On August 4, 2023 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based in vivo gene editing and ex vivo allogeneic CAR T therapies, reported financial results for the second quarter ended June 30, 2023 and provided a business update (Press release, Precision Biosciences, AUG 4, 2023, View Source [SID1234633827]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The first half of 2023 has been a busy time at Precision. Key updates have continued to elucidate the development and potential regulatory pathway for our ex vivo portfolio as well as demonstrate the potential of our proprietary ARCUS genome editing platform as a differentiated technology for high efficiency in vivo gene insertion and excision," said Michael Amoroso, Chief Executive Officer at Precision BioSciences. "Following our recent CAR T data update in May 2023 and subsequent Type B End of Phase 1 meeting with the U.S. Food and Drug Administration (FDA), we are actively engaging with potential partners to advance azer-cel and PBCAR19B to the next stage of development. As we prioritize organic development of our in vivo portfolio, we continue to generate supportive preclinical data to leverage the core features of ARCUS and advance differentiated programs to the clinic. We look forward to providing further updates across our in vivo programs at our upcoming gene editing R&D event in September."

Ex Vivo Allogeneic CAR T Platform

In July 2023, Precision received final meeting minutes from its June 2023 Type B meeting with the FDA for azer-cel. The objective of the meeting was to gain further clarity on the potential registration path for azer‑cel including study design, endpoints, and the recommended phase 2 dose in the CAR T relapsed patient setting. The discussion with the FDA provided clarity on azer-cel development, including a potential pathway toward registration. Based on the advice received from the FDA and clinical data shared during the May 2023 CAR T update, Precision is currently advancing discussions with multiple potential strategic partners for its cell therapy assets, including hematologic and non-hematologic applications.

In Vivo Gene Editing Platform

ARCUS may have broad utility in many diseases and the Company believes ARCUS is uniquely suited for in vivo gene editing, including the potential to produce a profound impact on diseases that are best treated by therapeutic gene insertion or excision of large defective gene sequences.

Chronic Hepatitis B: Precision is developing PBGENE-HBV for the treatment of patients with chronic hepatitis B with the goal of submitting a clinical trial application (CTA) and/or investigational new drug (IND) application in 2024. Hepatitis B virus (HBV) causes inflammation and damage to the liver, which can lead to chronic infection and increased risk of death from liver cancer or cirrhosis. There is no cure for chronic hepatitis B and current treatments rarely result in a functional cure, primarily due to persistence of viral DNA in the liver. In patients with chronic HBV infection, genetic material of the virus is converted within infected liver cells into covalently closed circular DNA (cccDNA) that acts as a template to make HBV copies. HBV also inserts its DNA into the human genome of infected liver cells. This integrated HBV DNA is a primary source of the viral protein, hepatitis B surface antigen (HBsAg), which is secreted in the blood. The presence of HBsAg is associated with poorer outcomes, and elimination of HBsAg, along with loss of circulating HBV DNA, is necessary for achieving a functional cure of chronic hepatitis B. Using ARCUS, Precision scientists have generated a highly specific nuclease designed to eradicate chronic HBV infection. The Company believes PBGENE-HBV is the only approach designed to inactivate and immediately eliminate cccDNA with direct edits as well as to inactivate integrated HBV DNA with the goal of long-lasting reductions in HBsAg and HBV DNA.

In June 2023, the Company presented data at the European Association for Study of the Liver (EASL) Congress. In an episomal adeno-associated virus (AAV) mouse model, Company researchers demonstrated that administration of lipid nanoparticles containing mRNA encoding an HBV-targeted ARCUS nuclease resulted in a 96% reduction in serum HBsAg. In a follow-on experiment, treatment of HBV-infected primary human hepatocytes with the HBV-targeted ARCUS nuclease resulted in a 90% reduction of cccDNA and high specificity. The Company plans to present additional data during its in vivo gene editing R&D event in September and at subsequent scientific conferences in 2023.

Novartis Partnered Program: Precision continues to advance an in vivo gene insertion program with Novartis to develop a custom ARCUS nuclease for patients with hemoglobinopathies, such as sickle cell disease and beta thalassemia. The collaborative intent is to insert a therapeutic transgene in vivo as a potential one-time transformative treatment administered directly to the patient that, if successful, would overcome many of the hurdles present today with other therapeutic technologies, including those that are utilizing an ex vivo gene editing approach.

Prevail Partnered Programs: Precision continues to progress its in vivo gene editing collaboration with Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company, in applying ARCUS nucleases to three initial targets, including Duchenne muscular dystrophy (DMD) in muscle, a central nervous system directed target, and a liver directed target. The goal of the PBGENE-DMD program is to utilize a pair of ARCUS nucleases, delivered by a single AAV, that are designed to excise an approximately 500,000 base pair mutation "hot spot" region from the dystrophin gene to generate a variant of the dystrophin protein that is functionally competent. In May 2023, the Company presented in vivo proof-of-concept data in preclinical models at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting demonstrating the therapeutic potential of PBGENE-DMD, including excision and repair of large sections of DNA. Precision scientists observed the edited dystrophin variant in multiple tissue types frequently involved in progression of DMD, including skeletal muscle, heart, and diaphragm, enabling significant functional muscle improvement.

In June 2023, Precision and Prevail entered into an amended and restated development and license agreement to continue to collaborate on developing the Company’s ARCUS nucleases for potential in vivo therapies for genetic disorders. Precision will continue to oversee creation, selection, in vitro development, and optimization of ARCUS nucleases with respect to the gene targets subject to the collaboration. Prevail will oversee and fund preclinical research and IND-enabling activities which were previously to be conducted by the Company at its expense. Prevail retains responsibility for conducting clinical development and commercialization activities for products from the collaboration. The Company will be eligible to receive milestone payments of up to an aggregate of $390 million to $395 million per licensed product, a decrease from $420 million as provided in the Original Agreement. This change reflects Prevail’s increased involvement in pre-clinical activities.

Ornithine Transcarbamylase (OTC) Deficiency: Led by iECURE, an ARCUS-mediated gene insertion approach is being pursued as a potential treatment for neonatal onset OTC deficiency. Non-human primate (NHP) data has been presented by researchers from the University of Pennsylvania’s Gene Therapy Program demonstrating sustained gene insertion of a therapeutic OTC transgene one-year post-dosing in newborn and infant NHP with high efficiency. iECURE is targeting submission of a CTA and/or IND in the second half of 2023.

Quarter Ended June 30, 2023 Financial Results:

Cash and Cash Equivalents: As of June 30, 2023, Precision had approximately $137.8 million in cash and cash equivalents. The Company expects that existing cash and cash equivalents, expected operational receipts, and available credit will be sufficient to fund its operating expenses and capital expenditure requirements through the first quarter of 2025.

Revenues: Total revenues for the quarter ended June 30, 2023 were $19.8 million, as compared to $3.8 million for the same period in 2022. The increase of $16.0 million was the result of a $11.2 million increase in revenue recognized from Prevail, $10.7 million of which was the result of a cumulative catch-up adjustment under the amended development and license agreement, and an increase of $4.8 million in revenue recognized under the Novartis Agreement.

Research and Development Expenses: Research and development expenses were $21.9 million for the quarter ended June 30, 2023, as compared to $22.9 million for the same period in 2022. The decrease of $1.0 million was primarily due to decreases in PBCAR19B external development, outsourced research and development, employee-related costs, and contract manufacturing organization expenses partially offset by increases in in vivo external development and azer-cel external development costs.

General and Administrative Expenses: General and administrative expenses were $9.8 million for the quarter ended June 30, 2023, as compared to $10.4 million for the same period in 2022. The decrease of $0.6 million was primarily driven by a decrease in share-based compensation expense and expense management, including a reduction in director and officer insurance premiums.

Net Loss: Net loss was $11.9 million, or $(0.10) per share (basic and diluted), for the quarter ended June 30, 2023, as compared to a net loss of $31.0 million, or $(0.46) per share (basic and diluted), for the same period in 2022. Weighted average shares of common stock outstanding were approximately 114.1 million for the quarter ended June 30, 2023, as compared to approximately 68.0 million for the quarter ended June 30, 2022. The increase in weighted average shares of common stock outstanding was primarily due to a $50 million underwritten offering of common stock and Novartis’ $25 million equity investment in 2022.

On August 4, 2023 Palatin Technologies, Inc. (NYSE American: PTN), a biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin receptor system, reported that management will participate at the Canaccord Genuity 43rd Annual Growth Conference on Wednesday, August 9, 2023 at 5:00 p.m. ET in an Analyst led Fireside Chat (Press release, Palatin Technologies, AUG 4, 2023, View Source [SID1234633826]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast of the Fireside Chat will be available on the Investors section of Palatin’s website at View Source A replay of the webcast will be available for 30 days following the presentation.

On August 4, 2023 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC) reported that Chief Executive Officer Dr. Matt Coffey will participate in a fireside chat at Canaccord Genuity’s 43rd Annual Growth Conference, which is taking place August 7-10, 2023 at the InterContinental Boston in Boston, MA (Press release, Oncolytics Biotech, AUG 4, 2023, View Source [SID1234633825]). Additional details on the fireside chat can be found below.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Date: Thursday, August 10, 2023
Time: 12:00 p.m. ET
Location: InterContinental Boston, Abigail Adams C Room
Webcast Link: Available by clicking here

Company management will also be participating in one-on-one investor meetings at the conference. To schedule a meeting, please submit a request on the conference website, contact your Canaccord representative, or email [email protected].

A live webcast of the Company’s presentation will also be available on the Investor Relations page of Oncolytics’ website (LINK) and will be archived for three months.

On August 4, 2023 Mersana Therapeutics, Inc. (NASDAQ:MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported that on August 1, 2023, an authorized sub-committee of the Board of Directors of Mersana granted inducement awards, consisting of stock options to purchase an aggregate of 38,775 shares of its common stock and restricted stock unit awards (RSUs) to acquire an aggregate of 27,655 shares of its common stock, to two new employees whose employment commenced in July 2023 (Press release, Mersana Therapeutics, AUG 4, 2023, View Source [SID1234633824]). The awards were granted pursuant to terms and conditions fixed by the Compensation Committee and as an inducement material to each new employee entering employment with Mersana in accordance with Nasdaq Listing Rule 5635(c)(4).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The option awards have an exercise price of $1.17 per share, which is equal to the closing price of Mersana’s common stock on August 1, 2023. Each option has a 10-year term and will vest over a period of four years, with 25% of the shares vesting on the one-year anniversary of the commencement of the employee’s employment and the remainder vesting in equal quarterly installments over the following three years, subject to the applicable employee’s continued service with Mersana on each such vesting date. The options are subject to the terms and conditions of Mersana’s 2022 Inducement Stock Incentive Plan and the terms and conditions of a stock option agreement covering each grant.

The RSUs will vest in equal annual installments on the first four anniversaries of August 15, 2023, subject to the applicable employee’s continued service with Mersana on each such vesting date. The RSUs are subject to the terms and conditions of Mersana’s 2022 Inducement Stock Incentive Plan and the terms and conditions of an RSU agreement covering each grant.

On August 4, 2023, iBio, Inc. (the "Company") reported to have entered into a purchase agreement, dated as of August 4, 2023 (the "Purchase Agreement"), with Lincoln Park Capital Fund, LLC ("Lincoln Park"), pursuant to which, under the terms and subject to the satisfaction of specified conditions set forth therein, the Company may sell to Lincoln Park up to $10.0 million (subject to certain limitations) of the Company’s common stock, par value $.001 per share (the "Common Stock"), from time to time during the term of the Purchase Agreement (Filing, 8-K, iBioPharma, AUG 4, 2023, View Source [SID1234633823]). Additionally, on August 4, 2023, the Company entered into a registration rights agreement, dated as of August 4, 2023 (the "Registration Rights Agreement"), with Lincoln Park., pursuant to which the Company agreed to file a registration statement with the Securities and Exchange Commission (the "SEC"), to register under the Securities Act of 1933, as amended (the "Securities Act"), the resale by Lincoln Park of shares of Common Stock that have been or may be issued and sold by the Company to Lincoln Park under the Purchase Agreement. The Company cannot sell any shares of Common Stock to Lincoln Park under the Purchase Agreement until such time that all of the conditions to Lincoln Park’s purchase obligation set forth in the Purchase Agreement, including that the resale registration statement that the Company is required to file with the SEC under the Registration Rights Agreement is declared effective by the SEC and a final prospectus relating thereto is filed with the SEC (the date on which all of such conditions are satisfied, the "Commencement Date").

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Beginning on the Commencement Date and for a period of up to 24 months thereafter, under the terms and subject to the conditions of the Purchase Agreement, from time to time, at the Company’s discretion, the Company has the right, but not the obligation, to sell to Lincoln Park, and Lincoln Park is obligated to purchase, up to $10 million of shares of Common Stock, subject to certain limitations set forth in the Purchase Agreement. Specifically, from time to time from and after the Commencement Date, the Company may, at its discretion, on any single business day on which the closing price of the common stock on the NYSE American is equal to or greater than $0.15, by written notice delivered to Lincoln Park, direct Lincoln Park to purchase up to 100,000 shares of Common Stock on such business day, at a purchase price per share that will be determined and fixed in accordance with the Purchase Agreement at the time the Company delivers such written notice to Lincoln Park (each, a "Regular Purchase"); provided, however, that the maximum number of shares the Company may sell to Lincoln Park in a Regular Purchase may be increased to up to (i) 150,000 shares, if the closing sale price of the Common Stock on the NYSE American on the applicable purchase date is not below $1.00, and (ii) 200,000 shares, if the closing sale price of the Common Stock on the applicable purchase date is not below $2.00; provided, however, that Lincoln Park’s maximum purchase commitment in any single Regular Purchase may not exceed $500,000. The foregoing share amounts and per share prices will be adjusted for any reorganization, recapitalization, non-cash dividend, stock split, reverse stock split or other similar transaction occurring after the date of the Purchase Agreement with respect to the Common Stock. The purchase price per share of Common Stock sold in each such Regular Purchase, if any, will be based on market prices of the Common Stock immediately preceding the time of sale, calculated as set forth in the Purchase Agreement.

In addition, provided that the Company has directed Lincoln Park to purchase the maximum amount of shares that the Company is then able to sell to Lincoln Park in a Regular Purchase on a particular business day on which the closing price of the common stock on the NYSE American is equal to or greater than $0.20, then in addition to such Regular Purchase, the Company may, in its sole discretion, also direct Lincoln Park to purchase additional shares of Common Stock in an "accelerated purchase," and one or more "additional accelerated purchases" on the business day immediately following the purchase date for such Regular Purchase, as provided in the Purchase Agreement. The purchase price per share of Common Stock sold to Lincoln Park in each accelerated purchase and additional accelerated purchase, if any, will be based on market prices of the Common Stock at the time of sale on the applicable purchase date for such accelerated purchase and such additional accelerated purchase(s), as applicable, calculated as set forth in the Purchase Agreement. There are no upper limits on the price per share that Lincoln Park must pay for shares of Common Stock in any purchase under the Purchase Agreement.

The Company will control the timing and amount of any sales of Common Stock to Lincoln Park pursuant to the Purchase Agreement. Lincoln Park has no right to require the Company to sell any shares of Common Stock to Lincoln Park, but Lincoln Park is obligated to make purchases as the Company directs, subject to certain conditions.

Actual sales of shares of Common Stock to Lincoln Park will depend on a variety of factors to be determined by the Company from time to time, including, among others, market conditions, market prices of the Common Stock from time to time during the term of the Purchase Agreement and determinations by the Company as to the appropriate sources of funding for the Company and its operations. The net proceeds under the Purchase Agreement to the Company will depend on the frequency and prices at which the Company sells shares of Common Stock to Lincoln Park. The Company expects that any proceeds received by the Company from such sales to Lincoln Park will be used for working capital and general corporate purposes.

As consideration for Lincoln Park’s commitment to purchase shares of Common Stock at the Company’s direction pursuant to the Purchase Agreement, the Company issued 211,473 shares of Common Stock to Lincoln Park as commitment shares (the "Initial Commitment Shares") and agreed to issue 211,474 additional shares of Common Stock to Lincoln Park as commitment shares (the "Additional Commitment Shares" and, collectively with the Initial Commitment Shares, the "Commitment Shares") at such time as the Company has received an aggregate of $5,000,000 in cash proceeds from Lincoln Park from sales of Common Stock to Lincoln Park, if any, that the Company elects, in its sole discretion, to make from time to time from and after the Commencement Date, pursuant to the Purchase Agreement.

Under applicable rules of the NYSE American, the Company may not issue or sell to Lincoln Park under the Purchase Agreement more than 4,474,945 shares (inclusive of the Commitment Shares), subject to adjustment as described above, of Common Stock (which is equal to approximately 19.99% of the shares of Common Stock outstanding immediately prior to the execution of the Purchase Agreement) (the "Exchange Cap"), unless stockholder approval is obtained to issue shares of Common Stock in excess of the Exchange Cap in accordance with the applicable rules of the NYSE American. In any event, the Company may not issue or sell any shares of Common Stock under the Purchase Agreement if such issuance or sale would breach any applicable rules or regulations of the NYSE American.

The Purchase Agreement also prohibits the Company from directing Lincoln Park to purchase any shares of Common Stock if those shares, when aggregated with all other shares of Common Stock then beneficially owned by Lincoln Park (as calculated pursuant to Section 13(d) of the Securities Exchange Act of 1934, as amended, and Rule 13d-3 thereunder), would result in Lincoln Park beneficially owning more than 4.99% of the outstanding shares of Common Stock.

There are no restrictions on future financings, rights of first refusal, participation rights, penalties or liquidated damages in the Purchase Agreement or Registration Rights Agreement, other than a prohibition (with certain limited exceptions) on entering into specified "Variable Rate Transactions" (as such term is defined in the Purchase Agreement) for a period specified in the Purchase Agreement. Such transactions include, among others, any sale of debt or equity securities that are convertible into or exercisable for shares of Common Stock at a conversion price or exercise price that is based upon and/or varies with the trading prices of the Common Stock after the initial issuance of such securities, or effecting or entering into an agreement to effect an "equity line of credit" or other substantially similar continuous offering with a third party, in which we may offer, issue or sell Common Stock or any securities exercisable, exchangeable or convertible into Common Stock at a future determined price. During any "suspension event" under the Purchase Agreement, the Company may not initiate any Regular Purchase, accelerated purchase or additional accelerated purchase of Common Stock by Lincoln Park, until such suspension event is cured.

Lincoln Park has agreed not to engage in or effect, directly or indirectly, for its own principal account or for the principal account of any of its affiliates, any short sales of the Common Stock or hedging transaction that establishes a net short position in the Common Stock during the term of the Purchase Agreement. The Company has the right to terminate the Purchase Agreement at any time with one business day’s notice, at no cost or penalty.

The Purchase Agreement and the Registration Rights Agreement contain customary representations, warranties, conditions and indemnification obligations of the parties. The representations, warranties and covenants contained in such agreements were made only for purposes of such agreements and as of specific dates, were solely for the benefit of the parties to such agreements and may be subject to limitations agreed upon by the contracting parties.

The foregoing descriptions of the Purchase Agreement and the Registration Rights Agreement are qualified in their entirety by reference to the full text of such agreements, copies of which are attached hereto as Exhibits 10.1 and 10.2, respectively, and each of which is incorporated herein in its entirety by reference.