On November 8, 2023 Lipocine Inc. (NASDAQ: LPCN), a biopharmaceutical company focused on treating Central Nervous System (CNS) disorders by leveraging its proprietary platform to develop differentiated products, reported financial results for the third quarter and nine months ended September 30, 2023, and provided a corporate update (Press release, Lipocine, NOV 8, 2023, View Source [SID1234637266]).

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Clinical Program Highlights

Neuroactive Steroids

● On October 18, Lipocine completed a successful meeting with the FDA on LPCN 1154, which is in development for postpartum depression (PPD). The FDA agreed on Lipocone’s proposal for a 505(b)(2) NDA filing based on a single pivotal study comparing exposure of LPCN 1154 with the approved IV infusion of brexanolone

○ Lipocine anticipates initiating the pivotal study program in Q1 2024 with the LPCN 1154 "to be marketed" formulation
○ Top line results from the study are expected by Q2 2024, with a goal of filing a New Drug Application (NDA) in 2024
○ If approved, LPCN 1154, has the potential to be a differentiated preferred treatment option for PPD with rapid and high remission/response rates with short treatment duration

LPCN 1148 in liver cirrhosis

● In July, Lipocine announced positive topline results from its Phase 2 proof-of-concept ("POC") study evaluating LPCN 1148 in cirrhosis

○ Study met primary endpoint: treatment with LPCN 1148 increased L3 skeletal muscle index (L3-SMI) relative to placebo (P <0.01)
○ Fewer hepatic encephalopathy (HE) events of grade >1 in the LPCN 1148 treatment arm relative to placebo (P < 0.05)
○ More patients on LPCN 1148 reported symptom improvement compared to placebo (P < 0.05)
○ LPCN 1148 was well-tolerated, with AE rates and severities similar to placebo
○ Lipocine plans to meet with the FDA to discuss the development path to NDA filing

Quarter Ended September 30, 2023 Financial Results

Lipocine reported a net loss of $6.7 million, or ($1.27) per diluted share, for the three months ended September 30, 2023, compared with a net loss of $2.4 million or ($0.52) per diluted share, in the three months ended September 30, 2022.

During the three months ended September 30, 2023, the Company recognized a non-cash minimum guaranteed royalties revenue reversal of variable consideration revenue of $3.1 million related to the termination of the Antares License Agreement. The reversal of revenue is due to the fact that Lipoocine will not receive anticipated royalties that were previously recorded for the Antares License Agreement due to the termination of the agreement.

Research and development expenses were $2.9 million during the three months ended September 30, 2023, as compared with $2.1 million in the three months ended September 30, 2022. The increase in research and development expenses was a result of an increase in costs related to the LPCN 1154 clinical studies, an increase in TLANDO manufacturing related costs, and an increase in personnel related costs, offset by a decrease in LPCN 1111 scale up costs in 2022, a decrease in contract research organization expense related to the LPCN 1148 Phase 2 POC study in male subjects with cirrhosis, a decrease in contract research organization expense and outside consulting costs related to the completion of the LPCN 1144 LiFT study in 2022, and a decrease in LPCN 1107 PK and food effect studies and other research and development costs in 2022.

General and administrative expenses were $1.0 million during the three months ended September 30, 2023, as compared to $0.8 million in the three months ended September 30, 2022. The increase in expenses is mainly due to increases in business development expenses, and an increase in professional services and legal fees. These increases were offset by a decrease in corporate insurance expense.

As of September 30, 2023, Lipocine had $23.8 million of unrestricted cash, cash equivalents and marketable investment securities compared to $32.5 million at December 31, 2022.

Nine Months Ended September 30, 2023 Financial Results

Lipocine reported a net loss of $14.1 million, or ($2.72) per diluted share, for the nine months ended September 30, 2023, compared with a net loss of $8.5 million or ($1.72) per diluted share, in the nine months ended September 30, 2022

The Company recognized a non-cash minimum guaranteed royalties revenue reversal of variable consideration revenue of $3.1 million related to the termination of the Antares License Agreement during the nine months ended September 30, 2023. The reversal of variable consideration revenue is offset by license revenue of approximately $55,000 for payments received from Spriaso, a related party, under a licensing agreement for the cough and cold field during the nine months ended September 30, 2023. The Company recognized revenue related to a non-refundable cash fee of $500,000 received from Antares for consideration of a 90-day extension for Antares to exercise its option to license LPCN 1111 during the nine months ended September 30, 2022.

Research and development expenses were $8.5 million and $6.9 million, respectively, for the nine months ended September 30, 2023, and 2022. The increase was due to an increase in costs related to the LPCN 1154 clinical studies, an increase in TLANDO manufacturing related costs, an increase in contract research organization expense related to the LPCN 1148 Phase 2 POC study in male subjects with cirrhosis, and an increase in personnel salaries and benefits. These increases were offset by a decrease related to LPCN 1111 scale up costs in 2022, a decrease in contract research organization expense and outside consulting costs related to the completion of our LPCN 1144 LiFT study, a decrease related to the completion of our LPCN 1107 PK and food effect studies and a decrease in other research and development activities.

General and administrative expenses were $3.8 million and $3.2 million, respectively, for the nine months ended September 30, 2023, and 2022. The increase consisted of an increase in business development expenses, an increase in professional and legal fees and an increase in other general and administrative expenses. These increases were offset by decrease resulting from a decrease in corporate insurance expense and a decrease in various other consulting fees.

For more information on Lipocine’s financial results for the three and nine months ended September 30, 2023, refer to Form 10Q filed with the SEC.

On November 8, 2023 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported financial results for the three and nine months ended September 30, 2023, and provided an operating forecast and business updates (Press release, Ligand, NOV 8, 2023, View Source [SID1234637265]). Ligand management will host a conference call today beginning at 4:30 p.m. Eastern Time to discuss this announcement and answer questions.

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"We’re pleased to report another quarter of strong financial results and we are now actively executing on our investment strategy as evidenced by the recent Tolerance, Ovid, Novan and Primrose transactions, in which we collectively invested over $75 million in Q3 and early Q4 2023," said Todd Davis, CEO of Ligand. "We have a strong balance sheet and are generating positive cash flow which positions us advantageously in this market to further build a robust portfolio of assets that are expected to drive future revenue growth. We look forward to sharing our long term financial forecast at our upcoming Investor and Analyst Day scheduled for December 12th in New York City."

Third Quarter 2023 Financial Results

Total revenues for the third quarter of 2023 were $32.9 million. Revenues for the same period in 2022 excluding sales related to COVID-19 were $26.9 million. Total revenues for the third quarter of 2022 including COVID-19 related sales were $59.2 million. Royalties for the third quarter of 2023 were $23.9 million, compared with $19.3 million for the same period in 2022, with the increase primarily attributable to Amgen’s (Nasdaq: AMGN) Kyprolis, Jazz Pharmaceuticals’ (Nasdaq: JAZZ) RYLAZE, Merck and Co.’s (NYSE: MRK) Vaxneuvance and Travere Therapeutics’ (Nasdaq: TVTX) FILSPARI. Core Captisol sales were $8.6 million for the third quarter of 2023, compared with $3.6 million for the same period in 2022, with the increase due to the timing of customer orders. There were no Captisol sales related to treatments for COVID-19 for the third quarter of 2023, compared with $32.4 million for the same period in 2022. Contract revenue was $0.4 million for the third quarter of 2023, compared with $4.0 million for the same period in 2022, with the difference due to the timing of partner milestone events.

Cost of Captisol was $3.5 million for the third quarter of 2023, compared with $14.2 million for the same period in 2022, with the decrease due to lower total Captisol sales. Amortization of intangibles was $8.2 million, compared with $8.6 million for the same period in 2022. Research and development expense was $5.5 million, compared with $9.2 million for the same period in 2022, with the decrease attributed to lower stock-based compensation, employee related expenses and lab supply expenses. General and administrative expense was $14.7 million, compared with $14.9 million for the same period in 2022, with the transaction costs associated with the Novan acquisition and Pelican spin out included in the third quarter of 2023.

Net loss from continuing operations for the third quarter of 2023 was $12.8 million, or $0.74 per share, compared with net income from continuing operations of $9.6 million, or $0.56 per diluted share, for the same period in 2022. The decrease in net income from the prior year period is due primarily to the decrease in COVID-19 related Captisol sales and the non-cash unrealized loss from short-term investments associated with Viking Therapeutics (Nasdaq: VKTX) stock of $11.5 million. Adjusted net income from continuing operations for the third quarter of 2023 was $18.0 million, or $1.02 per diluted share, compared with $10.5 million, or $0.60 per diluted share, for the same period in 2022. See the table below for a reconciliation of net income from continuing operations to adjusted net income from continuing operations.

As of September 30, 2023, Ligand had cash, cash equivalents and short-term investments of $191.1 million. On October 12, 2023, Ligand entered into a credit agreement with Citibank, N.A., which provides for a $75 million revolving credit facility with a maturity date of October 12, 2026.

Year-to-Date Financial Results

Total revenues for the nine months ended September 30, 2023 were $103.2 million. Revenues for the same period in 2022 excluding sales related to COVID-19 were $81.4 million. Revenues for the nine months ended September 30, 2022 including COVID-19 related sales were $145.9 million. Royalties for the nine months ended September 30, 2023 were $61.4 million, compared with $50.5 million for the same period in 2022, with the increase primarily attributable to Kyprolis, Rylaze, Vaxnuevance, Pneumsil, and FILSPARI. Core Captisol sales were $24.5 million for the nine months ended September 30, 2023, compared with $13.1 million for the same period in 2022. The difference in sales was due to the timing of customer orders. There were no Captisol sales related to COVID-19 for the nine months ended September 30, 2023, compared with $64.5 million for the same period in 2022. Contract revenue was $17.3 million for the nine months ended September 30, 2023, compared with $17.7 million for the same period in 2022.

Cost of Captisol was $8.9 million for the nine months ended September 30, 2023, compared with $31.2 million for the same period in 2022, with the decrease due to lower total Captisol sales. Amortization of intangibles was $25.3 million for the nine months ended September 30, 2023, compared with $25.7 million for the same period in 2022. Research and development expense for the nine months ended September 30, 2023 was $19.0 million, compared with $26.9 million for the same period in 2022, with the decrease attributed to lower employee related expenses and lab supply expenses. General and administrative expense for the nine months ended September 30, 2023 was $36.8 million, compared with $38.9 million for the same period in 2022, with the decrease primarily attributable to lower legal expenses.

Net income from continuing operations for the nine months ended September 30, 2023 was $33.1 million, or $1.86 per diluted share, compared with net income from continuing operations of $9.3 million, or $0.54 per diluted share, for the same period in 2022. The increase in net income was driven by a gain from short term investments of $30.3 million in the current year period compared to a loss from short term investments of $15.7 million for the same period in 2022. Adjusted net income from continuing operations for the nine months ended September 30, 2023 was $83.0 million, or $4.71 per diluted share, compared with $28.9 million, or $1.69 per diluted share, for the same period in 2022. See the table below for a reconciliation of net income from continuing operations to adjusted net income from continuing operations.

On May 15, 2023, the maturity date of the convertible senior unsecured notes due 2023 (the 2023 Notes), we paid off the remaining balance in amount of $77.1 million (including interest). In the second quarter of 2023, Ligand put in place a $50.0 million share repurchase program that expires in April 2026. The timing and amount of repurchase transactions, if any, will be determined by the Company’s management based on its evaluation of market conditions, share price, legal requirements and other factors.

2023 Financial Guidance

Ligand is increasing its 2023 revenue guidance to be in the range of $126 million to $129 million (previously $124 million to $126 million) and is raising adjusted EPS guidance. Sales of Captisol are now expected to range from $27 million to $28 million (previously $25 million). Guidance for royalties is unchanged at $82 million to $84 million and contract revenue guidance is unchanged at $17 million. We now expect 2023 adjusted diluted EPS of $5.25 to $5.40 (previously $5.10 to $5.25). The increase in EPS guidance is driven primarily by the increase in revenue and lower operating expenses. Due to the unpredictable nature of the pandemic and related Captisol sales, Ligand excludes Captisol sales related to COVID-19 from guidance and will update investors each quarter as orders are received and shipped.

Third Quarter 2023 Business Highlights

On September 18, Ligand spun-out its Pelican subsidiary through a merger with Primordial Genetics, to form a privately held company, Primrose Bio. Ligand retained existing royalty rights from the Pelican Expression Technology, including Jazz’s Rylaze, Merck’s Vaxneuvance and V116 vaccines, Alvogen’s teriparatide, and Serum Institute of India’s Pneumosil and MenFive vaccines. Additionally, Ligand owns 49.9% of the equity of Primrose Bio. Simultaneous with the merger, Ligand entered into a Purchase and Sale Agreement with Primrose Bio and invested $15 million to acquire economic rights in future programs including two contracts previously entered into by Primordial Genetics and an economic interest in future revenue generated from PeliCRM197. Primrose Bio is a leading synthetic biology company with solutions to the industry’s protein design, formulation and expression challenges. The transaction is expected to reduce ongoing cash expenses and be immediately accretive to Ligand’s adjusted EPS.

On October 18, Ligand invested $30 million to acquire 13% of Ovid Therapeutics’ interest in the royalties and milestones owed to Ovid Therapeutics Inc. on soticlestat, a program Takeda Pharmaceutical Company is developing in two pivotal Phase 3 trials in Lennox-Gastaut and Dravet syndromes, respectively, both rare disease conditions. Under the terms of the 2021 agreement between Ovid and Takeda, Ovid is eligible to receive regulatory and commercial milestone payments of up to $660 million, as well as tiered royalties on global net sales of soticlestat at percentages ranging from the low double-digits up to 20%. If soticlestat is approved. Ligand’s 13% purchase entitles the Company to receive up to $86 million in regulatory and commercial milestones, and tiered royalties up to 2.6%.

On September 27, Ligand acquired certain assets of Novan Inc. for $12.2 million. As part of the transaction, Ligand acquired the NDA-stage berdazimer gel 10.3% program, all the assets related to the NITRICIL delivery technology platform, and the rights to the Sitavig program. Berdazimer gel 10.3% remains on track for a PDUFA goal date of January 5, 2024, as the first potential at-home treatment for molluscum contagiosum. The Novan team is preparing to commercialize berdazimer gel 10.3% in the second half of 2024. Ligand is incubating the business to prepare for a spin-out or strategic partnering.

On October 31, Ligand acquired Tolerance Therapeutics, a private company which owns a less than 1% royalty on worldwide net sales of TZIELD (teplizumab-mzwv). Ligand invested $20 million to acquire Tolerance Therapeutics and expects it to be immediately accretive to Ligand’s royalty revenue. TZIELD is the first disease-modifying therapy in type 1 diabetes ("T1D"). It is a CD3-directed antibody indicated to delay the onset of Stage 3 T1D in adults and in children ages 8 years and older with Stage 2 T1D. TZIELD was granted Breakthrough Therapy Designation in 2019 and was approved by the U.S. Food and Drug Administration ("FDA") in November 2022. TZIELD is marketed by Sanofi S.A. following its acquisition of Provention Bio, Inc., in 2023 for $2.9 billion. Sanofi recently announced new data from TZIELD’s PROTECT Phase 3 trial which showed TZIELD’s potential to slow the progression of Stage 3 type 1 diabetes in newly diagnosed children and adolescents. TZIELD met the study’s primary endpoint, significantly slowing the decline of C-peptide levels, compared to placebo.

Portfolio Updates

On November 7, 2023 Travere Therapeutics (Nasdaq: TVTX) announced that 430 new patient start forms (PSFs) were received in the third quarter and a total of 990 PSFs have been received since the accelerated approval of FILSPARI was obtained in the first quarter of 2023. Additionally, Travere previously announced topline two-year confirmatory secondary endpoint results from the pivotal Phase 3 PROTECT Study of FILSPARI versus irbesartan in IgA nephropathy ("IgAN"). FILSPARI demonstrated long-term kidney function preservation and achieved a clinically meaningful difference in estimated glomerular filtration rate ("eGFR") total and chronic slope versus irbesartan, narrowly missing statistical significance in eGFR total slope while achieving statistical significance in eGFR chronic slope for purposes of regulatory review in the EU. All topline efficacy endpoints favored FILSPARI and patients treated with FILSPARI over two years exhibited one of the slowest annual rates of kidney function decline seen in a clinical trial of IgAN patients. Travere will engage with regulators and expects to submit a supplemental New Drug Application ("sNDA") in the first half of 2024 for full approval in the U.S.

On October 26, 2023 Merck (NYSE: MRK) announced third quarter 2023 Vaxneuvance sales of $214 million. Merck previously reported Vaxneuvance sales of $168 million and $106 million in the second and first quarter of 2023, respectively. Additionally, Merck previously announced its Phase 3 clinical trial of V116, an investigational 21-valent pneumococcal conjugate vaccine, met key immunogenicity and safety endpoints in two Phase 3 trials. If approved, V116 would be the first pneumococcal conjugate vaccine specifically designed for adults. Results from the STRIDE-3 trial demonstrated statistically significant immune responses compared to PCV20 (pneumococcal 20-valent conjugate vaccine) in vaccine-naïve adults for serotypes common to both vaccines. Positive immune responses were also observed for serotypes unique to V116. Additionally, results from STRIDE-6 demonstrated that V116 was immunogenic for all 21 pneumococcal serotypes in the vaccine among adults who previously received a pneumococcal vaccine at least one year prior to the study. In both studies, V116 had a safety profile comparable to the comparator in the studies.

Jazz Pharmaceuticals announced that the European Commission has granted marketing authorization for Enrylaze for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia and lymphoblastic lymphoma in adult and pediatric patients (1 month and older) who developed hypersensitivity or silent inactivation to E. coli-derived asparaginase. Enrylaze, approved as Rylaze in the US and Canada, is a new Erwinia-derived asparaginase developed using the Pfenex Expression Technology with a safety profile consistent with that of other asparaginase preparations. Enrylaze may be given by either intravenous infusion or intramuscular injection and is dosed on either alternate days (every 48 hours) or via a Monday/Wednesday/Friday dosing schedule. The use of the Pfenex Expression Technology to manufacture Enrylaze delivers a scalable supply, able to meet global demand, and a ready-to-use solution that avoids the need for reconstitution in the clinic.

Verona Pharma plc (Nasdaq: VRNA) announced that the FDA has accepted for review its New Drug Application seeking approval of ensifentrine for the maintenance treatment of patients with COPD. The FDA has assigned a PDUFA date of June 26, 2024, and is not currently planning to hold an advisory committee meeting to discuss the application.

Anebulo Pharmaceuticals Inc. (Nasdaq: ANEB) announced positive feedback from the FDA following a Type B meeting in July. The FDA indicated that a single well-controlled study of ANEB-001 in Acute Cannabinoid Intoxication patients presenting to the emergency department combined with a larger THC challenge study in volunteers could potentially provide substantial evidence to support a new drug application.

Adjusted Financial Measures

Ligand reports adjusted net income and adjusted net income per diluted share in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company’s financial measures under GAAP include share-based compensation expense, amortization of debt-related costs, amortization related to acquisitions and intangible assets, changes in contingent liabilities, mark-to-market adjustments for amounts relating to its equity investments in public companies, excess tax benefit from share-based compensation, income tax affect of adjusted reconciling items and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included at the end of this press release. However, the Company does not provide reconciliations of such forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in contingent liabilities, changes in the market value of its investments in public companies, share-based compensation expense and the effects of any discrete income tax items. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing the Company’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

Conference Call

Ligand management will host a conference call today beginning at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss this announcement and answer questions. To participate via telephone, please dial (888) 350-3452 using the conference ID 6501694. Callers outside the U.S. may dial 1 (646) 960-0369. To participate via live or replay webcast, a link is available at www.ligand.com.

On November 8, 2023 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), reported financial results for the three months ended September 30th, 2023 and provided an update on key corporate milestones (Press release, Lexicon Pharmaceuticals, NOV 8, 2023, View Source [SID1234637264]).

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"We achieved important milestones this quarter in commercially launching INPEFA (sotagliflozin) in the U.S. for the treatment of heart failure while simultaneously advancing LX9211 into late-stage development for diabetic peripheral neuropathic pain," said Lonnel Coats, Lexicon’s chief executive officer. "We saw increasing prescription demand and clinical utilization for INPEFA throughout the third quarter notwithstanding limited access, and are now beginning to see important formulary wins, with some of the more notable becoming effective on November 1st. With additional formulary wins expected, coupled with increasing uptake from a broadening base of priority physicians, we are confident the momentum behind INPEFA will continue to accelerate as we enter 2024."

"We remain focused on showcasing the growing breadth of INPEFA’s clinical evidence and strengthening value proposition at major medical meetings, further demonstrating its differentiated profile for heart failure patients."

"We are also continuing to present data regarding LX9211, for which we have commenced late-stage development in diabetic peripheral neuropathic pain, with patient enrollment expected to begin this quarter in a Phase 2b clinical trial. We remain excited about the clinical profile of LX9211 and believe it could become the first new, non-opioid drug for neuropathic pain in over two decades – an area where new treatment options are urgently needed. Our continued progress with the launch of INPEFA coupled with the advancement of LX9211 into late-stage development are both major milestones in Lexicon’s path to fulfilling its mission of pioneering medicines that transform patients’ lives."

Third Quarter Highlights

INPEFA (sotagliflozin)

Launch Progress

•On June 26, Lexicon commenced the U.S. commercial launch of INPEFA (sotagliflozin) for the treatment of heart failure, with product made available in pharmacies and sales representatives deployed across the U.S.

•On October 9, Lexicon announced that Express Scripts, the pharmacy benefits management (PBM) business of The Cigna Group’s Evernorth, determined that it would place INPEFA on its Premier Access

and Premier Performance national formularies for Medicare patients on November 1, 2023. On November 1, Lexicon announced that Express Scripts had also determined that it would place INPEFA on its Basic and High Performance formularies for commercially insured patients on that date. These placements provide access to INPEFA for Express Scripts Medicare and commercial patients in plans that utilize these formularies and who meet the parameters of INPEFA’s FDA-approved indication.

Publications and Data

•On August 8, Lexicon announced that a post hoc analysis of data from the SOLOIST-WHF Phase 3 outcomes study of sotagliflozin was published by the Journal of the American College of Cardiology (JACC): Heart Failure. The main endpoint of the analysis was first occurrence of cardiovascular death or heart failure-related event (hospitalizations or urgent care visits for heart failure) within 30 and 90 days after hospital discharge (not randomization) in the subgroup of patients who began study treatment on or before discharge. Treatment with sotagliflozin resulted in significant relative risk reductions of approximately 50% for readmission for non-fatal heart failure events and for the composite of cardiovascular death and readmission for heart failure at 30- or 90-days following hospital discharge versus placebo.

•On August 26, two analyses of clinical study results relating to sotagliflozin and Lexicon-supported data were presented during the European Society of Cardiology (ESC) Congress 2023 in Amsterdam, the Netherlands. Bertram Pitt, M.D., FACC, professor of medicine emeritus at the University of Michigan, School of Medicine, Ann Arbor, MI conducted an oral presentation entitled Mediators of the benefit of sotagliflozin in patients with worsening heart failure in SOLOIST-WHF. In addition, Marc Bonaca, M.D., M.P.H., University of Colorado Anschutz Medical Campus, Aurora, CO presented a moderated ePoster entitled Temporal shift in heart failure diagnoses among hospitalized patients within a large US integrated health system.

•On October 4, Craig Granowitz, M.D., Ph.D., Lexicon’s senior vice president and chief medical officer, conducted an oral presentation entitled Temporal shift in heart failure medications prescribed to hospitalised patients with and without diabetes in a large US integrated health system at the 59th Annual Meeting of European Association for the Study of Diabetes (EASD) in Hamburg, Germany and online.

•On October 8, Michael J. Davies, Ph.D., Lexicon’s executive director of clinical development, presented a new analysis of clinical trial data for sotagliflozin entitled Sotagliflozin Reduces the Risk of Cardiovascular Events In Patients With Left Ventricular Hypertrophy Without Hypertension: A Post Hoc Analysis From SCORED was presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting in Cleveland, Ohio.

LX9211

•In June, Lexicon announced the planned advancement of AAK1-inhibitor LX9211 into late-stage development in diabetic peripheral neuropathic pain. During Q3, our teams have continued the preparations for the Phase 2b dose optimization study, with the first patient expected to be enrolled in early December 2023.

•On September 7, Lexicon announced a series of presentations relating to LX9211 occurring throughout the third and fourth quarters. Data were or will be shared in four oral presentations and one poster presentation at various global congresses, including NeuPSIG 2023 International Congress on Neuropathic Pain, held September 7-9 in Lisbon, Portugal; 33rd Annual Meeting of the Diabetic Neuropathy Study Group (NeuroDiab), held September 28-October 1 in Thessaloniki, Greece; 59th Annual Meeting of the European Association for the Study of Diabetes (EASD), held October 2-6 in Hamburg, Germany and online; 17th Annual Pain Therapeutics Summit (Arrowhead

Conference), held October 19-20 in San Diego, California; and 2nd World Brain Disorders and Neuroscience Summit (BDNS), being held November 9-11 in Singapore and online.

Third Quarter 2023 Financial Highlights

Revenues: Revenues for the third quarter of 2023 were $0.2 million, primarily from the recent commercial launch of INPEFA. INPEFA net product revenues from launch (late June) through September 30, 2023 total $0.4 million.

Research and Development (R&D) Expenses: Research and development expenses for the third quarter of 2023 increased to $17.6 million from $10.6 million for the corresponding period in 2022, primarily due to higher manufacturing costs and higher external research and development expenses related to the LX9211 program, partially offset by lower professional and consulting fees.

Selling, General and Administrative (SG&A) Expenses: Selling, general and administrative expenses for the third quarter of 2023 increased to $32.2 million from $12.6 million for the corresponding period in 2022, primarily due to increases in salaries and benefits, professional and consulting costs and marketing costs relating to preparations for the commercial launch of INPEFA.

Net Loss: Net loss for the third quarter of 2023 was $50.5 million, or $0.21 per share, as compared to a net loss of $23.4 million, or $0.13 per share, in the corresponding period in 2022. For the third quarters of 2023 and 2022, net loss included non-cash, stock-based compensation expense of $3.9 million and $2.6 million, respectively.

Cash and Investments: As of September 30, 2023, Lexicon had $218.4 million in cash and investments, as compared to $138.4 million as of December 31, 2022.

Conference Call and Webcast Information

Lexicon management will hold a live conference call and webcast today at 8:00 am ET / 7:00 am CT to review its financial and operating results and to provide a general business update. The dial-in number for the conference call is 888-317-6003 and the conference ID for all callers is 2598226. The live webcast and replay may be accessed by visiting Lexicon’s website at www.lexpharma.com/events. An archived version of the webcast will be available on the website for 14 days.

On November 8, 2023 Lantern Pharma Inc. (NASDAQ: LTRN), an artificial intelligence ("AI") company developing targeted and transformative cancer therapies using its proprietary RADR AI and machine learning ("ML") platform with multiple clinical-stage drug programs, reported operational highlights and financial results for the third quarter ended September 30, 2023 (Press release, Lantern Pharma, NOV 8, 2023, View Source [SID1234637263]).

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"Lantern had a very productive and efficient third quarter where the team made excellent and continued progress across our lead clinical programs, launched a new program into the clinic, and accelerated our efforts to ensure that our AI platform for cancer drug development, RADR, maintains its industry-leading position. We now have three active clinical programs that we are confident will make significant strides in Q4 and throughout 2024 – with multiple readouts expected during 2024. In addition, we continued to maintain a financially disciplined operation that will allow us to achieve milestones in both our drug programs and our AI platform over the next several years. Our RADR AI platform is revolutionizing the way we understand and predict drug-cancer interactions, enabling us to advance our newly developed drug programs from initial AI insights to first-in-human clinical trials in 2 to 3 years and at a cost of roughly $1 to 2.5 million per program – a milestone unheard of in the realm of oncology drug discovery," said Panna Sharma, CEO of Lantern Pharma.

Sharma continued, "This past quarter we launched another first-in-human, Phase 1 program, with LP-284, a synthetically lethal small-molecule, in refractory NHL where there is significant patient need for improved therapies. Therapies that can work with proven monotherapy efficacy and in combination with existing standard-of-care agents are critically needed in cancers where relapse from existing treatments can be a dire consequence. Computational and AI-driven approaches are increasing their ability to predict meaningful and clinically relevant combination regimens for cancer, and our team continues to increase the value of our platform in this regard while helping to also de-risk and sharpen the focus of our existing clinical drug candidates. Our leadership in the innovative use of AI and machine learning to transform costs and timelines in the development of precision oncology therapies should yield significant returns for investors and patients as our industry matures and adopts an AI-centric approach to drug development."

Highlights of AI-Powered Pipeline:

● LP-284 – Launched the first-in-human Phase 1 clinical trial with LP-284 targeting recurrent non-Hodgkin’s lymphomas (NHL). LP-284 has shown nanomolar potency across multiple in vitro and in vivo studies, including mantle cell lymphoma (MCL), double hit lymphoma (DHL), and other advanced NHL cancer subtypes with DNA damage response deficiencies, notably those with compromised functioning of the ataxia-telangiectasia mutated (ATM) gene due to mutations or deletions.

In xenograft PDX models of high-grade B cell lymphomas (HGBL), LP-284 showed synergistic and significantly enhanced anti-cancer activity when used in combination with rituximab. In in-vivo PDX models, the combined synergy of rituximab with LP-284 was 63% more effective in destroying HGBL tumors—93% tumor growth inhibition with both rituximab and LP-284 versus 57% tumor growth inhibition with rituximab alone. Rituximab is a standard-of-care approved therapy used in a wide range of B-cell cancers and non-Hodgkin’s lymphomas. Lantern plans to release additional details and data on this set of results with LP-284 in this setting in the coming month.

Nearly all MCL, DHL, and HGBL patients relapse from the current standard-of-care agents and there is an urgent and unmet need for novel improved therapeutic options for these patients. In the US and Europe, MCL, DHL, and HGBLs are diagnosed in 16,000-20,000 patients each year and have an estimated annual market potential of over USD 3+ billion.

● LP-184 – Dosed the first patient in Phase 1A clinical trial – a first-in-human Phase 1 basket trial across multiple solid tumor indications that are advanced and refractory to existing standard-of-care therapies. The trial is anticipated to enroll patients that have relapsed/refractory advanced solid tumors, such as pancreatic cancer, glioblastoma (GBM), brain metastases (brain mets.), lung, triple-negative breast cancer, and multiple other solid tumor types with DNA damage response deficiencies. Lantern expects to continue Phase 1 enrollment throughout the remainder of 2023 and the first half of 2024 across a growing number of US clinical trial sites, including Fox Chase Cancer Center and Johns Hopkins Medicine.

The dosage and safety data obtained in the Phase 1 trial will be used to advance the central nervous system (CNS) indications for a future Phase 2 trial to be sponsored by Lantern’s wholly-owned subsidiary, Starlight Therapeutics. Globally, the aggregate annual market potential of LP-184’s target indications is estimated to be approximately $11+ billion, consisting of $5+ billion for CNS cancers and $6+ billion for solid tumors.

● LP-300 – Activated additional sites in the US which will increase the potential for dosing additional patients in the Phase 2 Harmonic trial during 2023. The Harmonic trial is assessing the effect of LP-300 in combination with standard-of-care chemotherapy in never-smoker patients with relapsed non-small cell lung cancer (NSCLC). In addition to the dosed patients, more than two dozen potential patients have been pre-screened and are being monitored for possible enrollment during Q4 across 10 clinical sites in the US. The Company is also actively advancing the Harmonic clinical trial to countries in Asia that are known to have a significantly higher incidence of never-smokers with NSCLC – Taiwan, Japan, and South Korea. In these countries, the incidence of never-smokers with NSCLC is double or higher than that of patients in the US.

Dr. Joseph Treat MD of Fox Chase Cancer Center has been appointed the lead principal investigator of the Harmonic study. Dr. Treat is a leading expert in lung malignancies, including NSCLC in never smokers, and has dedicated his career, since 1991, to serving patients with lung cancer.

Globally, never-smokers with NSCLC are a growing population of patients and do not respond well to PD-1/PD-L1-based therapies, leaving them with reduced treatment options. In the US, there are approximately 20,000-40,000 never-smokers with NSCLC diagnosed annually, representing an estimated US annual market potential of $1.5 billion and a global estimated annual market potential of over $2.6 billion. Additional information on the Harmonic trial can be found at the Harmonic website and clinicaltrials.gov.

RADR Platform Growth and Development:

● RADR continues to advance in size, scope, and capabilities and is also progressing towards becoming a standard for AI-driven drug development in oncology – for both early-stage development and later-stage patient biomarker and combination therapy identification. RADR has now surpassed 36 billion oncology-focused datapoints and is projected to reach over 50 billion datapoints by the end of 2023. The scope of RADR’s data has broadened with a strategic focus on additional classes of compounds, including antibodies, checkpoint inhibitors, and DNA-damaging agents. Additionally, data from clinical studies such as those being obtained from liquid biopsy, and data from preclinical combination studies that aim to define drug interaction and optimal dosage are being incorporated into the datapoints and data sets powering RADR.

● These datapoints, the associated advancements in automation, along with algorithms and code comprise a functional module and have advanced RADR’s drug development capabilities. Key modules that are being advanced are those for 1) predicting patient responses and identifying optimal combination regimens for immuno-oncology (IO) drugs such as immune checkpoint inhibitors, 2) predicting the BBB permeability, with 89% to 92% accuracy, of any compound at a scale and speed that allows the analysis of tens of thousands of compounds a day, and 3) accelerating the design and development of drug-conjugate templates for next-generation antibody-drug conjugates (ADCs) that have increased potential for improved safety and efficacy. These 3 additional modules exemplify the type of rapid and meaningful progress the RADR platform is expected to make over the next several quarters as it aims to improve the speed and reduce the costs and risks associated with creating cancer medicines.

Starlight Therapeutics:

● In Q1 2023, Lantern formed a wholly-owned subsidiary, Starlight Therapeutics Inc. ("Starlight"), for the clinical development of drug candidate LP-184’s central nervous system (CNS) and brain cancer indications – including GBM, brain mets., and several rare pediatric CNS cancers. Starlight will refer to the molecule LP-184, as it is developed in CNS indications, as "STAR-001".

● Lantern expects to recruit additional management focused on Starlight operations during Q4, 2023. Lantern has also begun discussions with leading clinicians and key opinion leaders at CNS-focused cancer centers to serve as clinical trial sites for planned upcoming clinical trials in adult and pediatric CNS cancers.

Additional Operational Highlights:

● During the 3rd quarter of 2023, Lantern filed 4 new patent applications for LP-184 and LP-284 relating to breast, liver, and blood cancers and an additional application directed to lyophilized formulations of these molecules.

● New data and scientific findings along with AI platform updates to be presented at several upcoming conferences:

SNO (Society for Neuro-Oncology) 28th Annual Meeting and Education Day in Vancouver, Canada

➢ Date: November 17, 2023, 10:55a-11:05a PST
➢ Presentation Title: LP-184, an MGMT-agnostic small molecule, has potent synergy with Spironolactone to effectively inhibit orthotopic GBM xenograft tumors
➢ Presenter: Dr. John Laterra (clinician-scientist collaborator from Johns Hopkins Medicine & Kennedy Krieger Institute)

Bengaluru Tech Summit 23 in Bengaluru, India

➢ Date: December 1, 2023, 12p-12:50p IST
➢ Presentation Topic: Biotech Future Forward – Pharma 4.0 & How AI is changing the playing field in Biopharma
➢ Presenter: Panna Sharma (President & CEO)

5th Annual CNS Drug Delivery Summit in Boston, MA

➢ Date: December 5, 2023 at 1:30p EST
➢ Presentation Topic: Leveraging AI & Machine Learning to Accelerate the Development of CNS & Brain Cancer Molecules
➢ Presenter: Kishor Bhatia, Ph.D. (CSO)

Third Quarter 2023 Financial Overview:

● Balance Sheet: Cash, cash equivalents, and marketable securities were approximately $44.9 million as of September 30, 2023, compared to approximately $55.2 million as of December 31, 2022. The quarterly cash burn rate continues to reflect our capital-efficient, collaborator-centered business model.

● R&D Expenses: Research and development expenses were approximately $2.2 million for the quarter ended September 30, 2023, compared to approximately $0.7 million for the quarter ended September 30, 2022. R&D expenses for the 3rd quarter of 2022 were significantly reduced, by $0.9 million, due to a one-time payment received from a service provider to resolve a difference of views regarding the service agreement.

● G&A Expenses: General and administrative expenses were approximately $1.3 million for the quarter ended September 30, 2023, compared to approximately $1.4 million for the quarter ended September 30, 2022.

● Net Loss: Net loss was approximately $3.2 million (or $0.29 per share) for the quarter ended September 30, 2023, compared to a net loss of approximately $2.3 million (or $0.21 per share) for the quarter ended September 30, 2022.

Earnings Call and Webinar Details:

Lantern will host its third quarter 2023 earnings call and webinar today, November 8, 2023, at 4:30 p.m. ET.

● View Source
● Related presentation materials will be accessible at: View Source
● A replay of the third quarter earnings call and webinar will be available at View Source

On November 8, 2023 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that the Company’s senior management team will participate in the following investor conferences in November (Press release, Karyopharm, NOV 8, 2023, View Source [SID1234637262]):

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Jefferies 2023 London Healthcare Conference
Format: Fireside chat
Date: Wednesday, November 15, 2023
Time: 8:00 a.m. ET / 1:00 p.m. GMT

Piper Sandler 35th Annual Healthcare Conference
Format: Fireside chat
Date: Wednesday, November 29, 2023
Time: 4:00 p.m. ET

A live webcast of these events, along with accompanying slides, can be accessed under "Events & Presentations" in the Investor section of the Company’s website, View Source, and will be available for replay for 90 days following each presentation.