On June 29, 2022 Codiak BioSciences, Inc. (NASDAQ: CDAK), a clinical-stage biopharmaceutical company pioneering the development of exosome-based therapeutics as a new class of medicines, reported the initiation of patient dosing in its Phase 1 clinical trial of exoASO-STAT6, an engineered exosome precision medicine candidate designed to selectively deliver antisense oligonucleotides to disrupt STAT6 signaling in tumor associated macrophages (TAMs) and induce an anti-tumor immune response (Press release, Codiak Biosciences, JUN 29, 2022, View Source [SID1234616363]). exoASO-STAT6 is Codiak’s third clinical program and the first to evaluate a systemically administered exosome-based drug candidate .

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"Targeting macrophages is the next great frontier in cancer immunotherapy and we are encouraged by the monotherapy anti-tumor activity exhibited by exoASO-STAT6 in preclinical models, which has not been observed among other approaches to date. We believe this may indicate the potential to bring transformative treatments to patients facing intractable forms of cancer," said Douglas E. Williams, Ph.D., CEO, Codiak. "The initiation of this trial is also a significant milestone for our company, as the advancement of exoASO-STAT6 into the clinic highlights the versatility of our engineering platform. This candidate is the first of our programs to target macrophages, the first to carry a nucleic acid and the first to be administered intravenously."

The Phase 1 clinical trial will evaluate the safety, tolerability, biomarkers and preliminary anti-tumor activity of exoASO-STAT6 in patients with advanced hepatocellular carcinoma (HCC), patients with liver metastases from primary gastric cancer and colorectal cancer (CRC). The study is anticipated to enroll patients across four cohorts at sequentially escalating dose levels, with subjects in the initial cohorts receiving biweekly exoASO-STAT6 administered intravenously over the course of 28 days. Ultimately the trial may enroll up to 30 patients. Initial Phase 1 data are expected in the first half of 2023.

TAMs promote tumor growth by exhibiting an immune suppressive M2 phenotype. Reprogramming TAMs toward a pro-inflammatory M1 phenotype may be a compelling approach to induce anti-tumor immunity. The M2 phenotype is controlled by key transcription factors such as STAT6, which have proven difficult to drug selectively in TAMs using prior approaches. Codiak plans to initially develop exoASO-STAT6 for primary cancers of the liver, where STAT6 expression has been correlated with poor survival.

In multiple in vivo preclinical studies, exoASO-STAT6 demonstrated potent single agent activity, including >90% tumor growth inhibition and 50-80% complete responses. In HCC models, exoASO-STAT6 induced significant knockdown of STAT6 mRNA, attenuated tumor growth and induced complete remission of tumor lesions in 50% of mice. This anti-tumor activity was enhanced (75% complete remissions) when exoASO-STAT6 was administered with anti-PD1 antibodies. The monotherapy activity was accompanied by remodeling of the tumor microenvironment including significant expansion of M1-like macrophages and induction of an adaptive anti-tumor immune response, enabling tumor elimination.

About the engEx Platform

Codiak’s proprietary engEx Platform is designed to enable the development of engineered exosome therapeutics for a wide spectrum of diseases and to manufacture them reproducibly and at scale to pharmaceutical standards. By leveraging the inherent biology, function and tolerability profile of exosomes, Codiak is developing engEx exosomes designed to carry and protect potent drug molecules, provide selective delivery and elicit the desired pharmacology at the desired tissue and cellular sites. Through its engEx Platform, Codiak seeks to direct tropism and distribution by engineering exosomes to carry on their surface specific targeting drug moieties, such as proteins, antibodies/fragments, and peptides, individually or in combination. Codiak scientists have identified two exosomal proteins that serve as surface and luminal scaffolds. By engineering the exosome surface or lumen and optimizing the route of administration, Codiak aims to deliver engEx exosomes to the desired cell and tissue to more selectively engage the drug target, potentially enhancing the therapeutic index by improving potency and reducing toxicity.

On June 29, 2022 M.S.Q. Ventures ("MSQ") reported that its client, BioLineRx Ltd. ("BioLineRx") (NASDAQ/TASE: BLRX), has successfully entered into a co-development agreement with GenFleet Therapeutics, Inc., an immuno-oncology focused biopharmaceutical company based in Shanghai, China, for the development of Motixafortide in pancreatic ductal adenocarcinoma ("PDAC") (Press release, BioLineRx, JUN 29, 2022, View Source [SID1234616362]).

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Under the terms of the agreement, GenFleet will design and execute a randomized Phase 2b clinical trial that will enroll approximately 200 first-line metastatic PDAC patients in China. This randomized controlled study will aim to evaluate the superiority of Motixafortide in combination with an anti-PD-1 and chemotherapy compared to chemotherapy alone, the current standard of care.

"This collaboration is based on the highly encouraging results from our Phase 2a COMBAT/KEYNOTE-202 study of Motixafortide in combination with an anti-PD-1 and chemotherapy, which provide strong support for continued development in this very challenging disease. With its broad solid tumor oncology pipeline and highly experienced development team, we believe we have found an outstanding partner in GenFleet to execute a rigorously designed randomized Phase 2b trial," said Philip Serlin, CEO of BioLineRx. "The MSQ team helped to navigate through the complexity of cross-border transactions and secured this transaction by deploying their deep knowledge of the global market. We are highly impressed with MSQ’s "thinking outside the box" approach and their expertise in deal structuring and negotiation."

"The results of the COMBAT/KEYNOTE-202 Phase 2a study demonstrate the benefit of combining the CXCR4 inhibitor Motixafortide with an anti-PD-1 and chemotherapy in a second-line setting," said Qiang Lu, Chairman of GenFleet. "We believe that this combination could be beneficial to patients in a first-line setting as well, and we hope to confirm this in a randomized trial. We are thrilled to be partners with BioLineRx in the development of this late-stage clinical asset, and look forward to initiating this important trial as quickly as possible. Throughout this journey, MSQ team has demonstrated professionalism and dedication in helping both of our teams to reach the finish line in a timely fashion."

Echo Hindle-Yang, CEO of MSQ, reflects on the transaction, "This collaboration demonstrates another excellent example of global co-development, which leverages each company’s regional expertise to accelerate the development of new treatments. We are excited to see the alliance between BioLineRx and Genfleet to pursue the treatment of PDAC, which has proven to be incredibly challenging. I am impressed by Phil’s commitment to developing Motixafortide in PDAC which is also shared by GenFleet’s Dr. Lu. With such a strong commitment from each team, we believe that patients will benefit from this collaboration. We are honored to move forward with this cross-border partnership, and help achieve future success.

On June 29, 2022 Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) reported a new vaccine supply agreement with the U.S. government to support the continued fight against COVID-19 (Press release, BioNTech, JUN 29, 2022, View Source [SID1234616361]). Under the agreement, the U.S. government will receive 105 million doses (30 µg, 10 µg and 3 µg). This may include adult Omicron-adapted COVID-19 vaccines, subject to authorization from the U.S. Food and Drug Administration (FDA). The doses are planned to be delivered as soon as late summer 2022 and continue into the fourth quarter of this year.

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The U.S. government will pay the companies $3.2 billion upon receipt of the first 105 million doses. Under this agreement, the U.S. government also has the option to purchase up to 195 million additional doses, bringing the total number of potential doses to 300 million.

"As the virus evolves, this new agreement will help ensure people across the country have access to vaccines that may provide protection against current and future variants," said Albert Bourla, Chairman and Chief Executive Officer, Pfizer. "Vaccines have been and will remain critical to protecting people of all ages against COVID-19. We remain proud of our long-term partnership with the U.S. government in helping to address this pandemic, and of the ongoing impact of vaccination efforts in the U.S. and around the world."

"This agreement will provide additional doses for U.S. residents and help cope with the next COVID-19 wave. Pending regulatory authorization, it will also include an Omicron-adapted vaccine, which we believe is important to address the rapidly spreading Omicron variant," said Sean Marett, Chief Business and Chief Commercial Officer of BioNTech. "We appreciate the continued partnership of the U.S. government in our shared goal to help end this pandemic."

On June 25, 2022, Pfizer and BioNTech reported pivotal data demonstrating the safety, tolerability and immunogenicity of two Omicron-adapted vaccine candidates. These data have been shared with regulators, including the FDA, and a request for U.S. Emergency Use Authorization is planned. The companies have begun manufacturing the Omicron-adapted vaccine candidates at risk so that they can begin deliveries rapidly upon authorization or approval and subsequent recommendation by the U.S. Centers for Disease Control and Prevention’s (CDC), if received, and as directed by the U.S. government. Eligible U.S. residents will continue to receive the vaccine for free, consistent with the U.S. government’s commitment for free access to COVID-19 vaccines.

The Pfizer-BioNTech COVID-19 Vaccine, which is based on BioNTech’s proprietary mRNA technology, was developed by both BioNTech and Pfizer. BioNTech is the Marketing Authorization Holder in the United States, the European Union, the United Kingdom, Canada and other countries, and the holder of emergency use authorizations or equivalents in the United States (jointly with Pfizer) and other countries. Submissions to pursue regulatory approvals in those countries where emergency use authorizations or equivalent were initially granted are planned.

U.S. Indication & Authorized Use

Pfizer-BioNTech COVID-19 Vaccine is FDA authorized under Emergency Use Authorization (EUA) for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 6 months of age and older.

Pfizer-BioNTech COVID-19 Vaccine is FDA authorized to provide:

Primary Series

a 3-dose primary series to individuals 6 months through 4 years of age
a 2-dose primary series to individuals 5 years of age and older
a third primary series dose to individuals 5 years of age and older with certain kinds of immunocompromise
Booster Series

a single booster dose to individuals 5 through 11 years of age who have completed a primary series with Pfizer-BioNTech COVID-19 Vaccine
a first booster dose to individuals 12 years of age and older who have completed a primary series with Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY (COVID-19 Vaccine, mRNA)
a first booster dose to individuals 18 years of age and older who have completed primary vaccination with a different authorized or approved COVID-19 vaccine. The booster schedule is based on the labeling information of the vaccine used for the primary series
a second booster dose to individuals 50 years of age and older who have received a first booster dose of any authorized or approved COVID-19 vaccine
a second booster dose to individuals 12 years of age and older with certain kinds of immunocompromise and who have received a first booster dose of any authorized or approved COVID-19 vaccine
COMIRNATY INDICATION
COMIRNATY (COVID-19 Vaccine, mRNA) is a vaccine approved for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 16 years of age and older.

COMIRNATY is administered as a 2-dose primary series
COMIRNATY AUTHORIZED USES
COMIRNATY (COVID-19 Vaccine, mRNA) is FDA authorized under Emergency Use Authorization (EUA) to provide:

Primary Series

a 2-dose primary series to individuals 12 through 15 years of age
a third primary series dose to individuals 12 years of age and older with certain kinds of immunocompromise
Booster Dose

a first booster dose to individuals 12 years of age and older who have completed a primary series with Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY
a first booster dose to individuals 18 years of age and older who have completed primary vaccination with another authorized or approved COVID-19 vaccine. The booster schedule is based on the labeling information of the vaccine used for the primary series
a second booster dose to individuals 50 years of age and older who have received a first booster dose of any authorized or approved COVID-19 vaccine
a second booster dose to individuals 12 years of age and older with certain kinds of immunocompromise and who have received a first booster dose of any authorized or approved COVID-19 vaccine
Emergency Use Authorization
Emergency uses of the vaccine have not been approved or licensed by FDA, but have been authorized by FDA, under an Emergency Use Authorization (EUA) to prevent Coronavirus Disease 2019 (COVID 19) in individuals 6 months of age and older. The emergency uses are only authorized for the duration of the declaration that circumstances exist justifying the authorization of emergency use of the medical product under Section 564(b)(1) of the FD&C Act unless the declaration is terminated or authorization revoked sooner.

INTERCHANGEABILITY
FDA-approved COMIRNATY (COVID-19 Vaccine, mRNA) and the Pfizer-BioNTech COVID-19 Vaccine FDA authorized for Emergency Use Authorization (EUA) for individuals 12 years of age and older can be used interchangeably by a vaccination provider when prepared according to their respective instructions for use.

The formulations of the Pfizer-BioNTech COVID-19 Vaccine authorized for use in individuals 6 months through 4 years of age, 5 through 11 years of age, and 12 years of age and older are different and should therefore not be used interchangeably.

IMPORTANT SAFETY INFORMATION

Tell your vaccination provider about all the vaccine recipient’s medical conditions, including if the vaccine recipient:

has any allergies
has had myocarditis (inflammation of the heart muscle) or pericarditis (inflammation of the lining outside the heart)
has a fever
has bleeding disorder or is on a blood thinner
is immunocompromised or is on a medicine that affects the immune system
is pregnant, plan to become pregnant, or are breastfeeding
has received another COVID-19 vaccine
has ever fainted in association with an injection
Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY (COVID-19 Vaccine, mRNA) may not protect all vaccine recipients
The vaccine recipient should not receive Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY (COVID-19 Vaccine, mRNA) if the vaccine recipient had a severe allergic reaction to any of its ingredients or had a severe allergic reaction to a previous dose of Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY
There is a remote chance that Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY (COVID-19 Vaccine, mRNA) could cause a severe allergic reaction. A severe allergic reaction would usually occur within a few minutes to 1 hour after getting a dose of the vaccine. For this reason, your vaccination provider may ask the vaccine recipient to stay at the place where the vaccine was administered for monitoring after vaccination. If the vaccine recipient experiences a severe allergic reaction, call 9-1-1 or go to the nearest hospital
Seek medical attention right away if the vaccine recipient has any of the following symptoms:

difficulty breathing, swelling of the face and throat, a fast heartbeat, a bad rash all over the body, dizziness, and weakness
Myocarditis (inflammation of the heart muscle) and pericarditis (inflammation of the lining outside the heart) have occurred in some people who have received the vaccine, more commonly in adolescent males and adult males under 40 years of age than among females and older males. In most of these people, symptoms began within a few days following receipt of the second dose of the vaccine. The chance of having this occur is very low.
Seek medical attention right away if the vaccine recipient has any of the following symptoms after receiving the vaccine, particularly during the 2 weeks after receiving a vaccine dose:

Chest pain
Shortness of breath or difficulty breathing
Feelings of having a fast-beating, fluttering, or pounding heart
Fainting
Unusual and persistent irritability
Unusual and persistent poor feeding
Unusual and persistent fatigue or lack of energy
Persistent vomiting
Persistent pain in the abdomen
Unusual and persistent cool, pale skin
Fainting can happen after getting injectable vaccines, including Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY (COVID-19 Vaccine, mRNA). Sometimes people who faint can fall and hurt themselves. For this reason, your vaccination provider may ask the vaccine recipient to sit or lie down for 15 minutes after receiving the vaccine.
Some people with weakened immune systems may have reduced immune responses to Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY (COVID-19 Vaccine, mRNA).
Additional side effects include rash, itching, hives, swelling of the face, injection site pain, tiredness, feeling weak or lack of energy, headache, muscle pain, chills, joint pain, fever, injection site swelling, injection site redness, nausea, feeling unwell, swollen lymph nodes (lymphadenopathy), decreased appetite, diarrhea, vomiting, arm pain, fainting in association with injection of the vaccine, and irritability.

These may not be all the possible side effects of the vaccine. Call the vaccination provider or healthcare provider about bothersome side effects or side effects that do not go away.

You should always ask your healthcare providers for medical advice about adverse events. Report vaccine side effects to the US Food and Drug Administration (FDA) and the Centers for Disease Control and Prevention (CDC) Vaccine Adverse Event Reporting System (VAERS). The VAERS toll-free number is 1‐800‐822‐7967 or report online to www.vaers.hhs.gov/reportevent.html. You can also report side effects to Pfizer Inc. at www.pfizersafetyreporting.com or by calling 1-800-438-1985

On June 29, 2022 Avid Bioservices, Inc. (NASDAQ:CDMO), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality development and manufacturing services to biotechnology and pharmaceutical companies, reported financial results for the fourth quarter and full fiscal year, ended April 30, 2022 (Press release, Avid Bioservices, JUN 29, 2022, View Source [SID1234616360]).

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Highlights from the Quarter and Fiscal Year Ended April 30, 2022, and Other Events:

"We made considerable progress during fiscal 2022. During the year, we achieved revenue of $120 million, representing a doubling of revenues recorded in fiscal 2020. Notably, Q4 fiscal 2022 was the eighth consecutive quarter of operational profitability for the company. The company signed net new project orders for $155 million in fiscal 2022, leading to a backlog of $153 million; Avid’s largest backlog to-date. Supporting this growth, as well as that which we anticipate in the coming years, our facilities and service expansions continue to proceed according to plan," stated Nick Green, president and chief executive officer of Avid Bioservices.

"Our momentum in fiscal 2022 was driven in part by the exceptional performance of our enhanced commercial team. Over the last six months, we expanded the sales teams for both our mammalian and our cell and gene therapy businesses. We also expanded our business operations team to best support our growing project pipeline. This new organization has had great success, highlighted by the fact that it signed the same number of new projects in the second half of fiscal 2022 as signed in all of fiscal 2021.

"During the period, our facilities and service expansions continued to advance on a timeline that will allow us to meet the demand of existing customers that are expanding their manufacturing work with us as well as our newest and prospective customers. With respect to our 53,000 square foot cell and gene therapy facility, we recently completed the first phase of a two-phase construction plan, opening our new analytical and process development laboratories eight months to the day after we announced our intention to expand into the cell and gene therapy business. Construction of the CGMP suites for our cell and gene therapy facility, the second phase of this expansion, remains on track and those manufacturing suites are expected to come online in mid-calendar 2023. The expansion of our Myford facility, which houses our mammalian operations, is also being constructed in a two-phase process. As reported last quarter, the company completed the first phase with the opening of a new downstream suite. The second phase of this project is focused on the Myford South facility and is on track to come online at the beginning of calendar year 2023, which at our current growth rate will be ideally timed to provide much needed capacity for fiscal 2024.

"Finally, we are delighted to announce a further expansion of our process development capacity for our mammalian cell business. Once complete at the end of this calendar year, these new suites will double our current process development capacity which came online in October of 2019. This new line will significantly increase capacity at the front-end of our mammalian cell business, which is critical to the efficient on-boarding of new clients. It is expected that this expansion will cost approximately $6 million, and upon completion, will have the potential to generate approximately $20 million in additional revenue capacity.

"Given the growth momentum achieved during fiscal 2022, our significant year-end backlog, and the increase in demand anticipated during fiscal 2023, we are pleased to announce revenue guidance for fiscal 2023 of between $140 and $145 million."

Financial Highlights and Guidance

The company is providing revenue guidance for fiscal 2023 of $140 million to $145 million, a 17% – 21% increase over fiscal 2022.

Revenues for the fourth quarter of fiscal 2022 were $31.2 million, representing a 13% increase compared to $27.6 million recorded in the prior year period. The increase in revenues for the quarter can primarily be attributed to an increase in the scope of in-process and completed manufacturing runs and an increase in process development revenues primarily associated with services provided to new customers as compared to the prior year period. For the 2022 full fiscal year, revenues were $119.6 million, a 25% increase compared to $95.9 million in the prior year period. The increase in revenues for the 2022 full fiscal year, as compared to the prior year period can primarily be attributed to an increase in the number and scope of in-process and completed manufacturing runs, unutilized reserved capacity fees, and process development revenues.

As of April 30, 2022, revenue backlog was $153 million, representing a net increase of 30% compared to $118 million at the end of fiscal 2021. The company expects to recognize the majority of this backlog during fiscal 2023.

Gross margin for the fourth quarter of fiscal 2022 was 22%, compared to a gross margin of 29% for the fourth quarter of fiscal 2021. Factors impacting the gross margin for the quarter were primarily from increases in costs associated with the growth of our business and our facility expansions including compensation and benefit expenses as well as increases in facility and related expenses, partially offset by higher revenues during the period. Gross margin for the 2022 full fiscal year was 31%, consistent with 31% for the prior year period.

Selling, general and administrative expenses ("SG&A") for the fourth quarter of fiscal 2022 were $5.9 million, an increase of 17% as compared to $5.1 million recorded for the fourth quarter of fiscal 2021. The increase in SG&A for the fourth quarter was primarily due to higher compensation and benefit expenses as well as increased facility and related expenses. For the 2022 full fiscal year, SG&A expenses were $21.2 million as compared to $17.1 million for the prior year. The increase in SG&A during the 2022 full fiscal year was primarily due to compensation and benefit expenses, facility and related expenses, advertising costs and legal and accounting fees.

During the fourth quarter of fiscal 2022 we recorded a non-cash income tax benefit of $115 million, or $1.63 per diluted share, due to release of our valuation allowance recorded against the company’s deferred tax assets (DTAs). The company previously maintained a valuation allowance on its DTAs until there is sufficient evidence to support the reversal of all or some portion of these allowances. On a periodic basis, the company reassesses the valuation allowance of its DTAs, weighing all positive and negative evidence, to assess if it is more-likely-than-not that some or all of the company’s DTAs will be realized. As of the fourth quarter of fiscal 2022, the company has demonstrated profitability and cumulative pretax income as well as forecasting revenue growth. After assessing both the positive and negative evidence, the company determined that it was more-likely-than-not that its DTAs would be realized and released the valuation allowance related to federal and state DTAs as of April 30, 2022.

For the fourth quarter of fiscal 2022, the company recorded net income attributable to common stockholders of $115.6 million or $1.87 per basic and $1.65 per diluted share, as compared to a net loss attributable to common stockholders of $2.7 million or $0.04 per basic and diluted share, for the fourth quarter of fiscal 2021. For the 2022 full fiscal year, the company recorded net income attributable to common stockholders of $127.7 million or $2.08 per basic and $1.84 per diluted share, compared to net income attributable to common stockholders of $3.3 million or $0.06 per basic and diluted share, for the 2021 full fiscal year. Excluding the non-cash income tax benefit of $115.0 million recorded during the fourth quarter of fiscal 2022, the company’s net income attributable to common stockholders was approximately $600 thousand or $0.01 per basic and diluted share for the quarter, and $12.7 million or $0.21 per basic and diluted share for the full fiscal year 2022.

The company reported $126.2 million in cash and cash equivalents as of April 30, 2022 compared to $169.9 million as of the prior fiscal year ended April 30, 2021.
More detailed financial information and analysis may be found in Avid Bioservices’ Annual Report on Form 10-K, which will be filed with the Securities and Exchange Commission today.

Recent Corporate Developments

The company’s commercial team signed multiple new project orders during the fourth quarter, totaling approximately net $44 million. For the 2022 full fiscal year, the company signed new project orders totaling approximately net $155 million. These projects span all areas of the business, from process development to commercial manufacturing.

The company continues to make progress with both the Myford South expansion, as well as the construction of its new dedicated cell and gene therapy facility. The company currently expects to complete the second phase of its Myford South expansion, which includes both upstream and downstream CGMP manufacturing suites, during the first quarter of calendar 2023. With respect to the cell and gene therapy business, the company brought its process and analytical development capacity online in mid-June 2022. The company remains on track to bring the CGMP manufacturing suites online in mid-calendar 2023. Please visit the Avid website Facilities page for more information about the company’s expansions and videos documenting progress (View Source).
Statement Regarding Use of Non-GAAP Financial Measures

The company uses certain non-GAAP financial measures such as non-GAAP adjusted net income, free cash flow, as well as adjusted EBITDA. The company uses these non-GAAP financial measures for financial and operational decision making and as a means to evaluate period-to-period comparisons. The company believes that they provide useful information about operating results, enhance the overall understanding of our operating performance and future prospects, and allow for greater transparency with respect to key metrics used by management in our financial and operational decision making. These non-GAAP financial measures exclude amounts that the company does not consider part of ongoing operating results when planning and forecasting and when assessing the performance of the organization and our senior management. The company computes non-GAAP financial measures using the same consistent method from quarter to quarter and year to year, and may consider whether other significant items that arise in the future should be excluded from our non-GAAP financial measures.

The company reports non-GAAP financial measures in addition to, and not as a substitute for, or superior to, measures of financial performance prepared in accordance with U.S. generally accepted accounting principles (GAAP). These non-GAAP financial measures are not based on any comprehensive set of accounting rules or principles, differ from GAAP measures with the same names, and may differ from non-GAAP financial measures with the same or similar names that are used by other companies. The company believes that non-GAAP financial measures should only be used to evaluate our results of operations in conjunction with the corresponding GAAP financial measures, and encourages investors to carefully consider our results under GAAP, as well as the supplemental non-GAAP information and the reconciliations between these presentations, to more fully understand our business.

Non-GAAP net income excludes stock-based compensation; business transition and related costs including corporate initiatives into new business activities such as our expansion into viral vectors for the cell and gene therapy sector of the market and other costs directly associated with such activities, and severance and related expenses; non-cash interest expense on convertible senior notes for the accretion of the issuance costs associated with our convertible senior notes; and other income or expense items. Adjusted EBITDA excludes non-cash operating charges for stock-based compensation, depreciation and amortization as well as non-operating items such as interest income, interest expense, and income tax expense or benefit. For the reasons explained above, adjusted EBITDA also excludes certain business transition and related costs. The company also uses measures such as free cash flow, which represents cash flow from operations less cash used in the acquisition and disposition of capital.

Additionally, non-GAAP net income and adjusted EBITDA are key components of the financial metrics utilized by the company’s compensation committee to measure, in part, management’s performance and determine significant elements of management’s compensation. The company encourages investors to carefully consider its results under GAAP, as well as its supplemental non-GAAP information and the reconciliation between these presentations, to more fully understand its business. Reconciliations between GAAP and non-GAAP financial measures included at the end of this press release.

Conference Call

Avid will host a conference call and webcast this afternoon, June 29, 2022, at 4:30 PM EST (1:30 PM PST).

To listen to the conference call, please dial (877) 312-5443 or (253) 237-1126 and request the Avid Bioservices conference call. To listen to the live webcast, or access the archived webcast, please visit: View Source

On June 29, 2022 Aileron Therapeutics (Nasdaq: ALRN), a chemoprotection oncology company that aspires to make chemotherapy safer and thereby more effective to save more patients’ lives, reported interim data from its Phase 1b chemoprotection trial of patients with advanced p53-mutated NSCLC undergoing treatment with first-line carboplatin plus pemetrexed with or without immune checkpoint inhibitors (Press release, Aileron Therapeutics, JUN 29, 2022, View Source [SID1234616359]). Aileron plans to stop further enrollment in the NSCLC trial and to apply key learnings from the interim analysis to strengthen the Phase 1b breast cancer trial in accordance with clinical and regulatory precedents.

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The interim analysis consisted of the first 20 patients randomized to ALRN-6924 0.3 mg/kg plus carboplatin/pemetrexed (n=11) or placebo plus carboplatin/pemetrexed (n=9).1,2 ALRN-6924-treated patients were able to stay on chemotherapy treatment longer, completing 93% of the first 4 cycles of carboplatin/pemetrexed administered compared to 78% on placebo.2,3 This imbalance of completed cycles between the treatment arms may have introduced a bias against ALRN-6924 on the composite primary endpoint. The imbalance increases further when looking at percentages of patients completing 6 cycles of treatment (79% on ALRN-6924 versus 57% on placebo). This is reflected in the progression free survival, which was 4.6 months in the ALRN-6924 arm versus 3.2 months in the placebo arm. The composite primary endpoint consisted of the proportion of treatment cycles free of Grade ≥3 neutropenia, Grade ≥3 thrombocytopenia, Grade ≥3 anemia, blood transfusions, and the use of growth factors, as well as dose reductions or dose delays within the first 4 cycles of treatment. ALRN-6924-treated patients demonstrated 56% of cycles free from these Grade ≥3 hematologic toxicities and related events compared to 50% on placebo.

"We remain passionate about advancing ALRN-6924 for patients with p53-mutated cancer, and these interim NSCLC findings have significantly helped to clarify our development path toward that goal. We are very encouraged by the finding that ALRN-6924-treated patients were able to complete more cycles of chemotherapy in the NSCLC trial, but unfortunately it also appears that this may have worked against us given the nature of the exploratory composite primary endpoint. The more cycles patients completed the more opportunity they had to experience toxicities. This introduced an imbalance of toxicities between the active and placebo arms and, may have resulted in a bias against ALRN-6924 on the composite primary endpoint," said Manuel Aivado, M.D., Ph.D., President and CEO of Aileron.

While the ALRN-6924 0.3 mg/kg dose previously demonstrated protection against topotecan-induced hematologic toxicities in Aileron’s small cell lung cancer trial, Aileron believes that a higher dose level could provide more durable cell cycle arrest and, thus more chemoprotection against certain chemotherapies, including carboplatin/pemetrexed. This is supported by the recently generated data from the company’s healthy volunteer study. In that study, serum MIC-1 levels were measured as an indicator of the duration of effect of ALRN-6924, including the duration of cell cycle arrest. Increasing dose levels of ALRN-6924 elicited more durable p53 activation, which correlates with cell cycle arrest in the bone marrow. Cell cycle arrest is a basis for protecting cells from chemotherapy.

Dr. Aivado continued, "By stopping the NSCLC trial, we plan to fully focus our resources on our Phase 1b breast cancer trial to continue our development of ALRN-6924 to protect p53-mutated cancer patients from chemotherapy-induced side effects. Neoadjuvant chemotherapy for breast cancer is associated with frequent severe neutropenia in cycle 1, and we believe this offers a well-established endpoint, which has been used to secure FDA approval of multiple supportive care drugs. This endpoint also obviates any potential imbalance in the number of cycles completed on ALRN-6924 versus placebo. The breast cancer trial also gives us the ability to evaluate protection against alopecia, which occurs in more than 90% of breast cancer patients on neoadjuvant chemotherapy compared to less than 10% of patients receiving carboplatin/pemetrexed."

The learnings from the NSCLC interim analysis create an opportunity for Aileron to take several steps to strengthen the Phase 1b breast cancer trial, including revising the primary endpoint to duration of severe neutropenia in cycle 1 and changing the chemotherapy regimen to a simultaneous administration of doxorubicin plus cyclophosphamide and docetaxel, referred to as TAC. Additionally, the company plans to modify the dosing strategy for the trial and will not further enroll additional patients in the ongoing 0.3 mg/kg and 0.6 mg/kg dose cohorts.

Additional NSCLC Trial highlights (Cycles 1-6)

Of the 83 cycles of carboplatin/pemetrexed administered, Grade ≥3 hematologic toxicities were observed in 25 cycles (30%): 18 cycles with 28 instances4 of hematologic toxicities on ALRN-6924, and 7 cycles with 10 instances of hematologic toxicities on placebo.
One patient receiving ALRN-6924 accounted for 15 of the total 28 Grade ≥3 hematologic instances observed on that arm, or 53%.
Grade 4 events5 were infrequent, occurring in 1 patient on ALRN-6924 and 2 patients on placebo.
5 of 11 patients treated with ALRN-6924 completed 6 planned cycles (45%) versus 1 out of 9 placebo patients (11%).
Frequency of patients experiencing Grade ≥3 hematologic events:
Treatment
(n of patients) Patients with grade≥3
neutropenia
n (%) Patients with grade ≥3
thrombocytopenia
n (%) Patients with grade ≥3
anemia
n (%)
ALRN-6924 (n=11) 5 (45%) 5 (45%) 1 (9%)
Placebo (n=9) 2 (22%) 4 (44%) 2 (22%)

Investor Webcast Details

Aileron will host a conference call and webcast today at 8:00 am ET to discuss these data and the company’s planned strategic prioritization of its breast cancer trial. The conference call can be accessed by dialing 844-838-0770 (United States) or 213-320-2558 (International) with the conference code 3218779. A live webcast may be accessed using the link here, or by visiting the "Events and Presentations" page in the investors section of the Aileron website at www.aileronrx.com. After the live webcast, the event will be archived on the Company’s website for approximately 30 days after the call.