On January 09, 2023 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported preliminary, unaudited ORLADEYO (berotralstat) net revenue for the fourth quarter and full year 2022 and provided guidance for full year 2023 ORLADEYO net revenue and expected peak ORLADEYO sales (Press release, BioCryst Pharmaceuticals, JAN 9, 2023, View Source [SID1234626035]).

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"In our second year on the market we more than doubled our first year ORLADEYO sales and are more than a quarter of the way towards $1 billion in peak sales. This continues to be an exceptional launch of an oral rare disease drug and we expect this success to continue creating real value for patients and for shareholders this year and for many years to come," said Jon Stonehouse, president and chief executive officer of BioCryst.

Preliminary, unaudited ORLADEYO net revenue in the fourth quarter of 2022 was $70.7 million (+53 percent y-o-y). Preliminary, unaudited ORLADEYO net revenue for full year 2022 was $251.6 million (+105 percent y-o-y).

Fourth Quarter and Full Year 2022 ORLADEYO Revenue Dynamics
"The number of patients on therapy at the end of 2022 was in-line with our expectations as patients continued to switch to ORLADEYO in the fourth quarter. Revenue in the quarter lagged our expectations slightly as we saw fewer paid product shipments based on seasonal factors and lower conversion from free to paid product than expected. We see continued growth in 2023 in the U.S. and around the world as we advance toward $1 billion in peak ORLADEYO sales," said Charlie Gayer, chief commercial officer of BioCryst.

2023 Financial Outlook
The company expects full year 2023 global net ORLADEYO revenue to be no less than $320 million. As in 2022, due to the seasonal impact of managed care reauthorizations in the first quarter of the year, the company expects revenue in the first quarter of 2023 to be similar to revenue in the fourth quarter of 2022.

Presentation Tomorrow at 41st Annual J.P. Morgan Healthcare Conference
On Tuesday, January 10, 2023 at 6:00 p.m. ET, the company will present at the 41st Annual J.P. Morgan Healthcare Conference in San Francisco. Links to a live audio webcast and replay of the presentation may be accessed in the Investors section of BioCryst’s website at https://www.biocryst.com/.

On January 9, 2023 Bristol-Myers Squibb presented its corporate presentation (Presentation, Bristol-Myers Squibb, JAN 9, 2023, View Source [SID1234626039]).

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On January 9, 2023 Blueprint medicine presented its corporate presentation (Presentation, Blueprint Medicines, JAN 9, 2023, View Source [SID1234626038]).

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On January 9, 2023 Beam therapeutics presented its corporate presentation (Presentation, Beam Therapeutics, JAN 9, 2023, View Source [SID1234626034]).

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On January 9, 2023 Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, reported that it has signed a multi-year research collaboration and option agreement with ReCode Therapeutics (Press release, Bayer, JAN 9, 2023, View Source [SID1234626033]). Under this agreement, the companies will work together to potentially discover precision genetic medicines through the development of a novel platform for full gene insertion by single vector delivery of gene editing and DNA cargoes. This represents another step forward for AskBio in developing its gene editing and other nonviral delivery technologies and expertise.

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"The collaboration pairs AskBio’s expertise in synthetic DNA and CRISPR applications with ReCode’s novel SORT LNP delivery technology to potentially discover new genetic medicines," said AskBio’s Chief Executive Officer Sheila Mikhail. "Through this collaboration, we have an opportunity to advance AskBio’s gene editing and nonviral delivery efforts, which support and align with our commitment to serve as an industry-leading engine of gene therapy innovation."

Under the agreement, AskBio will combine its synthetic DNA and gene editing nucleases with ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) technology to potentially create an all-in-one solution that enables full gene insertion by delivering with precision both the gene editing tool and DNA as mixed cargo in a single LNP to desired targets. This collaboration could extend the reach of gene editing for liver and lung disease targets.

"AskBio’s pioneering gene editing technology is a powerful complement to our modular SORT LNP genetic medicines platform, which enables the precise delivery of a wide variety of genetic cargoes to specific organs and cells, including large, complex genetic cargoes and mixed payloads," said Shehnaaz Suliman, M.D., MBA, M.Phil., Chief Executive Officer of ReCode Therapeutics. "We are excited to combine these unique platforms to develop next-generation genetic therapies with the potential to transform the lives of patients suffering from a wide range of debilitating genetic diseases."

In June 2022, ReCode announced the closing of a $200 million Series B extension financing round, co-led by Leaps by Bayer, the impact investment unit of Bayer AG, to further strengthen Leaps by Bayer’s innovation portfolio in gene therapies. The funds raised are being used to advance the development of ReCode’s platform and pipeline to selectively deliver genetic medicines to target organ and cell types in a predictable and programmable fashion.