On May 16, 2022 Nuvo Pharmaceuticals Inc. (TSX:MRV; OTCQX:MRVFF) d/b/a Miravo Healthcare (Miravo or the Company), a Canadian-focused healthcare company with global reach and a diversified portfolio of commercial products, reported its financial and operational results for the three months ended March 31, 2022 (Press release, Nuvo Pharmaceuticals, MAY 16, 2022, View Source [SID1234614654]). For further details on the results, please refer to Miravo’s Management, Discussion and Analysis (MD&A) and Condensed Consolidated Interim Financial Statements for the three months ended March 31, 2022, which are available on the Company’s website (www.miravohealthcare.com). All figures are in Canadian dollars, unless otherwise noted.

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Key Developments

Three months ended March 31, 2022 include the following:

Total revenue was $15.6 million, an increase of 8% compared to $14.4 million for the three months ended March 31, 2021. Adjusted total revenue(1) was $15.7 million, an increase of 8% compared to $14.5 million for the three months ended March 31, 2021.
Net income was $2.0 million compared to net loss of $18.0 million for the three months ended March 31, 2021. Adjusted EBITDA(1) was $3.1 million, a decrease of 28% compared to $4.4 million for the three months ended March 31, 2021.
Revenue related to the Blexten franchise, Cambia and Suvexx was $8.1 million, an increase of 38% compared to revenue of $5.9 million for the three months ended March 31, 2021. Total Canadian prescriptions of Blexten, Cambia and Suvexx increased by 21%, 1% and 120%, respectively compared to the three months ended March 31, 2021.
The Company repaid $3.5 million (US$2.8 million) of the Amortization Loan to Deerfield Management Company, L.P. (Deerfield).
As at March 31, 2022, cash and cash equivalents were $26.5 million.
(1)

Non-IFRS financial measure. These measures are not recognized under IFRS and do not have standardized meanings prescribed by IFRS. See the Non-IFRS Measures section for definitions, reconciliations and the basis of presentation of the Company’s non-IFRS measures.

Business Update

In May 2022, Miravo become aware that the United States’ Food & Drug Administration (FDA) approved Apotex Inc.’s abbreviated new drug application for a generic version of Pennsaid 2% on May 6, 2022. Miravo’s partner in the United States owns the Pennsaid 2% intellectual property rights in the United States and has carriage and full decision-making authority with respect to any litigation related to this matter. Miravo is the exclusive manufacturer of Pennsaid 2% for its U.S. partner. The entry of a generic version of Pennsaid 2% may have a material and adverse impact on Miravo’s Production and Service Business segment.
In May 2022, Miravo filed U.S., Canadian, European and PCT patent applications for a reformulated and improved version of Resultz. This new formulation maintains the original 5-minute treatment claim, but is now enhanced with a 100% effectiveness claim for killing nits (the lice eggs) in addition to head lice. The Company believes this enhanced efficacy against nits adds value to existing Resultz partners, as well as other companies active in the head lice category globally who may be interested in licensing the technology. The Company will begin the partnering process for this new intellectual property during Q2 2022. Additional basic development work is anticipated to be conducted to support the new product.
In February 2022, the United States District Court for the District of New Jersey granted a motion for summary judgment filed by Dr. Reddy’s Laboratories Inc. (Dr. Reddy’s). As a result, the asserted claims of Nuvo Pharmaceuticals (Ireland) DAC’s (Miravo Ireland) U.S. Patent Nos. 8,858,996 (the ‘996 Patent) and 9,161,920 (the ‘920 Patent) related to Vimovo in the U.S. were found to be invalid. Both the ‘996 and the ‘920 patents were to expire May 31, 2022. Miravo Ireland and its partner have not appealed this decision.
In February 2022, Blexten for pediatric use in patients 4 years of age and older* was commercially launched in Canada. The pediatric use includes two new dosage formats; a 2.5mg/mL oral solution and a 10mg orodispersible tablet (quick melt) for the treatment of the symptoms of seasonal allergic rhinitis and chronic spontaneous urticaria (such as itchiness and hives). The pediatric formats are available to patients with a prescription from their healthcare provider.
"Our Commercial Business segment continues to perform well with Q1 year-over-year adjusted total revenue growth driven by increased Blexten, Suvexx and Cambia prescriptions. Our final $375,000 sales-based milestone for the Blexten business was triggered during the quarter. This payment recognized the achievement of $60 million in cumulative net sales of Blexten in Canada – a milestone we are very proud of. As we look to the remainder of 2022, we anticipate continued year-over-year quarterly growth in the Commercial Business segment consistent with the seasonality this business has experienced in the past," said Jesse Ledger, Miravo’s President & CEO. "The financial impact of generic competition on our U.S. Vimovo royalty stream is now fully realized resulting in revenue stabilization in our Licensing and Royalty Business segment moving forward. Certain shipments to international customers were produced, but due to COVID-related delays, were not delivered in time to be recognized as revenue during Q1 in our Production and Service Business segment. While our business continues to be impacted by the COVID-19 pandemic, we are encouraged to see increasing numbers of patient/physician visits and look forward to a return to pre-COVID activity levels in the coming quarters."

First Quarter 2022 Financial Results

Adjusted total revenue was $15.7 million for the three months ended March 31, 2022 compared to $14.5 million for the three months ended March 31, 2021. The $1.2 million increase in adjusted total revenue in the current quarter was primarily attributable to an increase of $2.4 million of revenue in the Commercial Business segment, offset by a decrease of $0.9 million of revenue in the Production and Service Business segment combined with a decrease of $0.3 million in the Licensing and Royalty Business segment.

Revenue attributable to the Commercial Business segment increased during the three months ended March 31, 2022 due to a $2.2 million increase in sales of the Company’s promoted products (Blexten, Cambia, Suvexx and NeoVisc) and a $0.2 million increase in sales of the Company’s mature products.

* Blexten (bilastine) is indicated for the symptomatic relief of nasal and non-nasal symptoms of seasonal allergic rhinitis and chronic spontaneous urticaria (e.g. pruritus and hives) in patients 4 years of age and older with a body weight of at least 16 kg.

The Production and Service Business segment revenue decreased during the three months ended March 31, 2022, primarily due to a decrease in Pennsaid 2% product sales, slightly offset by an increase in sales of Pennsaid and Resultz.

The decrease in revenue attributable to the License and Royalty business segment during the three months ended March 31, 2022 was primarily attributable to a $0.5 million reduction in U.S. Vimovo royalty revenue due to a step-down in royalty to 5% of net sales compared to 10% of net sales in the comparative quarter, as well as a $0.1 million reduction in the Company’s Vimovo ex-U.S. royalties. In the current quarter, the decline in license revenue was offset by a $0.2 million milestone payment related to a sublicensee agreement for Vimovo ex-U.S.

Adjusted EBITDA was $3.1 million for the three months ended March 31, 2022 compared to $4.4 million for the three months ended March 31, 2021. During the three months ended March 31, 2022, a $0.9 million increase in gross profit from the Commercial Business segment was more than offset by a $0.3 million decrease in the contribution from the License and Royalty Business segment, a $0.7 million decrease in gross profit contribution from the Production and Service Business segment and a $0.9 million increase in G&A expenses.

Non-IFRS Measures

The Company discloses non-IFRS financial measures (adjusted total revenue, adjusted EBITDA, and cash value of loans) and non-IFRS ratios (adjusted EBITDA per share and net debt leverage ratio) that are not recognized under and do not have standardized meanings prescribed by IFRS. Accordingly, such measures are not necessarily comparable and may not have been calculated in the same way as similarly named financial measures presented by other companies. These measures should be considered as supplemental in nature and not as a substitute for related financial information prepared in accordance with IFRS. The Company believes that shareholders, investment analysts and other readers find such measures and ratios helpful in understanding and assessing the Company’s financial performance. We utilize these measures in managing our business, including as means of performance measurement, cash management, debt compliance and assessing leverage and borrowing capacity. Because non-IFRS financial measures and non-IFRS ratios do not have standardized meanings prescribed under IFRS, securities regulations require that such measures be clearly defined, identified, and for non-IFRS financial measures, reconciled to their nearest IFRS measure. The applicable definition, calculation and reconciliation of each such measure used in this press release is provided below.

Adjusted Total Revenue

The Company defines adjusted total revenue as total revenue, plus amounts billed to customers for existing contract assets, less revenue recognized upon recognition of a contract asset. Management believes adjusted total revenue is a useful supplemental measure to determine the Company’s ability to generate cash from its customer contracts used to fund its operations.

The following is a summary of how adjusted total revenue is calculated, reconciled to the nearest IFRS measure:

EBITDA refers to net income (loss) determined in accordance with IFRS, before depreciation and amortization, net interest expense (income) and income tax expense (recovery). The Company defines adjusted EBITDA as EBITDA, plus amounts billed to customers for existing contract assets, inventory step-up expenses, stock-based compensation expense, loss on fair value of derivative liabilities, loss on fair value of contingent and variable consideration, impairment loss, foreign currency loss, other losses less revenue recognized upon recognition of a contract asset, stock-based compensation recovery, gain on fair value of derivative liabilities, gain on fair value of contingent and variable consideration, impairment recovery, foreign currency gain and other income. Management believes adjusted EBITDA is a useful supplemental measure to determine the Company’s ability to generate cash available for working capital, capital expenditures, debt repayments, interest expense and income taxes.

The following is a summary of how EBITDA and adjusted EBITDA are calculated, reconciled to the nearest IFRS measure:

(1) Income tax expense for the three months ended March 31, 2022 includes $0.3 million for deferred income tax due to the utilization of loss carryforwards that were previously recognized [$0.3 million for the three months ended March 31, 2021].

(2) The Company’s derivative liabilities are measured at fair value through profit or loss at each reporting date. As a result of the decrease in the share price in the current quarter and a decrease in the volatility of the Company’s shares, amongst other inputs, the value of the Company’s derivative liabilities decreased and the Company recognized a net non-cash gain of $3.3 million on the change in fair value of derivative liabilities for the three months ended March 31, 2022.

Management to Host Conference Call/Webcast

Miravo’s 2022 Annual Meeting of Shareholders (Meeting) will be held as an online meeting only. The Meeting will take place on Monday, May 16, 2022 (today) at 9:00 a.m. Registered shareholders can attend the Meeting online, vote shares electronically if they have not voted by proxy in advance of the Meeting in accordance with the proxy instructions, and submit questions during the Meeting. You will need to have your 16-digit Control Number (the Control Number) to participate in the Meeting. If you are a shareholder and do not have a Control Number or if you are not a Miravo shareholder, you can attend the Meeting as a guest, but you will not be able to vote at the Meeting.

The link to participate in the Meeting is: www.virtualshareholdermeeting.com/mrv2022. If you encounter any difficulties accessing the virtual meeting during the check-in or meeting time, please call the technical support number that will be posted on the Virtual Shareholder Meeting log in page.

On May 16, 2022 Vyant Bio, Inc. ("Vyant Bio", "Company") (Nasdaq: VYNT) is an innovative biotechnology company reinventing drug discovery for complex neurodevelopmental and neurodegenerative disorders (Press release, Vyant Bio, MAY 16, 2022, View Source [SID1234614670]). The Company’s central nervous system ("CNS") drug discovery platform combines human-derived organoid models of brain disease, scaled biology, and machine learning. Today, Vyant Bio expects to file its Form 10-Q for the First Quarter 2022 with the Securities and Exchange Commission and will report its First Quarter 2022 highlights and business updates this afternoon in a conference call and webcast scheduled for 4:30 pm ET.

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"During the first quarter of 2022, we progressed our scientific work by using our proprietary drug discovery platform centered on human iPSC-derived neurospheroids to unveil novel targets and potentially disease-modifying therapeutics to treat patients suffering from severe neurodevelopmental and neurodegenerative diseases. We are validating important data that we believe will demonstrate our ability to de-risk the selection of therapeutics that can rescue a disease phenotype. We believe current preclinical models are not sufficiently predictive, and many current therapies for CNS disorders only address the symptoms and do not reverse the effects of neurological diseases. Our focus during the first 90 days of 2022 was to progress our platform validation efforts to advance our CNS programs that are designed to identify disease-modifying therapeutics, consistent with our plans and prior disclosures to our shareholders," stated Jay Roberts, the Company’s Chief Executive Officer.

"As we highlighted in our last earnings conference call, we believe our focus on complex neurodevelopmental and neurodegenerative disorders addresses significant unmet medical needs, and will lead to the identification of important, disease modifying therapies for major causes of death and disability worldwide. Our Rett patient-derived neural spheroids show a robust and reproducible disease-specific phenotype that can be quantified in an unbiased manner across dozens of endpoints. By phenotypic screening of our Rett neural spheroids, we identified VYNT-0126 which has consistently shown a dose-dependent unique rescue of the RTT functional phenotype with a differentiated mechanism of action from other Rett syndrome clinical candidates. Our ongoing work on CDKL5 and familial Parkinson’s disease has further established that our discovery platforms represent a new, unique robust model for human-first CNS drug discovery" stated Dr. Robert Fremeau, Chief Scientific Officer of Vyant Bio.

FIRST QUARTER 2022 FINANCIAL RESULTS

Cash and cash equivalents totaled $16.4 million as of March 31, 2022. The Company implemented two new vehicles to facilitate the raising of additional equity capital at the Company’s option with the finalization of the Lincoln Park Equity line of credit allowing access to raise up to $15 million, as well as signing a $14.5 million ATM with Canaccord Genuity. The Company’s current cash balances, future proceeds from the sale of vivoPharm and future proceeds from the equity line of credit and ATM are expected to fund operations well into 2023.

During the first quarter of 2022, the Company continued the process of divesting the vivoPharm business which is expected to complete in 2022. Therefore, the vivoPharm business is classified as a "held-for-sale" asset, and its financial information as "discontinuing operations".

The Company’s loss from continuing operations aggregated $4.4 million in the first quarter of 2022 and included non-cash depreciation and amortization as well as stock-based compensation expenses of $98 thousand and $278 thousand, respectively, and one-time severance charges of $437 thousand. Discontinuing operations net loss for the March 31, 2022 quarter aggregated $4.8 million and included a non-cash impairment charge of $4.3 million resulting from changed market conditions for contract research organizations from December 31, 2021 to March 31, 2022.

Total revenue from continuing operations increased 49.3%, or $100 thousand, to $303 thousand for the three months ended March 31, 2022, as compared with $222 thousand for the three months ended March 31, 2021.

Cost of goods sold – service from continuing operations totaled $38 thousand and $64 thousand, respectively, for the three months ended March 31, 2022 and 2021, resulting in a cost of goods sold of 40% and 66%, respectively, of service revenue.

Cost of goods sold – product costs decreased by 12%, or $48 thousand to $348 thousand for the three months ended March 31, 2022, as compared with $396 thousand for the three months ended March 31, 2021.

Research and development expenses increased by 89%, or $731 thousand, to $1.6 million for the three months ended March 31, 2022 from $820 thousand for the three months ended March 31, 2021. This increase is principally due a $336 thousand increase in payroll-related and consulting expenses, a $315 thousand increase in research and development activities at our Maple Grove facility, and $48 thousand related to moving to a new facility in California.

Selling, general and administrative expenses increased by 128%, or $1.5 million, to $2.8 million for the three months ended March 31, 2022, as compared with $1.2 million for the three months ended March 31, 2021. The 2021 period reflects the Company as a privately-held company whereas the 2022 period reflect the Company as a publicly-held company. The quarter ended March 31, 2022 includes incremental $564 thousand of payroll-related expenses, including one-time contractual severance benefits for two former employees of $437 thousand. The Company incurred incremental professional services fees of $472 thousand in the first quarter of 2022 as compared with the same prior-year period related to accounting, audit and other professional services and incurred $418 thousand of additional insurance expense.

Vyant Bio’s Conference Call and Webcast and Information

Vyant Bio’s management will host a conference call on Monday, May 16, 2022 at 4:30pm ET to discuss the first quarter 2022 results and provide strategic business updates as well as answer questions. Event information is below:

The live event will be recorded and available for replay. The conference call and webcast details are also included inside the Investors section of the Vyant Bio corporate website at www.vyantbio.com.

On May 16, 2022 Orna Therapeutics, a biotechnology company pioneering a new class of fully engineered circular RNA (oRNA) therapies, reported that data from its lead isCAR program that validates the potential of the company’s novel oRNA technology and LNP delivery platform (Press release, Orna Therapeutics, MAY 16, 2022, View Source [SID1234614686]). Based on advances in the expression of oRNA as well as its delivery to immune cells, Orna has demonstrated tumor suppression and eradication in an animal model pointing to the possibility that oRNA-LNP based cancer therapies could eventually overtake cell therapies .

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Additional data to be presented demonstrate the utility of Orna’s proprietary FoRCE screening platform which has enabled the company to discover and characterize a major new resource for protein expression based on internal ribosome entry sites (IRESs). Orna will also present data showing the development of novel immunotropic lipid nanoparticles (LNPs), and the potential of oRNA in genetic muscle disease and vaccines. View full presentations from ASGCT (Free ASGCT Whitepaper) on our website here.

"At Orna, we’ve created the world’s leading circular RNA company and are building a platform and pipeline with the potential to change the way we treat disease," said Tom Barnes, PhD, Orna’s Chief Executive Officer and oral presenter at ASGCT (Free ASGCT Whitepaper). "Presented for the first time, these data suggest our oRNA technology and LNP delivery favorably combine to maximize our reach into multiple therapeutic areas – validating our expanded pipeline beyond cancer to include muscle genetic diseases and vaccines."

isCAR: Revolutionizing CAR-T Cell Therapy with the Possibility of Eradicating Tumors
Our lead program is an in situ CAR therapy that combines oRNA and custom engineered LNPs to create modified immune cells within the patient. This easily redosable format would not require patient lymphodepletion and would allow for reliable dose control, overcoming barriers of ex vivo CAR-T therapies. Data being presented demonstrate that oRNA-LNP can eradicate cancer cells in an animal model. Additional iterative data in rodents and non-human primates gives Orna confidence that this may successfully translate into humans.

"We are excited about the preclinical results in our lead isCAR program as we clearly see the opportunity to overcome significant hurdles in current ex vivo approaches, suggesting that oRNA-LNP based cancer therapies may eventually overtake cell therapies," said Robert Mabry, PhD and Chief Scientific Officer at Orna. "We believe that data from iterative animal studies can support our plans to deliver in situ CAR-T therapies to the clinic."

FoRCE: Formulated oRNA Cell-based Evaluation Platform
Orna is also presenting new data from our proprietary FoRCE screening platform, which captures the entire oRNA production, formulation, and evaluation process in an arrayed and automated format. In a first application, Orna has screened and characterized thousands of IRES elements in multiple primary human cell types. IRES identification and development is critical for optimizing oRNA function via tunable protein expression. Orna has discovered many novel IRES elements that drive oRNA expression to levels well above those of standard IRES elements, including some that show differential activity across cell types. These results open a new technological toolkit for driving protein expression from circular RNA.

Breadth of Platform
Orna has extended its oRNA-LNP technology into several other indications including Duchenne Muscular Dystrophy and vaccines and believes there are many additional opportunities this technology can bring to existing therapeutics.

Duchenne Muscular Dystrophy (DMD):
Orna will present data highlighting the ease of working with very large oRNAs. Data demonstrate, for the first time, non-viral delivery of a large, full-length, dystrophin-encoding RNA in human cells, as well as in vivo delivery of smaller length versions in mouse models. These data are an encouraging first step on the path to delivering full-length gene therapy to patients with DMD.

Vaccines:
Orna is investigating the suitability of oRNA combined with intramuscularly administered immunotropic LNPs for vaccine applications, including for COVID-19. The oRNA half-life observed in muscle and immune cells, combined with the intramuscular administration of immunotropic lipids, suggests that oRNA-LNP technology may be beneficially applied to vaccine development.

Conference presentation details are shared below.

Oral Presentations:
In situ CAR Therapy Using oRNA Lipid Nanoparticles Regresses Tumors in Mice
Presenter: Tom Barnes, Ph.D., CEO
Date/Time/Location: Monday, May 16, 2022 from 9:10 – 9:45 a.m. ET in Room 207
Session: Scientific Symposium: Function and Therapeutics Applications of Circular RNAs (circRNAs)

Discovery of Translation Initiation Elements Enabled by a Parallel Arrayed Screen of Full-length Viral UTRs in Synthetic Circular RNA
Presenter: Alexander Wesselhoeft, Ph.D., Director, Molecular Biology
Date/Time/Location: Monday, May 16, 2022 from 11:30 – 11:45 a.m. ET in Salon H
Session: Oral Abstract Session: Oligonucleotide Therapeutics

Poster Presentations:
Improved Immune Cell Expression with Circular RNA (oRNA) in vivo
Presenter: Kevin Kauffman, Ph.D., Principal Scientist
Date/Time/Location: Monday, May 16, 2022 at 5:30 p.m. ET in Hall D
Session: Poster Session: Oligonucleotide Therapeutics I

Systemic Delivery of Circular RNA Encoding Partial Dystrophins and Expression in Skeletal Muscle
Presenter: Tatiana Fontelonga, Ph.D., Scientist
Date/Time/Location: Tuesday, May 17, 2022 at 5:30 p.m. ET in Hall D
Session: Poster Session: Oligonucleotide Therapeutics II

On May 16, 2022 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported financial results and business highlights for the first quarter of 2022 (Press release, Hookipa Biotech, MAY 16, 2022, View Source [SID1234614590]).

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"We observed strong external validation of our novel arenaviral platform in the first quarter with our collaboration agreement with Gilead and our capital raise, which drew funding from new and existing top-tier investors," said Joern Aldag, Chief Executive Officer at HOOKIPA. "On the heels of our AACR (Free AACR Whitepaper) data presentations, which showcased T cell and tumor response with our technology alone and in novel combinations, we remain focused on advancing our portfolio across cancer types. We look forward to sharing Phase 1 data from our HB-200 program mid-year and early Phase 2 data on HB-200 in combination with pembrolizumab in late 2022, as well as progressing with IND preparations for our HB-300 program in prostate cancer."

Quarter Highlights

In January 2022, the first patient was dosed in the Phase 2 trial to assess HB-200 in combination with pembrolizumab as 1st-line and 2nd-line treatment for advanced head and neck squamous cell carcinoma (HNSCC). Results from the ongoing Phase 1 study have highlighted the potential additive benefits of this combination to improve anti-tumor response. Preliminary data is anticipated in the second half of 2022.

In February 2022, HOOKIPA and Gilead agreed to advance its partnered HIV program, triggering a $54 million commitment from Gilead. HOOKIPA assumed development responsibility for the HB-500 program through the completion of a Phase 1b clinical trial; Gilead has the exclusive right for further development thereafter. Financial terms included a $4 million preclinical milestone, a $15 million non-refundable initiation fee and $35 million equity commitment at a premium to market price. The $35 million equity commitment includes a first tranche of $5 million (purchased at a $3 share price on February 15) and the remaining $30 million can be drawn at a 30 percent premium in a second tranche or at market price in a third tranche. If Gilead pursues further development, HOOKIPA is entitled to potential development and sales milestone payments exceeding $237 million, as well as royalties on net product sales.

Klaus Orlinger, Ph.D. was named Chief Scientific Officer. He was promoted from his previous role as Executive Vice President, Research. Klaus has played a leading role in the development of novel arenaviral immunotherapies and advancing them to the clinic since he joined the company in 2012.

In March 2022, HOOKIPA announced the acceptance of four poster presentations on preclinical, translational and clinical biomarker data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April. The data provided further evidence of the potential of the arenaviral platform in various cancers, either alone or with other modalities. Specifically, the data showed:

the combination of co-stimulatory 4-1BB agonists with arenaviral immunotherapy in a preclinical setting increased tumor control and resulted in a higher cure rate than arenaviral immunotherapy alone;
replicating immunotherapy sequentially combined with adoptively transferred TCR transgenic T cells resulted in tumor cures in a preclinical setting;
arenaviral immunotherapy was able to overcome immune tolerance, induce potent T cell responses against two different tumor self-antigens and reduce tumor growth in these cancers in a preclinical setting;
HB-200 induced robust antigen-specific T cells that were high quality, expanding on previously reported data in patients with Human Papillomavirus 16-positive (HPV16+) head and neck cancer. Additional Phase 1 data, including the recommended Phase 2 dose for HB-202/HB-201 was recently accepted for presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in June.

In April, HOOKIPA reported the addition of Tim Reilly, Ph.D. to its Board of Directors. Tim brings extensive product development experience to the Board.
Upcoming Milestones

Phase 1 HB-200 data in HPV16+ head and neck cancer: Mid-2022
Phase 2 HB-200 data in combination with pembrolizumab in HPV16+ head and neck cancer:
1st-line initial data: 2H 2022
2nd-line initial data: 2H 2022
Randomized Phase 2 HB-200 study in combination with pembrolizumab in 1st line for HPV16+ HNSCC: 1H 2023 (Fast Track designation)
Prostate cancer IND: 3Q 2022
Hepatitis B therapeutic IND: 2022 (Gilead-led)
First Quarter 2022 Financial Results
Cash Position: HOOKIPA’s cash, cash equivalents and restricted cash as of March 31, 2022 was $141.8 million compared to $66.9 million as of December 31, 2021. The increase was primarily attributable to funds resulting from the amended and restated Gilead collaboration agreement, and the follow-on financing in March 2022, partly offset by cash used in operating activities.

Revenue was $1.4 million for the three months ended March 31, 2022, and $5.3 million for the three months ended March 31, 2021. The decrease was primarily due to lower cost reimbursements received under the Collaboration Agreement with Gilead and the fact that the $4.0 million milestone payment and the $15.0 million initiation fee received in the three months ended March 31, 2022 were mostly recorded as deferred revenue to be recognized in future accounting periods.

Research and Development Expenses: HOOKIPA’s research and development expenses were $16.6 million for the three months ended March 31, 2022, compared to $20.2 million for the three months ended March 31, 2021.

The decrease for the three months ended March 31, 2022 compared to the three months ended March 31, 2021 was attributable to a decrease in direct research and development expenses, partially offset by an increase in indirect research and development expenses.

The decrease in direct research and development expenses was primarily driven by lower manufacturing expenses for our HB-200 and Gilead partnered programs and lower clinical study expenses due to the completion of patient enrollment of the Phase 2 study for our HB-101 program. Indirect research and development expenses increased slightly because of an increase in professional and consulting fees, partially offset by a decrease in personnel related costs.

General and Administrative Expenses: General and administrative expenses for the three months ended March 31, 2022 were $5.0 million, compared to $4.3 million for the three months ended March 31, 2021. The increase was primarily due to an increase in professional and consulting fees, and an increase in personnel-related expenses, partially offset by a decrease in other expenses. The increase in personnel-related expenses resulted from increased stock compensation expenses, a growth in headcount along with increased salaries in our general and administrative functions. The increase in professional and consulting fees was primarily attributable to intellectual property costs incurred in connection with filing and prosecuting patent applications as well as third-party license fees.

Net Loss: HOOKIPA’s net loss was $18.0 million for the three months ended March 31, 2022 compared to a net loss of $17.2 million for the three months ended March 31, 2021. This increase was due to a decrease in revenues from collaboration and licensing, a decrease in grant income, an increase in general and administrative expenses, partially offset by a decrease in research and development expenses.

On May 16, 2022 Celularity Inc. (Nasdaq: CELU) ("Celularity" or the "Company"), a clinical-stage biotechnology company developing placental-derived off-the-shelf allogeneic cell therapies, reported financial results for the first quarter ended March 31, 2022, and provided a corporate update (Press release, Celularity, MAY 16, 2022, View Source [SID1234614639]).

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"We have continued to achieve multiple transformational milestones and make significant progress this year with three ongoing Phase 1 clinical trials of two investigational drugs, CYNK-001 and CYNK-101, both of which have been granted Fast Track and Orphan Drug Designations for certain indications," said Robert J. Hariri, M.D., Ph.D., Founder, Chairperson and Chief Executive Officer of Celularity. We expect to have data readouts for all three ongoing Phase 1 programs later this year. As such, we believe we are well-positioned to continue executing our business strategy and developing cellular therapies for cancer, infectious and degenerative diseases that leverage our proprietary placental-based technology platform."

First Quarter Clinical and Regulatory Updates

CYNK-001 for the Treatment of AML and GBM:

CYNK-001 is Celularity’s unmodified cryopreserved human placental hematopoietic stem cell-derived NK cell therapy candidate that is enriched with CD56+/CD3- NK cells and expanded from human placental CD34+ cells. CYNK-001 is currently being investigated in two Phase 1 clinical trials, in AML and in GBM, with data readouts expected in the second half of 2022.
Celularity continues to enroll new cohorts in both arms of the Phase 1 AML study with clinical trial protocol adjustments communicated in December 2021, which include an addition of interleukin-2 (IL-2) to the treatment regimen; a fourth dose on day 21; and an increase in the dose of NK cells.
CYNK-101 for the Treatment of Gastric Cancer:

CYNK-101 is a novel allogeneic off-the-shelf human placental CD34+-derived NK cell product candidate that is genetically modified to express high-affinity and cleavage-resistant CD16 (FCGRIIIA) variant to drive antibody-dependent cell-mediated cytotoxicity. CYNK-101 is currently being investigated in the Phase 1 portion of a Phase 1/2a clinical trial in advanced HER2+ gastric cancer.
In January 2022, the FDA granted Fast Track designation to CYNK-101, which is being developed in combination with standard chemotherapy, trastuzumab and pembrolizumab in first-line locally advanced unresectable or metastatic HER2/neu positive gastric/gastroesophageal (G/GEJ) adenocarcinoma.
In February 2022, the FDA granted Orphan Drug Designation to CYNK-101 for the treatment of G/GEJ cancer.
CYCART-19 for the Treatment of B-Cell Malignancies:

CYCART-19 is an allogeneic Chimeric Antigen Receptor (CAR) engineered human placental-derived T cell that is a potential drug candidate in B-cell malignancies.
Preclinical data demonstrating the feasibility and functionality of expressing a CAR directed to CD19 on placental CD34+derived, cryopreserved, off-the-shelf, allogeneic CYNK cells were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2021.
Celularity submitted an Investigational New Drug (IND) application to FDA to investigate CYCART-19 for the treatment of B-cell malignancies in the first quarter of 2022 and in April 2022, received email notification from FDA that it will be requesting additional information before Celularity can proceed with the planned Phase 1/2 clinical trial. Celularity anticipates a formal written communication by late May 2022 and plans to work with the FDA in an effort to resolve its questions as promptly as possible and, if the IND is cleared, commence a Phase 1/2 clinical trial of CYCART-19 in B-cell malignancies in the second half of 2022.
First Quarter 2022 Financial Results

Cash and Cash Equivalents: Cash, cash equivalents and marketable securities were $48.0 million as of March 31, 2022, compared to $37.2 million as of December 31, 2021. In March 2022, we amended and restated certain warrants to reduce the exercise price per share to $3.50 per share, among other items, following which the holders exercised such warrants in full for cash for approximately $46.5 million and we issued the holders an aggregate 13,281,386 shares of our Class A common stock.
Total Revenues: Total revenues were $5.9 million for the first quarter of 2022, compared to $2.7 million for the first quarter of 2021. This increase in revenues was primarily driven by sales activity from supply and distribution agreements for Celularity’s degenerative disease products.
Research & Development (R&D) Expenses: R&D expenses were $21.7 million for the first quarter of 2022, compared to $17.0 million for the first quarter of 2021. The increase was primarily driven by higher clinical development expenses.
Selling, General & Administrative (SG&A) Expenses: SG&A expenses were $16.5 million for the first quarter of 2022, compared to $7.6 million for the first quarter of 2021. The increase in SG&A expenses was primarily caused by higher expenses related to being a public company, such as stock-based compensation, insurance and consulting fees.
Net loss: Net loss for the first quarter of 2022 was $62.9 million, or $(0.48) per share for both basic and diluted shares. Net loss for the first quarter of 2021 was $81.5 million, or $(3.40) per share for both basic and diluted shares.