Bussarin Rinchumrus/iStock by way of Getty Photographs
Annexon, Inc. (NASDAQ:ANNX) has made nice progress in having the ability to advance its pipeline, that is as a result of it has been in a position to receive alignment with the FDA to advance its complement inhibitor ANX007 ahead for the remedy of sufferers with Geographic Atrophy [GA]. It was in a position to garner the power for the company to permit for one more endpoint for the remedy of this affected person inhabitants for the design of its soon-to-be initiated ARCHER examine. That is excellent news for the biotech, as a result of it is not going to have to discover one other endpoint of lesion progress that’s sometimes used for these GA sufferers to garner regulatory approval.
Such a section 3 examine is predicted to start out in late 2024. The latest scientific replace of ANX007 achieved an excellent higher end result for the European territory. Why is that? That is as a result of the European Medicines Company [EMA] goes to permit for a worldwide sham-controlled trial, often called Archer II. The importance of this examine is that Annexon would possibly be capable to have a faster pathway in the direction of regulatory approval of ANX007 for the remedy of GA sufferers within the European territory. Such a section 3 sham-controlled examine is predicted to start out in mid-2024.
ANX007 For The Therapy Of Sufferers With Geographic Atrophy
As I said above, Annexon has been in a position to receive a optimistic scientific growth replace with respect to its program advancing the usage of ANX007 for the remedy of sufferers with Geographic Atrophy [GA]. That is as a result of it got here in alignment with two agencies so as to presumably finally transfer this program ahead in the direction of regulatory approval. The primary development particularly handled talks that this biotech had with the FDA. Such talks went very effectively, as a result of it was in a position to agree with the FDA {that a} section 3 head-to-head examine, shall be sufficient to file for regulatory approval of ANX007 for the remedy of sufferers with GA. That is going to be via a late-stage examine, often called ARROW, which goes to check ANX007 to a different authorized drug for these sufferers, often called SYFOVRE. For my part, I contemplate this can be a enormous win for this firm. Why do I imagine that? That is as a result of such late-stage approval research for GA usually need to see slowing of lesion progress. The win for the corporate is that will probably be in a position to as a substitute use one other major endpoint for this examine often called prevention of ≥15-letter lack of BCVA assessed via 12 months.
A second motive why this can be a good end result is as a result of a previous section 2 examine, often called ARCHER, achieved a statistically important preservation of BCVA in comparison with baseline when these sufferers obtained month-to-month ANX007 injections. Such a distinction in comparison with baseline was achieved with a statistically important p-value of p = 0.006. One other means of it’s that the drug was able to achieve a 72% reduction in the risk of 15-letter loss compared to baseline. Thus, if the section 3 ARROW examine achieves this major endpoint, then Annexon shall be in fine condition to file for FDA approval of this drug for the remedy of sufferers with GA.
A second optimistic scientific replace was achieved with respect to a faster approval pathway of ANX0067 for the European territory. The explanation why is as a result of it could be doable for Annexon to submit a regulatory utility on the idea of one other sort of section 3 examine, often called ARCHER II. This late-stage trial goes to enroll about 400 Geographic Atrophy [GA] sufferers who’re to be randomized 1:1 to obtain both a month-to-month dose of ANX007 or sham process. This examine goes to make use of the same endpoint as the opposite examine, which is the prevention of a ≥15-letter lack of finest corrected visible acuity [BCVA]. Discussions proceed with the European Medicines Company [EMA] to provoke a pivotal section 3 examine for this program within the 1st half of 2024. Not solely that, however ANX007 was given PRIME designation by this regulatory physique. This was the primary drug for GA to obtain this sort of designation as effectively.
Financials
In accordance with the 10-Q SEC Filing, Annexon had money and money equivalents and short-term investments of $192.9 million as of June thirtieth 2023. It believed that it will have sufficient money to fund its operations into 2025. Nevertheless, regardless of this money runway, it enacted the pricing of a $125 million underwritten public providing of widespread inventory. That is whereby it will promote 25,035,000 shares of its widespread inventory and pre-funded warrants to buy 18,379,861 shares of its widespread inventory. The share of widespread inventory themselves had been offered at a value of $2.88 per share, and the pre-funded warrants are being offered at a value of $2.879 per share. It ought to be effectively capitalized for fairly a while now, particularly after this most up-to-date money increase.
Nevertheless, simply in case it is not, it does have an ATM program it may faucet into if mandatory. This may be with respect to a 2021 ATM program that was established again in August of 2021 with Cowen and Firm LLC, or Cowen. Such an ATM settlement was made whereby there may very well be an mixture most providing value of $100 million price of inventory to be offered as wanted. As of September thirtieth of 2023, it’s stated that roughly $82 million stays beneath this ATM Gross sales Settlement with Cowen. Thus, it’s my perception that this stays one other viable choice sooner or later, ought to Annexon want to boost extra money.
Dangers To Enterprise
There are a number of dangers that traders ought to pay attention to earlier than investing in Annexon. The primary threat to think about can be with respect to the development of ANX-007 for the remedy of sufferers with Geographic Atrophy [GA] within the ongoing section 3 ARCHER II examine. That is as a result of not solely is there no assure that the first endpoint shall be met with statistical significance, however this once more goes to be necessary for presumably permitting this biotech to have a faster pathway in the direction of European approval. The second threat to think about can be with respect to the opposite section 3 ARROW examine, which goes to check ANX-007 to SYFOVRE for the remedy of sufferers with GA. I imagine that there are two factors to think about right here with respect to this threat. The primary in fact is whether or not or not the first endpoint is in the end achieved with statistical significance.
The second level would take care of a doable aggressive benefit. That is as a result of SYFOVRE was the first FDA-approved drug for the remedy of sufferers with GA. The factor is that this complement inhibitor drug was authorized based mostly on two late-stage research that confirmed SYROVRE was in a position to scale back the speed of GA lesion progress in comparison with sham in a statistically important method. What Annexon is hoping to show in its section 3 ARROW examine, is that its drug ANX-007 performs higher when it comes to preserving visible perform based mostly on BCVA measurement.
It hopes to indicate that long-term remedy with its drug does higher in preserving GA affected person’s imaginative and prescient, in comparison with SYFOVRE. A 3rd threat to think about can be with respect to ANX-005, which is being developed for the remedy of sufferers with Guillain-Barre Syndrome [GBS]. A pivotal section 3 examine utilizing this drug to deal with this affected person inhabitants is predicted to have an information readout within the 1st half of 2024. There is no such thing as a assure that the first endpoint shall be met for this late-stage trial, nor that the corporate will in the end be capable to file for regulatory approval of ANX-005 for these GBS sufferers.
Conclusion
Annexon has been in a position to make important progress in advancing its scientific pipeline. That is as a result of it has been in a position to obtain optimistic suggestions from each regulatory companies in regards to the trial designs essential to in the end obtain approval of ANX-007 for the remedy of sufferers with Geographic Atrophy [GA]. This may be with the development of two section 3 research, often called ARCHER II and ARROW. It’s fairly doable that the ARCHER II examine may present for a faster pathway in the direction of regulatory approval within the European territory, although. The flexibility for Annexon to realize the first endpoint of finest corrected visible acuity ≥ 15-Letter Loss, wouldn’t solely simply imply the power to have the ability to file regulatory approvals for its C1q inhibitor ANX-007.
It may additionally finally imply a aggressive benefit of this drug having the ability to obtain superior preservation of imaginative and prescient loss over SYFOVRE. Within the meantime, 2024 will not be with out another catalysts, although. It’s anticipated that within the coming yr that the biotech will launch outcomes from the section 3 examine utilizing ANX-005 for the remedy of sufferers with Guillain-Barre Syndrome. With section 3 program development of ANX-007 for the remedy of sufferers with GA, plus an information readout from one other program anticipated within the coming yr, I imagine that traders would possibly be capable to capitalize on any potential beneficial properties made.