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 Oncopeptides reported its interim Report Q1 2022 (Presentation, Oncopeptides, MAY 4, 2022, View Source [SID1234615403]).

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On March 4, 2022 AstraZeneca reported it’s supplemental Biologics License Application (sBLA) for Imfinzi (durvalumab), in combination with standard-of-care chemotherapy, has been accepted and granted Priority Review in the US for patients with locally advanced or metastatic biliary tract cancer (BTC) (Press release, AstraZeneca, MAY 4, 2022, View Source [SID1234613466]).

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The Food and Drug Administration (FDA) grants Priority Review to applications for medicines that, if approved, would offer significant improvements over available options by demonstrating safety or efficacy improvements, preventing serious conditions, or enhancing patient compliance.1 The Prescription Drug User Fee Act date, the FDA action date for their regulatory decision, is during the third quarter of 2022.

BTC is a group of rare and aggressive cancers that occur in the bile ducts and gallbladder. 2,3 Approximately 23,000 people in the US are diagnosed with BTC each year.2 These patients have a poor prognosis, with approximately 5% to 15% of patients with BTC surviving five years.4

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: "People with advanced biliary tract cancer have faced poor outcomes and limited treatment options for too long, and today’s news for the TOPAZ-1 trial underscores the urgency to deliver new, effective therapies in this setting. We are working closely with the FDA to bring the first immunotherapy-based option to patients with this devastating cancer and potentially set a new standard of care with Imfinzi plus chemotherapy."

The sBLA was based on results from an interim analysis of the TOPAZ-1 Phase III trial presented during the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers (ASCO GI) Symposium. The data showed Imfinzi plus chemotherapy (gemcitabine plus cisplatin) reduced the risk of death by 20% versus chemotherapy alone (based on a hazard ratio [HR] of 0.80; 95% confidence interval [CI], 0.66-0.97; 2-sided p=0.021). An estimated one in four (25%) patients treated with Imfinzi plus chemotherapy were alive at two years compared to one in 10 (10%) treated with chemotherapy alone.

Results also showed a statistically significant 25% reduction in the risk of disease progression or death with Imfinzi plus chemotherapy (HR, 0.75; 95% CI, 0.64-0.89; 2-sided p=0.001). The Imfinzi combination was generally well tolerated and did not increase the discontinuation rate due to adverse events compared to chemotherapy alone.

In December 2020, Imfinzi was granted Orphan Drug Designation in the US for the treatment of BTC.

Notes

Biliary tract cancer
Biliary tract cancer (BTC) is a group of rare and aggressive gastrointestinal (GI) cancers that form in the cells of the bile ducts (cholangiocarcinoma), gallbladder or ampulla of Vater (where the bile duct and pancreatic duct connect to the small intestine).2,3

Cholangiocarcinoma is more common in China and South-East Asia and is on the rise in Western countries.2,4 Gallbladder cancer is more common in certain regions of South America, India and Japan.5

Early-stage BTC affecting the bile ducts and gallbladder often presents without clear symptoms and most new cases of BTC are therefore diagnosed at an advanced stage, when treatment options are limited and the prognosis is poor.4-6

TOPAZ-1
TOPAZ-1 is a randomised, double-blind, placebo controlled, multicentre, global Phase III trial of Imfinzi in combination with chemotherapy (gemcitabine plus cisplatin) versus placebo in combination with chemotherapy as a 1st-line treatment in 685 patients with unresectable advanced or metastatic BTC including intrahepatic and extrahepatic cholangiocarcinoma, and gallbladder cancer. Patients with ampullary carcinoma were excluded.

The primary endpoint is overall survival and key secondary endpoints included progression-free survival, objective response rate and safety. The trial was conducted in 105 centres across 17 countries including in the US, Europe, South America and several countries in Asia including South Korea, Thailand, Japan and China.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

Imfinzi is the only approved immunotherapy in the curative-intent setting of unresectable, Stage III non-small cell lung cancer (NSCLC) in patients whose disease has not progressed after chemoradiotherapy, and is the global standard of care in this setting based on the PACIFIC Phase III trial.

Imfinzi is also approved in the US, EU, Japan, China and many other countries around the world for the treatment of extensive-stage small cell lung cancer (ES-SCLC) based on the CASPIAN Phase III trial. In 2021, updated results from the CASPIAN trial showed Imfinzi plus chemotherapy tripled patient survival at three years versus chemotherapy alone.

Imfinzi is also approved for previously treated patients with advanced bladder cancer in several countries.

Since the first approval in May 2017, more than 100,000 patients have been treated with Imfinzi.

As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with small cell lung cancer (SCLC), NSCLC, bladder cancer, several GI cancers, ovarian cancer, endometrial cancer, and other solid tumours.

In the past year, Imfinzi combinations have demonstrated clinical benefit in multiple additional cancer settings with positive Phase III trials in unresectable advanced liver cancer (HIMALAYA) and metastatic NSCLC (POSEIDON).

AstraZeneca in GI cancers
AstraZeneca has a broad development programme for the treatment of GI cancers across several medicines and a variety of tumour types and stages of disease. In 2020, GI cancers collectively represented approximately 5.1 million new cancer cases leading to approximately 3.6 million deaths.6

Within this programme, the Company is committed to improving outcomes in gastric, liver, BTC, oesophageal, pancreatic, and colorectal cancers.

Imfinzi is being assessed in combinations in liver, BTC, oesophageal and gastric cancers in an extensive development programme spanning early to late-stage disease.

The Company aims to understand the potential of Enhertu (trastuzumab deruxtecan), a HER2-directed antibody drug conjugate, in the two most common GI cancers, colorectal and gastric cancers. Enhertu is jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.

Lynparza (olaparib) is a first-in-class PARP inhibitor with a broad and advanced clinical trial programme across multiple GI tumour types including pancreatic and colorectal cancers. Lynparza is developed and commercialised in collaboration with MSD (Merck & Co., Inc. inside the US and Canada).

AstraZeneca in immunotherapy
Immunotherapy is a therapeutic approach designed to stimulate the body’s immune system to attack tumours. The Company’s Immuno-Oncology (IO) portfolio is anchored in immunotherapies that have been designed to overcome evasion of the anti-tumour immune response. AstraZeneca is invested in using IO approaches that deliver long-term survival for new groups of patients across tumour types.

The Company is pursuing a comprehensive clinical-trial programme that includes Imfinzi as a single treatment and in combination with tremelimumab and other novel antibodies in multiple tumour types, stages of disease, and lines of treatment, and where relevant using the PD-L1 biomarker as a decision-making tool to define the best potential treatment path for a patient.

In addition, the ability to combine the IO portfolio with radiation, chemotherapy, and targeted small molecules from across AstraZeneca’s oncology pipeline, and from research partners, may provide new treatment options across a broad range of tumours.

AstraZeneca in oncology
AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.

The Company’s focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience.

AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.

On May 4, 2022 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 first quarter of 2022 (Press release, Morphic Therapeutic, MAY 4, 2022, View Source [SID1234613482]).

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First Quarter 2022 and Recent Corporate Highlights

Launched EMERALD-1 (MORF-057-201) phase 2a study
EMERALD-1 is an open-label multi-center study of MORF-057 enrolling up to 35 patients with moderate to severely active ulcerative colitis (UC) who will be treated with 100 mg BID (twice daily)
EMERALD-2 (MORF-057-202), a global phase 2b double-blind randomized placebo-controlled trial of MORF-057, is expected to begin in the third quarter of 2022 and then run in parallel with EMERALD-1
Presented new preclinical data at ECCO 2022 expanding understanding of α4β7-expressing immune cells and further describing MORF-057 dose-response
Data from multiple preclinical studies in animals strongly correlated with human trials and demonstrate clear MORF-057 dose-response
Results also show changes in lymphocyte populations and CCR9 transcripts consistent with α4β7 inhibition mechanism of action
Announced several key leadership appointments
Dr. Brihad Abhyankar, MS, FRCS, FFPM was appointed Vice President, Clinical Development and will lead the EMERALD-1 and EMERALD-2 phase 2 trials of MORF-057 in UC
Dr. Abhyankar has directly relevant experience to the MORF-057 development program after serving as executive medical director of clinical science at Takeda, where he directed strategy for global clinical development and played a key role in the development, approval, and the life cycle management of ENTYVIO or vedolizumab
Dr. Abhyankar has over 20 years of biopharmaceutical industry experience and 10 years of training and practice in surgery and medicine
Aaron Pelta was promoted to Senior Vice President, Business and Corporate Development
Mr. Pelta previously served as Morphic’s Vice President of Businesses Development and leads partnering, commercial planning and portfolio strategy
Mr. Pelta has 20 years of biopharmaceutical business experience including leadership roles at Arsanis, Shire and Cubist Pharmaceuticals
Focused the Company’s research and development collaboration efforts with AbbVie and Janssen on higher-potential integrin targets in multiple undisclosed therapeutic areas
Thanked Nilesh Kumar, Ph.D., for his contributions as a member of the Morphic Board of Director upon his departure after five years of leadership and collaboration
"Morphic made the most significant clinical advance in our Company’s history with the launch of the EMERALD phase 2a study of MORF-057 in UC during the first quarter of 2022," commented Praveen Tipirneni, MD, President and Chief Executive Officer of Morphic Therapeutic. "In addition, we bolstered the Morphic team with the addition of Brihad Abhyankar, a proven leader in the GI development arena an expert in α4β7 for the treatment of UC. We will continue to make important progress in the MORF-057 development program with the launch of the EMERALD-2 global randomized controlled phase 2b study in the coming months."

Financial Results for the First Quarter 2022

Net loss for the quarter ended March 31, 2022 was $31.5 million or $0.85 per share compared to a net loss of $21.3 million or $0.63 per share for the same quarter last year.
Revenue was $2.4 million for the quarter ended March 31, 2022, compared to $3.3 million for the same quarter last year.
Research and development expenses were $26.5 million for the quarter ended March 31, 2022, as compared to $18.6 million for the same quarter last year. The increase was primarily attributable to higher manufacturing and development costs along with higher pre-clinical and phase 2 clinical trial costs to support our lead product candidate MORF-057.
General and administrative expenses were $7.6 million for the quarter ended March 31, 2022, compared to $6.0 million for the same quarter last year. The increase was due to increased non-cash stock-based compensation expense and higher payroll costs.
As of March 31, 2022, Morphic had cash, cash equivalents and marketable securities of $380.7 million, compared to $408.1 million as of December 31, 2021. The Morphic believes its cash, cash equivalents and marketable securities as of March 31, 2022, will be sufficient to fund operating expenses and capital expenditure requirements through year-end 2024.

On May 4, 2022 Exelixis, Inc. (Nasdaq: EXEL) reported that members of the company’s management team will participate in fireside chats at the following investor conferences in May (Press release, Exelixis, MAY 4, 2022, View Source [SID1234613514]):

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BofA Securities 2022 Healthcare Conference: Exelixis is scheduled to present at 1:40 p.m. ET / 10:40 a.m. PT on Wednesday, May 11, 2022 in Las Vegas.
2022 RBC Capital Markets Global Healthcare Conference: Exelixis is scheduled to present at 8:30 a.m. ET / 5:30 a.m. PT on Tuesday, May 17, 2022 in New York.
To access the webcast links, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the presentations to ensure adequate time for any software download that may be required to listen to the webcasts. Replays will also be available at the same location for at least 30 days.