Samumed osteoarthritis phase 2b data

Samumed
CEO Osman Kibar, CFO Cevdet Samikoglu, and chief medical officer
Yusuf Yazici
Diana Yukari/Business
Insider; photos courtesy Samumed

• $12 billion biotech Samumed on Wednesday is presenting data
  from a clinical trial looking at how one of its lead drugs works
  in patients with osteoarthritis. 
• The results, which Samumed plans to use to inform its phase 3
  trial it plans to start next year, show that the company’s drug
  worked in two doses to reduce pain and increase function in
  patients with osteoarthritis, a common form of arthritis in which
  cartilage wears down over time.
• The company is also planning additional mid-stage trials to
  get a better sense of how the drug might protect or regenerate
  cartilage, and what effects it might have on bone
  health.  

Samumed, a San Diego-based biotech that’s one
of the highest valued healthcare startups in the US with
a $12 billion valuation, just presented new data on one of its
lead programs.

The drug is a treatment for osteoarthritis, a common form of
arthritis in which cartilage wears down over time.

It’s part of Samumed’s
 pipeline of what
could be revolutionary treatments to regenerate hair, skin,
bones, and joints.

The company found that the treatment managed to reduce pain and
improve function in the treated knees, at two of the four doses
it tested, according to a presentation its scheduled to give
Wednesday at the
American College of Rheumatology’s annual meeting.  The
phase 2b trial looked at 700 patients who were given either the
drug injected once into the knee, or were given a placebo
treatment. 

At the six-month mark, the patients who received the drug didn’t
have any improvement in their medial joint space width, a measure
of how much cartilage is between the bones surrounding the knee,
when compared to those who received the placebo.

That’s a departure from what the company saw in its
phase 2a trial, which looked at the effects of the drug over
a full year. In that study, the company saw an improvement in
medial joint space width compared to placebo in the subgroup that
had received the .07 mg dose. Samumed Chief Medical Officer Yusuf
Yazici told Business Insider that the six-month cut-off was too
early to see a difference on an X-Ray. 

Samumed said Wednesday that it plans to move into two phase 3
clinical trials in 2019 at the .07 mg dose, which could set the
company up for an approval from the FDA if the trial shows the
drug works at reducing pain, improving knee function, and having
an impact on how the disease is progressing. Yazici said the
company is also planning on running two smaller trials aimed at
getting a better picture through MRIs of the drug’s effects on
cartilage — is it protecting or regenerating the cartilage — as
well as making sure the drug isn’t hurting patients’ bone health.

Phase 2 trials are used to determine which dose of a particular
medication might work best to test in a larger phase 3 trial. At
that point, the drug company is looking to make sure the drug
works the way it’s intended to, which it can then present to the
FDA for a potential approval.

Samumed’s approach

The company’s pipeline contains a number of experimental
treatments that offer the promise of reversing conditions related
to aging by regrowing hair on balding heads, smoothing out
wrinkles, and regenerating cartilage to worn-down joints in
people with osteoarthritis.

That happens through technology that targets certain proteins
that scientists think play a critical role in the development and
renewal of stem
cells, which give rise to other types of specialized cells,
from eye cells to skin and hair cells.

Your body is equipped with something called progenitor stem
cells. These cells are in charge of repairing and replenishing
specific organs in the body. For example, a mesenchymal stem cell
of the osteoblast lineage can go in and repair bone that’s
damaged. That process has something to do with the WNT pathway, a
set of proteins that tell these stem cells to spring into action.

“By dialing up or down various WNT genes or WNT processes, you
can trigger any one of these progenitor stem cells down a certain
lineage,” Kibar told
Business Insider in 2017.

As we get older, our WNT levels start to get out of balance,
Kibar said. Take the example of mesenchymal stem cells. “If the
WNT activity levels can no longer increase such that it’s not
making enough bone, now you develop osteoporosis.”

What Samumed hopes to do is manipulate the pathway that
makes these progenitor stem cells spring into action, so that
they don’t cause these diseases.

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