Breaking News

New inroads for CF patients, Karuna repurposing Goldfinch assets, & earnings earnings earnings

February 3, 2023
Biotech Correspondent
We're happy to see that cystic fibrosis patients who have felt left behind are now being prioritized by drugmakers. Also, there were earnings aplenty this week, but many pharma stocks dropped as a result. Have a nice weekend!

earnings

A précis of a big Big Pharma earnings day

If you were so inclined, five-ish hours of pharma earnings conference calls were on offer yesterday morning. Maybe you had other things to do. If so, here are some highlights:

Financial guidance for 2023 offered by Merck, Roche, Eli Lilly, and Bristol Myers Squibb generally bracketed Street consensus. But with large pharma already trading at high multiples, investors viewed "in line" guidance as a disappointment. Pharma stocks fell, with Bristol the lone exception.

Lilly, in particular, came up on the short end of the expectation game. It reported strong sales for its closely tracked GLP-1 class diabetes drugs Trulicity and Mounjaro, with the latter already on pace for blockbuster status just six months after launch. Investors, however, were looking for more. Cautious comments made by Lilly management about rates of insurer coverage and continued manufacturing shortfalls didn't help either. Lilly shares fell 6%.


ear candy

Is the next Lipitor on the way?

When is a pandemic over-over? And what's a Texas two-step? We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast. 

STAT Washington correspondent Rachel Cohrs joins us to explain the looming end of Covid-19's status as a federal emergency and what that does and doesn't mean for public health. We also dig into the most interesting stories from a busy week of pharmaceutical earnings and discuss a legal setback for Johnson & Johnson.

Listen here.



rare disease

CF patients, once left behind, may have a shot at treatment

Vertex Pharmaceuticals has nearly cornered the cystic fibrosis market: Its medications can work wonders in as much as 90% of patients with the inherited respiratory disease. But what of the rest? About 10% of cystic fibrosis patients have seen the lives of their peers change dramatically, while they have had to continue using old medications that do not work well. 

Vertex hasn't forgotten about these patients. The company is beginning to test an mRNA-based drug that might target mutations that have yet to respond to treatments. And it's not just Vertex: A $500 million effort from the Cystic Fibrosis Foundation is emboldening other companies to use new approaches — including mRNA, gene therapy, even gene editing — to find one that might work. 

"For so long, it felt like those of us in the 10% were watching innovation happening around us," one CF patient and advocate told STAT. "And for the first time, it feels like we're the focus." 

Read more.


future pandemics

Karuna repurposing Goldfinch assets from kidney to psychiatric indications

Goldfinch Bio sputtered to a close this past week after running low on capital, and has begun distributing its various assets. In a $520 million deal, Karuna Therapeutics just acquired exclusive rights to Goldfinch's experimental drug GFB-887, along with other assets in a class of drugs called TRPC4/5 channel candidates. GFB-887 has been tested in Phase 2 studies for a kidney disease, but Karuna has other plans. 

Karuna plans to use GFB-887 to treat psychiatric conditions, noting that the class of drugs its in demonstrated "anxiolytic and antidepressant properties" in preclinical and clinical data. The drug's mechanism "could represent a completely novel approach to treating mood and anxiety disorders," CEO Bill Meury said in a statement. 


More around STAT
Check out more exclusive coverage with a STAT+ subscription
Read premium in-depth biotech, pharma, policy, and life science coverage and analysis with all of our STAT+ articles.

More reads

  • Eye drops linked to U.S. drug-resistant bacteria outbreak, STAT

  • Layoffs continue to batter biotech, with Big Pharmas piling on the pain, FierceBiotech

  • How quickly does Covid immunity fade? What scientists know, Nature


Thanks for reading! Until next week,


Enjoying The Readout? Tell us about your experience
Continue reading the latest health & science news with the STAT app
Download on the App Store or get it on Google Play
STAT
STAT, 1 Exchange Place, Boston, MA
©2023, All Rights Reserved.

No comments