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Not sure about the switches but for sure the news benefits bi- specifics. The companies you mentioned and AMGN as well
Thank you poorgrad and the others for the comments on TGTX. if the other competitors are behind in development, they still have that advantage IMO.
Interesting the Jakafi combination study . As early discussed I am INCY investor since early days too...
To postgradstudent: TGTX
I saw a comment from you on twitter regarding TGTX, could you elaborate more on your opinion of TGTX´s dual pi3k (delta y gamma)?
thanks
Achillion Presents Interim Phase 1 Results for ACH-4471, a Novel Orally-Administered Factor D Inhibitor, at the 21st Congress of the European Hematology Association
- Results indicate up to 100% inhibition of alternative-pathway (AP) activity in hemolysis and Wieslab assays after oral dosing of ACH-4471 -
- Results support initiation of phase 1 multiple-ascending trial during the second quarter and phase 2 studies for paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy (C3G) by year-end 2016 -
NEW HAVEN, Conn., June 10, 2016 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (ACHN) today presented interim results from a phase 1 study with its novel small molecule factor D inhibitor, ACH-4471, at the 21st Congress of the European Hematology Association in Copenhagen, Denmark.
The e-poster entitled, "An Orally Administered Small Molecule Factor D Inhibitor (ACH-4471) For Treatment of PNH, C3G and Complement – Mediated Diseases: Interim Phase 1 Results In Healthy Volunteers” describes results from an ongoing phase 1 study led by Roderick B. Ellis-Pegler and Christian Schwabe of Auckland Clinical Studies Ltd, Auckland, New Zealand and a group of researchers from Achillion.
Across all four groups, ACH-4471 achieved peak plasma concentrations between 1 and 2.5 hours after oral dosing. Up to 100% inhibition of complement activity was achieved in all dose groups, and duration of inhibition was dose dependent. In addition, Group 4, which evaluated 1,200 mg of ACH-4471 given every twelve hours for 2 doses, achieved a median 99.5% inhibition (range 96 – 100%) of hemolysis at 24 hours.
"We are very encouraged by these data, which support our focus on developing potent, specific factor D inhibitor compounds, including ACH-4471, as a potentially novel approach to treating alternative pathway complement-mediated diseases such as PNH and C3G,” commented Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion. “Achieving sustained suppression of AP activity, as measured by the AP hemolysis and Wieslab assays, combined with modeling from these interim study results, suggest the potential for twice daily dosing to achieve sustained inhibition of AP activity."
The phase 1 study, initiated in February 2016, aims to assess the safety and tolerability of single ascending oral doses of ACH-4471 in healthy volunteers and to evaluate its pharmacokinetic (PK) and pharmacodynamic (PD) profile and PK/PD relationship as measured by the serum alternative pathway (AP) activity ex vivo.
In the study, 36 subjects have been dosed and evaluated. Trial participants were assigned to one of four groups:
Group 1: 200 mg, single dose (6 active + 6 placebo subjects)
Group 2: 600 mg, single dose (6 active + 2 placebo subjects)
Group 3: 1200 mg, single dose (6 active + 2 placebo subjects)
Group 4: 1200 mg x 2 doses (Q12H) (6 active + 2 placebo subjects)
For all groups, inhibition of serum AP activity was evaluated using the AP Wieslab and hemolysis assays. Blood samples were collected from days 1 through 7 to determine plasma concentrations. Trial participants were monitored through the last scheduled visit at day 28 for any adverse events.
The interim study data indicate ACH-4471 was well-tolerated at all dose levels examined with no safety trends noted. Evaluation of serum AP activity by ex vivo assessment suggests rapid inhibition of AP activity after oral dosing. Furthermore, the concentrations of Bb, the cleavage product of factor B by factor D, were determined for the assessment of the inhibitory effect of ACH-4471 on in vivo factor D activity. Bb levels were shown to decline after dosing with ACH-4471 with the lowest Bb levels observed at 16 hours post-dosing in Groups 3 and 4 and a gradual return to baseline by 48 hours after dosing.
Achillion plans to begin dosing in a phase 1 multiple-ascending dose study evaluating 14-days of dosing in healthy volunteers in June. The Company is also planning to initiate phase 2 studies of ACH-4471 in two complement-mediated diseases, PNH and C3G, by the end of 2016.
Abstracts for the 2016 Congress of the European Hematology Association are available online and can be accessed at http://www.ehaweb.org/. A reprint of the late breaking poster presentation will be available from the Resources section of the Achillion website at http://www.achillion.com.
About the Achillion Complement Factor D Platform
Achillion has leveraged its internal discovery capabilities and a novel complement-related platform to develop drug candidates that are oral inhibitors of complement factor D. Factor D is an essential serine protease involved in the complement pathway, a part of the innate immune system. Achillion's complement platform is focused on seeking to advance small molecule compounds that inhibit factor D and can potentially be used in the treatment of immune-related diseases in which complement plays a critical role. Potential indications being evaluated for these compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 Glomerulopathy (C3G), dry age-related macular degeneration (dry AMD), and chronic obstructive pulmonary disease (COPD). Achillion anticipates that its platform could play a role in addressing the needs of all PNH patients, including patients who have suboptimal response to, or fail to respond to, the currently available treatments, as well as for patients suffering from other alternative pathway complement-mediated diseases. Achillion nominated ACH-4471 for clinical development in December 2015, and initiated clinical development in February 2016.
ACHN at EHA (PlatformD)
Achillion Presents Interim Phase 1 Results for ACH-4471, a Novel Orally-Administered Factor D Inhibitor, at the 21st Congress of the European Hematology Association
- Results indicate up to 100% inhibition of alternative-pathway (AP) activity in hemolysis and Wieslab assays after oral dosing of ACH-4471 -
- Results support initiation of phase 1 multiple-ascending trial during the second quarter and phase 2 studies for paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy (C3G) by year-end 2016 -
NEW HAVEN, Conn., June 10, 2016 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (ACHN) today presented interim results from a phase 1 study with its novel small molecule factor D inhibitor, ACH-4471, at the 21st Congress of the European Hematology Association in Copenhagen, Denmark.
The e-poster entitled, "An Orally Administered Small Molecule Factor D Inhibitor (ACH-4471) For Treatment of PNH, C3G and Complement – Mediated Diseases: Interim Phase 1 Results In Healthy Volunteers” describes results from an ongoing phase 1 study led by Roderick B. Ellis-Pegler and Christian Schwabe of Auckland Clinical Studies Ltd, Auckland, New Zealand and a group of researchers from Achillion.
Across all four groups, ACH-4471 achieved peak plasma concentrations between 1 and 2.5 hours after oral dosing. Up to 100% inhibition of complement activity was achieved in all dose groups, and duration of inhibition was dose dependent. In addition, Group 4, which evaluated 1,200 mg of ACH-4471 given every twelve hours for 2 doses, achieved a median 99.5% inhibition (range 96 – 100%) of hemolysis at 24 hours.
"We are very encouraged by these data, which support our focus on developing potent, specific factor D inhibitor compounds, including ACH-4471, as a potentially novel approach to treating alternative pathway complement-mediated diseases such as PNH and C3G,” commented Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion. “Achieving sustained suppression of AP activity, as measured by the AP hemolysis and Wieslab assays, combined with modeling from these interim study results, suggest the potential for twice daily dosing to achieve sustained inhibition of AP activity."
The phase 1 study, initiated in February 2016, aims to assess the safety and tolerability of single ascending oral doses of ACH-4471 in healthy volunteers and to evaluate its pharmacokinetic (PK) and pharmacodynamic (PD) profile and PK/PD relationship as measured by the serum alternative pathway (AP) activity ex vivo.
In the study, 36 subjects have been dosed and evaluated. Trial participants were assigned to one of four groups:
Group 1: 200 mg, single dose (6 active + 6 placebo subjects)
Group 2: 600 mg, single dose (6 active + 2 placebo subjects)
Group 3: 1200 mg, single dose (6 active + 2 placebo subjects)
Group 4: 1200 mg x 2 doses (Q12H) (6 active + 2 placebo subjects)
For all groups, inhibition of serum AP activity was evaluated using the AP Wieslab and hemolysis assays. Blood samples were collected from days 1 through 7 to determine plasma concentrations. Trial participants were monitored through the last scheduled visit at day 28 for any adverse events.
The interim study data indicate ACH-4471 was well-tolerated at all dose levels examined with no safety trends noted. Evaluation of serum AP activity by ex vivo assessment suggests rapid inhibition of AP activity after oral dosing. Furthermore, the concentrations of Bb, the cleavage product of factor B by factor D, were determined for the assessment of the inhibitory effect of ACH-4471 on in vivo factor D activity. Bb levels were shown to decline after dosing with ACH-4471 with the lowest Bb levels observed at 16 hours post-dosing in Groups 3 and 4 and a gradual return to baseline by 48 hours after dosing.
Achillion plans to begin dosing in a phase 1 multiple-ascending dose study evaluating 14-days of dosing in healthy volunteers in June. The Company is also planning to initiate phase 2 studies of ACH-4471 in two complement-mediated diseases, PNH and C3G, by the end of 2016.
Abstracts for the 2016 Congress of the European Hematology Association are available online and can be accessed at http://www.ehaweb.org/. A reprint of the late breaking poster presentation will be available from the Resources section of the Achillion website at http://www.achillion.com.
About the Achillion Complement Factor D Platform
Achillion has leveraged its internal discovery capabilities and a novel complement-related platform to develop drug candidates that are oral inhibitors of complement factor D. Factor D is an essential serine protease involved in the complement pathway, a part of the innate immune system. Achillion's complement platform is focused on seeking to advance small molecule compounds that inhibit factor D and can potentially be used in the treatment of immune-related diseases in which complement plays a critical role. Potential indications being evaluated for these compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 Glomerulopathy (C3G), dry age-related macular degeneration (dry AMD), and chronic obstructive pulmonary disease (COPD). Achillion anticipates that its platform could play a role in addressing the needs of all PNH patients, including patients who have suboptimal response to, or fail to respond to, the currently available treatments, as well as for patients suffering from other alternative pathway complement-mediated diseases. Achillion nominated ACH-4471 for clinical development in December 2015, and initiated clinical development in February 2016.
Not sure of these two specific names. Could be many targets in the onco space. Probably, not the ones everybody thinks
GILD revelations at yesterday´s GS conference.
They were very clear they are evaluating companies/compounds/programs to build up their ONCOLOGY franchise (not in NASH so ICPT or Genfit are out of the picture of possible BO)
Transcript:
"with our cash flow we're able to thankfully to both fund our internal programs, but also look for external opportunities and I am spending about 30% of my time, but we look at external opportunities. And as we have said, I have said and as well as our CEO, John Milligan has said if we find the opportunity where the science is right, the price is right, and the organizational fit is right, then we will embark on that.
In terms of NASH I think we don’t have a need anymore because we have four different mechanisms that are all in clinical development and we think that will suffice for now. The biggest need I would say or the need where we are looking into is in oncology because I think we have to fully build out the oncology branch as we need a few more development programs or products."
ZIOP: Thanks DFRAI. I am currently long the stock, but contrary to other stocks, I swing this one because is such a volatile stocks , is better if you trade it.
he seems to be pumping SRPT these days
What are your thoughts on ZIOP?
Add also the REGN-NTLA deal. agree, both companies have done extraordinary well after IPO. I actually posted here I was doing DD on NTLA to get in at IPO, after successful EDIT.
My concern was they are all in preclinical yet. Ups and downs should come.
GILD: I think in terms of management changes, it is more remarkable that Kevin Young is back to the company. This is a good thing for GILD. Closing +3,5%
I don't know what the "financial community" thinks, but it is clear that J&J thinks JNJ the market being large enough for it to enter
Nice to see JNJ looking at a doublet as well as the triplet.
Synthetic skin: Interesting. Do you know if they have a company spin out of the research lab to use the IP?
INCY/ARIA I think this deal makes a lot of sense for both companies. I like the move.
NTLA. NICE debut. Eom
Yes, NTLA IPO today. I decided to watch from the sidelines for the moment, as it is not yet clear to me the IP situation with NTLA and EDIT, and because pipelines still not in the clinic, but I will definitely will follow them closely and I wish them good luck and a successful IPO debut!
L.
Why is -10% ?
I visited them around 8 years ago.
can you elaborate? you mean he was fired? thanks
"personal reasons" could be anything. it is an excuse for not explaining further of the real reasons. It is really odd he only stayed for 4 months and resigned IMO.
SGYP. As you are long the stock, what is your opinion on CFO resign news?
He came from Shire and kept the job only 5 months
Is he leaving because Shire BO is not going to happen? lack of communication on the convert exchange?
XON what is your take in this sope opera? The article seems designed to attack the stock as it also says 7 more remaining short articles are coming. You can like the company or not but the SA publication smells fishy IMO.
If a piece of opinion can make the stock dip 25% in a day, non biotech speculative stock is safe. This should be investigated for investors safety as it is an coordinated attack, it should be punished
Yes, I am thinking of buying $BLUE if it goes again around the 52 week low this or next week.
Good luck
SGYP That was almost a sure thing so I sold 1/4 of my shares at $3.75 earlier today. Nevertheless the trend should be up from here. GL
COMPARING GENE EDITING COMPANIES
I am currently analyzing gene editing companies for possible investment. After successful Editas (EDIT) IPO, we have discussed the current share price does not justify the preclinical pipeline.
Nevertheless those investors who bought shares at IPO at $16, made a good deal as the stock was at $46 only one month after. (today share price is $38)
Bearing this in mind, there are two other interesting companies in the field, also disputing their IP; that plan to go public in the future: INTELLIA and CRISPR.
INTELLIA plans its IPO around June. As we mentioned here, they just closed the deal for $75 with REGN for several targets in liver and they have a program in orphan drug TTP (preclinical), where ALNY already has a PhIII program on same indication.
Intellia says the key for gene editing to succeed is the delivery, and they claim to have the technology to do that, with vivo Lipid Nanoparticle (LNP) delivery technology.
Investors in Intellia are Novartis, Atlas Venture (17%) and Caribou
The question is if Intellia would be able to replicate the successful IPO EDIT performed, as in that case it would make sense an investment in Intellia at IPO.
All comments are welcome, thank you.
COMPARING GENE EDITING COMPANIES
I am currently analyzing gene editing companies for possible investment. After successful Editas (EDIT) IPO, we have discussed the current share price does not justify the preclinical pipeline.
Nevertheless those investors who bought shares at IPO at $16, made a good deal as the stock was at $46 only one month after. (today share price is $38)
Bearing this in mind, there are two other interesting companies in the field, also disputing their IP; that plan to go public in the future: INTELLIA and CRISPR.
INTELLIA plans its IPO around June. As we mentioned here, they just closed the deal for $75 with REGN for several targets in liver and they have a program in orphan drug TTP (preclinical), where ALNY already has a PhIII program on same indication.
Intellia says the key for gene editing to succeed is the delivery, and they claim to have the technology to do that, with vivo Lipid Nanoparticle (LNP) delivery technology.
Investors in Intellia are Novartis, Atlas Venture (17%) and Caribou
The question is if Intellia would be able to replicate the successful IPO EDIT performed, as in that case it would make sense an investment in Intellia at IPO.
All comments are welcome, thank you.
Intellia, "private" to IPO...
Another Gene-Editing IPO
Intellia Therapeutics, which seeks to develop CRISPR-based technologies to target rare diseases, is hoping to raise $120 million in an initial public offering.
By Bob Grant | April 12, 2016Editas Medicine made headlines earlier this yearwhen it snagged more than $94 million in its initial public offering (IPO), selling 5.9 million shares at $16 apiece. Now, another CRISPR-focused firm, Intellia Therapeutics, is throwing its hat into the IPO ring. Intellia, which is based in Cambridge, Massachusetts, filed the paperwork required to go public yesterday (April 11). According to The Boston Globe, Intellia is seeking to raise about $120 million with its IPO, but the details of how many shares it will offer and at what price have not yet been announced.
The juxtaposition of Intellia’s IPO and that of Editas is made all the more intriguing because the companies were founded by CRISPR-Cas9 pioneers who remain locked in a patent dispute over the technology. Intellia was spun out of Caribou Biosciences, which was founded by University of California, Berkeley researcher Jennifer Doudna, while Editas was founded by the Broad Institute’s Feng Zhang, who currently holds key patents regarding the development of CRISPR.
Meanwhile, Intellia yesterday (April 11) announced a $75 million licensing deal with Regeneron Pharmaceuticals. Regeneron is paying the hefty sum so that it can develop new CRISPR-based therapies that target genes expressed in the liver.
“We are excited to be partnering with Regeneron, an industry leader in human genetics research,” Nessan Bermingham, Intellia CEO and cofounder, said in a statement. “Regeneron’s focus on advancing science to medicine is an excellent fit with Intellia’s approach, and together, we aim to bring potential cures to patients who are suffering from life-threatening rare diseases and genetic diseases.”
ALNY has also a program for TTR (Transthyretin amyloidosis) target. It is currently in PhII. not sure which approach is better (RNAi vs CRISPR)
Can this compound be found in any food? Is it sold as supplement? I am already buying the arterial protect pills from Life Extension, for my parents. Thanks
Thanks. I think Also that the Favorable vote from FDA panel on ICPT's OCA, will be good for CNAT and other NASH players.
CNAT: expecting good data possibly? Is up +13% more today
Would they raise money soon? Before data release?
CNAT was up 20% on friday AND today 18% more. Could be related to the recent deal on NASH from GILD or is just stock manipulation?
ARRY it actually closed green, so probably a non event?
ARRY Only 2% down, so probably was already discounted as ovarian is difficult indication...
Array BioPharma (ARRY +3.1%) inks a strategic collaboration with Tokyo-based Asahi Kasei Pharma (OTC:AHKSF)(OTCPK:AHKSY) to develop and commercialize certain Tropomyosin receptor kinase A (TrkA) inhibitors, including ARRY-954, for pain, inflammation and other non-cancer indications in Asia.
• Under the terms of the deal, Array will receive an upfront payment of $12M, up to $64M in milestones and double-digit royalties on net sales. Asahi will have exclusive rights to develop and commercialize the products in Japan, Korea, Taiwan and China for indication mentioned above. Array retains the rights to all compounds ex-Asia. Within Asia, it retains the rights to all cancer indications for all compounds, exclusive of those licensed to Asahi.
• TrkA is the highest affinity receptor for Nerve Growth Factor (NGF). It is widely expressed on peripheral pain-sensing neurons. Preclinical studies have shown that blocking NGF/TrkA signaling with small molecule kinase inhibitors blocks peripheral pain responses, with therapeutic potential for a range of difficult-to-treat pain conditions like cancer, osteoarthritis, chronic low back pain and diabetic peripheral neuropathy.
RDUS submits New Drug Application to FDA for Abaloparatide-SC
Just in time, as they announced they will submit the NDA in late Q1
ALDR agree with you EOM
ALDR is 30% premkt on positive data for their phase IIb for the migraine drug at 100 and 300mg