Bellicum and MD Anderson Announce Additional License Agreement for Use of CaspaCIDe® Safety Switch

On September 1, 2021 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, and The University of Texas MD Anderson Cancer Center reported a global option and license agreement covering certain intellectual property and technology rights regarding Bellicum’s CaspaCIDe (inducible caspase-9, or iC9) safety switch and related technologies, and the use of rimiducid, an agent used to activate the safety switch (Press release, Bellicum Pharmaceuticals, SEP 1, 2021, View Source [SID1234587106]). Under this agreement, MD Anderson will have the option to incorporate CaspaCIDe into certain cellular therapy programs.

Bellicum’s CaspaCIDe safety switch may facilitate the use of cell therapies where cytokine release syndrome and neurotoxicities have been observed, in pursuit of novel targets with on-target/off-tumor safety concerns, and in conjunction with next-generation higher potency cell therapy constructs.

"We are excited to expand our CaspaCIDe agreement with MD Anderson to include a broader set of programs to benefit cancer patients," said Rick Fair, President and CEO of Bellicum Pharmaceuticals. "We believe that our switch technology may enhance the benefit/risk profile of cell therapies. We intend to continue to pursue opportunities to expand its use via external collaborations with other leaders in the field."

Upon exercise of each option – typically expected to be upon out-license of an MD Anderson program that incorporates iC9 – Bellicum will receive an upfront payment and will be entitled to a percentage of certain consideration paid to MD Anderson by the third party. Bellicum also will receive a single-digit-percent royalty on global sales of the product. Additional details of the financial arrangements are not disclosed. Bellicum and MD Anderson have agreed on the first two programs for development concurrent with the execution of the agreement. This agreement expands upon a previous one, which covers the use of CaspaCIDe in a specific MD Anderson cell therapy program.

"The unique inducible caspase-9 technology covered by this agreement has the potential to reduce the risk of serious adverse events associated with cellular therapies and to improve patient outcomes," said Katy Rezvani, M.D., Ph.D., professor of Stem Cell Transplantation and Cellular Therapy at MD Anderson. "We have successfully applied the technology to existing cell therapies, and we look forward to the potential future applications made possible by this agreement."

About CaspaCIDe

CaspaCIDe (inducible caspase-9, or iC9) is Bellicum’s chemical induction of dimerization (CID) safety switch technology designated to eliminate cells in the event of toxicity. The CaspaCIDe switch consists of the CID-binding domain coupled to the signaling domain of caspase-9, an enzyme that is part of the apoptotic pathway. Infusion of rimiducid is designed to trigger activation of this domain of caspase-9, which in turn leads to selective apoptosis of the CaspaCIDe-containing cells. In clinical studies, use of CaspaCIDe has resulted in clinical improvement in most patients as early as 24 hours after rimiducid administration. Further, because CaspaCIDe is designed to be permanently incorporated into Bellicum’s cellular therapies, the safety switch has the potential to be available for use when needed long after the initial therapy is delivered.

MAX BioPharma receives a Direct-to-Phase II SBIR award from the NIH

On September 1, 2021 MAX BioPharma reported that it has been awarded an SBIR Direct-to-Phase II grant from the NIH (NIAMS) titled: "Therapeutic Development of Osteogenic Oxysterol, Oxy133, for Spine Fusion" (R44AR079335-01) (Press release, MAX BioPharma, SEP 1, 2021, View Source [SID1234587151]). With limited options on the market, the medical community is excited about the prospect of a safe, efficacious and less-costly product .

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Biogen to Participate in the Morgan Stanley 19th Annual Global Healthcare Conference

On September 1, 2021 Biogen Inc. (Nasdaq: BIIB) reported that it will participate in a live Q&A at the Morgan Stanley 19th Annual Global Healthcare Conference (Press release, Biogen, SEP 1, 2021, View Source [SID1234587186]). The webcast will be live on Thursday, September 9, at 9:30 a.m. ET. To access the live webcast, please visit Biogen’s Investors section at investors.biogen.com. An archived version of the webcast will be available following the presentation.

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Exelixis to Webcast Fireside Chats as Part of Virtual Investor Conferences in September

On September 1, 2021 Exelixis, Inc. (Nasdaq: EXEL) reported that members of the company’s management team will participate in fireside chats at the following virtual investor conferences in September (Press release, Exelixis, SEP 1, 2021, View Source [SID1234587094]):

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Morgan Stanley 19th Annual Global Healthcare Conference: Exelixis is scheduled to present at 1:15pm EDT / 10:15am PDT on Thursday, September 9, 2021.
H.C. Wainwright 23rd Annual Global Investment Conference: Exelixis’ pre-recorded session will be available on-demand beginning 7:00am EDT / 4:00am PDT on Monday, September 13, 2021.
BofA Securities Global Healthcare Conference: Exelixis is scheduled to present at 5:35pm BST / 12:35pm EDT / 9:35am PDT on Wednesday, September 15, 2021.
Cantor 2021 Virtual Global Healthcare Conference: Exelixis is scheduled to present at 2:40pm EDT / 11:40am PDT on Wednesday, September 29, 2021.
To access the webcast links, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the presentations to ensure adequate time for any software download that may be required to listen to the webcasts. Replays will also be available at the same location for 14 days.

Autolus Therapeutics announces publication of obe-cel (AUTO1) Phase 1 ALLCAR19 data in adults with relapsed/ refractory B-ALL in Journal of Clinical Oncology

On September 1, 2021 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported publication of the obecabtagene autoleucel (obe-cel) Phase 1 ALLCAR19 data in Journal of Clinical Oncology1 (Press release, Autolus, SEP 1, 2021, View Source [SID1234587114]). Obe-cel is a fast off-rate CD19 CAR-T therapy, designed to reduce toxicity and improve engraftment1,2. ALLCAR19 is a clinical study in collaboration with Autolus’ academic partner, UCL [NCT02935257].

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"Currently there are no CD19 CAR T therapies approved for use in adult B-ALL and there exists a significant unmet need for r/r B-ALL patients," said Dr. Claire Roddie, Consultant Hematologist, UCL Cancer Institute and University College London Hospital. "We are very encouraged by the clinical profile of obe-cel, which demonstrates high sustained responses rates with remarkably little immunotoxicity despite high disease burden in many patients."

"We designed obe-cel, a unique rapid binding off rate CD19 CAR-T therapy, to specifically tackle the challenges of existing CD19 CAR T therapies, namely toxicity and lack of durable responses, by reducing the magnitude of T cell activation from each target cell interaction," said Dr. Martin Pulé, chief scientific officer of Autolus. "We have seen our design features play out in the clinical setting and these data further support our decision to progress obe-cel into the ongoing pivotal FELIX study [NCT04404660]."

Obe-cel demonstrated an excellent safety profile, with no patients experiencing high grade (≥ grade 3) cytokine release syndrome (CRS), despite the majority having a high disease burden prior to lymphodepletion1. In 8/20 patients developing grade 2 CRS, 7 patients received tocilizumab. No patients on study received corticosteroids for management of CRS1. Three of 20 patients experienced grade 3 immune effector cell-associated neurotoxicity syndrome (ICANS) and all resolved within 24-72 hours to grade 1 or less with corticosteroids1.

CAR T cell concentration reached very high levels at peak and persistence in peripheral blood was evident in 15/20 (75%) patients at a median of 166.5 days, with 4/20 (20%) patients having follow-up duration over 2 years, and 3/4 of these with ongoing CAR persistence at data cut off1. Interestingly, B-cell aplasia was ongoing in 15/20 patients at last observation1.

Of the 20 patients treated in the ALLCAR19 study, 85% patients achieved minimal residual disease (MRD) negative complete response (CR) at month 11. Duration of response remains highly encouraging. With a data cut-off date of May 17, 2021 and as presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress in June 2021, event free survival (EFS) at 12 months and 24 months was 50.2%, with median EFS not reached across all patients treated3.

Overall, obe-cel’s profile of durability and favorable toxicity is consistently observed across the B cell malignancies tested so far, with data on a total of 50 patients reported to date, including patients with adult ALL1 in ALLCAR19 [NCT02935257], pediatric ALL2 [NCT02443831] and indolent B-cell lymphomas3 [NCT02935257]. Alongside the FELIX study, a potential pivotal study ongoing in adult ALL, we continue to explore the profile of obe-cel in patients with other B-cell lymphomas through additional cohorts in the ALLCAR19 study and in patients with primary CNS lymphomas (PCNSL) through the CAROUSEL study [NCT04443829].

Citations (and hyperlinks)

Roddie et al. "Durable responses and low toxicity after fast off-rate CD19 CAR-T therapy in adults with relapsed/ refractory B-ALL." DOI: 10.1200/JCO.21.00917 Journal of Clinical Oncology – published online before print August 31, 2021
Ghorasian et al. "Enhanced CAR T cell expansion and prolonged persistence in pediatric patients with ALL treated with a low-affinity CD19 CAR." Nature Medicine volume 25, pages1408–1414(2019) (Sept 25, 2019)
Roddie et al. "Early Safety and Efficacy Findings of AUTO1 (CAT19), a Fast-Off Rate CD19 CAR, in Relapsed/Refractory Indolent B Cell Lymphomas." EHA (Free EHA Whitepaper) annual meeting, June 11 2021, abstract EP788. EHA (Free EHA Whitepaper) Investor slide presentation