Sarepta Therapeutics Inc. (NASDAQ:SRPT) declined to comment on Friday after FDA vigorously defended itself in front of the Duchenne muscular dystrophy (DMD) community over the agency's advice and treatment of the company's eteplirsen (AVI-4658). In a rare statement on a compound still in development, FDA said it has been consistent in the advice it has provided to Sarepta on the development and regulatory pathway for the phosphorodiamidate morpholino oligomer (PMO) targeting exon 51.
On Monday, Sarepta said it would delay an NDA submission to mid-2015 because the agency last week requested additional data following a pre-NDA meeting in September. In July, the company had said it planned to submit the NDA by year end (see BioCentury Extra, Oct. 27).
In its Thursday statement, FDA said its "most recent advice was consistent with the advice provided" following an April meeting with Sarepta.
The agency said its recent recommendations include how the company can improve dystrophin analyses because the agency is "concerned that the methods used to measure dystrophin were not adequately robust to support an NDA submission."
According to FDA, it "has consistently stated that it would be necessary to include data" in an NDA showing eteplirsen increases production of the muscle protein.
FDA said it has also been consistent in its guidance that Sarepta would need to submit data from natural history studies and that data from patients in addition to those enrolled in the company's open-label extension Study 4658-US-202 "would be critical to our assessment of the safety and efficacy of eteplirsen."
On a Monday conference call, President and CEO Christopher Garabedian told investors Sarepta plans to start enrolling 12-24 new patients next month. He said the company has not been able to obtain direct access to natural history data sets from academic sources.
Following Sarepta's update on Monday, Parent Project Muscular Dystrophy sent a letter to FDA stating it was "terribly disappointed" by the delay.
In an email following FDA's statement, Pat Furlong, president and CEO of the not-for-profit advocacy group, told BioCentury that while PPMD is "not privy to all details between sponsors and the agency this community stands at the ready to assist both Sarepta and the agency to expedite treatments."
Sarepta fell $7.65 (32%) to $15.91 on Monday. On Friday, the company added $0.44 to $16.17, but still closed the week off $7.39 (31%).
Two other companies developing DMD therapies also gained on Friday. Prosensa Holding N.V. (NASDAQ:RNA) was up $1.06 (10%) to $11.79 and PTC Therapeutics Inc. (NASDAQ:PTCT) was up $0.14 to $40.87.
Prosensa has begun a rolling NDA submission to FDA for drisapersen, an antisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene. The company on Friday told BioCentury it has "had frequent interactions with the Agency to discuss our submission and are satisfied with the consistency of their advice."
PTC said it plans to provide guidance next week on Translarna ataluren, but declined to comment on its interactions with FDA. The small molecule that facilitates complete translation of proteins containing nonsense mutations has conditional approval in the EU for DMD.
With 3Q14 earnings that beat guidance, AbbVie Inc. (NYSE:ABBV) CEO Richard Gonzalez said the pharma still has an appetite for M&A, but declared it's unlikely to find more $50 billion opportunities like the attempted inversion deal with Shire plc (LSE:SHP; NASDAQ:SHPG).
Gonzalez said AbbVie's performance shows "that we're a company that's in an even stronger position today than we were before we announced this deal."
Management focused the conference call on the upcoming launch of the company's interferon-free oral HCV combination, and alluded to its potential in hard-to-treat populations, but otherwise deflected questions about plans for issuing guidance or pricing. AbbVie did point investors to its plans to read out HCV pipeline data at the American Association for the Study of Liver Diseases (AASLD) meeting Nov. 7-11.
The company also singled out its management of the Humira adalimumab franchise. The anti-TNF antibody added $3.2 billion in sales globally in 3Q14, up 18% on an operating basis.
Adjusted EPS of $0.89 was up 9% over 3Q13 and eclipsed the top of the pharma's previous guidance of $0.79. The company boosted its full year adjusted EPS guidance to $3.25-$3.27 from $3.06-$3.16. On a GAAP basis, diluted EPS is forecast at $1.15-$1.17 including the costs of the Shire transaction, which collapsed on Oct. 20.
Citing AbbVie's cash flow, Gonzalez said, "we clearly have the wherewithal to be active in the M&A front." But, he said, "it's unlikely that we would do another $50 billion deal."
Gonzalez said the strategy is "to build leadership positions in specialty focused areas," citing opportunities to build out in existing franchises like immunology, especially where products can add different mechanisms of action.
He also cited oncology, and in the rare disease space said, "hepatology is the area that would be of strong interest."
Aegerion (NASDAQ:AEGR) plunged $14.02 (41%) to $20.19 on Friday as investors reponded to the company's FY14 guidance post-market on Thursday that fell short of expectations.
Citing slower than expected growth and higher than expected patient dropouts, the company slashed expectations for Juxtapid lomitapide to treat homozygous familial hypercholesterolemia (hoFH). Aegerion now expects FY14 Juxtapid sales of $150-$160 million, down from prior guidance at the lower end of $180-$200 million.
In 3Q14, the drug posted sales of $43.7 million, which missed consensus estimates of $49 million.
Chimerix Inc. (NASDAQ:CMRX) gained $1.15 to $31.04 after completing its second big follow-on in 2014. The infectious disease company raised $105.9 million through the sale of 3.65 million shares at $29 in a deal underwritten by Morgan Stanley; JP Morgan; Cowen; Piper Jaffray; Stifel, Nicolaus; Cantor Fitzgerald; and Brean Capital.
The company proposed to raise about $105 million after market close on Wednesday when its stock price was $29.79. In May, Chimerix raised $119.4 million through the sale of 8.4 million shares at $14.22.
The biotech's brincidofovir is in Phase III trials to prevent cytomegalovirus (CMV) infection in adults undergoing hematopoietic stem cell transplant (HSCT). The compound also is in Phase III testing to treat adenovirus infection and in Phase II to treat Ebola virus infection.
Myeloid malignancy company Imago BioSciences Inc. (San Francisco, Calif.) raised $26.5 million in a series A round led by Clarus Ventures. Frazier Healthcare; Amgen Ventures; and Merck Research Labs Venture Fund also participated. CEO Hugh Rienhoff did not disclose if the round was tranched, but told BioCentury it was "fully committed."
Imago is focused on therapeutics that alter gene expression. Its lead program is in preclinical development for acute myelogenous leukemia (AML), myelodysplastic syndrome (MDS) and other diseases.
Clarus' Dennis Henner and Frazier's Patrick Heron joined the Imagos' board.
Virobay Inc. (Menlo Park, Calif.) withdrew its proposed IPO on NASDAQ, citing market conditions. Earlier this month, the company amended its offering to sell 3.8 million shares at $12-$14. At $13, it would have raised $50 million and been valued at $169 million. Piper Jaffray; JMP Securities; Cantor Fitzgerald; and Summer Street Research Partners were underwriters.
The company expects to begin Phase II trials of its oral cathespin S inhibitor VBY-036 to treat pain and Crohn's disease in 1H15 and 2H15, respectively, and a Phase II trial of VBY-891 to treat psoriasis in 1H15.
PolyPid Ltd. (Petach Tikva, Israel) amended its proposed IPO on NASDAQ and now plans to sell 1.8 million shares at $10-$12. At $11, PolyPid would raise $20 million and be valued at $103 million. Aegis Capital; MLV; and Chardan are underwriters.
PolyPid's lead candidate is BonyPid, a biodegradable bone filler combined with an antibiotic release platform designed to treat long-bone fractures and prevent bone infection. The company plans to begin a confirmatory trial of BonyPid-1000 and a pilot program for dental counterpart BonyPid-500 in 2H15. PolyPid hopes to launch the products in 1H17.
Clarus Ventures named Barry Gertz a venture partner. He also will become chief medical advisor for Clarus portfolio company Relay Pharmaceuticals Inc., which finances late-stage clinical studies of pharma assets. Gertz previously was SVP of global clinical development at Merck & Co. Inc. (NYSE:MRK).
Veloxis Pharmaceuticals A/S (CSE:VELO) plunged DKK0.63 (31%) to DKK1.40 on Friday after the company said FDA's tentative approval of immunosuppressant Envarsus XR tacrolimus will not become finalized until exclusivity expires for Astagraf XL tacrolimus from Astellas Pharma Inc. (Tokyo:4503) on July 19, 2016.
Veloxis President and CEO William Polvino told BioCentury the NDA for once-daily Envarsus XR was submitted through the 505(b)(2) pathway and relied on data for Astellas' twice-daily Prograf tacrolimus, not the pharma's once-daily extended release Astagraf.
Prograf does not have exclusivity.
Polvino said Veloxis is appealing and that it is too soon to announce a timeline.
"We have great respect for the FDA and are confident that they will be able to correct this decision, which we believe is grossly mistaken, quickly," he told BioCentury.
Envarsus XR is approved in Europe, where partner Chiesi Farmaceutici S.p.A. (Parma, Italy) has commercialization rights. The product delivers tacrolimus using Veloxis' MeltDose technology.
Joseph Griffin joined regulatory consulting firm Greenleaf Health LLC as EVP of drug and biological drug products. He was associate director for policy development in the Office of Medical Policy at FDA's Center for Drug Evaluation and Research (CDER).
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