Novus Ther­a­peu­tics plunges deep in­to pen­ny stock ter­ri­to­ry af­ter failed ear tri­al

Af­ter a more than 15-year run, a Cal­i­for­nia-based biotech is ex­plor­ing op­tions, in­clud­ing a sale, af­ter its lead ex­per­i­men­tal ther­a­py failed an ex­plorato­ry mid-stage study in pa­tients with mid­dle ear in­fec­tions char­ac­ter­ized by a build-up of flu­id be­hind the eardrum.

The com­pa­ny, ini­tial­ly called Tokai Phar­ma­ceu­ti­cals but which sub­se­quent­ly changed its name to Novus Ther­a­peu­tics in 2017, saw its shares more than halve on Mon­day af­ter the drug — OP0201— did not pass muster as an ad­junct ther­a­py to oral an­tibi­otics in in­fants and chil­dren aged 6 to 24 months with acute oti­tis me­dia (OM).

Oti­tis me­dia is of­ten caused by a block­age in the pas­sage­way that con­nects the mid­dle ear to the back of the nasal cav­i­ty called the Eu­stachi­an tube. Com­prised of a sur­fac­tant (di­palmi­toylphos­phatidyl­choline) and a spread­ing agent (cho­les­teryl palmi­tate) sus­pend­ed in a pro­pel­lant, OP0201 is ad­min­is­tered in­tranasal­ly via a pres­sur­ized me­tered-dose in­haler to “de-stick” the tube re­store ven­ti­la­tion in the mid­dle ear.

In the tri­al, pa­tients were treat­ed twice dai­ly for 10 days and fol­lowed up to one month. There were two main goals: the res­o­lu­tion of bulging tym­pan­ic mem­brane and res­o­lu­tion of mid­dle ear ef­fu­sion.

Source: Novus Ther­a­peu­tics, 2020

Click on the im­age to see the full-sized ver­sion

So far, there have been no ther­a­pies ap­proved to specif­i­cal­ly treat OM. An­tibi­otics are used to ad­dress bac­te­r­i­al in­fec­tions that may arise as a re­sult of the con­di­tion, but they have no im­pact on vi­ral in­fec­tions. In or­der to deal with re­cur­rent or chron­ic oti­tis me­dia,  surgery is rec­om­mend­ed in which the tym­pan­ic mem­brane is per­fo­rat­ed to im­prove drainage and ven­ti­la­tion of the mid­dle ear.

Af­ter the failed study, Novus’ stock closed down about 52% at 62 cents.

Gre­go­ry Flesh­er

“We rec­og­nize that con­tin­ued de­vel­op­ment of our sur­fac­tant-based nasal aerosol will re­quire sig­nif­i­cant time and cap­i­tal,” said com­pa­ny chief Gre­go­ry Flesh­er in a state­ment. “We must fund the com­pa­ny for sev­er­al years in or­der to com­plete ad­di­tion­al for­mu­la­tion and de­vice de­vel­op­ment, ini­ti­ate the next oti­tis me­dia clin­i­cal tri­al, and ul­ti­mate­ly de­liv­er clin­i­cal da­ta. Giv­en our cur­rent val­u­a­tion rel­a­tive to our cap­i­tal needs, and giv­en the op­er­a­tional chal­lenges with the on­go­ing COVID-19 pan­dem­ic, we be­lieve that ex­plo­ration of all op­tions is the ap­pro­pri­ate course of ac­tion.”

In Jan­u­ary, the com­pa­ny said it had enough in the cof­fers to keep the lights on un­til the fourth quar­ter of 2020.

There is now a crowd­ed pipeline ad­dress­ing ail­ments of the ear, giv­en that most prod­ucts such as hear­ing aids and cochlear im­plants ad­dress ear dam­age caused by ge­net­ics, noise, ag­ing, or drugs, but noth­ing quite cures or in­deed tar­gets the bi­o­log­i­cal un­der­pin­nings of hear­ing loss. Vy­ing to fill that gap, a pletho­ra of de­vel­op­ers is work­ing on ther­a­peu­tics in a bur­geon­ing field.

Boston-based Ak­ou­os is work­ing on a ther­a­py de­signed to help in­di­vid­u­als with sen­sorineur­al hear­ing loss due to mu­ta­tions in the otofer­lin (OTOF) gene. Al­so in Boston is Deci­bel Ther­a­peu­tics, which is work­ing on re­gen­er­a­tion by tar­get­ing tiny hairs that grow in the in­ner ear to ad­dress con­gen­i­tal hear­ing loss or age-re­lat­ed bal­ance dis­or­ders. Fre­quen­cy Ther­a­peu­tics has a mid-stage hair cell re­gen­er­a­tion pro­gram us­ing prog­en­i­tor cells.

Across the At­lantic, UK-based Rin­ri Ther­a­peu­tics is work­ing on treat­ing hear­ing loss by trans­plant­i­ng ot­ic neur­al prog­en­i­tor cells in­to the in­ner ear. Am­s­ter­dam-based Au­dion Ther­a­peu­tics has a com­pound in-li­censed from Eli Lil­ly, which is de­signed to turn on a chem­i­cal switch to pro­duce new sen­so­ry hair cells from oth­er cells in the in­ner ear to im­prove hear­ing.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Isaac Veinbergs, Libra CEO

With $29M in Se­ries A, Boehringer-backed Li­bra looks to tack­le neu­rode­gen­er­a­tion through cel­lu­lar clean­ing

Can the natural process by which cells clean out toxic proteins be harnessed to create potential treatments for neurodegenerative disorders?

That’s the question Libra Therapeutics will be trying to answer, as the new biotech officially launched Wednesday morning with $29 million in Series A financing. The company has three preclinical programs at the ready, with its lead candidate targeting ALS and frontotemporal dementia. But CEO Isaac Veinbergs said he hopes to develop therapies for a wide range of diseases, including Parkinson’s, Alzheimer’s and Huntington’s.

Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

Gene Wang, Immetas co-founder and CEO (file photo)

Im­metas Ther­a­peu­tics nabs $11M Se­ries A to nar­row their bis­pe­cif­ic work tar­get­ing in­flam­ma­tion in age-re­lat­ed dis­eases

How does a biotech celebrate its two-year anniversary? For Immetas Therapeutics, it’s with an $11 million Series A round and a game plan to fight age-related disease.

Co-founders Gene Wang and David Sinclair came together years ago around the idea that inflammation is the ultimate process driving age-related illnesses, including cancer. The duo launched Immetas in 2018 and packed the staff with industry experts. Wang, who says he’s always had an entrepreneurial spirit, has held lead roles at Novartis, GSK, Bristol Myers Squibb and Merck. He’s worked on blockbuster drugs like Humira, Gardasil, Varubi and Zolinza. And now, he’s channeling that spirit as CEO.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.