10:00 AM (EST) to discuss financial results and provide a corporate update (Press release, Pieris Pharmaceuticals, NOV 2, 2017, View Source [SID1234521487]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

To access the call, participants may dial 877-407-8920 (US & Canada) or 412-902-1010 (Intern(Press release, Pieris Pharmaceuticals, NOV 2, 2017, View Source [SID1234521487])ational) at least 10 minutes prior to the start of the call.

An archived replay of the call will be available for 30 days by dialing (Toll Free US & Canada): 877-660-6853, (International): 201-612-7415, Conference ID #: 13661472.