Pfizer punts pair of orphan drugs amid rare disease R&D rethink

 Pfizer, amid a rethink of its rare disease R&D strategy, has punted (PDF) a pair of orphan drug candidates from its clinical development pipeline in a cull that also narrowed the opportunities open to the near-approval JAK3/TEC inhibitor ritlecitinib.

Recifercept is the most advanced rare disease candidate to become surplus to requirements in the latest pipeline update. The molecule is designed to bind to fibroblast growth factor isoforms and thereby act as a decoy that reduces FGFR3 signaling. Seeing that FGFR3 mutations drive inhibitory signaling in the rare disease achondroplasia, Pfizer made the condition its focus indication for recifercept development.

The program looked to be on track late last year, when Pfizer began a pilot project to check the feasibility of constructing a concurrent external control for recifercept, but has now hit the skids. The termination of development comes weeks after Pfizer wrapped up a phase 2 trial of the long-term safety and efficacy of recifercept.

Pfizer is kicking the candidate to the curb along with PF-06755347, a recombinant Fc fusion protein that is the product of a licensing agreement the Big Pharma formed with Gliknik in 2013. Gliknik received $15 million from Pfizer when the candidate entered phase 1 four years ago.

PF-06755347 was in development as a treatment for primary immune thrombocytopenia and chronic inflammatory demyelinating polyneuropathy, an indication in which it has orphan drug status in the U.S. 

Away from rare diseases, Pfizer dropped a midphase rheumatoid arthritis candidate, ritlecitinib, and a trio of phase 2 cancer programs. Ritlecitinib is closing in on approval decisions in alopecia areata on both sides of the Atlantic and is in phase 2 and 3 development as a treatment for vitiligo, ulcerative colitis and Crohn’s disease. But after studying the candidate with the IRAK4 inhibitor zimlovisertib and JAK inhibitor Xeljanz, Pfizer has decided to abandon plans to develop it as a treatment for rheumatoid arthritis. 

The three early-phase cancer candidates that failed to make the cut at Pfizer are: the therapeutic vaccine PF-07263689 in solid tumors; the BRAF kinase inhibitor PF-07284890 in melanoma; and the CDK2/4/6 inhibitor PF‑06873600 in metastatic breast cancer.

Pfizer's discontinuation drive also included ending phase 3 trials for the company's blockbuster COVID-19 pill Paxlovid in standard risk populations and as a prophylactic treatment. The antiviral is already authorized in high risk populations but had flopped in a post-exposure trial.

https://www.fiercebiotech.com/biotech/pfizer-punts-pair-orphan-drugs-amid-rare-disease-rd-rethink

Taysha: FDA seeks more dosing for CNS candidate

 Type B end-of-Phase 2 meeting with U.S. Food and Drug Administration (FDA) provided additional clarity for TSHA-120 for the treatment of giant axonal neuropathy (GAN) ultra-rare disease program 

- FDA acknowledged MFM32 as an acceptable endpoint with a recommendation to dose additional patients in a double-blind, placebo-controlled design to support Biologics License Application (BLA) submission

Organizational and business review by new management with operational, structural and personnel changes implemented to enhance execution

Dosing of first adult patient with Rett syndrome from ongoing trial in Canada expected in H1 2023; update of initial available clinical data anticipated in H1 2023 with quarterly updates primarily on safety thereafter

https://finance.yahoo.com/news/taysha-gene-therapies-provides-tsha-210100596.html

FBI searched Penn Biden Center in mid-November

 The Federal Bureau of Investigation searched the Penn Biden Center offices in mid-November, CBS News reported Tuesday, citing two sources familiar with the investigation. The search came after lawyers for President Joe Biden found about 10 documents marked classified there on Nov. 2. It's not clear whether the FBI personnel found any additional classified or presidential material during the mid-November sweep, the report said. The search wasn't previously disclosed by the White House, Biden's attorneys or the Justice Department.

https://www.morningstar.com/news/marketwatch/20230131323/fbi-searched-penn-biden-center-in-mid-november-report

Moderna's stock is down as Pfizer lowers revenue estimates for its COVID vaccine for 2023

 Moderna Inc.'s (MRNA) shares were down 2.8% in trading on Tuesday. Pfizer Inc. (PFE), which makes a competing COVID-19 vaccine to Moderna's Spikevax, on Tuesday put out guidance for 2023, saying it expects a total of $13.5 billion in sales of its COVID shot for the year. That's a much lower projection than the $37.8 billion in revenue that was generated by Pfizer and BioNTech's (BNTX) COVID vaccine in 2022. Moderna's COVID vaccine is expected to bring in $18.1 billion in sales for 2022 and $7.3 billion in 2023, according to the FactSet consensus. The company is set to report its fourth-quarter results on Feb. 23. Its stock is up 3.3% over the past year, while the broader S&P 500 is down 7.8%.

https://www.morningstar.com/news/marketwatch/20230131398/modernas-stock-is-down-as-pfizer-lowers-revenue-estimates-for-its-covid-vaccine-for-2023

Public health emergency for mpox to end Tuesday

 The U.S. government does not plan to renew the public-health emergency for mpox, the virus that was previously called monkeypox, and Tuesday is the final day of the declaration. The Department of Health and Human Services had notified states in December about its plan not to renew the emergency. "Given the low number of cases today, HHS did not renew the emergency declaration," officials said in a statement. "We will continue to monitor the case trends closely and encourage all at-risk individuals to get a free vaccine." More than 30,000 people in the U.S. contracted the mpox virus this year as part of an outbreak that began this summer and has had at least 85,000 confirmed cases worldwide. However, the number of new mpox infections in the U.S. has since declined.

https://www.morningstar.com/news/marketwatch/20230131400/public-health-emergency-for-mpox-to-end-tuesday

China is a 'clear and present danger' to American cybersecurity: Gov. Tate Reeves

 Gov. Tate Reeves, R-Miss., announced he plans to ban TikTok from all government devices in Mississippi on Monday, telling "Cavuto: Coast to Coast" the "future of warfare" is not necessity boots on the ground, but battle information - and that America's interests must be protected.

GOV. TATE REEVES: We know that the Chinese Communist Party has a history of stealing American intellectual property, they have a history of stealing America's personal information, and we want to be proactive in ensuring that they do not have the ability to steal Mississippi state government data. That is the reason I outlawed all TikTok on all state-owned devices because we know that the Chinese often use social media platforms such as TikTok to get that data, get that information, and we’re going to be proactive in Mississippi. 

No doubt there is opposition to what we’re doing, but we have got to be clear in recognizing that China is not our friend this is a clear and present danger to cybersecurity in our state and honestly across America. We’ve got to be cognizant of that, we have to be willing to take sometimes difficult decisions to make sure we protect ourselves. When we think about warfare in the future, warfare is not necessarily troops on the ground or boots on the ground anymore. There is a real battle going on for information in cybersecurity. We’ve got to do everything we can as a state, ultimately as this nation to protect America’s interests, and we’re willing to do that.

https://www.foxbusiness.com/politics/china-clear-present-danger-american-cybersecurity-gov-tate-reeves

FibroGen upped to Outperform by Blair

 from Market Perform 

https://finviz.com/quote.ashx?t=FGEN&p=d