On October 3, 2023 Immix Biopharma, Inc. ("ImmixBio", "Company", "We" or "Us"), reported presentation of additional AL Amyloidosis clinical data from its Phase 1b/2a NEXICART-1 (NCT04720313) study of novel, autologous, BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, NXC-201, at an oral presentation at the 20th International Myeloma Society Annual (IMS) Meeting being held in Athens, Greece on September 27-30 2023 (Press release, Immix Biopharma, OCT 3, 2023, View Source [SID1234635595]). One new patient and additional follow-up data from an additional 8 patients (9 total) are included in this update. All patients were DARZALEX (daratumumab) combination therapy relapsed/refractory and experienced a median of 6 earlier treatments that failed to stop worsening of disease (lines of therapy) prior to receiving NXC-201.

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"There are no approved drugs for relapsed/refractory AL Amyloidosis," said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, and principal study investigator. "NXC-201’s 100% response rate in relapsed/refractory AL amyloidosis patients, including t(11;14) and cardiac involved, indicates a potential broad mechanism of action. Additionally, as a one-time treatment, NXC-201 would present an attractive alternative to multi-drug combination, long-term daily or weekly regimens for relapsed/refractory AL amyloidosis patients."

"Currently, AL Amyloidosis treatment involves repeat dosing and weekly distant travel to academic medical centers," said Ilya Rachman, M.D., Ph.D., Chief Executive Officer of Immix Biopharma. "One-time treatment with NXC-201 could fill the void for relapsed/refractory AL Amyloidosis, where there are no therapies approved today, while restoring quality of life."

Gabriel Morris, Chief Financial Officer of Immix Biopharma, added, "NXC-201’s uniquely favorable CAR-T tolerability profile and an apparent ability to clear disease-causing amyloid chains from the body within ~30 days could make it particularly suitable for treatment in a potential outpatient setting."

Data were presented from 9 relapsed/refractory AL amyloidosis patients (including one new patient) in the ongoing Phase 1b/2a NEXICART-1 study of NXC-201 (formerly HBI0101). Patients were infused with CAR+T cells at doses of 150 x 106 (n=1), 450 x 106 (n=2), and 800 x 106 (n=6).
Of the 9 patients, 7/9 patients had cardiac involvement and 4/9 patients were t(11;14) relapsed/refractory. Median follow-up was 7.3 months (range: 2.5 – 16.5 months) as of the September 20, 2023 data cutoff. Data highlights:

9 relapsed/refractory AL amyloidosis patients (median 6 lines of prior therapy):

Overall response rate of 100% (9/9)
Complete response rate of 67% (6/9) (MRD 10-5)
Organ response rate of 56% (5/9)
Best responder had a duration of response of 19.2 months as of the data cutoff of September 20, 2023, with response ongoing
There were no immune effector cell-associated neurotoxicity syndrome (ICANS) events or grade 4 cytokine release syndrome (CRS) events reported
44% (4/9) had MAYO-stage IIIa/IIIb disease
For the 7 relapsed/refractory AL Amyloidosis patients with cardiac involvement:

Overall response rate of 100% (7/7)
Complete response rate of 57% (4/7) (MRD 10-5)
Organ response rate of 57% (4/7)
4 patients with t(11;14) relapsed/refractory AL Amyloidosis:

Overall response rate of 100% (4/4)
Complete response rate of 75% (3/4) (MRD 10-5)
Organ response rate of 50% (2/4)
The 20th IMS AL Amyloidosis presentation can be accessed on the ImmixBio corporate website at this link: View Source
Oral Presentation:

Event 20th International Myeloma Society Annual Meeting, Athens, Greece
Title "Feasibility of a novel academic anti-BCMA chimeric antigen receptor T-cell (CART) (HBI0101) for the treatment of relapsed and refractory AL amyloidosis"
Presentation
Date/Time (EEST)
September 27, 2023 9:00am – 14:30pm;
September 28, 2023 10:00 – 13:30pm;
September 29, 2023 9:30 – 14:15pm
About NEXICART-1

NEXICART-1 (NCT04720313) is an ongoing Phase 1b/2a, open-label study evaluating the safety and efficacy of NXC-201 (formerly HBI0101), in adults with relapsed or refractory multiple myeloma and AL amyloidosis.

The primary objective of the Phase 1b portion of the study was to characterize the safety and confirm the recommended Phase 2 dose (RP2D) and Phase 2 dose of NXC-201. The Phase 2 portion of the study will evaluate the efficacy and safety of NXC-201 with endpoints of overall survival, progression-free survival and response rates, according to International Myeloma Working Group (IMWG) Uniform Response Criteria.

The Phase 1b portion of the ongoing Phase 1b/2a clinical trial has been successful in determining the recommended Phase 2 dose (RP2D) of 800 million CAR+T cells. The expected primary endpoint for the Phase 2 portion of the ongoing Phase 1b/2a clinical trial of NXC-201 in relapsed/refractory multiple myeloma is overall response rate and duration of response. ImmixBio subsidiary Nexcella plans to submit data to the FDA in multiple myeloma once 100 patients are treated with NXC-201. The expected primary endpoint for NXC-201 in relapsed/refractory AL Amyloidosis is overall response rate. ImmixBio subsidiary Nexcella plans to submit data to the FDA in AL amyloidosis once 30-40 patients are treated with NXC-201.

About NXC-201

NXC-201 (formerly HBI0101) is a BCMA-targeted investigational chimeric antigen receptor T (CAR-T) cell therapy that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and AL amyloidosis.

About AL Amyloidosis

AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded amyloid proteins produced by plasma cells buildup in and around tissues, nerves and organs, gradually affecting their function. This can cause progressive and widespread organ damage, and high mortality rates.

AL amyloidosis affects roughly 30,000 – 40,000 patients in total throughout the U.S. and Europe, and it is estimated that there are approximately 3,000 – 4,000 new cases of AL amyloidosis annually in the U.S. The annual global incidence of AL Amyloidosis is ~15,000 patients.

The Amyloidosis market was $3.6 billion in 2017, expected to reach $6 billion in 2025, according to Grand View Research.

On October 3, 2023 Eli Lilly and Company (NYSE: LLY) and POINT Biopharma Global, Inc. (NASDAQ: PNT) reported a definitive agreement for Lilly to acquire POINT, a radiopharmaceutical company with a pipeline of clinical and preclinical-stage radioligand therapies in development for the treatment of cancer (Press release, Eli Lilly, OCT 3, 2023, View Source [SID1234635594]). Radioligand therapy can enable the precise targeting of cancer by linking a radioisotope to a targeting molecule that delivers radiation directly to cancer cells, enabling significant anti-tumor efficacy while limiting the impact to healthy tissue.

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POINT’s lead programs are in late-phase development. PNT20021 is a prostate-specific membrane antigen (PSMA) targeted radioligand therapy in development for patients with metastatic castration-resistant prostate cancer (mCRPC) after progression on hormonal treatment. Topline data from this study are expected in the fourth quarter of 2023. PNT20031 is a somatostatin receptor (SSTR) targeted radioligand therapy in development for the treatment of patients with gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Beyond the late-stage clinical pipeline, POINT has several additional programs in earlier stages of clinical and preclinical development. Additionally, POINT operates a 180,000-square-foot radiopharmaceutical manufacturing campus in Indianapolis, as well as a radiopharmaceutical research and development center in Toronto. These facilities will be utilized alongside POINT’s extensive network of supply chain partners for sourcing radioisotopes and their precursors.

"Over the past few years, we have seen how well-designed radiopharmaceuticals can demonstrate meaningful results for patients with cancer and rapidly integrate into standards of care, yet the field remains in the early days of the impact it may ultimately deliver," said Jacob Van Naarden, President of Loxo@Lilly, the oncology unit of Eli Lilly and Company. "We are excited by the potential of this emerging modality and see the acquisition of POINT as the beginning of our investment in developing multiple meaningful radioligand medicines for hard-to-treat cancers, as we have done in small molecule and biologic oncology drug discovery and development. We look forward to welcoming POINT colleagues to Lilly and working together to build upon their achievements as we develop a pipeline of meaningful new radioligand treatments for patients."

Joe McCann, Ph.D., CEO of POINT added: "The combination of POINT’s team, infrastructure and capabilities with Lilly’s global resources and experience could significantly accelerate the discovery, development and global access to radiopharmaceuticals. I look forward to a future where patients all over the world can benefit from the new cancer treatment options made possible by the joining of our two companies today."

Terms of the Agreement

Lilly will commence a tender offer to acquire all outstanding shares of POINT for a purchase price of $12.50 per share in cash (an aggregate of approximately $1.4 billion) payable at closing. The transaction has been approved by the boards of directors of both companies.

The transaction is not subject to any financing condition and is expected to close near the end of 2023, subject to customary closing conditions, including the tender of a majority of the outstanding shares of POINT’s common stock, and license transfer approval from the U.S. Nuclear Regulatory Commission. Following the successful closing of the tender offer, Lilly will acquire any shares of POINT that are not tendered in the tender offer through a second-step merger at the same consideration as paid in the tender offer.

The purchase price payable at closing represents a premium of approximately 87% to POINT’s closing stock price on Oct. 2, 2023, the last trading day before the announcement of the transaction, and 68% to the 30-day volume-weighted average price. POINT’s board of directors unanimously recommends that POINT’s stockholders tender their shares in the tender offer.

Lilly will determine the accounting treatment of this transaction as a business combination or an asset acquisition, including any related acquired in-process research and development charges, according to Generally Accepted Accounting Principles (GAAP) upon closing. This transaction will thereafter be reflected in Lilly’s financial results and financial guidance.

For Lilly, Goldman Sachs & Co. LLC is acting as exclusive financial advisor and Kirkland & Ellis LLP is acting as legal counsel. For POINT, Centerview Partners LLC is acting as exclusive financial advisor and Skadden, Arps, Slate, Meagher & Flom LLP is acting as legal counsel.

On October 3, 2023 Clarity Pharmaceuticals (ASX: CU6) ("Clarity"), a clinical stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for children and adults with cancer, reported the dosing of the first patient in its theranostic 64Cu/67Cu SAR-Bombesin Phase I/II trial in metastatic castrate resistant prostate cancer (mCRPC) (Press release, Clarity Pharmaceuticals, OCT 3, 2023, View Source [SID1234635593]). No issues were observed during the administration of 6GBq of 67Cu SAR-Bombesin and the participant continues to be followed for further safety and efficacy assessments as per protocol.

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COMBAT, which derives from "Copper-67 SAR Bombesin in metastatic castrate resistant prostate cancer" (NCT05633160)1 is a dose escalation and cohort expansion trial for up to 38 participants. 64Cu SAR-Bombesin is used to visualise gastrin-releasing peptide receptor (GRPr)-expressing lesions and select candidates for subsequent 67Cu SAR-Bombesin therapy. The aim for the trial is to determine the safety and efficacy of 67Cu SAR-Bombesin in participants with GRPr expressing mCRPC who are ineligible for therapy with 177Lu PSMA-617. These patients are unlikely to benefit from prostate-specific membrane antigen (PSMA)-targeted agents and represent a significant unmet need for both imaging and therapy. Doses of up to 14GBq of 67Cu SAR-Bombesin, in up to four cycles, are planned to be investigated in this trial (pending safety reviews).

Clarity’s Executive Chairperson, Dr Alan Taylor, commented, "SAR-Bombesin has already resulted in improvements to the management of prostate cancer for patients with PSMA-negative or low PSMA expressing lesions through diagnostic trials and we hope to confirm its safety and efficacy in this theranostic trial. We look forward to progressing the COMBAT trial and building on the compelling data from our preclinical and clinical studies to date. Combined with the logistical and manufacturing benefits of Targeted Copper Theranostics and with commercial quantities of the 67Cu radioisotope now being routinely produced domestically in the US, we see a clear path to bringing SAR-Bombesin and SAR-bisPSMA to the prostate cancer patient population in need of novel treatments."

On October 3, 2023 Century Therapeutics presented its corporate presentation (Presentation, Century Therapeutics, OCT 3, 2023, View Source [SID1234635592]).

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On October 3, 2023 BridgeBio Pharma, Inc. (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, and National Resilience, Inc. (Resilience), a technology-focused biomanufacturing company dedicated to broadening access to complex medicines, reported a strategic collaboration to manufacture and advance BBP-812, an investigational adeno-associated virus (AAV) 9 gene therapy for Canavan disease, and BBP-631, an investigational AAV 5 gene therapy for congenital adrenal hyperplasia (CAH) (Press release, BridgeBio, OCT 3, 2023, View Source [SID1234635591]).

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The two companies have developed a novel manufacturing and aligned incentive business model to drive these gene therapies forward with an emphasis on sustainability and capital-efficiency. Under the terms of the collaboration, BridgeBio will transfer its manufacturing process for its lead AAV-based gene therapy candidates to Resilience’s network of gene therapy sites. As part of an innovative cost and risk-sharing framework, Resilience will provide in-kind manufacturing services and will receive future development and approval milestones and low-to-mid single digit royalties on BBP-631 and BBP-812. Resilience will support the ongoing clinical development manufacturing needs and will serve as the primary commercial manufacturer for both programs if successful.

"Our partnership with BridgeBio seeks to accelerate development of innovative therapeutic options for patients in need," said Rahul Singhvi, Sc.D., Chief Executive Officer of Resilience. "We are pleased to partner with a gene therapy and rare disease leader, and we are inspired by their passion to deliver medicines to patients."

Beyond BBP-812 and BBP-631, Resilience will also be the primary manufacturer for future clinical projects across BridgeBio’s gene therapy portfolio. The agreement will reduce manufacturing uncertainty for these programs and is expected to help BridgeBio expedite development of gene therapies going forward.

"Manufacturing is the most critical and costly aspect of developing gene therapy for patients with a serious unmet need. We conduct process development, analytical development and optimization in our own labs, and with this partnership we can now hand these programs off to one of the most trusted partners in the industry for scale up and commercial manufacturing. This allows us to accelerate the development of our gene therapy portfolio in a capital-efficient and sustainable way with the hope of providing medicines more quickly," said Eric David, M.D., J.D., chief executive officer of BridgeBio Gene Therapy.

"We appreciate the opportunity to collaborate with the experienced and knowledgeable team at Resilience on the manufacturing of our gene therapies. We hope this expedites our path to benefiting as many patients as possible, as soon as possible," said Neil Kumar, Ph.D., founder and CEO of BridgeBio.