Die Vamorolone Wette

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01.05.21 17:52

838 Postings, 2021 Tage EvidencebasedDie Vamorolone Wette

m.M.n. die interessanteste "Wette" in den nächsten Wochen. die Zukunft von Santhera hängt an den baldigen Phase 2B Daten von Vamorolone. Bei 2,50 CHF eine gute Wette...oder wie immer Totalverlust.  
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244 Postings ausgeblendet.

17.10.22 10:01

838 Postings, 2021 Tage EvidencebasedIrgendwie erinnert mich

Deflazacort immer an Martin Shkreli und Marathon ? wollte ich schreiben  

17.10.22 16:02

838 Postings, 2021 Tage EvidencebasedMal sehen

wie  lange hier noch nach unten gedrückt werden kann?  

27.10.22 07:58

63 Postings, 399 Tage TruelinerKurs

Keine unmittelbare Auswirkung auf Aktienurs.  

31.10.22 15:05

11 Postings, 549 Tage mampfraupeWarum steigt der Kurs nicht?

Angenommen Vamolorone wird zugelassen in den USA?
Würde der Kurs steigen? Ja oder Nein.
Danke  

01.11.22 16:11

838 Postings, 2021 Tage EvidencebasedKurs

Vamorolone hat allein in DMD Peak Sales Potential von ca. 500 mio USD , Becker Muskel Dystrophie und weitere Indikationen mal ausgelassen. Zusätzlich sollten noch ein paar Milestone Zahlungen von Sperogenix zu erwarten sein und anteilig auch ein paar Millionen vom RPD Voucher (bei Zulassung Vamorolone.)
Auslizensierungen (außerhalb EU/USA ?) wären ebenfalls denkbar (s.a. Halbjahresbericht gestern )
Santhera ist leider immer noch in der Zange von den Investoren und deshalb braucht man Geduld und etwas Mut (natürlich mit Risiko verbunden)  und  wenn man mal jetzt schon mal in die Zukunft schaut und über den Daumen mit 100 mio ausstehenden Aktien rechnet , müsste der Kurs irgendwo bei 5,- chf landen. Denke das spätestens wenn priority Review durchgeht der knoten platzt  

01.11.22 16:38

838 Postings, 2021 Tage EvidencebasedNachtrag

Die 5,- CHF wären für mich ein Bereich kurz vor FDA Zulassung  Mitte 2023 ( priority review vorausgesetzt) Nach Zulassung, oder bei Übernahme kann ich mir durchaus auch höhere Kurse vorstellen.
Bis Ende des Jahres hoffe ich auf die ein oder andere Good News und damit auch wieder auf Kurse deutlich über 1,- CHF.  Mal sehen ?  

03.11.22 12:06

63 Postings, 399 Tage TruelinerForget it

Vergiss es einfach, dass hier etwas abgeht.
Die Bude ist zum Untergang gedeiht, da niemand eine Finanzierung für 2023 eingeht.  

03.11.22 12:13

63 Postings, 399 Tage TruelinerKurs

Werden vermutlich schon bald neue Tiefst sehen.  

03.11.22 14:02
1

1635 Postings, 2666 Tage Glatzenkogelwas

jetzt aktuell hier fehlt ist die News, dass die FDA den Zulassungsantrag annimmt, dieser als vollständig gewertet wird und der Status priority review vergeben wird.  Diese News ist innerhalb von 60 Tagen nach Einreichung, also bis Ende des Jahres, zu erwarten
Ich denke, man hat bereits Gespräche mit Lizenzpartnern Ex US/EU geführt. Tinte unter den Vertrag kommt vermutlich erst, wenn NDA angenommen wird. Dann ist auch ein BO denkbar.
Wenn es so kommt, dürfte Trueliner seinen letzten Post hier abgesetzt haben. Aktuell muss er natürlich weiterhin miese Stimmung machen, damit er sich bis Jahresende noch günstig eindecken kann. Seine Short Position scheint recht groß zu sein.
Wird der Zulassungsantrag nicht angenommen oder es gibt weitere Verzögerungen, dann wird´s dunkel in der Bude. Es steht jetzt Spitz auf Knopf. Hoffen wir also, dass diesmal keine formalen Katastrophen (wie beim CMO) passieren.
An Vamolorone glauben wir ja alle. Aber ein guter Wirkstoff allein reicht halt nicht um kommerziell erfolgreich zu sein.
   

04.11.22 08:17

63 Postings, 399 Tage TruelinerGeld nötig

Und schon wieder eine Kapitalerhöhung.
Kurz gesagt, einfach unfähige Bude.
Als nächstes kommt dann der Antrag nicht durch.  

04.11.22 20:17
1

132 Postings, 2365 Tage TraderderHerzenNachkauf

Heute noch ein paar Stk ins Körbchen gelegt. Sehe hier in relativ kurzer Zeit viele Katalysatoren (Finanzierung, Fast Track, Zulassung) auf und zu kommen.

Schluss ist erst bei 0 - schon klar. Aber der Kurs ist mittlerweile so ausgebombt ich sehe hier mittlerweile ein attraktives Chancen-Risiko-Verhältnis.  

05.11.22 12:12
1

838 Postings, 2021 Tage EvidencebasedSehe ich auch so

Extrem viele Katalysatoren bei außerordentlich niedriger Bewertung.  Mein Wunschszenario wäre eine Kombination aus Auslizensierung und einem private placement von einer starken Hand und vorallem nicht zu diesen unterirdischen Kursen . Wahrscheinlich wäre dieses Szenario erst  nach priority review denkbar (oder gleich ein Verkauf nach Zulassung ) Es ist einfach ein schlechter Witz als Investor die Gehälter in dieser Branche (bei so mieser Performance) ertragen zu müssen.  Falls Santhera anfangen sollte es komplett selbst zu versuchen (wovon ich jetzt mal nicht ausgehe) werden wir in 10 Jahren noch bei 0,7 chf rumdümpeln.  Die Hoffnung stirbt bekanntlich zuletzt und das gute bei Santhera ist, dass Vamorolone sehr viel Potential hat, also die Vermarktung wird nach Zulassung eher kein Problem werden  

05.11.22 20:17

63 Postings, 399 Tage TruelinerNoch mehr

Und am 29.11. möchte das Management noch ein bisschen mehr ( 2.5 Millionen) von den Aktionären.
Einfach zum davonlaufen.
Überall sonst muss man zuerst etwas leisten...  

06.11.22 11:07
1

132 Postings, 2365 Tage TraderderHerzen@Evidencebased

Sehe ich ähnlich, aber zwischen den Zeilen lese ich immer wieder was von "Weiterentwicklung der bestehenden Pipeline" und "Aufbau von Vertriebsstrukturen in USA etc.".

Also für mich klingt das nicht nach Auslizensierung oder gar Verkauf/Übernahme. Das Management hat natürlich auch keine Interesse an einem Buy-Out. Käme ein großer Pharma-Player, würde er das Board durch eigene Leute ersetzen.

Analysiert man also das Wording so muss ich In der Hinsicht Trueliner schon recht geben... Die Aktionäre werden weiter gemolken bis es nicht mehr geht und man zu anderen Maßnahmen greifen muss.

Ich werde hier maximal bis zur Zulassung dabei bleiben. Santhera hat kein Geschäftsmodell und den Turnaround zur profitablen Biotech Perle kann ich im Moment wirklich nicht erkennen.  

07.11.22 10:51
1

838 Postings, 2021 Tage EvidencebasedAuslizenzierung und Pipeline

@traderderher. Sehe ich etwas anders:
- Lonodelestat erstmal auf Eis gelegt
- idebenone LHON an chiesi  (das einzige Fragezeichen ist, ob sich Santhera nochmal traut bzw das Rsiko eingeht , anhand der positiven LEROS  Daten , Idebenone bei der FDA einzureichen. Idebenone ist genauso wenig innovativ wie Deflazacort ? ich vermute die FDA wird alles dafür tun, dass Medikament zu zerlegen.
- Vamorolone auslizensiert Region greater China an Sperogenix
- Gentechnik deal mit Seal
- FDA gefördertere Studie Vamorolone in BMD
Wenn ich CEO wäre würde ich auch den Vertrieb für Vamorolone  in den USA aufbauen , v.a. wenn in ca. 6 Monaten die  Zulassung erwarte und ja ich würde mir auch die Möglichkeit einer potentiellen KE freizeichnen lassen, um flexibler agieren bzw verhandeln zu können. Das heißt jetzt nicht, dass ich mit der Leistung von Santheras Management zufrieden bin , aber ich glaube hier geht noch was  und im Endeffekt kann ja jeder selbst entscheiden ob er in Santhera investiert oder tradet , oder einfach nur beobachtet. Man lernt ja auch durchs zuschauen und am meisten aus Fehlern.  

10.11.22 08:52

63 Postings, 399 Tage TruelinerFDA

Die Bude wird mit diesem Management auch mit einer Zulassung bald dicht machen.
Die einzige Hoffnung wäre eine Übernahme oder Auswechslung des Managements.  

10.11.22 09:23

63 Postings, 399 Tage TruelinerGV

Höchst suspekt, dass nur bei Santhera keine physische GV abgehalten wird. Das sieht nach Machenschaften aus...  

12.11.22 16:25

132 Postings, 2365 Tage TraderderHerzenSparen

Als Aktionär begrüße ich es, wenn das knappe Kapital nicht für das leibliche Wohl von "GV Touristen" verfeuert wird.  

12.11.22 16:43

63 Postings, 399 Tage TruelinerGeschäftsleitung

Dann musst du unbedingt zu allem ja stimmen damit sich die teure Geschäftsleitung noch ein bisschen mehr den Bauch füllen können.
Eine solch miserable Performance für Aktionäre noch belohnen???  

21.11.22 13:44

63 Postings, 399 Tage TruelinerUui

Heute wird wieder mal abverkauft!!!  

03.12.22 11:15

1635 Postings, 2666 Tage GlatzenkogelTS

Santhera Pharmaceuticals Holding AG (OTC:SPHDF) Q2 2022 Earnings Conference Call November 7, 2022 8:30 AM ET

Company Participants

Dario Eklund - CEO

Andrew Smith - CFO

Shabir Hasham - Chief Medical Officer

Conference Call Participants

Bob Pooler - valuationLAB

Boobalan Pachaiyappan - H.C. Wainwright & Co.

Operator

Ladies and gentlemen, good morning or good afternoon. Welcome to the Santhera Pharmaceuticals. I am Myra, the Chorus Call operator. [Operator Instructions]. The conference must not be recorded for publication or broadcast. This conference call may contain certain forward-looking statements based on current assumptions and forecasts made by Santhera Pharmaceuticals. Such statements involve certain risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of Santhera Pharmaceuticals to be materially different from those expressed or implied by such statements. These factors include those discussed in the comprehensive risk factor disclosure on the company's website at www.santera.com. Santhera disclaims any obligation to update any forward-looking statements. The conference may be downloaded on Santhera's website during the 2 weeks following the call. At this time, it's my pleasure to hand over to Mr. Dario Eklund, CEO. Please go ahead, sir.

Dario Eklund

Thank you, Myra. Good afternoon and good morning, everyone, joining us from the U.S. Thanks for joining today's call and on this occasion of our half-year results publication. Here with me today, I have our CFO, Andrew Smith as well as Dr. Shabir Hasham, who's our Chief Medical Officer, to answer any questions you may have later on. We'll briefly review the performance in 2022 to date, give you an outlook across various milestones and into 2023 before taking your questions at the end. Let me start with the highlight of 2022 that everyone at Santhera is very thrilled about. At the end of September, we submitted a marketing authorization application to the European Medicines Agency for vamorolone in DMD.

One month later, the submission was validated which confirms that the dossier is complete and the review by the CHMP has started. Completion of the CHMP review is expected in Q3 of 2023, which could allow us first launch in Europe towards the end of next year. In October, we completed the rolling submission of an NDA to the U.S. FDA for vamorolone in DMD.

After 60 days or around year-end, we expect to reply from the FDA on whether priority review will be granted. Subject to priority review, vamorolone could be approved as early as mid-2023. Either way, vamorolone is set to become available to patients in the U.S. in the second half of next year. Successfully accomplishing these 2 major projects and laying the groundwork for potential approvals in the U.S. and Europe was a major achievement for Santhera. It required everybody's full dedication over many months for which I'm very thankful to all of our employees. In the course of this year and while preparing submissions, we have assembled and presented compelling new data indicating that vamorolone displays anti-inflammatory efficacy with a potentially superior safety profile compared to standard of care.

Amongst others, differentiating characteristics were observed in the areas of bone health, preservation of normal growth, and less frequent and less severe mood disturbances. The Bone-specific findings were presented at several congresses, including the international conference on Children's Bone Health and the World Muscle Society Congress, and published in the peer review journal, the neurology of â?? the Journal of Neurology of the American Medical Association. You find papers and posters on the Santhera website or we are very happy to send them to you if you're interested.

In 2022, we also started 2 new studies, one in Becker muscular dystrophy, a milder form of muscular dystrophy and one in a wider age range of DMD patients. These studies are a logical next step within the original area of neuromuscular disease. But of course, the potential benefits of vamorolone may also be highly relevant to other indications beyond DMD in which chronic use of steroids as standard practice and where vamorolone efficacy and safety profile can make a remarkable difference. vamorolone is the main strategic focus in the near term and will consume the lion's share of financial and human resources.

Our focus will be on advancing vamorolone through the regulatory process towards approval and in parallel on preparations for market entry. As a regretful consequence of this focus, we were forced to pause the development program for lonodelestat. Phase II studies in an acute pulmonary indication as well as in cystic fibrosis were almost ready to go but had to be postponed subject to funding. Owing to the limited resources, partnering remains our priority. We explore various opportunities via collaborations and/or partnerships with vamorolone, lonodelestat and also Raxone. Our first agreement was concluded in early '22 with Sperogenix with vamorolone for the Greater China region. Such deals give access to non-dilutive funding. At the same time, we opened possibilities to exploit the product's potential to a much broader extent than Santhera would be able to do alone. With equally high priority, we are pursuing additional near-term financing primarily to bridge the gap to approval of a moral and to fund market entry preparations.

And with this, let me hand over to Andrew for comments on the financial results and our financing activities. Andrew, please?

Andrew Smith

Thank you, Dario. I'll start with some comments on the financial performance following -- followed by a status update of our financing activities. Let me first turn to the half-year results. Total revenue amounted to CHF 63 million. This is up 40% year-on-year from $5.5 million in the first half of '21. Included in total revenue is revenue from outsourcing transactions in the total of $11.2 million compared to nil for the same period last year.

This largely reflects an initial income from Sperogenix who in licensed for morale for all rare disease indications in Greater China region. Also included in total revenue and recorded as negative sales. There's an additional accrual of $6 million in relation to ongoing uncertainties around pricing and reimbursement of Raxone in France. Also to remember, Santhera voluntarily supplies Raxone to patients for free already since August 21. We do this purely as of goodwill to patients who have no other therapeutic options and will continue to do so until conclusion of the negotiations with the French government.

Operating expenses amounted to CHF 30 million compared to GBP 21.9 million in the same period last year. Development expenses increased to $16.9 million compared to $13.6 million last year. This $3 million increase is primarily related to the preparation of regulatory submissions in the U.S. and Europe for . Marketing and sales increased to $5.9 million compared to $2 million in the same period last year. The main contributor to the $3.9 million increase is a provision of $2.1 million related to Raxone in France. In total, we have made an additional accrual of CHF 8.1 million in relation to Raxone in France in the first half of the year. Dario mentioned $6 million recorded under revenue and $2.1 million under marketing and sales expenses.

This brings total accrual included in non-current liabilities for this purpose to CHF 25 million. We expect to finally settle the dispute including final pricing for Raxone in early '23. And once this agreement is reached, we will resume sales and expect to be able to settle the liability from future sales of Raxone in France. G&A expenses rose by $1 million from $6.3 million to $7.2 million, and this was primarily a result of the additional â?? addition of key personnel in U.S. subsidiary in preparation of vamorolone launch in '23.

The net financial expense amounted to $3.6 million compared to $0.4 million in the same period last year. The change is largely a reflection of a recognized gain on exchange of the 2017 '22 bonds in the first half of '21, which have been partially offset by the costs associated with financing transactions. Bottom line, the company recorded a net loss of $29.7 million for the half year, which includes a provision of $8.1 million related to France, as discussed earlier.

This compares to a loss of GBP 20.5 million in the same period last year. Cash outflows for operating activities in the first 6 months of '22 decreased by 1/3 year-on-year and amounted to CHF 12 million. In the same time period, cash inflows from financing activities were $3.5 million compared with $14.3 million in the same period last year. In summary, this resulted in the 6-month decrease in cash and cash equivalents by $8.5 million or from $21.2 million at the start of the year to $12.7 million as at the end of June 22.

Let me move on to financing. Our efforts to secure sufficient funding focused on 3 dimensions: First, by reducing the high overall historic debt. This is -- this in combination with difficult market conditions hindered the ability to raise adequate funds. We accomplished this in early '22 when we fully repaid the 2017 '22 bonds and further reduced outstanding convertible bonds to CHF 31.5 million maturing in August 24. Second, by extending cash reach through a reduction of near-term financial commitments as well as internal project prioritization and cost control. We did this by renegotiating the timing of vamorolone approval milestone payment, which reduced near-term financial obligations of the company by CHF 20 million. In addition, we decided to prioritize pipeline projects, as Dario mentioned already. Third, by securing additional funds to finance the company through to approval of vamorolone or mid-23 at least. In June, the company upsized its existing financing arrangement with certain funds managed by hybrid capital management by up to an additional CHF 40 million. As of June 30th, 2022, CHF 20 million remained available subject to certain conditions on the consent of Hybridge.

Additionally, Santhera look to create approved capital in supporting and satisfying its funding needs. Where do we stand as of November? Given our cash runway into Q1 '23, it is clear that we will need to secure additional funds in the short term. We are pursuing all avenues and strategic options. This includes, but is not limited to non-dilutive funding in the form of out-licensing agreements and/or the monetization of assets, debt financing, royalty financing, a standby equity distribution agreement or depending on market conditions, also equity-based funding.

With regard to equity-based funding, we are asking shareholders for additional capital increase at an upcoming EGM to be held at the end of November. Whilst further dilution and a placement that low share values are, of course, not our preferred avenue, we hope that shareholders will approve the capital increase as it provides us with additional options and flexibility with regard to short-term financing.

Thank you. I'll now turn you back to Dario.

Dario Eklund

Thanks, Andrew. So with this, I'll close our summary remarks and hand over to the operator for the Q&A session, please.

Question-and-Answer Session

Operator

[Operator Instructions]. The first question is from Bob Pooler from valuationLAB.

Bob Pooler

Congratulations on the progress you made so far this year. A few questions on my side, first starting off with Raxone. What are actually the remaining issues of the French authorities regarding the reimbursement of Raxone in LHON?

Dario Eklund

This is Dario. Thanks for the question. With regards to Rexton in LHON in France, we're at the very late stages of negotiations. I was personally in Paris a few weeks ago to wrap this up. So now it's really down to formalities of the agreements. And I expect these to probably be wrapped up in the first weeks of the next year or so. So I think we will be able to get reimbursement for Raxone again in France in Q1 of next year.

Bob Pooler

Okay. Would that have an impact on your cash reach?

Dario Eklund

Andrew?

Andrew Smith

We don't think so of it currently because the discussions around the clawback really provide for and future revenue from Raxone more or less match the period over time. So we don't think this is a near-term impact on cash requirements.

Bob Pooler

Okay. Just then your plan on Raxone the U.S., good data that you have?

Dario Eklund

Yes. So following the readout of the post-marketing study, LEROS and PAROS, where we had very, very convincing data around both safety and efficacy. We now think we have enough on the file to approach the FDA. Originally, we were planning to do that towards the end of this year, but due to the resource requirements of our teams here to submit both EMA and FDA for almost at the same time, we just haven't had the bandwidth to approach them. So I think that's something we'll do early next year, see if we can get a pre-NDA meeting scheduled, and provided that meeting with the FDA suggests that the file is something that can be approved. We would then submit for U.S. approval, and we would have that 7 years of orphan exclusivity in the U.S.

Bob Pooler

Then switching to vamorolone. You've completed the NDA submission in DMD in October. When do you expect the FDA acceptance of filing and a potential PDUFA date? And then just on the acceptance of filing, how important is that for potential partner agreements outside rare diseases?

Dario Eklund

So we submitted end of October, as you know, and the acceptance of filing comes by day 60. I don't have a lot of experience of how the FDA operates around Christmas vacations, but technically speaking, it should be between Christmas and New Year's. It could come before or it could come right after, but around there by the book, it should be 60 days later. And that acceptance of filing comes together with a PDUFA date and the most really relevant information there is do we get the pediatric priority review designation or not from the FDA. If we do get the priority review, that will be a 6-month review from the date of acceptance. So that will be an approval or PDUFA date somewhere towards the end of June. And if it's a standard review, it will be 4 months later, that would be end of October.

Bob Pooler

But I think to now, all of the DMD drugs got priority review.

Dario Eklund

I think most -- all of them or at least most of them have received a priority review, and we also expect to get that.

Bob Pooler

Just coming back to the acceptance of filing on the importance of potential port agreements outside there to see such DMD.

Dario Eklund

I think the acceptance of filing is important because of the reasons that I just mentioned, the PDUFA date and the priority review designation. But I don't think it's on the critical path for partnering with -- in other indications, co-promotions or partnering. Having said that, though, I think you asked the question outside of rare diseases. And I think right now, we're still focused on rare diseases where the price point is similar to what we are expecting to get as a price point in Duchenne. It will be very difficult to launch something in a different area with a different price point with the same substance.

Bob Pooler

No, that makes sense. Just you've been to many more data specialist on long-term data. What is the feedback for KOLs? Basically, what do they like to the data and what do they not like?

Dario Eklund

I could answer the question, but I think Shabir who is on the line, who will have daily contact with our key opinion leaders and the medical community is probably a better equipped. Shabir, do you want to take this question?

Shabir Hasham

Yes Thanks, Dario. Thanks, Bob. We presented that a number of conferences this year amongst 4 general themes. We presented the durable efficacy of the vamorolone 6 milligrams per kilogram over the 4-week time point for the first time. We've also been able to demonstrate comparable efficacy to standard of care with a comparison to an external control asset. We've shown a reduction in terms of factor risk over a long-term treatment with 2.5 years of the compared to the external data set as well.

And we've also been able to show the preservation of growth as we had with our earlier data set but also the consistent and more benign safety profile we've seen in vamorolone . So really, the conversations with KOLs have focused very much around the in that we've not shown any growth standing over 2.5 years, whereas you get considerable growth on in the standard care, then the reduction in Spain or facture risk is very important. Of course, it's a major impact with longer-term steroid treatment. We also had conversations around the more benign safety profile.

What I've heard from KOLs but also investigators is that they see the children or the more appear differently other than being they just appear on the distribution in terms of that distribution is more rather than being tends to be more diffused and their appearance is more normal. They tend to be able to maintain higher doses of steroids than they would have expected with standard of care. And we also see that now with 153 boys of the 164 who participated in trials still in expanded access programs.

We're seeing a very good tolerability profile, very consistent with this more benign safety protocol that we've seen within the clinical trial. So overall, it's been very encouraging. Of course, for physicians, it's extremely important that we can maintain a higher effective dose range for longer. That seems to be a part within the data set. But really, parents are coming in less frequently in terms of concerns. Some physicians said that they expect frequent visits from parents and children on standard of care, whereas these are the more tend to be doing very well and coming in less frequently, which is exactly the target product profile we've been aiming for with the vamorolone .

Bob Pooler

That's basically also one of the triggers why you're going for an expanded a wider age group of patients or younger patients and patients in the older group. How much do you expand the market by adding a despite the age group?

Dario Eklund

So we're -- I'll take that, . So we're expecting a broad label in terms of age group. And the way we've looked at the market is that there's basically 3 segments on which one segment will be the early revenue and then the other 2 segments will come slower. That's how we built our forecast. So the first segment is the -- obviously, the naive â?? steroid naive patients, the new starts.

The second segment are the ones that are currently on steroids but are not happy with the way they're being treated. They have tolerability issues or safety concerns or they are significantly down-titrated due to those issues. And then the third segment are those that have stopped taking steroids altogether because of the tolerability issues and safety concerns. The one that I think is the obvious first to go at the [indiscernible] is the middlemen that I just mentioned. The ones who are currently on the existing steroids that are unhappy or unsuccessful with them.

Over time, as becomes more standard of care in this community, you will see more early starts or naive patients being put on drug that will also require some negotiation with payers to be allowed to start on vamorolone. And I believe also the last segment that one could have stopped taking steroids in the past, but would still benefit from steroids, what are going to be starting to use vamorolone in the future. But if you look at these 3 segments, it's about 70% to 75% of the expected patients 5 years from launch would be in the segment of switchers, the middle segment that I mentioned. And only about 25% would be distributed between the naive patients or the ones that have discontinued steroids. So as data matures in these patient populations and vamorolone becomes more standard of care, we would then expand also our share of those segments into those other 2 segments.

Bob Pooler

Okay. How many reps do you plan to hire for the U.S. and Europe?

Dario Eklund

So this is a market that is quite concentrated, as you probably know, both in Europe and the U.S. And the number of reps is typically in the rare orphan disease space, determined by 3 factors in the market. The first factor is our patients diagnosed at all. In rare and orphan diseases, you often find situations where you have to do a lot of medical education around the disease and people to then identify the disease and diagnose the disease. But in Duchenne, that's taken care of all the patients get diagnosed.

The second element you need to look at is whether these patients are being treated in different specialties, are they being referred back and forth between specialties? Where do these patients -- where do you find these patients? And where do you find the HCPs and to treat these patients? And in Duchenne, particularly in the U.S., it's quite easy. There's about 90 centers of excellence in the U.S. that are treating the vast maturity of patients. About 90% of patients are treated -- and 28 -- sorry, I think it's over 30 now, patients then are excellence of those are roughly patient advocacy group certified by PPMD. So the targeting, so to speak, is quite easy. And then the third element is, are you introducing something into the practice, that inconvenience is them? Are you introducing something that changes their treatment algorithms changing the way they do their daily work?

And in the case of vamorolone, we're not doing any of that. This is steroids are a well-established market where clinicians know how to titrate them up and down, have to treat patients with them, and also how to fight for medical exceptions, prior authorizations, et cetera, of our patients. So all we're doing is introducing an option into their armamentarium where they have the same efficacy that they see with existing steroids, but with a much more benign safety profile, particularly as it pertains to growth in bone and mood disorders. So it's a long way to answer your question, but I think it's a relatively small sales force. It's going to be more than 10 and less than 20. We're still refining that because it's also -- it goes hand in time with the planning around the MSLs, on the MSLs . It's a very medical education-intensive launch. So you may need more MSLs and less reps in the beginning and then time.

Bob Pooler

Okay. That's actually a relatively low number. So 20 probably to a very high-margin product, if price is in the range of the current treatments for BMD.

Dario Eklund

Correct.

Bob Pooler

And then just one final, if I may. You have the FDA funding Phase II trial for Becker muscular dystrophy. Any idea when your get those results? And also, what is the percentage market side or improvement BMD?

Dario Eklund

So in Beckers, to answer your first question, I think the current plan is to wrap up the study towards the end of '24, with a readout at the end of '24. So the study would close somewhere mid-24% with a readout in '24. And as it pertains to the market size, the number of Becker's patients is slightly less than the number of Duchenne patients but they don't all take steroids today. Many of them would benefit from it, but their course of the disease is not as severe as Duchenne. So the benefit-risk in many cases is not seeing the same way as it is in Duchenne. So the ones that are taking steroids today who have Beckers muscular dystrophy are there are real severe cases, which are much closer in their to Duchenne muscular dystrophy than the Becker's muscular dystrophy. We have internally in terms of market size, estimated the BMD market for us to be about 1/3 of the size of the Duchenne market.

Bob Pooler

Okay, that's quite sizable.

Operator

The next question is from Boobalan Data Japan from HC Wing.

Boobalan Pachaiyappan

Can you hear me okay?

Dario Eklund

Yes. We can, Boobalan.

Boobalan Pachaiyappan

Congrats on the progress. So just wanted to follow up on Becker muscular dystrophy, the Phase II study that's coming. So maybe to get started, can you discuss the baseline characteristics of the BMD patients who will enroll in your Phase II study? And maybe like you can talk about the typical NSAA score?

Dario Eklund

That's a question. I would pass on to Shabir, please.

Shabir Hasham

Yes. So the basic criteria are the Beckers patients were now to steroid 12 to 65 years of age, with an NSAA score less than 30. And I think there's a time to walk criteria as well of less than 30 seconds. So NSAA so is less than 32. [indiscernible] less than 30 seconds.

Boobalan Pachaiyappan

Thanks for the color and then with respect to the efficacy benchmark for . Did I said one in terms of NSAA go drop or maybe time to run or walk?

Andrew Smith

So bear in mind that this is a Phase II study. The benchmark we're looking at is an improvement of time to run a walk of about 2.5 is a second, which is consistent with what we saw in Duchenne, but it's also the NCIB that's generally accepted.

Boobalan Pachaiyappan

That's clear. And then so you already talked about the possible data release date. So I'm not going to focus on that. So with respect to combination with existing therapies, do you think mechanism of action allows for potential combination with existing therapies that are used both on and off-label in BMD patients?

Shabir Hasham

In terms of the mechanism of action. Yes. So the mechanism of action, we don't see anything in terms of safety profile that gives us concern. It's very consistent with what we see with steroids other than the bone health, preservation of height and maybe the multiline safety profile. So there's no reason we would expect any issues in terms of the combinations.

Dario Eklund

I think it's important for the listeners on the call to understand that steroids are today used in conjunction with exon skippers and gene therapies that are in clinical trials. So for instance, the gene therapy trials that are ongoing right now, they all have steroids as an inclusion criteria in the study. And as these new modalities come to market, they're not going to replace steroids. It's going to be an additive therapy and you would want to have the best steroid for your patients, particularly if you're going to be spending hundreds of thousands, if not millions of gene therapies, for instance.

It will be a real pity to dose a young child with a gene therapy and then need to have that child in a wheelchair early on because of bone fractures and other bond-related health issues. If you're going to be â?? the current gene therapies are not curative. They will just modify the disease from a Duchenne genotype to more of a Becker phenotype. It will extend their life, but it's still not a cure for these boys and adolescents. And so they will continue benefiting from taking steroids. And you wouldn't want to give a steroid that causes osteoporosis and spinal fractures and fund growth to a boy or a child is expected not to live much longer. So I think that's an important nuance to understand in the big picture.

Boobalan Pachaiyappan

Thanks for the color. And then I'm sure you probably saw this -- one of your competitors in the BMD indication, therapeutics. So they recently reported a 6-month interim results involving the asset of 2506. So what are your thoughts on this 6-month interim results of your company ?

Dario Eklund

I think it's just a little bit too early for us to comment on that. I would like to see the outcomes of this before we say anything.

Boobalan Pachaiyappan

Understood. And then maybe 1 or 2 in the segment for wrap-up. So are there any particular biomarkers that are interesting to you in the BMD indication maybe creatine or kinase levels or ? And are you planning to evaluate the number of severe adverse events as part of your endpoint analysis in your Phase II BMD study?

Shabir Hasham

So we are looking at a couple of endpoints. We're looking at CK. We're also looking at microRNA 16, and we will be looking at SAEs.

Operator

[Operator Instructions]. There are no more questions at this time.

Dario Eklund

Okay. So I'll close the call with a few comments here. I want to thank you all for joining us today and your interest in Santhera. We believe that, as you've heard now with vamorolone, we have an anti-inflammatory drug with a unique bone-sparing profile. It has the potential to change the lives of boys with DMD and down the line potentially in other pediatric indications. And our ambition is to make it available to patients as soon as possible. On the back of this, I trust that we will be able to secure sufficient funding to take the company to approval of vamorolone. First and foremost, I thank our employees for their dedication and persistence and our investors for their support. Please stay tuned for updates on our progress in this exciting journey. Thanks again for joining today and speak to you next time. Bye-bye.

Operator

Ladies and gentlemen, the conference is now over. Thank you for choosing Chorus Call, and thank you for participating in the conference. You may now disconnect your lines. Goodbye.  

03.12.22 12:26
1

1635 Postings, 2666 Tage Glatzenkogelbig picture gene therapy

interessante Aussage:

Eklund
I think it's important for the listeners on the call to understand that steroids are today used in conjunction with exon skippers and gene therapies that are in clinical trials. So for instance, the gene therapy trials that are ongoing right now, they all have steroids as an inclusion criteria in the study. And as these new modalities come to market, they're not going to replace steroids. It's going to be an additive therapy and you would want to have the best steroid for your patients, particularly if you're going to be spending hundreds of thousands, if not millions of gene therapies, for instance.

It will be a real pity to dose a young child with a gene therapy and then need to have that child in a wheelchair early on because of bone fractures and other bond-related health issues. If you're going to be â?? the current gene therapies are not curative. They will just modify the disease from a Duchenne genotype to more of a Becker phenotype. It will extend their life, but it's still not a cure for these boys and adolescents. And so they will continue benefiting from taking steroids. And you wouldn't want to give a steroid that causes osteoporosis and spinal fractures and fund growth to a boy or a child is expected not to live much longer. So I think that's an important nuance to understand in the big picture.
 

03.12.22 12:31
1

1635 Postings, 2666 Tage Glatzenkogelzwischen

Weihnachten und Neujahr erhält Santhera also Feedback von der FDA hinsichtlich
- Annahme NDA Antrag
- Priority Review Y/N (very likely Yes)
- PDUFA Date (voraussichtlich Ende Q2 23)

mE ist Santhera dann spätestens ab 01.01.23 ein heißer BO Kandidat (oder Pleite und ein Totalverlust, wenn NDA nicht angenommen wird, was nach der Annahme seitens der EMA jedoch eher unwahrscheinlich ist).  

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