Posts by pee198

    Wurde verschoben....


    • Aprocitentan – NDA under review with the US FDA – new PDUFA date of March 19, 2024 – MAA under review with the European Medicines Agency.

    Jean-Paul Clozel, concluded:

    “The review process with the US FDA is progressing well, though it is likely to require an extension to the review period of up to 3 months as the company will provide additional Risk Evaluation and Mitigation Strategy (REMS) materials to support a streamlined REMS which is designed specifically for patients taking aprocitentan.”


    Idorsia | Media release

    Zur Erinnerung:


    Prescription Drug User Fee Act (PDUFA) current date: December 19, 2023, and the market authorisation application (MAA) was submitted to the EMA at the end of January 2023.

    Stock Isn't Priced For FDA Approval

    I do not believe that the current market cap of $419 million assumes near-term profitability. Essentially, the stock is priced for failure.


    From a valuation standpoint, assigning a direct price target with so much up in the air wouldn't be fair. However, I wanted to extend my forecast into a simplified DCF to see just how far off the market cap might be. I made the following conservative assumptions:


    Q4 performance in 2024 annualized to 2025

    A long-term growth rate of 3% essentially pacing with inflation

    Cost of capital at 10%

    With those assumptions and the forecast above, the value of the company would be $5.8 billion versus $419 million today.


    To be clear, I am not suggesting a $5.8 billion valuation because there is a lot of execution timing at play. However, even considering timing and execution risk, I do not feel this is even close to a $400 million company.


    In my opinion, the stock is priced for an IP sale. A competitor would likely scoop up existing therapeutics if FDA approval were denied or delayed. The book value of bluebird is closer to $200 million, and therapeutic premiums have been running at 50-60%, giving a floor value of approximately $300 million.



    Verdict

    Assuming FDA approval, bluebird's current market cap at $419 million seems undervalued, given the potential growth trajectory. The simplified DCF valuation, albeit with conservative assumptions, suggests a $5.8 billion valuation, starkly contrasting its current standing. Even considering execution timing and potential risks, the company appears significantly undervalued.


    Even in the event of an FDA approval delay or denial, the IP sale and therapeutic premiums provide a floor value of around $300 million, implying limited downside risk. Thus, the stock seems priced more for an IP sale rather than profitability through FDA approval.


    With asymmetric risk in mind, I cautiously recommend a 'Buy' on bluebird. The potential upside appears substantial compared to the current market cap. However, investors should remain cognizant of the inherent risks and uncertainties associated with FDA approval and the execution of business growth plans.

    bluebird bio Isn't Priced For Success


    Summary


    bluebird bio is a clinical-stage biotechnology company specializing in developing and commercializing gene therapies for severe genetic and rare diseases.

    bluebird has had a rough run, down nearly 100% from its 2018 high and down 49% across the past year with a limited cash runway.

    The downside risk for this stock is material, from both FDA approval and cash flow. However, the floor isn't $0, as competitors would more than likely buy out the company.

    With a floor on the downside and the potential for an outsized return on IP, I believe this is an asymmetric buy opportunity that will likely only exist through December 20th.

    bluebird bio (NASDAQ:BLUE) is a clinical-stage biotechnology company specializing in developing and commercializing gene therapies for severe genetic and rare diseases. Their business model hinges on establishing and scaling the commercial model for ex-vivo gene therapy. In 2023, they strengthened their commercial presence through multiple product launches. One key pillar of bluebird's commercial strategy is educating families and healthcare providers about their innovative treatments. Additionally, bluebird has formed strategic alliances to foster product and manufacturing innovation in cell therapy.


    bluebird has had a rough run, down nearly 100% from its 2018 high and down 49% across the past year. In addition, short interest is running shy of 24%, and the company only has a cash runway through Q2 to Q4 2024.


    However, bluebird is also within three weeks (December 20th) of potential FDA approval for a gene therapy to treat Sickle Cell. In addition, they have positioned themselves for growth related to existing treatments and can ramp up quickly for newly approved therapies.


    The downside risk for this stock is material, from both FDA approval and cash flow. However, the floor isn't $0, as competitors would more than likely buy out the company for IP.


    With a floor on the downside and the potential for an outsized return on IP, I believe this is an asymmetric buy opportunity that will likely only exist through December 20th. While investors should proceed cautiously with government approval as the primary driver of value, this could be a worthwhile bet.



    FDA Approval Likely Based On Signals

    The biggest downside risk and, conversely, upside potential is FDA approval of bluebird's Lovo-cel treatment for Sickle Cell Disease on December 20th.


    While never definite, signals are pointing to approval. First and foremost, the FDA approved Lovo-cel for accelerated approval. Once converted to accelerated approval, nearly every therapy is approved unless the company itself withdraws to the traditional process following an advisory committee recommendation. bluebird did not have to undergo an advisory committee review. In fact, many have criticized the FDA for this pathway to approval being too easy. Also, treatments put through accelerated approval have a strong track record, with only 13% being later withdrawn from the market.


    The next signal is the robust follow-up and strong results from the clinical trial. There was a 90% improvement in the target symptom with limited adverse effects. Gene therapies were first approved in 2017, and since then, the clinical trials have become more comprehensive, and the FDA's approval rate has increased.


    Lastly, management has put significant resources into preparing for a launch. They started 2023 with ten qualified treatment centers or QTC and are growing to 40-50 by the end of the year.


    Given the precarious cash position, investing in growth signals that management is confident in approval.


    Financials Tight, But Manageable

    The Q3 earnings call, especially Q&A was heavily focused on revenue and expense expectations following FDA approval in addition to cash flow.


    Looking first at cash flow, unrestricted cash gets bluebird to Q2 FY24. Restricted cash of $50 million and an FDA certificate sale of $100 million buy bluebird until mid Q4 FY24, again at current burn rate. With that in mind, bluebird needs to be on a path to profitability by Q3 FY24 at the latest, or at least be on the right path to secure additional financing.


    Assuming FDA approval, I wanted to see if this was possible. Here are some key assumptions I lifted from the Q3 earnings discussion:


    Existing therapies will grow linearly, with cost of revenue improving to a 30-40% margin

    Lovo-cel will grow linearly starting from Q2 FY24 except at a 10x size based on eligible patients and expanded treatment centers

    S,G,&A investments are largely in place for the expansion (I added 5% q-o-q growth as a buffer)

    R&D will drop materially following FDA approval (I cut R&D in half for 2024 although management signaled it could be more

    Here is a rough forecast based on year-to-date 2023 performance and management's implied guidance:


    A lot still has to go right, but based on current trends, bluebird could become profitable before running out of cash or at least set up for additional financing as the business grows.

    vllt sehe ich falsch, aber ich würde sagen, dass der Kurs nach mehreren Versuchen einfach nicht unter CHF 1.5 gepusht werden konnte...

    Eine "Bodenbildung" auf diesem sehr tiefen Niveau war eigentlich schon seit ein paar Tagen zu sehen...

    kann sein, dass eine evtl. EU-Zulassung teilweise schon eingepreist ist, bzw. einige Teilnehmer nur auf das richtige Moment warten SANN-Aktien loszuwerden? z.B: HB, JPM, Catalyst, oder evtl. auch Idorsia, sie brauchen auch dringend Geld...

    ich bin kein Medi Profi, aber die Daten (oder PR) von Newron von 9.10.23 scheinen deutlich stärker zu sein; dort kann man keine Bedenken erkennen.


    09.10.2023 - Newron Pharmaceuticals SpA

    EQS-Adhoc: Newron TRS-Studie, 6-Monatsergebnisse: Evenamide verbessert Patienten in einem Ausmaß, dass sie nicht länger die Auswahlkriterien der Studie erfüllen


    40% der Patienten erfüllen nicht länger die TRS-Diagnose-Kriterien


    Auf allen Wirksamkeitsskalen zeigte sich im selben Zeitraum eine kontinuierliche und anhaltende Verbesserung; der Nutzen der Behandlung nahm im Laufe der Zeit weiter zu und viele Patienten, die zu Beginn nicht ansprechen, erzielen später einen klinisch bedeutsamen Nutzen.


    Noch der Artikel zu Kia`s Post:


    FDA kicks off review of Karuna’s schizophrenia drug KarXT | pharmaphorum


    Das wichtigste davon:

    A lack of data after five weeks is a concern, according to the review, which says the drug appears to be “promising” but with an inconclusive net health benefit compared to older drugs. Karuna is currently conducting two phase 3 studies looking at the long-term safety and efficacy of the drug.

    GlobalData has predicted that KarXT will launch in the US next year, and achieve sales of $1.1 billion by 2031 thanks to its safety profile and ability to broadly target symptoms of schizophrenia, i.e. both positive (delusions and hallucination) symptoms and negative (social withdrawal and apathy).

    Looking at its rivals, ulotaront – an agonist of 5-HT receptor 1A and trace amine-associated 1 (TAAR-1) receptors – stumbled in phase 3, failing to meet the primary endpoints in the DIAMOND 1 and 2 trials reported earlier this year.

    That makes KarXT’s closest rival evenamide, which modulates glutamate and blocks voltage-gated sodium channels and is currently in late-stage testing for patients with treatment-resistant schizophrenia as an add-on to current therapies, and as a monotherapy for those who don’t respond to current drugs.

    interessant ist das in Orderbuch (welche übrigens völlig Computergesteuert ist) der erste Verkaufsauftrag plötzlich über CHF 4.8 liegt. Sicher auch rein Zufall... :) (Oder wir sind nicht allein, die die heutige News irgendwie spannend finden...)

    wie sollte sie denn reagieren deiner meinung nach? :)

    ich selber würde Newron Aktien sicher nicht verkaufen wenn der Konkurrent solche Bedenken äussert und sowieso bald neue Daten zu Evenamide zu erwarten sind... Jeder muss aber natürlich selber wissen...

    übrigens; wenn Lennych Recht hat, dann sollten die Daten von Newron diese Woche kommen, oder?

    Seit Wochen dasselbe Muster:


    Sehr starker Verkaufsdruck bis ca. 11.00 Uhr, und danach eine genau so starke Erholung bis Mittagszeit; dann am Nachmittag eine langsame Konsolidierung.


    Heute ging es aber bis 10.50 Uhr nur bis CHF 1.60 runter, und nicht tiefer, ausserdem war heute das Handelsvolumen bei dem Druck auch sehr gering (Umsatz CHF 178`276 im Vergleich zu gestern über 1 Mio in derselben Zeit...


    Es sind leider (noch) keine fundamentale Gründe, aber trotzdem - m.M.n würde diese Entwicklung auf eine Bodenbildung hindeuten...


    Die Frage ist ob Clozel den Markt mit einer neuen Partnerschaft und daraus resultierender Finanzierung überraschen kann oder die KE bis in Q1/2024 "verzögern" könnte.


    Nach der letzten Mitteilung würde Idorsia das Cash bis Ende März 2024 ausreichen.


    US PDUFA für Aprocitentan ist für 19. März 2024 geplant.

    In Europa wurde MAA Ende Januar 2023 eingereicht. Mit etwas Glück könnte dort eine Zulassung kommen, und damit würde natürlich eine KE ganz anders aussehen. Das erwartete Potenzial ist für das Medi gross; es könnte Idorsia noch retten und die Umsätze und Cashflow deutlich verbessern.


    "A new drug application (NDA) for aprocitentan was accepted for review by the US FDA. Following the provision of additional Risk Evaluation and Mitigation Strategy (REMS) materials to support a streamlined REMS designed specifically for aprocitentan, the company is working towards a PDUFA date of March 19, 2024. A market authorisation application (MAA) was submitted to the EMA at the end January 2023."


    New Phase 3 data with aprocitentan for patients with resistant hypertension has been presented at the American Society of Nephrology Kidney Week 2023 (idorsia.com)