On May 12, 2022 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported financial results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Vincerx Pharma, MAY 12, 2022, View Source [SID1234614364]).

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"In April, we presented positive preclinical and preliminary clinical data on PTEFb/CDK9 inhibitor VIP152 at AACR (Free AACR Whitepaper). These data, coupled with our findings presented at ASH (Free ASH Whitepaper) last year, suggest that VIP152 has the potential to provide new treatment options for patients across various MYC and MCL-1-driven tumor types," said Ahmed Hamdy M.D., Chief Executive Officer of Vincerx.

"We continue dosing patients in our VIP152 clinical program and are seeing improvements in trial enrollment following a challenging pandemic year," added Dr. Hamdy. "This June, we are excited for our poster presentation at the annual European Hematology Association (EHA) (Free EHA Whitepaper) meeting, which will show data from patients with lymphoma treated with VIP152. We remain on track to initiate Phase 2 studies of VIP152 in the second half of this year."

"Looking to the second half of this year and into 2023, we look forward to continuing to advance our preclinical bioconjugation platform – a diverse, modular platform of linkers and payloads that can be conjugated with antibodies and small molecules to create novel targeted therapeutics for a broad range of solid tumors and hematologic malignancies. We remain on track to file an IND in the second half of this year for VIP236, a small molecule drug conjugate (SMDC) engineered to bind an αvß3 integrin adhesion molecule that is abundantly expressed in advanced metastatic solid tumors. αvß3 is a hallmark of aggressive cancers and poor prognosis. We also remain on-target to file INDs in the second half of 2023 for our two initial antibody drug conjugates (ADCs), VIP943, an anti-IL3RA-KSPi, and VIP924, an anti-CXCR5-KSPi, both with a CellTrapper moiety. From a financial perspective, our current cash resources position us to continue to pursue our upcoming regulatory and clinical milestones," concluded Dr. Hamdy.

RECENT CORPORATE HIGHLIGHTS

VIP152

Abstract accepted for poster presentation at the upcoming European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting, titled "VIP152 is a novel CDK9 inhibitor with improved selectivity, target modulation, and cardiac safety in patients with lymphoma."

Presenting author: Melanie Frigault, PhD

Abstract number: P1269

Session date and time: Friday, June 10, 2022; 16:30-17:45 CEST

Presented poster, "VIP152, a selective CDK9 inhibitor, demonstrates sensitivity in gynecologic cell lines that are cisplatin sensitive or resistant and delivers in vivo antitumor efficacy," at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2022.

Bioconjugation Platform

Invited talk on the Company’s modular assets that can build innovative and high-potency ADCs titled, "ADCs with KSP-Inhibitor Payloads and a Tailored Design of Linker and Metabolite Profile," at the Festival of Biologics meeting March 2022.

Continue to advance our next generation modular bioconjugation platform, comprised of a first-in-class SMDC for solid tumors (VIP236) and two best-in-class preclinical-stage assets for hematologic malignancies (VIP943 and VIP924).

VIP236 consists of an αvß3 integrin binder and a linker that is cleavable by neutrophil elastase. The payload is an optimized camptothecin derivative designed for high cellular permeability and low efflux. IND filing in solid tumors expected in 2H 2022.

VIP943 (anti-IL3RA) and VIP924 (anti-CXCR5) are two antibodies linked to a KSP inhibitor – a novel payload class in ADCs. These ADCs also have a stable linker specifically cleaved by legumain, a tumor associated protease. The payload (i.e., KSP inhibitor) is modified to be trapped in the cell by the CellTrapper moiety. Manufacturing is underway and IND filings expected in 2H 2023.

FIRST QUARTER FINANCIAL RESULTS

Vincerx Pharma had $96.5 million in cash as of March 31, 2022, as compared to $111.5 million as of December 31, 2021. Based on its current business plans and assumptions, Vincerx believes its available cash will be sufficient to meet its operating requirements through 2023.

Research and development (R&D) expenses for the quarter ended March 31, 2022 were $16.0 million, as compared to $4.8 million for the same period in 2021. The increase was primarily related to increases in manufacturing services of approximately $4.0 million, including the initiation of manufacturing associated with our ADC program, new employee salaries of approximately $2.5 million, third party research and preclinical work of approximately $2.0 million, clinical services of approximately $1.4 million and stock-based compensation of approximately $0.8 million.

General and administrative (G&A) expenses for the quarter ended March 31, 2022 were $5.6 million, as compared to $4.8 million for the same period in 2021. The increase was primarily related to new employee salaries and increases in legal (patent protection and filings), accounting and other professional services in support of our operations as a public company.

For the quarter ended March 31, 2022, Vincerx reported a net loss of $16.4 million, or $0.79 per share. For the quarter ended March 31, 2021, Vincerx reported a net loss of $6.3 million, or $0.46 per share.

On May 12, 2022 Day One Biopharmaceuticals (Nasdaq: DAWN), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, reported financial results for the first quarter of 2022 and highlighted recent corporate achievements (Press release, Day One, MAY 12, 2022, View Source [SID1234614363]).

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"We continue to make excellent progress across our clinical programs and look forward to reporting initial data from our pivotal Phase 2 FIREFLY-1 trial in relapsed pLGG next month," said Jeremy Bender, Ph.D., chief executive officer of Day One. "These initial data will provide preliminary insights into the potential of tovorafenib to transform patient care for the most common childhood brain cancer, which currently has no approved therapies. Beyond FIREFLY-1, we are preparing to initiate a pivotal Phase 3 study, FIREFLY-2, for the first-line treatment of pLGG patients and recently initiated the combination portion of our Phase 2 FIRELIGHT-1 study with tovorafenib and our investigational oral MEK inhibitor, pimasertib. As our clinical programs advance, we continue to accelerate planning for our first potential regulatory submission for tovorafenib in 2023 and execute on our business strategy to make an impact for patients of all ages with life threatening diseases."

Program Highlights

Initial data from FIREFLY-1, a pivotal Phase 2 clinical trial of tovorafenib in relapsed pLGG, is expected in June 2022.

Day One anticipates releasing topline results from the fully-enrolled pivotal study in the first quarter of 2023. Pending positive results from FIREFLY-1, Day One anticipates filing a new drug application (NDA) with the U.S. Food and Drug Administration (FDA) in 2023.

Day One has expanded the FIREFLY-1 study to include two additional study arms:

An expanded access arm that enables treatment for eligible patients once the primary cohort has completed enrollment; and
An advanced solid tumor arm to evaluate the preliminary efficacy of tovorafenib in patients aged 6 months to 25 years with a relapsed or progressive extracranial solid tumors with activating RAF fusion.

Day One plans to initiate a pivotal Phase 3 clinical trial (FIREFLY-2) evaluating tovorafenib as a front-line therapy in pLGG in the second quarter of 2022.

Day One is enrolling patients in the Phase 2 FIRELIGHT-1 trial evaluating tovorafenib monotherapy in adults with recurrent, progressive, or refractory solid tumors harboring MAPK pathway aberrations. Day One recently expanded FIRELIGHT-1 to include a Phase 1b/2 portion to evaluate tovorafenib in combination with pimasertib, Day One’s investigational MEK inhibitor.
First Quarter 2022 Financial Highlights

Cash Position: Cash and cash equivalents totaled $262.7 million on March 31, 2022. Based on Day One’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into 2024.

R&D Expenses: Research and development expenses were $15.0 million for the first quarter of 2022 compared to $12.6 million for the first quarter of 2021. The increase was primarily due to additional employee compensation costs, clinical trial and product development expenses which were offset by a decrease in milestone payments for licensing agreements.

G&A Expenses: General and administrative expenses were $12.7 million for the first quarter of 2022 compared to $3.5 million for the first quarter of 2021. The increase was primarily due to additional employee compensation costs, initial commercial buildout, and professional expenses to support company growth.

Net Loss: Net loss totaled $27.7 million for the first quarter of 2022 compared to $16.1 million for the first quarter of 2021 with non-cash stock compensation expense of $6.2 million and $0.5 million for the first quarters of 2022 and 2021, respectively.
Upcoming Events

2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting
A trial-in-progress poster on Day One’s FIREFLY-1 pivotal study, abstract number TPS10062, will be presented at the ASCO (Free ASCO Whitepaper) Annual Meeting on Monday, June 6, 2022, from 8 to 11 a.m. CST.

The 20th International Symposium on Pediatric Neuro-Oncology (ISPNO) Annual Meeting
An educational exhibit on Day One’s pipeline will be displayed at Booth #F.05, at ISPNO’s Annual Meeting, which is being held June 12-15, 2022.
About Tovorafenib
Tovorafenib is an investigational, oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway, which is being investigated in primary brain tumors or brain metastases of solid tumors. Tovorafenib has been studied in over 250 patients to date. Currently tovorafenib is under evaluation in a pivotal Phase 2 clinical trial (FIREFLY-1) among pediatric, adolescent and young adult patients with pediatric low-grade glioma (pLGG), which is an area of considerable unmet need with no approved therapies. Tovorafenib is also being evaluated alone or as a combination therapy for adolescent and adult patient populations with recurrent or progressive solid tumors with MAPK pathway aberrations (FIRELIGHT-1). Tovorafenib has been granted Breakthrough Therapy and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pLGG harboring an activating RAF alteration. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma, and from the European Commission (EC) for the treatment of glioma.

About Pimasertib
Pimasertib is an investigational, oral, highly selective, small molecule inhibitor of mitogen‐activated protein kinases 1 and 2 (MEK-1/-2) within the MAPK signaling pathway. Pimasertib has been dosed in over 850 patients to date for various tumor types. Preclinical data indicates that the combination of a MEK inhibitor, such as pimasertib, and a type II RAF inhibitor, such as tovorafenib, has synergistic anti-tumor activity.

Day One is conducting a Phase 1b/2 study (FIRELIGHT-1) to evaluate the safety, tolerability, and preliminary efficacy of combining pimasertib with tovorafenib in adolescent and adult patients (≥12 years of age) with recurrent, progressive, or refractory solid tumors with MAPK pathway aberrations.

On May 12, 2022 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported financial results and recent corporate highlights for the first quarter ended March 31, 2022 (Press release, Mustang Bio, MAY 12, 2022, View Source [SID1234614361]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "Mustang started strong in 2022, with multiple data announcements and key clinical milestones achieved across our robust pipeline of CAR T cell and gene therapies. In the first quarter, data were presented on three of our CAR T cell therapies and our oncolytic virus clinical candidate at prestigious medical conferences by our academic collaborators at City of Hope and Fred Hutchinson Cancer Center ("Fred Hutch"). City of Hope’s Dr. Tanya Dorff presented Phase 1 clinical trial data on MB-105, our prostate stem cell antigen ("PSCA") chimeric antigen receptor ("CAR") T-cell therapy for treatment of PSCA-positive metastatic castration-resistant prostate cancer ("mCRPC") at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Genitourinary ("GU") Cancers Symposium that demonstrated its potential as a PSCA-targeted CAR T-cell therapy for prostate cancer and other solid tumors that express PSCA. Fred Hutch’s Dr. Mazyar Shadman presented updated interim Phase 1/2 clinical trial data on MB-106, our CD20-targeted, autologous CAR T cell therapy for treatment of relapsed or refractory B-cell non-Hodgkin lymphomas ("NHL") and chronic lymphocytic leukemia ("CLL"), at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy and Center for International Blood & Marrow Transplant Research ("2022 Tandem Meetings") and at the 4th International Workshop on CAR-T and Immunotherapies ("iwCAR-T"). The data demonstrated high efficacy and a favorable safety profile, with an overall response rate ("ORR") of 96% and complete response ("CR") rate of 72% observed in all patients across all dose levels. The data included two patients previously treated with CD19-directed CAR T therapy who relapsed and responded to MB-106, demonstrating its potential as an immunotherapy option for these patients. Additionally, City of Hope’s Dr. Christine Brown presented Phase 1 clinical trial data from a late-breaking poster regarding MB-108 (C134 oncolytic virus licensed from Nationwide Children’s Hospital) and MB‐101 (IL13Rα2‐targeted CAR T cell therapy licensed from City of Hope) for the treatment of recurrent glioblastoma ("rGBM"), at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Annual Meeting 2022 that support the safety of administering these two therapies sequentially to optimize treatment in a regimen designated as MB-109."

"Looking ahead, we have additional upcoming data presentations for MB-106 at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 ("EHA2022") Hybrid Congress and for our MB-107 gene therapy for the treatment of X-linked severe combined immunodeficiency ("XSCID") in newly diagnosed infants under the age of two at the American Society of Gene & Cell Therapy ("ASGCT") 25th Annual Meeting. We plan to dose the first patient under Mustang’s IND in a multicenter Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 for relapsed or refractory B-NHL and CLL at the end of this quarter. In the second half of 2022, we plan to file an IND to initiate a Phase 1 clinical trial for MB-109 for the treatment of rGBM and enroll the first patient in a pivotal multicenter Phase 2 clinical trial under Mustang’s IND to evaluate MB-107. It’s an incredibly exciting time for our team and we look forward to continuing to advance our cell and gene therapies to help meet the needs of patients with hard-to-treat diseases."

Financial Results:

●As of March 31, 2022, Mustang’s cash and cash equivalents and restricted cash totaled $123.2 million, compared to $110.6 million as of December 31, 2021, an increase of $12.6 million year-to-date.
●Research and development expenses were $16.3 million for the first quarter of 2022, compared to $11.6 million for the first quarter of 2021. Non-cash, stock-based expenses included in research and development were $0.5 million for the first quarter of 2022, compared to $0.7 million for the first quarter of 2021.
●General and administrative expenses were $3.3 million for the first quarter of 2022, compared to $3.5 million for the first quarter of 2021. Non-cash, stock-based expenses included in general and administrative expenses were $1.0 million for the first quarter of 2022, compared to $1.5 million for the first quarter of 2021.
●Net loss attributable to common stockholders was $19.8 million, or $0.20 per share, for the first quarter of 2022, compared to a net loss attributable to common stockholders of $15.0 million, or $0.19 per share, for the first quarter of 2021.
Recent Corporate Highlights:

●In February 2022, Mustang was selected as the Bronze winner for the Central region in the Eighteenth Annual Team Massachusetts Economic Impact Awards presented by MassEcon. The award winners were honored at Gillette Stadium on April 7, 2022.
●Also in February 2022, Phase 1 data on MB-105, a PSCA-targeted CAR T cell therapy administered systemically to patients with PSCA-positive mCRPC, were presented by City of Hope at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium. The data indicated that PSCA-targeted CAR T-cell therapy is feasible in patients with mCRPC with dose-limiting toxicity ("DLT") of cystitis and is associated with preliminary anti-tumor effect at a dose of 100 million cells plus lymphodepletion. It was concluded that escalation up to the next dose level of 300 million cells plus modified lymphodepletion can proceed in the trial.
●In March 2022, Mustang completed a $75 million long-term debt facility with Runway Growth Capital LLC (with $30.0 million funded on the closing date and the remaining $45.0 million becoming available if Mustang achieves certain predetermined milestones).
●In April 2022, Mustang announced that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell NHL and CLL, were presented at the 2022 Tandem Meetings. Data demonstrated high efficacy and a favorable safety profile in all patients (n=25). Five dose levels were used during the study, and complete responses were observed at all dose levels. Durable responses were observed in a wide range of hematologic malignancies including follicular lymphoma ("FL"), CLL, diffuse large B-cell lymphoma ("DLBCL"), and Waldenstrom macroglobulinemia. An ORR of 96% and CR rate of 72% was observed in all patients across all dose levels. Additionally, two patients had been previously treated with CD19-directed CAR T therapy and subsequently relapsed, and both responded to treatment, one patient with FL with a CR and the other with DLBCL with a partial response. A copy of the abstract can be viewed on the meeting website here. Mustang expects to dose the first patient in a multicenter Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 for relapsed or refractory B-NHL and CLL later this quarter.
●Also in April 2022, MB-106 data focused on CLL were presented at the 4th iwCAR-T.
●Additionally, in April 2022, Mustang announced its plan to file an IND in the second half of 2022 to initiate a Phase 1 clinical trial combining CAR T cells and an oncolytic virus for the treatment of rGBM, supported by interim data from two ongoing investigator-sponsored Phase 1 clinical trials evaluating two clinical candidates, MB‐101 (IL13Rα2‐targeted CAR T cell therapy licensed from City of Hope) and MB-108 (C134 oncolytic virus licensed from Nationwide Children’s Hospital). The data are from a late-breaking poster presented at the AACR (Free AACR Whitepaper) Annual Meeting 2022. Preclinical data also presented support the safety of administering these two therapies sequentially to optimize treatment in a regimen designated as MB-109.
●In May 2022, Mustang announced that MB-106 CD20-targeted CAR T therapy data were selected for an oral presentation at EHA (Free EHA Whitepaper)2022 taking place June 9 – 12 in Vienna. Dr. Mazyar Shadman of Fred Hutch will present updated interim data from the ongoing Phase 1/2 clinical trial for B-NHL and CLL. A copy of the abstract can be viewed online through the EHA (Free EHA Whitepaper)2022 website here.
●In May 2022, Mustang announced that interim Phase 1/2 data on MB-107, Mustang’s lentiviral gene therapy for the treatment of XSCID, also known as bubble boy disease, in newly diagnosed infants under the age of two, were selected for an oral presentation during the Clinical Trials Spotlight Symposium at the ASGCT (Free ASGCT Whitepaper) 25th Annual Meeting taking place May 16-19, 2022, both virtually and in Washington, D.C. The presentation will include updated data from a multicenter Phase 1/2 clinical trial for XSCID in newly diagnosed infants under the age of two at St. Jude Children’s Research Hospital, UCSF Benioff Children’s Hospital in San Francisco and Seattle Children’s Hospital. The abstract can be viewed on the meeting website here.
●In the second half of 2022, Mustang expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial under Mustang’s IND to evaluate MB-107, a lentiviral gene therapy for the treatment of infants under the age of two with XSCID.
●Mustang filed an IND application in December 2021 for its pivotal multicenter Phase 2 clinical trial of MB-207, a lentiviral gene therapy for the treatment of patients with XSCID who have been previously treated with hematopoietic stem cell transplantation ("HSCT") and for whom re-treatment is indicated. The trial is currently on hold pending CMC clearance from the FDA and, based on feedback from the Agency, Mustang Bio expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial in the first quarter of 2023.

On May 12, 2022 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported financial results for the first quarter of 2022 (Press release, CASI Pharmaceuticals, MAY 12, 2022, View Source [SID1234614360]).

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Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, "We are pleased to report $9.0 million in EVOMELA sales revenue for the first quarter of 2022. This is an increase of 58% compared to the same period last year. Our first commercial product, EVOMELA has been instrumental in treating patients with multiple myeloma prior to stem cell transplantation in China. We have achieved our goal for full-year 2021 EVOMELA revenue to reach 100% growth. Our achievements in 2021 provide a great foundation that we plan to build upon throughout 2022 and beyond."

Dr. He continued, "Our strategic focus in 2022 will continue to advance the development and commercialization of the portfolio. Through our partner Juventas, the CNCT-19 NDA submission to the National Medical Products Administration (NMPA) is on track, and we are in preparation for the anticipated launch in China; We expect the start of the BI-1206 phase I trial in China; We expect CB-5339 to receive Clinical Trial Application approval from the NMPA during 2022; Meanwhile, our CID-103’s Phase I study continues. We are excited by our momentum and will continue to execute on several key milestones across our broad portfolio in the quarters ahead."

First Quarter 2022 Financial Highlights

Revenues consist primarily of product sales of EVOMELA. Revenue was $9.0 million for the three months ended March 31, 2022, compared to $5.7 million for the three months ended March 31, 2021.
Costs of revenues were $3.8 million for the three months ended March 31, 2022, compared to $2.4 million for the three months ended March 31, 2021, which included royalty payment of $1.8 million and $1.1 million, respectively.
Research and development expenses for the three months ended March 31, 2022, were $4.0 million, compared with $5.3 million for the three months ended March 31, 2021.
General and administrative expenses for the three months ended March 31, 2022, were $5.3 million, compared with $5.5 million for the three months ended March 31, 2021.
Selling and marketing expenses for the three months ended March 31, 2022, were $3.3 million, compared with $2.7 million for the three months ended March 31, 2021.
As of March 31, 2022, CASI had cash and cash equivalent of $29.3 million.
Further information regarding the Company, including its Quarterly Report on Form 10-Q for the quarter ended March 31, 2022, can be found at www.casipharmaceuticals.com.

Conference Call

The conference call can be accessed by dialing 1-866-218-2402 (U.S.) or 1-412-902-6605 (international) and ask to be joined into the CASI Pharmaceuticals call to listen to the live conference call. Confirmation #: 10165613

This call will be recorded and available for replay by dialing 1-877-344-7529 (U.S.) or 1-412-317-0088 (international) and enter 2360248 to access the replay.

On May 12, 2022 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a precision oncology medicine company pioneering the discovery and development of MasterKey therapies, reported the publication of data identifying new oncogenic HER2 allosteric mutations that support the Mutation-Allostery-Pharmacology (MAP) discovery engine’s capabilities and further suggest the need for novel inhibitors to treat HER2-mutant cancers (Press release, Black Diamond Therapeutics, MAY 12, 2022, View Source [SID1234614359]). The paper, titled "Computational and Functional Analyses of HER2 Mutations Revealing Allosteric Activation Mechanisms and Altered Pharmacologic Effects" by Ishiyama et al. was published online by the American Association for Cancer Research (AACR) (Free AACR Whitepaper)’s Cancer Research Journal on May 3, 2022.

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"At the core of our precision medicine approach to cancer treatment is the ability to identify new, full spectrum oncogenic mutations. This study provides strong rationale for the power of our MAP discovery engine as we identified new oncogenic HER2 allosteric mutations that further suggest the need for novel treatment options. These findings support our overall approach to cancer treatment by demonstrating the value and importance of oncogenicity prediction, biological validation, protein conformation-based drug design and MasterKey inhibitor development against mutation families," said Elizabeth Buck, Chief Scientific Officer of Black Diamond Therapeutics. "In this study, Black Diamond’s proprietary MAP-scoring and functional validation analyses were able to provide new insights into the oncogenic activity and therapeutic targeting of HER2 mutations in cancer in addition to identifying 22 new oncogenic HER2 mutations. As the number of unique mutations across cancers is expected to rise over time, there remains a need for an effective means of identification of oncogenic mutations. We believe that our MAP technology has the potential to fill this gap and provide critical insights for the use of precision medicine to treat cancers driven by rare oncogenic mutations."

The peer-reviewed paper describes computational and functional analyses of HER2 mutations showing that Black Diamond’s MAP discovery engine has the ability to identify and experimentally validate 22 new oncogenic HER2 driver mutations. By applying its computational approach to 820 single-nucleotide variants, a list of 222 known mutations and potential driver mutations was produced. Of these 222 mutations, 37 HER2 mutations were experimentally determined to be driver mutations, comprised of 15 previously characterized and 22 newly identified oncogenic mutations. Black Diamond researchers found that these oncogenic mutations mostly affected allosteric sites in the extracellular domain (ECD), transmembrane domain (TMD), and kinase domain (KD) of HER2. In addition, Black Diamond was able to describe the unique pharmacological characteristics of these new HER2 driver mutations that render them susceptible to unique drug discovery screening strategy.