On November 4, 2022 Philogen reported that it will hold a virtual briefing on 1 December 2022 at 10:00 ET / 15:00 BST / 16:00 CEST following the Press Release of the Third Quarter Financial Results (9 November 2022) (Press release, Philogen, NOV 4, 2022, View Source [SID1234623108]).

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On November 4, 2022 Galapagos NV (Euronext & NASDAQ: GLPG) reported that held its R&D Day 2022, featuring presentations of key opinion leaders and company management on the strategic, scientific, and commercial progress at the company (Press release, Galapagos, NOV 4, 2022, View Source [SID1234623107]). The company also presented its financial results for the third quarter of 2022.

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Paul Stoffels3, CEO and Chairman of the Board of Directors of Galapagos, commented: "Today we presented our strategic vision for our future as a financially sustainable biopharma company. Building on our strong fundamentals, we are convinced that we are taking the right steps to deliver on our core mission: adding years of life and improving the quality of life of patients globally.

Focusing on our strategic therapeutic areas, we strive to push forward our deep, differentiated portfolio in immunology, build out our oncology franchise, and execute on business development opportunities with the aim to accelerate innovation and speed up time-to-patients, while creating long-term value for our stakeholders."

Strategic update

Building on our strong fundamentals, we aim to build a financially sustainable biopharma company, accelerating cutting-edge therapies to market to address unmet medical needs in our core key therapeutic areas of oncology and immunology.

Our renewed focus on two strategic therapeutic areas goes hand in hand with redirecting our resources. To further streamline our portfolio, we have decided to discontinue activities in fibrosis and kidney disease to allow for increased investments in our oncology franchise.
To shape our portfolio of innovative drugs with accelerated time-to-market, we intend to combine internal innovation with smart business development and apply deep clinical expertise to push programs through an optimal development process.

Based on these guiding principles, we presented our Vision 2028 portfolio outlook. Over the course of the next five years, we aim to build a portfolio comprising:

over 10 assets in lead optimization and five in preclinical development across different modalities (small molecules, cell therapy, biologics),
five pivotal-stage candidates forming a solid late-stage pipeline in immunology and cell therapy, and
a growing commercial presence with additional marketed indications for Jyseleca and one CAR-T therapy approved in multiple indications.

Financial vision

Aligned with our Vision 2028, we presented our financial outlook and capital allocation strategy at our R&D Day 2022. Discontinuation of our fibrosis and kidney efforts allows for reinvesting in oncology.

With Jyseleca potentially breaking even in 2024, and projected peak sales of €500 million in Europe and first oncology revenues potentially contributing to our topline later this decade, we are committed to building a sustainable financial future, with a significantly reduced cash burn by 2028.

Oncology

We also presented our Vision 2028 roadmap for oncology at our R&D Day 2022. Our core mission in the field is to broaden patient access and improve clinical outcomes by bringing best-in-class medicines to patients.

With the combined acquisitions of CellPoint and AboundBio announced earlier this year, we positioned ourselves in the cell therapy space, combining the potentially disruptive manufacturing and delivery model of CellPoint (Lonza’s Cocoon, a closed, automated manufacturing platform for cell therapy) with the ability to develop next-generation CAR-Ts, small molecules and biologics.

Short term, our aim is to validate the decentralized CAR-T delivery model with proven CAR-T therapies, and we announced the addition of a BCMA Phase 1/2 trial on the Cocoon platform. This complements the currently ongoing CD19 Phase1/2 programs in recurring/refractory Non-Hodgkin Lymphoma (rrNHL; ATALANTA study) and recurring/refractory Chronic Lymphocytic Leukemia (rrCLL; EUPLAGIA study).

In the coming years, we intend to build a pipeline of best-in-class cell therapies for hematologic malignancies and leverage our capabilities to rapidly address unmet needs in oncology.

Today, we showcased the robust process performance of the Cocoon system as well as first encouraging biomarker patient data of the CAR-T expansion profile with a 7 day-point-of-care vein-to-vein treatment. Topline Phase 1 results of both the rrNHL and rrCLL study are expected in the first half of 2023, followed by initial data of the BCMA study later in the year.

Next year, we also aim to broaden the studies to include U.S. patients, and we plan to submit Investigational New Drug (IND) applications for both the CD19 and BCMA CAR-T programs with the FDA.

Immunology

We have built up over 20 years of expertise in immunology, resulting in a deep and growing pipeline with multiple modes-of-action candidate medicines across all phases of development, from preclinical to Phase 4.

We are excited about our selective TYK2 inhibitor, ‘3667, for which we presented new data elucidating its potentially differentiating selectivity and potency profile. Ex vivo and in vivo data show that ‘3667 fully blocks the INFa pathway with once-daily dosing, while in our assays JAK2 and JAK1/3 dependent pathways are not affected. Further, no effects on hematological parameters, lipids and creatine phosphokinase (CPK) were observed, suggesting that ‘3667 does not show JAKi ‘fingerprints’ at therapeutic doses. Also based on the positive results in psoriasis patients, we presented our development plan for ‘3667, confirming the start of a Phase 2 study in dermatomyositis (GALARISSO) around year-end and the intention to start a Phase 2 study in systemic lupus erythematosus (SLE) in 2023.

We also presented an update on our SIKi portfolio, a potential novel mode-of-action in immunology. With targeted investments, we continue to make important progress in developing next-generation best-in-class candidates that reach optimal target coverage, teasing apart selectivity profiles that indicate the potential of SIK3i for rheumatological indications and SIK2/3i for inflammatory bowel disease. Our most advanced candidate, SIK3 inhibitor ‘4399, has shown strong preclinical evidence for potential in rheumatoid arthritis (RA) (EULAR 20224), with a promising pharmacological and safety profile. We are planning to start a proof-of-mechanism study in RA patients with ‘4399 mid 2023.

For filgotinib, our selective JAK1 inhibitor, we are completing a Phase 3 program in Crohn’s disease (CD), with results expected in the first half of 2023. Given the sales momentum and supportive long-term safety and efficacy data generated for filgotinib, we are currently exploring additional indications for filgotinib, and plan to start a Phase 3 study in axial spondyloarthritis (AxSpA) in 2023.

Commercial progress

Since becoming European marketing authorization holder (MAH) of Jyseleca, we successfully set up our own commercial capabilities and are currently operational throughout Europe in the current approved indications RA and ulcerative colitis (UC).
The financial results for the third quarter of 2022 show continued strong sales momentum for Jyseleca, and we further raise our net sales guidance 2022 from €75-€85 million at H1 2022 to €80-€90 million.

In the future, we aim to leverage our European commercial infrastructure beyond Jyseleca, in line with our Vision 2028, to have at least one CAR-T cell therapy on the market within five years.

Jyseleca is a trademark of Galapagos NV and Gilead Sciences, Inc. or its related companies. Except for filgotinib’s approval as Jyseleca for the treatment of moderately to severely RA and UC by the relevant regulatory authorities in the European Union, Great Britain, and Japan, our drug candidates are investigational; their efficacy and safety have not been fully evaluated by any regulatory authority.

On November 4, 2022 Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of refractive and retinal eye disorders, announces financial results for the third quarter ended September 30, 2022 and provides a corporate update (Press release, Ocuphire Pharma, NOV 4, 2022, View Source [SID1234623106]).

"During the third quarter, Ocuphire continued to execute elements of our strategic plan to bring innovative treatments to patients with highly prevalent refractive and diabetic retinal diseases," said Mina Sooch, MBA, founder and CEO of Ocuphire Pharma. "With enrollment and 24-week treatment completed in over 100 patients in our ZETA-1 Phase 2b trial of APX3330, we look forward to sharing topline results in early 2023, bringing us closer to delivering a potential oral option for diabetic retinopathy patients. We are on track for NDA submission for Nyxol for reversal of mydriasis in the fourth quarter 2022. We have strong momentum and are poised to deliver on multiple catalysts going forward that we believe will create significant value for our company and shareholders."

Key Anticipated Future Milestones

Reversal of Mydriasis (RM): Plan to submit New Drug Application (NDA) with the FDA for Nyxol in RM indication in Q4 2022, with potential approval and commercial launch as first dilation reversal drop in 2023.

Diabetic Retinopathy (DR) and Diabetic Macular Edema (DME): Plan to report top-line results from the ZETA-1 Phase 2b trial of APX3330 in early 2023. APX3330 is a novel oral therapy with a dual mechanism of action in validated pathways, decreasing both abnormal angiogenesis and inflammation.

Presbyopia: Plan to initiate VEGA-2 Phase 3 trial in Q4 2022 investigating Nyxol alone and Nyxol with 0.4% low-dose pilocarpine (LDP) as adjunctive therapy. In addition, VEGA-3 (2nd Phase 3) and LYRA-1 (1-year safety) trials are planned to begin in 2023.

Third Quarter and Recent Business Highlights

Clinical and Regulatory Development

In September, the Company announced that the last of the 103 enrolled patients in the ZETA-1 Phase 2b trial of oral APX3330 for the treatment of diabetic retinopathy (DR) completed the final visit of the 24-week study.

In September, the Company announced that U.S. Food and Drug Administration (FDA) has granted a small-business waiver of the Prescription Drug User Fee Act (PDUFA) fee of $3.1 million for the 505(b)(2) NDA for Nyxol.

Presentations, Publications, and Conferences

Year to date, Ocuphire was represented at multiple key ophthalmological conferences with updates on Nyxol in RM, presbyopia and night vision disturbances, as well as masked safety data for APX3330 in DR. In total, more than 25 papers, posters, and panel talks were presented over 20 medical and industry conferences. Highlights in October and early November 2022:

Mitchell Jackson, MD presented a poster highlighting presbyopia data at the American Academy of Ophthalmology Annual Meeting in Chicago, IL.

Prominent optometry thought leaders and clinical trial investigators Justin Schweitzer, OD, Mitch Ibach, OD, Leslie O’Dell, OD, Shane Foster, OD, Doug , Devries, OD and Shane Kannarr, OD presented six posters on Nyxol and APX3330 at the American Academy of Optometry Annual Meeting in San Diego, CA.

The Company announced publication of an earlier Phase 2 clinical trial in patients with severe night vision disturbances in the BMC Ophthalmology peer-reviewed journal. The publication can be accessed here.

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In October, the Company held a Key Opinion Leader (KOL) webinar on oral APX3330. The event featured presentations by KOLs Peter Kaiser, MD, from the Cleveland Clinic, Caroline Baumal, MD, from Tufts Medical Center, and David Lally, MD, from New England Retina Consultants. KOL. The discussion highlighted the unmet need and current treatment landscape for DR/DME and included new data on study demographics and 24-week masked safety data from the ZETA-1 trial. A replay of the event can be found on the company’s corporate website here.

Corporate

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On August 2, 2022, Ocuphire was granted extended intellectual property protection for Nyxol with the issuance of U.S. Patent No. 11,400,077 with claims directed to methods for mydriasis treatment using phentolamine, extended by 5 years into 2039.

In September, the Company appointed seven new Key Opinion Leaders (KOLs) across retina, cornea/refractive, and medical optometry to its Medical Advisory Board (MAB): Anat Loewenstein, MD, PhD, Caroline Baumal, MD, Zaina Al-Mohtaseb, MD, Inder Paul Singh, MD, Leslie O’Dell, OD, Selina McGee, OD, Justin Schweitzer, OD.

Third Quarter Ended September 30, 2022, Financial Highlights

As of September 30, 2022, Ocuphire had cash and cash equivalents of approximately $13.9 million. Based on current projections, management believes the current cash on hand will be sufficient to fund operations into the fourth quarter of 2023. Net cash used in operating activities in the third quarter of 2022 was $4.5 million, with a cumulative total for the nine months ended September 30, 2022, of $14.5 million.

General and administrative expenses for the three and nine months ended September 30, 2022, were $1.7 million and $5.2 million, respectively, compared to $1.6 million and $6.7 million, respectively, for the three and nine months ended September 30, 2021. The increase from the comparable quarter in 2021 was largely attributed to an increase in legal costs on a net basis. The decrease from the comparable nine months in 2021 was largely attributed to a non-cash settlement with certain investors in the comparable prior year period, offset by a slight increase in general and administrative expenses attributed to higher payroll and other operating costs in the current year period when compared to the comparable prior year period.

Research and development expenses for the three and nine months ended September 30, 2022, were $2.8 million and $10.8 million, respectively, compared to $3.1 million and $10.4 million, respectively, for the three and nine months ended September 30, 2021. The decrease from the comparable quarter in 2021 was primarily attributable to the completion of clinical trials and the timing of manufacturing activities for Nyxol and APX3330. The increase from the comparable nine months in 2021 was primarily attributable to the timing of clinical trials and manufacturing activities for Nyxol and APX3330 as well as regulatory, preclinical and other development activities.

The total loss from operations for the three and nine months ended September 30, 2022, was $4.5 million and $16.0 million, respectively, compared to $4.2 million and $16.6 million, respectively, for the three and nine months ended September 30, 2021.

Net loss for the three and nine months ended September 30, 2022, was $4.5 million and $16.1 million, respectively, compared to $4.2 million and $50.4 million, respectively, for the three and nine months ended September 30, 2021. Net loss per share for the three and nine months ended September 30, 2022, was ($0.22) and ($0.82) per share, respectively, compared to ($0.25) and ($3.64) per share, respectively, for the comparable periods in 2021.

For further details on Ocuphire’s financial results, refer to the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2022, to be filed with the Securities and Exchange Commission.

On November 4, 2022 Pieris Pharmaceuticals, Inc. (the "Company") reported that entered into an Open Market Sale Agreement (the "Sales Agreement"), dated August 9, 2019, with Jefferies LLC ("Jefferies"), pursuant to which the Company may offer and sell shares of its common stock, from time to time, up to an aggregate amount of gross sales proceeds of $50.0 million through an "at the market offering" program (the "2019 ATM Program"), under a shelf registration statement on Form S-3 (File No. 333-226725) (Filing, 8-K, Pieris Pharmaceuticals, NOV 4, 2022, View Source [SID1234623105]).

In August 2021, the 2019 ATM Program expired, and the Company established a second ATM offering program (the "2021 ATM Program") under the Sales Agreement, pursuant to which the Company may offer and sell shares of its common stock, from time to time, up to an aggregate amount of gross sales proceeds of $50.0 million. As of November 4, 2022, the Company had offered and sold shares of its common stock with an aggregate offering price of approximately $33.6 million pursuant to the 2021 ATM Program.

On November 4, 2022, the Company and Jefferies entered into an amendment to the Sales Agreement (the "Amendment No. 1" and together with the Sales Agreement, the "Amended Sales Agreement") to provide for an increase in the aggregate offering amount under the Sales Agreement, such that as of November 4, 2022, the Company may offer and sell shares of its common stock having an aggregate offering price of up to $75.0 million pursuant to the prospectus supplement filed on November 4, 2022, exclusive of shares previously sold under the 2021 ATM Program. The material terms and conditions of the Sales Agreement otherwise remain unchanged.

Sales, if any, of the Company’s shares of common stock through Jefferies will be made by any method permitted by law deemed to be an "at the market offering" as defined in Rule 415(a)(4) under the Securities Act of 1933, as amended, including without limitation sales made directly on the Nasdaq Capital Market or any other existing trading market for its common stock. Jefferies will use commercially reasonable efforts to sell the shares from time to time, based upon instructions from the Company (including any price, time or size limits or other customary parameters or conditions it may impose). The Company is not obligated to make any sales of shares under the Amended Sales Agreement.

The foregoing description of the material terms of the Amendment No. 1 is qualified in its entirety by reference to the full texts of each of the Sales Agreement, a copy of which was filed as Exhibit 10.1 to the Company’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2019, filed with the SEC on August 9, 2019 and is incorporated herein by reference, and the Amendment No. 1, which is attached as Exhibit 1.1 hereto and is incorporated herein by reference.

The shares to be offered under the Amended Sales Agreement have been registered pursuant to the Company’s shelf registration statement on Form S-3 (File No. 333-258497) (the "Registration Statement"), and offerings for such shares will be made only by means of a prospectus supplement. This Current Report on Form 8-K shall not constitute an offer to sell or solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities law of such state or jurisdiction.

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On November 4, 2022 Amgen (NASDAQ:AMGN) reported that it will present at the Credit Suisse 31st Annual Global Healthcare Conference at 3:50 p.m. ET on Wednesday, November 9, 2022 (Press release, Amgen, NOV 4, 2022, View Source [SID1234623104]). David M. Reese, M.D., executive vice president of Research and Development and Peter H. Griffith, executive vice president and chief financial officer at Amgen will present at the conference. The webcast will be broadcast over the internet simultaneously and will be available to members of the news media, investors and the general public.

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The webcast, as with other selected presentations regarding developments in Amgen’s business given by management at certain investor and medical conferences, can be found on Amgen’s website, www.amgen.com, under Investors. Information regarding presentation times, webcast availability and webcast links are noted on Amgen’s Investor Relations Events Calendar. The webcast will be archived and available for replay for at least 90 days after the event.