On May 12, 2020 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer reported first quarter financial results for the period ended March 31, 2020 (Press release, SpringWorks Therapeutics, MAY 12, 2020, View Source [SID1234557583]).

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"We are pleased with our execution in the first quarter of 2020, which included building upon our existing intellectual property portfolio by extending the patent protection for our lead product candidate, nirogacestat, to 2039, while continuing to advance our diversified pipeline of targeted oncology programs," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our focus for the remainder of the year continues to be on enrolling patients in our ongoing clinical trials, advancing nirogacestat as a cornerstone of BCMA combination therapy for patients with multiple myeloma, and further expanding our portfolio through additional business development activities."

Recent Business Highlights

In March 2020, the United States Patent and Trademark Office issued a new composition of matter patent that covers the polymorphic form of nirogacestat that is currently in clinical development. This patent expires in 2039.

In March 2020, MapKure LLC, a clinical-stage company that is jointly owned by SpringWorks and BeiGene, Ltd., announced that the first patient was dosed in Australia in a Phase 1 clinical trial of BGB-3245, a selective RAF dimer inhibitor. This ongoing Phase 1 trial is enrolling adult patients with biomarker-defined advanced or refractory solid tumors that may benefit from BGB-3245 treatment. The companies also announced that the U.S. Food and Drug Administration has allowed the Investigational New Drug application submitted for BGB-3245 to proceed, which will enable this study to expand to U.S. sites as well.

In January 2020, SpringWorks entered into a clinical collaboration agreement with Allogene Therapeutics to evaluate nirogacestat in combination with ALLO-715, an investigational anti-B-cell maturation antigen (BCMA) allogeneic CAR T cell therapy, in patients with relapsed or refractory multiple myeloma. A Phase 1 study is expected to commence in the second half of 2020.

COVID-19 Update

To date, the COVID-19 pandemic has had a relatively modest impact on SpringWorks’ business operations, in particular on SpringWorks’ clinical trial programs, and the company is undertaking considerable efforts to mitigate the various challenges presented by this crisis. For further details and descriptions of the risks associated with the COVID-19 pandemic, please see the Risk Factors in SpringWorks’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 12, 2020 and refer to the Forward-Looking Statements section in this press release.

First Quarter 2020 Financial Results

 Research and Development (R&D) Expenses: R&D expenses were

$9.7 million for the first quarter, compared to $8.4 million for the comparable period of 2019. The increases in R&D expenses were primarily attributable to growth in employee costs, including non-cash share-based compensation associated with increases in the number of R&D personnel.

 General and Administrative (G&A) Expenses: G&A expenses were $6.4 million for the first quarter, compared to $3.3 million for the comparable period of 2019. The increases in G&A expenses were primarily attributable to growth in employee costs, including non-cash share-based compensation associated with increases in the number of G&A personnel, and increases in consulting and professional services related to the expansion of our business activities.

 Net Loss Attributable to Common Stockholders: SpringWorks reported net loss of $15.3 million, or a loss of $0.37 per share, for the first quarter of 2020. This compares to net loss of $11.4 million, or a loss of $5.41 per share, for the comparable period of 2019.

Cash Position: Cash and cash equivalents were $311.1 million as of March 31, 2020.

On May 12, 2020 Gossamer Bio, Inc. (Nasdaq: GOSS), a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutics in the disease areas of immunology, inflammation and oncology, reported its financial results for the first quarter 2020 and provided a corporate update (Press release, Gossamer Bio, MAY 12, 2020, View Source [SID1234557600]).

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"We are very pleased today to share initial results of the Gossamer team’s great execution and hard work, including that we have successfully completed the interim analysis of the LEDA study of GB001," said Sheila Gujrathi, M.D., Co-Founder and Chief Executive Officer of Gossamer. "We have begun initial Phase 3 planning and supportive activities, while awaiting final data from the study which will inform our decision to proceed to Phase 3."

"We are also excited to share topline results from our four-week Phase 1b study of GB004 in patients with active mild-to-moderate ulcerative colitis," said Dr. Gujrathi. "The safety and tolerability data, in addition to the promising efficacy data observed in the study, give us confidence as we move into Phase 2."

"The trends observed in the clinical endpoints from a 28-day study with a limited number of patients, especially those endpoints that reflect GB004’s novel mechanism of action, such as histologic remission and mucosal healing, are very exciting," said William Sandborn, M.D., Chief of the Division of Gastroenterology of University of California San Diego. "An oral, gut-targeted therapy with a non-immunosuppressive mechanism of action and a robust effect on mucosal healing would be a very meaningful addition to the treatment options for patients with ulcerative colitis. These early signals of activity, combined with the tolerability data generated to date, position GB004 as a promising and differentiated potential treatment for IBD."

Clinical-Stage Product Candidate Updates

GB001: Oral DP2 Antagonist for Eosinophilic Asthma and Chronic Rhinosinusitis (CRS)

Gossamer recently completed a pre-specified interim analysis of LEDA, its Phase 2b clinical study of GB001 in moderate-to-severe eosinophilic asthma. The interim analysis was based on approximately the first two thirds (~320) of patients who either completed or withdrew from the study. The Independent Data Monitoring Committee (IDMC) reviewed results from the interim analysis and recommended continuation of the study to its completion without modification. Based on the results of the interim analysis and the IDMC recommendation, Gossamer has commenced initial Phase 3 planning and supportive activities in anticipation of the completion of the study and final analysis of the study data. The final decision to proceed to Phase 3 will be made based on the totality of the final data from the LEDA study, as well as discussions with global regulatory authorities. Topline results from LEDA continue to be expected in the second half of 2020.

On April 21, 2020, the United States Patent and Trademark Office issued patent US 10,626,089. This key patent protects the lysine salt that is being studied in the clinical development of GB001. These compound claims further enhance the intellectual property protection around GB001. This patent is not due to expire before 2037.

The TITAN Phase 2 clinical study of GB001 in CRS, both with and without nasal polyps, is on track to report topline data in the second half of 2020.

GB004: Oral HIF-1α Stabilizer for Inflammatory Bowel Disease

Gossamer reported promising topline results from its Phase 1b study of GB004 in patients with active mild-to-moderate ulcerative colitis (UC). GB004 is designed to restore intestinal epithelial barrier integrity and function in patients with inflammatory bowel disease.

The Phase 1b study was designed to evaluate the safety, tolerability, and pharmacokinetics of a 120mg once-daily dose of GB004 in a solution formulation over a 28-day treatment period in UC patients with active disease despite treatment with 5-ASA therapy. In addition, pharmacodynamics and certain outcomes related to clinical activity were studied as exploratory measures. Thirty-four patients were randomized 2:1 to receive either GB004 (n=23) or placebo (n=11).

Safety and Tolerability: GB004 was well tolerated during the study with no effects on systemic erythropoietin or vascular endothelial growth factor observed. The most frequent adverse events experienced by patients on the GB004 arm were nausea and dysgeusia, all of which were mild in severity aside from one case of moderate nausea. All patients completed the study, except for a single patient on the GB004 arm who experienced a serious adverse event of worsening UC, which was deemed by the investigator to be unrelated to study drug.

PK / PD and Target Engagement: The gut-targeted pharmacokinetic (PK) profile of GB004 was shown with rapid clearance from systemic circulation and multi-fold higher concentrations of drug in the gut as compared to the plasma after eight hours of dosing. Preliminary data from gut biopsies showed increased expression of genes

associated with HIF-1α stabilization and enhanced epithelial barrier function, such as TJP1 and CLDN1, and evidence of reduced gut epithelial neutrophil activity in the GB004 arm compared to the placebo arm.

Clinical Activity: While this four-week study was not powered to show differences in clinical outcomes, several encouraging trends related to treatment with GB004 were observed at Day 28. Mucosal healing, defined as the achievement of both histologic remission and endoscopic improvement in the sigmoid or rectum, was observed in 4 of 23 patients (17%) in the GB004 arm compared to 0 of 11 patients in the placebo arm. Ten of 23 patients (43%) in the GB004 arm achieved histologic remission in either the sigmoid or rectum compared to 2 of 11 patients (18%) in the placebo arm. Favorable trends were also observed in clinical response (6/20 [30%] vs. 2/11 [18%]) and improvement in the rectal bleeding sub-score (13/21 [62%] vs. 5/11 [45%]). One patient in the GB004 arm achieved clinical remission; no patients in the placebo arm achieved clinical remission.

Further data from the Phase 1b study will be presented at future medical conferences.

Gossamer also recently completed a Successful Phase 1 clinical study in healthy volunteers to support the selection of a tablet formulation to be used in future clinical studies of GB004. In the study, the tablet formulation showed improved tolerability compared to solution at higher doses.

Subject to the developments in the ongoing COVID-19 viral pandemic, Gossamer plans to initiate a Phase 2 study of GB004 in UC with an oral tablet formulation of the product candidate in a 12-week induction setting in the second half of 2020.

Gossamer gives its thanks to the patients and physicians that participated in its Phase 1b study of GB004. We are grateful for the opportunity to take this novel mechanism to patients in need.

GB002: Inhaled PDGFR Inhibitor for Pulmonary Arterial Hypertension (PAH)

GB002 is currently being evaluated in an ongoing Phase 1b study in PAH. Given COVID-19 related delays in study enrollment, Gossamer now anticipates reporting initial results from this study in the second half of this year.

Subject to the developments in the ongoing COVID-19 viral pandemic, Gossamer plans to commence a Phase 2 study in functional class II and III PAH patients in the second half of 2020. The primary endpoint for this 24-week study will be change in pulmonary vascular resistance (PVR) from baseline. A key secondary endpoint will be change from baseline in 6-minute walk distance at week 24.

GB1275: Oral CD11b Modulator for Oncology Indications

Enrollment continues in the KEYNOTE-A36 Phase 1/2 study to evaluate GB1275 as a monotherapy and in combination with either KEYTRUDA (pembrolizumab) or chemotherapy in patients with selected solid tumors.

The American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) accepted an abstract for poster presentation (abstract 3085; poster 149) containing initial dose-escalation data from the KEYNOTE-A36 study, which will be presented in the Developmental Therapeutics—Immunotherapy Poster Session at the ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program. Gossamer also expects to present further data from the study in the second half of the year.

During the first quarter 2020, the European Medicines Agency granted orphan designation to GB1275 for the treatment of pancreatic cancer. GB1275 had previously received orphan drug designation from the U.S. Food and Drug Administration for the treatment of pancreatic cancer.

Corporate Updates

On May 11, 2020, Gossamer paid Aerpio Pharmaceuticals, Inc. $15 million as part of an amendment to the GB004 license agreement. Under the amended terms, all development milestones have been obviated, total remaining milestones were reduced from $400 million to $90 million, and royalties on worldwide net sales now range from a low- to mid-single digit percentage. Gossamer continues to be responsible for the remaining development, regulatory, and commercialization expenses for GB004. Aerpio’s participation right on a disposition of GB004 remains.

Chief Medical Officer Jakob Dupont, M.D. will depart Gossamer to pursue oncology opportunities closer to his family in the San Francisco Bay Area. He will serve as a consultant to Gossamer to support the development of GB1275 through a transitional period. Chief Executive Officer Sheila Gujrathi, M.D., together with Richard Aranda, M.D., Senior Vice President for Clinical Development, Caryn Peterson, Senior Vice President for Regulatory and Quality, Heather Smith, Senior Vice President for Clinical Operations, and Matt Cravets, Vice President for Biometrics will assume Dr. Dupont’s responsibilities.

"I would like to thank our Chief Medical Officer, Jakob Dupont, for his contributions to Gossamer, especially with respect to our GB1275 program, which Jakob will continue to support as a consultant," said Dr. Gujrathi. "I respect and support his personal decision to be closer to his family in the Bay Area in this unprecedented time," she added.

Gossamer will participate in the upcoming Bank of America Securities Virtual Healthcare Conference. Chief Executive Officer Dr. Sheila Gujrathi will present at the conference on Thursday, May 14, 2020, at 6:40 p.m. ET. A live webcast will be available on the "Events & Presentations" page within the Investors section of the Gossamer website and a replay will be available for 30 days following the event.

Financial Results for the Quarter Ended March 31, 2020

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of March 31, 2020, were $346.2 million. The Company expects the combination of current cash, cash equivalents and marketable securities, and access to our debt facility will be sufficient to fund its operating and capital expenditures to mid-2022.

Research and Development (R&D) Expenses: For the quarter ended March 31, 2020, R&D expenses were $41.4 million, compared to R&D expenses of $25.0 million for the same period in 2019. This increase reflects a continued ramp-up of clinical expenses in connection with

the further advancement of the GB001, GB002, GB004 and GB1275 programs and increased expenses related to the development of proprietary pre-clinical programs.

In-Process Research and Development (IPR&D) Expenses: For the quarter ended March 31, 2020, IPR&D expenses were $2.8 million, compared to $1.0 million for the same period in 2019.

General and Administrative (G&A) Expenses: For the quarter ended March 31, 2020, G&A expenses were $10.7 million, compared to $8.0 million for the same period in 2019.

Net Loss: Net loss for the quarter ended March 31, 2020, was $54.1 million, or $0.87 per share, compared to a net loss of $32.6 million, or $0.90 per share, for the same period in 2019.

Conference Call and Webcast

Gossamer’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Tuesday, May 12, to discuss its first quarter 2020 financial results and provide a corporate update.

The live audio webcast may be accessed through the Events/Presentations page in the Investors section of the Company’s website at www.gossamerbio.com. Alternatively, the conference call may be accessed through the following:

A replay of the audio webcast will be available for 30 days on the Investors section of the Company’s website, www.gossamerbio.com.

About the GB004 Phase 1b Study

The Phase 1b study of GB004 was a multi-center, randomized, double-blind, placebo-controlled study which enrolled 34 patients with active mild-to-moderate ulcerative colitis. Patients were randomized 2:1 to receive either a 120mg once-daily dose of a solution formulation of GB004 (n=23) or placebo (n=11). The primary objective of the study was to evaluate the safety and tolerability of GB004 administered over 28 days. Pharmacokinetics were evaluated as a secondary objective, while exploratory objectives included measurements of pharmacodynamics and clinical outcomes. Histology, endoscopic improvement, and mucosal healing were evaluated individually in two segments of the large intestine: the sigmoid colon and rectum.

Exploratory clinical outcomes in the study were defined as follows:

Histology: evaluated using the Robarts Histopathology Index, or RHI.

Histologic remission: RHI ≤ 3 with lamina propria neutrophils sub-score = 0 and neutrophils in epithelium sub-score = 0, among patients with baseline RHI > 3 and baseline lamina propria neutrophils and neutrophils in epithelium sub-scores > 0.

Endoscopic improvement: endoscopic sub-score of 0 or 1 if baseline endoscopic sub-score > 1, or 0 if baseline endoscopic sub-score = 1.

Mucosal healing: achievement of both histologic remission and endoscopic improvement in the same segment.

Clinical response: reduction in Mayo score of ≥ 3 points and ≥ 30% from baseline with an accompanying decrease in rectal bleeding sub-score of ≥ 1 point or absolute rectal bleeding sub-score of ≤ 1 point, among patients with baseline rectal bleeding sub-score ≥ 1 and baseline sigmoid endoscopy sub-score ≥ 1.

Clinical remission: Mayo score ≤ 2, with no individual sub-score > 1, among patients with baseline sigmoid endoscopy sub-score ≥ 1.

Improvement in rectal bleeding: reduction from baseline in rectal bleeding sub-score of ≥ 1, among patients with a baseline rectal bleeding sub-score ≥ 1.

On May 12, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, reported positive topline results from its Phase 3 clinical study evaluating the safety and efficacy of omidubicel, an investigational advanced cell therapy in development as a potential life-saving treatment option for patients in need of bone marrow transplant (Press release, Gamida Cell, MAY 12, 2020, View Source [SID1234557616]). The median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p<0.001). Neutrophil engraftment is a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells, and rapid neutrophil engraftment has been associated with fewer infections and shorter hospitalizations.1

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Despite the curative potential of bone marrow transplant, it is estimated that more than 40 percent of eligible patients in the United States do not receive a transplant for various reasons, including the lack of a matched donor.2 Even for patients who do receive a transplant, treatment is not always effective and can lead to serious complications that can dramatically affect their quality of life.3 Omidubicel is intended to address the current limitations of bone marrow transplant by providing a therapeutic dose of stem cells while preserving the cells’ functional therapeutic characteristics.

"I’m very encouraged by the data from this rigorous, Phase 3 study that was conducted at more than 50 centers around the world, as there is a significant need for new bone marrow transplant graft modalities," said Mitchell Horwitz, M.D., principal investigator and professor of medicine at the Duke Cancer Institute. "These results have the potential to substantially move the field forward and represent an important step toward making stem cell transplantation more accessible and more successful for patients with lethal blood cancers. Shortening the time to engraftment is clinically meaningful, as it can reduce a patient’s time in the hospital and decrease likelihood of infection."

"Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has the potential to be the first FDA-approved bone marrow transplant graft. We are very pleased with the results of the Phase 3 data reported today, which move us one step closer toward bringing potentially curative therapies to patients. We expect to begin to submit our biologics license application for omidubicel to the FDA on a rolling basis in the fourth quarter of this year," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We deeply appreciate the participation of patients in this trial and the support we have received from investigators and their teams."

Topline Phase 3 Data

The international, multi-center, randomized Phase 3 study (NCT02730299) was designed to evaluate the safety and efficacy of omidubicel in patients with high-risk hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. The primary endpoint was time to neutrophil engraftment. The intent-to-treat analysis included 125 patients aged 12–65 years with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma and was conducted at more than 50 clinical centers in the United States, Latin America, Europe and Asia. The demographics and baseline characteristics were well-balanced across the two study groups.

The study achieved its primary endpoint (p<0.001). In the intent-to-treat analysis, the median time to neutrophil engraftment was significantly shorter for patients who received omidubicel (12 days; 95% CI: 10-15 days) compared to the comparator group (22 days; 95% CI: 19-25 days). Omidubicel was generally well tolerated. Among patients who were transplanted per protocol, 96 percent of patients who received omidubicel achieved successful neutrophil engraftment, compared to 88 percent of patients in the comparator group.

"We are pleased with the outcome of this global, well-designed study in patients with life-threatening blood cancers who were in need of a bone marrow transplant and did not have an available matched donor," said Ronit Simantov, M.D., chief medical officer of Gamida Cell. "Importantly, these data confirmed the results from our earlier Phase 1/2 clinical study and demonstrated that patients who received omidubicel had more rapid recovery of neutrophils, which are key infection-fighting white blood cells."

The data reported today are consistent with results from a multi-center, Phase 1/2 study in 36 patients with advanced hematologic malignancies, which showed that patients treated with omidubicel demonstrated more rapid neutrophil engraftment compared to a concurrent cohort of 146 patients treated with standard umbilical cord blood as reported by the Center for International Blood and Bone Marrow Transplant Research.4 In the Phase 1/2 study, the median time to engraftment was 11.5 days (95% CI: 9-14 days) for omidubicel recipients compared to 21 days (95% CI: 20-23 days) for the CIBMTR cohort (p<0.001).

Gamida Cell expects to report full efficacy and safety results at a medical conference later this year.

Conference Call Information

Gamida Cell will host a conference call today, May 12, 2020, at 8:30 a.m. ET to discuss the Phase 3 study results. A live webcast of the conference call can be accessed in the "Investors" section of Gamida Cell’s website at www.gamida-cell.com. To participate in the live call, please dial 1-866-930-5560 (domestic toll-free), 1-409-216-0605 (international) and refer to conference ID number 5454076. A recording of the webcast will be available approximately two hours after the event, for approximately 30 days.

About Omidubicel

Omidubicel, the company’s lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In both Phase 1/2 and Phase 3 clinical studies, omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.5 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On May 12, 2020 Invivoscribe reported that their LeukoStrat CDx FLT3 Mutation Assay was submitted to regulatory authorities in China in April in support of the Astellas New Drug Application (NDA) submission of XOSPATA (gilteritinib) for treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation (Press release, Invivoscribe Technologies, MAY 12, 2020, View Source [SID1234557639]). Invivoscribe also announced expansion of its wholly-owned company, Invivoscribe Diagnostic Technologies (Shanghai) Co. Ltd., adding laboratory testing services to support clinical trials and pharmaceutical partners.

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Invivoscribe developed the LeukoStrat CDx FLT3 Mutation Assay in partnership with Astellas as the companion diagnostic supporting use of gilteritinib to treat adult patients with relapsed/ refractory FLT3-mutated AML.

"This is an announcement of two significant milestones: Submission of our LeukoStrat CDx Assay to the NMPA, and the expansion of our laboratory network into China. The submission itself is a milestone that builds on the LeukoStrat CDx FLT3 Mutation Assay as the international gold standard signal ratio assay for comprehensive assessment of activating mutations in the FLT3 gene; one of the most important biomarkers in AML", said Jeffrey Miller, Invivoscribe’s CSO and CEO. "Further, the expansion of our company to include a laboratory was done to provide comprehensive test support for our partners conducting trials in China. Testing will include simultaneous assessment of minimal residual disease by both flow cytometry and next-generation sequencing technologies conducted on exactly the same subject specimen. These paired analyses have been requested by regulatory authorities and partners, but have been missing in clinical studies. Invivoscribe’s LabPMM laboratories will bring this long-desired capability to fruition in our laboratories in China, Japan, Europe, and the US."

The LeukoStrat CDx FLT3 Mutation Assay is the only internationally standardized signal ratio assay that identifies both ITD and TKD mutations of the FLT3 biomarker. It has been approved as a companion diagnostic by regulatory authorities in the US, Australia, and Japan, and is available as a CE-marked kit in Europe and Switzerland. The LeukoStrat CDx FLT3 Mutation Assay served as the companion diagnostic in the ADMIRAL, RATIFY and QuANTUM-R clinical trials, which supported approvals of gilteritinib (XOSPATA), midostaurin (RYDAPT) and quizartinib (VANFLYTA). The assay is available both as a service and as a kit. LeukoStrat CDx FLT3 Mutation Assay test services are available from LabPMM locations in Japan, Germany, and the United States.

On May 12, 2020 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported additional data from multiple oral and poster presentations during the ongoing 23rd American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting as well as its first quarter 2020 financial results (Press release, Beam Therapeutics, MAY 12, 2020, View Source [SID1234557584]).

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"The start to 2020 has been one of focused execution across our business and continued advancement of our proprietary base editing platform and portfolio, even with the uncertainty around the COVID-19 pandemic," said John Evans, chief executive officer of Beam. "We are pleased to have a strong showing during ASGCT (Free ASGCT Whitepaper), in which we are presenting our first in vivo proof of concept for base editing in alpha-1 antitrypsin deficiency as well as two potentially best-in-class approaches for base editing in sickle cell disease, including the direct correction of the HbS point mutation. These data are a testament to the compelling therapeutic potential of base editors and, coupled with our financial strength following our initial public offering, position us to continue our comprehensive investment to develop base editors as a new class of precision genetic medicines for patients."

Updated Data Presented at ASGCT (Free ASGCT Whitepaper)

Alpha-1 Antitrypsin Deficiency Program Demonstrates Proof-of-Concept in a Mouse Model: Beam reported additional data from its direct editing program for Alpha-1 in a poster at ASGCT (Free ASGCT Whitepaper) titled "Use of Adenine Base Editors to Precisely Correct the Disease-Causing PiZ Mutation in Alpha-1 Antitrypsin Deficiency." Updated data demonstrate that using Beam’s adenine base editors (ABEs) to directly correct the PiZ mutation resulted in an average of 16.9% correction of beneficial alleles at seven days and 28.8% at three months. This significant increase over the period suggests that corrected hepatocytes may have a proliferative advantage relative to uncorrected cells. In addition, treated mice demonstrate decreased alpha-1 antitrypsin (A1AT) globule burden within the liver and a durable increase in serum A1AT, roughly 4.9-fold higher than in controls, was observed at three months. These data support the potential for base editing to treat both the lung and liver manifestations of Alpha-1.

HbG-Makassar Program Demonstrates Significant In Vitro Direct Editing for Sickle Cell Disease: Beam reported additional data from its direct correction program for sickle cell disease in a poster titled "A Novel Base Editing Approach to Directly Edit the Causative Mutation in Sickle Cell Disease." Updated data show conversion of the causative HbS point mutation to HbG-Makassar, a naturally-occurring human variant that does not cause hemoglobin to polymerize or red cells to sickle, at levels greater than 80%. A simultaneous reduction of HbS globin to less than 20% of control levels was observed in edited in vitro-differentiated erythroid cells from a homozygous sickle cell disease patient. In addition, more than 70% of erythroid colonies derived from edited patient cells showed biallelic editing, with 20% monoallelic and 2% unedited. Further, when in vitro-differentiated erythroid cells were subjected to hypoxia, a very significant reduction in sickling was observed. These data demonstrate therapeutic levels of correction and support advancement of this program to potentially address the underlying genetic cause of sickle cell disease.

HPFH Program Demonstrates Therapeutically Relevant Increase in Gamma Globin Protein: Beam will present findings from its HPFH program in an oral presentation titled, "Base Editing of Gamma Globin Gene Promoters Generates Durable Expression of Fetal Hemoglobin for the Treatment of Sickle Cell Disease." Beam’s HPFH program aims to recreate naturally-occurring single base changes in the gamma globin gene promoters (HBG1 and HBG2) that disrupt repressor binding and lead to increased expression of gamma globin, which is half of the fetal hemoglobin (HbF) tetramer. Beam observed more than 90% editing and a more than 65% increase in gamma globin levels compared to less than 1.5% in unedited cells in mice at 16 weeks post-transplant. Beam was able to replicate these findings with a second donor at 18 weeks post-transplant. In addition, the data showed successful editing of CD34+ cells from a homozygous sickle patient, demonstrating greater than 60% increase in gamma globin levels with a concomitant decrease to less than 40% in sickle beta globin levels in vitro. The data demonstrate that ex vivo delivery of ABEs achieved precise editing, resulting in long-term engraftment and therapeutically relevant increases in target gene expression.

Business Continuity Plans

Beam continues to execute its business objectives for 2020, while closely monitoring the impact of the evolving COVID-19 pandemic. The company continues to operate under a remote model while advancing critical research and development activities to support an initial wave of Investigational New Drug (IND) applications beginning in 2021.

First Quarter 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $253.4 million as of March 31, 2020, which includes $188.3 million in net proceeds from the company’s initial public offering completed in February 2020.

Research & Development (R&D) Expenses: R&D expenses were $21.5 million for the quarter ended March 31, 2020, compared to $9.2 million for the quarter ended March 31,

2019. This increase was primarily due to the growth in the number of research and development employees and their related activities, as well as the expense allocated to R&D related to Beam’s leased facilities.

General &Administrative (G&A) Expenses: G&A expenses were $6.8 million for the quarter ended March 31, 2020, compared to $3.9 million for the quarter ended March 31, 2019. This increase was primarily a result of increased personnel related costs due to an increase in general and administrative employees and other administrative expenses.

Net Loss: Net loss attributable to common stockholders was $31.7 million, or $1.03 per share, for the quarter ended March 31, 2020, compared to $16.6 million for the quarter ended March 31, 2019.