On January 13, 2020 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, reported the appointment of Dr. Elliot Norry as Senior Vice President and Chief Medical Officer (CMO) effective immediately, and additional changes to its R&D organization (Press release, Adaptimmune, JAN 13, 2020, View Source [SID1234553082]).

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"Elliot has done a fantastic job as acting CMO over the past few months. This builds on his impact leading the ADP-A2AFP program in liver cancer as well as leading our safety and pharmacovigilance team over the last four years. I look forward to continuing to work with Elliot to deliver cell therapy treatments to patients with cancer," said Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer. "In addition, we have made changes to our R&D leadership to accelerate how our products move from research to late stage development, including rapid application of translational learnings that are crucial to bringing cell therapies to patients."

Dr. Elliot Norry

Dr. Norry has served as Head of Clinical Safety and Pharmacovigilance and leader of the ADP-A2AFP program since 2015. He has been acting Chief Medical Officer since August 2019. Prior to joining Adaptimmune, he served as Safety Development Leader and was Chair of the Hepatic Safety Panel at GSK. Prior to his work in the biotech and pharmaceutical industries, Dr. Norry practiced adult internal medicine at Abington Memorial Hospital in Abington, Pennsylvania for 13 years. He holds a B.A. from Columbia College and an M.D. from New York University. He performed his residency in Internal Medicine at Temple University Hospital, Philadelphia and GI fellowship at Thomas Jefferson University Hospital, Philadelphia.

R&D leadership

Changes to the R&D Leadership that will strengthen the end-to-end scientific and clinical development from early to late stage, include:

·An Early Stage Development group, led by Mark Dudley, will evaluate therapies in Phase 1 studies for safety as well as determining their potential for efficacy and further clinical development. Mark becomes Senior Vice President (SVP), Early Stage Development and previously served as SVP, Product Development.
·A Late Stage Development group, led by Dennis Williams, will take products through Phase 2/3 trials and registration. The first of these trials is SPEARHEAD-1 with ADP-A2M4 – currently open for recruitment of patients with synovial sarcoma or myxoid/round cell liposarcoma. Dennis has been promoted to SVP, Late Stage Development and will continue leading the regulatory affairs team.
·Joanna Brewer has been promoted to SVP, Allogeneic Research and will continue leading the allogeneic discovery work.
·The Pipeline Research team will continue to be led by Karen Miller, SVP Pipeline Research.

On January 13, 2020 SELLAS Life Sciences Group, Inc. (Nasdaq: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications, reported its clinical development progress for galinpepimut-S (GPS) and nelipepimut-S (NPS) and outlined expected milestones for 2020 (Press release, Sellas Life Sciences, JAN 13, 2020, View Source [SID1234553081]).

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"We made significant clinical and corporate advances in 2019 and early this month, most notably in our preparations for our Phase 3 registrational study (REGAL study) for GPS in acute myeloid leukemia in patients who achieved complete remission after second-line anti-leukemic therapy (CR2) which has now commenced with patient screening underway," said Angelos M. Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "We are excited to continue progressing our GPS program in 2020; in addition to the REGAL study, we remain focused on advancing GPS in combination with PD-1 agents, pembrolizumab and nivolumab. We are also expecting guidance from the U.S. Food and Drug Administration (FDA) within this quarter regarding our NPS program in triple negative breast cancer after recent discussions with the agency. We are indeed excited about the progress we made in 2019 and early 2020 both, clinically as well as on the corporate level, and look forward to advancing our pipeline of immunotherapies and creating value for our shareholders."

Dr. Stergiou will present a corporate overview at the Biotech Showcase on Monday, January 13, 2020 at 9:30 a.m. PT (12:30 p.m. ET) in San Francisco, CA. A live audio webcast of the presentation will be available under "Events & Presentations" in the Investors section of SELLAS’ website at www.sellaslifesciences.com/investors. A replay of the webcast will be available for up to 30 days on SELLAS’ website following the presentation.

2019 and Early 2020 Highlights

Galinpepimut-S (GPS)

In January 2020, the Company announced that it commenced the Phase 3 (REGAL) clinical trial of GPS in AML and has begun patient screening. The Company expects that the first patient will be treated in February. The pivotal Phase 3 REGAL study is a 1:1 randomized, open-label study comparing GPS monotherapy in the maintenance setting to investigators’ choice of best available treatment in AML patients who have achieved hematologic complete remission, with or without thrombocytopenia (CR2/CR2p), after second-line antileukemic therapy and who are deemed ineligible for or unable to undergo allogeneic stem-cell transplantation. The study is expected to enroll approximately 116 patients across approximately 50 clinical sites in the United States and Europe. The primary endpoint is overall survival (OS) from the time of study entry. The Phase 2 study in AML CR2 patients, which is the same indication as the Phase 3 REGAL study, showed a 10.9 months median OS benefit (16.3 months vs. 5.4 months in contemporaneously treated patients with best standard therapy; p-value=0.0175).

In November 2019, SELLAS announced that follow-up data from its Phase 1 clinical trial of GPS in combination with nivolumab to treat Wilms Tumor 1 (WT1) positive patients with ovarian cancer in second- or third-line remission continues to support the development of GPS in combination with PD-1 inhibitors. Topline data from this study at 10 months had been presented at the June 2018 meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). The follow-up data show that three of the 11 patients enrolled in the study have continued to show no signs of disease progression. The mean progression free survival (PFS) for these three patients is 35.4 months from the initiation of salvage chemotherapy or mean PFS of 30.1 months from the first administration of GPS plus nivolumab. Based on this follow-up information, the estimated two-year PFS rate for this study is now 27.3% for the intent-to-treat (ITT) patients (n=11) and approximately 30% for patients who received greater than two doses of GPS and nivolumab (n=10), as compared to a historical 3% to 10% PFS rate for patients receiving only salvage chemotherapy. No new serious adverse events were noted during the longer follow-up period.

In November 2019, SELLAS hosted its first R&D KOL Symposium with internationally renowned experts on immuno-oncology and myeloid malignancies, including Dr. Hagop M. Kantarjian, MD, Professor and Chair of the Department of Leukemia at the University of Texas MD Anderson Cancer Center, and global principal investigator of the Phase 3 AML REGAL clinical trial.

In July 2019, SELLAS announced the dosing of the first patient in its Phase 1/2 open-label study of GPS in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab), in patients with selected WT1-positive advanced cancers, including both solid tumors and hematologic malignancies. The two initial indications being studied in this basket study, with approximately 30 patients in total in these indications, are ovarian cancer (second or third line) and colorectal cancer (third or fourth line). The Phase 1/2 open-label, multicenter, multi-arm study is being conducted under a Clinical Trial Collaboration and Supply Agreement with Merck (known as MSD outside the United States and Canada) to assess the efficacy and safety of the combination of GPS and KEYTRUDA. The primary endpoints of the study include safety and overall response rate, while secondary endpoints include progression-free survival, overall survival and immune response correlates. The study is expected to enroll approximately 90 patients across all arms at up to 20 centers in the U.S. SELLAS expects an interim analysis from this study in the second half of 2020.

In April 2019, SELLAS announced an agreement with Memorial Sloan Kettering Cancer Center (MSK) for the conduct of an investigator-sponsored clinical trial (IST) of GPS in combination with Bristol-Myers Squibb’s anti-PD-1 therapy, nivolumab, in patients with malignant pleural mesothelioma (MPM). The Phase 1 open-label clinical study will enroll patients with MPM who harbor relapsed or refractory disease after having received frontline standard of care multimodality therapy. SELLAS has been informed by MSK that it expects the first patient to be dosed in this study in January 2020.
Nelipepimut-S (NPS)

Based on promising Phase 2b data presented in 2018, SELLAS is in continued active discussions with the FDA regarding the optimal development path for NPS in triple negative breast cancer (TNBC). The Company provided supplemental information to the FDA in September 2019 and, after recent discussions with the agency, expects feedback from the FDA within the first quarter of 2020.

In 2019, SELLAS presented immune response data from the TNBC cohort of the Phase 2b study at the 55th Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and presented final results from an efficacy and safety data analysis of the Phase 2b study at the 2019 ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium.

In August 2019, SELLAS announced the completion of enrollment in a Phase 2 randomized IST of NPS in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) in women with ductal carcinoma in situ (DCIS) of the breast who are HLA-A2+ or A3+ positive, express HER2 at IHC 1+, 2+, or 3+ levels, and are pre- or post-menopausal. Initial data from this trial are expected in the first quarter of 2020.
Expected 2020 Clinical Milestones

First patient dosed in the Phase 3 registration-enabling study of GPS in AML patients in February 2020.
First patient dosed in the Phase 1 open-label study of GPS with nivolumab in MPM patients in January 2020.
Interim analysis of the Phase 1/2 basket study of GPS with pembrolizumab in multiple tumor types in the second half of 2020.
Guidance from the FDA on the regulatory and development pathway for NPS in TNBC patients in the first quarter of 2020.
Initial data from Phase 2 trial of NPS in DCIS in the first quarter of 2020.
Corporate

On January 9, 2020, the Company announced that it had entered into a securities purchase agreement with institutional investors to purchase approximately $6.5 million of its common shares (or pre-funded warrants to purchase common shares in lieu thereof) in a registered direct offering priced at-the-market under Nasdaq rules and warrants to purchase common shares in a concurrent private placement. The combined purchase price for one common share (or pre-funded warrants to purchase common shares in lieu thereof) and a warrant to purchase 0.5 common shares is $3.9825.
In June 2019, SELLAS announced the closing of an underwritten public offering of (i) 527,344 shares of common stock together with common stock warrants to purchase 527,344 shares of common stock and (ii) 1,472,656 pre-funded warrants, with each pre-funded warrant exercisable for one share of common stock, together with common warrants to purchase 1,472,656 shares of common stock, for net proceeds of approximately $13.4 million.
Keytruda is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, N.J., USA., and is not a trademark of SELLAS. The manufacturer of this brand is not affiliated with and does not endorse SELLAS or its products.

On January 13, 2020 Cerus Corporation (Nasdaq:CERS) reported preliminary product revenue for the fourth quarter and full year 2019 and provided 2020 product revenue guidance (Press release, Cerus, JAN 13, 2020, View Source [SID1234553080]).

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Cerus’ unaudited preliminary product revenue for the fourth quarter of 2019 totaled $20.9 million, an increase of 27%, compared to the $16.5 million recognized during the same period in the prior year. Based on its fourth quarter unaudited preliminary product revenue, the Company expects full year 2019 product revenue of $74.6 million, at the top end of the Company’s most recent 2019 product guidance range of $72 million to $75 million. The preliminary product revenue results have not been audited and are subject to change.

Preliminary fourth quarter product revenue would represent the highest quarterly product revenue ever reported by Cerus.

"We finished 2019 with momentum with preliminary fourth quarter product revenue up 27% driven by strong INTERCEPT platelet kit demand in North America and platelet and plasma kit demand in EMEA. We expect the robust sales growth experienced during the past year to continue into 2020 driven in part by the need for U.S. blood centers and hospitals to comply with the final FDA guidance document on bacterial safety," said William ‘Obi’ Greenman, Cerus’ president and chief executive officer.

"We are looking forward to an exciting year as we continue to help our blood center partners safeguard their blood component production against known and unknown infectious disease. With the planned increase in product revenue and margins, and continued leverage we expect from our SG&A investments, we anticipate lower cash use from operations during 2020 compared to 2019. In addition, we are looking forward to the anticipated expansion of our product portfolio with our first biologic therapeutic product, pathogen-reduced cryoprecipitate, which has been granted FDA breakthrough device designation for the treatment of critically bleeding patients. An anticipated U.S. regulatory submission is planned for the first half of 2020, with a potential approval decision in the second half of the year," continued Greenman.

Given the 2019 strength and anticipated increase in platelet kit demand as U.S. blood centers become compliant with the FDA guidance document, the Company expects full year 2020 product revenue will be in the range of $89 million to $93 million, representing growth of approximately 20% to 25% compared to preliminary unaudited 2019 full year results.

Cerus will provide complete fourth quarter and full year 2019 financial results and host a call to discuss both 2019 results and 2020 expectations in late February.

On January 13, 2020 Coherus BioSciences, Inc. ("Coherus" or the "Company", Nasdaq: CHRS), reported the Company has entered into a licensing agreement with Innovent Biologics, (Suzhou) Co., Ltd., ("Innovent"), a leading biopharmaceutical company headquartered in China, to commercialize Innovent’s biosimilar candidate to Avastin (bevacizumab) in the United States and Canada (Press release, Coherus Biosciences, JAN 13, 2020, View Source [SID1234553079]). Coherus plans to file a Biologics License Application ("BLA") with the U.S. Food and Drug Administration ("FDA") in late 2020 or early 2021 depending on FDA interaction timing, and to launch directly upon approval. The Company anticipates completing a single dose pharmacokinetic clinical study and certain analytical/bioanalytical exercises to support the U.S. filing. Innovent’s Avastin biosimilar successfully completed a large Phase 3 safety and efficacy study in China, and the application was filed for approval and was accepted by the National Medical Products Administration ("NMPA") in China in January 2019, and subsequently granted priority review status. Also, under the terms of the agreement, Coherus acquired an option to commercialize Innovent’s biosimilar to Rituxan (rituximab) in the United States and Canada. Innovent’s Rituxan biosimilar was filed for approval and accepted by the NMPA in China in June 2019, and subsequently granted priority review status.

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The agreement furthers the Company’s previously-stated strategic objective to expand its oncology franchise and leverage its commercial infrastructure in the United States. Coherus expects to apply its biosimilar regulatory expertise to convert the Chinese regulatory filing into a FDA biologics application.

"We are excited to enter into a strategic collaboration with one of the premier Chinese biologics companies," said Denny Lanfear, Chairman and Chief Executive of Coherus. "Innovent is an impressive fully-integrated organization delivering substantial benefits to the healthcare system and patients in China with their first approved and successfully commercialized PD-1. Its oncology therapeutics complement UDENYCA and advance our core mission to expand choice, improve patient access and lower healthcare costs in the United States."

"UDENYCA has been the most successful biosimilar launch in the United States and has made Coherus the obvious partner of choice," said Michael Yu, Ph.D., Founder, Chairman and CEO of Innovent. "Coherus and Innovent share a very similar mission, vision and set of values. We are pleased and proud to be working together."

Oncology is one of three focus areas for the Company along with ophthalmology and immunology. In the fourth quarter of 2019, Coherus announced it had acquired rights to commercialize BioEq’s Lucentis biosimilar in the United States, the lead asset in Coherus’ ophthalmology franchise.

According to the terms of the agreement with Innovent, Coherus will pay up to $45 million in milestones, including the upfront, for its Avastin biosimilar upon achieving certain regulatory and commercialization goals, as well as customary double-digit royalty payments. Financial terms for Innovent’s Rituxan biosimilar will be the same when optioned.

On January 13, 2020 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update that will be presented at the 38th Annual J.P. Morgan Healthcare Conference on Wednesday, January 15th at 8:30 a.m. PST. Stuart W. Peltz, Ph.D., PTC’s Chief Executive Officer, will provide an update on the 2019 accomplishments and highlight value creating events in the upcoming year (Press release, PTC Therapeutics, JAN 13, 2020, View Source [SID1234553078]). Additionally, the Company is providing preliminary 2019 financial results and 2020 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:

Gene Therapy Platform Update:

PTC has submitted a Marketing Authorization Application (MAA) for the potential approval of a gene therapy treatment, PTC-AADC, for AADC deficiency with the European Medicines Agency (EMA). PTC expects the Committee for Medicinal Products for Human Use (CHMP) opinion in 2H 2020.

In a recent interaction with the U.S. Food and Drug Administration (FDA) there was a request for additional information concerning the use of the commercial delivery system for PTC-AADC in young patients. Patients have been identified who are immediately available for treatment with the commercial delivery system. We anticipate that this will cause a short delay of the Biologics License Application (BLA) and should allow for submission to the FDA in 2Q 2020.

PTC has identified 200 AADC patients to date and continues to anticipate that over 300 AADC patients will be identified by launch.

PTC-FA gene therapy for Friedreich ataxia is progressing and is anticipated to enter the clinic in 3Q 2020.

In order to control and accelerate its gene therapy platform, PTC secured a 15-year lease on ~185,000 sq. ft. of space, which includes a state-of-the-art biologics production facility with supporting research and operations buildings in NJ. PTC expects manufacturing to begin at this facility in 2020.

Risdiplam Regulatory & Clinical Updates:

The risdiplam Prescription Drug User Fee Act (PDUFA) date for a decision by the FDA is May 24, 2020. Risdiplam is expected to be indicated for spinal muscular atrophy (SMA) type 1, 2 & 3 patients, if approved.

In November 2019, PTC announced that the pivotal part of the SUNFISH trial in SMA type 2/3 patients was successful, meeting its primary endpoint. Data from the pivotal part of SUNFISH will be presented at the SMA Europe Conference from February 5 – 7, 2020.

Topline results from the pivotal portion of the open label FIREFISH study in type 1 SMA patients are anticipated in 1Q 2020.

The SMA program has studied the broadest cohort of SMA patients in clinical trials to date. This program is a collaboration between PTC, Roche and the SMA Foundation.

Expanding commercial platform:

The Duchenne muscular dystrophy (DMD) franchise, consisting of Translarna and Emflaza, continues to grow with 2019 unaudited revenue of approximately $291 million.

Translarna continues to grow with further penetration into existing territories, geographic expansion into new territories, increased awareness and earlier diagnosis. PTC plans to re-submit the Translarna New Drug Application (NDA) to the FDA with the data from the dystrophin study in mid-year 2020.

Growth in Emflaza revenue was based on a recent label expansion in the 2-5-year-old patient population, and reduced payer restrictions resulting from multiple publications showing benefit of Emflaza relative to prednisone. One such recent publication, which included real-world data from Cincinnati Children’s hospital, specifically highlighted the benefit of switching from prednisone to Emflaza.

Tegsedi (inotersen) received approval from the Brazilian health regulatory authority (ANVISA) for the treatment of stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR), to delay disease progression and improve quality of life. The commercial launch for Tegsedi is ongoing with patient and physician services to support the launch, including medical education and diagnosis efforts.

Growing pipeline and R&D capabilities:

In 2020, PTC plans to initiate three trials in its newly acquired redox platform with two unique compounds that regulate inflammation and oxidative stress.

These include potential registrational trials in mitochondrial epilepsy and Friedreich ataxia with PTC743. Additionally, a Phase 1 trial with PTC857 is also planned targeting GBA Parkinson’s disease.

In late 2020, PTC expects to file an investigational new drug (IND) application for its development candidate in Huntington disease. PTC has several additional programs from the splicing platform in development with undisclosed targets.

Preliminary Unaudited 2019 Financial Results

Total unaudited revenue for 2019 was approximately $306 million. This includes expected net product revenue for the Duchenne muscular dystrophy franchise of approximately $291 million for the full year 2019 and a $15 million milestone payment from Roche for the risdiplam NDA acceptance.

PTC expects to report net product revenue for Translarna of approximately $190 million for full year 2019. As a reminder, the ANVISA approval in Brazil should lead to expanded market access in 2020 and beyond but led to a price discount that impacted 2019 net sales.

Additionally, PTC expects to report net product revenue for Emflaza of approximately $101 million for the full year 2019.

PTC expects to report 2019 year-end cash and cash equivalents of approximately $686 million.

PTC is currently in the process of finalizing its financial results for the 2019 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2019, 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 2019.

2020 Guidance

PTC anticipates full year net product revenues for the DMD franchise to be between $320 and $340 million. New product launches, including Tegsedi, Waylivra, PTC-AADC and risdiplam, could contribute revenue in 2020. Remaining milestones and royalties from Roche on the SMA program are outlined in the table below.

PTC anticipates GAAP R&D and SG&A expense for the full year 2020 to be between $610 and $640 million.

PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2020 to be between $545 and $575 million, excluding estimated non-cash, stock-based compensation expense of approximately $65 million.

The increase in R&D and SG&A expense are based in part on highly leverageable and scalable investments towards the $1.5 billion projected revenue in 2023, including gene therapy manufacturing, an increase in the number of programs advancing into the clinic and launches.