Lager en tråd for Orphazyme. Dansk selskap med to Fase 3-readouts i 1H 2021. Market cap ca 2,4 mrd DKK.
Redeye har bear case på 70. Stor oppside.
Melding fra november:
9.11.2020, 07:20 · Ritzau Finans
Orphazyme søger om markedsføringstilladelse i Europa
Biotekselskabet Orphazyme har ansøgt om markedsføringstilladelse i Europa for lægemiddelkandidaten Arimoclomol til behandling af den sjældne arvelige hjernesygdom, Niemann-Pick type C, NPC
Det fremgår af en meddelelse.
Ansøgningen var oprindeligt stillet i udsigt i andet halvår, så Orphazyme følger planen.
I USA venter Orphazyme en tilbagemelding fra den amerikanske lægemiddelstyrelse, FDA, på en tilsvarende ansøgning senest den 17. marts.
- Indsendelsen i Europa er en væsentlig milepæl for Orphazyme, mens vi arbejder mod vores første potentielle godkendelse af Arimoclomol i større markeder, siger administrerende direktør i Orhazyme Kim Stratton ifølge meddelelsen.
Der lever ifølge Orphazyme omkring 1800 personer i USA og Europa med hjernesygdommen, der giver anledning til ophobning af kolesterol i kroppens organer. Sygdommen vurderes at ramme omkring én ud af 100.000 nyfødte.
Orphazyme is developing arimoclomol in four indications: Niemann-Pick disease Type C (NPC), Gaucher disease, Amyotrophic Lateral Sclerosis (ALS), and Inclusion Body Myositis (IBM).
In our Phase 2/3 clinical trial of arimoclomol in NPC, we have observed evidence of slowing of disease progression. We completed submission of a rolling New Drug Application (NDA) in the US in July 2020. This was accepted in September 2020 with priority review and a target action date of March 17, 2021 under the Prescription Drug User Fee Act (PDUFA). We submitted for marketing approval in Europe in November 2020.
In a Phase 2 study in Gaucher disease Type 1 and Type 3, arimoclomol demonstrated marked improvements in key clinical markers including liver and spleen size.
A Phase 2/3 trial in IBM and a Phase 3 trial in ALS are all fully enrolled and topline results are anticipated in H1 2021
Pusher videre, jeg kjøpte på 64 og 65 forrige uke, steg til 69 i går og 74 i dag.
Sterk avslutning i dag nær HOD. Nærmer vi oss 80 før nyttår?
Hm…denne var interessant. Hvor god er medisinen sammenlignet med dagens behandling?
Finnes vel ingen godkjente behandlinger i US for NPC. Her har selskapet Priority Review og får svar fra FDA innen 17.mars.
Finnes også særs få godkjente behandlinger innen både ALS og IBM. Her skal selskapet lese av topline data fra registreringsstudie i begge indikasjoner i 1H2021.
Orphazyme provides regulatory update on arimoclomol for NPC
Company Registration No. 32266355
Copenhagen, Denmark – December 27, 2020 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, announced today the U.S. Food and Drug Administration (FDA) has extended the review period of the New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC) by a standard extension period of three months. This extension is necessary for the FDA to complete its review. The updated Prescription Drug User Fee Act (PDUFA) target action date is June 17, 2021.
The FDA has confirmed the NDA remains under Priority Review, and the extension does not impede eligibility for a Pediatric Rare Disease Priority Review Voucher. The FDA grants Priority Review to applications for potential therapies that, if approved, could offer a significant improvement in safety or effectiveness, diagnosis, or prevention of serious conditions.
“Orphazyme is working closely with the FDA to support the final review of the new drug application for arimoclomol,” said Molly Painter, US President, Orphazyme. “There is significant unmet medical need for the treatment of NPC, and we are committed to bringing arimoclomol to patients in the U.S. and Europe as soon as possible.”
“We have responded to all FDA information requests and submitted all outstanding information regarding the arimoclomol NDA for NPC,” said Thomas Blaettler, Chief Medical Officer, Orphazyme. “The Phase 3 trials for Amyotrophic Lateral Sclerosis and Inclusion Body Myositis remain on track for read-out in the first half of 2021 and we look forward to providing an update on our progress.”
Arimoclomol has received FDA Fast-Track and Breakthrough Therapy Designations for NPC, as well as Orphan Drug and Rare Pediatric Disease Designations. If approved in the US, arimoclomol will be the first and only approved medicine for NPC, a rare, relentlessly progressive, neurodegenerative disease with an estimated incidence of one in 100,000 live births. In November 2020, the company also submitted a Marketing Authorisation Application to the European Medicines Agency for arimoclomol in NPC.
For additional information, please contact
Copenhagen: Anders Vadsholt, CFO +45 28 98 90 55
Chicago: Molly Carey Poarch +1 773-770-6888
About Orphazyme A/S
Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat-Shock Proteins (or HSPs) to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S., Switzerland, France and Germany. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA.CO).
Arimoclomol is an investigational drug candidate that amplifies the production of Heat-Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally, crosses the blood-brain barrier, and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, IBM, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC, IBM, and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC.
About Niemann-Pick disease Type C (NPC)
Niemann-Pick disease Type C (NPC) is a rare, genetic, progressively debilitating, and often fatal neurovisceral disease. It belongs to a family known as lysosomal storage diseases and is caused by mutations leading to defective NPC protein. As a consequence, lipids that are normally cleared by the lysosome accumulate in tissues and organs, including the brain, and drive the disease pathology. We estimate the incidence of NPC to be one in 100,000 live births and the number of NPC patients in the United States and in Europe to be approximately 1,800 individuals. There are no approved treatments for NPC in the U.S.
This company announcement may contain certain forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.
Tanker om meldingen? Litt kjedelig med 3mnd utsettelse.
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.
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.
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.
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.
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.
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
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…
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…