Rubius Therapeutics surges 84% on encouraging results from early-stage testing of cancer treatment

NYSE traders
  • Rubius Therapeutics shares surged 84% during Monday’s trading session.
  • The biotech firm outlined encouraging data from testing of RTX-240, a potential cancer treatment.
  • Rubius said data collected provide initial proof-of-concept of its red blood cells generation system Red Platform.
  • See more stories on Insider’s business page.

Rubius Therapeutics shares more than doubled during Monday’s session after investors received the biotech firm’s outline of results from early-stage tests of a potential cancer drug.

The company said data from an ongoing clinical trial of patients with advanced solid tumors contributed to providing initial proof-of-concept for its platform to generate red blood cells.

Shares of Rubius jumped as much as 136% to $38.71 then trimmed closed the session up by 84%. Trading was heavy, with volume of nearly 38 million shares outrunning the average daily volume of about 1.5 million shares.

Among the data from an ongoing phase 1/2 trial of RTX-240, the company’s cellular therapy product candidate, there was a 54% reduction in target lesions in a patient with metastatic anal cancer.

Rubius said the data collected “provide clinical validation” of its Red Platform red blood cells generation system, said Pablo Cagnoni, chief executive at Rubius, in a statement.

With “encouraging” initial safety and preliminary efficacy data for RTX-240, he said the company plans to initiate a phase 2 expansion cohort in the first quarter of 2022 and a new phase 1 arm of the RTX-240 trial to evaluate it in combination with another therapy during the second half of 2021.

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New data on Eli Lilly’s experimental Alzheimer’s drug backs a hotly debated scientific theory, but shows mixed benefits for patients

FILE PHOTO:  Patients with Alzheimer's and dementia are sit inside the Alzheimer foundation in Mexico City April 19, 2012. REUTERS/Edgard Garrido
Patients sit inside the Alzheimer’s Foundation in Mexico City

  • Eli Lilly’s experimental treatment slowed down cognitive decline by 32% in a midstage trial.
  • But the drug missed most of the secondary trial goals, including one watched by experts.
  • An Eli Lilly executive told Insider the data was mixed, but very exciting.
  • See more stories on Insider’s business page.

The latest test of Eli Lilly’s Alzheimer’s disease drug ended with a mixed bag of results, but experts say there are signs the experimental treatment may curb the disease’s decimation of cognition and memory.

Lilly gave an in-depth look at the performance of its drug, called donanemab, on Saturday at the International Conference on Alzheimer’s and Parkinson’s Diseases. The drug met the trial’s primary goals, reducing a harmful substance that builds up in the brains of patients with Alzheimer’s disease. It also slowed down the rate of cognitive decline by 32%, according to data scheduled to be published Saturday in a medical journal. But it missed the mark on several secondary tests.

The Alzheimer’s drug-development field has been marked by failure after failure, so the news that Lilly may have a working drug sent a surge of energy through the field when the drugmaker first disclosed some information about the results in January.

Those earlier failures – even within Lilly’s own drug-development pipeline – may have tempered expectations for donanemab. Dr. Mark Mintun, the vice president of the Alzheimer’s disease unit at Lilly, said he couldn’t think of a similar Alzheimer’s disease trial that hit its primary goal.

At this point, any news is good news, Dr. Howard Fillit, executive director of the Alzheimer’s Drug Discovery Foundation, told Insider.

“We’ve basically had a 100% failure rate on drug approvals since 2003. The thing that we’re going for is incremental benefits,” he said.

The donanemab data may give hope to the scientific community and other drug companies, but Fillit said the jury is still out on how effective the drug will be for patients themselves.

Donanemab missed the mark on a memory assessment experts were watching

Donanemab was designed to target a plaque that builds up in the brains of Alzheimer’s patients called amyloid beta. The hope is that getting rid of the plaque will reduce the rate of decline of a person’s memory and cognition.

Scans of trial participants’ brains showed that people taking donanemab did see their plaques fade away. That’s not only a boon to Eli Lilly, but other drugmakers that are working on treatments targeting amyloid beta, such as Biogen and Roche.

FILE - In this May 19, 2015, file photo, a doctor points to PET scan results that are part of a study on Alzheimer's disease at a hospital in Washington. Scientists know that long before the memory problems of Alzheimer's become obvious, people experience more subtle changes in their thinking and judgment. (AP Photo/Evan Vucci, File)
FILE – In this May 19, 2015, file photo, a doctor points to PET scan results that are part of a study on Alzheimer’s disease at a hospital in Washington. Scientists know that long before the memory problems of Alzheimer’s become obvious, people experience more subtle changes in their thinking and judgment. (AP Photo/Evan Vucci, File)

It wasn’t as clear how much benefit donanemab had on subjects’ ability to recall words, follow instructions and other abilities lost to dementia.

The drug passed the main test of memory and cognition.

But Alzheimer’s experts had their eyes on one datapoint in particular: the scores from a widely accepted dementia assessment known as CDR-SB. The tool is used to measure things like memory and judgement, as well as a person’s ability to do household chores and maintain their personal hygiene. The higher the score, the more skills have been lost to dementia like Alzheimer’s.

Eli Lilly’s drug didn’t meet expectations there.

The company reported that trial participants taking donanemab had a 0.36 point score difference compared to the participants that received a placebo treatment.

For comparison, Dr. Lon Schneider, director of the California Alzheimer’s Disease Center at University of Southern California, had hoped to see at least a one-point difference, according to the Wall Street research firm Mizuho.

Mintun, the Eli Lilly executive, said he hadn’t expected that donanemab would pass all five dementia tests, calling it statistically impossible. But giving patients more time to hold onto memories is meaningful for them, he said.

The clinical trial took a ‘Goldilocks approach,’ and more testing is needed

The donanemab trial assessed a small and very specific group of patients. The trial was limited to participants with amyloid plaques, as well as another protein linked to Alzheimer’s called tau. Fillit called it a Goldilocks approach – in the end, Lilly ended up with participants whose Alzheimer’s disease wasn’t too early or too advanced.

Fillit and Alzheimer’s disease researcher Rudy Tanzi said that Lilly will need to run another clinical trial, preferably a much larger late-stage test with participants from multiple countries, if it wants to bring the treatment to the US Food and Drug Administration for approval. In particular, the company would need to show the drug works, as measured by the CDR-SB score, Tanzi said.

Eli Lilly’s current plan is to run at least one more mid-stage trial. The format of that trial had already been set, but Mintun said the drug giant is discussing possible changes with the FDA, including potentially removing a significant CDR-SB change as the main goal.

Changing that CDR-SB requirement, particularly given that the original Phase 2 trial failed on that regard, would make investors skeptical of the drug, according to a note from Morgan Stanley analysts before the full results were announced.

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