On January 10, 2021 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported preliminary, unaudited ORLADEYO (berotralstat) revenue for the fourth quarter and full year 2021 and provided new guidance for full year 2022 ORLADEYO net revenue and expected peak ORLADEYO sales (Press release, BioCryst Pharmaceuticals, JAN 10, 2022, View Source [SID1234598509]).

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"Following 12 months of a successful launch through a global pandemic, we have a clear picture of the continued commercial trajectory for ORLADEYO based on a very attractive product profile, leading to strong patient demand to switch from injectable therapies to our oral, once-daily medicine, with 70 percent patient retention through the first year. Building on our substantial 2021 patient base, we are confident that ORLADEYO will achieve no less than $250 million of net revenue in 2022 and that ORLADEYO will become the market leader as the most prescribed prophylactic therapy with peak sales of $1 billion," said Jon Stonehouse, president and chief executive officer of BioCryst.

Fourth Quarter 2021 (Q4 2021) ORLADEYO Launch Dynamics

Preliminary, unaudited ORLADEYO net revenue in Q4 2021 was $45.6 million. Preliminary, unaudited ORLADEYO net revenue for full year 2021 (FY 2021) was $122 million.

New patient demand for ORLADEYO remains strong and consistent, with a similar number of new patients added in Q4 2021 as in each of the previous three quarters of the year. Patients switching from other prophylactic therapies and acute-only therapy continue to drive the launch. More than half of patients new to ORLADEYO since launch had a previous prophylactic medicine prior to ORLADEYO and most of the remainder were from acute-only treatment.

Most patients are well-controlled on ORLADEYO and remain on therapy. Approximately 70 percent of patients starting ORLADEYO, including those switching from injectable prophylaxis, remain on ORLADEYO in the first year.

ORLADEYO is now covered by all major payors and national and regional pharmacy benefit managers, which will lead to more patients being reimbursed quickly.

The ORLADEYO prescriber base continues to grow significantly. The number of new physicians prescribing ORLADEYO in Q4 2021 was similar to the number added in Q3 2021. In market research, 60 U.S. physicians, who treat an average of seven HAE patients each, reported that they expect to double their use of ORLADEYO, and that ORLADEYO will become their most prescribed prophylactic treatment in the next 12 months.
"We expect ORLADEYO revenues in 2022 to more than double in our second year of launch as we benefit from a full year of reimbursement and continued strong demand from patients and physicians. ORLADEYO is transforming the lives of HAE patients, which is why ORLADEYO is on a trajectory to become the market leader in HAE prophylaxis," said Charlie Gayer, chief commercial officer of BioCryst.

Pipeline Update: BCX9930 Pivotal Trials in PNH Now Enrolling

BioCryst is currently enrolling patients in two global pivotal trials, REDEEM-1 and REDEEM-2, with the company’s oral Factor D inhibitor, BCX9930 (500 mg bid), in patients with paroxysmal nocturnal hemoglobinuria (PNH). The company also has begun screening patients in a proof of concept (PoC) basket trial of BCX9930 (500 mg bid) in patients with C3 glomerulopathy (C3G), IgA nephropathy (IgAN) and primary membranous nephropathy (PMN).

BioCryst plans to further advance and expand its Factor D program over the next two years by achieving the following:

Complete and report data from REDEEM-1 and REDEEM-2
Prepare to submit regulatory approval filings in PNH
Complete the renal PoC basket trial and advance to pivotal trials in C3G, IgAN and PMN
Commence PoC trials in other complement-mediated diseases
"Following the discovery, development and commercialization of ORLADEYO, the BioCryst team plans to repeat this success as we leverage our platform to bring new oral medicines to patients suffering from other rare diseases. BCX9930 is especially exciting because the clinical data we have reported so far provides confidence that we can help patients in PNH, and across many complement-mediated diseases, with this pipeline in a molecule," said Dr. Helen Thackray, chief research and development officer of BioCryst.

Presentation Today at 40th Annual J.P. Morgan Healthcare Conference

Today at 9:45 a.m. ET, the company will present at the 40th Annual J.P. Morgan Healthcare Conference, which is being conducted as a virtual event. Links to a live audio webcast and replay of the presentation may be accessed in the Investors section of BioCryst’s website at https://www.biocryst.com/.

About ORLADEYO (berotralstat)
ORLADEYO (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.

U.S. Indication and Important Safety Information

INDICATION
ORLADEYO (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

Limitations of use
The safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation.

IMPORTANT SAFETY INFORMATION
An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent.

The most common adverse reactions (≥10 percent and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.

A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C) and in patients taking chronically administered P-glycoprotein (P-gp) or breast cancer resistance protein (BCRP) inhibitors (eg, cyclosporine).

Berotralstat is a substrate of P-gp and BCRP. P-gp inducers (eg, rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO.

ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO.

The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established.

There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production.

On January 10, 2022 Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") reported the exercise of its option to acquire Adaptate Biotherapeutics ("Adaptate"), a UK company focused on developing antibody-based therapeutics for the modulation of variable delta 1 (Vδ1) gamma delta (γδ) T cells (Press release, Takeda, JAN 10, 2022, View Source [SID1234598526]). Through the acquisition, Takeda will obtain Adaptate’s antibody-based γδ T cell engager platform, including pre-clinical candidate and discovery pipeline programs. Adaptate’s γδ T cell engagers are designed to specifically modulate γδ T cell-mediated immune responses at tumor sites while sparing damage to healthy cells.

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The planned acquisition of Adaptate follows Takeda’s recently exercised option to acquire GammaDelta Therapeutics ("GammaDelta") and is intended to further accelerate the development of innovative γδ T cell-based therapies. Combining GammaDelta’s cell therapy-based platform and Adaptate’s antibody-based γδ T cell engager platform with Takeda’s strong research and development organization positions Takeda to be at the leading edge in deploying the full potential of γδ T cells in the fight against cancer. The planned acquisition complements Takeda’s ongoing efforts to research and develop cell engagers for solid tumor applications, bolstered by the novel T cell engager COBRA platform, which was acquired from Maverick Therapeutics in another successful build-to-buy collaboration.

"Partnering with early-stage innovators to access cutting-edge platforms in the fight against cancer is at the center of our R&D strategy," said Christopher Arendt, Ph.D., Head of Oncology Cell Therapy and Therapeutic Area Unit of Takeda. "Adaptate’s γδ T cell engager platform and the team’s deep understanding of γδ T cell biology gives us an opportunity to develop a new class of therapeutics that tap into powerful innate immune mechanisms. The planned acquisition will strengthen our immuno-oncology R&D efforts as part of our ongoing pursuit of life-transforming medicines for patients with cancer."

Adaptate was formed in 2019 as a spin-out company from GammaDelta with investment from Abingworth LLP and Takeda, in which Takeda received an exclusive right to purchase Adaptate for a pre-negotiated upfront payment. The acquisitions of Adaptate and GammaDelta are expected to be finalized in Q1 of Takeda’s fiscal year 2022, pending completion of review under applicable antitrust laws, including the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976 in the U.S.

"Our acquisition by Takeda recognizes the tremendous work put in over the last two years by Adaptate’s incredibly talented team," said Dr. Natalie Mount, CEO of Adaptate. "We have rapidly demonstrated, in preclinical models, the therapeutic potential of our novel Vδ1-targeting antibodies, and this move brings us an exciting step closer to realizing the full potential for Vδ1 T cell targeted therapies to improve treatment outcomes for cancer patients."

In addition, Tim Haines, Chair & Managing Partner at Abingworth noted, "Having played an instrumental role in creating Adaptate, we are delighted to see the impressive developments of its γδ T cell therapeutic antibody portfolio to date, under the leadership of Natalie Mount. We look forward to seeing Takeda progress Adaptate’s very promising therapeutic antibodies into the clinic."

Takeda’s oncology pipeline focuses on novel strategies that leverage the power of the immune system, with a focus on innate immunity. Innate immune responses serve as the body’s first defense mechanism against disease and involve the orchestration of a broad arsenal of mechanisms and cell types, including γδ T cells and natural killer (NK) cells, that may help to overcome cancer’s ability to evade immune recognition. Adaptate has discovered a unique set of antibodies that selectively modulate γδ T cell activity in the tumor microenvironment. The antibodies provide a precisely targeted signal to the immune system, thereby offering the opportunity for superior efficacy and safety compared to conventional immuno-oncology approaches in solid tumors.

Takeda’s Commitment to Oncology

Our core R&D mission is to deliver novel medicines to patients with cancer worldwide through our commitment to science, breakthrough innovation and passion for improving the lives of patients. Whether it’s with our hematology therapies, our robust pipeline, or solid tumor medicines, we aim to stay both innovative and competitive to bring patients the treatments they need. For more information, visit www.takedaoncology.com

On January 10, 2022 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for people with cancer, reported that updates on clinical programs and key milestones anticipated in 2022 (Press release, Arcus Biosciences, JAN 10, 2022, View Source [SID1234598542]).

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"Our priorities for 2022 are clear and unambiguous—to flawlessly execute on the expansion of our global clinical programs which will include more than 10 randomized Phase 2 and 3 studies. We also expect to present randomized datasets from ARC-7 and ARC-8 at medical meetings and generate early data for AB521 that will clarify its potential as a best-in-class molecule," said Terry Rosen, Ph.D., Chief Executive Officer of Arcus Biosciences. "Our strong cash position and the support from our partner Gilead Sciences enable earlier investment to intelligently advance a broad development plan for our novel and potentially practice-changing combinations to treat cancer."

Program Updates and 2022 Milestones

Anti-TIGIT program (domvanalimab and AB308)

Recent Updates:

Taiho Pharmaceutical Co., Ltd., exercised its option for domvanalimab and AB308 in Japan and certain other territories in Asia (excluding China). In exchange for the exclusive license, Taiho will make an option exercise payment, as well as additional payments upon achievement of clinical, regulatory and commercialization milestones, and, if any products from the program are approved, will pay royalties on net sales of such products.
Anticipated 2022 Milestones:

Data from ARC-7, an ongoing randomized 150-patient three-arm study in first-line PD-L1≥50% NSCLC, including progression-free survival data, are expected to be presented in 2H22.
In addition to ARC-10, an ongoing registrational study in 1L PD-L1≥50% NSCLC, we and Gilead plan to initiate two new Phase 3 studies in lung and gastrointestinal (GI) cancers, as well as additional clinical studies of domvanalimab-based combinations, in 2022.
AstraZeneca and Arcus will initiate the PACIFIC-8 registrational Phase 3 study in January to evaluate domvanalimab plus durvalumab, an anti-PD-L1 antibody, in unresectable Stage 3 NSCLC with curative intent, where durvalumab is standard of care.
Data from the Phase 1/1b ARC-12 study evaluating AB308, an Fc-enabled anti-TIGIT antibody, plus zimberelimab in advanced malignancies will inform future development plans.
Etrumadenant (A2a/A2b adenosine receptor antagonist)

ARC-4 Update:

The randomized Phase 1/1b study ARC-4 did not show differentiated clinical activity for etrumadenant plus zimberelimab and pemetrexed/carboplatin compared to that of zimberelimab and pemetrexed/carboplatin in patients with metastatic, EGFRm+ NSCLC who progressed after one or more TKI therapies. Arcus is conducting exploratory biomarker analyses to assess whether sub-populations of patients in the study derive benefit from etrumadenant-based treatment, such as those with PD-L1 high tumors. Final data from the study will be presented when available.
All ongoing studies for etrumadenant will continue unchanged. These studies are in settings where encouraging clinical activity has been observed, such as PD-L1≥50% NSCLC (ARC-7), castrate-resistant prostate cancer (CRPC; ARC-6), and colorectal cancer (CRC; ARC-9). Patients whose tumors harbor EGFRm+ characteristics are excluded from the ARC-7 and ARC-10 studies.
Anticipated 2022 Milestones:

Data from the etrumadenant-containing arm of ARC-7 are anticipated to be presented in 2H22, as noted above.
Data from the randomized cohort of ARC-6 evaluating etrumadenant plus zimberelimab and docetaxel versus docetaxel in second-line (2L) metastatic CRPC are anticipated in 2H22.
Additional clinical studies for etrumadenant-based combinations, including the "triplet" of etrumadenant plus domvanalimab and zimberelimab, are being planned for 2022.
Quemliclustat (small molecule CD73 inhibitor)

Recent Updates:

Completed enrollment of 90 patients into the randomized portion of ARC-8, a Phase 1 study evaluating quemliclustat plus zimberelimab and gemcitabine/nab-paclitaxel vs quemliclustat plus gemcitabine/nab-paclitaxel in 1L pancreatic cancer.
Anticipated 2022 Milestones:

Results from the randomized portion of ARC-8, including data on progression-free survival, are expected to be presented in 2H22.
Enrollment of the cohort in 2L pancreatic cancer, an area of high unmet need, is on track to be completed in 1H22.
Additional clinical studies for quemliclustat are being planned for 2022.
AB521 (HIF-2a inhibitor)

Recent Updates:

Initiated ARC-14, a study to investigate the safety, tolerability, and pharmacokinetic profile of AB521 in healthy volunteers.
Anticipated 2022 Milestones

Share pharmacokinetic and safety data from ARC-14 in 1H22, which may demonstrate competitive advantages to other HIF-2a inhibitors.
Initiate Phase 1/1b study in oncology patients in mid-2022.
Discovery Programs:

Added a research collaboration to the existing agreement with Gilead under which Arcus will lead the discovery and early development of drug candidates against two novel research targets jointly selected by the parties.
Selected AB598 (CD39 antibody) as a development candidate, which is advancing into IND-enabling studies; several other oncology discovery programs continue to progress.
In 1H22, expect to select the first development candidate for a non-oncology target. This small molecule may have first-in-class potential in several inflammatory diseases.
Financial Guidance

Arcus’s cash position will nearly double to $1.4 billion, upon receiving the option payment totaling $725 million for three programs from Gilead. With this cash position and 50/50 cost sharing with Gilead for the joint development programs, Arcus plans to expand its clinical development programs and anticipates cash utilization of $275-325 million in 2022.

Arcus Clinical Study Overview

Trial

Name

Arms

Setting

Status

NCT No.

ARC-4

etruma + zim + carbo/pem vs.

zim + carbo/pem

TKI R/R EGFRmut NSCLC

Ongoing

Randomized

Phase 1/2

NCT03846310

ARC-6

etruma + zim + SOC vs. SOC

2L/3L CRPC

Ongoing

Randomized

Phase 2

NCT04381832

ARC-7

zim vs. zim + dom vs. zim +

dom + etruma

1L NSCLC (PD-L1 ≥ 50%)

Ongoing

Randomized

Phase 2

NCT04262856

ARC-8

quemli + zim + gem/nab-pac

vs. quemli + gem/nab-pac

1L PDAC

Ongoing

Randomized

Phase 1/1b

NCT04104672

ARC-9

etruma + zim + mFOLFOX

vs. SOC

2L/3L/3L+ CRC

Ongoing

Randomized

Phase 2

NCT04660812

ARC-10

chemo vs. zim vs. zim + dom

1L NSCLC (PD-L1 ≥ 50%)

Ongoing

Registrational

NCT04736173

ARC-12

AB308 + zim

Advanced Malignancies

Ongoing

Phase 1/1b

NCT04772989

ARC-14

AB521

Healthy Volunteer

Ongoing

NCT05117554

PACIFIC-8

durva ± dom

Curative-Intent Stage 3 NSCLC

Planned

Registrational

NA

Carbo/pem: carboplatin/pemetrexed; dom: domvanalimab; durva: durvalumab; etruma: etrumadenant; gem/nab-pac: gemcitabine/nab-paclitaxel; quemli: quemliclustat; R/R: relapsed/refractory; SOC: standard of care; zim: zimberelimab CRC: colorectal cancer; CRPC: castrate-resistant prostate cancer; NSCLC: non-small cell lung cancer; PDAC: pancreatic ductal adenocarcinoma

On January 10, 2022 Celsion Corporation (NASDAQ: CLSN), a clinical-stage development company focused on DNA-based immunotherapy and next-generation vaccines, reported that it has entered into a securities purchase agreement with certain institutional investors to purchase 50,000 shares of Series A convertible redeemable preferred stock and 50,000 shares of Series B convertible redeemable preferred stock (Press release, Celsion, JAN 10, 2022, View Source [SID1234598559]). Each share of Series A and Series B preferred stock has a purchase price of $285, representing an original issue discount of 5% of the $300 stated value of each share. Each share of Series A preferred stock is convertible into shares of Celsion’s common stock at an initial conversion price of $0.91 per share. Each share of Series B preferred stock is convertible into shares of Celsion’s common stock at an initial conversion price of $1.00 per share. Shares of the Series A and Series B preferred stock are convertible at the option of the holder at any time following the Company’s receipt of stockholder approval for a reverse stock split of the Company’s common stock. Celsion will be permitted to compel conversion of the Series A and Series B preferred stock after the fulfillment of certain conditions and subject to certain limitations. Total net proceeds from the offerings, before deducting the placement agent’s fees and other estimated offering expenses, is approximately $28.5 million.

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The Series A and Series B preferred stock permit the holders thereof to vote together with the holders of the Company’s common stock on a proposal to effectuate a reverse stock split of the Company’s common stock at a special meeting of Company stockholders. The Series A preferred stock permits the holder to vote on such proposal on an as-converted to common stock basis. The Series B preferred stock permits the holder to cast 45,000 votes per share of Series B preferred stock on such proposal. The Series A and Series B preferred stock will not be permitted to vote on any other matter. The holders of the Series A and B preferred stock agreed not to transfer their shares of preferred stock until after the special meeting of Company stockholders. The holders of the Series A preferred stock agreed to vote their shares in favor of that proposal and the holders of the Series B preferred stock agreed to vote their shares in the same proportions as the shares of common stock and Series A preferred stock are voted on that proposal. The holders of the Series A and Series B preferred stock have the right to require the Company to redeem their shares of preferred stock for cash at 105% of the stated value of such shares commencing after the earlier of the Company’s stockholders’ approval of the reverse stock split and 90 days after the closing of the issuances of the Series A and Series B preferred stock and until 120 days after such closing.

The closing of the offerings is expected to occur on or about January 13, 2022, subject to the satisfaction of customary closing conditions. Additional information regarding the securities described above and the terms of the offering are included in a Current Report on Form 8-K to be filed with the United States Securities and Exchange Commission ("SEC").

A.G.P. /Alliance Global Partners is acting as the sole placement agent in connection with the offering.

The Series A and Series B preferred stock and shares of common stock into which such preferred stock are convertible are being offered pursuant to a registration statement on Form S-3 (333-254515), which was declared effective by the Securities and Exchange Commission on March 30, 2021. The offerings will be made only by means of prospectus supplements and a prospectus that form a part of the registration statement. Copies of the final prospectus supplements and accompanying prospectus relating to the registered direct offering may be obtained from A.G.P./Alliance Global Partners, 590 Madison Avenue, 28th Floor, New York, NY 10022 at (212) 624-2060.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On January 10, 2022 Atara Biotherapeutics, Inc. (the "Company") Presented the Corporate Presentation (Presentation, Atara Biotherapeutics, JAN 10, 2022, View Source [SID1234598476]).

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