Scientist turned investor at Bigger Capital
Las Vegas
Joined October 2013
- Tweets130
- Following161
- Followers373
- Likes12.6K
$NWBO “The Prime Minister has taken a personal interest in this and wants to assure an action is taking as soon as possible”
Let’s go Braelyn! @MHRAgovuk it’s time to take action.
Mathias Bigger retweeted
While I'm excited about today's announcement from uniQure, I have very mixed emotions.
I believe AMT-130 works. I believe in its potential to change the future of Huntington's disease. But the road to this point has been an emotional roller coaster for our community, and we're still nowhere near the finish line.
I've lost count of how many advocates and families have asked me, "Why is it so hard for us to get this treatment?"
And the heartbreaking part is that I don't have an answer. Because it shouldn't be this hard.
I'm relieved that the FDA has finally accepted the application for Priority Review, and I welcome the opportunity for an Advisory Committee to examine the evidence and hear directly from patients and families.
However, I still have serious concerns about the proposed confirmatory trial, which I plan to address separately.
We are not asking for shortcuts. We are asking for a regulatory pathway that recognizes the reality of a relentlessly progressive disease and the human cost of every delay.
I'm also deeply disappointed by uniQure's communication with the very community that has fought so hard to get us here. Patients and families deserve transparency, consistency, and respect throughout this process.
But despite all the frustration, today is a step in the right direction.
And as I've said before, we have to allow ourselves to celebrate the wins along the way.
So today, I'll celebrate this progress. Tomorrow, I'll get back up, dust myself off, and continue fighting.
Because progress deserves to be celebrated, but our fight is far from over.
And until we have meaningful treatments available to the people who desperately need them, we cannot afford to stop.
#HuntingtonsDisease #raredisease #TimeMatters #ProgressionDoesntPause
@FDA_KyleD @FDA @FDADrugs @uniQure_NV
"uniQure Announces FDA Acceptance and Priority Review of the Biologics License Application for ifezuntirgene inilparvovec for the Treatment of #HuntingtonsDisease" Check out the full press release on the Huntington's Post! #HDResearch
help4hd.org/post/uniqure-ann…
$CLPT $QURE great decision by the FDA. Major Revenue catalyst for Clearpoint: more spending by UniQure to reach commercial scale, new sites being initiated, and increased enrollment effort on the confirmatory trial (a couple million in revenue to CLPT).
For $QURE this means approval is, in my opinion, highly likely. The four year data is supportive of effectiveness and the TFC benefit is real. Doctors and patient advocates will have to step up for the Adcom which has not been a problem in the slightest for AMT130, special shout to @laurencurehd
UniQure $QURE announces FDA acceptance and Priority Review of BLA for AMT-130 for treatment of Huntington's disease
PDUFA May 1, 2027
"In its acceptance letter, the FDA stated that they are tentatively planning to discuss this application at an Advisory Committee meeting. The Company will be notified once the date and timelines have been finalized."
Mathias Bigger retweeted
We are thrilled to announce that the FDA has accepted for review the BLA for ifezuntirgene inilparvovec (AMT-130), an investigational #genetherapy for #HuntingtonsDisease. The application has been granted Priority Review with a PDUFA date of May 1, 2027. uniqure.gcs-web.com/news-rel…
Mathias Bigger retweeted
Yesterday, I had the privilege of attending the FDA’s Rare Disease Framework Initiative public meeting.
I had brain surgery for recurrent Glioblastoma (GBM) just a few weeks ago, but I flew to Maryland for this meeting because this new framework has the potential to change the future of GBM.
I’ve fought this disease for 4.5 years, using every tool available. But my best chance now, at recurrence, is a treatment that is currently only accessible in the UK, a personalized cancer vaccine called DCVaxL.
I made sure to freeze my tumor tissue specifically so I could have a chance at getting this vaccine. I should find out within the next week or two whether my tissue sample is adequate for vaccine manufacturing. More details to come.
@HollensbeAmanda @LoriMills4CA42 @andrewcaravello @intrstedobservr
Mathias Bigger retweeted
$CLPT — MASSIVE CATALYSTS AHEAD!
Following the company? You don’t want to miss this one:
investingwithsalva.substack.…
Mathias Bigger retweeted
🇺🇸❤️ WE MET PRESIDENT TRUMP TONIGHT! ❤️🇺🇸
What an extraordinary evening for our family!
Tonight, Braelyn, Ben, and I had the incredible opportunity to meet President Trump at the rally in Pendleton, South Carolina!
We had only about 20–30 seconds with him, and I knew I needed to make every second count.
I introduced him to Braelyn and thanked him for telling Dr. Oz about her brain cancer story.
I explained that Braelyn had been diagnosed with glioblastoma, the deadliest type of brain tumor, but that today she is CANCER-FREE after receiving DCVax-L, a personalized cancer vaccine developed right here in America!
Yet we must travel all the way to England for her ongoing treatments because we cannot access it here in the United States.
Then something pretty cool happened!
President Trump turned to one of his staffers and instructed them to get me in touch with Dr. Oz!
I also expressed my hope that we could connect with Secretary Kennedy.
As our brief time together came to an end, I made one final appeal:
“Please help bring this American-made treatment back home to America.”
President Trump was incredibly gracious to our family and very kind to Braelyn. We are so grateful for the opportunity to speak with him!
Earlier in the evening, I was also able to give a member of his staff a detailed packet about Braelyn’s journey, DCVax-L, and the obstacles American patients face in accessing promising investigational treatments.
Now we are praying that the connection with Dr. Oz comes through!
Three years ago, Braelyn was given just months to live. Today, she’s 19, attending college, and her latest MRI shows NO EVIDENCE OF DISEASE RECURRENCE!
Tonight, we had the opportunity to bring her story directly to the President of the United States. ❤️
My prayer is that this opens the door to a much bigger conversation—one that could change the future for countless American cancer patients.
📸 We were not permitted to bring our phones into the private meeting with President Trump, so we couldn’t take our own pictures with him. His staff took an official photograph of us together, which we’re told will be emailed to us. I can’t wait to share it! In the meantime, here are some pictures from our unforgettable evening.
Thank you to EVERYONE who helped make tonight possible and to all of you who continue to share Braelyn’s story. You helped us get this far, and we’re not stopping now! ❤️🙏
@realDonaldTrump @DrOzCMS @WhiteHouse
#DCVaxForBraelyn #DCVax
$NWBO American GBM patients should have the right to access DCVax. Dr. Bosch presented this past Sunday at the Atria Health and Research Institute in NYC. 5 month OS benefit, tail is long, easy injection to the arm, no toxicity, and the combination potential is extraordinary.
Mathias Bigger retweeted
Let Dr. Liau have her All Solid Tumors Cancer Moonshot. @houmanhemmati @ScottGottliebMD
Mathias Bigger retweeted
❤️ IT’S OFFICIAL! WE’RE MEETING PRESIDENT TRUMP TOMORROW! 🇺🇸
Braelyn and I have been invited to attend tomorrow’s rally for Darline Graham in Clemson! We’ll be seated in the VIP section AND have the opportunity to meet President Trump in the “click line”!
Ben is cutting his work trip short, and we’re hoping he’ll make it home in time to join us!
We’ll have only seconds with the President, but I intend to make every one of them count. I’ll also be bringing information for him and his staff about Braelyn, DCVax-L, and the barriers American patients face trying to access promising treatments developed right here in the United States.
This is an extraordinary opportunity, but we’re hoping it can become something even bigger.
PLEASE HELP US GET THE ATTENTION OF THE WHITE HOUSE PRESS TEAM! 🙏
We would love the opportunity for a longer conversation with President Trump or his administration about how we can make these treatments accessible to American patients.
Please SHARE, REPOST, and TAG anyone who might help get Braelyn’s story in front of the right people!
Three years ago, Braelyn was given just months to live. Today she’s 19, attending college, and her latest MRI shows NO EVIDENCE OF DISEASE RECURRENCE!
Her story deserves to be heard. And so do the stories of countless other patients fighting for access to promising treatments.
@realDonaldTrump @DrOzCMS @WhiteHouse
#DCVaxForBraelyn #DCVax #PresidentTrump
My blog post on Alpha Cognition’s long-term Alzheimer’s care treatment ZUNVEYL. I will appreciate any of your feedback or criticism. $ACOG
biggercapital.com/blog-posts…
Mathias Bigger retweeted
Still waiting for @VPrasadMDMPH to to sit down for a real interview or podcast where the host(s) don't kiss his ass with softball questions.
We published this blog post on $SNWV. Check it out.
Wow shocker. Who could’ve guessed?
Ahead of the highly anticipated AMT130 4 year data readout from $QURE @uniQure_NV delivered by $CLPT @ClearPointNeuro I like to rewatch this excellent video by Dr. Ben Miles about the history of HD and the science behind AMT130. The repeat CAG seq was only discovered in 1993, BY HAND! Wow we have come far.
While acknowledging entropical factors are at play in data readouts, the MOA of AMT130 is extremely robust in my opinion. Hoping for the best for the patients, scientists and investors.
youtu.be/g66x9funPc0?si=dfCV… via @YouTube
