On November 8, 2022 OPKO Health, Inc. (NASDAQ: OPK) reported that business highlights and financial results for the three months ended September 30, 2022 (Press release, Opko Health, NOV 8, 2022, View Source [SID1234623396]).

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Business highlights for the third quarter of 2022 and subsequent weeks include the following:

Pfizer’s NGENLA (somatrogon) launched in Japan, Germany and other global markets; Pfizer seeking pricing approvals in additional European countries and continuing to work with FDA to obtain approval in the U.S. NGENLA treats pediatric patients with decreased growth due to insufficient growth hormone and reduces the injection frequency from once daily to once weekly. NGENLA is the first once weekly product approved for the treatment of pediatric growth hormone deficiency in Japan, Canada, Australia, Taiwan, UAE and Brazil. Its European Union marketing authorization is valid in all EU Member States as well as Iceland, Norway and Liechtenstein. In Australia, NGENLA is now offered through the Pharmaceutical Benefits Scheme, Australia’s national drug subsidy program.

ModeX Therapeutics, Inc. (ModeX) progresses proprietary immunotherapy pipeline. ModeX continues to advance its pipeline of antiviral and immune-oncology products utilizing its next generation multispecific antibodies. In addition, ModeX is pursuing potential licensing and collaboration arrangements with strategic partners for certain of its early stage pipeline candidates.

Innovation and cost cutting efforts continue at BioReference Laboratories (BRL). BRL continues significant cost reduction and business rationalization efforts following the decline of COVID testing volume. These efforts have resulted in a reduction of more than $100 million in annualized costs to date. Furthermore, BRL is exploring other revenue sources to work toward returning BRL to profitability.
Third Quarter Financial Results

Pharmaceuticals: Revenue in the third quarter of 2022 decreased to $32.4 million from $36.9 million in the third quarter of 2021, driven by foreign currency exchange and lower sales of RAYALDEE. During the third quarter of 2022, OPKO received gross profit share and royalty payments from Pfizer related to sales of NGENLA. We expect these payments to increase as Pfizer receives additional pricing approvals and launches the product in other geographies. Revenue from sales of RAYALDEE in the third quarter of 2022 was $6.9 million compared with $8.5 million in the prior-year period; however, we also began to receive royalty payments from sales of RAYALDEE by CSL Vifor in Germany and Switzerland during the third quarter of 2022. We expect these payments to increase as CSL Vifor launches RAYALDEE in additional territories throughout Europe. There were several significant events that positively impacted third quarter 2021 results, and should be considered when comparing those results to 2022. Revenue from the transfer of intellectual property was $4.5 million in the third quarter of 2022 compared with $8.8 million in the 2021 period. The third quarter of 2021 also included $4.9 million related to an exclusive license agreement with CAMP4 Therapeutics Corp. and $1.0 million due to the joint venture with LeaderMed Health Group Ltd. Total costs and expenses were $65.2 million in the third quarter of 2022 compared with $17.0 million in the prior-year period, which included a $31.5 million gain on the sale of OPKO’s fill-finish manufacturing facility in Ireland to Horizon Therapeutics. In addition, the increase in total costs and expenses in the third quarter of 2022 was primarily attributable to higher amortization expenses related to the reclassification of NGENLA’s in-process research and development upon its approval in Europe and Japan, higher employee expenses associated with the ModeX acquisition and higher research and development costs for somatrogon, partially offset by foreign currency exchange. The operating loss of $28.3 million in the third quarter of 2022 compared to operating income of $28.6 million in the third quarter of 2021 reflects these events.

Diagnostics: Revenue from services in the third quarter of 2022 was $142.8 million compared with $340.1 million in the prior-year period, the decrease primarily due to lower COVID-19 testing volume. BRL processed approximately 0.2 million COVID-19 PCR tests in the third quarter of 2022 versus 2.2 million tests in the third quarter of 2021. Total costs and expenses were $192.3 million in the third quarter of 2022 compared with $320.5 million in the third quarter of 2021, resulting in an operating loss of $49.5 million compared with operating income of $19.7 million in the 2021 period. BRL continues to implement significant cost-reduction initiatives and scaled back digital health investments as it looks to return to profitability following the buildup and then decline of COVID-related testing. The third quarter of 2022 included $5.1 million in severance costs as the company reduced employee costs on an annualized basis by over $46 million.

Consolidated: Consolidated total revenues for the third quarter of 2022 were $179.7 million compared with $385.8 million for the comparable period of 2021. Operating loss for the third quarter of 2022 was $87.8 million compared with operating income of $37.8 million for the 2021 quarter. Net loss for the third quarter of 2022 was $86.1 million, or $0.11 per share, compared with net income of $28.7 million, or $0.04 per diluted share, for the 2021 quarter. Net loss for the third quarter of 2022 included a non-cash expense of $30.6 million due to a decrease in the fair value of OPKO’s Sema4 investment, while the comparable quarter of 2021 included a $31.5 million gain on the sale of the facility in Ireland.

Cash and cash equivalents: Cash and cash equivalents were $180.8 million as of September 30, 2022.
Conference Call and Webcast Information

OPKO’s senior management will provide a business update, discuss third quarter financial results and answer questions during a conference call and live audio webcast today beginning at 4:30 p.m. Eastern time. Participants are encouraged to pre-register for the conference call using this link. Callers who pre-register will receive a unique PIN to gain immediate access to the call and bypass the live operator. Participants may register at any time, including up to and after the call start time. Those unable to pre-register may participate by dialing (833) 630-0584 (U.S.) or (412) 317-1815 (International). A webcast of the call can also be accessed at OPKO’s Investor Relations page and here.

A telephone replay will be available until November 15, 2022 by dialing (877) 344-7529 (U.S.) or (412) 317-0088 (International) and providing the passcode 5757952. A webcast replay will be available beginning approximately one hour after the completion of the live conference call here.

On November 8, 2022 Athenex (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, reported that management will participate in the 5th Annual Evercore ISI HealthCONx Conference, taking place virtually November 29 to December 1, 2022 (Press release, Athenex, NOV 8, 2022, https://ir.athenex.com/news-releases/news-release-details/athenex-participate-5th-annual-evercore-isi-healthconx [SID1234623395]).

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Dr. Johnson Lau, Chief Executive Officer of Athenex, and Dr. Dan Lang, President of Athenex Cell Therapy, will be participating in a fireside chat on Wednesday, November 30, 2022, beginning at 3:05pm Eastern Time. Athenex management will also be available for one-on-one investor meetings during the event. Please contact your representative at Evercore ISI to schedule a virtual one-on-one meeting with management during the conference.

On November 8, 2022 Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to transforming the lives of those affected by cancer, reported recent business progress and third-quarter 2022 financial results (Press release, Kronos Bio, NOV 8, 2022, View Source [SID1234623394]).

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In a press release issued earlier this afternoon, the company announced the prioritization of its clinical portfolio to focus on its next-generation SYK inhibitor, lanraplenib, and its CDK9 inhibitor, KB-0742, and the decision to discontinue the Phase 3 entospletinib trial. Kronos Bio believes that focusing on lanraplenib and KB-0742 will allow the company to direct its resources to the highest value programs and deliver on its mission of bringing cancer drugs to the patients with the greatest need.

Third Quarter and Recent Company Updates

Entospletinib
After a recent review of enrollment data that projected significant delays, Kronos Bio will discontinue the Phase 3 entospletinib trial and close enrollment in the fourth quarter of 2022. The trial is not being discontinued due to adverse events or lack of efficacy signals.
Lanraplenib
The company anticipates sharing initial data from the Phase 1b/2 study of lanraplenib in combination with gilteritinib in patients with relapsed/refractory FLT3-mutated acute myeloid leukemia (AML), along with a recommended Phase 2 dose (RP2D), in the fourth quarter of 2023 or first quarter of 2024.
KB-0742
Kronos Bio remains on track to report pharmacokinetic (PK), pharmacodynamic (PD) and safety data, as well as the RP2D, from the Phase 1/2 study of KB-0742 in solid tumors in the fourth quarter of 2022.
After reaching RP2D, the company plans to enroll two cohorts of patients in the next stage of the trial: patients with MYC-amplified solid tumors and patients with transcriptionally addicted cancers.
Initial KB-0742 efficacy data are anticipated in the second half of 2023.
Third Quarter 2022 Financial Highlights

Cash, Cash Equivalents and Investments: With its ongoing and currently planned clinical programs and $270.3 million in cash, cash equivalents and investments as of September 30, 2022, the company anticipates sufficient resources to fund its planned operations into the second quarter of 2025.

R&D Expenses: Research and development expenses were $23.4 million for the third quarter of 2022, which includes non-cash stock-based compensation expense of $3.5 million.

G&A Expenses: General and administrative expenses were $10.1 million for the third quarter of 2022, which includes non-cash stock-based compensation expense of $4.0 million.

Net Loss: Net loss for the third quarter of 2022 was $32.3 million, or $0.57 per share, including non-cash stock-based compensation expense of $7.5 million.

On November 8, 2022 Omega Therapeutics, Inc. (Nasdaq: OMGA) ("Omega"), a clinical-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as a new class of programmable epigenetic medicines by leveraging its OMEGA Epigenomic Programming platform, reported financial results for the third quarter ended September 30, 2022 and highlighted recent Company progress (Press release, Omega Therapeutics, NOV 8, 2022, View Source [SID1234623393]).

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"The significant progress we are making on all fronts across our development pipeline is exciting, including the initiation of the MYCHELANGELO clinical program for OTX-2002, which represents the first-ever dosing of an epigenomic controller in a patient and marks a significant milestone in our journey to bring novel and programmable mRNA therapeutics to patients," said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. "We were also delighted to announce our next development candidate, OTX-2101 for the treatment of patients with MYC-driven non-small cell lung cancer. Our focus is on advancing our lead programs, OTX-2002 and OTX-2101, as well as driving additional discovery and preclinical assets forward to further expand our pipeline."

Recent Corporate Highlights

Development Pipeline and Platform

First Patient Dosed in Landmark MYCHELANGELO I Clinical Trial for OTX-2002, the First-Ever Omega Epigenomic Controller (OEC): The Phase 1/2 study is the first-ever study to evaluate this new class of programmable mRNA therapeutics designed to treat or cure serious diseases through precision genomic control. The study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of OTX-2002 as a monotherapy (Part 1) and in combination with standard of care therapies (Part 2) in patients with relapsed or refractory hepatocellular carcinoma (HCC) and other solid tumor types known for association with the c-Myc (MYC) oncogene. The study is expected to enroll approximately 190 patients at clinical trial sites in the United States, Asia, and Europe.

OTX-2002 Granted Orphan Drug Designation by U.S. Food and Drug Administration (FDA) for Hepatocellular Carcinoma (HCC): OTX-2002 is a rationally engineered, novel and programmable mRNA therapeutic designed to downregulate MYC expression pre-transcriptionally through epigenetic modulation while potentially overcoming MYC autoregulation. The FDA’s Orphan Drug Designation Program provides orphan status to drugs intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States.

OTX-2101 for MYC-Driven Non-Small Cell Lung Cancer (NSCLC) Selected as Second Omega Epigenomic Controller Development Candidate: OTX-2101 is the second candidate in this new class of programmable mRNA therapeutics designed to downregulate MYC expression pre-transcriptionally through epigenetic modulation while potentially overcoming MYC autoregulation. Preclinical data presented at the 2022 American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting demonstrated OTX-2101 potently downregulates MYC in multiple NSCLC cell lines. OTX-2101 effectively reduced tumor growth in vivo and was well tolerated in murine xenograft models, further supporting its clinical potential. Investigational New Drug (IND)-enabling studies for OTX-2101 are underway.

Completed Development Candidate-enabling Activities for Several OECs: Beyond HCC and NSCLC, the Company continues to advance multiple OECs from the OMEGA Epigenomic Programming platform through preclinical studies. The CXCL 1-8-targeting OEC has been characterized in preclinical studies and has potential in several indications including neutrophilic asthma, acute respiratory distress syndrome (including COVID-related), oncology, and dermatological and rheumatological indications, representing a potential franchise opportunity. The Company continues additional preclinical work for its OEC development programs spanning oncology, multigenic diseases including immunology, regenerative medicine, and select monogenic diseases.
Corporate

Rainer Boehm Appointed to Board of Directors: Mr. Boehm joined the Board on August 30, 2022. He serves on the Company’s audit and compensation committees. He brings over 30 years of successful and diverse clinical, managerial, drug development, and commercialization experience to Omega.
Third Quarter 2022 Financial Results

As of September 30, 2022, the Company had cash, cash equivalents and marketable securities totaling $148.3 million.

Research and development (R&D) expenses for the third quarter of 2022 were $20.7 million, compared to $12.3 million for the third quarter of 2021. The $8.4 million increase in R&D expense was primarily driven by an increase in personnel-related expenses, external manufacturing costs, and study costs in support of the advancement of our programs.

General and administrative (G&A) expenses for the third quarter of 2022 were $5.2 million, compared to $4.5 million for the third quarter of 2021. The $0.7 million increase in G&A expense was primarily driven by an increase in personnel-related expenses to support business growth.

Net loss for the third quarter of 2022 was $25.8 million, compared to $18.5 million for the third quarter of 2021, driven predominantly by increased R&D and G&A expenses to support the Company’s growth and operations as a public company.

On November 8, 2022 ALX Oncology Holdings Inc., ("ALX Oncology") (Nasdaq: ALXO), a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, reported financial results for the third quarter ended September 30, 2022 and provided clinical development and operational highlights (Press release, ALX Oncology, NOV 8, 2022, View Source [SID1234623392]).

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"We continued to expand the clinical development of our lead program, evorpacept, during the third quarter of 2022, with the announcement of a new investigational treatment arm in the I-SPY-P1 TRIAL for the treatment of patients with unresectable or metastatic HER2-positive and HER2-low breast cancer in partnership with Quantum Leap Healthcare Collaborative," said Jaume Pons, Ph.D., Founder, President and Chief Executive Officer of ALX Oncology. "With our recently announced non-dilutive term loan facility expected to extend our cash runway to mid-2025, we are focused on the advancement of evorpacept through multiple clinical milestones over the next two years."

Recent Clinical Developments for Evorpacept

Abstract Data for Phase 1 Dose Escalation Portion of ASPEN-05 Study in acute myeloid leukemia ("AML") Released as Part of the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") Annual Meeting
In November 2022, ALX Oncology’s ASH (Free ASH Whitepaper) abstract was released online with initial results of the Phase 1 dose escalation portion of ASPEN-05, a Phase 1/2 clinical trial of evorpacept in combination with venetoclax and azacitidine for the treatment of patients with AML. Session 616: Poster III; Publication Number: 4076.
The results presented were as of July 8, 2022. 14 subjects were treated at evorpacept doses of 20 mg/kg Q2W (N=4), 30 mg/kg Q2W (N=4), and 60 mg/kg Q4W (N=6). The addition of evorpacept to standard dose venetoclax and azacitidine for AML was well tolerated with no maximum tolerated dose reached. Preliminary dose-proportional pharmacokinetics was seen along with full CD47 target occupancy in both peripheral blood and bone marrow across all dose levels evaluated. Initial anti-leukemic activity was observed in subjects with both newly diagnosed and relapse refractory AML. These initial results support further evaluation of evorpacept in myeloid malignancies, including AML.
A poster presentation from the dose escalation portion of ASPEN-05 with updated results will be presented at the ASH (Free ASH Whitepaper) Annual Meeting on Monday, December 12, 2022, 6:00pm to 8:00pm CT, at the Ernest N. Morial Convention Center, Hall D, New Orleans, LA.
Trials in Progress Abstracts Related to Data from ASPEN-03 and ASPEN-04 Clinical Trials Accepted for Poster Presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) ("SITC") 37th Annual Meeting
In October 2022, ALX Oncology announced the acceptance of two Trials in Progress abstracts related to ASPEN-03 and ASPEN-04, the Company’s Phase 2 head and neck squamous cell carcinoma ("HNSCC") studies which will be presented at SITC (Free SITC Whitepaper) in Boston, MA, from November 10–11, 2022 (abstracts 678 and 676).
ALX Oncology continues to advance ASPEN-03 and ASPEN-04, which are two distinct randomized Phase 2 studies for the treatment of patients with advanced HNSCC in combination with KEYTRUDA (pembrolizumab) with or without chemotherapy. Patient enrollment for ASPEN-03 and ASPEN-04 continues as planned with results expected to be presented by the middle of 2024.
Clinical Trial Agreement in Combination with ENHERTU (Fam-trastuzumab deruxtecan-nxki) Entered with Quantum Leap
In August 2022, ALX Oncology entered into a collaboration and supply agreement with Quantum Leap Healthcare Collaborative ("Quantum Leap") to evaluate evorpacept for a new investigational treatment arm in the I-SPY-P1 TRIAL for the treatment of patients with unresectable or metastatic HER2-positive and HER2-low breast cancer.
Sponsored by Quantum Leap, this Phase 1 (open-label), multi-center study arm will investigate evorpacept in combination with ENHERTU (fam-trastuzumab deruxtecan-nxki), a HER2 directed antibody-drug conjugate, to determine the safety, tolerability and efficacy of this drug combination.
First Patient Dosed in Phase 2 Investigator-sponsored Trial of Evorpacept in Combination with ERBITUX (Cetuximab) and KEYTRUDA (Pembrolizumab) in Patients with Advanced Colorectal Cancer
In August 2022, ALX Oncology announced the initiation of a Phase 2 investigator-sponsored study of evorpacept in combination with ERBITUX (cetuximab) and KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, in patients with refractory microsatellite stable metastatic colorectal cancer who have progressed on at least two lines of systemic therapy. This trial, managed by Criterium, Inc., is led by the Academic GI Cancer Consortium.
Additional Clinical Program Updates for Evorpacept
In June 2022, ALX Oncology announced the initiation of ASPEN-07, a Phase 1 trial of evorpacept for the treatment of patients with urothelial cancer ("UC"), with the first patient expected to be dosed in the fourth quarter of 2022. ASPEN-07 will investigate evorpacept in combination with an antibody drug conjugate, PADCEV (enfortumab vedotin-ejfv), for the treatment of patients with UC.
ALX Oncology continues to advance ASPEN-06, a randomized Phase 2 (open-label) / Phase 3 (double-blind), international, multi-center study to evaluate the efficacy of evorpacept and ramucirumab added to trastuzumab and paclitaxel for the treatment of patients with HER-positive gastric cancer or gastroesophageal junction cancer whose tumors have progressed following treatment with HER2-targeted therapy and chemotherapy. ASPEN-06 is being conducted in collaboration with Eli Lilly and Company. Patient enrollment continues to progress and results from the Phase 2 portion of ASPEN-06 are expected to be presented in 2023.
Recent Corporate Updates

Loan Facility Agreement with Oxford Finance LLC and Silicon Valley Bank of up to $100 Million of Non-dilutive Financing
In October 2022, ALX Oncology drew $10 million of an initial $50 million tranche at closing, with the remaining $40 million available at its discretion through the end of 2023. ALX Oncology also has access up to an additional $50 million with $12.5 million available in each of two tranches based upon the achievement of milestones related to the development of evorpacept and one pre-clinical product candidate, and $25 million available at the lenders’ discretion.
Third Quarter 2022 Financial Results:

Cash, Cash Equivalents and Investments: Cash, cash equivalents and investments were $293.1 million as of September 30, 2022. ALX Oncology believes its cash, cash equivalents, investments and the ability to draw down up to $50 million of its term loan are sufficient to fund planned operations through mid-2025.
Research and Development ("R&D") Expenses: These expenses for the three months ended September 30, 2022 were $29.4 million, compared to $18.2 million for the prior-year period. Research and development expenses increased by $11.2 million during the three months ended September 30, 2022 compared to the three months ended September 30, 2021. The increase was primarily attributable to an increase of $6.0 million in clinical and development costs primarily due to clinical costs from an increase in the number of active trials and patient enrollment as well as manufacturing of clinical trial materials to support a higher number of active clinical trials and future expected patient enrollment related to the advancement of our lead product candidate, as well as expenses related to the Tallac Collaboration for costs related to the IND filing planned for 2023, an increase of $0.9 million in preclinical costs primarily related to development of new targets, an increase of $2.0 million in personnel and related costs primarily due to an increase driven by headcount growth and a portion of a retention bonus payable to ScalmiBio stockholders, an increase of $1.4 million in stock-based compensation expense due to additional awards granted since September 30, 2021 and an increase of $0.9 million in other research costs due primarily to an increase in facility costs related to the expansion of our new laboratory space.
General and Administrative ("G&A") Expenses: These expenses for the three months ended September 30, 2022 were $7.3 million, compared to $6.4 million for the prior-year period. General and administrative expenses increased by $0.9 million during the three months ended September 30, 2022 compared to the three months ended September 30, 2021. The increase was primarily attributable to an increase of $0.6 million in stock-based compensation expense due to additional stock option awards granted since September 30, 2021 and an increase in facility and information technology costs.
Net loss: GAAP net loss was $35.3 million for the third quarter ended September 30, 2022, or $0.87 per basic and diluted share, as compared to a net loss of $24.6 million for the third quarter ended September 30, 2021, or $0.61 per basic and diluted share. Non-GAAP net loss was $29.1 million for the third quarter ended September 30, 2022, as compared to a net loss of $20.4 million for the third quarter ended September 30, 2021. A reconciliation of GAAP to non-GAAP financial results can be found at the end of this news release.