On February 2, 2024 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 first quarter of fiscal 2024 ended December 31, 2023 (Press release, Twist Bioscience, FEB 2, 2024, View Source [SID1234639822]).

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"We’re starting fiscal 2024 off strong with record revenue of $71.5 million for the first fiscal quarter, coming in above our projection, with a gross margin of 40.5%," said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. "In SynBio, after a limited launch of Express Genes in November, we expanded the offering to include midi and Maxi preps at Express speed and over time, we expect this offering will allow us to both expand our share of market and convert the Maker’s Market of researchers who make their own genes into DNA buyers."

Dr. Leproust continued, "NGS had a strong quarter, with our top ten customers accounting for 44% of NGS revenue. As we move through the year, we plan to introduce new products and differentiated solutions to our customers and remain laser focused on revenue growth, margin expansion and financial discipline as we progress on our path to profitability."

FISCAL 2024 FIRST QUARTER FINANCIAL RESULTS

•Orders: Total orders received for the first quarter of fiscal 2024 increased to $77.5 million compared to $64.7 million for the same period of fiscal 2023.
•Revenue: Total revenues for the first quarter of fiscal 2024 increased to $71.5 million compared to $54.2 million for the same period of fiscal 2023.
◦Synbio revenue increased to $26.8 million for the first quarter of fiscal 2024 compared to $21.7 million for the same period of fiscal 2023.
▪Synthetic genes revenue increased to $19.7 million for the first quarter of fiscal 2024 compared to $16.2 million for the same period of fiscal 2023.
▪Oligo Pools revenue increased to $4.2 million for the first quarter of fiscal 2024 compared to $3.7 million for the same period of fiscal 2023.

▪DNA libraries revenue increased to $2.9 million for the first quarter of fiscal 2024 compared to $1.8 million for the same period of fiscal 2023.
◦NGS revenue increased to $39.4 million for the first quarter of fiscal 2024 compared to $24.4 million for the same period of fiscal 2023.
◦Biopharma revenue was $5.2 million for the first quarter of fiscal 2024 compared to $8.2 million in the same period of fiscal 2023.
•Cost of Revenues: Cost of revenues for the first quarter of fiscal 2024 was $42.5 million compared to $29.4 million for the same period of fiscal 2023.
•Research and Development Expenses: Research and development expenses for the first quarter of fiscal 2024 were $23.1 million compared to $31.2 million for the same period of fiscal 2023.
•Selling, General and Administrative Expenses: Selling, general and administrative expenses for the first quarter of fiscal 2024 were $52.8 million compared to $42.3 million for the same period of fiscal 2023.
•Net Loss: Net loss attributable to common stockholders for the first quarter of fiscal 2024 was $43.0 million, or $0.75 per share, compared to $41.8 million, or $0.74 per share, for the same period of fiscal 2023.
•Cash Position: As of December 31, 2023, the company had $311.1 million in cash, cash equivalents and short-term investments.

Recent Highlights:

•Shipped products to 2,140 customers in the first quarter of fiscal 2024, compared to approximately 2,060 customers in the first quarter of fiscal 2023.
•Shipped approximately 171,000 genes during the first quarter of fiscal 2024, compared with approximately 134,000 genes during the first quarter of fiscal 2023.
•On January 24, 2024, expanded Express Genes rapid gene synthesis service to all prep scales.
•Appointed Adam Laponis as chief financial officer.

Updated Fiscal 2024 Financial Guidance

The following statements are based on Twist’s current expectations for fiscal 2024, including the second quarter of fiscal 2024. 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 2024, Twist provided the following updated financial guidance:

•Total revenue is expected to be in the range of $288 million to $293 million, growth of 18 to 20 percent, and includes the following:

◦SynBio revenue is expected to be in the range of $114 million to $117 million compared to the previous estimate of $113 million to $116 million, indicating year over year growth of 16 to 19 percent
◦NGS revenue is expected to be in the range of $150 million to $152 million compared to the previous estimate of $147 million to $149 million, indicating year over year growth of 21 to 23 percent
◦Biopharma revenue is expected to be approximately $24 million compared to the previous estimate of approximately $25 million, indicating year over year growth of approximately 3 percent
•Gross margin is expected to be approximately 40% to 41% for fiscal 2024, an increase across the range
•Loss from operations before taxes of approximately $189 million to $194 million, an increase from $180 million to $188 million provided previously
◦Capital expenditure of $15 million, a decrease of $5 million from prior guidance
◦Ending cash, cash equivalents and short-term investments at September 30, 2024 of approximately $245 million, unchanged from previous guidance

For the second quarter of fiscal year 2024, Twist provided the following financial guidance:

•Total revenue of approximately $70 million to $71 million
◦SynBio revenue of approximately $28.5 million
◦NGS revenue of approximately $37 million to $38 million
◦Biopharma revenue of approximately $4.5 million
•Gross margin of 39%

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 February 2, 2024 Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, reported that it has entered into a securities purchase agreement with institutional and accredited investors to sell securities in a private placement financing (the PIPE) for gross proceeds of approximately $200 million (Press release, Tyra Biosciences, FEB 2, 2024, View Source [SID1234639824]).

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The financing was led by RA Capital Management, with participation by new and existing institutional investors, including Boxer Capital, BVF Partners, Nextech Invest Ltd (on behalf of one or more funds managed by it), OrbiMed, 5AM Ventures, a large investment management firm and a life-sciences focused institutional investor.

"We appreciate the support of RA Capital and our outstanding group of current and new investors as we further our mission of developing next-generation precision medicines that target FGFR biology," said Todd Harris, CEO of TYRA. "The additional funding strengthens our balance sheet at a time of strong momentum. We are focused on developing TYRA-300, an oral FGFR3-selective inhibitor, to become a best-in-class agent for achondroplasia, NMIBC and metastatic urothelial carcinoma, while advancing TYRA-200 and leveraging our SNÅP platform to discover new drug candidates."

In the PIPE, TYRA is selling an aggregate of approximately 15.4 million shares of its common stock (or pre-funded warrants in lieu thereof) at a price of $13.01 per share (or $13.009 per pre-funded warrant). The pre-funded warrants will have an exercise price of $0.001 per share of common stock, will be immediately exercisable and will not expire. The PIPE is expected to close on February 6, 2024, subject to customary closing conditions. The financing was priced at-the-market under Nasdaq rules.

TYRA intends to use the net proceeds from the PIPE to advance the clinical development of TYRA-300 and TYRA-200, to advance its preclinical programs, and for drug discovery, working capital and general corporate purposes.

The securities described above have not been registered under the Securities Act of 1933, as amended. Accordingly, these securities may not be offered or sold in the United States, except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act. TYRA has agreed to file a registration statement with the Securities and Exchange Commission (SEC) registering the resale of the shares of common stock and shares of common stock issuable upon the exercise of the pre-funded warrants issued in this PIPE.

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 jurisdiction.

On February 2, 2024 Qilu Pharmaceutical reported that results of the phase II study on novel anticancer drug iparomlimab and tuvonralimab (QL1706) were published online in Signal Transduction and Targeted Therapy (Impact Factor=39.3) (Press release, Qilu Pharmaceutical, FEB 2, 2024, View Source [SID1234639825]). The study investigated the use of QL1706 in combination with chemotherapy, with or without bevacizumab, for the treatment of non-small cell lung cancer (NSCLC). The publication of the phase II study results signifies broader recognition of QL1706 in the international clinical research community and expands the drug’s impact within the global academia.

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The study, led by Prof. Li Zhang from the Sun Yat-sen University Cancer Center, focused on using QL1706 in combination with chemotherapy, with or without bevacizumab, as a first-line treatment for advanced NSCLC. The research also examined the efficacy of QL1706 in combination with chemotherapy and bevacizumab in patients with epidermal growth factor receptor (EGFR)-mutant NSCLC that progressed after tyrosine kinase inhibitor (TKI) therapy. The findings indicated that QL1706, when used with chemotherapy, with or without bevacizumab, exhibited improved tolerability and promising anti-tumor activity in first-line treatment of patients with EGFR wild-type NSCLC. The combination of QL1706 with chemotherapy and bevacizumab showed notable anti-tumor effects in patients with EGFR-mutant NSCLC who did not respond to TKI therapy, presenting a potential new treatment option.

NSCLC is the predominant pathological type of lung cancer. The systemic management of advanced NSCLC has been revolutionized through advances in chemotherapy, targeted therapy, and immunotherapy, leading to extended overall survival for patients. Among immunotherapies, PD-1/PD-L1 and CTLA-4 inhibitors have become crucial in the first-line treatment of NSCLC, showing remarkable effects on survival outcomes. Despite their efficacy, these treatments can often present safety concerns. Currently, EGFR-TKIs are the standard therapy for EGFR-mutant advanced NSCLC. Despite the high initial objective response rate (ORR), tumors inevitably become resistant to first- and second-generation EGFR-TKIs. The secondary EGFR mutations are considered one possible resistance mechanism. For instance, the T790M mutation impairs binding between first-/second-generation EGFR-TKIs and mutated EGFR. Osimertinib, a third-generation EGFR-TKI, is used for treating NSCLC with EGFR T790M mutation, but its benefits in terms of overall survival are limited.

This trial was designed based on current evidence, to investigate whether dual immunotherapy plus chemotherapy (2 or 4 cycles), with or without bevacizumab, followed by maintenance therapy, could provide durable response and survival benefits. QL1706, targeting both PD-1 IgG4 and CTLA-4 IgG1, blocks both pathways. In a phase I study, QL1706 demonstrated robust anti-tumor effects in patients with advanced solid tumors, including those with NSCLC. Building on these findings, the multi-cohort phase II study assessed the effectiveness of QL1706 in combination with chemotherapy, with or without bevacizumab, in treating both EGFR wild-type and mutant advanced NSCLC. Out of the 112 patients screened in the trial, 91 were enrolled and divided into five distinct cohorts based on genotype: cohorts 1-4 comprised EGFR wild-type individuals, while cohort 5 included EGFR-mutant patients who had experienced disease progression on EGFR-TKI therapy.

The study’s findings revealed that the combination of QL1706 with chemotherapy, with or without the addition of bevacizumab, demonstrated promising efficacy in the treatment of advanced NSCLC. For patients with EGFR wild-type NSCLC, the ORR was 45%, and the median progression-free survival (mPFS) was 6.8 months. Notably, the median duration of response (mDOR) was not reached, and for those who responded to the treatment, the median immune duration of response (iDOR) was 11.5 months. These outcomes provide strong support for the efficacy of QL1706. Regarding safety, grade 3 or higher treatment-related adverse events (TRAEs) were reported in 31.7% of the patients, but no grade 4 or 5 TRAEs occurred.

In patients with EGFR-mutant NSCLC that progressed after EGFR-TKI therapy, the combination of QL1706 with bevacizumab and chemotherapy resulted in an impressive ORR (54.8%) and mPFS (8.5 months). This suggests the potential advantage of the combination therapy over the use of PD-1 inhibitor combined with chemotherapy and bevacizumab. In the study, 18 (58.1%) of the EGFR-mutant patients experienced immune-related adverse events (irAEs), the most common being hypothyroidism (29%), increased aspartate transaminase (AST) level (25.8%), and increased alanine transaminase (ALT) level (22.6%). Despite the irAEs, the combination therapy was deemed safe and effective, providing a valuable treatment option for patients with EGFR-mutant NSCLC.

On February 2, 2024 Hanmi reported its fourth quarter and full year 2023 financial results (Presentation, Hanmi, FEB 2, 2024, View Source [SID1234644665]).

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On February 2, 2024 Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, reported it has received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for RZ-001, for the treatment of patients with Hepatocellular carcinoma (HCC) (Press release, Rznomics, FEB 2, 2024, View Source [SID1234639826]).

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The ODD granted to RZ-001 in HCC may allow it to receive 7 years of marketing exclusivity upon product approval, exemption from user fee and tax credits. This also may provide access to specialized regulatory assistance from FDA’s Office of Orphan Products Development (OOPD).

The trans-splicing ribozyme is derived from the self-splicing Tetrahymena group I intron, which both recognizes and reprograms the target RNA into the therapeutic transcript of interest. Ribozyme-based RNA editing technology developed by Rznomics has unique features, differentiating it from other nucleic acid-based editing approaches, as follows: (1) A single RNA molecule is catalytically capable of both suppressing target RNA expression and simultaneously expressing a therapeutic RNA. Thus, this dual activity potentially requires no extra proteins or cofactors. (2) Safety can be improved by selectively inducing therapeutic RNA expression only in cells/tissues where the target gene is expressed. (3) Therapeutic gene expression can be regulated proportionally to endogenous cellular target RNA levels. (4) Editing occurs at the RNA level, not the genomic level, thus eliminating concerns about genomic toxicity and eternal genome changes. (5) Indications with multiple mutation sites scattered throughout a target RNA can be edited with a single RNA designed to react upstream of all mutations and by replacing and editing large stretches of RNA. (6) Additional safety can be conferred by building control mechanisms into the ribozyme itself, without the need to modulate intrinsic cellular mechanisms or external proteins.

More specifically, RZ-001 engenders effective anti-HCC activity by suppressing hTERT expression selectively in cancer cells, which over-express hTERT, and simultaneously inducing a cytotoxic effect by trans ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. Moreover, the result of such editing efficiently induces immune cell infiltrations into HCC tumors in preclinical animal models. (View Source).

"This FDA Orphan Drug Designation further underlines the potential of our pipeline to expeditiously address the current unmet medical needs of patients with Hepatocellular Carcinoma," said Seong-Wook Lee, CEO and founder of Rznomics.

Rznomics received Phase I/IIa IND approvals for RZ-001 in Hepatocellular Carcinoma (HCC) both from the FDA and the South Korean Ministry of Food and Drug Safety (MFDS), and this will be a dose escalation/expansion study to investigate the safety, tolerability, and efficacy of RZ-001 in HCC patients with no extrahepatic metastasis. In addition to HCC, Rznomics received Phase I/IIa IND approvals for RZ-001 GBM (Both in Korea and the U.S.) and Fast Track.

Most recently, Rznomics has entered into an HCC clinical collaboration agreement with F.Hoffmann-La Roche Ltd (Roche) to study RZ-001, in combination with Roche’s atezolizumab.