On January 08, 2023 Castle Biosciences, Inc. (Nasdaq: CSTL), a company improving health through innovative tests that guide patient care, reported certain unaudited preliminary performance results for the fourth quarter and full-year ended Dec. 31, 2022 (Press release, Castle Biosciences, JAN 8, 2023, View Source [SID1234626043]).

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"2022 was a year of focused investment, and we believe our strong success across the organization, including in revenue and test report volume growth, was a result of exceptional execution on our initiatives," said Derek Maetzold, president and chief executive officer of Castle Biosciences. "These initiatives include our acquisitions and planned integration of both Cernostics and AltheaDx, continued evidence development to support our commercial and pipeline tests and the expansion of our commercial team. And as such, we expect to meet or exceed the top end of our 2022 revenue guidance of $132-137 million."

Preliminary, Unaudited Fourth Quarter Ended Dec. 31, 2022, Highlights
•Delivered 12,563 total test reports in the fourth quarter of 2022, compared to 8,242 in the same period of 2021, an increase of 52%:
◦DecisionDx-Melanoma test reports delivered in the quarter were 7,295, compared to 5,635 in the fourth quarter of 2021, an increase of 29%.
◦DecisionDx-SCC test reports delivered in the quarter were 1,844, compared to 1,265 in the fourth quarter of 2021, an increase of 46%.
◦MyPath Melanoma and DiffDx-Melanoma diagnostic gene expression profile (GEP) aggregate test reports delivered in the quarter were 822, compared to 904 in the fourth quarter of 2021, a decrease of 9%.
◦DecisionDx-UM test reports delivered in the quarter were 432, compared to 438 in the fourth quarter of 2021, a decrease of 1%.
◦TissueCypher Barrett’s Esophagus test reports delivered in the quarter were 996. No test reports were delivered by Castle in the fourth quarter of 2021.
◦IDgenetix test reports delivered in the quarter were 1,174. No test reports were delivered by Castle in the fourth quarter of 2021.

Preliminary, Unaudited Year- Ended Dec. 31, 2022, Highlights
•The Company expects to meet or exceed the top end of its previously guided range of $132–137 million for full-year 2022 total revenue.
•Total test reports delivered in 2022 were 44,338, compared to 28,118 in the same period of 2021, an increase of 58%:
◦DecisionDx-Melanoma test reports delivered in 2022 were 27,797, compared to 20,328 in 2021, an increase of 37%.
◦DecisionDx-SCC test reports delivered in 2022 were 5,966 compared to 3,510 in 2021, an increase of 70%.

◦MyPath Melanoma and DiffDx-Melanoma diagnostic GEP aggregate test reports delivered in 2022 were 3,561, compared to 2,662 in 2021, an increase of 34%.
◦DecisionDx-UM test reports delivered in 2022 were 1,711, compared to 1,618 in 2021, an increase of 6%.
◦TissueCypher Barrett’s Esophagus test reports delivered in 2022 were 2,094. No test reports were delivered by Castle in 2021.
◦IDgenetix test reports delivered in 2022 were 3,209. No test reports were delivered by Castle in 2021.

Revenues and Test Report Volumes Disclaimer

The effects of macroeconomic events and conditions, including inflation, the COVID-19 pandemic and geopolitical events, among others, continue to evolve and bring along with them a high level of uncertainty surrounding potential future effects. Therefore, trends in revenues and test report volumes are not necessarily indicative of the Company’s results of operations that can be expected for future fiscal periods.

Cash, Cash Equivalents and Marketable Investment Securities

Year-end 2022 cash and cash equivalents are expected to be approximately $123 million. Additionally, the Company expects to hold approximately $136 million in short-term investments.

Castle Biosciences has not completed the preparation of its financial statements for the fourth quarter or full-year-ended Dec. 31, 2022. The preliminary, unaudited information presented in this press release for the quarter and year-ended Dec. 31, 2022, is based on management’s initial review of the information presented and its current expectations, and is subject to adjustment as a result of, among other things, the completion of the Company’s end-of-period reporting processes and related activities, including the audit by the Company’s independent registered public accounting firm of the Company’s financial statements. As such, any financial information contained in this press release may differ materially from the information reflected in the Company’s financial statements as of and for the year-ended Dec. 31, 2022. Additional information and disclosures would be required for a more complete understanding of the Company’s financial position and results of operations as of and for the quarter and year-ended Dec. 31, 2022. Accordingly, undue reliance should not be placed on this preliminary information.

On January 8, 2023 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its preliminary fourth quarter and full year 2022 global net product revenues for ONPATTRO, AMVUTTRA, GIVLAARI, and OXLUMO and provided additional updates on the products’ commercial launches (Press release, Alnylam, JAN 8, 2023, View Source [SID1234626028]).

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Preliminary Fourth Quarter and Full Year 2022 Commercial and Financial Performance*

Total TTR: ONPATTRO (patisiran) & AMVUTTRA (vutrisiran)

Preliminary global net product revenues for ONPATTRO and AMVUTTRA for the fourth quarter were approximately $122 million and $69 million, respectively, representing 12% total TTR quarterly growth compared to Q3 2022, and for the full year 2022 were approximately $558 million and $94 million, respectively, representing 37% total TTR annual growth compared to full year 2021.

As of year-end 2022, over 2,975 patients worldwide were receiving commercial ONPATTRO or AMVUTTRA.

* The preliminary selected financial results are unaudited, subject to adjustment, and provided as an approximation in advance of the Company’s announcement of complete financial results in February 2023.

** CER = Constant Exchange Rate, representing growth calculated as if the exchange rates had remained unchanged from those used during 2021. CER is a Non-GAAP financial measure.

GIVLAARI (givosiran)

Preliminary global net product revenues for the fourth quarter and full year 2022 were approximately $47 million and $173 million, respectively, representing quarterly and annual growth of 3% and 35% compared to Q3 2022 and full year 2021, respectively.

As of year-end 2022, over 520 patients worldwide were receiving commercial GIVLAARI.

OXLUMO (lumasiran)

Preliminary global net product revenues for the fourth quarter and full year 2022 were approximately $24 million and $70 million, respectively, representing quarterly and annual growth of 45% and 17% compared to Q3 2022 and full year 2021, respectively.

As of year-end 2022, over 280 patients worldwide were receiving commercial OXLUMO.

Further, at December 31, 2022, Alnylam had preliminary cash, cash equivalents, and marketable securities of approximately $2.2 billion, as compared to $2.4 billion at December 31, 2021.

"We are pleased to have closed out 2022 on a very strong note with continued execution across our commercial portfolio, delivering top-line revenue in line with our guidance range. These preliminary results reflect healthy patient demand for our transformative products and strong commercial execution by our teams in delivering these important medicines to patients in need around the world. In 2022 we were particularly excited to celebrate the approval and global launch of AMVUTTRA, which has demonstrated an impressive commercial performance in its first two full quarters of launch," said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. "As we consider our commercial and financial performance, combined with robust execution on the R&D front, we believe we are well on our way to achieving our Alnylam P5x25 goals, positioning Alnylam as a top-tier, global, multi-product commercial company with a broad pipeline and organic platform poised to deliver sustainable innovation well into the future, a profile rarely seen in our industry."

Alnylam management will discuss these preliminary selected financial results and commercial updates during a webcast presentation at the 41st Annual J.P. Morgan Healthcare Conference in San Francisco, California tomorrow, Monday, January 9, 2023 at 9:45 a.m. PT (12:45 p.m. ET).

About RNAi Therapeutics

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

On January 8, 2023 Akoya Biosciences, Inc. (Nasdaq: AKYA) ("Akoya"), The Spatial Biology Company, reported preliminary unaudited revenue for the fourth quarter and full year ended December 31, 2022 (Press release, Akoya Biosciences, JAN 8, 2023, View Source [SID1234626024]).

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Akoya reported the following preliminary financial results for the fourth quarter and full year 2022, which remain subject to quarter end closing adjustments:

Revenue for the fourth quarter of 2022 is expected to be between $20.7 million and $21.2 million, as compared to $16.2 million for the corresponding quarter of last year
For the fiscal year of 2022, revenue is expected to be between $74.3 million and $74.8 million, as compared to $54.9 million for fiscal year 2021
"The fourth quarter of 2022 was another record quarter for Akoya, demonstrating our continued business momentum and commercial execution," said Brian McKelligon, Chief Executive Officer. "In 2022, we delivered strong financial performance and saw growing adoption of our spatial biology platforms across the discovery, translational, and clinical markets."

A link to a webcast replay of Akoya’s 2nd annual Spatial Day, which took place virtually on December 15, 2022, can be found below:

Akoya Biosciences Spatial Day 2022 | Akoya

The financial results in this press release reflect expectations based on currently available information. The company has yet to complete its quarter end closing and actual results are therefore subject to change.

On January 08, 2023 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported several developments in its infectious disease and oncology portfolios (Press release, Molecular Partners, JAN 8, 2023, View Source [SID1234626011]). These include:

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DARPin Radioligand Therapy platform progressing well. Disclosure of the tumor-associated protein Delta-like ligand 3 (DLL3) as the first target. Expression of DLL3 is low in healthy tissue but significantly increased in certain tumor types, providing an opportunity for selective targeting.
Year-end 2022 unaudited cash and short-term deposits ~CHF249m. Company continues to maintain expected cash runway into 2026.*
Molecular Partners and Novartis signed a non-binding letter of intent to negotiate a Research Framework Agreement with a primary focus on emerging infectious global health threats. The Parties agree that the Definitive Agreement if concluded, shall contain provisions covering research plans, governance, development and commercialization, intellectual property, and the potential to prioritize future pandemic projects
The company expects milestones in 2023 to include:

Q1 2023: The recruitment of the first patient in the company’s phase 1 study of MP0533 (CD33 X CD123 X CD70 X CD3), a novel tri-specific t-cell engager for the treatment of AML and High Risk MDS
1H 2023: Complete recruitment in the dose escalation of the Phase 1 trial of the company’s MP0317 (FAP X CD40) program for the treatment of solid tumors
H1 2023: Scientific presentations of DARPin radioligand therapies and their potential differentiation as tumor targeting moiety
Formal selection of DLL3 DARPin RLT clinical candidate
Q4 2023: A first data readout from the Phase 1 study of MP0533
Ensovibep Update:

Molecular Partners was informed by its partner Novartis that it has submitted a request to withdraw the Emergency Use Authorization (EUA) application from the U.S. Food and Drug Administration (FDA) for ensovibep, a DARPin therapeutic candidate to treat COVID-19.
As previously disclosed, ensovibep is not presently in clinical development.
Patrick Amstutz, CEO of Molecular Partners, will present at the 41st Annual JP Morgan Healthcare Conference on Wednesday, January 11 at 10:30 AM ET (4:30 PM CET). A webcast will be accessible on the Molecular Partners website, under the Events tab.

*Unaudited financials. YE audited results will be available March 9, 2023

On January 8, 2023 Genexine (KOSDAQ: 095700), a publicly traded, clinical-staged Korean biopharmaceutical company committed to the discovery and development of novel biologics for the treatment of unmet medical needs, reported its updated results following the completion of its Phase 2 clinical study using GX-188E, its first-in-class proprietary DNA vaccine, in combination with KEYTRUDA (pembrolizumab), MSD’s (Merck & Co., Inc., Rahway, NJ., USA) anti-PD-1 therapy (Press release, Genexine, JAN 8, 2023, View Source [SID1234626009]).

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Having recently received the completed Clinical Study Report (CSR), Genexine has updated its primary efficacy numbers in the Phase 2 trial which evaluated the efficacy and safety of the combination of GX-188E and KEYTRUDA in a total of 65 patients (safety population) with HPV 16- and/or HPV 18- positive recurrent or metastatic advanced cervical cancer. The final efficacy analysis evaluated in 60 patients (efficacy evaluable population) showed a Objective Response Rate (ORR) of 35.0% (21 of 60 patients) indicating that of the 60 patients with advanced cervical cancer, 21 patients saw either an over 30% reduction in tumor size or complete remission.

Patients with a CPS<1 showed a response rate of 29.2%, while the patients with a CPS≥1 showed a response rate of 38.9% in the 60 patient evaluable population. With a disease control rate (DCR) of 57.0%, the combination therapy was found effective in more than half of the patients. The overall survival (OS) was 16.7 months which compares favorably to other agents that have been granted the accelerated approval by U.S. FDA in 2nd line cervical cancer treatment. The combination therapy was also safe and well tolerated. Genexine management will present these data together with a corporate update at various meetings during the upcoming JP Morgan conference to be held January 9-13, 2023 in San Francisco.

"We are pleased to be able to offer much-needed hope to patients showing an impressive response rate regardless of PD-L1 expression and extended survival of 16.7 months in advanced cervical cancer patients," said Neil Warma, President and CEO of Genexine. "After completing the final Blinded Independent Central Review (BICR) of the data, the results show an improvement over the top-line results we presented last September at ESMO (Free ESMO Whitepaper) and reinforce our belief that GX-188E could open up new treatment opportunities to all cervical cancer patients, especially PD-L1 negative patients who currently have limited options. We also appear to be extending patient survival beyond that of currently marketed drugs which should position us well as we move into larger Phase 3 studies in our effort to become a leader in the oncology DNA vaccine market."

GX-188E is a therapeutic DNA vaccine that encodes the E6/E7 fusion protein of human papillomavirus (HPV) subtypes 16 and 18, which are known to be a cause of over 70% of cervical cancer cases and is administered intramuscularly by electroporation. GX-188E induces immune cells to detect E6 and E7 proteins that only exist in cervical cancer cells, inducing apoptosis of tumor cells in an immune-oncological response without affecting healthy cells. Genexine is also conducting two ongoing clinical studies with GX-188E in combination with a check point inhibitor and a long-acting interleukin 7 in squamous cell carcinoma of head and neck (SCCHN). Results from these trials are expected in 2023.