BMYMP - Autolus: Investment Case Further Entrenched With Latest Interim FELIX Data

Summary

• Primary endpoint met with interim analysis released from phase 1b/2 FELIX study using obe-cel for the treatment of patients with relapsed/refractory Acute Lymphoblastic Leukemia; 70% Overall Remission Rate achieved.
• Recently released positive interim analysis results with obe-cel for relapsed/refractory Acute Lymphoblastic Leukemia to be presented at medical conference mid-2023; Final results to be released by end of 2023.
• Biologics Licensing Application to the FDA of obe-cel for the treatment of patients with relapsed/refractory Acute Lymphoblastic Leukemia to be filed before end of 2023.
• Autolus has been able to establish three partnerships based on its two CAR technologies it has, which are with Moderna, Bristol Myers Squibb and Cabaletta Bio.

Autolus Therapeutics ( AUTL ) is a great speculative biotech play to look into. The reason why I state that is because it has gone on and accomplished what I talked about in the prior article I wrote , which is titled " Autolus Therapeutics: Near-Term Catalyst Plus Several Bio Deals Could shift Momentum" . The reason why I now I believe that this investment case is now further entrenched all has to do with it achieving positive interim results from the ongoing phase 1b/2 FELIX study, which used obe-cel for the treatment of patients with relapsed/refractory Acute Lymphoblastic Leukemia [ALL]. These were analysis of pre-planned interim results that were to be released for this pivotal trial. It was noted that the phase 1b/2 FELIX study had met the primary endpoint of Overall Remission Rate (ORR) of 70% in an interim analysis of 50 patients with relapsed/refractory ALL. This interim analysis is very important and now indicates that the chance for Autolus to submit a Biologics Licensing Application (BLA) to the FDA by the end of 2023 is much higher now. Not only that, but it creates a few other catalyst opportunities for investors to look forward to as well. That is, it expects to report additional results from this phase 1b/2 FELIX study in adult r/r ALL patients by end of 2023. Before then, these most recently released positive interim analysis results are to be presented at a medical conference in mid-2023. The final reason why I believe this biotech is a good one to look at is because it has already raised the cash necessary to get it past these catalysts in 2023. With proof of concept in r/r adult ALL patients using obe-cel, plus the ability to move it towards treating other B-cell malignancies, these are the reasons why I believe it is a good speculative biotech play to look into. Speaking of partnerships, Autolus has a safety switch for its CAR technology which appears to be in high demand, along with another type of Chimeric Antigen Receptor [CAR] technology which is Dual Targeting CARs. It formed a partnership with Cabaletta Bio ( CABA ) just last week so the biotech could use its tech. Prior to that, it was able to establish a partnership with a big pharmaceutical company like Bristol Myers Squibb ( BMY ) and an option was taken from Moderna ( MRNA ) in a prior deal made a few years back.

Obe-Cel Proven In Pre-Planned Interim Analysis In Relapsed/Refractory Adults With Acute Lymphoblastic Leukemia

As I highlighted in the beginning above, Autolus accomplished the catalyst I had noted in the prior article, which was the expected released of results from the FELIX study. Not only did it release interim results from the pivotal phase 1b/2 FELIX study , but it noted that it had met the primary endpoint. This late-stage pivotal study recruited 50 adults with relapsed/refractory Acute Lymphoblastic Leukemia ((ALL)). It is ultimately expected to enroll a total of 100 adults with r/r ALL. This is a highly underserved patient population, as such, the trial was designed to be a single-arm one. That is, all these patients had only received treatment with obe-cel. The primary endpoint of this clinical trial was ORR up to 24 months. The ORR was set as those patients who had either achieved a complete response [CR] or complete response with incomplete blood count recovery [CRI] which was assessed by an independent response review committee (IRRC). The primary endpoint was met, because the interim analysis showed that obe-cel was able to achieve an ORR of 70% in the 50 r/r Adult ALL patients who were evaluated. On the safety side of things obe-cel was tolerable and had a manageable safety profile. About 3% of patients had ? Grade 3 Cytokine Release Syndrome [CRS] and 8% ?Grade 3 Immune effector cell-associated neurotoxicity syndrome (ICANS). The positive interim analysis brings about multiple catalysts for investors to look forward to during this year. For instance, in mid-2023 it intends to present these results at a medical conference. A second catalyst would be the release of full results from the phase 1b/2 FELIX study by the end of 2023. The final and likely more important catalyst would be Autolus Therapeutics filing its Biologics Licensing Application (BLA) to the FDA for the treatment of patients with ALL, which is expected before the end of 2023.

Safety Switch Technology In High Demand

The long-term potential with Autolus Therapeutics doesn't only lie off of it advancing its own clinical products. It has been able to generate several partnership deals over the last several months with respectable pharmaceutical companies. The partnerships developed are with the likes of Bristol-Myers Squibb, Moderna and Cabaletta Bio as I noted above. The most recent partnership includes a deal that Autolus generated with Cabaletta Bio . With respect to the Cabaletta Bio partnership deal, it wants to use Autolus' proprietary RQR8 safety switch for use in autoimmune diseases. This is going to be the initial development indication to be advanced using this switch, however, Cabaletta Bio has the option to use the "Safety Switch" technology in up to 4 additional therapy target indications should it choose to do so. This deal wasn't the first to be made for the RQR8 safety switch for use in advanced cell therapies. One deal was established with Bristol Myers Squibb to access the RQR8 safety switch for its cell therapy programs. Then, another license option with Moderna was taken, so that it could obtain Autolus' binder technology . What Autolus has done with respect to its technology is that it has incorporated two different types of Chimeric Antigen Receptors (CARs). The first is known as "Fast Off Rate" CARs, which in essence means how quickly binders are released off of a cell. This is the tech that Moderna wanted to get for its undisclosed immuno-oncology target. The second type of technology is known as "Dual Targeting CARs", which as the name suggests, targets two proteins at the same time.

Financials

According to the 6-K SEC Filing , Autolus Therapeutics had $163.1 million as of September 30, 2022. However, since the last time I noted this cash on hand, it has been able to achieve/complete several transactions to raise funds for its pipeline. I wrote last time about the Blackstone Life Sciences transaction whereby $250 million in equity and product financing was announced . However, more recently Autolus received $75 million based on this deal. Why? Because it accomplished two key items in the contract. The first item achieved was the release of the positive pre-planned interim analysis results from the pivotal phase 1b/2 FELIX study, using obe-cel for the treatment of adults with r/r ALL, as I went over above. This event itself triggered one of the $35 million milestone payments. Not only that, but it received another $35 million milestone payment for completing planned activities of the manufacturing process. In total, it received $70 million from Blackstone for achieving both of these milestones. With a few milestones left, there is some additional cash to be earned as obe-cel is advanced through the clinical pipeline. It had also gone on to file a $150 million ADS offering back on December 9, 2022 as well. The underwriters only partially exercised their option once all was said and done, but the key is that with all the shares sold under the offering, Autolus raised approximately $163.9 million in cash.

Risks To Business

There are several risks that traders/investors should be aware of before investing in this biotech. The first risk to consider is with respect to the ongoing pivotal phase 1b/2 FELIX study, which is using obe-cel for the treatment of Adults with relapsed/refractory Acute Lymphoblastic Leukemia [ALL]. There are two reasons why this program remains a big risk. The first reason is because only pre-planned interim analysis results were released. Having said that, final results from this pivotal clinical trial are not expected to be released until the end of 2023. There is no guarantee that the final results to be released from this study will turn out to be the similar or successful. Especially, since additional patients are to be recruited into this study. The second reason is because even if a Biologics Licensing Application (BLA) to the FDA is going to be filed by the end of 2023 for obe-cel, there is no guarantee that it will be accepted by it. Lastly, all regulatory applications must be evaluated for marketing approval, which means there is no assurance that this treatment will ultimately be approved for the treatment of this patient population.

The second risk would relate to all of the ongoing collaboration agreements that Autolus had made with several of the pharmaceutical companies I highlighted above. While these programs are currently being explored, any one of these partners could choose to end their respective deals for any reason. it could be either due to lack of safety/efficacy of the program itself or if there is a shift in its intended direction. This means, that Autolus could lose out on several milestone payments along with possible future royalties on net sales of marketed products. The final risk to consider would be with respect to the financials. While it was able to just complete a cash raise of $163.9 million, the biotech has relied on the need to raise cash to fund its pipeline and pay for its manufacturing plant. I expect another cash raise to happen again at least by the end of 2023. More than likely, I believe that another cash raise would be done after the release of final results from the ongoing pivotal phase 1b/2 FELIX trial by end of 2023.

Conclusion

The final conclusion is that Autolus Therapeutics is a good speculative biotech play to look into. The reason why I believe that is because of what I highlighted in the beginning above, which is that it was able to establish proof of concept in a pre-planned interim analysis from the phase 1b/2 FELIX study. It showed that an Overall Remission Rate (ORR) of 70% was achieved when obe-cel was given to adults with relapsed/refractory Acute Lymphoblastic Leukemia (r/r ALL). The good news is that this biotech is developing obe-cel to be used against other B-cell malignancies such as B-cell Non-Hodgkin's Lymphoma (B-cell NHL), chronic lymphocytic leukemia (CLL), and Primary CNS lymphoma. Even better than that, is it has the two types of technologies which I noted above. One tech is the Dual Targeting CARs and the other is the Fast Rate Off CARs. Not only has it established proof of concept with use of its technologies, but it has been able to form partnerships based on them. These include partnerships with the likes of Moderna, Bristol-Myers Squibb and Cabaletta Bio. With additional results to be released from the pivotal phase 1b/2 FELIX study by the end of 2023, along with the BLA filing to the FDA expected during the same time period, these are the reasons why I believe that Autolus Therapeutics is a great speculative biotech play to look into.

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