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]).

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!

"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]).

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!

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.

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!

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 Exscientia the world-leading Artificial Intelligence (AI)-driven drug discovery company reported it has raised US$26 million in a Series B financing round (Press release, Exscientia, JAN 7, 2019, View Source [SID1234539638]). This will be used to scale the company’s pipeline and advance selected programmes towards clinical development. The round included participation from new investors Celgene Corporation and specialist healthcare investor GT Healthcare Capital Partners, as well as existing investor Evotec AG.

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!

Alan Au, Managing Partner of GT Healthcare Capital Partners, commented: "We are very excited to back Exscientia and partner with a world-class team to further extend its business, including in Asia. We have a shared vision to revolutionize traditional drug development processes and believe Exscientia’s unique capabilities have the potential to deliver well beyond cost-effectiveness and speed, to first-in-class and best-in-class drugs that benefit patients in the near future."

Dr Rupert Vessey, President of Research and Early Development at Celgene, said: "Exscientia has demonstrated that AI in molecular design is here today. With the global pharmaceutical industry acknowledging the importance of incorporating AI-driven R&D approaches into their drug discovery processes, we see a huge growth opportunity ahead. We believe Exscientia is set to become a global leader in AI-driven drug discovery and are excited to participate in this investment round."

Professor Andrew Hopkins, CEO and founder of Exscientia, said: "This Series B marks a milestone in our development and enables us to drive the next phase of strong business growth. Over the past 12 months we have substantially expanded our operations and capabilities to become a full stack AI drug discovery company. Furthermore, our unique Centaur Chemist platform allows us to move rapidly from idea generation to new drug molecules ready for IND and clinical development. With this new funding Exscientia is positioned to become the dominant player in AI drug discovery, driving radical change in R&D productivity. We are excited Celgene and GT Healthcare have joined with existing investor Evotec on this exciting journey."

The company has made considerable progress during 2018 and anticipates its first programmes driven by AI to be IND-ready within the next 12 months. The speed and capital efficiency demonstrated by Exscientia is already revolutionising the economics of small molecule discovery and in the future, this improved throughput will deliver benefits to patients.

Dr Werner Lanthaler, Chief Executive Officer of Evotec, said: "We continue to be very impressed with the progress Exscientia has made over the past year. Through our partnership with Exscientia we have seen first-hand evidence that they can deliver the most productive drug discovery engine in the industry. This latest funding will allow Exscientia to apply its platform at scale, taking advantage of the efficiencies that its AI-driven systems provide."

Exscientia will use the proceeds of this financing round to grow its "full stack" AI drug discovery capability to dramatically expand its pipeline, with a target of establishing an expansive portfolio of projects, both in-house and with partners by the end of 2019.

Alex Snow, Executive Chairman of Exscientia, said: "Exscientia has validated its technology by delivering multiple pre-clinical drug candidates with unprecedented efficiency. Exscientia’s platform has the potential to bring a generation of new medicines to the market by offering a technologically and financially superior approach to drug discovery that will benefit of patients. I am personally very excited to be part of Exscientia’s next stage of development, and to have the backing of such prestigious investors."

Exscientia were advised by Rothschild & Co on this transaction.

On January 07, 2019 CytoReason, a leader in machine learning for drug discovery and development, reported that it has entered into a collaboration agreement with Pfizer Inc. that will leverage CytoReason’s cell-centered models of the immune system (Press release, CytoReason, JAN 7, 2019, View Source [SID1234619714]).

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!

CytoReason will receive from Pfizer payments potentially equaling up to low double digit millions of US$ for technology access fees, research support and certain success-based payments.

CytoReason’s proprietary platform helps rebuild lost cellular information from gene expression data and associates genes to specific cells. This information is then integrated with additional omics and literature data to create a cell-based model of the trial-specific immune response. Integration with the CytoReason disease model empowers the study analytics and allows the model to learn and improve, leading to robust target discovery, drug response biomarkers and indication selection.

"We believe that CytoReason’s platform has the potential to offer valuable insights that may be applied to our research into the human immune system," said Michael Vincent, Chief Scientific Officer, Inflammation & Immunology, Pfizer. "Leveraging technologies such as this can help us understand disease and prioritize targets, and support our mission of bringing innovative new therapies to patients who need them."

"The collaboration with Pfizer will further strengthen our models in our core therapeutic areas. This will be our fifth major partnership, which we believe will help make our model unparalleled in its accuracy for assets across the pipeline," said David Harel, CytoReason’s CEO. "CytoReason’s model brings together thousands of samples on a cell-protein-gene level, allowing for fast and accurate insights."

About CytoReason

Based on more than 10 years of research, CytoReason’s technology uses a proprietary data and machine learning model to reconstruct cellular information from bulk tissue, to train an immune-specific NLP engine, and to integrate multi-omics data. The company’s platform organizes and standardizes collaborators’ data (gene, protein, cell, and microbiome) and integrates it into CytoReason’s proprietary disease model to generate mechanistic understanding of the immune system, leading to novel insights.

CytoReason’s technology has yielded 2 pending patents, 10 commercial and scientific collaborations and 16 peer reviewed publications. Fully applicable to cancer immunotherapy, autoimmune, neurodegenerative and infectious disease research, CytoReason is at the cutting edge of society’s boldest attempts to improve health outcomes through better understanding of the immune system.