Agios Unveils 2023-2026 Value-driving Rare Disease Catalysts

 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced its anticipated 2023 milestones and significant value-driving catalysts through 2026 that support the company’s mission to transform patient outcomes in rare diseases. Agios will present at the 41st Annual J.P. Morgan Healthcare Conference on Wednesday, January 11, 2023, at 7:30 a.m. PT, and a live webcast will be available at investor.agios.com.

“Agios is poised for significant growth with the potential for approvals in two additional PYRUKYND^® indications by 2026, and is well capitalized to advance its robust existing clinical pipeline and expand its portfolio within our core areas of expertise,” said Brian Goff, chief executive officer at Agios. “As the pioneering leader in PK activation with more than seven years of clinical experience with PYRUKYND^®, we have generated an impressive body of consistent and compelling data across rare hematological diseases with shared underlying pathophysiology that further builds confidence in our five ongoing pivotal clinical trials in thalassemia, sickle cell disease and pediatric PK deficiency. Furthermore, we are executing our first rare disease U.S. product launch with PYRUKYND^® in adult PK deficiency, providing the first disease-modifying therapy for this patient community that previously had no treatment options and building the capabilities to set us up for success with our expected launches in meaningfully larger patient populations.”

Recent Highlights

• Adult PK Deficiency: Received marketing authorization for PYRUKYND^® in adults with PK deficiency in the EU and Great Britain
• Thalassemia: Enrolled approximately half of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND^® in not regularly transfused and regularly transfused adults with thalassemia, respectively
• Sickle Cell Disease: Closed screening in the Phase 2 portion of the RISE UP study of PYRUKYND^® in adults with sickle cell disease in December and expect to complete enrollment in January
• Data Presentations: Presented broad set of clinical and translational data at the 64^th American Society of Hematology (ASH) Annual Meeting & Exposition, including long-term PYRUKYND^® data in adults with non-transfusion-dependent thalassemia and in adults with PK deficiency
• Leadership:Appointed Tsveta Milanova to the role of chief commercial officer, bringing two decades of experience in rare disease commercial strategy and global market access

Anticipated 2023 Milestones

• Thalassemia: Complete enrollment of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND^® by mid-year
• Pediatric PK Deficiency: Enroll at least half of patients in the Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND^® by year-end
• Sickle Cell Disease: Announce data readout from Phase 2 portion of RISE UP study of PYRUKUND^® and go/no-go to Phase 3 decision by mid-year
• Lower-risk Myelodysplastic Syndromes (LR-MDS): Complete enrollment of Phase 2a study of novel PK activator AG-946 by year-end
• Earlier-stage Pipeline: File investigational new drug (IND) application for PAH stabilizer for the treatment of phenylketonuria (PKU) by year-end

https://www.biospace.com/article/releases/agios-unveils-2023-2026-value-driving-catalysts-enabling-company-s-vision-to-transform-patient-outcomes-in-rare-diseases/

Ipsen to acquire Albireo accelerating growth in rare disease

 

• Transaction focused on Bylvay® (odevixibat), the first-approved treatment in progressive familial intrahepatic cholestasis in U.S. and E.U., with potential in other rare diseases
• Acquisition aligned with Ipsen’s long-term strategy for expanding the scope of its Rare Disease portfolio and pipeline
• Ipsen to commence cash tender offer to acquire all issued and outstanding shares of Albireo for $42.00 per share plus a contingent value right (CVR) of $10.00 per share related to the U.S. FDA approval of Bylvay in biliary atresia

https://www.ipsen.com/press-releases/ipsen-to-acquire-albireo-accelerating-growth-in-rare-disease-with-treatments-for-several-pediatric-liver-diseases/

RxSight: Strong prelims

  RxSight, Inc., an ophthalmic medical device company dedicated to improving the vision of patients following cataract surgery, today announced certain preliminary unaudited financial and operational results for the fourth quarter and full-year 2022.

Preliminary Unaudited Results

• Preliminary unaudited fourth quarter 2022 revenue is expected to be approximately $16.1 million, representing growth of approximately 91% compared to the prior year period, driven by:

  • The sale of 57 Light Delivery Devices (LDD™s), expanding the installed base to 400 LDDs as of December 31, 2022; and

  • The sale of 9,123 Light Adjustable Lenses (LAL®s).

• Preliminary unaudited 2022 fiscal year revenue is expected to be approximately $49.0 million, representing growth of approximately 117% compared to the prior year, driven by:

  • The sale of 195 LDDs; and

  • The sale of 25,284 LALs.

• Preliminary unaudited cash, cash equivalents and short-term investments as of December 31, 2022, is expected to be $105.8 million, which includes $6.0 million in net proceeds from sales of shares of the Company’s common stock sold through the Company’s “at-the-market” (“ATM”) offering during the fourth quarter of 2022.

https://finance.yahoo.com/news/rxsight-inc-announces-preliminary-unaudited-210000454.html

Quidel prelims above views

 QuidelOrtho Corporation (Nasdaq: QDEL) (the "Company" or "QuidelOrtho"), a global provider of innovative in vitro diagnostics technologies designed for point-of-care settings, clinical labs and transfusion medicine, today announced preliminary unaudited revenue results for the fourth quarter and full year ended January 1, 2023.

The Company expects total revenues in the fourth quarter of 2022 to be in the range of $853 million to $868 million. COVID-19 product revenue is expected to be in the range of $124 million to $134 million and non-COVID-19 product revenue is expected to be in the range of $729 million to $734 million, representing 485% growth on a GAAP basis and 18% growth on a proforma constant currency basis at the midpoint.

The Company expects total revenues in the full year of 2022 to be in the range of $4,038 million to $4,053 million. COVID-19 product revenue is expected to be in the range of $1,441 million to $1,451 million and non-COVID-19 product revenue is expected to be in the range of $2,597 million to $2,602 million, representing 502% growth on a GAAP basis and 11% growth on a proforma constant currency basis at the midpoint.

https://finance.yahoo.com/news/quidelortho-reports-preliminary-revenue-results-210500790.html

Chiesi Farmaceutici S.p.A. to Acquire Amryt

 Transaction expands Chiesi’s rare disease medicine portfolio

- All cash acquisition at US$14.50 per ADS, plus Contingent Value Rights of up to an additional US$2.50 per ADS based on certain Filsuvez® milestones being achieved

- Total Transaction value of up to US$1.48 Billion with upfront consideration representing a 107% premium to Amryt ADS’ closing price on January 6, 2023

- Transaction unanimously approved and recommended by the Boards of both Chiesi and Amryt

https://finance.yahoo.com/news/chiesi-farmaceutici-p-acquire-amryt-200000567.html

Castle Biosciences Announces Preliminary Fourth Quarter and Full-Year 2022 Results

 2022 total revenue expected to meet or exceed top end of guided range of $132–137 million

Delivered 44,338 total test reports in 2022, an increase of 58% compared to 2021

Growth of 37% year over year in DecisionDx®-Melanoma test report volume

Year-end 2022 cash, cash equivalents and marketable investment securities expected to be approximately $259 million

https://finance.yahoo.com/news/castle-biosciences-announces-preliminary-fourth-000000241.html

Exelixis: 2022 prelims, 2023 guidance, at JPMorgan

 Cabozantinib franchise achieves approximately $1.4 billion in preliminary U.S. net product revenues for full year 2022, including approximately $375 million for fourth quarter 2022 –

– Full year 2023 net product revenues guidance of $1,575 million - $1,675 million –

– Corporate priorities for 2023 include readout of Phase 3 trials for cabozantinib, expansion of the Phase 3 program for zanzalintinib, progression of XB002 into full development, and advancement of Exelixis’ earlier stage pipeline –

– Presentation and webcast at 2023 J.P. Morgan Healthcare Conference on Monday, January 9th at 8:15 p.m. ET / 5:15 p.m. PT –

Exelixis President and Chief Executive Officer Michael M. Morrissey, Ph.D., will provide a corporate overview and discuss the company’s preliminary fourth quarter and full year 2022 financial results, 2023 financial guidance, and key priorities and milestones for 2023 during the company’s presentation at the J.P. Morgan Healthcare Conference beginning at 8:15 p.m. ET / 5:15 p.m. PT on Monday, January 9, 2022.

To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the presentation to ensure adequate time for any software download that may be required to listen to the webcast. A replay will also be available at the same location for at least 30 days.

https://finance.yahoo.com/news/exelixis-announces-preliminary-fourth-quarter-210000924.html