On June 12, 2023 Bluebird Bio presented its corporate presentation (Presentation, bluebird bio, JUN 12, 2023, View Source [SID1234632648]).

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On June 12, 2023 AIM ImmunoTech Inc. (NYSE American: AIM) ("AIM" or the "Company"), an immuno-pharma company focused on the research and development of therapeutics to treat multiple types of cancers, immune disorders and viral diseases, reported an update on its ongoing clinical development programs evaluating Ampligen (rintatolimod), a dsRNA and highly selective TLR3 agonist immune-modulator with broad spectrum activity (Press release, AIM ImmunoTech, JUN 12, 2023, View Source [SID1234632642]).

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AIM Chief Executive Officer Thomas K. Equels commented, "We have made promising progress across multiple clinical fronts, and I am proud of the dedication of our team and their evident operational execution. Ampligen continues to demonstrate significant potential across multiple types of cancers, immune disorders and viral diseases. We are committed to advancing Ampligen’s development and are poised to achieve multiple key milestones in 2023."

Ampligen Phase 2 Study as a Therapy for Locally Advanced Pancreatic Cancer ("LAPC") (AMP-270)

The AMP-270 clinical trial is the Company’s randomized, open-label, controlled, parallel-arm study with the primary objective of comparing the efficacy of Ampligen versus a no treatment control group following FOLFIRINOX for subjects with locally advanced pancreatic adenocarcinoma. Secondary objectives include comparing safety and tolerability. AMP-270 is expected to enroll approximately 90 subjects in up to 30 centers across the United States and Europe.

The Company is recruiting patients for its AMP-270 Phase 2 study of Ampligen as a therapy for LAPC. The lead site at the University of Nebraska Medical Center is now open and actively working to enroll patients. The Gabrail Cancer & Research Center in Canton, Ohio is also recruiting patients.

Kelsey Klute, MD, Medical Director of the Pancreatic Diseases Specialty Clinic at Nebraska Medicine, commented, "We are pleased to join into what we believe is an important Phase 2 study that has the potential to address a much-needed treatment option for LAPC. Our team is actively working to enroll and treat patients on the study in a joint effort with all other clinical trial sites. We are encouraged by the potential of Ampligen and look forward to further exploring its potential in the treatment of LAPC."

AIM continues its efforts to open additional clinical sites at premier cancer centers across the United States and Europe. The Company remains optimistic for first patient enrollment in the second quarter of this year.

For more information about the AMP-270 please visit ClinicalTrials.gov and reference identifier NCT05494697.

Ampligen Phase 1 Study for the Treatment of Early-Stage Triple Negative Breast Cancer ("TNBC")

Ampligen is currently being evaluated in a Phase 1 study for TNBC by Roswell Park Comprehensive Cancer Center. The study is designed to evaluate the safety and tolerability of a combination of Ampligen and celecoxib with or without Intron A, when given along with chemotherapy. The primary endpoint was safety and tolerability. Secondary endpoints included pCR rate. Tumor and blood biomarkers were analyzed in exploratory studies. The goal of this approach is to increase survival. In November 2022, Roswell presented positive data at the SITC (Free SITC Whitepaper) 37th Annual Meeting demonstrating the chemokine-modulating regimen including Ampligen was well tolerated, with promising clinical activity of pathologic complete response (pCR) + microinvasive residual disease (ypTmic).

Roswell has now completed the study and is currently analyzing data. Based on current timelines, the Company anticipates Roswell will release – via publications – full study results before the end of the year.

For more information about the Phase 1 study of Ampligen for the treatment of TNBC, visit ClinicalTrials.gov and reference identified NCT04081389.

Ampligen Analysis Demonstrating Potential as a Maintenance Therapy After Systemic Chemotherapy in Patients with Metastatic and LAPC

A new analysis was recently presented in an abstract titled, "Immune Response in Stable Pancreatic Cancer after Rintatolimod Treatment," by Professor C.H.J. van Eijck, MD, PhD of Erasmus Medical Center at the 2023 Annual Pancreas Club Meeting held in Chicago, Illinois. Findings from the analysis demonstrated that treatment of patients with LAPC after FOLFIRINOX with Ampligen (rintatolimod) may induce markers of dendritic cells and T cells in a subgroup of patients. The absence of these markers may predict tumor progression after FOLFIRINOX, providing the future possibility of identifying pancreatic cancer patients who could potentially respond to Ampligen.

The results from the new analysis build on the previously reported peer-reviewed article "Rintatolimod (Ampligen) Enhances Numbers of Peripheral B Cells and Is Associated with Longer Survival in Patients with Locally Advanced and Metastasized Pancreatic Cancer Pre-Treated with FOLFIRINOX: A Single-Center Named Patient Program," published in March 2022 in the journal Cancers.

Ampligen as a Potential Therapy Against Ebola Virus Disease ("EVD")

The South African Patent and Trademark Office (CIPC) has granted patent No. 2022/01079, titled "Compositions and Methods Useful for Ebola Virus Infection." This pioneering patent recognizes the efficacy of AIM’s tdsRNA drug family, of which Ampligen is a member, as a potential solution to combat the devastating impact of Ebola outbreaks. Under the newly granted patent, a composition comprising tdsRNA may be used in a method that involves administering the composition to a subject to prevent, treat, inhibit, or attenuate an Ebola virus infection in that subject. Significantly, the composition may be used as a treatment for individuals already infected with the Ebola virus, or the composition may be used to prevent an Ebola infection.

About Ampligen

Ampligen is AIM’s dsRNA product candidate being developed for globally important cancers, viral diseases and disorders of the immune system. Ampligen has demonstrated in the clinic the potential for standalone efficacy in a number of solid tumors. Additionally, Ampligen has shown success in increasing survival rates and efficacy in the treatment of animal tumors when used in combination with checkpoint blockade therapies.

Ampligen is currently being evaluated as a combinational therapy for the treatment of a variety of solid tumor types in multiple clinical trials – both underway and planned – at major cancer research centers around the country. Ampligen is being used to treat pancreatic cancer patients in an Early Access Program approved by the Inspectorate of Healthcare in the Netherlands at Erasmus Medical Center. Additionally, Ampligen is also approved in Argentina for the treatment of severe chronic fatigue syndrome and is currently being evaluated in SARS-CoV-2/COVID-19, myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) and Post COVID Conditions.

On June 11, 2023 Chinook Therapeutics, Inc. (Nasdaq: KDNY), a biopharmaceutical company focused on the discovery, development and commercialization of precision medicines for kidney diseases, reported that it has entered into an agreement and plan of merger with Novartis AG pursuant to which Novartis will acquire Chinook for $40 per share in cash, or a total of $3.2 billion (Press release, Chinook Therapeutics, JUN 11, 2023, View Source [SID1234632641]). This offer represents a premium of 83 percent to Chinook’s 60-day volume-weighted average stock price and 67 percent to Chinook’s closing price on June 9, 2023. In addition, Chinook shareholders will receive contingent value rights (CVRs) providing for payment of up to $4 per share upon the achievement of certain future regulatory milestones with respect to Chinook’s lead product candidate, atrasentan. Total consideration including the contingent value right, if the milestones are achieved, would be approximately $3.5 billion. The transaction has been unanimously approved by the Boards of Directors of both companies.

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"We are pleased that Novartis recognizes the significant value that the Chinook team has built with our pipeline of clinical and preclinical programs for patients with rare, severe chronic kidney diseases," said Eric Dobmeier, president and chief executive officer of Chinook Therapeutics. "We believe this transaction is great news for kidney disease patients and the programs we have built at Chinook. Through this merger, Novartis can apply its substantial resources to pursue broader development efforts and commercialization of atrasentan, zigakibart (BION-1301) and other programs in our pipeline to build its global renal therapeutic area."

Completion of the transaction is expected in the second half of 2023, pending approval by Chinook’s stockholders and satisfaction of other customary closing conditions. Until that time, Chinook will continue to operate as a separate and independent company.

Centerview Partners LLC and MTS Health Partners, L.P. are serving as financial advisors, and Fenwick & West LLP is serving as legal counsel to Chinook.

Transaction Details

Under the terms of the merger agreement, Novartis will acquire all of the outstanding shares of Chinook through a subsidiary for a price of $40 per share in cash at closing. The CVRs to be issued to Chinook shareholders will provide for payments of up to an additional $4 per share with respect to specific regulatory approvals for atrasentan, $2 of which is related to IgA nephropathy and $2 of which is related to focal segmental glomerulosclerosis. The closing of the proposed transaction is subject to certain conditions, including approval by Chinook’s stockholders, the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and other customary conditions. A copy of the merger agreement will be filed with the Securities and Exchange Commission ("SEC") and will be publicly available.

On June 11, 2023 Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of oncology, autoimmune, metabolic, ophthalmology and other major diseases, reported that Phase 1 clinical data of the globally first anti-CD47/PD-L1 bispecific antibody IBI322 for the treatment of anti-PD-(L)1-resistant patients with classic Hodgkin lymphoma was presented in oral presentation at the 28th Annual Meeting of the European Society of Hematology (EHA 2023) (Press release, Innovent Biologics, JUN 11, 2023, View Source [SID1234632627]). The EHA (Free EHA Whitepaper) Annual Meeting was held in Frankfurt, Germany, from June 8 to 15, 2023.

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Title: CD47/PD-L1 bispecific antibody (IBI322) in anti-PD-1 or PD-L1 treatment-resistant classical Hodgkin lymphoma: a Phase 1 study
Session: s445 Hodgkin lymphoma – Clinical, 11/06/2023, 11:30 – 12:45 (CEST)
Final Abstract Code: S216
Presenting Author: Dr. Jingwei Yu, Tianjin Medical University Cancer Institute & Hospital, Tianjin, China

The presented data was based on an dose-expansion cohort from a Phase 1 study (NCT04795128) aiming to evaluate the safety and preliminary antitumor activity of IBI322 in patients with anti-PD-(L)1 treatment-resistant classic Hodgkin lymphoma. Eligible patients receive IBI322 (45 mg/kg intravenous Q2W) until unacceptable toxicity or disease progression, or up to 24 months. A total of 24 patients currently included, of which 23 patients were efficacy evaluable.

Among the 23 patients, the objective response rate (ORR) and disease control rate (DCR) were 47.8% (95% CI: 26.8-69.4) and 91.3% (95% CI: 72.0-98.9), respectively. Among the 7 patients with primary resistance, the ORR was as high as 57.1% (95% CI: 18.4-90.1), and 3 patients achieved complete response (CR).

In terms of safety, the incidence of treatment-related adverse reactions (TRAE) was 91.7%, and the most common TRAE were decreased lymphocyte count (62.5%), anemia (62.5%), decreased white blood cell count (20.8%), and decreased platelet count (20.8%). The incidence of grade ≥ 3 TRAE was 41.7%, with the most common ≥ grade 3 TRAE (>5%) associated with decreased lymphocyte count (29.2%). There were no TRAE leading to permanent treatment discontinuation or death. As of the data cut-off, 12 patients were still receiving IBI322 monotherapy.

Overall, IBI322 monotherapy showed promising anti-tumor efficacy with a manageable safety profile in anti-PD-1 or PD-L1 treatment-resistant classical Hodgkin lymphoma patients. Abstract Link

Professor Ting Niu, the principal investigator of this study, West China Hospital of Sichuan University, said: "This phase 1 study shows that IBI322 monotherapy is effective and safe in the treatment of PD-1 or PD-L1 treatment-resistant classical Hodgkin lymphoma, and we look forward to longer follow-up results and larger sample sizes in the future. IBI322 has the potential to be an effective treatment option for the treatment of immunotherapy-resistant classical Hodgkin lymphoma and other types of lymphoma to meet the unmet clinical needs. "

Dr. Hui Zhou, Senior Vice President of Innovent, said: "With the widespread application of anti-PD-(L)1 monoclonal antibodies in the field of classical Hodgkin lymphoma, immunotherapy resistance has emerged as an urgent clinical need. IBI322, as globally the first anti-CD47/PD-L1 bispecific antibody for the treatment of anti-PD-1 or PD-L1 treatment-resistant classic Hodgkin lymphoma, showed encouraging anti-tumor efficacy and a manageable safety profile in the Phase 1 clinical study, exhibiting its potential to overcome immunotherapy resistance for patients with classical Hodgkin lymphoma. "

About Hodgkin lymphoma

Hodgkin lymphoma is a special type of lymphoma containing Reed-Sternberg cells, which accounts for about 8.54% of lymphoma. The current incidence rate is about 0.6 cases per 100,000 population. After first-line radiotherapy + chemotherapy ABVD (doxorubicin + bleomycin + vincristine + dacarbazine), ASCT (autologous stem cell transplantation) has achieved a 5-year survival rate of more than 80% for patients, but the treated rate is limited in China due to several reasons, including the poor tolerability for certain patients, the relatively low mental acceptance, and limited medical resources; Another 15-20% of patients do not respond to initial treatment and develop relapsed or refractory classical Hodgkin lymphoma after immunotherapy resistance, which is currently an urgent clinical need.

About IBI322

IBI322 is a recombinant anti-human CD47/PD-L1 bispecific antibody developed by Innovent Biologics. As a bispecific antibody, IBI322 targets CD47 on the surface of tumor cells, blocks SIRPα/CD47 pathway and activates macrophages to attack the tumor cells. Furthermore, IBI322 target PD-L1 on the surface of tumor cells, blocks the PD-1/PD-L1 pathway, which counteracts the inhibition of T cells and activates the T cells to attack the tumor cells. By inhibiting two different targets, IBI322 can not only activate both innate immune pathway and adaptive immune pathway, which provides synergistic effect, but also reduce the red blood cell destruction.

On June 11, 2023 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported updated clinical data from KOMET-001, a Phase 1/2 clinical trial of the Company’s potent and selective menin inhibitor, ziftomenib, including significant clinical activity in patients with heavily pretreated and co-mutated relapsed/refractory NPM1-mutant acute myeloid leukemia (AML) (Press release, Kura Oncology, JUN 11, 2023, View Source [SID1234632626]).

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The updated clinical data are being featured during a late-breaking oral session today at the 2023 European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress in Frankfurt, Germany. A copy of the presentation is available in the Posters and Presentations section on Kura’s website.

"Our goal with our ziftomenib program is to transform the standard of care for patients with acute leukemias," said Stephen Dale, M.D., Chief Medical Officer of Kura Oncology, "and we are delighted to share new clinical and preclinical data that we believe further demonstrate its potential best-in-class product profile. The emerging data for ziftomenib include: high clinical activity in relapsed/refractory NPM1-mutant AML patients, including 35% achieving durable complete remissions (CR) with maintained full count recovery on ziftomenib monotherapy; a lower frequency of MEN1 resistance mutations; a favorable safety and tolerability profile; strong evidence of mechanistic synergy with standards of care such as venetoclax and FLT3 inhibitors; and convenient once-daily, oral dosing and optimal pharmaceutical properties for combination. We believe ziftomenib has the ideal properties to become a cornerstone of therapy across the continuum of treatment, and we intend to build on the growing momentum as we continue to execute on our registration-enabling study in NPM1-mutant AML and move rapidly into combinations."

As of the data cutoff on April 12, 2023, seven of the 20 patients (35%) with NPM1-mutant AML treated at the recommended Phase 2 dose (RP2D) of 600 mg achieved a CR with full count recovery. Notably, 33% (2/6) of patients with FLT3 co-mutations and 50% (4/8) of patients with IDH co-mutations achieved a CR on ziftomenib. Two patients underwent a stem cell transplant (SCT) and remain in remission as of the data cutoff, including one on post-SCT ziftomenib maintenance therapy. An eighth patient who had a CR with incomplete recovery (CRi) at the time of transplant subsequently evolved to a CR and remains on study.

The median duration of response for all NPM1-mutant patients was 8.2 months (95% CI: 1.0 to NE), with a median follow-up of 8.8 months. The median duration of response for patients censored at SCT was 5.6 months (95% CI: 1.0 to NE). As of the cutoff date, three patients treated at 600 mg remain on study and in CR; an additional NPM1-mutant patient treated at 200 mg remained on ziftomenib for 36 cycles.

As part of an ongoing analysis, the resistance mutation MEN1-M3271 has been detected in three patients treated with ziftomenib: in two patients, the mutation was detected at study entry after the patients had progressed on a prior menin inhibitor, and in the third patient, the mutation was detected after four cycles of ziftomenib therapy and, despite the mutation, the patient was maintained in a condition of stable disease through cycle 7. These data show that MEN1 mutations developed in just 3% (1/29) of patients analyzed following treatment with ziftomenib and suggest that resistance mutations are less likely to evolve after prolonged exposure to ziftomenib monotherapy. A key new biochemical finding, confirmed by crystal structure, demonstrates that ziftomenib retains full activity against the MEN1-T349M mutation, detected in two-thirds of patients who acquired menin resistance mutations on another recent menin inhibitor trial.

"NPM1-mutant AML accounts for approximately 30% of AML cases annually and represents a disease of significant unmet need for which no approved targeted therapy exists," said Amir Fathi, M.D., Director of the Leukemia Program at the Massachusetts General Hospital. "The clinical data presented today continue to demonstrate the ability of ziftomenib to drive durable responses as a monotherapy in heavily pretreated patients with NPM1-mutant AML. In addition, data appears to suggest that ziftomenib is less likely to induce common MEN1 resistance mutations, coupled with emerging data showing the retention of activity against other key resistance mutants, are exciting, as we look to advance ziftomenib into combinations and treat patients in earlier lines of therapy."

Continuous daily dosing of ziftomenib was well tolerated and the safety profile remains consistent with features of underlying disease. The on-target effect of differentiation syndrome was manageable, with 15% of patients experiencing Grade 1 or 2 events and 5% experiencing a Grade 3 event.

Enrollment in a Phase 2 registration-directed study of ziftomenib in patients with relapsed/refractory NPM1-mutant AML continues to outperform projections. The study is expected to enroll a total of 85 patients at 62 U.S. and European sites. Kura is also preparing to initiate a series of studies to evaluate ziftomenib in combination with current standards of care in earlier lines of therapy and across multiple patient populations, including NPM1-mutant and KMT2A-rearranged AML. The Company has begun site activation in the first of these studies, KOMET-007, and is on track to dose the first patients this quarter.

Virtual Investor Event

Management will host a virtual investor event featuring company management and investigators from the Phase 1 trial of ziftomenib at 8:00 a.m. ET on Monday, June 12, 2023. The event will be webcast live and can be accessed on the Investors section of Kura’s website at www.kuraoncology.com. An archived replay will be available shortly after the conclusion of the live event.

About Acute Myeloid Leukemia

AML is the most common acute leukemia in adults and begins when the bone marrow makes abnormal myeloblasts (white blood cells), red blood cells or platelets. Despite the many available treatments for AML, prognosis for patients remains poor. NPM1-mutations are among the most common genetic alterations, representing approximately 30% of AML cases. While patients with NPM1-mutant AML have high response rates to frontline therapy, relapse rates are high and survival outcomes are poor, with only 30% overall survival at 12 months in the relapsed or refractory setting. Additionally, NPM1 mutations frequently occur with co-mutations in other disease-associated genes, including FLT3, DNMT3A and IDH1/2, with prognosis heavily influenced by the occurrence of co-occurring mutations. Median overall survival is only approximately six months following relapse for NPM1-mutant patients. KMT2A-rearrangements are less frequent, representing approximately 5-10% of AML. No FDA-approved therapies targeting NPM1-mutant and KMT2A-rearranged AML currently exist.

About Ziftomenib

Ziftomenib is a novel, once-daily, oral investigational drug candidate targeting the menin-KMT2A/MLL protein-protein interaction for treatment of genetically defined AML patients with high unmet need. In preclinical models, ziftomenib inhibits the KMT2A/MLL protein complex and exhibits downstream effects on HOXA9/MEIS1 expression and potent anti-leukemic activity in genetically defined preclinical models of AML. Ziftomenib has received Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of AML. Additional information about clinical trials for ziftomenib can be found at kuraoncology.com/clinical-trials/#ziftomenib.