Inspirna Announces Phase 1b RGX-202 Clinical Trial Data Selected for Poster Presentation at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting?

On April 27, 2022 Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and biologic cancer therapeutics, reported that it will present a poster at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held June 3-7 in Chicago, Illinois (Press release, Inspirna, APR 27, 2022, View Source [SID1234613028]).

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Poster Presentation Details
Title: Phase 1b study of RGX-202-01, a first-in-class oral inhibitor of the SLC6A8/CKB pathway, in combination with FOLFIRI and bevacizumab (BEV) in second-line advanced colorectal cancer (CRC)
Date and time: Saturday, June 4, 2022, 8:00 a.m. CDT
Session: Gastrointestinal Cancer—Colorectal and Anal
Abstract ID: 3579

Alkermes plc Reports First Quarter 2022 Financial Results

On April 27, 2022 Alkermes plc (Nasdaq: ALKS) reported financial results for the first quarter of 2022 (Press release, Alkermes, APR 27, 2022, View Source [SID1234613044]).

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"Our strong first quarter results reflect continued momentum across the business, and a sharp operational focus that provides a solid foundation to drive further growth of our proprietary products and advance our pipeline of development programs in 2022. As we execute on our launch strategy for LYBALVI, we are particularly encouraged by early utilization trends and feedback from healthcare providers that underscore LYBALVI’s value proposition in the oral antipsychotic market," said Richard Pops, Chief Executive Officer of Alkermes. "With our focus on disciplined allocation of capital, strong corporate governance, and our commitment to long-term profitability targets, we are delivering on our commitment to efficiently drive growth and actively managing the business to create value for our shareholders in 2022 and beyond."

Quarter Ended March 31, 2022 Financial Results

Revenues

– Total revenues for the quarter were $278.5 million, compared to $251.4 million for the same period in the prior year.

– Net sales of proprietary products for the quarter were $171.3 million, compared to $130.0 million for the same period in the prior year.

Net sales of VIVITROL were $84.9 million, compared to $74.5 million for the same period in the prior year, representing an increase of approximately 14%.
Net sales of ARISTADAi were $72.5 million, compared to $55.4 million for the same period in the prior year, representing an increase of approximately 31%.
Net sales of LYBALVI were $13.9 million, following its commercial launch in October 2021.
– Manufacturing and royalty revenues for the quarter were $105.2 million, compared to $119.8 million for the same period in the prior year.

Royalty revenues from INVEGA SUSTENNA/XEPLION, INVEGA TRINZA/TREVICTA and INVEGA HAFYERA (the long-acting INVEGA products) were $37.1 million, compared to $61.6 million for the same period in the prior year. This includes approximately one month of royalty payments related to sales of the long-acting INVEGA products in the United States (U.S.), compared to three months in the same period in the prior year. This decrease was driven primarily by Janssen Pharmaceutica N.V.’s (Janssen) partial termination of the license agreement related to sales of long-acting INVEGA products in the U.S., which took effect starting in February of 2022.
° In April 2022, the company commenced binding arbitration proceedings related to, among other things, Janssen’s partial termination of the license agreement in the U.S. and Janssen’s royalty and other obligations under the agreement.

Manufacturing and royalty revenues from RISPERDAL CONSTA were $17.4 million, compared to $14.2 million for the same period in the prior year.
Manufacturing and royalty revenues from VUMERITY were $30.6 million, compared to $13.4 million for the same period in the prior year.
Costs and Expenses

– Total operating expenses for the quarter were $305.1 million, compared to $267.9 million for the same period in the prior year, primarily reflecting increased investment to support the commercial launch of LYBALVI.

Cost of Goods Manufactured and Sold were $55.2 million, compared to $41.0 million for the same period in the prior year.
Research and Development (R&D) expenses were $96.0 million, compared to $92.3 million for the same period in the prior year.
Selling, General and Administrative (SG&A) expenses were $145.1 million, compared to $125.2 million for the same period in the prior year.
– Other Expense, Net for the quarter included a reduction of $19.1 million in the fair value of contingent consideration related to increased risk of non-payment of certain milestone payments by Baudax Bio, Inc. in light of its disclosures regarding its ability to continue as a going concern.

Profitability

– Net loss according to generally accepted accounting principles in the U.S. (GAAP) was $35.9 million for the quarter, or a basic and diluted GAAP loss per share of $0.22. This compared to GAAP net loss of $22.4 million, or a basic and diluted GAAP loss per share of $0.14, for the same period in the prior year.

– Non-GAAP net income was $19.6 million for the quarter, or a non-GAAP basic and diluted earnings per share of $0.12. This compared to non-GAAP net income of $17.8 million for the quarter, or a non-GAAP basic and diluted earnings per share of $0.11, for the same period in the prior year.

Balance Sheet

– At March 31, 2022, the company recorded cash, cash equivalents and total investments of $758.7 million, compared to $765.7 million at Dec. 31, 2021. The company’s total debt outstanding as of March 31, 2022 was $295.2 million.

"Our first quarter results demonstrate the strength of our proprietary commercial product portfolio and our continued focus on efficient management of our cost structure. We are in a strong financial position to execute on our strategic priorities and work toward achievement of our long-term profitability targets," commented Iain Brown, Chief Financial Officer of Alkermes. "Today, we are reiterating our financial expectations for 2022, as we focus on efficiently driving growth of LYBALVI, ARISTADA and VIVITROL, and advancing our development pipeline."

Financial Expectations for 2022

Alkermes reiterates its financial expectations for 2022, and the assumptions underlying such expectations, as set forth in its press release dated Feb. 16, 2022.

Recent Events:

Oncology

In February 2022, the company presented new data from the ongoing phase 1/2 ARTISTRY-1 clinical trial for nemvaleukin alfa (nemvaleukin), the company’s novel, investigational, engineered interleukin-2 (IL-2) variant immunotherapy, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary (GU) Cancers Symposium. The presentation included updated data from the monotherapy arm of ARTISTRY-1 in patients with advanced renal cell carcinoma (RCC), including patients who were checkpoint inhibitor-pretreated.
In March 2022, the company presented nemvaleukin data from ARTISTRY-1 in patients with platinum-resistant ovarian cancer (PROC) in an oral plenary session at the Society of Gynecologic Oncology (SGO) 2022 Annual Meeting on Women’s Cancer. The company also presented a trial-in-progress poster from the ongoing phase 3 ARTISTRY-7 global study evaluating the efficacy, safety and tolerability of IV nemvaleukin in combination with pembrolizumab compared to investigator’s choice chemotherapy in patients with platinum-resistant epithelial ovarian, fallopian tube or primary peritoneal cancer.
Psychiatry

In April 2022, the company presented new research from its psychiatry portfolio at the 2022 Congress of the Schizophrenia International Research Society (SIRS). The presentations included detailed results from the recently completed ENLIGHTEN-Early study of LYBALVI (olanzapine and samidorphan), a phase 3b study that evaluated the effect of LYBALVI compared to olanzapine on body weight in young adult patients (ages 16 to 39; mean age: 26 years) with schizophrenia, schizophreniform disorder or bipolar I disorder who were early in their illness.
Other

In April 2022, the company commenced binding arbitration proceedings related to, among other things, Janssen’s partial termination of two license agreements with the company in the U.S. and Janssen’s royalty and other obligations under the agreements. Under these agreements, Janssen received access and rights to Alkermes’ small particle pharmaceutical compound technology, known as NanoCrystal Technology, which enabled the development and commercialization of a number of successful products, such as INVEGA SUSTENNA, INVEGA TRINZA, INVEGA HAFYERA and CABENUVA. Janssen partially terminated these agreements in the United States effective as of February 2022.
Conference Call

Alkermes will host a conference call and webcast presentation with accompanying slides at 8:00 a.m. ET (1:00 p.m. BST) on Wednesday, April 27, 2022, to discuss these financial results and provide an update on the company. The webcast may be accessed on the Investors section of Alkermes’ website at www.alkermes.com. The conference call may be accessed by dialing +1 877 407 2988 for U.S. callers and +1 201 389 0923 for international callers. In addition, a replay of the conference call will be available from 11:00 a.m. ET (4:00 p.m. BST) on Wednesday, April 27, 2022, through Wednesday, May 4, 2022, and may be accessed by visiting Alkermes’ website or by dialing +1 877 660 6853 for U.S. callers and +1 201 612 7415 for international callers. The replay conference ID is 13727838.

Charles River and Valo Launch Logica, an Integrated AI-Powered Drug Discovery Solution to Rapidly Deliver Optimized Preclinical Assets

On April 27, 2022 Charles River Laboratories International, Inc. (NYSE: CRL) and Valo Health, Inc ("Valo") have launched LogicaTM, an artificial intelligence (AI) powered drug solution that directly translates clients’ biological insights into optimized preclinical assets (Press release, Charles River Laboratories, APR 27, 2022, View Source [SID1234613061]). Logica leverages Valo’s AI-powered Opal Computational Platform and Charles River’s leading preclinical expertise, providing clients with transformed drug discovery with a single integrated offering seamlessly translating targets to candidate nomination.

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"Through our partnership with Valo, we’re integrating Charles River’s laboratory capabilities with industry-altering AI-driven molecular design to provide our clients with highly advanceable leads and candidates, while tying client costs to value generation," said Birgit Girshick, Corporate Executive Vice President & Chief Operating Officer, Charles River. "AI-powered solutions are driving drug discovery forward and this is the kind of innovation our industry needs to ultimately bring effective treatments to patients faster."

"Valo is thrilled to partner with Charles River on this first of its kind offering," added David Berry, MD, Ph.D., founder, and CEO of Valo. "We are creating an opportunity to fully outsource small molecule discovery while providing a true risk-sharing model with advanceable leads and candidates with the goal of helping companies scale faster, translating more biological insight into candidates to make better drugs for patients, faster."

Logica’s Advanceable Lead (Logica-AL) and Candidate (Logica-C) programs, which are available exclusively from Charles River, are part of a multi-year strategic partnership that Charles River and Valo announced earlier this year. Logica-AL aims to provide customers with highly advanceable, potent series with desired characteristics, such as in vitro absorption, distribution, metabolism, and excretion (ADME) and selectivity profiles understood. In addition, the Logica-C offering will use the advanceable leads and trained predictive models to rapidly progress the program, ultimately aiming to deliver a development candidate that has undergone necessary safety and efficacy tests and is ready for IND-enabling studies.

Logica utilizes industry-leading predictive models, chemical design and synthesis capabilities, DNA-encoded libraries, in silico high throughput screening from Valo’s Opal Computational Platform as well as Charles River’s leading capabilities in all aspects of discovery optimization including high throughput screening, medicinal chemistry, ADME, biology, pharmacology, and ultimately safety testing and IND submission, joining together for the first time to create a computation-powered, unified target-to-candidate offering. Logica combines approaches that have a demonstrated ability to produce an advanceable lead series 90 percent of the time and deliver development candidates 58 percent of the time. The products will be offered with most of the client cost tied to success.

PTC ANNOUNCES SECOND FISCAL QUARTER 2022 RESULTS

On April 27, 2022 PTC (NASDAQ: PTC) reported financial results for its second fiscal quarter ended March 31, 2022 (Press release, PTC Therapeutics, APR 27, 2022, View Source [SID1234613077]).

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"In the second quarter we continued to see our key operating and financial metrics showing strong performance. We delivered organic constant currency ARR growth of 13% year over year to end Q2 at $1.56 billion. In Q2, our cash from operations was $142 million, up 17% year over year, and our adjusted free cash flow was $158 million, up 22% year over year. The strength in Q2 was broad-based across all segments and geographic regions, driven by demand for digital transformation and SaaS," said James Heppelmann, President and CEO, PTC.

"Our differentiated product portfolio and growing SaaS capabilities position PTC to drive superior value for customers. Our market position coupled with our subscription model, which took us years of hard work to put in place, is highly resilient and positions us to continue to deliver strong double-digit ARR growth. Based on our strong performance in the first half of the year and the momentum we have created, we are raising our fiscal 2022 guidance for ARR and free cash flow," concluded Heppelmann.

Second Quarter 2022 Highlights 1
Key operating and financial highlights are set forth below. For additional details, please refer to the Q2’22 earnings presentation and financial data tables that have been posted to the Investor Relations section of our website at investor.ptc.com. Revenue and, as a result, operating margin and earnings per share are impacted by revenue recognition under ASC 606.

ARR was $1,532 million at the end of Q2’22, up 11% compared to Q2’21. On a constant currency basis, ARR was $1,564 million, up 13% compared to Q2’21, and above guidance of $1,540 million to $1,550 million. ARR at the end of Q2’22 includes a $4 million reduction associated with discontinuing our business operations in Russia.
Cash flow from operations was $142 million, free cash flow was $140 million, and adjusted free cash flow was $158 million in Q2’22, compared to Q2’21 cash flow from operations of $122 million, free cash flow of $116 million, and adjusted free cash flow of $130 million.
Revenue was $505 million in Q2’22 compared to $462 million in Q2’21, representing growth of 9%. On a constant currency basis, year-over-year revenue growth in Q2’22 was 13%.
Operating margin was 32% in Q2’22, compared to 22% in Q2’21. Non-GAAP operating margin in Q2’22 was 42%, compared to 37% in Q2’21. GAAP and non-GAAP operating margin expanded year over year due to higher revenue and operational discipline.
Earnings per share was $0.76 in Q2’22, compared to $0.92 in Q2’21. Non-GAAP earnings per share in Q2’22 was $1.39, compared to $1.08 in Q2’21. GAAP EPS in Q2’22 was negatively impacted by a non-cash charge within other expense due to a decline in value of a publicly traded equity investment, which was sold in Q2’22.
Total cash and cash equivalents as of the end of Q2’22 was $307 million. Gross debt was $1.28 billion as of the end of Q2’22. During Q2’22, we repaid $175 million on our revolving credit facility and we had proceeds of $43 million from the sale of the aforementioned equity investment.
Stock repurchases were $5 million in Q2’22, reflecting the settlement of repurchases that were initiated in Q1’22
1 We include operating and non-GAAP financial measures in our operational highlights. The detailed definitions of these items and reconciliations of non-GAAP financial measures to comparable GAAP measures are included below and in the reconciliation tables at the end of this press release.

Fiscal 2022 and Q3’22 Guidance
"PTC delivered strong second quarter results that exceeded our expectations," said Kristian Talvitie, EVP and CFO, PTC. "Based on our Q2 performance and our forecast for the remainder of the year, we are raising our guidance for fiscal 2022 ARR, Free Cash Flow, and Revenue. Despite significant foreign exchange headwinds and the impact of exiting our business in Russia, our strong execution and operational discipline have helped us to deliver solid financial performance thus far in fiscal 2022, and we believe we are well positioned to deliver on our updated targets for the year."

(1)On a constant currency basis, using our FY’22 Plan foreign exchange rates (rates as of September 30, 2021) for all periods

(2)FY’22 cash from operations and free cash flow guidance include expected restructuring payments of $45 million (current estimate is $40 million to $45 million) and transaction-related payments of approximately $5 million (already incurred in 1H’22), both of which are excluded from FY’22 adjusted free cash flow guidance; Q3’22 cash from operations and free cash flow guidance include expected restructuring payments of approximately $10 million and transaction-related payments of approximately $5 million (already incurred in 1H’22), both of which are excluded from Q3’22 adjusted free cash flow guidance

(3)Free cash flow and adjusted free cash flow guidance are net of expected capex of approximately $25 million (previously $30 million) in FY’22 and $5 million in Q3’22

Our FY’22 and Q3’22 financial guidance includes the assumptions below:

We provide ARR guidance on a constant currency basis, using our FY’22 Plan foreign exchange rates (rates as of September 30, 2021) for all periods. Based on foreign exchange rate fluctuations as of the end of Q2’22, we currently expect a $34 million headwind (previously $13 million), relative to our constant currency ARR guidance for FY’22, and a $32 million headwind, relative to our constant currency ARR guidance for Q3’22.
We expect FY’22 organic churn to improve by approximately 100 basis points over FY’21.
Due to invoicing seasonality, the majority of our collections occur in the first half of our fiscal year. Q4 is our lowest cash flow generation quarter.
Costs are expected to ramp throughout FY’22 due to hiring and increased SaaS investments. At the mid-point of ARR guidance, we expect FY’22 GAAP operating expenses to increase approximately 3% to 4% (previously 4% to 5%) and non-GAAP operating expenses to increase approximately 2% to 3% over FY’21.
FY’22 GAAP results are expected to include the items outlined below, totalling $293 million to $308 million (previously $275 million to $280 million), as well as their related tax effects:
$160 million to $170 million of stock-based compensation expense (previously $178 million)
$58 million of intangible asset amortization expense
$35 million to $40 million of restructuring charges (previously $40 million to $45 million)
$35 million of FY’22 net realized losses from the sale of an equity investment (new)
Approximately $5 million of transaction-related charges (new)
Our FY’22 guidance does not reflect operating results of the Intland acquisition and the ITCI transaction, the impact of business combination accounting, incremental interest expense, or transaction-related charges not incurred as of the end of Q2’22.
Related to restructuring, for FY’22 we expect:
P&L charges of $35 million to $40 million (previously $40 million to $45 million), of which $32 million was incurred in the first half of FY’22.
Cash outflows for restructuring payments of $40 million to $45 million (previously $45 to $50 million), of which $28 million was paid in the first half of FY’22. We expect the majority of remaining payments to be made in Q3’22. Restructuring payments in FY’22 include $5 million related to prior period actions, primarily the relocation of our headquarters in FY’19.
Our FY’22 GAAP tax rate is expected to be approximately 20% and our non-GAAP tax rate is expected to be approximately 19%.
FY’22 capital expenditures are expected to be approximately $25 million (previously $30 million).
For the remainder of FY’22, we plan to focus on de-levering. In FY’23 and on a go-forward basis, assuming our Debt/EBITDA ratio is below 3x, our goal is to return approximately 50% of our free cash flow to shareholders via share repurchases.
PTC’s Fiscal Second Quarter Results Conference Call
The Company will host a conference call to discuss results at 5:00 pm ET on Wednesday, April 27, 2022. To participate in the live conference call, dial (888) 330-2508 or (240) 789-2735 and provide the passcode 7328695, or log in to the webcast, available on PTC’s Investor Relations website. A replay will also be available.

Important Disclosures
Important Information About Our Non-GAAP Financial Measures
PTC provides supplemental non-GAAP financial measures to its financial results. We use these non-GAAP financial measures, and we believe that they assist our investors, to make period-to-period comparisons of our operating performance because they provide a view of our operating results without items that are not, in our view, indicative of our operating results. These non-GAAP financial measures should not be construed as an alternative to GAAP results as the items excluded from the non-GAAP financial measures often have a material impact on our operating results, certain of those items are recurring, and others often recur. Management uses, and investors should consider, our non-GAAP financial measures only in conjunction with our GAAP results.

Non-GAAP operating expense, non-GAAP operating margin, non-GAAP gross profit, non-GAAP gross margin, non-GAAP net income and non-GAAP EPS exclude the effect of the following items: stock-based compensation; amortization of acquired intangible assets; acquisition-related and other transactional charges included in general and administrative expenses; restructuring and other charges, net; certain non-operating charges and credits; and income tax adjustments. Additional information about the items we exclude from our non-GAAP financial measures and the reasons we exclude them can be found in "Non-GAAP Financial Measures" on page 24 of our Annual Report on Form 10-K for the fiscal year ended September 30, 2021. In FY’21, we incurred tax expense related to a South Korean tax matter which is excluded from our non-GAAP financial measures as it is related to prior periods and not included in management’s view of results for comparative purposes. We also recorded a tax benefit in FY’21 related to the release of our U.S. valuation allowance as a result of the Arena acquisition and our conclusion that it is now more likely than not that we will realize the majority of our deferred tax assets in the U.S. As the non-GAAP tax provision is calculated assuming that there is no valuation allowance, this benefit has been excluded from our non-GAAP financial measures.

Free Cash Flow and Adjusted Free Cash Flow – PTC provides information on free cash flow and adjusted free cash flow to enable investors to assess our ability to generate cash without incurring additional external financings and to evaluate our performance against our announced long-term goals and intent to return approximately 50% of our free cash flow to shareholders via stock repurchases. Free cash flow is cash provided by (used in) operations net of capital expenditures. Adjusted free cash flow is free cash flow net of restructuring payments, acquisition-related payments, and non-ordinary course tax-related payments or receipts. Free cash flow and adjusted free cash flow are not measures of cash available for discretionary expenditures.

Constant Currency (CC) Change Metric – We present CC information to provide a framework for assessing how our underlying business performed excluding the effects of foreign currency rate fluctuations. To present CC information, FY’22 and comparative prior period results for entities reporting in currencies other than United States dollars are converted into United States dollars using the foreign exchange rate as of September 30, 2021, rather than the actual exchange rates in effect during that period.

Operating Measures
ARR – To help investors understand and assess the performance of our business as a SaaS and on-premise subscription company we provide an ARR (Annual Run Rate) operating measure. ARR represents the annualized value of our portfolio of active subscription software, cloud, SaaS, and support contracts as of the end of the reporting period. ARR includes orders placed under our Strategic Alliance Agreement with Rockwell Automation, including orders placed to satisfy contractual minimum commitments.

We believe ARR is a valuable operating metric to measure the health of a subscription business because it captures expected subscription and support cash generation from customers.

Zentalis Pharmaceuticals Announces $25 Million Equity Investment from Pfizer

On April 27, 2022 Zentalis Pharmaceuticals, Inc. (Nasdaq: ZNTL), a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapeutics targeting fundamental biological pathways of cancers, reported that it has agreed to sell 953,834 of its common shares at a price of $26.21 per share to Pfizer for gross proceeds of $25.0 million (the "Transaction") (Press release, Pfizer, APR 27, 2022, View Source [SID1234613277]). The common shares were offered and sold to Pfizer in a registered direct offering conducted without an underwriter or placement agent. The offering is expected to close on or about April 29, 2022, subject to customary closing conditions. Zentalis intends to use the net proceeds of the offering to help fund ongoing and planned clinical trials, including studies of ZN-c3, its Wee1 inhibitor, and ZN-d5, its BCL-2 inhibitor, and for working capital and general corporate purposes. With prioritization of the clinical development of ZN-c3 and ZN-d5, budget reallocation and Pfizer’s investment, the Company extends current cash runway into Q1 2024.

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Pfizer also expects to leverage its global development capabilities and expertise to enhance Zentalis’ clinical development program. The parties have entered into an agreement to collaborate to advance the clinical development of ZN-c3, a selective Wee1 inhibitor designed to induce synthetic lethality in cancer cells.

Zentalis will maintain full economic ownership and control of ZN-c3 and the rest of its pipeline.

Dr. Anthony Sun, Chairman and Chief Executive Officer of Zentalis, commented, "Given Pfizer’s deep expertise in developing treatments for genitourinary cancers, we are proud not only to receive capital support but to partner with them to develop a potential treatment option for patients living with cancer. We look forward to advancing our promising therapeutics to hopefully realize the full potential of our internally discovered oncology pipeline."

"We are proud to support Zentalis and to assist in the clinical advancement of its pipeline," said Chris Boshoff, M.D., Ph.D., FMedSci, Chief Development Officer, Pfizer Oncology. "We believe Zentalis’ commitment to developing innovative treatments holds promise, and we are looking forward to continuing our relationship to potentially help better the lives of people with cancer."

In addition, Adam Schayowitz, Ph.D., MBA, Vice President & Medicine Team Group Lead for Breast Cancer, Colorectal Cancer and Melanoma, Pfizer, will join Zentalis’ Scientific Advisory Board. Dr. Schayowitz will work closely with Zentalis’ senior management team to provide input on the Company’s clinical strategy.

The securities described above were offered by means of a prospectus supplement dated April 26, 2022, and accompanying prospectus dated May 4, 2021, forming a part of the Company’s effective shelf registration statement (File No. 333-255769). The prospectus supplement and accompanying prospectus relating to this offering will be filed with the U.S. Securities and Exchange Commission (the "SEC") and will be available on the SEC’s website at www.sec.gov.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy the common shares, nor shall there be any sale of the common shares 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.