Ja kortsiktig er dette negativt noe dagens startkurs viser. Det går ikke frem årsaken til de ekstra 3 månedene, så det kan være alt fra at FDA har for mye å gjøre med vaksinegodkjenninger til kompleksitet vedr akkurat denne medisinen.
Det er flere selskaper som har fått utsettelse på behandling av FDA. Mener Biovica fikk utsatt sin 510K søknad også. Det ble opplyst at årsaken var høy belastning på FDA. Såfremt ikke annet blir kommunisert, så skal ikke en utsettelse i seg selv være problematisk. Men det er allikevel slik at ingen liker forsinkelser.
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Orphazyme: Our View to the Regulatory Update in NPC
Redeye Research Note 2020/12/28
Redeye reiterates its probability rate for arimoclomol in NPC. While the extended NDA review, from previously March 17, 2021, updated to June 17, 2021, is initially disappointing, we also note that all outstanding information has now been submitted to the FDA. We consequently see a small risk for additional delays.
Background
The FDA has extended the NDA review period for arimoclomol in Niemann-Pick Disease (NPC) with a standard 90 days extension. The NDA remains under Priority Review, and the extension does not impact the eligibility for a potential Priority Review Voucher (PRV). The updated PDUFA date is set to June 17, 2021, extended from previously March 17, 2021.
Our View
We had looked forward to March 17, and believe we share the initial disappointment with the management team and investors on this delay. In the press release, we learn that Orphazyme has “responded to all FDA information requests and submitted all outstanding information regarding the arimoclomol NDA for NPC.” It also encompasses the crucial QTc clinical data. Thus, we see a small risk for additional delays. Based on the arguments below, we even believe that a potential decision by the FDA could come earlier than June 17:
- It is important to stress that the NDA remains under priority review
- Also, we want to highlight the urgent medical need in NPC
- The FDA does not plan to hold an advisory committee with expert panelists
Naturally, other factors speak against an earlier approval. It attributes not least to the ongoing pandemic that complicates, for instance, site visits. Further, we cannot rule out that additional factors come up during the extensive NDA review, which could impact the timeline.
Impact on Valuation
Orphazyme is a relatively small biopharma company, with not unlimited resources and we also see a risk that the US delay impacts the timeline for the EMA process. Hence, we don’t factor in any sales in the next year in Europe (a minor adjustment). For the US market, we factor in slightly lower sales for the coming years. The estimate changes fall under our margin of safety in our valuation, and we reiterate our Base Case of DKK 145 per share.
When delays occur, it is relevant to emphasize exclusivity protection. Arimoclomol in NPC has Orphan Drug Designation. From approval, it entitles to seven years of market exclusivity in the US and ten years of market exclusivity in the EU. Orphazyme also has a method of use patent for arimoclomol in NPC that expires in 2029, with a potential extension to 2032 (US) and 2034 (EU).
It is no surprise that the market trades the stock down on today’s news, currently in the range – 5-10%. Delays and uncertainties do seldom appeal to investors. Based on what we know today, our stance is that this news is about FDA needs sufficient time to review the NDA and that a decision will come on June 17 at the latest.
Anders Hedlund
Equity Analyst
Har ikke kjøpt selv ennå, men må se om eg klarer få den inn en plass. så ikke mere stigning nå på en stund 
fra i dag - redeye.
Orphazyme
We chose to include Orphazyme, a pure orphan play, in our 2021 top picks for the following reasons:
- The current valuation represents a significant EV discount to other late-stage, Scandinavian orphan companies
- It is well-funded (est. > DKK 700 mn at 2020 yearend), which could be strengthened by another USD 100 million in non-dilutive cash. This follows if arimoclomol gets approved in NPC by the FDA this year. Orphazyme could then be entitled a Pediatric Review Voucher (PRV), which can be sold to a third party for about USD 100 million
- Orphazyme has a news flow in the first half of 2021 that other biotech companies could dream of. It includes:
- Two phase 3 readouts (in ALS and sIBM)
- Potential approval in NPC, the furthest advanced indication, in both the US and the EU
- Potential advancement into a pivotal stage in a fourth rare indication; Gaucher disease
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Noen som har en viss innsikt i fase 2 resultatene sett ift dagens soc? Bør jo kunne gi ett visst hint ift godkjenning osv…
Et ordentlig hak på min gamle aktie, som jeg solgte for tidligt men alligevel bedre end nu.
Jeg fikk heldigvis solgt meg ut tidligere denne uken…
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Jeg hoppet også av alt for tidlig her. Heldigvis. Caset med to Fase 3 utlesninger så fristende ut, men resultatene viser at dette er en bransje med mye risiko.
Ja det er helt sikkert, biotek er ikke for pyser…
Den går da fuldstændig amok!
Some investors are hinting that it might be up 900% due to a short squeeze. The company has 34.95M outstanding shares with a float of 24.59M shares and only 65k volume in the last 3 months. A very low float can cause the stock to skyrocket.
Now, you’re asking what caused people to buy if there’s no news?
Well, one poster on the Yahoo Finance Board posted;
“In 2011, CytRx sold the rights to arimoclomol to Orphazyme A/S (formerly Orphazyme ApS) in exchange for a one-time, upfront payment of $150,000 (USD) and the right to receive up to a total of $120 million (USD) in milestone payments upon the achievement of certain pre-specified regulatory and business milestones, as well as royalty payments based on a specified percentage of any net sales of products derived from arimoclomol. Orphazyme, a public company trading on the Nasdaq Copenhagen exchange, is testing arimoclomol in three additional indications beyond ALS, including Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM). Orphazyme anticipates announcing top-line results for their pivotal Phase 2/3 trial in NPC during 2018, and if positive, expects to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration and a Marketing Authorization Application (MAA) to the Europeans Medicines Agency (EMA) during the second half of 2019”
Yahoo Finance Board User
Although its reported that, They failed their Phase 1 for the treatment of ALS, its reported that the PDUFA date for Niemann-Pick is June 17, 2021. They are pivoting towards Niemann-Pick.
In January 2019, the medication narrowly missed its first primary objective in a 50-person Phase II/III NPC research study. The severity of the condition was reduced by 74%, and the p-value was 0.0506. Disease development slowed in individuals over the age of 4 and in children who had been receiving an Actelion medicine, which is sometimes prescribed for the disease, according to two pre-specified subgroup analyses. However, the business did not reveal any data for this round of ALS trials, putting the public in the dark about how it performed in the experiment.
The company said in a statement that topline data will be provided during the virtual European Network to Cure ALS meeting from May 12-14, while detailed data will be released later in the year.
Au. Da har vel Orphazyme misset på alle 3 skuddene de hadde på mål, og er vel sånn sett «ferdig»
Ingen penger og ingen dokumenterbare prosjekter så spøker det ja.
Ny base case fra Redeye 15DK, Bear Case 0Dk…
Biotek er binært…
Og alligevel har man kunne lave 100 % hvis man havde mod til det i morges de første 700000 aktier kostede 22-26 kr…
Her er det nok tradere som kjører opp aksjen for å komme ut på en gunstig kurs. Jeg blir forbløffet om denne står over 30 om en uke etter alle bomskuddene dette halvåret.
Ja jeg havde heller ikke set den over 30 i dag, men den bliver kørt op.
også er det hele USA delen de får godt nok omsat nogle aktier.