On May 4, 2022, Turning Point Therapeutics, Inc. (the "Company") reported that entered into a license agreement (the "LaNova License Agreement") with LaNova Medicines Limited ("LaNova") for an exclusive, royalty-bearing license to intellectual property related to LM-302, a clinical stage anti-Claudin18.2 antibody drug conjugate (the "Product"), on a worldwide basis excluding Greater China and South Korea (the "Company Territory") (Filing, 8-K, Turning Point Therapeutics, MAY 4, 2022, View Source [SID1234613640]). Under the LaNova License Agreement, the Company has the exclusive right to research, develop, use, register, offer for sale, import and otherwise commercialize the Product in the Company Territory and non-exclusive rights to manufacture the Product worldwide in support of activities in the Company Territory.

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Pursuant to the LaNova License Agreement, the Company will pay LaNova an upfront cash payment of $25.0 million and may be obligated to pay milestone payments, which include up to $195.0 million in development and regulatory milestones and up to $880.0 million in sales milestones, and tiered royalty payments based on percentages (ranging from the mid-single digits to the mid-teens) of net sales (subject to customary deductions).

Subject to specified exceptions, for a period of time, the Company has agreed that neither it nor its controlled affiliates or sublicensees will engage in any clinical development, use or commercialization of specified products that would compete with the Products in the Company Territory, and LaNova has agreed that neither it nor its affiliates, licensees and its sublicensees will conduct any clinical development, use or commercialization of specified products that would compete with the Products in the Company Territory, other than expressly permitted activities.

The LaNova License Agreement will continue in effect until expiration of the last royalty term for the Product in any country in the Company Territory, where the royalty term for a Product in a given country in the Company Territory continues until the later of (i) the date of the last-to-expire valid claim within the Company’s patent rights that covers the Product in such country; (ii) the expiry of the regulatory exclusivity for such Product in such country; or (iii) 10 years after the date of the first commercial sale of the Product in such country. Subject to the terms of the LaNova License Agreement, the Company may terminate the LaNova License Agreement for convenience by providing written notice to the Company, which termination will be effective following a prescribed notice period. In addition, either party may terminate the LaNova License Agreement for the other party’s uncured material breach of the LaNova License Agreement, with a customary notice and cure period, or for the other party’s insolvency. LaNova may also terminate the agreement if the Company is acquired by a third party and the acquired party is engaged in activities with competing products that are not divested or discontinued, upon notice of termination to the Company within a specific period following closing of such acquisition. In addition, LaNova may terminate the LaNova License Agreement under specified circumstances if the Company or certain other parties challenge LaNova’s patent rights. If the Company terminates the agreement for convenience, the Company will grant to LaNova a non-exclusive, worldwide license, which may be royalty-bearing in certain circumstances, to intellectual property owned by the Company that is necessary for and was used by the Company to commercialize the Product.

The Company is responsible for conducting the development and commercialization activities in the Company Territory related to the Products at its own expense. The Company and LaNova will collaborate on a global development plan under which both parties will conduct global clinical studies in their respective territories. The Company and LaNova will each be responsible for the costs allocated to them in accordance with the agreed budget under the global development plan. Both the Company and LaNova have the ability to conduct local studies outside of the global development plan at their own expense.

Both the Company and LaNova have the obligation to use commercially reasonable efforts to conduct development activities under the agreed-upon global development plan. The Company has the obligation to use commercially reasonable efforts to perform local studies independent of the global development plan and to obtain regulatory approvals for the Product in the Company Territory.

LaNova has an initial obligation to supply the Company with the Product. After a specified period, the Company will assume responsibility for supply of the Product.

As part of the LaNova License Agreement, the Company also obtained the right of first negotiation for an exclusive license to develop, use, manufacture and commercialize other products containing components of the Product. In addition, the Company has the option to collaborate with LaNova on up to three additional antibody drug conjugate programs, initiated or proposed by either the Company or LaNova.

The foregoing description of the material terms of the Agreement does not purport to be complete and is qualified in its entirety by reference to the complete text of the Agreement, a copy of which the Company intends to file, with confidential terms redacted, with the Securities and Exchange Commission as an exhibit to the Company’s Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2022.

On May 4, 2022 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a biotechnology company developing novel LAG-3-related immunotherapy treatments for cancer and autoimmune disease, reported new biomarker and exploratory analysis data from its Phase IIb AIPAC trial (Press release, Immutep, MAY 4, 2022, View Source [SID1234613616]). The data was presented at ESMO (Free ESMO Whitepaper)’s Breast Cancer Congress in a poster presentation which is available on ESMO (Free ESMO Whitepaper)’s website and at View Source

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The double blind and randomised AIPAC trial evaluated efti in combination with paclitaxel chemotherapy (efti group) compared to placebo plus paclitaxel (placebo group) in 227 patients with HER2-negative/HR positive metastatic breast cancer. Final OS results were reported in November 2021 in a late breaking abstract at SITC (Free SITC Whitepaper) showing encouraging efficacy in multiple patient subgroups.

Dr Frederic Triebel, Immutep’s CSO and CMO said: "The biomarker analysis is highly valuable for two key reasons. Firstly, the statistically significant difference in the immune response between the efti and placebo patients confirms efti is activating the immune system and helping patients live longer. This is demonstrated by the increase in circulating monocytes, CD8 T cells and a serum Th1 marker, CXCL10, plus the absolute lymphocyte count (ALC), and correlation of these improved immune parameters with overall survival. Secondly, the early rise in ALC in patients treated with efti provides clinicians with a potential predictor of improved survival, helping them to determine early on if continued treatment with efti is potentially beneficial."

"The exploratory analysis showing statistically significant improvements in OS in different patient subgroups is also very important as we work towards the optimal design of the planned registrational trial in breast cancer. This all continues to be consistent with our long-held belief that efti, with its unique mechanism of action, should be able to help diverse sets of patients, including those who fail to respond to current immunotherapy options," he concluded.

Increase in immune response biomarkers linked to improved overall survival

Biomarker analysis showed efti, in combination with weekly paclitaxel, significantly increased the number of circulating immune cells (monocytes, activated CD8 T cells) and CXCL10 serum levels, compared to baseline. The increase was not observed in the placebo group (see Table 1). An increase in activated CD4 cells was also observed. The increase in these pharmacodynamic markers (monocytes, CD8 T cells and CXCL10) was significantly linked to improved OS in the efti group, but not in the placebo group. These findings of an improved immune status are also relevant for the anti-PD-1 combinations with efti.

Notably, in the biomarker study, patient blood samples were drawn 13 days after efti injection. This was to ensure that the study would show the minimum residual pharmacodynamic effect from therapy with efti and paclitaxel, since efti disappears from the blood in only 3-4 days.

Thus, since efti is administered every 14 days, we conclude that that the observed increase in the number of circulating immune cells was sustained throughout the course of treatment (or even greater at the peak of the immune response).

Potential predictive biomarker for improved survival

The absolute lymphocyte count (ALC) was shown to increase early and sustainably in patient’s treatment regimen in the efti group, but not the placebo group. The increase in ALC was also significantly linked to improved OS. ALC can be measured easily in every hospital laboratory, making it a practical potential predictive biomarker for efti efficacy and therefore improved survival.

Univariate and multivariate analysis indicates subgroups for future studies

Through exploratory analyses, six subgroups of patients showed an improvement in OS in the efti group, compared to placebo (see Table 2). Five of the six subgroups (< 65 years, low baseline monocytes, high neutrophil to lymphocyte ratio (NLR), < 5 years since diagnosis and luminal B) showed a statistically significant improvement in OS and in the ‘no prior taxane therapy subgroup’ a statistically significant increase was also seen in ORR.

The subgroups will be considered for patient population selection for future studies, such as Immutep’s planned Phase III AIPAC-003 trial.

Multivariate analysis showed patients entering the trial with high body mass index (BMI) and prior CDK4/6 treatment had significantly poorer PFS and OS outcomes irrespective of the therapy they received. High BMI and prior CDK4/6 treatment are therefore considered independent poor prognostic markers and will be considered as stratification factors for future studies. Multivariate analysis also showed that low monocytes and no prior taxane therapy were independent significant predictive factors for improved OS.

Table 2. Six favourable subgroups with improved OS based on univariate analysis

Active Immunotherapy Paclitaxel (AIPAC) was a multicentre, placebo-controlled, double-blind, 1:1 randomised Phase IIb clinical trial in HER2-negative/HR positive metastatic breast cancer.

The study evaluated the combination of Immutep’s lead product candidate, eftilagimod alpha (efti, LAG-3Ig or IMP321), and paclitaxel chemotherapy. 227 HER2-negative/HR positive metastatic breast cancer patients were randomised 1:1 to a chemo-immunotherapy arm (efti plus paclitaxel) or to a comparator arm (placebo plus paclitaxel). Patients received weekly paclitaxel at days 1, 8 and 15, with either efti or placebo injected subcutaneously on days 2 and 16 of each 4-week cycle, repeated for 6 cycles. Thereafter, patients passed over to the maintenance phase with efti alone.

On May 4, 2022 Signify Health, Inc. (NYSE: SGFY), a leading healthcare platform that leverages advanced analytics, technology and nationwide healthcare networks to create and power value-based payment programs, reported the Company’s financial results for the first quarter 2022 (Press release, Signify Health, MAY 4, 2022, View Source [SID1234613595]).

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"Our strong first quarter results demonstrate the significant value we are delivering to our clients as we continue to execute our growth strategy and make strategic investments in our business," said Kyle Armbrester, Chief Executive Officer of Signify Health. "The investments we have made to support increased customer demand for our comprehensive member evaluations enabled us to reach a record level of 564 thousand evaluations in the first quarter. Investment in people and processes also helped our Episodes of Care and BPCI-A program partners to improve health outcomes for patients and deliver meaningful savings as we continue to manage through the COVID-19 environment and its impact on program size and savings rate."

Mr. Armbrester continued, "The integration of Caravan Health is progressing well and clients are responding favorably to how the combined capabilities of Signify and Caravan can help them be more successful in their alternative payment strategies. Clients also are expressing interest in tapping into the significant access and scale we have in homes as part of their efforts to manage total cost of care. Caravan Health adds to our revenue diversification while supporting our mission to accelerate the transformation of the U.S. healthcare system from fee-for-service to value-based care."

First Quarter 2022 Financial Results

Total revenue for the first quarter increased 20% to $216.5 million, up from $180.0 million in the same period a year ago. Overall growth in the first quarter of 2022 was driven by a 23% increase in Home & Community Services (HCS) segment revenue to $186.9 million and a 7% increase in Episodes of Care Services (ECS) segment revenue to $29.6 million compared to a year ago.
The strong HCS revenue was a quarterly record and is attributable to an increase in in-home evaluations (IHE) volume, which grew to approximately 564 thousand from approximately 462 thousand in the first quarter of 2021.
ECS first quarter revenue growth was driven by an increase in program size and revenue generated by Caravan Health for one-month post acquisition.
First quarter total net loss improved to $16.3 million compared to a net loss of $51.7 million for the same period a year ago. Net loss included $28.9 million of other expense related to the remeasurement of the fair value of our Equity Appreciation Rights (EAR) due to an increase in the Company’s stock price in the first quarter.
Non-GAAP Adjusted EBITDA1 for the first quarter increased 31% to $45.0 million, compared to $34.4 million for the first quarter 2021, driven primarily by HCS revenue growth.
Non-GAAP Adjusted EBITDA margin1 for the first quarter was 20.8%, a 170-basis point improvement from the comparable year ago period.
2022 Outlook

The Company is maintaining its full year 2022 estimates as follows:

Total revenue in the range of $948 million to $971 million; and
Total adjusted EBITDA1 in the range of $212 million to $222 million.
1Adjusted EBITDA and Adjusted EBITDA margin are non-GAAP financial measures. Refer to the reconciliation in "Non-GAAP Financial Measures." We have not reconciled 2022 guidance for adjusted EBITDA to net income (loss), the most directly comparable GAAP measure, and have not provided forward-looking guidance for net income (loss) because of the uncertainty around certain items that may impact net income (loss), including, among others, stock-based compensation and the fair valuation of the EARs, that are not within our control or cannot be reasonably estimated.

Conference Call Information

Signify Health will host a conference call to discuss the Company’s first quarter 2022 results on May 5, 2022 at 8:30am ET. A live audio webcast of the conference call may be accessed through the investor relations section of Signify Health’s website at investors.signifyhealth.com/events/default.aspx and will be available for replay through July 5, 2022.

On May 4, 2022 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the first quarter ended March 31, 2022 and provided a general business update (Press release, Ophthotech, MAY 4, 2022, View Source [SID1234613593]).

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"During the first quarter, we continued the momentum of 2021 for GATHER2, our second Phase 3 clinical trial for Zimura (avacincaptad pegol), a novel complement inhibitor, for the treatment of geographic atrophy (GA)," stated Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio. "We look forward to the exciting opportunities that lie ahead in 2022, including reporting topline data from GATHER2 and being closer to reaching our goal of providing patients and physicians with a treatment for GA for which there are currently no treatments options available."

•The Company expects topline GATHER2 data to be available in the third quarter of this year, approximately one year after the enrollment of the last patient plus the time needed for database lock and analysis. If the results from GATHER2 are positive, the Company plans to submit a new drug application (NDA) with the U.S. Food and Drug Administration (FDA) and a marketing authorization application (MAA) with the European Medicines Agency.

•Patient retention for the GATHER2 clinical trial, as measured by the injection fidelity rate, continues to exceed the Company’s expectations. The Company is targeting an injection fidelity rate for GATHER2, as measured through month 12, of greater than 90%. Injection fidelity is calculated by dividing the total number of actual injections (drug and sham) for all patients by the total number of expected injections (drug and sham) based on the total number of patients enrolled in the trial. The Company considers injection fidelity to be the most important and stringent measure of patient retention because it reflects the timely administration of the drug or sham into the patient’s eye.

•The Company is currently more than 94% complete with year one of the GATHER2 clinical trial, based on the number of scheduled patient visits.

"We are pleased with the strength of our current cash position and with the progress achieved during this first quarter," stated Pravin U. Dugel, MD, President of Iveric Bio. "As we get closer to reporting the GATHER2 topline data, we continue to prepare for a potential submission of an NDA in the U.S. for Zimura for the treatment of GA as efficiently as possible. We are well-positioned with an established medical affairs team in place, and we continue to build out our commercial infrastructure with a team that has extensive experience in launching drugs to treat retinal diseases with large market potential. Additionally, during this year we continue to provide additional exploratory analyses from GATHER1, our first pivotal clinical trial for Zimura in GA, which analyses we believe further support the consistency of the positive data previously reported for GATHER1 and inform future potential development opportunities for Zimura in other indications."

Zimura (avacincaptad pegol): Complement C5 Inhibitor

•Today, the Company provided results from a post-hoc analysis of GATHER1 evaluating the reduction in GA lesion growth observed for patients receiving Zimura as compared to patients receiving sham in a subset of patients based on the distance of a patient’s GA lesion to the foveal center at baseline. See the press release issued earlier today. The Company believes the results of this post-hoc analysis are consistent with the other results previously reported for GATHER1.

•In March 2022, the U.S. Patent and Trademark Office (USPTO) granted the Company a patent with claims covering methods of using Zimura for the treatment of GA. Subject to any patent term adjustments or extensions the Company may obtain, the issued patent is expected to expire in 2034.

•In April 2022, the Company provided results from a post-hoc analysis of the 8 cases of choroidal neovascularization (CNV) reported for the Zimura 2 mg group (n=67 patients) in the GATHER1 trial. An independent and masked reading center reviewed the optical coherence tomography (OCT) images of those patients at the 12-month and 18-month timepoints and classified the cases as non-exudative macular neovascularization (MNV) (2 cases at 12 months and 18 months) and exudative MNV (4 cases at 12 months and 6 cases at 18 months). The reading center also found that among the 6 patients who developed exudative MNV at 18 months, 5 of those patients had a double layer sign at baseline. See the Company’s Form 8-K filed on April 4, 2022, for full results from this analysis. Based on scientific literature and clinical understanding among the retinal community, the Company believes that the presence of a double-layer sign on OCT may be a useful biomarker to predict the future onset of cases of exudative MNV.

•In February 2022, results from a post-hoc analysis that evaluated various GA growth parameters to explore the rate of disease progression within various regions in the fovea among a subset of patients from GATHER1 were presented at the Angiogenesis, Exudation and Degeneration conference. Consistent with the overall results of GATHER1, in the post-hoc analysis a reduction in lesion growth in five standardized regions surrounding and including the central foveal area was observed for patients receiving Zimura 2 mg as compared to patients receiving sham over a period of 18 months. The Company believes preserving the central fovea region may be associated with clinical outcomes important to GA patients.

•The Company plans to initiate a clinical trial studying Zimura in patients with intermediate AMD in the fourth quarter of 2022, following planned interactions with the FDA and other regulatory authorities. The Company’s development strategy in this indication is subject to regulatory feedback.

•Patient enrollment in STAR, the Company’s Phase 2b screening clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease (STGD1), is ongoing. The results of this clinical trial are expected after the topline results of GATHER2.

IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor
•The Company is planning for IND-enabling toxicology studies for IC-500. The Company expects to submit an investigational new drug application (IND) to the FDA for IC-500 during mid-2023.

Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)
•As the Company focuses its efforts and resources on the development and potential commercialization of Zimura, the Company is exploring potential collaborations for the future development and potential commercialization of IC-100, the Company’s product candidate for Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP) and IC-200, the Company’s product candidate for BEST1-Related IRDs.
•The Company is continuing its minigene programs for Leber’s Congenital Amaurosis type 10 (CEP290), autosomal recessive Stargardt Disease (ABCA4) and Usher’s syndrome (USH2A).

Corporate Updates
The Company expanded its Board of Directors by adding Christine Ann Miller, a pharmaceutical veteran, to the Company’s board of directors in January 2022.

First Quarter 2022 Financial Update and 2022 Cash Guidance

•As of March 31, 2022, the Company had approximately $345.7 million in cash, cash equivalents and available-for-sale securities.

•The Company estimates its year-end 2022 cash, cash equivalents and available-for-sale securities to range between $215 and $225 million. The Company also estimates that its cash, cash equivalents and available-for-sale securities will be sufficient to fund its planned capital expenditure requirements and operating expenses through at least mid-2024. These estimates are based on the Company’s current business plan, including the continuation of its ongoing clinical development programs for Zimura in GA and STGD1 and the initiation of an intermediate AMD clinical trial, preparation and potential filing of an NDA and MAA for Zimura in GA, continuing preparations for potential commercial launch of Zimura in GA, investing in sustained release delivery technologies for Zimura, and the advancement of its IC-500 development program. Excluded from these estimates are any potential approval or sales milestones payable to Archemix Corp. or any potential expenses for actual commercial launch of Zimura, such as associated sales force expenses, any additional expenditures related to potentially studying Zimura in indications outside of GA, STGD1 and intermediate AMD, or resulting from the potential in-licensing or acquisition of additional product candidates or technologies, or any associated development the Company may pursue.

2022 Q1 Financial Highlights

•R&D Expenses: Research and development expenses were $22.6 million for the quarter ended March 31, 2022, compared to $18.5 million for the same period in 2021. Research and development expenses increased primarily due to the continued progress and patient recruitment activities of our GATHER2 clinical trial, increased manufacturing activities for Zimura, and increases in personnel costs, including share-based compensation associated with additional research and development staffing.

•G&A Expenses: General and administrative expenses were $12.1 million for the quarter ended March 31, 2022, compared to $8.3 million for the same period in 2021. General and administrative expenses increased primarily due increases in personnel costs, including share-based compensation associated with preparations for potential commercial launch of Zimura in GA.

•Net Loss: The Company reported a net loss for the quarter ended March 31, 2022 of $34.5 million, or ($0.29) per diluted share, compared to a net loss of $26.8 million, or $(0.29) per diluted share, for the same period in 2021.

Conference Call/Web Cast Information
Iveric Bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for May 4, 2022, at 8:00 a.m. Eastern Time. To participate in this conference call, dial 1-888-317-6003 (USA) or 1-412-317-6061 (International), passcode 1313914. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the Iveric Bio website at www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 1-877-344-7529 (USA Toll Free), passcode 9999784.

On May 4, 2022 Northwest Biotherapeutics (OTCQB: NWBO) ("NW Bio"), a biotechnology company developing DCVax personalized immune therapies for solid tumor cancers, reported that a presentation entitled "Autologous Tumor Lysate-Loaded Dendritic Cell Vaccination for Glioblastoma" will be made on May 10, 2022 at 11:10 a.m., by Dr. Linda Liau at the Frontiers of Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) of the New York Academy of Sciences (Press release, Northwest Biotherapeutics, MAY 4, 2022, View Source [SID1234613592]).

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This presentation can be viewed virtually by registering online at the Academy’s website at View Source