On January 10, 2022 Selecta Biosciences, Inc. (Nasdaq: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses, and Ginkgo Bioworks (NYSE: DNA), the leading horizontal platform for cell programming, reported a collaboration to develop safer and more effective next-generation gene therapy viral capsids (Press release, Selecta Biosciences, JAN 10, 2022, View Source [SID1234598506]). This partnership leverages the unique platforms of both companies as they seek to develop next-generation viral capsids with improved transduction, enhanced tissue tropism and reduced immunogenicity. Ginkgo plans to design and engineer the capsids and Selecta will conduct all pre-clinical and clinical studies thereafter.

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"We are excited to continue our partnership with Ginkgo in a second collaboration that leverages Ginkgo’s leading cell engineering platform to design viral vector capsids with the goal of improved transduction, enhanced tissue tropism and reduced immunogenicity," said Carsten Brunn, CEO of Selecta Biosciences. "By combining ImmTOR with next-generation, fit-for-purpose AAV vectors, we hope to further improve the safety and efficacy of AAV-mediated gene therapies."

Jason Kelly, CEO of Ginkgo Bioworks, added, "Gene therapies utilize naturally occurring viral capsids that elicit immune responses and often exhibit safety and toxicity issues. We believe our collaboration with Selecta has the potential to confront major challenges within the gene therapy space and represents an important application of our cell programming platform. We look forward to executing on our shared vision and offering a potential solution for challenges in gene therapies in order to enable treatment for millions of patients."

Under the terms of the collaboration, Ginkgo is eligible to earn upfront research and development fees and milestone payments, including certain milestone payments in the form of Selecta common stock. In addition, Ginkgo is eligible to earn clinical, regulatory and commercial milestone payments of up to $200 million in cash for each of a specified number of products, which have the potential to total, in the aggregate, up to $1.1 billion. Ginkgo is also entitled to potential further downstream value in the form of royalties on sales.

On January 10, 2022 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a commercial-stage biopharmaceutical company developing innovative medicines to improve the lives of people with cancer, reported a corporate update and highlighted key 2022 milestones in conjunction with its presentation at the 40th Annual J.P. Morgan Healthcare Conference (Press release, Deciphera Pharmaceuticals, JAN 10, 2022, View Source [SID1234598505]). The Company will webcast its presentation today at 9:00 AM ET at View Source

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"Deciphera is well-positioned as we enter 2022 with multiple high-value clinical programs and a successful commercial franchise in QINLOCK, all generated by our proven and productive research engine," said Steve Hoerter, President and Chief Executive Officer of Deciphera Pharmaceuticals. "We are excited about our leading portfolio of novel kinase inhibitors, including our first-in-class autophagy pathway inhibitor, DCC-3116, and our newly disclosed pan-RAF research program, both of which seek to address the challenges of drug resistance in patients with cancer. We believe that DCC-3116, designed to inhibit the autophagy pathway by inhibiting the ULK kinase, has the potential to address a broad range of human cancers, and planning is now underway to expand this development program to include a KRAS G12C inhibitor combination in non-small cell lung cancer (NSCLC). We plan to present initial data from the single agent dose escalation portion of this Phase 1 study later this year. We also announced today our plans to nominate a new clinical development candidate from our pan-RAF research program later this year, which we believe has the potential to be a best-in-class agent that can address unmet medical needs as a single agent and in combination."

Mr. Hoerter continued, "We kicked off 2022 with the launch of QINLOCK in Germany and we plan to transition to a post-approval paid access program in France in the first half of this year. Our existing QINLOCK commercial footprint with sarcoma specialists also serves as an excellent foundation for the future commercialization of our potential best-in-class CSF1R inhibitor, vimseltinib. The Phase 3 MOTION study of vimseltinib in tenosynovial giant cell tumor (TGCT) is currently underway and we expect to present updated data from our Phase 1/2 study in patients with TGCT later this year."

In 2022, the Company seeks to achieve the following milestones:

QINLOCK (ripretinib)

Execute on the commercial launch of QINLOCK in fourth-line GIST in Germany. Launch in Germany is ongoing as of January 1, 2022.
Transition to post-approval paid access program in France in the first half of 2022.
Present results of the Phase 3 INTRIGUE study in second-line GIST at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Plenary Series Session on January 25, 2022.
Vimseltinib

Enrollment underway in the Phase 3 MOTION study of vimseltinib, an orally administered, potent, and highly selective switch-control kinase inhibitor of CSF1R, for the treatment of TGCT.
Present updated data from the Phase 1/2 study in TGCT patients in the second half of 2022.
DCC-3116

Present data from the single agent dose escalation portion of this Phase 1 study of DCC-3116, a first-in-class ULK kinase inhibitor designed to inhibit autophagy for the treatment of patients with advanced or metastatic tumors with a mutant a RAS or RAF gene, in the second half of 2022. DCC-3116 is currently being investigated as a single agent and in combination.
Initiate Phase 1 study dose expansion in the second half of 2022 in combination with trametinib, a U.S. Food and Drug Administration-approved MEK inhibitor, in patients with selected mutations in advanced or metastatic pancreatic ductal adenocarcinoma, NSCLC, colorectal cancer, and melanoma.
Planning underway to add a combination with a KRAS G12C inhibitor in NSCLC to the ongoing Phase 1 study, subject to feedback from regulatory authorities.
Present additional preclinical data for DCC-3116 in 2022 and continue to explore preclinical combinations of DCC-3116 with multiple additional targeted oncology agents with diverse mechanisms of action.
Proprietary Drug Discovery Platform

Nominate a development candidate in 2022 from the pan-RAF inhibitor research program, discovered using the Company’s novel switch-control inhibitor platform.
Presentation at the 40th Annual J.P. Morgan Healthcare Conference

Deciphera will webcast its corporate presentation from the 40th Annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022 at 9:00 AM ET. A live webcast of the presentation can be accessed under "Events & Presentations" in the investors section of the Company’s website at deciphera.com. A replay of the webcast will be archived on the Company’s website for 90 days following the presentation. In conjunction with the conference, the Company has also updated its corporate presentation, which can be found here: View Source

On January 10, 2022 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported tthat it will present an update on its commercial progress and R&D pipeline at the 40th Annual J.P. Morgan Healthcare Conference today, Monday Jan. 10th at 7:30am EST (Press release, PTC Therapeutics, JAN 10, 2022, View Source [SID1234598504]). Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2021 accomplishments and highlight upcoming potential value-creating milestones. Preliminary 2021 unaudited financial results and 2022 financial guidance will also be provided. The presentation will be webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com.

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Key 2021 Corporate Highlights:

Unaudited net product revenue of $429 million in 2021 representing 29% year-over-year growth.
Strong year-over-year growth for the Duchenne muscular dystrophy (DMD) franchise, with unaudited net product revenue of $424 million for Translarna (ataluren) and Emflaza (deflazacort) in 2021. Cumulative net product revenue for Translarna exceeds $1 billion and Emflaza exceeds $500 million, since respective launches.
Translarna growth was driven by new patients and high compliance in existing geographies and continued geographic expansion.
Emflaza growth was due to continued new prescriptions, high compliance, less patient discontinuations and more favorable access.
Evrysdi (risdiplam) has shown continued rapid uptake in the United States and is now approved in all major markets including the European Union and Japan. Evrysdi is a product of the Spinal Muscular Atrophy (SMA) collaboration between PTC, the SMA Foundation and Roche.
Waylivra(volanesorsen), the only treatment for familial chylomicronemia syndrome was approved by Brazilian Health Regulatory Agency, Agência Nacional de Vigilância Sanitária (ANVISA), and both Waylivra and Tegsedi(inotersen) received Category 1 classification from Câmara de Regulação do Mercado de Medicamentos – CMED (Drug Market Regulation Chamber) in Brazil. Category 1 classification is given to innovative treatments that provide greater efficacy than the standard of care and allows for pricing in line with international markets.
PTC successfully advanced its clinical pipeline in 2021:
APHENITY, a Phase 3 registration-directed trial of PTC923 in phenylketonuria (PKU) was initiated.
Phase 1 healthy volunteer trials of PTC518 and PTC857 were completed.
Advanced the oncology platform with the completion of Phase 1b studies of unesbulin in leiomyosarcoma and diffuse intrinsic pontine glioma (DIPG).
Completed enrollment for the vatiquinone MOVE-FA registration-directed trial with results expected in the second quarter of 2023.
2022 Potential Key Value-Creating Milestones:

Results from Study 041 for Translarna are expected mid-year 2022 and if positive could support
re-submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA).
Results for MIT-E, the registration-directed study of vatiquinone in mitochondrial disease associated seizures, are expected in the fourth quarter of 2022.
Results are expected by year end 2022 for the Phase 3 registration-directed study, APHENITY, for PTC923 in patients with PKU.
From the splicing platform, the PIVOT-HD Phase 2 study of PTC518 in Huntington’s disease patients is planned to initiate in the first quarter of 2022.
From the Bio-e platform, the registration-directed CardinALS study of PTC857 in amyotrophic lateral sclerosis (ALS) patients is expected to be initiated in the second quarter of 2022.
Progress in the gene therapy platform is anticipated in 2022:
PTC expects an opinion from the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for PTC-AADC, the first gene therapy for AADC deficiency, in April 2022.
Submission of a Biologics License Application (BLA) to the FDA for PTC-AADC is expected in the second quarter of 2022.
Preliminary Unaudited 2021 Financial Results:

Total unaudited net revenue for full year 2021 was approximately $536 million.
Total unaudited net product revenue for full year 2021 was approximately $429 million.
DMD franchise revenue for year end 2021 included net product revenue for Translarna of approximately $236 million, with $70 million in revenue in the fourth quarter, and Emflaza of approximately $188 million, with $48 million in revenue in the fourth quarter.
PTC expects to report approximately $107 million in 2021 collaboration and royalty revenue associated with Evrysdi.
PTC expects to report 2021 year-end cash, cash equivalents and marketable securities of approximately $773 million.
PTC is currently in the process of finalizing its financial results for the 2021 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2021, subject to the completion of PTC’s financial closing procedures.

2022 Financial Guidance:

PTC anticipates total revenues for the full year 2022 to be between $700 and $750 million.
PTC anticipates net product revenues for the DMD franchise for the full year 2022 to be between $475 and $495 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2022 to be between $915 and $965 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2022 to be between $800 and $850 million, excluding estimated non-cash, stock-based compensation expense of $115 million.
Non-GAAP Financial Measures:

In this press release, the financial results and financial guidance of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.

On January 10, 2022 Invitae Corporation (NYSE: NVTA), a leading medical genetics company, reported that preliminary unaudited full-year 2021 revenue, billable volume and commercial metrics reporting approximately 64 percent growth in revenue and 76 percent growth in volume, signaling continued momentum into 2022 (Press release, Invitae, JAN 10, 2022, View Source [SID1234598502]).

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

"We are encouraged by the acceleration of our business in 2021, particularly in light of the direct and indirect impacts on the healthcare system throughout the year," said Sean George, Ph.D., co-founder and chief executive officer of Invitae. "Our results, which exceeded our outlook a year ago, underscore the strength of our unique model for engaging patients and physicians early, delivering new and efficient ways to interact and provide information to those patients and support their providers at every step along the way. The growth in testing during this period demonstrates the value of our differentiated strategy of expanding access to and use of genetic information for all patients as they plan for a healthier life. I am confident in our continued ability to execute and integrate new technologies to expand our menu, services and data platform as we enter into this next phase of our business model on our way to establishing genome management as the future of medicine."

Preliminary, unaudited financial results and other metrics for 2021

Generated revenue of more than $458 million in 2021, an approximate 64 percent increase from $279.6 million in 2020
Generated billable volume of more than 1.16 million in 2021, an approximate 76 percent increase from billable volume of 659,000 in 2020
Total active healthcare provider accounts are over 17,500, almost doubling since the beginning of 2020
Active pharma and commercial partnerships grew to 178, an increase of approximately 68 percent in 2021 driving continued revenue growth from Invitae’s data and data services platform to pharma, health system and software and services partners
Total patient population is more than 2.5 million with over 62 percent available for data sharing
Invitae has not completed preparation of its financial statements for the fourth quarter or full year 2021. The preliminary, unaudited results presented in this press release for the year ended December 31, 2021, are based on current expectations and are subject to adjustment. Actual results may differ materially from those disclosed in this press release. Invitae will report its full financial results and other metrics during its fourth quarter and year-end 2021 conference call in early March.

Invitae’s Presentation at 40th Annual J.P. Morgan Healthcare Conference
Invitae will present at the 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, 2022, at 9:45 a.m. Eastern Time/6:45 a.m. Pacific Time. The 2022 J.P. Morgan Healthcare Conference is being held virtually via webinar. A live webcast of both the presentation and a Q&A session may be accessed at the following direct link or by visiting the investors section of the company’s website at ir.invitae.com. Public listeners can access an audio and slide recording of the session, which will be available shortly after the conclusion of the presentation and Q&A session on the investors section of the company’s website at ir.invitae.com.

On January 10, 2022 Cardinal Health (NYSE: CAH) reported that provided a fiscal year 2022 financial update for the Medical segment (Press release, Cardinal Health, JAN 10, 2022, View Source [SID1234598501]). The company now expects additional inflationary impacts and lower volumes resulting primarily from global supply chain constraints, and a lower-than-expected offset from pricing actions . The company estimates this incremental impact will be approximately $150 million to $175 million to fiscal year 2022 Medical segment profit and approximately $0.40 to $0.45 per share to non-GAAP diluted EPS. Excluding this impact to the Medical segment and non-GAAP diluted EPS, the company’s other financial expectations1 for fiscal year 2022 remain unchanged.

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Cardinal Health continues to take actions to drive performance in the Medical segment, including: simplifying its operating model, evolving its commercial contracting strategies and driving mix, and making investments in its growth businesses. Additionally, the company reaffirms its previously announced long-term financial targets, expense reduction initiatives, and capital deployment priorities.

Cardinal Health will present at the 40th Annual J.P. Morgan Healthcare Conference today, January 10, 2022 at 10:30 a.m. Eastern. The presentation will be followed by a Q&A session with Mike Kaufmann, chief executive officer, and Jason Hollar, chief financial officer, and will include comments related to this update.

To listen to a live webcast of this event, please visit the Investor Relations page at ir.cardinalhealth.com.

The company is currently analyzing how this financial update to the Medical segment may impact other components of its second quarter GAAP financial statements, including goodwill. The financial update is based on preliminary results and remains subject to completion of the company’s standard quarter-end closing procedures.

The company plans to release its second-quarter fiscal year 2022 financial results on February 3 prior to the opening of trading on the New York Stock Exchange. The company will webcast a discussion of these results beginning at 8:30 a.m. Eastern.

The company does not provide forward-looking guidance on a GAAP basis as certain financial information, the probable significance of which cannot be determined, is not available and cannot be reasonably estimated. See "Use of Non-GAAP Measures" following the attached schedules for additional explanation.