On August 5, 2022 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 2022 ended June 30, 2022 (Press release, Twist Bioscience, AUG 5, 2022, View Source [SID1234617701]).

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"We are the high quality, low cost leader and as the macroeconomic environment continues to hold uncertainty, Twist’s product offerings are resonating with customers," said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. "We continue to see large accounts expanding their footprint while we also focus on diversifying our risk across an increasing number of customers, particularly in biopharma. We entered into our first exclusive option and license agreement for a Twist-discovered antibody and continue to build a robust ecosystem for DNA data storage through educational materials, including the most recently published white paper, and by driving toward an interoperable system through the development of industry standards."

Dr. Leproust continued, "Moving forward, we plan to launch the Factory of the Future in January 2023, which will facilitate growth across all areas of our business. We are well positioned to take market share in synbio and NGS, deliver new antibody targets for our biopharma customers and advance our efforts in DNA data storage toward our first commercial solution, all while targeting toward adjusted EBITDA breakeven at $300 million annual revenue for the core business."

FISCAL 2022 THIRD QUARTER FINANCIAL RESULTS

Orders: Total orders received for the third quarter of fiscal 2022 were $59.7 million compared to $39.1 million for the same period of fiscal 2021.
Revenue: Total revenues for the third quarter of fiscal 2022 were $56.1 million compared to $35.0 million for the same period of fiscal 2021.
Cost of Revenues: Cost of revenues for the third quarter of fiscal 2022 was $31.0 million compared to $20.9 million for the same period of fiscal 2021.
Research and Development Expenses: Research and development expenses for the third quarter of fiscal 2022 were $36.8 million compared to $19.8 million for the same period of fiscal 2021.
Selling, General and Administrative Expenses: Selling, general and administrative expenses for the third quarter of fiscal 2022 were $53.7 million compared to $34.5 million for the same period of fiscal 2021.
Net Loss: Net loss attributable to common stockholders for the third quarter of fiscal 2022 was $60.5 million, or $1.08 per share, compared to $40.0 million, or $0.82 per share, for the same period of fiscal 2021.
Cash Position: As of June 30, 2022, the company had $527.6 million in cash, cash equivalents and investments.
Recent Highlights:

SynBio and NGS

Shipped products to approximately 1,900 customers in the third quarter of fiscal 2022, versus approximately 1,800 in the third quarter of fiscal 2021.
Shipped approximately 163,000 genes during the third quarter of fiscal 2022, compared with approximately 107,000 in the third quarter of fiscal 2021.
Launched two human monkeypox synthetic DNA controls, expanding Twist’s portfolio of synthetic controls and reference standards.
Offered two new SARS-CoV-2 controls for the BA.2.12.1 sublineage of the omicron variant.
Received expanded Emergency Use Authorization from the U.S. Food and Drug Administration for the SARS-CoV-2 Next-Generation Sequencing (NGS) Assay developed with Biotia for the qualitative detection, identification and differentiation of SARS-CoV-2 lineages and identification of specific genomic mutations.
Launched Twist MRD Rapid 500 Panel to enable customers to implement fully customizable panels for $50 per sample.
Launched the Twist Human Methylome Panel covering 84.2% of GpC islands to support the discovery of differentially methylated regions and epigenetic target identification for cancer metastasis and human development research.
Entered into a collaboration with DNAnexus to integrate DNAnexus’ biomedical data analysis platform with Twist’s NGS Methylation Detection and Unique Molecular Identifier Adapter Systems.
BioPharma and DNA Data Storage

Entered into a research, exclusive option and license agreement with Astellas for a Twist-discovered antibody targeting an undisclosed checkpoint inhibitor.
Entered into a collaboration with Ildong Pharmaceutical, Twist’s first collaboration with a South Korea-based company, under which Ildong will license a suite of Twist VHH antibody libraries to use to discover and develop antibodies for applications in immuno-oncology.
Entered into an antibody discovery agreement with Xilio Therapeutics to advance the development of novel immunotherapies.
Hosted a ribbon-cutting ceremony for the new location of Twist Boston, also known as Abveris, in Quincy, MA.
The DNA Data Storage Alliance joined SNIA as a Technology Affiliate group to accelerate its mission to create an interoperable ecosystem for DNA-based data storage solutions.
Participated in panels and presentations at key data storage and industry conferences including Fujifilm’s 12th Annual Global IT Executive Summit, the 2022 MRS Spring Meeting and Exhibit, and the Flash Memory Summit.
Highlighted a DNA data storage white paper that quantifies the need for new storage mediums to address the zone of potential insufficiency where current methods of storing data, even when scaled, will not meet future demand.
Updated Fiscal 2022 Financial Guidance

The following statements are based on Twist’s current expectations for fiscal 2022. 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 2022, Twist provided the following updated financial guidance:

Revenue is now expected to be approximately $203 million, an increase from previous guidance of $191 million to $199 million
SynBio revenue including Ginkgo Bioworks is expected to be $80 million, an increase from previous guidance of $71 million to $73 million
NGS revenue is estimated to be approximately $97 million, an increase from previous guidance of $94 million to $96 million
Biopharma revenue is estimated to be approximately $26 million, at the low end of the previous guidance range of $26 million to $30 million
Gross margin is expected to be 40% for fiscal 2022
Operating expenses including R&D and SG&A are expected to be approximately $330 million for the year
Net loss is expected to be approximately $250 million, reflecting the impact of the Abveris acquisition in stock-based compensation and includes:
R&D expense is expected to be approximately $125 million, a decrease from previous guidance of $130 million
Approximately $40 million of R&D expense is expected to be for DNA data storage
Stock-based compensation is expected to be approximately $80 million
Depreciation is expected to be $14 million
Capital expenditures are expected to be in the range of $95 to $100 million, including building out the "Factory of the Future"
Conference Call Information

The company plans to hold a conference call and live audio webcast for analysts and investors at 8:00 a.m. Eastern Time today to discuss its financial results and provide an update on the company’s business. The conference call will be webcast live through the Investor Relations section under the "Company" tab at www.twistbioscience.com. Those parties interested in participating via telephone must register on the Company’s Investor Relations website or by clicking here. Upon registration, all telephone participants will receive the dial-in number along with a unique PIN number that can be used to access the call. To avoid delays, we encourage participants to dial into the conference call fifteen minutes ahead of the scheduled start time. The webcast replay will be available for two weeks.

On August 5, 2022 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that a conference call will be held on Monday, August 8, 2022 at 8:30 AM ET to discuss results for the second quarter of 2022 (Press release, TG Therapeutics, AUG 5, 2022, https://ir.tgtherapeutics.com/news-releases/news-release-details/tg-therapeutics-host-conference-call-second-quarter-2022 [SID1234617700]). Michael S. Weiss, Executive Chairman and Chief Executive Officer, will host the call.

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In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics. A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com. An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.

TG Therapeutics will announce its financial results for this period in a press release to be issued prior to the call.

On August 5, 2022 Nuvectis Pharma, Inc (NASDAQ: NVCT) ("Nuvectis" or the "Company"), a biopharmaceutical company focused on the development of novel therapies for the treatment of serious conditions of unmet medical need in oncology, reported its financial results for the second quarter of 2022 and provided an update on recent business progress (Press release, Nuvectis Pharma, AUG 5, 2022, View Source [SID1234617699]).

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"Nuvectis has made excellent progress this year towards its goal of becoming a leading precision medicine company with a robust, novel pipeline to treat unmet needs in oncology" said Ron Bentsur, Chairman and Chief Executive Officer of Nuvectis. "The NXP800 Phase 1a dose-escalation study is proceeding as planned with the start of the Phase 1b expansion phase expected in Q1 2023. With the U.S. Food and Drug Administration’s ("FDA") clearance of the NXP800 Investigational New Drug Application ("IND"), we now have clinical sites in the United States participating in the clinical program in addition to sites in the United Kingdom. We also look forward to sharing updates on new potential target indications, in addition to ovarian clear cell and ovarian endometrioid carcinoma, later this year."

Mr. Bentsur continued: "The body of evidence supporting the therapeutic potential of NXP900 continues to grow, and we are pleased with the recent preclinical data demonstrating robust activity of NXP900 in a xenograft model of Group 4 Medulloblastoma, a disease that affects mosrtly pediatric patients. We expect to complete the IND enabling studies by the end of this year."

Mr. Bentsur concluded: "The closing of a private placement earlier this month, coupled with our careful attention to spending, is expected to extend our cash runway into H2 2024. Based on the excellent second quarter progress, while leveraging our experienced team and expanded cash resources, I believe Nuvectis is well-positioned to potentially generate meaningful proof of concept data on its pipeline programs over the next year and beyond."

Second Quarter Highlights

NXP800 (our potentially first-in-class Heat Shock Factor 1 ("HSF1") pathway inhibitor): The Phase 1a dose escalation portion of the Phase 1 clinical program of NXP800 is ongoing and the Phase 1b dose expansion study is expected to begin in Q1 2023.

The IND for NXP800 was cleared by the FDA, including the Phase 1 clinical trial protocol which is now enrolling patients in both the United States and the United Kingdom. Additional preclinical studies to identify new target indications for NXP800 are ongoing.
An overview of the NXP800 drug discovery program was presented at the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Conference in the "New Drugs on The Horizon" oral session. The presentation included details on the unique program that led to the selection of NXP800 as a clinical candidate and on ovarian clear cell carcinoma and endometrioid ovarian carcinoma as the lead target indications.
NXP900 (our novel, selective SRC/YES1 kinase inhibitor): IND-enabling studies for NXP900 are ongoing with expected completion by the end of 2022.

Robust anti-cancer activity was demonstrated in a preclinical model of Group 4 Medulloblastoma (data presented at the European Society for Paediatric Oncology ("SIOPE") Brain Tumor Group meeting in Hamburg, Germany).
Preclinical data on NXP900 was presented at the 2022 AACR (Free AACR Whitepaper) Conference. The presentation included data demonstrating that NXP900 is potent against cancer cells of squamous origin (prevalent in lung, head and neck, cervical, skin and esophageal malignancies) and the identification of mutations that are associated with NXP900 sensitivity.
Financing

In August 2022, the Company completed a private placement raising approximately $15.9 million in gross proceeds which is expected to extend the cash runway into H2 2024.

Second Quarter 2022 Financial Results

Cash, cash equivalents, and short-term investments were $13.6 million as of June 30, 2022, compared to $5.7 million as of December 31, 2021. The increase of $7.9 million was primarily a result of the Company’s initial public offering in February 2022.

Research and development expenses were $2.5 million for the three months ended June 30, 2022, compared to $4.2 million for the three months ended June 30, 2021, a decrease of $1.7 million. The reduction in research and development expenses is primarily attributed to the payment of the NXP800 license fee in 2021. Research and development expenses for the second quarter 2022 included $0.3 million in non-cash expenses.

General and administrative expenses were $1.1 million for the three months ended June 30, 2022, compared to $1.7 million for the three months ended June 30, 2021, a decrease of $0.6 million. The reduction in general and administrative expenses is primarily attributed to a reduction in professional and consulting fees. General and administrative expenses for the second quarter 2022 included $0.2 million in non-cash expenses.

The Company’s net loss was $3.6 million for the three months ended June 30, 2022, compared to $5.9 million for the three months ended June 30, 2021, a decrease of $2.4 million. Net loss for the second quarter of 2022 included $0.5 million in non-cash expenses as well as $0.5 million in non-recurring expenses.

On August 5, 2022 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) ("Lexicon") reported the issuance and sale of an additional 2,526,540 shares of its common stock, par value $0.001 (the "Common Stock"), pursuant to the full exercise of the underwriters’ option to purchase additional shares in connection with Lexicon’s previously announced public offering of Common Stock (Press release, Lexicon Pharmaceuticals, AUG 5, 2022, View Source [SID1234617698]). After giving effect to the full exercise of the underwriters’ option to purchase additional shares, the total number of shares of Common Stock sold in the public offering was 19,370,140, and total gross proceeds from the public offering were approximately $48.4 million, before deducting underwriting discounts and commissions and other offering expenses.

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Two affiliates of Invus, L.P., Lexicon’s largest shareholder, have also exercised in full their options to purchase an additional 2,573,460 shares of Common Stock in the aggregate in connection with Lexicon’s previously announced concurrent private placement. After giving effect to the full exercises of the purchasers’ options to purchase additional shares, the total number of shares of Common Stock sold in the concurrent private placement was 19,729,860, and total gross proceeds from the concurrent private placement were approximately $49.3 million.

Lexicon currently intends to use the net proceeds from the public offering and the concurrent private placement, together with its existing cash and cash equivalents and short-term investments, for (i) funding pre-commercial and commercial launch activities for sotagliflozin in heart failure; (ii) funding continued development of sotagliflozin in heart failure and LX9211 in neuropathic pain; and (iii) working capital and other general corporate purposes.

Citigroup and Piper Sandler acted as joint book-running managers for the public offering.

A shelf registration statement on Form S-3 relating to the underwritten public offering was filed with the U.S. Securities and Exchange Commission ("SEC") on August 6, 2021 and declared effective by the SEC on September 14, 2021. The shares of Common Stock issued in the concurrent private placement have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or the securities laws of any state or other jurisdiction in the United States, and may not be offered, pledged, sold, delivered or otherwise transferred, directly or indirectly, in the United States except pursuant to registration under the Securities Act, or an applicable exemption from the registration requirements of the Securities Act and, in each case, in compliance with other applicable securities laws. A final prospectus supplement and accompanying prospectus relating to the public offering have been filed with the SEC and are available on the SEC’s website at www.sec.gov. Copies of the final prospectus supplement and accompanying prospectus may also be obtained from Citigroup Global Markets Inc., c/o Broadridge Financial Services, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at 1-800-831-9146, or by email at [email protected]; or Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, Attn: Prospectus Department, by telephone at 1-800-747-3924, or by email at [email protected].

This press release does not constitute an offer to sell, or the solicitation of an offer to buy, these securities, nor will there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale is not permitted.

On August 5, 2022 Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development company, reported the presentation of promising new data from an ongoing phase I clinical trial of paxalisib in combination with radiotherapy for the treatment of brain metastases, sponsored by Memorial Sloan Kettering Cancer Center in New York, NY (Press release, Kazia Therapeutics, AUG 5, 2022, View Source [SID1234617697]).

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Interim data from the first stage of the study reports that all 9 evaluable patients experienced complete or partial response, representing an overall response rate (ORR) of 100%. For comparison, a typical ORR associated with whole brain radiotherapy alone can commonly range from 20-45% in published studies, of which some representative examples are indicated below.

The data has been accepted for an oral presentation at the upcoming 2022 Annual Conference on CNS Clinical Trials and Brain Metastases, jointly organised by the Society for Neuro-Oncology (SNO) and the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper), and held in Toronto, Canada from 12-13 August 2022.

Key Points

Approximately 200,000 cancer patients develop brain metastases in the United States each year. Brain metastases are typically very challenging to treat and are associated with poor prognosis. Radiotherapy remains a mainstay of clinical management.

Dr. Jonathan Yang, Director, Metastatic Disease, Department of Radiation Oncology at Memorial Sloan Kettering Cancer Center is the Principal Investigator of this clinical trial that is examining the combination of paxalisib with whole brain radiotherapy for patients with brain metastases (NCT04192981). The trial is designed in two stages: an initial exploratory stage and a confirmatory expansion stage.

9 of 12 patients in the initial stage were evaluable for efficacy. All 9 patients exhibited complete or partial response, according to RANO-BM criteria, representing an ORR of 100%.

The patients comprised a range of primary tumours, with breast cancer the most common, representing one third of patients.

The safety profile of paxalisib in combination was broadly consistent with monotherapy experience in other clinical trials, and a maximum tolerated dose (MTD) of 45mg daily in combination with radiotherapy was confirmed.

Recruitment to the expansion stage has already commenced, with the objective of recruiting an additional 12 patients.
Kazia CEO, Dr. James Garner, commented, "We are encouraged by this data and by the potential benefit it may indicate to this substantial and high-need group of patients. Radiotherapy is a ubiquitous component of the treatment paradigm for brain metastases, but resistance is common. Dr. Yang’s study has shown a very promising signal that paxalisib may help to potentiate the effect of radiotherapy. We also learned recently that the ongoing Alliance study in brain metastases had graduated to an expansion stage in the breast cancer cohort, so this now represents the second positive signal for paxalisib in brain metastases, which we increasingly believe represents a very promising opportunity for the product candidate."

Brain Metastases

It is estimated that as many as 20% of all patients with cancer will develop brain metastases (secondary tumours in the central nervous system). The most common primary tumours that spread to the brain include lung, breast, colorectal, melanoma, and renal cell carcinoma. Median overall survival for patients diagnosed with brain metastases ranges from 2.3 to 7.7 months.[1] It is estimated that approximately 200,000 patients are diagnosed with brain metastases each year in the United States alone.[2]

Radiotherapy is the mainstay of treatment for brain metastases, and generally consists in either stereotactic radiosurgery (SRS) or whole brain radiotherapy (WBRT) or some combination thereof. The efficacy of WBRT differs according to the type of tumour and the number and volume of brain metastases, but several recent publications cite overall response rates of 20-45%.

Clinical Study Rationale and Design

Research by Dr. Yang and others has shown that activation of the PI3K pathway is common in brain metastases, even in some cases where it is not present in the primary tumour. Moreover, PI3K pathway activation appears to be induced by radiotherapy, and to confer upon the tumour resistance to radiotherapy. These observations provide a strong rationale for testing the combination of a brain-penetrant PI3K inhibitor with radiotherapy.

This phase I study is a single-arm prospective trial comprising patients with brain metastases or leptomeningeal metastases from any primary tumour. The primary objective is safety and tolerability. All patients have PI3K pathway mutations at baseline. The first stage of the study is intended to establish the MTD of paxalisib in combination with WBRT and to characterise safety and tolerability. The second stage is intended to elicit confirmatory signals of efficacy, with the intent to recruit a further 12 patients.

Next Steps

Enrolment to the second stage of the study is already underway and we currently estimate preliminary data from the second part of the phase I clinical trial in CY2023.

Kazia expects to discuss emerging data from this study, along with other research in brain metastases, with its scientific advisors and regulatory consultants in due course, with potential FDA consultation at a future date.

Summary of Abstract

Session 2: Multimodality Approaches to Primary and Secondary Brain Tumors – Invited
Speakers and Oral Abstracts
Friday, 12 August 2022
11am – 1pm

Abstract MMAP-05 – Phase I study of concurrent paxalisib and radiation therapy in patients with solid tumor brain metastases or leptomeningeal metastases harboring PI3K pathway mutations: results from the dose-escalation cohort (NCT04192981)
Lead Author: T Jonathan Yang, MD, PhD
Institution: Memorial Sloan Kettering Cancer Center, New York, NY