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Ascendis Pharma A/S Reports Full Year 2020 Financial Results

On March 10, 2021 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, reported financial results for the full year ended December 31, 2020 (Press release, Ascendis Pharma, MAR 10, 2021, View Source [SID1234576387]).

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"2020 was a year to remember for Ascendis as our global workforce delivered on time or ahead of schedule on all of our corporate milestones, advancing Vision 3×3, the company’s strategic roadmap through 2025 to build a leading biopharma company by achieving sustainable growth through multiple approaches," said Jan Mikkelsen, Ascendis’ President and CEO. "As we look to 2021, we expect to report important clinical updates across our entire investigational product portfolio, including Endocrinology Rare Disease and Oncology, while also achieving key commercial milestones including the anticipated regulatory approval and commercial launch in the United States, and regulatory approval in Europe, of TransCon hGH for pediatric growth hormone deficiency."

Company Highlights & Progress

TransCon hGH (lonapegsomatropin)
○ Continued to build the global commercial infrastructure in preparation for the PDUFA date of June 25, 2021, and expected subsequent commercial launch of lonapegsomatropin in the United States in the third quarter of 2021. European Commission decision is anticipated in the fourth quarter of 2021 for pediatric patients with growth hormone deficiency (GHD).
○ Reported two year data from the enliGHten long-term extension trial demonstrating children with GHD initially treated with lonapegsomatropin maintained an advantage in height SDS (standard deviation score) improvement.
○ Continued execution in the ongoing foresiGHt Trial, a global phase 3 study in adults with GHD, complete enrollment expected by late 2021 or early 2022.
○ Submitted Clinical Trial Notification (CTN) to initiate pediatric GHD phase 3 riGHt Trial in Japan.

TransCon PTH
○ Submitted regulatory filings in North America and Europe to initiate PaTHway, a phase 3 clinical trial evaluating the safety, tolerability and efficacy of TransCon PTH in adults with hypoparathyroidism (HP). Topline results are expected from PaTHway in the fourth quarter of 2021.
○ Announced six-month data from the PaTH Forward Trial open-label extension (OLE), which demonstrated TransCon PTH replaced standard of care, normalized quality of life and urinary calcium. These results will be presented in an oral presentation on March 23, 2021 at ENDO 2021.
○ As of March 10, 2021, 58 out of the 59 randomized subjects continue in the phase 2 PaTH Forward clinical trial.
○ On track to file CTN to initiate adult HP phase 3 trial in Japan in the second quarter of 2021.

TransCon CNP
○ Continued dose escalation in the phase 2 ACcomplisH Trial to evaluate the safety and efficacy of TransCon CNP in children ages two to ten with achondroplasia.
○ IND approved for the ACcomplisH China Trial, a phase 2 trial to enable dose expansion cohorts, in children ages two to ten, conducted in Greater China by Visen Pharmaceuticals (VISEN).
○ Clinical program update on both randomized, double-blind, placebo-controlled trials expected in the fourth quarter of 2021.

TransCon TLR7/8 Agonist
○ Filed IND application with the FDA to initiate the transcendIT-101 clinical trial.
○ Initial results from the first part of transcendIT-101, the monotherapy dose escalation, are expected in the fourth quarter of 2021.
○ Expect to initiate the second part of transcendIT-101, dose escalation of TransCon TLR7/8 Agonist in combination with a checkpoint inhibitor, in the second quarter of 2021.

TransCon IL-2 β/γ
○ New preclinical data demonstrated a single dose of TransCon IL-2 β/γ resulted in durable and robust increases in the ratios of CD8+ T cells and NK cells over Treg cells in non-human primates.
○ IND filing or similar planned in the third quarter of 2021.

Ended 2020 with cash, cash equivalents and marketable securities totaling €834.1 million.
Full Year 2020 Financial Results

For the full year 2020, Ascendis Pharma reported a net loss of €419.0 million, or €8.28 per share (basic and diluted) compared to a net loss of €218.0 million, or €4.69 per share (basic and diluted) for the same period in 2019.

Revenue for 2020 was €7.0 million compared to €13.4 million in 2019. The decrease was due to lower license and service revenue, partly offset by sale of clinical supply, to VISEN.

Research and development (R&D) costs for 2020 were €260.9 million compared to €191.6 million in 2019. Higher R&D costs in 2020 reflect an increase in personnel-related costs, expansion of R&D facilities and the continued progress in development of the company’s product candidates.

Selling, general and administrative expenses for 2020 were €76.7 million compared to €48.5 million in 2019. The increase is primarily due to higher personnel-related costs, higher IT costs and continued build out of the company’s commercial capabilities.

Net loss in associate was €9.5 million for 2020 compared to €8.1 million in 2019, representing the company’s share of net result from VISEN.

Net finance expenses were €79.0 million for 2020 compared to net finance income of €16.6 million in 2019, primarily reflecting negative exchange rate fluctuations compared to 2019 when the company recognized a gain.

As of December 31, 2020, Ascendis Pharma had cash, cash equivalents and marketable securities totaling €834.1 million compared to €598.1 million at the beginning of the year. As of December 31, 2020, Ascendis Pharma had 53,750,386 ordinary shares outstanding.

Conference Call and Webcast information

Ascendis Pharma will host a conference call and webcast today at 4:30 p.m. Eastern Time (ET) to discuss its full year 2020 financial results.

A live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will also be available on this website shortly after conclusion of the event for 30 days.

Lantern Pharma Reports Fourth Quarter and Year End 2020 Financial Results and Operational Highlights

On March 10, 2021 Lantern Pharma Inc. (NASDAQ: LTRN), a clinical stage biopharmaceutical company using its proprietary RADR artificial intelligence ("A.I.") platform to transform oncology drug discovery and development reported financial results for the fourth quarter and fiscal year ended December 31, 2020 (Press release, Lantern Pharma, MAR 10, 2021, View Source;utm_medium=rss&utm_campaign=lantern-pharma-reports-fourth-quarter-and-year-end-2020 [SID1234576386]).

"2020 was a pivotal year for Lantern Pharma, marked by a series of financial, operational and drug development achievements. We believe each of these achievements further validates our unique capital-efficient business model that leverages the power of our proprietary RADR A.I. platform with the knowledge and experience of our scientific team aimed at developing precision oncology drugs," stated Panna Sharma, President and CEO of Lantern Pharma. "We anticipate 2021 to be a transformational year for Lantern and our shareholders as each of our drug programs progresses towards key milestones, including the initiation of a Phase 2 trial of LP-300 in NSCLC among non-smokers, IND-enabling studies of LP-184 in multiple solid tumors, advancing our ADC program, and continued growth in the biologically-relevant and curated data that powers our RADR A.I. platform."

"As a result of our rapid development and operational progress after our June IPO, we were able to significantly strengthen our balance sheet with the closing of a $69.0 million public offering in January 2021. Our solid financial position is expected to fuel continued growth and evolution of our RADR A.I. platform, accelerate the development of our portfolio of targeted oncology drug candidates and allow us to introduce additional targeted opportunities in a capital efficient manner," continued Sharma. "In a very short time since our IPO in June 2020, we have:

More than doubled the number of programs in development, increasing our "shots on goal" and the number of opportunities for potentially accretive licensing or partnering opportunities.
Initiated a differentiated Antibody Drug Conjugate (ADC) program with novel linking technologies.
Grew by over 5x the number of datapoints that fuel our RADR A.I. platform.
Added significant additional functionality into our RADR A.I. platform.
Initiated multiple research and development collaborations with leading cancer centers, including: Johns
Hopkins in GBM and other brain cancers, Georgetown University in prostate cancer, and Fox Chase Cancer Center in pancreatic cancer.
The rapid development and capital-efficient, collaborative approach of our business showcases the power and potential of A.I. and machine learning to transform the pace, risk and cost of oncology drug discovery and development."

Lantern is developing three drug candidates and an ADC program across seven disclosed targets, including:

LP-100 (Irofulven), in a Phase 2 trial for the treatment of metastatic castration resistant prostate cancer (mCRPC) which is out-licensed to Allarity Therapeutics.
LP-300, a small molecule candidate that is preparing to enter a Phase 2 trial as a combination therapy in non-smokers with Non-Small Cell Lung Cancer (NSCLC).
LP-184, a small molecule DNA damaging candidate in preclinical development for genomically-defined prostate, pancreatic, glioblastoma multiforme (GBM), atypical teratoid rhabdoid tumors (ATRT) and other undisclosed tumors defined by overexpression of PTGR1.
Antibody Drug Conjugate (ADC) program leverages RADR and is aimed at identifying targeted or therapeutic antibodies, utilizing a unique library of linkers to conjugate with LP-184 and other compounds.
Below, a recap of milestones since the June 15, 2020 IPO that position Lantern for further achievements in 2021:

Drug Development Achievements:

Expanded the pipeline from three tumor targets in development to seven, including: LP-184 in prostate, pancreatic and multiple CNS tumors where PTGR1 is overexpressed, such as GBM and ATRT. We anticipate disclosing additional programs in the coming year.
Initiated an Antibody Drug Conjugate platform that adds a key dimension to Lantern’s focus on leveraging biological data and innovative platforms to accelerate cancer drug development.
RADR A.I. platform grew to over 1.2 billion datapoints at year end 2020 from 275 million at IPO.
Published several peer reviewed publications at ASCO (Free ASCO Whitepaper), AACR (Free AACR Whitepaper) and other symposia on the use of our RADR A.I. platform, including the development of RNA expression signatures for predicting response to our portfolio of oncology drug candidates.
Advanced LP-300 in NSCLC towards a planned launch of a Phase 2 trial in Q3 2021.
Operational Achievements:

Established manufacturing network for the company’s pipeline of targeted drug candidates.
Initiated R&D and CRO collaborations to support capital efficient pre-clinical validation and development of novel small molecule oncology drug candidates.
Initiated collaborations with recognized KOLs in prostate, pancreatic and CNS cancers.
Strengthened our intellectual property estate with over 15 new patent applications.
Significantly expanded our data sciences and research teams.
Financial Highlights:

Successfully completed a $26.3 million IPO on June 15, 2020.
Strengthened the Balance Sheet with a $69.0 million follow-on public offering in January 2021.
Extended the cash runway through mid-2025, allowing the company to focus on efficiently developing our portfolio of promising oncology therapeutics.
Fourth Quarter 2020 Financial Highlights

Cash Position: Cash and cash equivalents were $19.2 million as of December 31, 2020, compared to $20.8 million as of September 30, 2020 and $1.2 million as of December 31, 2019. The quarterly cash burn reflects our capital-efficient, collaborator-centered business model. The year-over-year increase in cash balance reflects proceeds from the June 2020 IPO. On January 20, 2021, we completed a follow-on public offering resulting in gross proceeds of $69.0 million.
R&D Expenses: Research and development expenses were $1,348,329 for the quarter ended December 31, 2020, compared to $177,467 for the quarter ended December 31, 2019. The increase was primarily attributable to increases in research studies, expansion of the company’s research team, and research and development related stock option compensation expense of $470,401 (a non-cash item) for the quarter ended December 31, 2020.
G&A Expenses: General and administrative expenses were $1,547,675 for the quarter ended December 31, 2020, compared to $497,700 for the quarter ended December 31, 2019. The increase was primarily attributable to expenses associated with operating as a public company and general and administrative related stock option compensation expense of $554,503 (a non-cash item) for the quarter ended December 31, 2020.
Net Loss: Net losses were $2,896,004 for the quarter ended December 31, 2020, or $0.47 per share, compared to a net loss of $675,167 for the quarter ended December 31, 2019, or $0.34 per share. The net losses include non-cash expenses related to employee stock options of $1,024,904 for the quarter ended December 31, 2020.
Fiscal Year 2020 Financial Highlights

R&D Expenses: Research and development expenses were $2,243,225 for the year ended December 31, 2020, compared to $953,185 for the year ended December 31, 2019. The increase was primarily attributable to research and development labor and research study expenses, as well as an increase of $470,401 (a non-cash item) in research and development related stock option compensation expense.
G&A Expenses: General and administrative expenses were $3,664,965 for the year ended December 31, 2020, compared to $1,475,000 for the year ended December 31, 2019. The increase was primarily attributable to expenses associated with transitioning to and becoming a public company, including corporate insurance expenses, general and administrative labor expenses, and $721,840 (a non-cash item) in general and administrative related stock option compensation expense.
Net Loss: Net loss was $5,908,190 for the year ended December 31, 2020, or $1.37 per share, compared to a net loss of $2,428,185 for the year ended December 31, 2019, or $1.23 per share. The net loss for the fiscal year ended December 31, 2020 includes non-cash expenses related to employee stock options of $1,192,241.
Mr. Sharma continued, "The golden age of A.I. in medicine is beginning, and Lantern Pharma is among the leaders in this paradigm shift to transform the pace, risk and cost of oncology drug discovery and development. With our RADR A.I. platform we are demonstrating the opportunity for attainment of significant efficiencies in the time and cost of oncology drug discovery and development. As our growing pipeline of oncology drug candidates demonstrates, the rapid, machine learning enabled identification and validation of molecular drivers of cancer provides the potential for more targeted and more effective oncology therapies. During the fourth quarter of 2020 we were able to identify and validate an entirely new indication for LP-184 in a type of ultra-rare brain cancer, ATRT, that presents primarily in children. This discovery along with additional CNS opportunities that we are in the process of validating was enabled by using our RADR A.I. platform. As our RADR A.I. platform grows over the coming year, we anticipate the identification of additional high value targets and indications as monotherapies, combination therapies or as part of our ADC program."

Conference Call

Lantern will host a conference call and webcast today, Wednesday, March 10 at 4:00 p.m. ET.

Avid Bioservices, Inc. Announces Pricing of $125 Million Offering of Exchangeable Senior Notes

On March 10, 2021 Avid Bioservices, Inc. (NASDAQ:CDMO) (NASDAQ:CDMOP) (the "company"), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality development and manufacturing services to biotechnology and pharmaceutical companies, reported that its wholly-owned subsidiary, Avid SPV, LLC (the "Issuer"), has priced its sale of $125 million aggregate principal amount of exchangeable senior notes due 2026 (the "notes") in a private placement to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act") (Press release, Avid Bioservices, MAR 10, 2021, View Source [SID1234576385]). The issuance and sale of the notes are scheduled to settle on March 12, 2021, subject to customary closing conditions. The Issuer also granted the initial purchasers of the notes a 13-day option to purchase up to an additional $18.75 million aggregate principal amount of the notes.

The notes will be senior, unsecured obligations of the Issuer, will be fully and unconditionally guaranteed by the company on a senior, unsecured basis, and will accrue interest at a rate of 1.250% per annum payable semi-annually in arrears on March 15 and September 15 of each year, beginning on September 15, 2021. The notes will mature on March 15, 2026, unless earlier repurchased, redeemed or exchanged. Before September 15, 2025, noteholders will have the right to exchange their notes only upon the occurrence of certain events. From and after September 15, 2025, noteholders may exchange their notes at any time at their election until the close of business on the second scheduled trading day immediately before the maturity date of the notes. The notes will be settled in cash, shares of the company’s common stock or a combination of cash and shares of the company’s common stock, at the Issuer’s election.

The initial exchange rate is 47.1403 shares of the company’s common stock per $1,000 principal amount of notes (which represents an initial exchange price of approximately $21.21 per share of the company’s common stock). The initial exchange price represents a premium of approximately 32.5% over the last reported sale price of $16.01 per share of the company’s common stock on March 9, 2021. The exchange rate and exchange price of the notes will be subject to adjustment upon the occurrence of certain events.

The notes will be redeemable, in whole or in part, for cash at the Issuer’s option at any time, and from time to time, on or after March 20, 2024 if the last reported sale price of the company’s common stock has been at least 130% of the exchange price then in effect for at least 20 trading days (whether or not consecutive) during any 30 consecutive trading day period (including the last trading day of such period) ending on, and including, the trading day immediately preceding the date on which the Issuer provides notice of redemption at a redemption price equal to the principal amount of the notes to be redeemed, plus accrued and unpaid interest, if any, to, but excluding, the redemption date.

If a "fundamental change" (as defined in the indenture for the notes) occurs, then noteholders may require the Issuer to repurchase their notes for cash. The repurchase price will be equal to the principal amount of the notes to be repurchased, plus accrued and unpaid interest, if any, to, but excluding, the applicable repurchase date.

The net proceeds from the offering are estimated to be approximately $120.6 million (or approximately $138.7 million if the initial purchasers fully exercise their option to purchase additional notes), after deducting the initial purchasers’ discounts and commissions and estimated offering expenses. The Issuer expects to make an intercompany loan to the company of all of the net proceeds from this offering. The company intends to use approximately $11.2 million of such loan to pay the cost of the capped call transactions described below, and to use up to approximately $41.3 million of such loan to redeem all of the company’s outstanding 10.50% Series E Convertible Preferred Stock (assuming such redemption occurs on April 10, 2021, all such shares remain outstanding through such date and none of such shares are converted into the company’s common stock prior to such redemption). The company intends to use the remaining net proceeds for working capital and other general corporate purposes. If the initial purchasers exercise their option to purchase additional notes, the Issuer expects to make an intercompany loan to the company of all of the net proceeds from the sale of additional notes, which the company intends to use to pay the cost of additional capped call transactions and for working capital and other general corporate purposes. The company may also use a portion of such loans for the acquisition of, or investment in, technologies, solutions or businesses that complement the company’s business, although it has no commitments to enter into any such acquisitions or investments at this time.

In connection with the pricing of the notes, the company entered into privately negotiated capped call transactions with the initial purchasers or their affiliates (the "option counterparties"). The capped call transactions cover, subject to customary adjustments, the number of shares of the company’s common stock that initially underlie the notes. The capped call transactions are expected to reduce or offset the potential dilution of the company’s common stock as a result of any exchange of the notes and/or offset any potential cash payments the Issuer is required to make in excess of the principal amount of exchanged notes, as the case may be, with such reduction and/or offset subject to a cap. If the initial purchasers exercise their option to purchase additional notes, the company expects to enter into additional capped call transactions with the option counterparties. The cap price of the capped call transactions will initially be approximately $28.02 per share of the company’s common stock, which represents a premium of approximately 75.0% over the last reported sale price of the company’s common stock of $16.01 per share on March 9, 2021, and is subject to certain adjustments under the terms of the capped call transactions.

In connection with establishing their initial hedges of the capped call transactions, the option counterparties and/or their respective affiliates may purchase shares of the company’s common stock and/or enter into various derivative transactions with respect to the company’s common stock concurrently with, or shortly after, the pricing of the notes, including with certain investors in the notes. This activity could increase (or reduce the size of any decrease in) the market price of the company’s common stock or the notes at that time.

In addition, the option counterparties and/or their respective affiliates may modify their hedge positions by entering into or unwinding various derivatives with respect to the company’s common stock and/or purchasing or selling the company’s common stock or other securities in secondary market transactions following the pricing of the notes and prior to the maturity of the notes (and are likely to do so on each exercise date for the capped call transactions, which are expected to occur during the 40 trading day period beginning on the 41st scheduled trading day prior to the maturity date of the notes). This activity could also cause or avoid an increase or decrease in the market price of the company’s common stock or the notes, which could affect the ability of noteholders to exchange the notes, and, to the extent the activity occurs during any observation period related to an exchange of notes, it could affect the number of shares of the company’s common stock and value of the consideration that holders of the notes will receive upon exchange of the notes.

Neither the notes, nor any shares of the company’s common stock potentially issuable upon exchange of the notes, have been, nor will be, registered under the Securities Act or any state securities laws and, unless so registered, may not be offered or sold in the United States absent registration or an applicable exemption from, or in a transaction not subject to, the registration requirements of the Securities Act and other applicable securities laws.

This press release is neither an offer to sell nor a solicitation of an offer to buy any securities, nor shall it constitute an offer, solicitation or sale of the securities in any 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. The notes will be offered to qualified institutional buyers pursuant to Rule 144A under the Securities Act.

Linnaeus Therapeutics Granted Orphan Drug Designation for LNS8801 for the Treatment of Patients with Metastatic Uveal Melanoma

On March 10, 2021 Linnaeus Therapeutics, Inc. (Linnaeus), a privately held clinical-stage biopharmaceutical company focused on the development and commercialization of novel small-molecule oncology therapeutics, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for LNS8801 for the treatment of patients with metastatic uveal melanoma (MUM) (Press release, Linnaeus Therapeutics, MAR 10, 2021, View Source [SID1234576383]).

The FDA’s Office of Orphan Drug Products grants orphan drug status to support drug candidates in development for underserved patient populations or rare disorders that affect fewer than 200,000 people in the United States. Orphan drug designation provides certain benefits, including market exclusivity upon FDA approval, exemption of FDA application fees, and tax credits for qualified clinical trials.

"We are extremely pleased to have received orphan drug designation for MUM from the FDA. This is an important milestone that has emerged from the very promising data we have seen in patients with MUM during dose escalation," commented Patrick Mooney, MD, CEO of Linnaeus. "We look forward to further exploring the preliminary results with LNS8801 alone and also in combination with pembrolizumab when we open additional cohorts soon."

Having completed dose escalation, Linnaeus is currently testing LNS8801 in its phase 1/2 adaptive-design clinical trial as a monotherapy and in combination with KEYTRUDA (pembrolizumab) in patients who had previous clinical benefit from immune checkpoint inhibitors and then subsequently progressed. This marks the first time any company has dosed a patient in a clinical trial specifically targeting the G protein-coupled estrogen receptor (GPER) in combination with pembrolizumab. Linnaeus intends to open a LNS8801 monotherapy cohort in patients with MUM and also a combination therapy cohort testing LNS8801 and pembrolizumab in patients with MUM in the near term as well as other targeted indications.

About LNS8801
LNS8801 is an orally bioavailable and highly specific and potent agonist of GPER whose activity is dependent on the expression of GPER. GPER activation by LNS8801 rapidly and durably depletes c-Myc protein levels. In preclinical cancer models, LNS8801 displays potent antitumor activities across a wide range of tumor types, rapidly shrinking tumors and inducing immune memory.

In the ongoing phase 1/2 study in humans, LNS8801 monotherapy has been safe and well tolerated. Additionally, LNS8801 has demonstrated target engagement, c-Myc protein depletion, and clinical benefit in patients with advanced cancer. Data from the phase 1/2 study are anticipated to be presented in a peer-reviewed setting in 2021.

Infinity to Present at the Oppenheimer & Co. Annual Healthcare Conference

On March 10, 2021 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI), a clinical-stage biotechnology company developing eganelisib, a potentially first-in-class, oral, immuno-oncology macrophage reprogramming therapeutic which addresses a fundamental biologic mechanism of immune suppression in cancer, reported that management will present at the Oppenheimer & Co. Annual Healthcare Conference which is being held Tuesday, March 16th – Thursday, March 18th, 2021 (Press release, Infinity Pharmaceuticals, MAR 10, 2021, View Source [SID1234576382]). Presentation details are as follows:

Oppenheimer & Co. Annual Healthcare Conference Company Presentation:

Date:

Wednesday, March 17

Time:

1:10 pm Eastern Time

Webcast:

View Source

The webcast of the presentation can be accessed in the Investors/Media section of Infinity’s website at www.infi.com and will be available on Infinity’s website for 30 days following the event.