On December 13, 2022 Champions Oncology, Inc. (Nasdaq: CSBR), a leading global technology-enabled biotech that is transforming drug discovery through innovative AI-driven pharmaco-pheno-multiomic integration, reported its financial results for its second quarter of fiscal 2023, ended October 31, 2022 (Press release, Champions Oncology, DEC 13, 2022, View Source [SID1234625191]).

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Second Quarter and Recent Highlights:

•Record quarterly revenue of $14.3 million, an increase of 21% year over year
•Adjusted EBITDA of $686,000
•Discovery targets progressing along the development timeline; expectation to advance to preclinical phase testing in calendar 2023

Ronnie Morris, CEO of Champions, commented, "We continued to deliver good financial results while simultaneously investing in the future growth of the business. Morris added, "while long term prospects remain strong, we experienced a slowdown in the pace of our bookings growth during the quarter. We attribute the slow down to the global economic environment as our customers reanalyzed their spending budgets. We’re monitoring the situation closely and we’re optimistic this is a short-term adjustment as we’ve seen a re-acceleration in the beginning of our third quarter."

David Miller, CFO of Champions, added, "We reached another revenue milestone for Champions exceeding $14 million for the first time. The increase to $14.3 million represents 21% year over year growth which was in-line with the projected growth rate for the year. However, due to the economic climate, we saw an increase in cancellations during the quarter which will impact the revenue in the second half of the year. Accordingly, we’re revising our full year growth target to be in the 10% – 15% range."

Exhibit 99.1
Second Fiscal Quarter Financial Results

Total revenue for the second quarter of fiscal 2023, was a record $14.3 million, an increase of 21.2%, compared to $11.8 million for the same period last year. The increase in revenue was due to continued demand for our services, leading to larger pharmacology studies in both our in-vivo and ex-vivo platforms. Total costs and operating expenses for the second quarter of fiscal 2023 were $14.3 million compared to $11.5 million for the second quarter of fiscal 2022, an increase of $2.8 million or 23.9%.

For the second quarter of fiscal 2023, Champions reported income from operations of $7,000, including $119,000 in stock-based compensation and $560,000 in depreciation and amortization expenses, compared to income from operations of $263,000, inclusive of $134,000 in stock-based compensation and $346,000 in depreciation and amortization expenses, in the second quarter of fiscal 2022. Excluding stock-based compensation, depreciation and amortization expenses, Champions reported adjusted EBITDA for the quarter of $686,000 compared to $743,000 in the prior year period.
Cost of oncology solutions was $7.4 million for the three-months ended October 31, 2022, an increase of $1.8 million, or 32.7% compared to $5.6 million for the three-months ended October 31, 2021. The increase in cost of sales was primarily from compensation, mice and lab supply expenses for pharmacology studies, and an increase in compensation expense for our SaaS platform. For the three- months ended October 31, 2022, total gross margin was 48% compared to 52% for the three-months ended October 31, 2021. Pharmacology services margin was 51% vs 53% in the year ago period. The lower margin resulted from an increase in study related expenses against lower than expected revenue conversion.

Research and development expense for the three-months ended October 31, 2022 was $2.6 million, an increase of $305,000 or 13.3%, compared to $2.3 million for the three-months ended October 31, 2021. The increase was primarily from compensation and lab supply expenses related to the investment in our therapeutic discovery platform. Sales and marketing expense for the three-months ended October 31, 2022 was $1.7 million, a slight increase of $60,000, or 3.7%, compared to $1.6 million for the three-months ended October 31, 2021. General and administrative expense for the three-months ended October 31, 2022 was $2.5 million, an increase of $552,000, or 27.9%, compared to $2.0 million for the three-months ended October 31, 2021. The increase was primarily due to depreciation and amortization expenses and IT related costs to support the growth of the business.

Net cash provided by operating activities was $3.3 million for the three months ended October 31, 2022. The cash generated from operating activities was primarily due to income from operations excluding stock-based compensation, depreciation and amortization expenses as well as an increase in accounts payable due to timing differences in the ordinary course of business. The Company ended in the quarter in a strong cash position with cash on hand of $10.8 million and no debt.

Year-to-Date Financial Results

For the first six months of fiscal 2023, revenue increased 21.6% to $28.0 million compared to $23.0 million for the first six months of fiscal 2022. The increase in revenue was due to the expansion of our platforms and business lines. Total costs and operating expenses for the first six months of fiscal 2023 were $28.3 million compared to $23.0 million for the first six months of fiscal 2022, an increase of $5.4 million or 23.3%.

For the first six months of fiscal 2023, Champions reported a net loss from operations of $277,000, including $325,000 in stock-based compensation and $1.1 million in depreciation and amortization expenses, compared to income from operations of $88,000, inclusive of $414,000 in stock-based compensation and $663,000 in depreciation and amortization expenses, in the first six months of fiscal 2022. Excluding stock-based compensation, depreciation and amortization expenses, Champions reported adjusted EBITDA of $1.1 million for the first six months of fiscal 2023 compared to adjusted EBITDA of $1.2 million in the first six months of fiscal 2022.

Cost of oncology solutions was $14.5 million for the six-months ended October 31, 2022, an increase of $3.5 million, or 31.7% compared to $11.0 million for the six-months ended October 31, 2021. For the six-months ended October 31, 2022, total gross margin was 48.3% compared to 52.2% for the six-months ended October 31, 2021. For the same respective periods, pharmacology services margin was 51.1% vs 52.8%. The decrease in gross margin was primarily attributable to an increase in study related expenses in advance of the revenue recognition and lower than expected revenue conversion.

Research and development expense for the six-months ended October 31, 2022 was $5.5 million, an increase of $888,000 or 19.3%, compared to $4.6 million for the six-months ended October 31, 2021. The increase was primarily from compensation, sequencing costs, and lab supplies as we increased investment in our therapeutic target discovery platforms. Sales and marketing expense for the six-months ended October 31, 2022 was $3.4 million, a slight increase of $178,000, or 5.5%, compared to $3.2 million for the six-months ended October 31, 2021. General and administrative expense for the six-months ended October 31, 2022 was $4.9 million, an increase of $800,000, or 19.3%, compared to $4.1 million for the six-months ended October 31, 2021. The increase was primarily due to an increase in compensation and IT related expenses to support the overall infrastructure growth of the organization as well as depreciation and amortization expenses.

Net cash provided by operating activities was $3.1 million for the six-months ended October 31, 2022. The cash generated from operating activities was primarily due to an increase in income from operations excluding stock-based compensation and depreciation and amortization expenses as well as an increase in accounts payable due to timing differences in the ordinary course of business. Net cash used in investing activities was $1.4 million and was primarily from investment in additional lab and computer equipment.

Conference Call Information:
The Company will host a conference call today at 4:30 p.m. EST (1:30 p.m. PST) to discuss its second quarter financial results. To participate in the call, please call 877-545-0523 (Domestic) or 973-528-0016 (International) and enter the access code 541646, or provide the verbal reference "Champions Oncology".
Full details of the Company’s financial results will be available by December 15, 2022 in the Company’s Form 10-Q at www.championsoncology.com.

* Non-GAAP Financial Information
See the attached Reconciliation of GAAP net income (loss) to Non-GAAP net income for an explanation of the amounts excluded to arrive at Non-GAAP net income and related Non-GAAP earnings per share amounts for the three and six months ended October 31, 2022 and 2021. Non-GAAP financial measures provide investors and management with supplemental measures of operating performance and trends that facilitate comparisons between periods before and after certain items that would not otherwise be apparent on a GAAP basis. Certain unusual or non-recurring items that management does not believe affect the Company’s basic operations do not meet the GAAP definition of unusual or non-recurring items. Non-GAAP net income and Non-GAAP earnings per share are not, and should not, be viewed as a substitute for similar GAAP items. Champions defines Non-GAAP dilutive earnings per share amounts as Non-GAAP net earnings divided by the weighted average number of diluted shares outstanding. Champions’ definition of Non-GAAP net earnings and Non-GAAP diluted earnings per share may differ from similarly named measures used by other companies.

On December 13, 2022 Cellectis presented its clinical trial update presentation (Presentation, Cellectis, DEC 13, 2022, View Source [SID1234625189]).

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On December 13, 2022 Cellectis S.A. (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported that it will host a live webcast reviewing updated clinical data on its Phase 1/2a BALLI-01 clinical trial (evaluating UCART22) and on its Phase 1 AMELI-01 clinical trial (evaluating UCART123) that were presented in an oral session on December 12, 2022 at the 64th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) (Press release, Cellectis, DEC 13, 2022, View Source [SID1234625188]).

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Preliminary Data from the BALLI-01 Clinical Study

BALLI-01 is a Phase 1/2a open-label dose-escalation trial evaluating the safety and clinical activity of UCART22 given at escalating dose levels after lymphodepletion (LD) with either fludarabine and cyclophosphamide (FC) or FC with alemtuzumab (FCA) in patients with relapsed or refractory acute lymphoblastic leukemia (r/r ALL). Alemtuzumab was added to the LD regimen to sustain host T-cell and Natural Killer (NK) cell depletion and to promote UCART22 cell expansion and persistence.

Compared to the last clinical update on BALLI-01 at ASH (Free ASH Whitepaper) 2021, the webcast presented data from five additional patients who received UCART22 at dose level 3 (DL3) 5×106 cells/kg after lymphodepletion with FCA. No dose limiting toxicities (DLTs), Grade 2 or higher cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS) or adverse events of special interest (AESI) were observed.

Evidence of UCART22 anti-tumor activity was observed in 60% (n=3) of the five patients at DL3 after lymphodepletion with FCA:

A patient experienced a durable minimal residual disease (MRD) negative complete response with incomplete count recovery (CRi) that continues beyond 6 months.

A patient experienced an MRD negative complete response (CR) that continues beyond Day 56.

A patient experienced a morphologic leukemia-free state (MLFS) that continues beyond Day 84 (MRD-negative until Day 84; MRD-positive at Day 117).

All three of the responders failed multiple lines of prior therapy including chemotherapy, CD19-directed autologous CAR T cell therapy, and allogeneic stem cell transplant. Additionally, the patient with the MRD negative CR also failed both prior blinatumomab (a CD19-directed bi-specific antibody) and inotuzumab (a CD22-directed antibody-drug conjugate).

"These treatment responses in combination with the safety data are very encouraging for patients with r/r B-cell ALL who have limited, if any, treatment options, especially for those who have failed prior CD19 directed CAR T-cell therapy and allogeneic stem cell transplant’, said Nitin Jain, M.D., The University of Texas MD Anderson Cancer Center, Department of Leukemia, and coordinating investigator for the BALLI-01 study.

Next Steps

Overall, these preliminary data support the continued administration of UCART22 after FCA lymphodepletion in patients with r/r ALL. The Company is now enrolling patients in BALLI-01 with product candidate manufactured fully in-house at DL2 after FCA lymphodepletion. The first patient has been dosed at dose level 2 (DL2) 1×106 cells/kg. The next data set is expected to be released in 2023.

Preliminary Clinical Data from the AMELI-01 Study Presented at ASH (Free ASH Whitepaper) 2022

AMELI-01 is a Phase 1 open-label dose-escalation trial evaluating the safety, tolerability, expansion and preliminary activity of UCART123 given at escalating dose levels after lymphodepletion (LD) with either fludarabine and cyclophosphamide (FC) or FC with alemtuzumab (FCA) in patients with relapsed or refractory acute myeloid leukemia (r/r AML).

The oral presentation reviewed preliminary data from patients who received UCART123 at one of the following dose levels: dose level 1 (DL1) 2.5×105 cells/kg; dose level 2 (DL2) 6.25×105 cells/kg; intermediate dose level 2 (DL2i) 1.5×106 cells/kg; or dose level 3 (DL3) 3.30×106 cells/kg after lymphodepletion with FC ([n=8], DL1 – DL3) or FCA ([n=9], DL2 & DL2i).

Preliminary Safety Data

The FCA LD regimen resulted in robust lymphodepletion for greater than 28 days in all patients. Seven out of nine patients demonstrated UCART123 expansion, with maximum concentration (C max) ranging from 13,177 to 330,530 copies/µg DNA, an almost nine-fold increase compared with FC LD, and a significant increase in area under the curve (AUC)(0-28 days) (p=0.04; FC 10.2±15.7 vs. FCA 34.9±28.4).

Cytokine release syndrome (CRS) occurred in eight patients in the FC arm and nine patients in the FCA arm. In the FC arm, one patient experienced Grade 3 immune effector cell-associated neurotoxicity syndrome (ICANS) and two patients experienced Grade 4 protocol-defined dose limiting toxicities (DLTs) secondary to CRS. In the FCA arm, two patients experienced Grade 5 DLTs secondary to CRS. Grade 4 toxicities are potentially life threatening and Grade 5 toxicities result in death.

Preliminary Efficacy Data

Evidence of UCART123 anti-tumor activity was observed in four patients out of fifteen at DL2 or above with best overall responses in the FCA arm. Two out of eight patients (25%) at DL2 with FCA arm achieved meaningful response:

•
A patient who failed five prior lines of therapy experienced a durable minimal residual disease (MRD) negative complete response (CR) with full count recovery at Day 56 that continues beyond one year.

•
A patient with stable disease achieved greater than 90% bone marrow blast reduction (60% to 5%) at Day 28.

"Exemplary activity was seen in a 64-year-old female with AML who had relapsed after allogeneic stem cell transplantation (allo-SCT) and has maintained a durable MRD-negative complete response for over one year without salvage donor lymphocyte infusion or second allo-SCT," said David A. Sallman, M.D., Moffit Cancer Center, Department of Malignant Hematology, Tampa, FL. "Overall, these encouraging clinical data are a meaningful step forward for patients and support further enrollment into the study. This trial addresses a patient population with severe unmet medical need, where a successful CAR T-cell product candidate could be a major breakthrough."

The preliminary data show that adding alemtuzumab to the FC LD regimen was associated with sustained lymphodepletion and significantly higher UCART123 cell expansion, which correlated with improved anti-tumor activity.

Next Steps: 2-Dose Regimen

Overall, these preliminary data support the continued administration of UCART123 after FCA lymphodepletion in patients with r/r AML. Based on observed UCART123 expansion patterns and cytokine profiles, pursuant to an amended protocol (as described below), a second dose of UCART123 will be given after 10-14 days to allow for additional UCART123 expansion and clinical activity without the use of additional lymphodepletion. The second expansion phase in the setting of reduced disease burden is expected to be safe and allow for clearance of residual disease.

After a protocol-based pause in patient recruitment following a Grade 5 event related to CRS, the protocol treatment strategy has been modified and AMELI-01 has now commenced enrolling patients in the FCA 2-dose regimen arm at DL2, a dose that has already been administered and cleared for safety as a single dose. The arm incorporates the use of prophylactic tocilizumab, which is associated with reduced incidence of CRS.

A copy of the ASH (Free ASH Whitepaper) oral presentation is available on Cellectis’ website.

"These clinically meaningful preliminary data from both the BALLI-01 and AMELI-01 studies are very encouraging for patients and for the future of allogeneic CART-cell therapy. Both ALL and AML are diseases with an urgent need for alternative treatment options for patients, and we are excited to be moving each of these studies forward," said Dr. Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis. "We are now implementing a two-dose regimen arm for our AMELI-01 trial, as well as enrolling patients with in-house manufactured product for our BALLI-01 trial. We look forward to sharing future updates as they become available for both of these clinical studies."

Pipeline Overview

The following chart highlights our and our licensee’s key product candidates:

(1)
ALLO-501 and ALLO-501A are exclusively licensed to Servier and under a joint clinical development program between Servier and Allogene. The ALPHA and ALPHA2 studies targets Diffuse Large B-Cell Lymphoma (DLBCL) and Follicular Lymphoma (FL) indications, which are subtypes of NHL.

(2)
Phase 3 may not be required if Phase 2 is registrational.

(3)
ALLO-715 and ALLO-605 target BCMA which is a licensed target from Cellectis. Allogene has an exclusive license to the Cellectis technology for allogeneic products directed at the BCMA target. Allogene holds global development and commercial rights for this Investigational candidate.

(4)
Allogene is promoting this clinical trial in combination with SpringWorks Therapeutics.

(5)
ALLO-316 targets CD70 which is a licensed target from Cellectis.. Allogene has an exclusive license to the Cellectis technology for allogeneic products directed at the CD70 target. Allogene holds global development and commercial rights for this investigational candidate.

As of September 21, 2022, Servier has notified Allogene that Servier was discontinuing involvement in the development of CD-19-targeting allogeneic CAR T-cell products.

NATHALI-01 Study (evaluating UCART20x22):

Cellectis is enrolling patients at dose level 1 (50×106 cells) with a fludarabine, cyclophosphamide, and alemtuzumab lymphodepletion regimen in the NATHALI-01 Phase 1 dose-escalation clinical study of UCART20x22. UCART20x22 is Cellectis’ first allogeneic dual CAR T-cell product candidate being developed for patients with relapsed or refractory non-Hodgkin lymphoma and fully designed, developed and manufactured in-house.

MELANI-01 Study (evaluating UCARTCS1):

Cellectis is enrolling patients at dose level 1 (1.0×106 cells/kg) with a fludarabine and cyclophosphamide (FC) lymphodepletion regimen in the MELANI-01 Phase 1 dose-escalation clinical study of UCARTCS1 for patients with relapsed or refractory multiple myeloma (MM).

ASH 2022 Poster Presentation on UCARTCS1, in Collaboration with Amsterdam UMC

On December 10, 2022, the Amsterdam University Medical Center (VUmc location), in collaboration with Cellectis, presented preclinical data in a poster session showcasing Cellectis’ UCARTCS1 product candidate. These initial preclinical data demonstrated anti-tumor activity in vitro and in vivo, supporting the potential benefit of Cellectis’ UCARTCS1 first in-human study MELANI-01.

Collectively, the preclinical data demonstrated that UCARTCS1 has potent anti-MM activity against MM cell lines and primary MM cells, as well as in a MM xenograft model. These preclinical data support the ongoing Phase 1 clinical trial with UCARTCS1 in heavily pretreated multiple myeloma patients.

A copy of the poster presentation is available here on Cellectis’ website.

Webcast Information

The event will feature presentations by the management team and will be followed by a live Q&A. A replay of the webcast will be made available under the "Events and Webcasts" section on the Investor page of the Company’s website: View Source

In this context, the listing of the Company’s ordinary shares on Euronext Growth will be suspended on December 13, 2022 until the opening of trading of Cellectis’ ADSs on the Nasdaq Global Market at 3:30 pm (Paris time)/ 9:30 a.m. (New York time).

On December 13, 2022 MAIA Biotechnology, Inc. (NYSE American: MAIA) ("MAIA," "the Company"), reported that regulatory authorities in three European countries, Hungary, Poland, and Bulgaria, have approved the implementation of THIO-101, MAIA’s Phase 2 clinical trial evaluating its lead therapeutic candidate, THIO, in patients with Non-Small Cell Lung Cancer (NSCLC) (Press release, MAIA Biotechnology, DEC 13, 2022, View Source [SID1234625166]). The first patients in THIO-101 were dosed in Australia earlier this year.

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"Adding EU sites to THIO-101 officially makes it a global trial. We believe the data that THIO-101 generates will further validate THIO and be a major step towards bringing effective therapies to lung cancer patients."

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THIO-101 is designed to evaluate THIO’s potential immune system activation effects in NSCLC patients by administering THIO in advance of administration of Regeneron’s anti-PD1 therapy, Libtayo (cemiplimab), allowing for immune system activation and sensitivity to the PD-1 inhibitor to take effect. The primary objectives of the trial are to evaluate the safety and tolerability of THIO administered as a direct anticancer and priming immune system agent prior to cemiplimab administration, as well as to evaluate the clinical efficacy of THIO in patients with advanced NSCLC who either progressed or relapsed through treatment with an immune-check point inhibitor alone or in combination with chemotherapy.

"Adding EU sites to the THIO-101 study increases patient access to THIO on a global scale. Lung cancer is the second most common cancer indication, so addressing it requires worldwide research efforts," said MAIA Chief Medical Officer Mihail Obrocea, M.D. "Adding EU sites to THIO-101 officially makes it a global trial. We believe the data that THIO-101 generates will further validate THIO and be a major step towards bringing effective therapies to lung cancer patients."

"THIO-101 is a critical component of THIO’s clinical development process and it is of the utmost importance that we collaborate with leading cancer institutes in Australia and now in Europe, for a target total of 30 clinical trial sites in six countries," said MAIA Chairman and Chief Executive Officer Vlad Vitoc, M.D. "European sites will be excellent additions to this clinical trial. We look forward to the role they will play in the evolution and expansion of this trial, which will continue validating our telomere-targeting approach."

On December 13, 2022 BeyondSpring Inc. (the "Company" or "BeyondSpring") (Nasdaq: BYSI), a clinical stage global biopharmaceutical company focused on developing innovative cancer therapies, reported data from the ESMO (Free ESMO Whitepaper) Asia Congress 2022 and the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting about the use of lead asset, plinabulin, for the prevention of docetaxel-induced neutropenia (DIN) in non-small cell lung cancer (NSCLC) patients. In addition, data was presented on plinabulin for the prevention of docetaxel-induced neutropenia in breast cancer (BC in the 105 study) at the 2022 San Antonio Breast Cancer Symposium (SABCS). The analyses in the NSCLC studies support the efficacy of plinabulin as a monotherapy in reducing the mean duration of severe neutropenia (DSN) with a >1 day benefit for patients receiving docetaxel and plinabulin (compared to patients not receiving plinabulin) in two independent randomized trials (study 101 and 103).

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"Docetaxel-induced neutropenia can cause life-threatening infections in cancer patients, and the current standard of care, prophylactic treatment with a G-CSF in high-risk patients, has limitations. It has to be administered 24 hours following chemotherapy, and patients can experience post-treatment bone pain, a mild reduction in platelet count and a decline in quality of life," said Dr. Douglas Blayney, professor of medicine (oncology) emeritus at Stanford University Medical School and global principal investigator for the plinabulin neutropenia prevention studies. "The data that we presented at these three conferences demonstrate that plinabulin can provide solutions for some of the challenges seen with G-CSF. Plinabulin is given on the same day as chemotherapy as a short infusion, has minimal associated bone pain, no reduction in platelet count and quality of life is maintained throughout the course of therapy. Importantly, the analyses showed a reduction in mean DSN of one day or more in NSCLC patients receiving plinabulin vs placebo (no G-CSF), which is the gold standard for regulatory review."

Trials Mentioned in the Abstracts

study 101: Phase 2 study of plinabulin and docetaxel (75 mg/m2) docetaxel alone (75 mg/m2) in 2nd/3rd line NSCLC (NCT00630110)
study 103: Phase 3 study of plinabulin and docetaxel (75 mg/m2) docetaxel alone (75 mg/m2) in 2nd/3rd line NSCLC (NCT02504489)
study 105: Phase 2/3 study of plinabulin vs. pegfilgrastim with docetaxel (75 mg/m2) treatment in NSCLC, breast cancer and prostate cancer (NCT03102606)
Poster Presentation at the ASH (Free ASH Whitepaper) Annual Meeting

Title: Prevention of Docetaxel (Doc)-Induced Neutropenia (DIN) with Single Agent Plinabulin (Plin) Versus (vs) Control (No-Treatment or Placebo) in Non-Small Cell Lung Cancer (NSCLC) in Two Randomized Trials

Presentation Number: 1094

Presenter: Dr. Douglas Blayney, professor of medicine (oncology) emeritus at Stanford University Medical School and global principal investigator for the plinabulin neutropenia prevention studies

The presentation summarized data from two randomized NSCLC studies (study 101 and 103), and DIN results were compared between the plinabulin arm and the control arm (placebo or no treatment). The DSN was calculated based on Day 8 absolute neutrophil count (ANC) values in the plinabulin and control arms.

In NSCLC patients, docetaxel 75 mg/m2 is typically used without G-CSF prophylaxis ("no treatment"). This analysis focuses on the effectiveness of plinabulin (20 mg/m2 or its pharmacokinetic equivalent exposure) control for the prevention of DIN in the 101 and 103 studies.
In summary, in these two independent randomized studies, plinabulin demonstrated a superior benefit for Gr4N, Gr3/4N, all GrN and DSN compared to the control. Importantly, in both these randomized studies, there was a reduction in mean DSN of >1 day for plinabulin vs. control.
Oral Presentation at ESMO (Free ESMO Whitepaper) Asia Congress 2022

Title: Superior single agent effectiveness with plinabulin (Plin) versus (vs) placebo (Plac) for docetaxel (Doc)-induced neutropenia (DIN) prevention in non-small cell lung cancer (NSCLC) patients (pts)

Presentation Number: 276MO

Presenter: Dr. Douglas Blayney, professor of medicine (oncology) emeritus at Stanford University Medical School and global principal investigator for the plinabulin neutropenia prevention studies

The presentation summarized DIN data from a non-randomized comparison derived from two different studies: plinabulin data from study 105 and the control (placebo or no treatment) data from study 103.

In the 105 study, NSCLC pts with at least one febrile neutropenia (FN) risk factor received docetaxel 75 mg/m2 with plinabulin (20 mg/m2 or its equivalent of 40 mg fixed dose, n=30).
In the 103 study, patients received docetaxel 75 mg/m2 without plinabulin (placebo; n=224).

In summary, plinabulin was superior for the prevention of DIN and hematologic complications vs control: Grade 4 Neutropenia: 17% with plinabulin vs 40% with placebo (p-value=0.02). In addition, the mean DSN was 0.43 days for plinabulin vs. 1.32 days for placebo (p-value=0.002). There was also a favorable quality of life and safety profile with plinabulin.
Poster Presentation at SABCS

Title: Superior effectiveness of Plinabulin (Plin) versus no-treatment for Docetaxel (Doc)-induced neutropenia (N) and other hematologic complication in breast cancer (BC) patients

Presentation Number: P1-12-10

Presenter: Dr. Douglas Blayney, professor of medicine (oncology) emeritus at Stanford University Medical School and global principal investigator for the plinabulin neutropenia prevention studies

The presentation summarized DIN data for plinabulin from study 105 and the control data (placebo or no treatment) was taken from the literature.

The hematologic complications endpoints from the 27 early breast cancer patients with at least one NCCN high FN risk factor (N=27) from the Phase 3 portion of 105 study were compared with the no-treatment studies from literature where patients were given 75 mg/m2 docetaxel without G-CSF (Harvey et al., JCO, 2006 and Dieras et al., Br J Ca, 1996). Blood sampling in the no-treatment studies (Harvey and Dieras) were infrequent and likely underestimated the true grade 4 neutropenia frequency.
In summary, despite a higher frequency of ANC sampling in cycle 1, plinabulin was superior vs no-treatment for DIN and hematologic complications. Quality of life was maintained, and there were minimal adverse effects including minimal bone pain burden in the plinabulin arm vs. no-treatment.

About Plinabulin

Plinabulin, BeyondSpring’s lead asset, is a selective immunomodulating microtubule-binding agent, which is a potent antigen presenting cell (APC) inducer that is being developed as an anticancer agent. Plinabulin triggers the release of the immune defense protein, GEF-H1, which leads to two distinct effects: first is a durable anti-cancer benefit due to the maturation of dendritic cells resulting in the activation of tumor antigen-specific T-cells to target cancer cells and the second is a CIN prevention benefit. Plinabulin has single agent anti-cancer activity in a number of cancers including small cell lung cancer (SCLC) and multiple myeloma (MM). Plinabulin also exerts early-onset of action in the prevention of chemotherapy-induced neutropenia (CIN) by boosting the number of hematopoietic stem/progenitor cells (HSPCs).