On January 7, 2019 Rainier Therapeutics, Inc., a privately-held clinical stage drug development company, reported that its targeted antibody vofatamab has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for treatment of patients with advanced or metastatic urothelial cell carcinoma (bladder cancer) that is positive for FGFR3 mutation and/or fusion (Press release, Rainier Therapeutics, JAN 7, 2019, View Source [SID1234532539]).

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"This Fast Track designation underscores the great unmet medical need that exists for the treatment of bladder cancer," said Scott Myers, Chairman and CEO of Rainier Therapeutics. "As the only antibody specifically targeted to FGFR3 we know to be in clinical development, we believe vofatamab offers a promising therapeutic option. We look forward to further data from our ongoing trials and working to advance our development efforts."

The FDA’s Fast Track designation is a process to facilitate development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Through the Fast Track program, more frequent meetings may be scheduled with the FDA to discuss the drug’s development plan and to ensure the collection of appropriate data needed to support approval. Additionally, the drug may qualify for accelerated approval and priority review and, at the time of a biologics license application (BLA) filing, the drug candidate’s sponsor may be eligible to submit completed sections of the BLA on a rolling basis before the complete application is submitted.

"Fast Track designation offers the potential to reduce development time and cost associated with bringing a drug to patients," said Valerie Fauvelle, Vice President, Regulatory Affairs at Rainier Therapeutics. "We look forward to working with the FDA to rapidly advance vofatamab through the clinical development and regulatory processes."

Vofatamab (B-701) is an antibody specifically targeted against the fibroblast growth factor receptor 3 (FGFR3), a known driver of bladder and other cancers. Vofatamab is the most advanced targeted biologic specific for FGFR3 known by Rainier to be in clinical development.

Rainier Therapeutics has ongoing Phase 1b and Phase 2 clinical studies of vofatamab in metastatic bladder cancer – the Fierce 21 and Fierce 22 studies. In addition, Rainier Therapeutics plans to study vofatamab in early stage bladder cancer – the Fierce 23 trial.

The Fierce 21 trial is evaluating vofatamab alone and in combination with docetaxel versus docetaxel alone to determine safety and efficacy in the treatment of patients with locally advanced or metastatic bladder cancer with FGFR3 mutant/fusion who have relapsed after, or are refractory to, at least one prior line of chemotherapy. For more on this trial, visit www.clinicaltrials.gov (NCT0240542).

The Fierce 22 trial is evaluating vofatamab in combination with pembrolizumab, an immune checkpoint inhibitor, to determine safety, tolerability and efficacy in the treatment of patients with locally advanced or metastatic bladder cancer, who have progressed following platinum-based chemotherapy and who have not received prior immune checkpoint inhibitor therapy. For more on this trial, visit www.clinicaltrials.gov (NCT03123055).

The Fierce 23 trial will evaluate vofatamab monotherapy in non-muscle invasive bladder cancer (NMIBC). This trial is planned to start in 2019.

On January 7, 2019 Unum Therapeutics Inc. (the "Company") is presented a corporate presentation (Presentation, Unum Therapeutics, JAN 7, 2019, View Source [SID1234532555]).

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On January 7, 2019 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update, which will be detailed as part of the company’s presentation at the 37th Annual J.P. Morgan Healthcare Conference on Tuesday, January 8th at 5:00 p.m. PT. Stuart W. Peltz, Ph.D., PTC’s Chief Executive Officer, will highlight the company’s growth and diversification focused on bringing clinically differentiated, life-changing treatments to patients affected by rare disorders (Press release, PTC Therapeutics, JAN 7, 2019, View Source [SID1234532571]). Additionally, an overview of the company’s strategic vision will be provided as well as preliminary 2018 financial results and 2019 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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Corporate Highlights

Advancing gene therapy portfolio

PTC plans to submit a BLA with the FDA followed by an MAA in Europe for the AADC deficiency gene therapy program in 2H 2019 with expected launch in 2020. Identification of patients with AADC deficiency has been a priority for the Company, with approximately 100 patients identified to date in the U.S. and Europe. PTC expects to screen about 100,000 patients who are at risk for AADC deficiency before the regulatory approval to maximize patient benefit at time of launch.

Friedreich’s ataxia program is advancing with an expected IND filing and entry into the clinic in 2019.

PTC is enhancing its internal research and in-house manufacturing capabilities with fully dedicated resources to maximize current and future programs.

Risdiplam regulatory filing progress. The SMA program is a collaboration between PTC, Roche and SMA Foundation.

Successfully completed enrollment of pivotal portion of FIREFISH trial in Type 1 SMA babies in 2018.

Regulatory submission targeted for the second half of 2019 based on recent feedback from the FDA & national health authorities in Europe that Part 1 of FIREFISH and SUNFISH may be sufficient to file NDA/MAA.

The goal of the clinical development program is to support the approval of a broad label so risdiplam can potentially benefit as many SMA patients as possible.

Short- and long-term cash milestones to PTC expected upon regulatory approval and commercial launches. PTC expects the peak annual royalties/milestones from risdiplam to exceed $200M.

Expanding commercial platform

TEGSEDI application filed with ANVISA- Brazilian regulatory authority. ANVISA granted priority review. PTC expects approval in Brazil by year end 2019. TEGSEDI has the potential to generate peak revenues of approximately $150M in LATAM.

Duchenne franchise expected to continue to grow over the next 5 years. Translarna ex-U.S. launch in patients 2 to 5 years of age now initiated. Non-ambulatory label expansion is currently under EMA regulatory review. Emflaza continues to accrue new patients in the U.S. with expected increased market share gained via differentiation and improved market access.

In recent interactions, the FDA invited PTC to submit a supplementary NDA (sNDA) for Emflaza for patients 2 to 5 years of age on the basis that existing data support its safety and efficacy in this population. PTC recently submitted the sNDA for potential approval in 2019. The previous written request from the FDA that a trial in patients 2 to 5 years of age be performed has been officially withdrawn and the trial will no longer be conducted. PTC now expects to launch Emflaza in this younger population before the end of 2019.

Growing pipeline and R&D capabilities

PTC’s alternative splicing platform has generated another development candidate. PTC258 was selected as a development candidate for Familial dysautonomia (FD), a rare genetic neurological disorder that effects the sensory and autonomic nervous systems, causing life-threatening medical complications from birth. PTC258 is advancing to IND enabling studies with the goal to enter the clinic in late 2019. This program is in collaboration with MGH and NYU.

Translarna’s dystrophin study was initiated in 4Q 2018 for potential U.S. regulatory submission in early 2020.

PTC’s oncology portfolio continues to advance with the initiation of a study in AML with PTC299 and a DIPG study for PTC596. PTC expects these studies to move to the expanded cohort stage in 2020. PTC596 is also being developed for the treatment of patients with Leiomyosarcoma (LMS) with the first patient in that trial expected to be dosed in Q1 2019.

Preliminary Unaudited 2018 Financial Results

PTC expects to report Translarna (ataluren) net product revenue for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) of approximately $171 million for full year 2018, an increase of 18% over the prior year.

PTC expects to report EMFLAZA (deflazacort) net product revenue for the treatment of Duchenne muscular dystrophy (DMD) of approximately $91 million for full year 2018, an increase from $28.8 million in the prior year.

PTC expects to report year-end cash and cash equivalents of approximately $227 million.

PTC is currently in the process of finalizing its financial results for the 2018 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2018, 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 2018.

2019 Guidance

PTC anticipates full year net product revenues to be between $285 and $305 million.

PTC anticipates GAAP R&D and SG&A expense for the full year 2019 to be between $395 and $405 million.

PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2019 to be between $360 and $370 million, excluding estimated non-cash, stock-based compensation expense of approximately $35 million.

On January 7, 2019 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immuno-oncology biopharmaceutical company focused on the acquisition, development and commercialization of novel treatments for patients with solid tumor cancers, reported that the ongoing multi-center clinical trial of CK-301 has been expanded to enroll patients in three cohorts intended to support requests for accelerated approval and Biologics License Application (BLA) submissions to the U.S. Food and Drug Administration (FDA) (Press release, Checkpoint Therapeutics, JAN 7, 2019, View Source [SID1234532508]). These cohorts include:

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• Microsatellite instability-high (MSI-H) endometrial cancer that has progressed following one or two prior anti-cancer therapies;

• Microsatellite stable (MSS) endometrial cancer that has progressed following one or two prior anti-cancer therapies; and

• MSI-H or mismatch repair deficient (dMMR) colorectal cancer that has progressed on or after, or been intolerant of, previous treatments, including a fluoropyrimidine-and oxaliplatin-and irinotecan-based chemotherapy.

Each cohort is evaluating a fixed dose of 800 mg CK-301 every two weeks (Q2W). The primary endpoint for each cohort is objective response rate (ORR), and secondary endpoints include duration of response (DOR), progression-free survival (PFS), and overall survival (OS). The ongoing trial is also enrolling cohorts of patients with non-small cell lung cancer (NSCLC) and cutaneous squamous cell carcinoma.

"We are excited to advance our first immuno-oncology drug candidate, CK-301, into these potentially registration-enabling cohorts, representing a significant milestone in the execution of our strategy to obtain multiple accelerated approvals for our anti-PD-L1 antibody," said James F. Oliviero, President and Chief Executive Officer of Checkpoint. "We look forward to presenting interim safety and efficacy data from the ongoing clinical trial in the coming months."

The Phase 1, open-label, multi-center trial is evaluating the safety and tolerability of ascending doses of CK-301 in checkpoint therapy-naïve patients with selected recurrent or metastatic cancers. Following completion of dose escalation in 2018, multiple dose expansion cohorts were initiated. Preliminary data from the ongoing trial suggest that CK-301 is safe and well-tolerated across dose levels ranging from 200 mg to 800 mg administered every two weeks and 1200 mg administered every three weeks, with treatment-related adverse events consistent with marketed anti-PD-1/PD-L1 antibodies.

About CK-301
CK-301 is a fully-human monoclonal antibody of IgG1 subtype that directly binds to Programmed Death Ligand-1 (PD-L1) and blocks the PD-L1 interaction with the Programmed Death Receptor-1 (PD-1) and B7.1 receptors. PD-L1 is an immune-inhibitory checkpoint molecule expressed on epithelial and vascular endothelial cells, as well as by a number of immune cells, and is utilized by tumor cells as an immune escape mechanism. CK-301’s primary mechanism of action is based on the inhibition of the interaction between PD-L1 and its receptors PD-1 and B7.1, which removes the suppressive effects of PD-L1 on anti-tumor CD8+ T-cells to restore the cytotoxic T cell response

On January 7, 2019 Coherus BioSciences, Inc. (Nasdaq: CHRS), a commercial biosimilar company, reported that it has entered into a $75 million senior secured credit facility agreement with Healthcare Royalty Partners (Press release, Coherus Biosciences, JAN 7, 2019, View Source [SID1234532524]).

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"We are pleased to have the renewed support of HealthCare Royalty Partners," said Denny Lanfear, President and CEO of Coherus. "This financing bolsters our cash position and working capital, enabling us to accelerate and enhance the manufacture and sale of UDENYCA (pegfilgrastim-cbqv), which we launched January 3, 2019. We look forward to providing significant value to cancer patients, oncology clinics and hospitals, as well as providing significant savings to payers."

"This investment reflects our continued confidence in the Coherus leadership team, as well as their ability to execute on their commercial plan," said Clarke Futch, Managing Partner and Chairman of the Investment Committee of Healthcare Royalty Partners. "We are honored to help Coherus in its mission to serve patients and deliver savings to the healthcare system. With this second investment in Coherus, we look forward to their continued growth and success."

The senior secured credit facility matures on January 7, 2025 and pays interest quarterly at a rate of 3–month LIBOR plus 7% per annum. Subject to other customary fees, the senior secured credit facility will also be subject to a 4% final payment premium owed on the entire principal amount funded.