On August 4, 2023 Twist Bioscience Corporation (NASDAQ: TWST), a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, reported financial results and business highlights for the third quarter of fiscal 2023 ended June 30, 2023 (Press release, Twist Bioscience, AUG 4, 2023, View Source [SID1234633830]).

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"We reported record revenue again this quarter, with our core business, comprised of SynBio and NGS, delivering very strong results," said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. "Significantly, our Wilsonville site manufactured and shipped the vast majority of our genes, gene fragments and oligo pools, with the new team performing well. We continued to expand our NGS portfolio with the launch of our RNA sequencing portfolio targeting the substantial research market."

Dr. Leproust continued, "As we look forward, we expect continued growth in the core business, driven by taking market share, an increasing customer base and expanding product portfolio. For Biopharma Solutions, we will continue to focus on hiring the right commercial leaders, serving our customers, and closing new deals both with existing and new partners while keeping a keen eye on evaluating, analyzing and managing this business to ensure value creation in the short, medium and long term. In data storage, we remain on track to demonstrate an end-to-end gigabyte Century Archive workflow by the end of calendar 2023."

FISCAL 2023 THIRD QUARTER FINANCIAL RESULTS

Orders: Total orders received for the third quarter of fiscal 2023 were $63.8 million compared to $59.7 million for the same period of fiscal 2022.
Revenue: Total revenues for the third quarter of fiscal 2023 were $63.7 million compared to $56.1 million for the same period of fiscal 2022.
Cost of Revenues: Cost of revenues for the third quarter of fiscal 2023 was $41.8 million compared to $31.0 million for the same period of fiscal 2022.
Research and Development Expenses: Research and development expenses for the third quarter of fiscal 2023 were $24.5 million compared to $36.8 million for the same period of fiscal 2022.
Selling, General and Administrative Expenses: Selling, general and administrative expenses for the third quarter of fiscal 2023 were $46.1 million compared to $53.7 million for the same period of fiscal 2022.
Net Loss: Net loss attributable to common stockholders for the third quarter of fiscal 2023 was $57.4 million, or $1.01 per share, compared to $60.5 million, or $1.08 per share, for the same period of fiscal 2022.
Cash Position: As of June 30, 2023, the company had $357.1 million in cash, cash equivalents and investments.
Recent Highlights:

Shipped products to approximately 2,200 customers in the third quarter of fiscal 2023, versus approximately 1,900 in the third quarter of fiscal 2022.
Shipped approximately 171,000 genes during the third quarter of fiscal 2023, compared with approximately 163,000 in the third quarter of fiscal 2022.
Expanded the executive leadership team with the appointments of Robert Werner as chief accounting officer and Chet Gandhi as chief information officer.
Announced Jim Thorburn, chief financial officer (CFO), will transition to a new role supporting commercial scaling, operational leverage and DNA data storage once a new CFO is hired.
Entered into a new market with the launch of a portfolio of RNA sequencing tools to enable targeted or whole transcriptome research across fields including precision medicine, biomarker discovery and immuno-oncology research.
Entered into a collaboration with Cancer Research Horizons, the innovation arm of Cancer Research UK, by which Cancer Research Horizons will license the entire Twist Biopharma Solutions Library of Libraries.
Published preclinical data supporting the potential use of the highly potent and optimized GLP-1R antagonist antibody, TB-222-023, as a treatment for congenital hyperinsulinism in the journal Diabetes.
Presented at Imec Technology Forum (ITF) World 2023 on how industry growth could be fueled by using CMOS-based chips for high-density DNA synthesis for data storage applications.
Fiscal 2023 Financial Guidance

The following statements are based on Twist’s current expectations for fiscal 2023. The following statements are forward-looking, and actual results could differ materially depending on market conditions and the factors set forth under "Forward-Looking Statements" below.

For the full fiscal year 2023, Twist provided the following updated financial guidance:

Revenue is expected to be approximately $241 million to $242 million, compared to previous guidance of $235 million to $238 million
SynBio revenue is expected to be approximately $98 million, at the high end of the previous guidance of $96 million to $98 million
NGS revenue is expected to be approximately $120 million, compared to previous guidance of $113 million to $114 million
Biopharma revenue is estimated to be approximately $23 million to $24 million compared to previous guidance of $26 million
Gross margin for fiscal 2023 is expected to be 36%, at the high end of the range of previous guidance of 35-36%
Operating expenses including R&D and SG&A are expected to be approximately $308 million, compared to previous guidance of $313 million to $319 million
R&D expense is expected to be approximately $109 million, compared to previous guidance of $112 million to $114 million
SG&A expense is expected to be $189 million, compared to previous guidance of $197 million to $200 million
Mark to market of contingent consideration and indemnity holdbacks is projected to be a credit of $6 million, compared to previous guidance of $5 million
Restructuring costs are expected to be approximately $14 million, which includes approximately $4 million of asset and lease hold impairment charges related to the transition of SynBio operations from South San Francisco to Wilsonville, compared to previous guidance of $9 million to $11 million
Other income and expense for the year is projected to be approximately $12 million
Operating loss is expected to be approximately $220 million inclusive of one-time charges for restructuring of $14 million, compared to our previous guidance of $230 million to $234 million and includes the following:
Stock-based compensation is expected to be approximately $32 million, compared to our previous guidance of $43 million
Depreciation and amortization are expected to be approximately $29 million, unchanged from previous guidance
Operating expenses for DNA data storage is expected to be approximately $40 million, unchanged from previous guidance
Capital expenditure is expected to be approximately $35 million, compared to previous guidance of $40 million
FY23 Year End Cash is projected to be $325 million, compared to previous guidance of $320 million
For the fourth quarter of fiscal 2023, Twist provided the following financial guidance:

Revenue is expected to be approximately $63 million to $64 million, compared to previous guidance of $62 to $63 million
Gross margin is expected to be approximately 36%
R&D expense is expected to be approximately $26 million
SG&A expense is expected to be approximately $47 million
Restructure expense is expected to be approximately $1 million
Fiscal 2024 Financial Guidance

For the full fiscal year 2024, Twist provided the following financial guidance:

Twist expects to exit fiscal 2024 with adjusted EBITDA breakeven for its core (SynBio, NGS) business in the fourth quarter
Operating expenses for DNA data storage are expected to be $40 million
Fiscal year end cash is projected to be $220 million
Twist expects to be delayed in reaching its initial timeline in achieving adjusted EBITDA breakeven for its biopharma business exiting the fourth quarter of fiscal 2024.

On August 4, 2023 Aptevo Therapeutics Inc. (NASDAQ:APVO) ("Aptevo" or the "Company"), a clinical stage biotechnology company focused on developing novel immuno-oncology therapeutics, reported the closing of its previously announced public offering of 2,221,550 shares of common stock at a purchase price of $0.62 per share, Pre-Funded Warrants to purchase up to an aggregate of 5,842,967 shares of common stock at a purchase price of $0.619 per Pre-Funded Warrant, Series A Warrants to purchase up to an aggregate of 8,064,517 shares of common stock and Series B Warrants to purchase up to an aggregate of 8,064,517 shares of common stock (Press release, Aptevo Therapeutics, AUG 4, 2023, View Source [SID1234633829]). The Pre-Funded Warrants have an exercise price of $0.001, are immediately exercisable and may be exercised at any time until exercised in full. The Series A and Series B Warrants have an exercise price of $0.62 per share, are immediately exercisable and expire in August 2028 and February 2025, respectively.

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Gross proceeds from the offering were approximately $5 million before deducting placement agent fees and other estimated offering expenses. The Company intends to use the net proceeds of this offering for the continued clinical development of its product candidates and for working capital purposes.

A.G.P./Alliance Global Partners acted as sole placement agent for the offering.

The securities described above were offered pursuant to a registration statement on Form S-1, as amended (File No. 333-273067) previously filed with the Securities and Exchange Commission (SEC) which became effective on August 1, 2023. The offering was made only by means of a prospectus forming part of the effective registration statement. A final prospectus relating to the offering has been filed with the SEC. Electronic copies of the final prospectus may be obtained on the SEC’s website located at View Source and may also be obtained, when available, from A.G.P./Alliance Global Partners, 590 Madison Avenue, 28th Floor, New York, NY 10022, or by telephone at (212) 624-2060, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation, or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On August 4, 2023 Vivesto AB, an oncology-focused research and development company, reported early termination of patient enrollment in the Docetaxel micellar advanced prostate cancer Phase 1b study with the Swiss Group for Clinical Cancer Research (SAKK) (Press release, Vivesto, AUG 4, 2023, View Source [SID1234633828]).

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The open-label, multicenter, single-stage Phase 1b study has closed its accrual after enrollment of 11 of the planned 18 patients, since Vivesto believes that the data generated will be sufficient ahead of switching into development with a new formulation based on its improved XR-18 micelle technology platform.

Docetaxel micellar has shown good tolerability at doses considered standard for conventional docetaxel formulations, as well as signs of clinical activity. Docetaxel micellar has been well received by investigators of the SAKK 67/20 and by participating prostate cancer patients.

"We are grateful for the general positive feedback from the principal investigators. Particularly, Docetaxel micellar was safely administered with no infusion reactions and without the requirement for steroid premedication. A thorough analysis of the data, including pharmacological aspects, is pending, and we will await its completion to gain a comprehensive understanding of the outcomes," said Heidi Ramstad, Vivesto’s CMO. "We are grateful to the enrolled patients and for the effort put in by SAKK and investigators in search for better treatment options for prostate cancer."

Vivesto’s Docetaxel micellar is a solvent-free formulation of docetaxel developed to avoid the need for the solubility enhancers in a solvent-based docetaxel and the mandatory high-dose steroid premedication. Patients treated with other existing formulations of docetaxel require steroid administration to avoid certain serious adverse events related to the formulations with solvent. The administration of steroids can lead to marked bone fragility, exacerbated by cancer metastases in the bone, or steroid-related metabolic issues.

Vivesto has progressed in the development of its XR-18 drug delivery platform and intends to use this next-generation improved technology in future development of Docetaxel micellar.

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]).

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"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]).

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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.