Design Therapeutics Azioni (NASDAQ: DSGN)

Design Therapeutics (NASDAQ:DSGN) Azioni

Design Therapeutics

Design Therapeutics Inc is a clinical-stage biopharmaceutical company engaged in the research and development of GeneTACTM molecules, which are a novel class of small-molecule gene-targeted chimera therapeutic candidates designed to be disease-modifying by addressing the underlying cause of diseases caused by inherited nucleotide repeat expansion mutations. The Company's product candidate is in Friedreich ataxia (FA), its second product candidate is in Fuchs endothelial corneal dystrophy (FECD), and it is also advancing its GeneTACTM program to address other serious nucleotide repeat-driven monogenic diseases.

Design Therapeutics Inc

Rosenblatt analyst Mike Genovese maintains Lumentum Holdings (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/LITE#NASDAQ">LITE</a>) with a Buy and maintains $105 price target.

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LITE

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CALX

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PLRZ

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NIVF

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AAOI

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OKLO

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ORCL

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GameStop shares are trading lower after the company announced a $1.75 billion proposed private offering of convertible senior notes.

AIHS

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CRMT

America's Car-Mart Q4 Sales Volumes Increased By 2.6% YoY To 15,649 Units; For Fiscal 2025 Sales Volumes Decreased 1.7% YoY To 57,022 Units

Disc Medicine, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/IRON#NASDAQ">IRON</a>), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today spotlights 5 posters presented at the EHA 2025 annual meeting in Milan, Italy. This year's presentations included data from HELIOS, an ongoing open-label extension study of bitopertin in EPP, which showed favorable long-term efficacy and safety with sustained protoporphyrin IX (PPIX) reductions, improvement in quality of life, and improved liver biomarkers. Disc is advancing development and registrational activities for bitopertin in EPP, with plans to submit an NDA in H2 2025. The company has initiated APOLLO, a confirmatory clinical trial of bitopertin in adults and adolescents with EPP. Disc recently launched a campaign to raise awareness of EPP among physicians, patients, and caregivers with emphasis on the causative role of PPIX accumulation in the disease. Learn more at PPIXisWhy.com.
Disc also presented longer term data from the continuation phase of its Phase 1b trial in MF anemia, showing sustained activity on key biomarkers and durable anemia response among major responders. Enrollment for the Phase 2 RALLY-MF trial of DISC-0974 is ongoing with the exploratory cohort for patients on concomitant momelotinib or pacritinib fully enrolled. The company expects to present initial RALLY-MF data in H2 2025. Additionally, Disc shared an update of the Phase 1 healthy volunteer trial of DISC-3405, initially presented at ASH 2024, which demonstrated deep, sustained reductions in serum iron and meaningful changes in hematologic parameters, establishing proof of mechanism. Based on these data, the company has initiated a Phase 2 trial of DISC-3405 in polycythemia vera (PV). Disc also presented an iron pulse study of DISC-3405 in healthy volunteers which showed inhibition of dietary iron uptake, further confirming the mechanism of action and supporting DISC-3405's potential in diseases of iron overload.
The collection of data presented at EHA adds to the evidence base supporting Disc's continued advancement of all three clinical candidates and provides support for expansion opportunities in new indications. Management will host a call to review these updates on June 16 at 8:00 am ET. Please register for the event on the Events and Presentations page of Disc's website (<a href="https://ir.discmedicine.com/">https://ir.discmedicine.com/</a>)
Bitopertin, DISC-0974, and DISC-3405 are investigational agents and are not approved for use as therapies in any jurisdiction worldwide.
Summary of Poster Presentations
Bitopertin:
HELIOS:
HELIOS is an ongoing Phase 2, open-label, long-term extension trial that enrolled 86 adult and adolescent patients with EPP from the BEACON and AURORA trials. Patients were randomized to receive 20 mg or 60 mg bitopertin in BEACON and 20 mg or 60 mg bitopertin or placebo in AURORA, with all patients transitioning to a 60 mg daily dose of bitopertin in HELIOS.
<ul>
<li>Longer term treatment with bitopertin was associated with sustained reductions in the disease-causing toxin PPIX, with additional benefit for patients receiving the 60 mg dose continuously</li>
<li>Continuous treatment with 60 mg of bitopertin reduced ALT and other exploratory hepatobiliary biomarkers</li>
<li>Nearly all participants reported substantial improvements in quality of life measures</li>
<li>Bitopertin exhibited a favorable longer-term safety profile with up to 2+ years of exposure and similar safety across adults and adolescents with EPP and XLP</li>
</ul>
DISC-0974:
DISC-0974 Phase 1b in MF anemia:
Data from the continuation phase of the Phase 1b trial of DISC-0974 in MF anemia as of October 2024 were presented. This multi-center, open-label trial included patients who were: non-transfusion dependent receiving no transfusions (nTD, n=23), transfusion dependent with low transfusion burden (TD Low, n=5) and transfusion dependent with high transfusion burden (TD High, n=7). The trial was comprised of both patients receiving concomitant JAK inhibitor therapy (n=13) and not receiving JAK inhibitor therapy (n=22).&nbsp;DISC-0974 was administered subcutaneously at 14 mg (n=1), 28 mg (n=7), 50 mg (n=12), 75 mg (n=9), or 100 mg (n=6) every 4 weeks for up to 6 treatments. Long-term results demonstrated:
<ul>
<li>Sustained hepcidin suppression, iron mobilization, and reduction in Zinc PPIX, a measure of iron restricted hemoglobin production</li>
<li>nTD patients:
<ul>
<li>50% of evaluable patients achieved major response of sustained mean hemoglobin increase of ≥1.5 g/dL</li>
<li>Hematologic improvement was durable through the continuation phase</li>
</ul>
</li>
<li>TD Low patients:
<ul>
<li>80% of evaluable patients achieved major response of transfusion independence over 16 weeks</li>
<li>Hematologic improvement was durable, and all major responders remained transfusion independent during the continuation phase</li>
</ul>
</li>
<li>TD High patients:
<ul>
<li>40% of evaluable patients achieved major response of transfusion independence over 12 weeks</li>
<li>1 of 2 patients with a major response entered the continuation phase and remained transfusion independent at Day 225, with follow-up ongoing</li>
</ul>
</li>
<li>DISC-0974 was safe and well-tolerated at all evaluated dose levels &nbsp;</li>
</ul>
DISC-0974 in combination with luspatercept and ESA (mouse model):
The effect of DISC-0974, luspatercept, and ESA as single agents and DISC-0974 combined with luspatercept or ESA on hematological parameters in wild type mice was evaluated. Results showed:
<ul>
<li>Treatment with DBIO-100 (a mouse analog of DISC-0974) suppressed hepcidin, enhanced iron availability, and boosted erythropoiesis in wild-type mice</li>
<li>Combining with DPO (ESA) or RAP-536 (mouse analog of luspatercept) led to additional hematological benefits beyond those agents alone</li>
</ul>
These results demonstrate that DISC-0974 has a distinct mechanism of action among anemia-targeted agents and highlight the potential for synergistic anemia benefits when combining DISC-0974 with other anemia-targeted agents.
DISC-3405:
Phase 1 Healthy Volunteer Study:
Updated SAD/MAD data from the Phase 1 trial of DISC-3405 in healthy volunteers were presented. In the SAD portion of this trial, healthy males and females ages 18 to 65 were given a single dose of placebo (n=10) or DISC-3405 at 75 mg intravenously (IV) (n=6), 37.5 mg subcutaneously (SC) (n=6), 75 mg SC (n=6), 150 mg SC (n=6), or 300 mg SC (n=6). The MAD portion included placebo (n=4), 75 mg SC (n=6), and 150 mg SC (n=6) cohorts dosed every 4 weeks for a total of 2 doses. Consistent with data presented at ASH 2024, results showed:
<ul>
<li>DISC-3405 produced dose-related increases in serum hepcidin with corresponding reductions in serum iron across all dose levels</li>
<li>DISC-3405 resulted in deep reductions in serum iron (ranging from 50-80% from baseline) that were sustained and support a once-monthly SC dosing regimen</li>
<li>Single and repeat dosing of DISC-3405 demonstrated meaningful reductions in hematologic parameters, including reticulocyte hemoglobin, hemoglobin, and hematocrit</li>
<li>DISC-3405 was generally well-tolerated at all evaluated dose levels, with no serious adverse events (AEs), greater than Grade 2 AEs, or AEs leading to study withdrawal</li>
</ul>
These results provide proof of mechanism and support the advancement DISC-3405 into proof of concept studies. Disc has now initiated a Phase 2 trial of DISC-3405 in PV with initial results expected in 2026.
Iron Pulse Study:
Disc conducted an iron pulse study in healthy volunteers to evaluate the effectiveness of DISC-3405 in inhibiting dietary iron uptake. N=8 healthy volunteers received placebo treatment followed by oral ferrous sulfate tablets on two sequential occasions to establish baseline iron absorption profiles. Participants were then given a single 150mg IV dose of DISC-3405, followed by oral ferrous sulfate "iron pulses" on the 2nd and 15th days following treatment. DISC-3405 resulted in an average 94% reduction in iron absorption at Day 2 and 68% at Day 15.
These results confirm the mechanism of DISC-3405 and demonstrate its ability to block dietary iron absorption, supporting the potential for treating diseases associated with iron overload.

Government

Regulations

IRON

Disc Medicine Highlights New Clinical Data on Three Drug Candidates for Rare Blood and Iron Disorders, Plans FDA Filing for Bitopertin After EPP Study

Cryptocurrency markets are slipping on Thursday, but the broader investor outlook remains optimistic, driven by growing institutional interest in Bitcoin.
<figure class="wp-block-table"> 
<table class="has-fixed-layout">
<tbody>
<tr>
<td>Cryptocurrency</td>
<td>Price&nbsp;&nbsp;&nbsp;</td>
<td>Gains +/-</td>
</tr>
<tr>
<td>Bitcoin (CRYPTO: <a class="ticker" href="https://www.benzinga.com/quote/btc/usd">BTC</a>)</td>
<td>$107,319.73</td>
<td>-1.8%</td>
</tr>
<tr>
<td>Ethereum (CRYPTO: <a class="ticker" href="https://www.benzinga.com/quote/eth/usd">ETH</a>)</td>
<td>$2,749.17</td>
<td>-0.8%</td>
</tr>
<tr>
<td>Solana (CRYPTO: <a class="ticker" href="https://www.benzinga.com/quote/sol/usd">SOL</a>)</td>
<td>$158.63</td>
<td>-3.8%</td>
</tr>
<tr>
<td>XRP (CRYPTO: <a class="ticker" href="https://www.benzinga.com/quote/xrp/usd">XRP</a>)</td>
<td>$2.23</td>
<td>-3.4%</td>
</tr>
<tr>
<td>Dogecoin (CRYPTO: <a class="ticker" href="https://www.benzinga.com/quote/doge/usd">DOGE</a>)</td>
<td>$0.1882</td>
<td>-6%</td>
</tr>
<tr>
<td>Shiba Inu (CRYPTO: <a class="ticker" href="https://www.benzinga.com/quote/shib/usd">SHIB</a>)</td>
<td>$0.00001263</td>
<td>-4.6%</td>
</tr>
</tbody>
</table>
</figure>
Notable Statistics:
<ul class="wp-block-list">
<li>IntoTheBlock data shows Bitcoin and Ethereum large transaction volume decreasing by 14.7% and 16.1%, respectively. Daily active addresses are down by 8.7% and 0.7%, respectively. ETH's exchange netflows are up by 88.3%.</li>
<li>Coinglass data shows 109,114 traders were liquidated in the past 24 hours for $327.18 million.</li>
<li>SoSoValue data shows net inflows of $164.6 million into spot Bitcoin ETFs, while spot Ethereum ETFs saw net inflows of $240.3 million.</li>
</ul>
Trader Notes: Market watcher More Crypto Online highlighted the importance of staying grounded during volatile cycles. Rather than reacting emotionally to dips or headlines, disciplined traders are focusing on key technical zones and upcoming retests — an approach that helps not only to survive the cycle but to grow through it.
Bitcoin hit a key bearish target, according to trader Carl The Moon, who emphasized in his latest update that the recent breakdown below crucial support levels opens the door for further downside.
<figure class="wp-block-embed is-type-rich is-provider-twitter wp-block-embed-twitter"> 
<div class="wp-block-embed__wrapper">
<blockquote class="twitter-tweet" data-width="500" data-dnt="true"><a href="https://twitter.com/hashtag/Bitcoin?src=hash&amp;ref_src=twsrc%5Etfw">#Bitcoin</a> has reached our bearish target from yesterday&#39;s post! But considering where the support was broken, <a href="https://twitter.com/search?q=%24BTC&amp;src=ctag&amp;ref_src=twsrc%5Etfw">$BTC</a> might fall even lower. <a href="https://t.co/cXOhAnnMTH">pic.twitter.com/cXOhAnnMTH</a>
&mdash; Carl Moon (@TheMoonCarl) <a href="https://twitter.com/TheMoonCarl/status/1933100973079068817?ref_src=twsrc%5Etfw">June 12, 2025</a></blockquote>
<script async src="https://platform.twitter.com/widgets.js" charset="utf-8"></script></div>
</figure>
Trader Degen Hardy <a href="https://x.com/Degen_Hardy/status/1933109145248707003">highlighted</a> that Bitcoin is nearing the $106,000–$107,000 value area high zone, suggesting a possible retest. 
He remains on the sidelines, with no active long positions, opting to wait for clearer confirmation before making moves.
On the Ethereum front, More Crypto Online is <a href="https://x.com/Morecryptoonl/status/1933101958929256642">watching</a> whether bulls can defend the critical $2,611–$2,772 support zone, which aligns with key wave (4) levels. So far, ETH has shown relative strength compared to BTC, suggesting potential resilience.
Solana is catching attention again — trader Posty predicts a rally toward the $180 mark, pointing to technical strength amid broader market caution.
<figure class="wp-block-embed is-type-rich is-provider-twitter wp-block-embed-twitter"> 
<div class="wp-block-embed__wrapper">
<blockquote class="twitter-tweet" data-width="500" data-dnt="true"><a href="https://twitter.com/search?q=%24SOL&amp;src=ctag&amp;ref_src=twsrc%5Etfw">$SOL</a> $180 soon <a href="https://t.co/HGYvYqd41t">pic.twitter.com/HGYvYqd41t</a>
&mdash; Posty (@PostyXBT) <a href="https://twitter.com/PostyXBT/status/1933082976939819085?ref_src=twsrc%5Etfw">June 12, 2025</a></blockquote>
<script async src="https://platform.twitter.com/widgets.js" charset="utf-8"></script></div>
</figure>
As for Dogecoin, trader Tardigrade <a href="https://x.com/TATrader_Alan/status/1933087157545226443">pointed</a> out a repeating pattern on the weekly chart, a series of small rallies leading into major breakouts. He believes the current move may follow this trend, with some market watchers forecasting a potential push toward the $1 milestone.
Read Next:
<ul class="wp-block-list">
<li><a href="https://www.benzinga.com/crypto/cryptocurrency/25/06/45887955/bitcoin-hovers-below-110000-is-this-the-calm-before-the-storm">Bitcoin Hovers Below $110,000: Is This The Calm Before The Storm?</a></li>
</ul>
Image: Shutterstock

Coverage of Bitcoin, Etherum, Ripple and all the latest digital currencies.

Cryptocurrency

$BTC

$DOGE

$ETH

$SHIB

$SOL

$XRP

Bitcoin, Ethereum, XRP, Dogecoin Slide On Thursday Morning: What's Going On?

Polyrizon Announces Preclinical Results From Study Evaluating Its Trap & Target Platform Showing Formulation Achieved Targeted Deposition In Upper Regions Of Nasal Cavity

– Encouraging clinical activity with deep responses demonstrated in the KOMET-007 trial with the combination of 600 mg ziftomenib with 7+3 in newly diagnosed patients with NPM1-m and KMT2A-r AML&nbsp;–
– 93% (41/44) and 89% (24/27) CRc observed in NPM1-m and KMT2A-r AML response-evaluable patients, respectively&nbsp;–
– 71% (24/34) and 88% (14/16) CR measurable residual disease (MRD)-negativity observed among responding NPM1-m and KMT2A-r AML patients, respectively –
– 96% (47/49) of NPM1-m and 88% (29/33) KMT2A-r AML patients remain alive and continue on-study –
– Combination was well tolerated with no additive myelosuppression&nbsp;–
– KOMET-017-IC (intensive chemotherapy) and NIC (non-intensive chemotherapy) randomized phase 3 studies (NCT07007312) expected to start in 2H 2025 –
–&nbsp;Kura Oncology&nbsp;to host virtual investor event to discuss results and broader ziftomenib development plan on June 18, 2025 at 4:30pm ET / 1:30pm PT&nbsp;–
SAN DIEGO and TOKYO, June 12, 2025 (GLOBE NEWSWIRE) -- &nbsp;Kura Oncology, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/KURA#NASDAQ">KURA</a>, "Kura"))) and Kyowa Kirin Co., Ltd. (TSE: 4151, "Kyowa Kirin") today provided positive updated clinical data from KOMET-007, a Phase 1a/1b trial of ziftomenib, a highly selective oral investigational menin inhibitor, in combination with standards of care in patients with newly diagnosed NPM1-mutant (NPM1-m) and KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML). The data for the combination with cytarabine/daunorubicin (7+3) were presented as an oral presentation at the European Hematology Association 2025 Congress (EHA2025) being held in Milan, Italy from June 12-15, 2025.

KURA

Kura Oncology And Kyowa Kirin Report Updated Clinical Data From KOMET-007, Phase 1a/1b Trial Of Ziftomenib, In Patients With Newly Diagnosed NPM1-M And KMT2A-R AML

Press Releases

Design Therapeutics to Participate in Leerink's Global Healthcare Conference

Earnings

Design Therapeutics Highlights Progress Across Lead GeneTAC® Programs and Reports Fourth Quarter and Full Year 2024 Financial Results

Management

Bright Peak Therapeutics Appoints John Schmid to its Board of Directors

Design Therapeutics Appoints Veteran Industry Executive Chris Storgard, M.D., as Chief Medical Officer

Health Care

Design Therapeutics to Present Phase 1 Data for Fuchs Endothelial Corneal Dystrophy Program at Eyecelerator @ Park City 2025

Design Therapeutics Announces Favorable Phase 1 Data for DT-168 Supporting Advancement into Phase 2 Biomarker Trial for Patients with Fuchs Endothelial Corneal Dystrophy

Design Therapeutics Highlights Momentum Across Lead GeneTAC® Programs and Reports First Quarter 2025 Financial Results

Design Therapeutics to Participate in 2025 RBC Capital Markets Healthcare Conference

Design Therapeutics to Participate in 2025 Jefferies Global Healthcare Conference

Design Therapeutics Announces Start of Friedreich Ataxia Patient Dosing Ex-U.S. in its RESTORE-FA Phase 1/2 Multiple-Ascending Dose Trial of DT-216P2

Comments

Design Therapeutics

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PARTNER/COLLABORATORE

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