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 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.

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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 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.

On January 7, 2019 Generex Biotechnology Corporation (OTCQB:GNBT) is reported that Joe Moscato, the company’s CEO is attending the annual J.P. Morgan Healthcare Conference from January 7 to 9, 2019 (Press release, Generex, JAN 7, 2019, View Source [SID1234532540]). Mr. Moscato is meeting with leading healthcare investors to present the corporate vision for Generex Biotechnology as an integrated healthcare holding company with a unique strategy to provide end-to-end, patient centric solutions to enhance the doctor patient relationship.

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With 9,000 attendees and representation from over 450 companies, the annual J.P. Morgan Healthcare Conference is the largest and most informative healthcare investment symposium in the industry, bringing together industry leaders, emerging fast-growth companies, innovative technology creators, and members of the investment community.

"We are pleased to unveil the "New" Generex to the biotech and healthcare investment community," stated Joe Moscato, CEO of Generex. "After two-plus years of reorganizing the operations, capital structure, and strategic vision for the company, Generex is poised to deliver on our corporate mission to provide physicians, hospitals, and healthcare providers an end-to-end solution for patient centric care from rapid diagnosis through delivery of personalized therapies, streamlining care processes, minimizing expenses, and delivering transparency for payers. Entering 2019, we are building value in Generex by executing on our strategic plan with our newly acquired MSO and pharmacy network, the advancement of our clinical stage assets in immune-oncology, and through acquisitions in cutting-edge areas like regenerative medicine."