Archemix: Initiates Phase II ARC1779 Study

CLSDF continues to cover Cambridge-based Archemix; find out why (here) and (here). The aptamer company announced initiation of a Phase II study of ARC1779. The trial is designed to evaluate the safety and efficacy of ARC1779 as a first-in-class anti-platelet agent in patients suffering from a rare, life-threatening blood disorder known as thrombotic thrombocytopenic purpura (TTP).

TTP is a rare blood disorder that arises from uncontrolled activation of von Willebrand Factor (vWF), a protein which under normal conditions plays a key role in maintaining the normal balance between bleeding and clotting. In patients suffering from TTP, vWF binds excessively to platelets, causing excessive blood clots. These clots form throughout the circulation and can lead to serious medical consequences such as strokes, seizures, kidney failure and heart attack. Each year in the United States, between four and eleven new cases of TTP per million of the total population are diagnosed. There is no approved drug treatment for patients with TTP.

Aptamers are synthetically-derived oligonucleotides that bind to protein targets with high affinity and specificity and can be designed to have a specified duration of action. Aptamers represent an emerging class of potential therapeutic agents that Archemix believes may have broad application to treat a variety of human diseases. ARC1779, a pegylated aptamer consisting of 40 nucleotides, is Archemix’ lead product candidate for the treatment of TTP. ARC1779 is designed to inhibit the function of vWF, which is, when activated, responsible for the adhesion, activation and aggregation of platelets.

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Archemix – Milestone Pandemonium

CLSDF has highlighted the history and relationship of Archemix to CO in previous posts; read about it (here). It has been a very busy 2008 thus far for the Cambridge-based aptamer therapeutics company who has now (I believe it is technically a - sub - based upon University Colorado orgins…?) licensed out the right to discover and develop therapeutic aptamers to Pfizer (NYSE: PFE), Merck (NYSE: MRK), Nuvelo (NASDAQ: NUVO), Takeda (TSE: 4502), Elan (NYSE: ELN), NascaCell Technologies and Ophthotech.

Most recently Archemix has been awarded an undisclosed milestone payment from Ophthotech Corp. for development of their aptamer candidate E10030 an anti-platelet derived growth factor being developed for age-related macular degeneration. Opthotech is now enrolling a Phase I study of E10030 and Archemix is eligible for additional follow-on milestone payments and royalties on future sales of E10030.

Archemix has also recently been awarded a $1M milestone payment by Nuvelo for development of their aptamer candidate NU172 for thrombin-inhibition and blood clot formation stimulation. Nuvelo is enrolling a Phase I study of NU172 and Archemix is eligible for additional follow-on milestone payments and royalties on future sales of NU172.

Finally, an expansion of the strategic alliance with NascaCell Technologies was announced on the use and development of aptamers for drug discovery applications. Under the terms of the new agreement, NascaCell receives non-exclusive rights to uses of aptamers for target validation, mode of action studies, drug discovery, and aptamer structure-based drug design. Specific financial terms were not disclosed

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Selecting a SELEX® Pathway

You may have seen the news yesterday on the Archemix and Merck KGaA deal for the use of the SELEX® technology in the oncology field. Well Cambridge and Germany are a long way from Colorado, or are they…

The origins of the Archemix/Merck deal may be traced back to the first floor of Porter Bioscience, home to the University Colorado, Boulder department of molecular, cell and developmental biology and laboratory of Dr. Larry Gold approaching some twenty years ago. Aptamers (a single-stranded oligonucleotide that assumes a specific sequence-dependent shape and binds to a target protein based on a lock-and-key fit between the two molecules) and the SELEX® process (an evolutionary, in vitro combinatorial chemistry process used to identify aptamers to a target from large pools of diverse oligonucleotides) were both discovered there and formed the basis of NeXagen which later merged with Vestar to become NeXstar Pharmaceuticals and in 1991 NeXstar was acquired in an all stock deal by Gilead.

Along the way the SELEX® technology created the aptamer pegaptanib, an anti-VEGF drug approved as Macugen in 2004 for wet age-related macular degeneration. Macugen was created by Eyetech Pharmaceuticals and where rights and ventures lead it through the halls of Gilead, OSI Pharmaceuticals and Pfizer.

Around 2001 Archemix successfully licensed rights to the intellectual property portfolio of Gilead covering aptamers and the SELEX® process for a wide variety of applications, including therapeutic uses. This portfolio included over 400 issued and pending patents resulting from work carried out at both Gilead and NeXstar in the 1990’s. This comprehensive patent estate has established Archemix as the leading aptamer therapeutics company.

Though Archemix is driving aptamers down the therapeutic field with pipeline products in cardiovascular, oncology and autoimmune disease Boulder-based Somalogic has licensed back from Gilead the diagnostic field of use of aptamers and SELEX® and are planning to transform medical practice through next generation disease screening, diagnosis, therapy selection and patient monitoring tools.