On February 23, 2023 OPKO Health, Inc. (NASDAQ: OPK) reports business highlights and financial results for the three months ended December 31, 2022 (Press release, Opko Health, FEB 23, 2023, View Source [SID1234627624]).

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Business highlights include the following:

Sales are underway by Pfizer for NGENLA (somatrogon) in 15 countries including Japan, Germany and the United Kingdom; Pfizer expects to launch in all priority ex-U.S. markets by year-end and is continuing to work with the 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, United Arab Emirates and Brazil. Its European Union marketing authorization is valid in all EU Member States, as well as in Iceland, Norway and Liechtenstein.

BioReference Health advances its cost-cutting initiatives. BioReference continues to implement significant initiatives to reduce costs and rationalize its business following the decline of COVID-19 testing volume. In addition, BioReference is driving innovation to its portfolio and exploring other revenue sources to return the business to profitability.
Fourth Quarter Financial Results

Pharmaceuticals: Revenue from products in the fourth quarter of 2022 increased to $37.9 million from $35.3 million in the fourth quarter of 2021, driven by sales in our international operating companies and revenue from sales of RAYALDEE. Revenue from sales of RAYALDEE in the fourth quarter of 2022 was $9.1 million compared with $7.7 million in the prior-year period. Revenue from the transfer of intellectual property was $8.1 million in the fourth quarter of 2022 compared with $3.2 million in the 2021 period. Furthermore, during the fourth quarter of 2022, OPKO received $2.5 million from Nicoya, tied to the first anniversary of the agreement’s effective date. Total costs and expenses were $68.0 million in the fourth quarter of 2022 compared with $53.3 million in the prior-year period. The increase 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 the impact of foreign exchange at our international operating companies. The operating loss was $22.0 million in the fourth quarter of 2022 compared with an operating loss of $14.8 million in the fourth quarter of 2021.

Diagnostics: Revenue from services in the fourth quarter of 2022 was $139.4 million compared with $362.8 million in the prior-year period. The decrease was primarily due to lower COVID-19 testing volume. BioReference processed approximately 0.1 million COVID-19 PCR tests in the fourth quarter of 2022 versus 2.7 million tests in the fourth quarter of 2021. Total costs and expenses were $162.5 million in the fourth quarter of 2022 compared with $381.4 million in the fourth quarter of 2021, resulting in an operating loss of $23.1 million compared with an operating income of $18.6 million in the 2021 period. BioReference continues to implement significant cost-reduction initiatives and has scaled back its digital health investments as it looks to return to profitability following the decline of COVID-19 testing.

Consolidated: Consolidated total revenues for the fourth quarter of 2022 were $185.4 million compared with $401.3 million for the comparable period of 2021. The operating loss for the fourth quarter of 2022 was $55.3 million compared with an operating loss of $63.1 million for the 2021 quarter. Net loss for the fourth quarter of 2022, which included a non-cash expense of $49.1 million due to a decrease in the fair value of OPKO’s GeneDx Holdings (formerly Sema4 Holdings Corp.) investment, was $85.2 million, or $0.11 per share, compared with a net loss of $73.8 million, or $0.11 per share, for the 2021 quarter. Net loss for the fourth quarter of 2021 included non-recurring legal expenses and expenses related to the sale of GeneDx, Inc.

Cash and cash equivalents: Cash and cash equivalents were $153.2 million as of December 31, 2022.
Conference Call and Webcast Information

OPKO’s senior management will provide a business update, discuss fourth quarter financial results, provide financial guidance 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 here. 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 March 2, 2023 by dialing (877) 344-7529 (U.S.) or (412) 317-0088 (International) and providing the passcode 3187296. A webcast replay will be available beginning approximately one hour after the completion of the live conference call here.

On February 23, 2023 VBL Therapeutics (Nasdaq:VBLT), a biotechnology company developing targeted medicines for immune-inflammatory diseases, and Notable Labs, Inc. ("Notable"), a clinical stage therapeutic platform company developing predictive precision medicines for cancer patients, reported they have entered into a definitive merger agreement (Press release, VBL Therapeutics, FEB 23, 2023, View Source [SID1234627620]). The combined company will focus on the advancement of Notable’s proprietary Predictive Precision Medicines Platform ("PPMP") and therapeutic pipeline focused on cancer patients with high unmet medical needs. Immediately after the merger, Notable stockholders are expected to own approximately 76% and VBL Therapeutics shareholders are expected to own approximately 24% of the combined company, each on a fully-diluted basis and subject to adjustment. The combined company is expected to operate under the name Notable Labs, Ltd. with its shares listed on the Nasdaq Capital Market under the ticker symbol ‘NTBL.’

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"Following an extensive process to identify the best possible partner, we are very encouraged to have arrived at this proposed merger with Notable," remarked Prof. Dror Harats, M.D., Chief Executive Officer of VBL Therapeutics. "The caliber and track record of Notable’s management and their deep industry experience further adds to our excitement about developing Volasertib with Notable’s predictive platform technology to create value for our shareholders."

"The operational infrastructure, intellectual resources and investment capital that this merger brings will serve as both the foundation and a driver of potential best-in-class therapeutic assets out of our PPMP platform," commented Dr. Thomas A. Bock, Chief Executive Officer of Notable. "We are excited about the tremendous support of Notable’s top-tier investors including Builders VC, B Capital Group, Y Combinator, First Round Capital, and Founders Fund, further fueling our ambition to revolutionize oncology drug development with our ability to predict patient responses. This merger will serve to advance our lead clinical program with Volasertib, while in parallel accelerating our pursuit of additional targeted in-licensing opportunities."

Notable’s PPMP combines multi-dimensional biological assays and machine learning to bio-simulate a patient’s cancer treatment and predict their clinical response to the actual treatment. Three clinical validation studies with recognized academic centers have demonstrated PPMP’s high precision in accurately predicting clinical responders.

PPMP has guided Notable in the selection and development of two clinical-stage therapeutic candidates in platform-predicted responding patients with AML. Notable’s lead asset derived from PPMP is Volasertib, a highly potent PLK1 inhibitor proven to induce cell cycle arrest and apoptosis in various cancer cells. Phase 2a results for Volasertib in adult AML are expected in 3Q 2024. Notable expects to use PPMP to identify, in-license, and fasttrack additional undervalued assets as it builds out its development pipeline.

Concurrent with the execution of the merger agreement, a healthtech-focused investor syndicate, including existing Notable stockholders Builders VC, B Capital Group, Y Combinator, First Round Capital, and Founders Fund, have entered into a definitive securities purchase agreement with Notable to invest $10.3 million in gross proceeds prior to the close of the proposed merger. After completion of the merger, the combined company is expected to be capitalized with a cash runway into 2025.

About the Proposed Merger

Under the terms of the merger agreement, pending approval of the transaction by VBL Therapeutics’ shareholders and Notable’s stockholders, Notable will merge with a wholly-owned Delaware subsidiary of VBL Therapeutics, and stockholders of Notable will receive newly issued ordinary shares. Immediately after the merger, Notable stockholders are expected to own approximately 76% and VBL Therapeutics shareholders are expected to own approximately 24% of the combined company, in each case on a fully diluted basis as set forth in the merger agreement. The percentage of the combined company that the respective companies’ shareholders will own after completion of the merger is subject to adjustment based on the amount of VBL Therapeutics’ net cash at the closing date and the net proceeds from Notable’s pre-merger financing, among other adjustments, in each case as described in the merger agreement.

The merger agreement has been unanimously approved by the Board of Directors of each company. The merger is expected to close in the second quarter of 2023, subject to receipt of necessary approvals by the stockholders of each company and other customary closing conditions.

Chardan is serving as exclusive financial advisor to VBL Therapeutics and Goodwin Procter LLP and Horn & Co. are serving as legal counsel to VBL Therapeutics. JMP Securities, a Citizens Company, is serving as exclusive financial advisor for Notable and Wiggin and Dana LLP and Meitar are serving as legal counsel to Notable.

Management and Organization

The combined company will be led by Notable’s current management team and will be headquartered in Foster City, California. The Board of Directors of the combined company is expected to consist of up to seven members, one of which will be designated by VBL Therapeutics and the remainder of which will be designated by Notable.

Conference Call and Webcast Information

VBL Therapeutics and Notable will host a conference call and webcast today at 8:30 AM Eastern Time. The call can be accessed by dialing (877) 407-9208 (U.S. and Canada) or (201) 493-6784 (international) and entering passcode 13736622. A link to the live webcast, including the presentation of corporate slides, may be found here. To access a subsequent archived recording, visit the "Events & Presentations" section of the VBL website at www.vblrx.com, or the "News & Events" section of the Notable website at www.notablelabs.com

On February 23, 2023 Morphic Therapeutic (Nasdaq: MORF), a biopharmaceutical company developing a new generation of oral integrin therapies for the treatment of serious chronic diseases, reported corporate highlights and financial results for the full year 2022 (Press release, Morphic Therapeutic, FEB 23, 2023, View Source [SID1234627619]).

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2022 and Recent Corporate Highlights

EMERALD phase 2 trials of MORF-057 in ulcerative colitis

•Completed enrollment of the EMERALD-1 open-label phase 2a trial of MORF-057 in patients with moderate to severe ulcerative colitis (UC)
◦Study met target enrollment in the third quarter of 2022
◦EMERALD-1 topline results expected in the second quarter of 2023 including primary endpoint (change in Roberts Histopathological Index (RHI)), key secondary measures including modified mayo clinical score (mMCS), safety and pharmacokinetic data
•Initiated EMERALD-2 phase 2b study of MORF-057 in patients with moderate to severe UC
◦EMERALD-2 is a global phase 2b randomized, double-blind, placebo-controlled trial of MORF-057 in patients with moderate-to-severe ulcerative colitis
◦The primary endpoint of EMERALD-2 is clinical remission rate as measured by mMCS at 12 weeks and is expected to report in the first half of 2025

MORF-057 Preclinical and Phase 1 Studies

•Presented new MORF-057 preclinical and clinical data at European Congress for Crohn’s and Colitis, the United European Gastroenterology Week and the American College of Gastroenterology further supporting the ongoing EMERALD phase 2 trials in UC

Integrin Science and Technology

•Celebrated the publication in the scientific journal Cell by Albert Lin, PhD and Timothy Springer, PhD, describing critical insights into the function of integrins, a central biologic receptor class, based on conformation changes
◦The discovery made by Drs. Lin and Springer led directly to the foundation of Morphic Therapeutic
◦Dr. Springer is the Latham Family Professor at Harvard Medical School and recipient of the 2022 Lasker Prize for his pioneering work with integrins, and a founder, Director, and SAB member of Morphic
◦Dr. Lin is Executive Director of Biology at Morphic and a founder of Morphic

Morphic Operations

•Strengthened the Morphic team across research, regulatory, clinical, and executive functions
◦Key additions in 2022 included: the appointment of Dr Brihad Abhyankar as Vice President, Clinical Operations. Dr. Abhyankar was recently promoted to Senior Vice President, Clinical Development, the appointment of Joanne Gibbons as Senior Vice President of Regulatory Affairs. Ms. Gibbons was recently promoted to Senior Vice President, Regulatory Affairs and Quality

"Morphic executed well against our corporate objectives in 2022 which has positioned us ideally for a strong 2023, a key year in the clinical development of MORF-057, our oral a4b7 inhibitor in phase 2 studies for UC", commented Praveen Tipirneni, Chief Executive Officer of Morphic. "We are looking forward to the EMERALD-1 readout in the second quarter while advancing our earlier stage pipeline across multiple therapeutic areas including pulmonary hypertensive diseases, myelofibrosis, and beyond."

Financial Results for the Full Year 2022

•Net loss for the year ended December 31, 2022, was $59.0 million or $1.55 per share compared to a net loss of $95.5 million or $2.67 per share for the year ended December 31, 2021.
•Revenue was $70.8 million for the year ended December 31, 2022, compared to $19.8 million for the year ended December 31, 2021. The change was primarily due to recognition of revenue due to the conclusion of the AbbVie collaboration and reduction in the scope of the Janssen collaboration.
•Research and development expenses were $102.1 million for the year ended December 31, 2022, as compared to $87.8 million for the year ended December 31, 2021. The increase was primarily attributable to higher manufacturing and development costs along with clinical trial costs to support our lead product candidate MORF-057.
•General and administrative expenses were $32.1 million for the year ended December 31, 2022, compared to $27.8 million for the year ended December 31, 2021. The increase was due to increased headcount and higher professional and consulting costs associated with ongoing business development activities and Morphic operating as a public company.

As of December 31, 2022, Morphic had cash, cash equivalents and marketable securities of $348.2 million, compared to $408.1 million as of December 31, 2021. In 2022, Morphic raised net proceeds of $39.2 million from the use of our At-The Market (ATM) facility through its use on a single day in the second quarter. To date in 2023, Morphic has not issued any stock through its ATM facility. On February 13, 2023, the Company entered into a securities purchase agreement pursuant with existing investors for a private placement where the Company agreed to sell and issue 848,655 shares of its common stock at a price of $35.35 per share and pre-funded warrants to purchase 1,980,198 shares of common stock at a purchase price of $35.3499 for an aggregate net proceeds of approximately $100.0 million. We believe that our cash, cash equivalents and marketable securities of $348.2 million as of December 31, 2022, together with the $100.0 million raised in our private issuance of common stock and pre-funded warrants in February 2023, will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2026.

About MORF-057

Morphic is developing MORF-057 as a selective, oral small molecule inhibitor of the α4β7 integrin for patients with inflammatory bowel disease (IBD). α4β7 has been clinically validated as a target for the treatment of IBD by the success of the approved injectable antibody therapeutic vedolizumab. MORF-057, like vedolizumab, is designed to block the interactions between α4β7 on the surface of lymphocytes and the mucosal endothelial cell ligand MAdCAM-1, substantially reducing lymphocyte migration from the bloodstream into intestinal mucosal tissues and avoiding inflammation that is associated with IBD.

About the EMERALD-1 Study

EMERALD-1 (MORF-057-201) is an open-label multi-center phase 2a trial designed to evaluate the efficacy, safety, and tolerability of MORF-057 in adults with moderate to severe ulcerative colitis. EMERALD-1 has completed enrollment of the main cohort of 30-35 patients who will be treated with 100 mg BID (twice daily) at sites in the United States and Europe. The primary endpoint of the trial is the change in Robarts Histopathology Index (RHI), a validated instrument that measures histological disease activity in ulcerative colitis at 12 weeks compared to baseline. Patients will then continue for an additional 40 weeks of maintenance therapy followed by a 52-week assessment. Secondary and additional outcome measures in the EMERALD-1 study include change in the modified Mayo clinic score, safety, pharmacokinetic parameters and key pharmacodynamic measures including α4β7 receptor occupancy and lymphocyte subset trafficking.

About the EMERALD-2 Study

EMERALD-2 (MORF-057-202) is a global phase 2b randomized, double-blind, placebo-controlled trial of MORF-057 that is currently enrolling patients with moderate-to-severe ulcerative colitis. The primary endpoint of EMERALD-2 is clinical remission rate as measured by the Modified Mayo Clinic Score (mMCS) at 12 weeks. EMERALD-2 will also measure several secondary and exploratory endpoints based on the mMCS as well as histologic, pharmacokinetic and pharmacodynamic measures, and safety parameters. Patients in the EMERALD-2 study will be randomized to receive either 200 mg BID (twice daily) MORF-057, 100 mg BID MORF-057, a QD (once daily) dose of MORF-057, or a placebo dose. Following the 12-week induction phase, all patients will receive MORF-057 for 40 weeks of maintenance dosing. For more information about the EMERALD clinical trials of MORF-057, please click here.

On February 23, 2023 MannKind Corporation (Nasdaq: MNKD) reported financial results for the fourth quarter and full year ended December 31, 2022 (Press release, Mannkind, FEB 23, 2023, View Source [SID1234627618]).

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"For 2022, we recognized almost $100 million in total revenues – an incredible triumph for our entire organization," said Michael Castagna, PharmD, Chief Executive Officer of MannKind Corporation. "With our revenues growing nicely, we are focusing on our product pipeline where our INHALE-1 Phase 3 trial for Afrezza in pediatrics reached 50% enrollment at December 31, 2022 and our inhaled clofazimine will move into an adaptive Phase 2/3 study in the second half of 2023."

Fourth Quarter 2022 Results

Revenue Highlights

For the Three Months Ended

December 31,

2022

2021

$ Change

% Change

Net revenue — Afrezza

$

12,006

$

11,340

$

666

6

%

Net revenue — V-Go

5,434

$

5,434

n/a

Revenue — collaborations and services

9,544

1,175

$

8,369

712

%

Royalties — collaborations

9,075

$

9,075

n/a

Total revenues

$

36,059

$

12,515

$

23,544

188

%

The increase in Afrezza net revenue was mainly a result of higher demand. V-Go was acquired in the second quarter of 2022. The increase in collaborations and services revenue reflected the fact that the manufacture of Tyvaso DPI for commercial sale had not yet commenced in the previous period. Royalties from United Therapeutics ("UT") for Tyvaso DPI continued to grow based on strong patient demand.

Afrezza gross margin in the fourth quarter of 2022 was 92% compared to 62% for the same period in 2021, mainly due to a decrease in cost of goods sold driven by lower inventory write offs, lower cost of production per unit and a higher amount of manufacturing activity in 2022 (which absorbed more cost to inventory). V-Go gross margin was 42% for the fourth quarter of 2022.

Cost of revenue – collaborations and services for the fourth quarter of 2022 was $12.0 million compared to $7.1 million for the same period in 2021, an increase of $4.9 million, primarily due to an increase in manufacturing activities associated with the production of Tyvaso DPI.

Research and development ("R&D") expenses for the fourth quarter of 2022 were $7.2 million compared to $3.9 million for the same period in 2021. This $3.3 million increase was primarily attributed to costs incurred to develop our product pipeline, including the Afrezza pediatrics clinical study (INHALE-1) and MNKD-101 (inhaled clofazimine).

Selling expenses for the fourth quarter of 2022 were $11.6 million compared to $13.5 million for the same period in 2021. This $1.9 million decrease was primarily attributable to the net impact of lower personnel-related costs associated with the first quarter of 2022 Afrezza sales force restructuring, and a pilot promotional effort aimed at primary care physicians that began in the fourth quarter of 2021 and ended in the third quarter of 2022, partially offset by V-Go promotional efforts.

General and administrative ("G&A") expenses for the fourth quarter of 2022 were $10.5 million compared to $9.2 million for the same period in 2021. This $1.3 million increase was primarily attributable to higher stock-based compensation and increased headcount.

Interest expense on financing liability was $2.5 million for the fourth quarter of 2022 and represented interest incurred on the sale lease-back transaction for our manufacturing facility in Danbury, CT, which was entered into in the fourth quarter of 2021.

Interest expense on notes was flat in the fourth quarter of 2022 compared to the same period in 2021 due to fixed interest rates and no changes in debt balances.

Year Ended December 31, 2022

Revenue Highlights

For the Year Ended

December 31,

2022

2021

$ Change

% Change

Net revenue — Afrezza

$

43,316

$

39,168

$

4,148

11

%

Net revenue — V-Go

12,931

—

$

12,931

n/a

Revenue — collaborations and services

27,924

36,274

$

(8,350

)

(23

%)

Royalties — collaborations

15,599

$

15,599

n/a

Total revenues

$

99,770

$

75,442

$

24,328

32

%

Afrezza net revenue increased year-over-year primarily due to higher price (including a more favorable gross-to-net adjustment), higher product demand, and a more favorable cartridge mix. Collaborations and services revenue decreased, primarily due to the completion of the R&D services associated with our collaboration with UT, which was partially offset by revenues associated with the manufacturing of Tyvaso DPI. As of December 31, 2022, $37.9 million of manufacturing revenue associated with Tyvaso DPI remains deferred and will be recognized as commercial product is sold to UT.

Afrezza gross margin for 2022 was 80% compared to 57% for the same period in 2021, driven primarily by a decrease in excess manufacturing capacity costs (as Tyvaso DPI was in commercial production in 2022), a decrease in inventory write offs and an amendment fee associated with our Insulin Supply Agreement in 2021. V-Go gross margin was 43% for 2022.

Cost of revenue – collaborations and services for 2022 was $41.5 million compared to $22.0 million for the same period in 2021, an increase of $19.5 million, primarily due to an increase in manufacturing activities for the production of Tyvaso DPI.

R&D expenses for 2022 were $19.7 million compared to $12.3 million for the prior year. This $7.4 million increase was primarily attributable to costs incurred to develop our product pipeline, including (INHALE-1) and MNKD-101. This $7.4 million increase was primarily attributable to costs incurred to develop our product pipeline, including INHALE-1 and MNKD-101.

Selling expenses for 2022 were $53.8 million compared to $45.5 million for the prior year. This $8.3 million increase was primarily attributable to the primary care pilot program, elimination of the Thyquidity co-promotion (which permitted some expenses associated with the sales force to be recognized as cost of revenue for collaborations and services in the same period of 2021), V-Go promotional efforts after the acquisition in the second quarter of 2022, and partially offset by the net favorable impact of personnel-related costs associated with the Afrezza sales force restructuring in 2022.

G&A expenses for 2022 were $37.7 million compared to $31.9 million for the prior year. This $5.8 million increase was primarily attributable to higher stock-based compensation, increased headcount, and higher professional fees.

Interest expense on the financing liability was $9.8 million for 2022 and represented interest incurred on the sale lease-back transaction for our manufacturing facility in Danbury, CT.

Interest expense on notes was flat for 2022 compared to 2021 due to fixed interest rates and no changes in debt balances.

Cash and cash equivalents and investments as of December 31, 2022 were $172.8 million.

Conference Call

MannKind will host a conference call and presentation webcast to discuss these results today at 5:00 p.m. Eastern Time. Those interested in listening to the conference call live via the Internet may do so by visiting the Company’s website at mannkindcorp.com under Events & Presentations. A replay will be available on MannKind’s website for 14 days.

On February 23, 2023 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported fourth quarter and full year 2022 financial results and provided a corporate update (Press release, Kura Oncology, FEB 23, 2023, View Source [SID1234627617]).

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"We continue to have strong conviction in ziftomenib and its potential to be the best-in-class menin inhibitor," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "The speed with which we have begun enrolling patients with NPM1-mutant acute myeloid leukemia (AML) in our registration-directed trial speaks to our impressive Phase 1 data in this population as well as the significant interest in ziftomenib among investigators. In addition, we believe ziftomenib is well positioned for future combination strategies, with no evidence of drug-induced QTc prolongation, no predicted adverse drug-drug interactions and oral daily dosing that should enable convenient administration with standards of care. We continue to prioritize investment in the program and look forward to sharing further updates as the year progresses, including the presentation of a more mature dataset from our Phase 1 trial of ziftomenib in NPM1-mutant AML patients at a medical meeting in mid-2023."

"Meanwhile, clearance of the IND for KO-2806 marks an important next step for our next-generation farnesyl transferase inhibitor (FTI) program," Dr. Wilson continued. "Our preclinical data is supportive of FTIs in combination with a growing number of targeted therapies, including EGFR inhibitors and PI3 kinase alpha inhibitors as well as tyrosine kinase inhibitors in renal cell carcinoma and KRAS G12C inhibitors in lung cancer, and we look forward to starting our first-in-human trial of KO-2806 in the coming months."

Recent Highlights

Updated clinical data from Phase 1 trial of ziftomenib at ASH (Free ASH Whitepaper) – In December, Kura reported updated data from its Phase 1 trial of ziftomenib, the Company’s potent and selective menin inhibitor, in an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The data highlighted the encouraging safety profile and clinical activity of ziftomenib in patients with relapsed/refractory AML, including a 30% complete response (CR) rate with full count recovery among 20 patients with NPM1-mutant AML treated at the 600 mg dose. Notably, two-thirds of NPM1-mutant AML patients who achieved a CR at 600 mg had IDH and/or FLT3 co-mutations, all of whom had failed prior treatment with IDH and/or FLT3 inhibitors. A median duration of response had not been reached as of the ASH (Free ASH Whitepaper) data cutoff on October 24, 2022.

Recommended Phase 2 dose for ziftomenib in NPM1-mutant AML – In December, Kura also announced that 600 mg once-daily dosing has been designated as the recommended Phase 2 dose and schedule for ziftomenib in NPM1-mutant AML following a positive Type C meeting with the U.S. Food and Drug Administration (FDA). Agreement was also reached on key elements of the Company’s Phase 2 registration-directed trial design.

First patients dosed in registration-directed trial of ziftomenib in NPM1-mutant AML – Earlier this month, Kura announced that multiple patients had been dosed in its Phase 2 registration-directed trial (KOMET-001) of ziftomenib in NPM1-mutant relapsed or refractory AML. The Company expects to enroll a total of 85 patients in the U.S. and Europe. The primary endpoint is CR or CR with partial hematologic recovery (CRh), and key secondary endpoints include duration of response, transfusion independence, safety and tolerability. NPM1-mutant AML accounts for approximately 30% of new AML cases annually and represents a disease of significant unmet need for which no approved targeted therapy exists.

Combination trials to support commercial potential for ziftomenib – Kura is preparing to initiate multiple Phase 1 trials to evaluate ziftomenib in combination with current standards of care in earlier lines of therapy and across multiple patient populations, including NPM1-mutant and KMT2A-rearranged AML. The Company intends to establish a foundation where ziftomenib can be combined safely with various commonly used regimens, such as venetoclax plus azacitidine, FLT3 inhibitors and standard induction cytarabine plus daunorubicin (7+3) chemotherapy, then prioritize those combinations that represent the largest populations and greatest potential commercial value. Kura expects to initiate the first of these trials, KOMET-007, in the first half of 2023.

Preliminary proof of mechanism of tipifarnib plus alpelisib in HNSCC – In October, Kura reported the first demonstration that the combination of tipifarnib and alpelisib can induce a durable clinical response in PIK3CA-dependent head and neck squamous cell carcinoma (HNSCC) at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Molecular Targets and Cancer Therapeutics Symposium. A patient with stage III squamous cell carcinoma of the tonsil with a PIK3CA mutation achieved a durable partial response in the Company’s KURRENT-HN trial and continued on-study for more than 27 weeks as of the data cutoff on September 14, 2022. Treatment-related adverse events in KURRENT-HN are consistent with the known safety profiles of each drug and are manageable, with no dose-limiting toxicities reported to date.

IND for KO-2806, a next-generation farnesyl transferase inhibitor – Last month, Kura announced FDA clearance of its Investigational New Drug (IND) application for KO-2806 for the treatment of advanced solid tumors. KO-2806 is a potent inhibitor of farnesyl transferase designed to improve upon potency, pharmacokinetic and physicochemical properties of earlier FTI drug candidates. The Company intends to evaluate safety, tolerability and preliminary antitumor activity of KO-2806 in a Phase 1 dose-escalation trial (FIT-001) as a monotherapy and in combination with other targeted therapies in adult patients with advanced solid tumors.

$25 million equity investment from Bristol Myers Squibb – In November, Kura sold 1,370,171 shares to Bristol Myers Squibb at a price of $18.25 per share for gross proceeds of $25 million. In connection with the equity investment, Bristol Myers Squibb has appointed a member to Kura’s Global Steering Committee. The equity investment further strengthens the relationship between the two organizations and enables Bristol Myers Squibb, a leader in the discovery and development of transformational cancer treatments, to provide valuable strategic input into Kura’s global development strategy.
Financial Results

Research and development expenses for the fourth quarter of 2022 were $22.7 million, compared to $21.0 million for the fourth quarter of 2021. R&D expenses for the full year 2022 were $92.8 million, compared to $84.7 million for the prior year.

General and administrative expenses for the fourth quarter of 2022 were $12.5 million, compared to $12.1 million for the fourth quarter of 2021. G&A expenses for the full year 2022 were $47.1 million, compared to $46.5 million for the prior year.

Net loss for the fourth quarter of 2022 was $33.1 million, compared to a net loss of $32.7 million for the fourth quarter of 2021. Net loss for the full year 2022 was $135.8 million, compared to a net loss of $130.5 million for the prior year. Net loss for the fourth quarter and full year 2022 included non-cash, share-based compensation expense of $6.8 million and $26.3 million, respectively, compared to $6.4 million and $23.6 million for the same periods in 2021.

Cash, cash equivalents and short-term investments totaled $438.0 million as of December 31, 2022, including the $25 million equity investment from Bristol Myers Squibb and a one-time $10 million draw from a term loan facility with Hercules Capital, compared with $518.0 million as of December 31, 2021. Based on its operating plan, management expects that cash, cash equivalents and short-term investments will fund current operations into the fourth quarter of 2025.
Forecasted Milestones

Dose the first patients in the KOMET-007 combination trial of ziftomenib in the first half of 2023.

Present updated data from the KOMET-001 trial of ziftomenib in NPM1-mutant AML at a medical meeting in mid-2023.

Dose the first patients in the KOMET-008 combination trial of ziftomenib in the second half of 2023.

Determine the optimal biologically active dose in the KURRENT-HN trial of tipifarnib in combination with alpelisib in mid-2023.

Dose the first patients in the FIT-001 dose-escalation trial of KO-2806 in the third quarter of 2023.
Conference Call and Webcast

Kura’s management will host a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT today, February 23, 2023, to discuss the financial results for the fourth quarter and full year 2022 and to provide a corporate update. The live call may be accessed by dialing (877) 407-4018 for domestic callers and (201) 689-8471 for international callers and entering the conference ID: 13735896. A live webcast and archive of the call will be available online from the investor relations section of the company website at www.kuraoncology.com.