On January 8, 2018 PTC Therapeutics, Inc. (NASDAQ: PTCT) today provided a corporate update, which will be detailed as part of the company’s presentation at the 36th Annual J.P. Morgan Healthcare Conference on Wednesday, January 10th at 2:30 pm PT (Press release, PTC Therapeutics, JAN 8, 2018, View Source [SID1234525048]). Stuart W. Peltz, Ph.D., PTC’s Chief Executive Officer, will highlight the company’s 20-year commitment to bring best-in-class therapies to patients affected by rare disorders, the company’s 2018 strategic priorities, preliminary 2017 financial results and 2018 financial guidance. The presentation will be webcast live on the Events and Presentations page under the investors section of PTC Therapeutics’ website at www.ptcbio.com.

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Preliminary 2017 Unaudited Financial Results

PTC expects to report Translarna (ataluren) net product revenue for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) of approximately $145 million for 2017, an increase of 78% over the prior year. This strong performance, which achieves the upper end of the company’s guidance for the full year 2017, reflects the rapid uptake and the high unmet need in this community. PTC continues to be pleased by the greater than 90% compliance rate of patients on therapy.
PTC expects to report EMFLAZA (deflazacort) net product revenue for the treatment of Duchenne muscular dystrophy (DMD) of approximately $29 million for 2017, 16% higher than the upper end of the company’s guidance for the full year 2017.
PTC expects to report year-end cash and cash equivalents of approximately $191 million.
2018 Guidance

PTC anticipates full-year net product revenues 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 (12/31/17-12/31/22) compound annual growth rate of 15% representing continued strong growth year-over-year of Translarna in existing countries and in expansion into new territories. PTC anticipates EMFLAZA net product revenue for the full year 2018 to be between $90 and $110 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2018 to be between $280 and $290 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2018 to be between $250 and $260 million, excluding estimated non-cash, stock-based compensation expense of approximately $30 million.
Corporate Highlights

Successful commercial launch of EMFLAZA for the treatment of Duchenne muscular dystrophy. PTC has established programs with the goal of ensuring that all eligible patients will have access to EMFLAZA regardless of financial or insurance status. PTC is committed to improving the standard of care for all Duchenne patients.
Continued strong growth of Translarna product revenue outside US in nonsense mutation Duchenne patients. PTC plans continued growth in Translarna ex-US business by increasing penetration in current countries, expanding into new geographies, and pursuing opportunities for label expansion.
As part of the US FDA appeal process for the Translarna NDA, a meeting is scheduled at the request of the Office of New Drugs and PTC plans to provide an update in the first quarter.
The SUNFISH trial in the spinal muscular atrophy (SMA) program transitioned to the pivotal portion in 2017 with FIREFISH anticipated to transition to the pivotal stage in the coming months. Survival data from FIREFISH study in Type 1 SMA patients will be presented at the upcoming SMA Europe International Scientific Congress in Krakow. The SMA program is a joint collaboration with Roche and the SMA Foundation.
PTC continues to expand its innovative pipeline with internal research programs in the company’s next generation readthrough platform, alternative splicing platform and key developments in oncology with two DHODH inhibitor compounds.
PTC to host an analyst day in the upcoming months to provide an update on its growing pipeline.
Non-GAAP Financial Measures:
In this press release, the unaudited 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 non-cash, stock-based compensation expense. This non-GAAP financial measure is provided as a complement to results 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 operating performance at PTC and the company’s future outlook. Quantitative reconciliations of these non-GAAP financial measures to GAAP financial measures are included in the table below.

PTC Therapeutics, Inc.

Reconciliation of Projected GAAP to Non-GAAP Full Year 2018 R&D and SG&A Expense (In thousands)

Low End of Range

High End of Range

Projected GAAP R&D and SG&A expense

280,000

290,000

Less: projected non-cash stock-based compensation expense

30,000

30,000

Total projected non-GAAP R&D and SG&A expense

$

250,000

$

260,000

Preliminary 2017 Financial Results:
PTC is currently in the process of finalizing its financial results for the 2017 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2017, subject to the completion of PTC’s financial closing procedures. In addition, the above information is subject to revision as PTC completes its financial closing procedures for fiscal 2017.

On January 8, 2018 Epizyme presented Company Slide Deck as of January 8, 2018 (Presentation, Epizyme, JAN 8, 2018, View Source [SID1234522982]).

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On January 8, 2018 TG Therapeutics, Inc. (NASDAQ:TGTX) reported that Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, will present at the 36th Annual J.P. Morgan Healthcare Conference, being held at the Westin St. Francis Hotel in San Francisco, CA (Press release, TG Therapeutics, JAN 8, 2018, View Source [SID1234523029]). The presentation is scheduled to take place on Thursday, January 11, 2018 at 7:30am PT. A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com.

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On January 8, 2018 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported that it has successfully manufactured the first SPEAR T-cells for a patient at its Navy Yard facility in Philadelphia (Press release, Adaptimmune, JAN 8, 2018, View Source;p=RssLanding&cat=news&id=2325396 [SID1234522941]). In addition, Adaptimmune announced an agreement with Cell and Gene Therapy Catapult for vector production in the UK, which will ensure vector supply for its ongoing and future clinical studies.

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"We are making great strides to becoming a fully integrated cell therapy company. Our Navy Yard facility is now fully operational producing SPEAR T-cells for patients. In addition, we have vector supply into 2019, and the initiation of our own vector manufacturing capability at the Catapult facility will extend vector supply further," said James Noble, Adaptimmune’s Chief Executive Officer. "We will continue to work with our cell manufacturing partner PCT, now part of Hitachi, where we have dedicated space and personnel for production of our SPEAR T-cells, as well as our other vector suppliers. Having these dedicated resources both in-house and through external partnerships is essential to ensure our future success as a fully integrated cell therapy company."

First SPEAR T-cells manufactured at the Navy Yard
The first SPEAR T-cells have been successfully manufactured by the Adaptimmune team at our own Navy Yard headquarters for a patient in the first dose cohort of the ongoing MAGE-A4 multiple tumor study in bladder, melanoma, head & neck, ovarian, non-small cell lung, esophageal, and gastric cancers.

The manufacturing facility at the Navy Yard can deliver cells for up to 300 patients per year, with the possibility of expansion that would enable manufacture for up to 1000 patients per year. In addition to production at its wholly-owned manufacturing facility at the Navy Yard, Adaptimmune will continue working with the PCT team to manufacture SPEAR T-cells.

Vector supply extended to beyond 2020
The agreement, which was executed on January 5, 2018 with Cell and Gene Therapy (CGT) Catapult, will enable Adaptimmune to have its own dedicated vector manufacturing space in the UK. It will ensure vector supply production beyond 2020 for ongoing studies with all three SPEAR T-cell therapies, MAGE-A4, MAGE-A10 and AFP.

The module, in which Adaptimmune will use its own novel vector manufacturing process and be responsible for operation of the manufacturing process, is located in the UK-based CGT Manufacturing Centre. The CGT manufacturing Centre is a Good Manufacturing Practice (GMP) facility designed to enable the development of commercial scale manufacturing systems in cell and gene therapy by offering a full suite of GMP facilities, support and expertise.

On January 8, 2018 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported the achievement of four development milestones for IPI-549, a first-in-class, oral, immuno-oncology product candidate targeting tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition, thereby reducing pro-tumor macrophage function and increasing anti-tumor macrophage function (Press release, Infinity Pharmaceuticals, JAN 8, 2018, View Source [SID1234522986]).

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First, the Phase1/1b monotherapy expansion component of the study has been fully enrolled. Second, the combination dose escalation component of the study has been completed. Third, six disease-specific combination expansion cohorts are open to enrollment at the recommended phase 2 dose of 40mg daily of IPI-549 plus Opdivo (nivolumab) at 240 mg every two weeks in patients with non-small cell lung cancer, melanoma, triple negative breast cancer, head and neck cancer, mesothelioma, and adrenocortical carcinoma. Fourth, the company announced today the expansion of its Phase 1/1b clinical trial of IPI-549 to include a combination cohort of IPI-549 plus Opdivo that will enroll patients with high baseline levels of myeloid derived suppressor cells (MDSCs). Studies have shown that poor response to checkpoint inhibitor therapy is correlated with the presence of high baseline levels of MDSCs in cancer patients.1 2 3 In addition, preliminary translational data from Infinity’s Phase 1/1b study demonstrated an association between high baseline levels of MDSCs and clinical responses. Enriching for patients with high MDSCs could lead to improved clinical activity for patients treated with the combination of IPI-549 and anti-PD1. Infinity expects to begin enrolling this combination expansion cohort in patients with high baseline levels of MDSCs in the first quarter of 2018.

Infinity also provided financial guidance for 2018 and outlined anticipated 2018 milestones for the development of IPI-549. During the year, the company expects to make substantial progress with the Phase 1/1b clinical study of IPI-549, which is designed to evaluate IPI-549 both as a monotherapy and in combination with Opdivo. Infinity plans to report data from the monotherapy expansion and combination dose escalation components and initial data from the combination expansion component of the Phase 1/1b study of IPI-549 with Opdivo in the second quarter of 2018. In the second half of 2018, Infinity expects to report more mature clinical data from the combination expansion component of the study including translational insights from paired tumor biopsies across multiple diseases.

"2018 will be a decisive year for both Infinity and IPI-549, as we look forward to reporting maturing data from the Phase 1/1b trial both in monotherapy and in combination with Opdivo at several medical meetings throughout the year, which will help to define our development and regulatory strategy for this first-in-class product candidate," stated Adelene Perkins, Infinity’s chair and chief executive officer. "IPI-549 represents a unique approach to targeting tumors through its effects on myeloid cells within the tumor microenvironment, and we are very pleased with the data to date. There is a significant need for better treatment options for patients, especially for patients who do not respond to, or develop resistance to, existing immunotherapies, as well as for types of cancer where there is limited benefit from treatment with checkpoint inhibitors. We look forward to presenting updates throughout 2018 from our Phase 1/1b clinical trial."

"As the majority of patients treated with IPI-549 monotherapy have advanced forms of cancer and received several therapies prior to enrollment in this study, it’s very encouraging to see single-agent activity, including a patient with a partial response who has remained on treatment for over a year and continues on study today," said Dr. David Hong from MD Anderson Cancer Center, Deputy Chair of the Department of Investigational Cancer Therapeutic. "IPI-549 has also been well tolerated with a favorable safety profile."

Infinity’s chair and chief executive officer, Adelene Perkins, will discuss the company’s continued execution on its corporate strategy and 2018 priorities as part of a podium presentation at the 36th Annual J.P. Morgan Healthcare Conference on Thursday, January 11, at 9:30 a.m. PST (12:30 p.m. EST). The presentation will be webcast on Infinity’s website, www.infi.com.

Anticipated Milestones in 2018

During 2018, Infinity expects to achieve the following IPI-549 data milestones:

• Report data from the monotherapy expansion component of the study in the second quarter of 2018

• Report data from the combination dose-escalation component of the study in the second quarter of 2018
• Report initial data from the combination expansion component of the study in the second quarter of 2018

• Report additional data from the combination expansion component, with more mature clinical and translational data, including insights from paired tumor biopsies, in the second half of 2018
2018 Financial Guidance

Infinity ended 2017 with approximately $57.6 million in cash and investments (unaudited) and plans to report its fourth quarter and full-year 2017 financial results in March. The company is providing the following financial guidance today:

• Net Loss: Infinity expects net loss for 2018 to range from $40 million to $50 million.

• Cash and Investments: Infinity expects to end 2018 with a year-end cash and investments balance ranging from $10 million to $20 million.

• Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities at December 31, 2017, will be adequate to satisfy the company’s capital needs into the first quarter of 2019.
Infinity’s financial guidance excludes additional funding or business development activities and does not include the potential $22 million payment from Verastem upon the first regulatory approval of duvelisib. Verastem has provided its expectation that it plans to submit a New Drug Application to the U.S. Food and Drug Administration for duvelisib in the first quarter of 2018.

About IPI-549

IPI-549 is an investigational first-in-class, oral, immuno-oncology product candidate targeting tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition, thereby reducing pro-tumor macrophage function and increasing anti-tumor macrophage function. In preclinical studies, IPI-549 reprograms macrophages from a pro-tumor (M2), immune suppressive function, to an anti-tumor (M1) immune activating function and can enhance the activity of, and overcome resistance to, checkpoint inhibitors.4 5 As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially additive or synergistic approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

The ongoing Phase 1/1b study being conducted by Infinity is designed to evaluate the safety, tolerability, activity, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with Opdivo in approximately 200 patients with advanced solid tumors.6 The four-component study includes monotherapy and combination dose-escalation components, in addition to monotherapy expansion and combination expansion components. The monotherapy dose-escalation component is complete and the monotherapy expansion component has been fully enrolled. The combination dose-escalation component is also complete, and combination expansion cohorts are open to enrollment.

The combination expansion component of the study includes multiple cohorts designed to evaluate IPI-549 in patients with specific types of cancer, including patients with non-small cell lung cancer (NSCLC), melanoma, and head and neck squamous cell carcinoma (HNSCC) whose tumors show initial resistance or initially respond to but subsequently develop resistance to immune checkpoint blockade therapy. The combination expansion component also includes a cohort of patients with triple negative breast cancer (TNBC) who have not been previously treated with immune checkpoint blockade therapy, a cohort of patients with mesothelioma, a cohort of patients with adrenocortical carcinoma and a cohort of patients with high baseline levels of MDSCs.

IPI-549 is an investigational compound, and its safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.