On October 26, 2023 Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-driven biopharmaceutical company, reported its sales performance for the year to date and the third quarter of 2023 (Press release, Ipsen, OCT 26, 2023, View Source [SID1234636374]).

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YTD
2023 YTD
2022 % change Q3
2023 Q3
2022 % change
€m €m Actual CER1 €m €m Actual CER1
Oncology 1,744.1 1,767.2 -1.3% 0.8% 574.5 603.1 -4.7% 0.8%
Neuroscience 489.0 407.7 19.9% 24.5% 164.8 160.7 2.5% 13.7%
Rare Disease 76.0 33.6 n/a n/a 33.2 11.0 n/a n/a
Total Sales 2,309.1 2,208.5 4.6% 7.1% 772.4 774.8 -0.3% 6.5%

Sales and pipeline highlights

Total-sales growth in the year to date of 7.1% at CER1, or 4.6% as reported, driven by the performance of the growth platforms2, up by 16.1%1, with Dysport (abobotulinumtoxinA) up by 24.7%1 and Cabometyx (cabozantinib) up by 24.4%1, respectively. The performance included contributions from new medicines Bylvay (odevixibat), Tazverik (tazemetostat) and Sohonos (palovarotene)
Further pipeline progress, including the regulatory approval and launch in the U.S. of Sohonos in Rare Disease and initial results from the CONTACT-02 Phase III trial of Cabometyx plus atezolizumab in Oncology
David Loew, Chief Executive Officer, commented:
"Ipsen’s strategic success has been reflected in further sales and pipeline progress so far this year. Our portfolio has performed well across the three therapy areas, driven by strengthened commercial execution and the results of our external-innovation strategy. Based on the solid sales momentum, today we are confirming our guidance for the full year.

Further good news from the pipeline, including the regulatory approval of Sohonos in the U.S., continue to provide additional options for patients with real unmet medical needs. In the final quarter of the year, we look forward to regulatory steps for elafibranor in primary biliary cholangitis, as well as sharing further details on sustainable growth opportunities across our portfolio and pipeline at our forthcoming capital-markets day."

Full-year 2023 guidance
Ipsen has confirmed its financial guidance for FY 2023:

Total-sales growth greater than 6.0%, at constant exchange rates. Based on the average level of exchange rates in September 2023, an adverse impact on total sales of around 3.5% from currencies is expected
Core operating margin greater than 30% of total sales
Pipeline development

In August 2023, it was announced that the U.S. Food and Drug Administration (FDA) had approved Sohonos, the first and only treatment for people with fibrodysplasia ossificans progressiva.

It was also announced that the global CONTACT-02 pivotal Phase III trial of Cabometyx plus atezolizumab in metastatic castration-resistant prostate cancer met one of two primary endpoints, demonstrating a statistically significant improvement in progression-free survival at the primary analysis.

In October 2023, the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products confirmed its negative opinion recommending not to maintain the orphan designation for Bylvay in Alagille syndrome (ALGS). This was despite a positive opinion from the Committee for Medicinal Products for Human Use in July 2023. To maintain Bylvay’s orphan designation in the approved treatment of progressive familial intrahepatic cholestasis, Ipsen is planning to resubmit to the EMA under a new brand name for the treatment of ALGS by the end of 2023.

Galderma partnership

In September 2023, the Arbitral Tribunal of the International Chamber of Commerce issued a final decision following a difference of opinion on the regulatory-submission strategy for the liquid botulinum toxin type A, QM1114. In October 2023, Ipsen announced that its partner, Galderma, had received a Complete Response Letter from the U.S. FDA related to its Biologics License Application for QM1114.

A second arbitration proceeding, related to the territorial scope of the Dysport/Azzalure aesthetics’ partnership, is anticipated to conclude next year.

AASLD call
To accompany the presentation of the ELATIVE Phase III trial results at the American Association for the Study of Liver Diseases (AASLD) 2023 Annual World Congress, Ipsen plans to host a conference call for analysts and investors on 14 November 2023, at 4.30pm CET. Participants can access the call and its details by registering here; webcast details can be found here. A recording will be available on ipsen.com.

Capital-markets day
The Company is planning to host a capital-markets event, starting at 12.30pm GMT on 7 December 2023 in London. The event will be webcast live and details will be available on ipsen.com in due course. In-person attendance will be by invitation only.

Calendar
Ipsen intends to publish its full-year and fourth-quarter results on 8 February 2024.

Conference call: YTD 2023
A conference call and webcast for investors and analysts will begin today at 2pm CET. Participants can access the call and its details by registering here; webcast details can be found here. A recording will be available on ipsen.com.

Notes

All financial figures are in € millions (€m). The performance shown in this announcement covers the nine-month period to 30 September 2023 (YTD 2023) and the three-month period to 30 September 2023 (Q3 2023), compared to nine-month period to 30 September 2022 (YTD 2022) and the three-month period to 30 September 2022 (Q3 2022), respectively, unless stated otherwise. Commentary is based on the performance in YTD 2023, unless stated otherwise.

On October 26, 2023 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, reported financial results for the third quarter ended September 30, 2023 and provided a business update (Press release, Insmed, OCT 26, 2023, View Source [SID1234636373]).

"Insmed made tremendous progress in the third quarter of 2023, punctuated by positive topline results from the ARISE study, encouraging blinded data from the Phase 2 trials of TPIP in PH-ILD and PAH, and the strongest quarter of ARIKAYCE revenues to date," said Will Lewis, Chair and Chief Executive Officer of Insmed. "Each of our mid- to late-stage pipeline assets continue to demonstrate strong performance in the clinic and Insmed’s enormous potential. We are extremely excited about the robust series of clinical, regulatory, and commercial catalysts that lie ahead as we strive to bring forward first-in-class or best-in-class medicines to patients in need."

Recent Pillar Highlights

Pillar 1: ARIKAYCE

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ARIKAYCE global revenue grew 17% in the third quarter of 2023 compared with the third quarter of 2022, reflecting the strongest quarter of sales since commercial launch and continued sequential quarterly revenue growth in the U.S. and Japan.

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Insmed continues to execute on its post-marketing, confirmatory trial program for ARIKAYCE, including the recently completed ARISE study and the ongoing ENCORE study in patients with newly diagnosed or recurrent Mycobacterium avium complex (MAC) lung infection who have not started antibiotics.

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The Company announced positive topline efficacy and safety data from the ARISE trial in September of 2023. The study met its primary objective of validating a patient-reported outcome (PRO) instrument in patients with MAC lung disease. In addition, ARIKAYCE-treated patients had nominally statistically significantly higher culture conversion rates at Month 7 versus the comparator arm. Based on the results of ARISE, Insmed plans to explore with global regulators accelerating the filing for approval of ARIKAYCE in newly infected patients with MAC lung disease.

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The Company remains on track to enroll 250 patients in the registrational ENCORE study by the end of 2023. Enrollment is expected to continue into 2024, pending additional discussions with the U.S. Food and Drug Administration (FDA). Insmed anticipates reporting topline data from the ENCORE study in 2025.

1
Pillar 2: Brensocatib

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Insmed continues to expect topline data from the ASPEN study, a global Phase 3 trial designed to assess the efficacy, safety, and tolerability of brensocatib in patients with non-cystic fibrosis bronchiectasis, in the second quarter of 2024.

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The Company has opened several study sites in the Phase 2b BiRCh trial of brensocatib in patients with chronic rhinosinusitis without nasal polyps (CRSsNP) and is nearing randomization of the study’s first patients.

Pillar 3: TPIP

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Insmed continues to enroll patients in a Phase 2 study of treprostinil palmitil inhalation powder (TPIP) in pulmonary hypertension associated with interstitial lung disease (PH-ILD) and a Phase 2 study in pulmonary arterial hypertension (PAH).

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In the ongoing studies, 80% of the first 10 PH-ILD patients and 83% of the first 24 PAH patients who reached the Week 5 visit, which is the last possible point at which the dose can be increased in the trial, were able to titrate up to the maximum dose of 640 micrograms or matching placebo.

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Adverse events observed to date in these trials have been consistent with the events commonly seen in patients with PAH or PH-ILD and with the known effects of inhaled prostacyclin therapies. In addition, adverse events related to cough have been mostly mild and there have been no instances of throat irritation or pain to date. A meeting of the Data Safety and Monitoring Board was held in October of 2023, where it was recommended that both studies continue as planned.

•
Based on a review of 22 patients who had completed 16 weeks of treatment in the ongoing PAH study, including patients receiving placebo, the average reduction in PVR from baseline was 21.5%. The average PVR reduction among the 64% of patients who had an improvement in PVR was 47%.

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Insmed remains on track to report topline results from the Phase 2 study of TPIP in PH-ILD in the first half of 2024.

Pillar 4: Early-Stage Research

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Insmed’s early-stage research efforts include more than 30 identified pre-clinical programs in development, all of which have the potential to become first-in-class or best-in-class therapies.

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The Company is continuing to advance its gene therapy program in Duchenne muscular dystrophy (DMD), including additional pre-clinical studies to further characterize its novel intrathecal route of gene therapy administration. Pending completion of this work, the Company expects to initiate clinical trials in patients. In parallel, the Company continues to advance its mid-length dystrophin DMD gene therapy program using its proprietary RNA-end-joining technology.

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The Company continues to anticipate the totality of its early-stage research programs will comprise less than 20% of overall spend.

2
Third-Quarter 2023 Financial Results

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Total revenue for the third quarter ended September 30, 2023 was $79.1 million, reflecting the Company’s strongest quarter of sales to date and 17% growth compared to total revenue of $67.7 million for the third quarter of 2022.

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Total revenue for the third quarter of 2023 was comprised of ARIKAYCE net sales of $59.2 million in the U.S., $16.0 million in Japan, and $3.8 million in Europe and rest of world. Third-quarter sales reflect year-over-year growth of 20% and 11% in the U.S. and Japan, respectively, as well as the highest quarter of sales to date in these two regions.

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Cost of product revenues (excluding amortization of intangibles) was $16.7 million for the third quarter of 2023, compared to $13.5 million for the third quarter of 2022, reflecting increased sales volumes of ARIKAYCE.

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Research and development (R&D) expenses were $109.1 million for the third quarter of 2023, compared to $99.9 million for the third quarter of 2022 and $197.0 million for the second quarter of 2023. R&D expenses in third-quarter 2023 reflected continued investment in the Company’s early and mid- to late-stage pipelines.

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Selling, general and administrative (SG&A) expenses for the third quarter of 2023 were $90.6 million, compared to $75.6 million for the third quarter of 2022 and $84.4 million for the second quarter of 2023. The year-over-year increase in SG&A expenses resulted primarily from an increase in compensation and benefit-related expenses and stock-based compensation costs due to an increase in headcount, as well as increased fees and expenses driven by commercial readiness activities for brensocatib.

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Insmed reported a net loss of $158.9 million, or $1.11 per share, for the third quarter of 2023, compared to a net loss of $131.1 million, or $1.09 per share, for the third quarter of 2022, and a net loss of $244.8 million, or $1.78 per share, for the second quarter of 2023.

Balance Sheet, Financial Guidance, and Planned Investments

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As of September 30, 2023, Insmed had cash, cash equivalents, and marketable securities totaling $786 million, down from $918 million as of June 30, 2023, reflecting the ongoing support of the ARIKAYCE franchise, commercial readiness activities for brensocatib, and clinical operations for its mid- to late-stage pipeline programs.

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Insmed is reiterating its sales guidance for full-year 2023 global revenues for ARIKAYCE in the range of $295 million to $305 million.

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Insmed continues to anticipate that over 80% of total expenditures will be on its mid- to late-stage and commercial programs (ARIKAYCE, brensocatib, and TPIP), and that less than 20% of overall spend will be on its early-stage research programs, reflecting the Company’s historical approach to spending.

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The Company plans to continue to invest in the following key activities during the remainder of 2023:

(i)
commercialization and expansion of ARIKAYCE globally;

(ii)
advancement of brensocatib, including the Phase 3 ASPEN study in patients with bronchiectasis, which is expected to be completed in the second quarter of 2024, and commercial launch readiness activities, as well as the Phase 2 trial in patients with CRSsNP, which is nearing randomization;

(iii)
advancement of the clinical trial program for ARIKAYCE (ARISE and ENCORE), which is intended to satisfy the post-marketing requirement for full approval of its current indication and potentially support label expansion to include all patients with a MAC lung infection;

(iv)
advancement of its Phase 2 clinical development programs for TPIP; and

(v)
development of its early-stage research platforms.

3
Conference Call

Insmed will host a conference call beginning today at 8:30 AM Eastern Time. Shareholders and other interested parties may participate in the conference call by dialing (888) 210-2654 (U.S. and international) and referencing access code 7862189. The call will also be webcast live on the Company’s website at www.insmed.com.

A replay of the conference call will be accessible approximately 1 hour after its completion through November 25, 2023, by dialing (800) 770-2030 (U.S. and international) and referencing access code 7862189. A webcast of the call will also be archived for 90 days under the Investor Relations section of the Company’s website at www.insmed.com.

On October 26, 2023 ImmunityBio, Inc. (NASDAQ: IBRX), a clinical-stage immunotherapy company, reported that the U.S. Food and Drug Administration (FDA) has accepted for review ImmunityBio’s resubmission of its Biologics License Application (BLA) for N-803, a first-in-class IL-15 superagonist, plus Bacillus Calmette-Guérin (BCG) for the treatment of BCG-unresponsive non-muscle-invasive bladder cancer carcinoma in situ (CIS) with or without Ta or T1 disease, and considered it as a complete response to the FDA’s May 9, 2023 complete response letter (Press release, ImmunityBio, OCT 26, 2023, View Source [SID1234636372]). The FDA has set a user fee goal date (PDUFA date) of April 23, 2024.

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"We are pleased that the FDA has accepted ImmunityBio’s resubmission of the BLA as a complete response, following our productive interactions leading up to the resubmission. We look forward to working closely with the Agency to finalize the review and to bringing this important immune-enhancing therapeutic to patients suffering from bladder cancer," said Patrick Soon-Shiong, M.D., Executive Chairman and Global Chief Scientific and Medical Officer at ImmunityBio.

ImmunityBio’s IL-15 superagonist N-803 (also called Anktiva and nogapendekin alfa inbakicept)

The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of the natural killer (NK) and T cells. N-803 is a novel investigational IL-15 superagonist complex consisting of an IL-15 mutant (IL-15N72D) bound to an IL-15 receptor α/IgG1 Fc fusion protein. Its proposed mechanism of action is direct specific stimulation of CD8+ T cells and NK cells through beta gamma T-cell receptor binding with generation of memory T-cells while avoiding T-reg stimulation. N-803 is designed to have improved pharmacokinetic properties, longer persistence in lymphoid tissues and enhanced anti-tumor activity compared to native, non-complexed IL-15 in vivo.

N-803 is currently being evaluated in adult patients in two clinical NMIBC trials. QUILT-2.005 is investigating use of N-803 in combination with BCG for patients with BCG-naïve NMIBC; QUILT-3.032 is studying N-803 in combination with BCG in patients with BCG-unresponsive NMIBC CIS and Papillary Disease.

N-803 is investigational. Safety and efficacy have not been established by any Health Authority or Agency, including the FDA.

On October 26, 2023 IDP Pharma, a clinical-stage biotechnology company pioneering the development of drugs that directly engage and degrade intrinsically disordered proteins (IDPs), reported that the first patient has been successfully dosed in its IDP-121-001 CASSANDRA trial, a multi-center, open-label Phase I/II study for the treatment of cMyc driven hematological malignancies (Press release, IDP Pharma, OCT 26, 2023, View Source;utm_medium=rss&utm_campaign=first-patient-dosed-in-a-phase-1-2-clinical-trial-of-idp-121-a-direct-cmyc-protein-inhibitor-and-degrader [SID1234636371]).

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The cMyc oncogene is activated in most cancers by genetic, epigenetic or post- translational mechanisms, and cMyc protein function is altered in approximately 70% of all tumours. The intrinsic connection between cMyc protein and tumorigenesis highlights the potential therapeutic significance of its inhibition. IDP-121 is designed to impair cMyc protein function and selectively degrade the target.

"IDP-121 has demonstrated the unique ability to directly engage cMyc protein, triggering antitumor response in several animal models. We are particularly interested in the exploration in hematological diseases, including multiple myeloma, that we know are heavily cMyc dependent and give us the opportunity to quickly measure the on-target effect. Ultimately, we hope to see biological and clinical response that will confirm the broad potential of this therapeutic approach across solid and liquid tumors" said Dr. Laura Nevola, IDP’s Chief Scientific Officer.

According to Valentin Cabañas, the PI of the Hospital Clínico Virgen de la Arrixaca (Murcia, Spain), where the first patient was dosed, the use of IDP-121 for the treatment of multiple myeloma "could potentially represent a major shift in the disease management due to the exploitation of a mechanism of action previously uncharted for targeting myeloma cells". The clinical trial will also include patients of non-Hodgkin lymphoma and chronic lymphocytic leukemia, as this new drug "acts on the protein involved in the development of several hematological cancers beyond multiple myeloma".

"This is an extremely important next step in the understanding and relevance of cMyc and its role in cancer development and progression. The ability to directly target cMyc protein in the clinic is very exciting and the exploration of IDP-121 in this Phase I study could be a landmark in oncology drug development", said Professor Dean Felsher, Scientific Adviser to IDP Pharma and Professor in the Division of Oncology within the Departments of Medicine and Pathology at Stanford University.

"IDP-121 is IDP Pharma’s lead compound and the announcement today represents a major step towards the systematic and direct inhibition of disease drivers in cancer mediated through IDPs" said Santiago Esteban, Chief Executive Officer of IDP Pharma.

The study will assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary clinical activity of IDP-121. The principal investigator is Professor Enrique Ocio, Head of Hematology of the Hospital Universitario Marqués de Valdecilla, and the study includes other four medical facilities in Spain: Hospital Vall d’Hebron, Hospital Universitario 12 de Octubre, Hospital Universitario of Salamanca, and Hospital Universitario Virgen de la Arrixaca.

To learn more about the IDP-121 clinical trial, visit clinicaltrials.gov (Identifier: NCT05908409).

On October 26, 2023 Evaxion Biotech A/S (NASDAQ: EVAX) ("Evaxion" or the "Company"), a clinical-stage TechBio company specializing in developing AI-Immunology-powered vaccines, reported that it will be presenting clinical readouts on its two personalized cancer vaccine trials at SITC (Free SITC Whitepaper)’s 38th annual meeting, taking place in San Diego, California from November 1-5, 2023 (Press release, Evaxion Biotech, OCT 26, 2023, View Source [SID1234636370]).

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The results will be presented in two posters:

1. Title: "AI-designed personalized neoantigen vaccine, EVX-02, induces robust T-cell responses in melanoma patients"

Poster #: 623
Location: Exhibit Hall B – San Diego Convention Center
Time: Friday, November 3, 9 a.m. – 7 p.m. PDT

This poster will be presented by Evaxion’s senior project manager, Daniela Kleine-Kohlbrecher. The abstract was also selected by the SITC (Free SITC Whitepaper) Communications Committee to be presented at the SITC (Free SITC Whitepaper) 2023 Annual Meeting Press Conference, scheduled for Wednesday, November 1, from 12:00–1:30 p.m. PDT.

2. Title: "Effects of an AI-generated personalized neopeptide-based immunotherapy, EVX-01, in combination with pembrolizumab in patients with metastatic melanoma. A clinical trial update"

Poster #: 782-H
Location: Exhibit Hall B – San Diego Convention Center
Time: Saturday, November 4, 9 a.m. – 8:30 p.m. PDT

This poster will be presented by principal investigator Professor Adnan Khattak from the Hollywood Private Hospital in Nedlands, Australia.

Christian Kanstrup, Chief Executive Officer at Evaxion, commented, "We are thrilled about the continuous buildup of positive clinical evidence for the unique predictive capabilities of our AI-immunology platform, which we believe holds promise for truly groundbreaking cancer treatments."

Birgitte Rønø, Chief Scientific Officer at Evaxion, expressed her enthusiasm about the upcoming conference, stating, "We are looking forward to sharing these promising clinical trial results with the SITC (Free SITC Whitepaper) community, showcasing the advantages of our AI-Immunology platform to design personalized cancer vaccines. We believe this takes us one step closer to bringing novel treatments to the market and meeting the pressing global medical need for cancer patients."

About EVX-01 Phase 2 Clinical Trial
EVX-01 is Evaxion’s lead clinical asset and constitutes a peptide-based personalized cancer vaccine. The Phase 2 clinical study is a self-sponsored open-label, single-arm, multi-center trial carried out in collaboration with Merck Sharp & Dohme LLC, together with leading principal investigators and research centers from Italy and Australia, and aims at evaluating the efficacy and safety of EVX-01 vaccination in combination with anti-PD1 treatment (pembrolizumab) in treatment-naive patients with metastatic or unresectable malignant stage III or IV melanoma. More information can be accessed under clinical trial ID NCT05309421.

About EVX-02 Phase 1/2a Clinical Trial

The EVX-02 Phase 1/2a represents Evaxion’s first-in-human DNA-based personalized cancer vaccine study. This open-label, multi-center study aims at assessing the safety, tolerability, pharmacodynamic responses, and efficacy of EVX-02 neoantigen vaccine and anti-PD-1 treatment (nivolumab) in patients who have had a complete resection of a Stage IIIB/IIIC/IIID or Stage IV melanoma with risk of recurrence. More information can be accessed under clinical trial ID NCT04455503.