Uncommon illnesses aren’t usually part of baseball’s information cycle. However late final summer time, Los Angeles Dodgers first baseman Freddie Freeman needed to step away from the sport for a number of weeks when his son turned in poor health with Guillain-Barré syndrome, a uncommon, doubtlessly life-threatening neurological dysfunction. The swell of assist for Freeman, who went on to develop into the 2024 World Collection MVP, instantly raised the profile of the illness.
In GBS, the immune system mistakenly assaults a part of the peripheral nervous system. Signs come on instantly and progress quickly, beginning with tingling and weak spot and typically resulting in paralysis.
“It begins in your ft and strikes up by means of your physique,” stated Douglas Love, president and CEO of Annexon Biosciences, which has a late-stage GBS candidate within the pipeline. “One in 4 sufferers are going to be on a ventilator inside a few weeks.”
Douglas Love, CEO, Annexon Biosciences
Permission granted by Annexon
That frequent trajectory was the identical for Freeman’s three-year-old son, Maximus. Inside a matter of days, he developed a limp, couldn’t sit up and finally couldn’t breathe on his personal.
Though the “why” behind GBS is unknown, it strikes days or perhaps weeks after one other sickness, resembling a respiratory or gastrointestinal bacterial or viral an infection, has cleared.
“There’s no illness that I am aware of that’s like Guillain-Barré syndrome. It occurs in sufferers who’re in any other case utterly wholesome,” stated Love. GBS impacts 3,000-6,000 individuals within the U.S. annually, in keeping with the CDC, and is commonly handled with off-label intravenous immunoglobulin remedy.
“It has some impact, relying upon how extreme the illness is. However the extra extreme the illness, the much less efficient IVIG is,” Love stated.
Nevertheless, there’s no FDA-approved remedy for the illness, and whereas most sufferers recuperate, the method might be lengthy and brutal.
“It is a neurological nightmare for sufferers that deserves an applicable remedy.”
Douglas Love
CEO, Annexon Biosciences
Annexon Biosciences desires to alter that. The corporate efficiently accomplished a pivotal section 3 trial for its lead candidate, ANX005, an immunotherapy that targets and blocks C1q, the molecule that initiates the classical complement pathway related to GBS. ANX005 obtained FDA quick observe and orphan drug designations.
“We’re pursuing what’s a singular or numerous method from different complement applications in that we’re concentrating on upstream complement exercise,” Love stated. “It localizes and prompts complement or irritation proper on illness tissue, so that you might be extra proximal in your method in concentrating on aberrant irritation.”
A June section 3 readout confirmed ANX005 had statistically vital outcomes on key endpoints, together with on the GBS incapacity scale.
Not like IVIG, which is run over 5 days, ANX005 is given by IV infusion inside a number of hours and works that rapidly, too, Love stated.
“Our drug is kind of easy. It’s designed to dam C1q instantly upon administration,” he stated. “It speaks to the ability of our method, which is rapidly shutting down C1q, shutting down the illness course of, and permitting sufferers to maneuver from a state of decline to stopping the illness course of.”
Love stated the corporate plans to file its biologics license utility for ANX005 within the first half of 2025.
Though the GBS market is small, Annexon can have it cornered as the primary and solely authorized remedy if it wins an FDA OK. As well as, Love stated there’s a 90% compliance fee in treating GBS, and 80% of the illness’s incidence happens in 5 massive states and in “only a few hundred hospitals.”
“Though [GBS] occurs acutely and sporadically, it occurs the place you’ve extra individuals,” he stated. “That enables us to have a really environment friendly business footprint.”
ANX005 isn’t the one product in Annexon’s pipeline concentrating on C1q. Its different flagship applications are in geographic atrophy and complement-mediated autoimmune illnesses, in addition to applications in Huntington’s illness, ALS and lupus nephritis.
“We are the first, and up to now, the one firm concentrating on C1q within the clinic,” Love stated. “By concentrating on C1q the place it initiates this inflammatory illness course of, it permits us to pursue a large swath of indications that you do not essentially all the time see with complement applications.”
First up, although, is an opportunity to alter the trajectory of GBS.
“It is a neurological nightmare for sufferers that deserves an applicable remedy,” Love stated.
