uniQure: An FDA Approval, An Advancing Pipeline And A Critical Data Readout

Trending 1 year ago

Red eritrosit humor count aesculapian concept



Partner CSL obtained an FDA support connected Hemgenix and uniQure (NASDAQ:QURE) will now beryllium eligible for milestones and double digit royalties reaching nan debased 20s. This gross will money uniQure's pipeline, which is expanding and supports a valuation in excess of nan existent stock price. This article is an update connected nan pipeline, an appraisal of nan commercialized scenery for Hemgenix and a position connected nan upcoming binary readout connected AMT-130. The captious catalyst for uniQure remains a information readout for AMT-130 successful Huntington's disease, which is owed successful 2023.

AMT-260 - The pipeline expands

At an R&D event, nan institution presented information connected cistron therapy asset, AMT-260, for nan curen of refractory temporal lobe epilepsy (rTLE). Patients pinch rTLE, which is nan astir communal shape of localized epilepsy, acquisition seizures. Patients acquisition mediocre successful value of life and accrued mortality and morbidity. Current modular of care treatments see medications and successful immoderate cases epilepsy room and implantable devices, which some person information issues. These procedures person nan imaginable for a nonaccomplishment of cognitive usability and location is variability successful outcomes. There are 800K patients successful nan US pinch rTLE and frankincense location is simply a important marketplace opportunity.

An IND is expected successful 2023 for AMT-260. It is an AAV9 cistron therapy pinch 2 miRNAs designed to trim nan look of nan GRIK2 gene, pinch nan extremity of reducing neuronal excitability. The first information successful humans is expected successful 2024.

Mice treated successful preclinical models showed a simplification successful seizure frequency, and nary toxicity aliases information issues were noted. Brain insubstantial resected from humans (post surgery) were transduced successful nan laboratory pinch AMT-260 and these pre-clinical experiments evaluated epileptic activity and biomarkers. Results propose AMT-260 is progressive and location is target engagement.

uniQure has group nan extremity of a 50% simplification successful seizures erstwhile tested successful quality subjects. They expect that nan study will enroll adults pinch a test of rTLE pinch terrible disease. The study will apt beryllium designed arsenic an open-label, controlled study wherever AMT-260 is compared to patients taking modular anti-seizure medication. The proceedings will trial 2 doses, and AMT-260 will beryllium administered by a neurosurgeon straight into nan hippocampus. Endpoints for nan study which whitethorn beryllium reported see safety, seizure wave and biomarkers.

In addition, uniQure precocious licensed an plus for ALS caused by nan SODI mutation and is moving connected an further pre-clinical asset, AMT-191 for Fabry's disease.

Hemgenix was approved by FDA

Hemgenix, a cistron therapy developed by uniQure was approved by FDA for nan curen of hemophilia B. Pivotal studies met nan superior non-inferiority endpoint and demonstrated superiority connected a secondary endpoint. The support included patients who presently usage FIX prophylaxis therapy, person existent aliases past life-threatening hemorrhage, aliases person repeated terrible spontaneous bleeding episodes. Because Hemgenix uses an AAV5 (rather than different AAVs wherever neutralizing antibodies whitethorn effect treatment) astir hemophilia B patients will beryllium eligible for treatment.

The database value of $3.5m was announced. ICER suggested nan precocious limits for nan value of Hemgenix to beryllium $2.9m, which reflects a value that delivers use to nan aesculapian system. Treating hemophilia B tin costs $20 m complete a patient's life and frankincense assuming nan cistron therapy is durable, important savings tin accrue for insurers arsenic they trim aliases destruct nan request for costly FIX and hospitalizations. It is notable that immoderate patients dosed early successful objective improvement person showed sustained efficacy retired to 10 years. Pricing, while incredibly expensive, is accordant pinch BioMarin's Roctavian for hemophilia A which was priced astatine $2.5 m.

uniQure is not responsible for immoderate of nan costs of commercialization, and partner CSL will incur these costs. However, Pfizer will apt beryllium bringing to marketplace a competing cistron therapy for hemophilia B patients.

Pfizer's BENEGENE-2 study met its superior endpoint of non-inferiority and superiority successful nan annualized bleeding complaint ('ABR'). Patients acquisition a mean ABR for each bleeds of 1.3 for nan 12 months from week 12 to period 15 compared to an ABR of 4.43 during nan pre-treatment period, resulting successful a 71% simplification successful ABR (p<0.0001). Data precocious released suggests that for Hemgenix, nan ABR for each bleeds was reduced by 64% during months 7-24 of nan study (mean ABR 1.51 vs. 4.18 during nan lead-in period; p=0.0002).

Pfizer's merchandise met nan barroom of superiority complete SoC, while Hemgenix was non-inferior connected nan superior endpoint. However, successful patients treated pinch Pfizer's cistron therapy, location were fourteen superior adverse events pinch 2 deemed related to treatment. In contrast, location were no serious adverse events successful patients treated pinch Hemgenix successful nan pivotal study. In addition, patients pinch existing antibodies to nan AAV were excluded from curen pinch Pfizer's cistron therapy. Given this, Hemgenix appears to connection comparable efficacy pinch a amended information floor plan and nary request to surface and exclude patients pinch neutralizing antibodies.

H.C. Wainwright expert Patrick Trucchio wrote that nan information for some cistron therapies demonstrated comparable ABR simplification but that Pfizer's pointed to "a much analyzable information profile. " He suggested that Hemgenix "should go nan starring HB cistron therapy connected nan market," and placed a Buy standing and $90 value target connected uniQure shares. Wells Fargo's expert described nan precocious accounted Pfizer information arsenic "competitive" and cited Pfizer's commercialized prowess arsenic an advantage. However, CSL presently markets aggregate hemophilia products and frankincense has existing relationships pinch nan mini cohort of prescribers for treated Hem B and frankincense should beryllium good positioned commercially.

AMT-130 - Huntington's Disease is simply a precocious consequence programme and competitors person not been successful successful nan clinic

For a elaborate explanation of Huntington's illness and AMT -130, please publication my anterior SA article. The erstwhile 2 years person been filled pinch disappointment for nan HD community. Roche's shape 3 tominersen study yielded disappointing results. Roche has revised their protocol to see younger, earlier shape patients and is moving a caller phase 2 trial. Wave Life Sciences had 2 early shape assets that failed. Novartis's branaplam produced broadside effects including peripheral nervus damage, and nan proceedings was halted. PTC Therapeutics halted their study while they reappraisal information protocols. Triplet Therapeutics, a backstage company, had early disappointing information and closed aft their supplier showed signs of toxicity and nan institution could not raise further capital.

While these failures item that nan chartless biology successful HD makes it difficult to creation therapeutics, it is notable that uniQure's AMT-130 is highly differentiated. The AMT-130 proceedings only enrolled patients early successful nan objective people of nan disease. AMT-130 was distributed straight and exclusively to nan tissues affected successful nan striatum via a neurosurgical process and critically, AMT-130 targets exon 1 HTT, a toxic macromolecule implicated successful nan illness which nary of nan different medicines target.

By mid 2023, uniQure will merchandise further information connected nan first cohort treated pinch AMT-130. There will beryllium information provided for sixteen patients who received nan precocious dose. This cohort includes 10 patients who were treated and six power patients pinch 1 twelvemonth of travel up. There will besides beryllium information connected 10 patients who received nan low-dose and 4 power patients pinch 2 years of follow-up.

This information readout will see functional and cognitive testing which tin beryllium compared to placebo patients and earthy history data. This will beryllium nan first information connected whether location is an efficacy signal. Importantly, volumetric MRIs will beryllium conducted to measure encephalon atrophy. Imaging studies conducted successful pre symptomatic and early shape HD patients person shown that quantitative measures of nan striatum are meticulous biomarkers measuring illness progression.


uniQure has $440 million successful cash, which they judge will beryllium capable done 2025. Milestone payments whitethorn adhd an further $175m further extending nan company's runway. Royalties will besides beryllium owed to uniQure. uniQure whitethorn ne'er request to raise costs successful nan superior markets again, fixed nan gross watercourse from nan CSL licensing deal.


AMT-130 remains a very precocious consequence program, but they now person a dependable gross watercourse which cushions nan rustle if it fails to show efficacy. At nan clip nan CSL woody was announced, shares tumbled arsenic uniQure nary longer appeared to beryllium an M & A target. However, now, they are positioned to reap nan rewards of this deal. Given nan very important rate position and gross stream, nan institution is apt to beryllium aforesaid sustaining and beryllium successful nan enviable position of not requiring backing from nan superior markets during this difficult time.

Critically, Goldman Sachs analysts person weighted nan hemophilia B royalty stream/milestone payments and propose it unsocial supports a $42/share valuation. Given this, astatine nan existent stock value of astir $20, location appears to beryllium constricted downside should AMT-130 neglect to show efficacy.

For investors, if AMT-130 is simply a breakthrough for Huntington's patients, Raymond James expert Danielle Brill estimates location is simply a $7B marketplace for HD medicines. Given this and nan unfortunate way grounds of competitors, investors would apt beryllium highly good rewarded if nan information successful mid 2023 for AMT-130 shows impervious of concept. While I would delegate a debased probability of occurrence fixed nan chartless biology, nan downside protection provided by nan CSL gross makes nan consequence reward look favorable.

This article was written by

Sage Advisors floor plan picture

M.A. Science. I usage my subject background, nan knowledge acquired while moving for a awesome pharmaceutical institution and entree to scientists pinch domain circumstantial knowledge to find nan champion opportunities successful biotech.

Disclosure: I/we person a beneficial agelong position successful nan shares of PFE either done banal ownership, options, aliases different derivatives. I wrote this article myself, and it expresses my ain opinions. I americium not receiving compensation for it (other than from Seeking Alpha). I person nary business narration pinch immoderate institution whose banal is mentioned successful this article.

Additional disclosure: I americium not a financial advisor and this article should not beryllium interpreted arsenic proposal to bargain aliases waste immoderate security. Investors should execute their ain owed diligence and consult their financial advisor.

Editor: Naga

Read other contents from at
More Source