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

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

Second Quarter Financial Highlights:

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

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

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

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

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

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

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

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

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

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

2018 Guidance

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

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

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

Key Second Quarter and Other Corporate Highlights:

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

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

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

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

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

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

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

Non-GAAP Financial Measures
In this press release, the financial results and financial guidance of PTC are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude stock-based compensation expense. This non-GAAP financial measure is provided as a complement to financial measures reported in GAAP because management uses this non-GAAP financial measure when assessing and identifying operational trends. In management’s opinion, this non-GAAP financial measure is useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating

performance of PTC and the company’s future outlook. Quantitative reconciliations of non-GAAP financial measures to their closest equivalent GAAP financial measures are included in the tables below.

Today’s Conference Call and Webcast Reminder:
Today’s conference call will take place at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 8284468. A live, listen-only webcast of the conference call and corresponding slides can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for two weeks. PTC’s current Investor Presentation and R&D Day slides are available at the same website location.

On August 7, 2018 Synlogic(Nasdaq:SYBX) reported that Aoife Brennan, M.B., B.Ch., Synlogic’s interim president and chief executive officer, and chief medical officer, will present a corporate update at the 2018 Wedbush PacGrow Healthcare Conference at 3:05pm ET on Tuesday, August 14, 2018, in New York City (Press release, Synlogic, AUG 7, 2018, View Source [SID1234528792]).

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A live webcast of the presentation can be accessed under "Event Calendar" in the Investors & Media section of the Company’s website. An archived webcast recording will be available on the Synlogic website for approximately 30 days after the event.

On August 7, 2018 Sesen Bio, Inc. (Nasdaq: SESN), a late-stage clinical company developing next-generation antibody-drug conjugate (ADC) therapies for the treatment of cancer, reported key leadership transitions as part of its evolution into a commercial-stage oncology company (Press release, Eleven Biotherapeutics, AUG 7, 2018, View Source [SID1234528926]). Thomas Cannell, DVM has been appointed chief executive officer and a member of the board of directors, bringing with him a wealth of leadership experience in building and overseeing strategic operations and global pharmaceutical commercialization for life science companies. Stephen Hurly left his employment with Sesen Bio effective August 7, 2018.

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"Over the last two years, Sesen Bio has undergone a unique evolution as a company. Following the acquisition of Viventia and the resulting transition from an ophthalmology organization to an oncology company, we have been wholly focused on developing Vicinium for high-grade non-muscle invasive bladder cancer," said Wendy Dixon, Ph.D., chair of Sesen Bio’s board of directors. "We are grateful to Steve for his many significant contributions in advancing Sesen Bio to where it is today, and we wish him all the best in his future endeavors. As we transition further as a company, Tom will be an exceptional strategic leader bringing deep experience in drug development and commercialization to drive Vicinium, our pipeline and the company through this important next chapter."
Thomas Cannell most recently served as chief operating officer and president of global commercial products at Orexigen Therapeutics, Inc., where he led the successful commercialization and profitability of Contrave. Prior to Orexigen, Dr. Cannell spent 27 years with Merck & Co., Inc., where he held senior leadership positions in global commercialization, consumer marketing, and sales operations and management for both development-stage programs and approved marketed products. While with Merck, he served as president of Merck Canada and head of marketing and strategy for Merck Sharp & Dohme Corp., Japan, a subsidiary of Merck & Co., where he was responsible for setting up a long-standing strategic process and plan, managed a multi-billion-dollar product portfolio and oversaw thousands of employees. In addition, he designed and successfully piloted an innovative, customer-centric commercial model for Merck’s U.S. business. Dr. Cannell received his DVM degree from Washington State University.
"My enthusiasm for joining Sesen Bio is based on the novel mechanism of Vicinium, its potential as a monotherapy and combination agent, particularly with checkpoint inhibitors, and

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the range of therapeutic opportunities with our novel fusion proteins," stated Dr. Cannell. "Over the next several months, we will be focused on several strategic objectives across our pipeline. These include: completing the Phase 3 registration trial for Vicinium with 12-month data expected by mid-2019; engaging with regulatory authorities to prepare for our first BLA submission; initiating the appropriate pre-commercial activities to support a potential future product approval and launch; and exploring new therapeutic opportunities in additional indications. I am excited to join the company at this pivotal time and believe strongly in our ability to execute these strategic priorities to make a meaningful difference in the lives of patients."
Sesen Bio also announced today that Abbie Celniker, Ph.D. and Paul Chaney have stepped down from the company’s board of directors, effective August 7, 2018. Dr. Celniker, who served on the board of directors since the company’s founding by Third Rock Ventures in 2011, is focusing on her partner role at Third Rock Ventures and early-stage portfolio-building efforts. Mr. Chaney joined the company’s board in 2014 as a leader in developing innovative ophthalmic therapeutics and is leaving to continue his efforts in that area of drug development.
"I am very proud of the progress and many milestones that the Sesen Bio team has achieved, and it has been a pleasure to serve on the board with this outstanding group of industry leaders," said Dr. Celniker. "The company is uniquely positioned with a Phase 3 trial that is well underway, established clinical data, a lead product candidate with a pipeline of opportunities and a strong balance sheet to fund its growing organization. I look forward to watching Sesen Bio’s continued success as a supportive investor with Third Rock Ventures."
In connection with the appointment of Dr. Cannell, Sesen Bio entered into an employment agreement with Dr. Cannell that, among other things, provides for the grant of a non-statutory stock option outside of the company’s 2014 Stock Incentive Plan as an inducement material to Dr. Cannell’s entering into employment with Sesen Bio in accordance with Nasdaq Stock Market Listing Rule 5635(c)(4). The stock option to purchase 1,350,000 shares of the company’s common stock is being granted effective as of August 7, 2018. The stock option grant was approved by the independent compensation committee of the board of directors in accordance with Nasdaq Stock Market Listing Rule 5635(c)(4). The stock option will have an exercise price per share equal to the closing price per share of Sesen Bio’s common stock on The Nasdaq Global Market on August 7, 2018. The stock option will have a ten-year term and will vest over a four-year period, with 25 percent of the shares underlying the stock option award vesting on the first anniversary of the date of grant and an additional 6.25 percent of the shares underlying the stock option vesting at the end of each successive three-month period following the one-year anniversary of the date of grant of the stock option, subject to Dr. Cannell’s continued service with the company through the applicable vesting dates.

On August 7, 2018 Teneobio, Inc., a next generation multi-specific antibody therapeutics company, and Poseida Therapeutics, Inc., a San Diego-based clinical-stage company translating best-in-class gene engineering technologies into lifesaving cell therapies, reported a new research collaboration and licensing agreement to develop novel CAR-T therapies using Teneobio’s heavy chain only domain antibodies (UniDabs) (Press release, TeneoBio, AUG 7, 2018, View Source [SID1234558319]).

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Poseida will apply UniDab binders, which demonstrate significant advantages over traditional single chain variable antibody fragment (scFv) binders, to the development of its next generation CAR-T therapies.

The new collaboration follows a commercial license agreement between the companies that was announced in May of 2017. Under the terms of the new agreement, Teneobio will generate multiple UniDab product candidates using its proprietary UniRat transgenic human antibody ‘heavy-chain only’ rodent platform and its state-of-the-art sequence-based discovery engine, TeneoSeek. Poseida will have exclusive global licensing rights for the clinical development and commercialization of specific UniDabs for CAR-T therapies.

Teneobio Inc. will receive an upfront payment and is eligible to receive future research, development and regulatory milestone payments per UniDab candidate, with total potential earnings of over $250 million for CAR-T therapies developed by Poseida. Teneobio would also receive royalties on worldwide net sales of each CAR-T therapy.

"We are delighted to partner with Poseida and to help create the next generation of cell therapies," said Roland Buelow, CEO of Teneobio. "Domain antibodies have been clinically validated as excellent targeting moieties in CAR-T cells. They confer robust in vivo specificity and efficacy. They are smaller in size, have greater humanicity, and superior developability relative to standard scFv’s. The use of UniDabs as binders in CAR-T products is predicted to result in a lack of tonic signaling and lower immunogenicity, thus solving some of the problems of the first-generation, scFv-based CAR-T therapies."

Eric Ostertag, CEO of Poseida, noted, "Teneobio’s UniDab binders are an ideal match for Poseida’s novel and industry-leading CAR-T platform technologies. Poseida has demonstrated that UniDabs can be engineered to serve as binding molecules for our CAR-T therapeutics and oftentimes may function better than other binders for use in CAR-T products."

"We are pleased to expand our existing partnership with Poseida, whose cutting-edge genetic engineering tools combined with our targeting UniDab candidates will enable the development of the next generation of superior CAR-T therapies to treat cancer. We believe that UniDabs provide differentiated advantages from other targeting moieties, and that their utility and reach will extend beyond antibody therapeutics to novel transformational cell therapy treatments," added Omid Vafa, CBO of Teneobio.

On August 7, 2018 Myovant Sciences (NYSE: MYOV), a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of women’s health and endocrine diseases, reported corporate updates and reported financial results for the first fiscal quarter ended June 30, 2018 (Press release, Myovant Sciences, AUG 7, 2018, View Source [SID1234528486]).

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"We are off to an excellent start to the year, highlighted by the completion of patient screening for our Phase 3 LIBERTY 1 trial evaluating relugolix in women with heavy menstrual bleeding associated with uterine fibroids, and screening in the replicate LIBERTY 2 trial expected to be completed this quarter," said Lynn Seely, M.D., President and Chief Executive Officer of Myovant Sciences. "We are at an exciting stage in the evolution of the Company, with top-line safety and efficacy data expected for relugolix in 2019 in three distinct indications, each with large markets and wholly owned North American and European rights."

Recent Business Highlights and Upcoming Milestones

On July 10, 2018, Myovant announced completion of patient screening for its LIBERTY 1 trial, the first of two Phase 3 replicate trials evaluating relugolix in women with heavy menstrual bleeding associated with uterine fibroids. Top-line safety and efficacy data from this trial are expected in the second quarter of 2019.
Screening for the LIBERTY 2 trial is anticipated to be completed this quarter.
Completion of enrollment and top-line safety and efficacy data from the two Phase 3 SPIRIT trials of relugolix in women with endometriosis-associated pain are expected in 2019.
Enrollment is expected to be completed in 2018 for the Phase 3 HERO trial of relugolix in men with advanced prostate cancer, and top-line safety and efficacy data are expected in 2019.
Myovant raised aggregate net proceeds of approximately $150.0 million during fiscal 2018 from the issuance and sale of 3,333,334 common shares in an underwritten public offering in July 2018, 1,110,015 common shares in a private placement to the Company’s controlling shareholder, Roivant Sciences, Ltd. in April 2018, and 2,767,129 common shares in an "at-the-market" equity offering program in April 2018.
On May 30, 2018, Myovant entered into a Commercial Manufacturing and Supply Agreement with Takeda Pharmaceutical Company Ltd. pursuant to which Takeda will manufacture and supply Myovant with relugolix drug substance to support the commercial launch of relugolix, if marketing authorization is granted. Takeda has also agreed to assist with the transfer of technology and manufacturing know-how to a second contract manufacturing organization of Myovant.
In May 2018, following the completion of a Phase 1 study, Myovant initiated a Phase 2a clinical trial in healthy female volunteers to characterize the dose-response curve in the controlled ovarian stimulation setting prior to studying MVT-602 in infertile woman seeking pregnancy.
On April 20, 2018, Myovant announced a partnership with PERIOD, Inc., a youth-led non-profit organization focused on menstrual equity, to elevate the conversation around period health, including an Ask Me About Periods campaign on college campuses around the country.
First Fiscal Quarter 2018 Financial Summary

Research and development (R&D) expenses for the quarter ended June 30, 2018, were $51.3 million compared to $17.7 million for the comparable period in 2017. The increase for the quarter primarily reflects the progress of Myovant’s ongoing Phase 3 clinical trials of relugolix, which were initiated in 2017, and additional personnel-related expenses.

General and administrative (G&A) expenses for the quarter ended June 30, 2018, were $8.7 million compared to $4.2 million for the comparable period in 2017. The increase for the quarter primarily reflects increases in personnel-related expenses, professional service fees, and other administrative expenses to support Myovant’s headcount growth and expanding operations.

Interest expense for the quarter ended June 30, 2018, was $1.6 million compared to no interest expense in the comparable prior year period. Interest expense for the quarter consisted of interest expense related to financing agreements with NovaQuest Pharma Opportunities Fund IV L.P. and Hercules Capital, Inc., as well as the associated non-cash amortization of debt discount and issuance costs.

Net loss for the quarter ended June 30, 2018, was $62.1 million, compared to $23.3 million for the comparable period in 2017. On a per common share basis, net loss was $0.98 and $0.39 for the quarters ended June 30, 2018, and 2017, respectively. The increases in the net loss and net loss per common share for the quarter were driven primarily by the increase in costs associated with Myovant’s ongoing Phase 3 clinical trials, which were initiated in 2017, as well as increased personnel-related expenses.

Capital resources: Cash and committed funding totaled $235.6 million at June 30, 2018, consisting of $143.6 million in cash and $92.0 million in remaining financing commitments available from NovaQuest under the NovaQuest Securities Purchase Agreement and the NovaQuest Equity Purchase Agreement. In the second quarter of fiscal year 2018, Myovant further strengthened its balance sheet, raising net proceeds of approximately $70.2 million from the issuance and sale of 3,333,334 common shares in an underwritten public offering. An additional $40.6 million of capacity remains available under the "at-the-market" equity offering program that Myovant initiated in April 2018.

About Relugolix

Relugolix is an oral, once-daily, small molecule that acts as a gonadotropin-releasing hormone, or GnRH, receptor antagonist that has been evaluated in approximately 1,600 study participants in Phase 1, Phase 2 and Phase 3 clinical trials. In these trials, relugolix has been shown to be generally well tolerated and to suppress estrogen and progesterone levels in women and testosterone levels in men. Common side effects observed were consistent with suppression of these hormones. In the ongoing Phase 3 LIBERTY clinical trials in women with heavy menstrual bleeding associated with uterine fibroids and the ongoing Phase 3 SPIRIT clinical trials in women with endometriosis-associated pain, relugolix is undergoing evaluation alone as monotherapy and in combination with estradiol and norethindrone acetate, a progestin. Myovant is studying whether the combination decreases estradiol levels to the range required to treat signs and symptoms of endometriosis and uterine fibroids while minimizing the side effects such as bone mineral density loss and hot flashes, associated with low estrogen levels. The ongoing Phase 3 HERO study is evaluating relugolix alone in men with advanced prostate cancer.